MDedge Neurology

This discussion was recorded on August 2, 2024. This transcript has been edited for clarity. 

Robert D. Glatter, MD: Today, we’ll be discussing the results of a new study published in The Journal of Emergency Medicine, looking at the incidence of delayed intracranial hemorrhage among older patients taking preinjury anticoagulants who present to the emergency department (ED) with blunt head trauma.

Joining me today is the lead author of the study, Dr. Richard Shih, professor of emergency medicine at Florida Atlantic University. Also joining me is Dr. Christina Shenvi, associate professor of emergency medicine at the University of North Carolina (UNC) Chapel Hill, with fellowship training in geriatric emergency medicine. 

Welcome to both of you.

Richard D. Shih, MD: Thanks, Rob. 

Christina L. Shenvi, MD, PhD, MBA: Thanks. Pleasure to be here. 
 

ICH Study Methodology

Dr. Glatter: It’s a pleasure to have you. Rich, this is a great study and targeted toward a population we see daily in the emergency department. I want you to describe your methodology, patient selection, and how you went about organizing your study to look at this important finding of delayed intracranial hemorrhage, especially in those on anticoagulants.

Dr. Shih: This all started for our research team when we first read the 2012 Annals of Emergency Medicine paper. The first author was Vincenzo Menditto, and he looked at a group of patients that had minor head injury, were anticoagulated, and had negative initial head CTs. 

There were about 100 patients, of which about 10 of them did not consent, but they hospitalized all these patients. These were anticoagulated, negative-first head CTs. They hospitalized the patients and then did a routine second CT at about 24 hours. They also followed them for a week, and it turned out a little over 7% of them had delayed head CT. 

We were wondering how many delayed intracranial hemorrhages we had missed because current practice for us was that, if patients had a good physical exam, their head CT was normal, and everything looked good, we would send them home.

Because of that, a number of people across the country wanted to verify those findings from the Menditto study. We tried to design a good study to answer that question. We happen to have a very large geriatric population in Florida, and our ED census is very high for age over 65, at nearly 60%. 

There are two Level I trauma centers in Palm Beach County. We included a second multicenter hospital, and we prospectively enrolled patients. We know the current state of practice is not to routinely do second CTs, so we followed these patients over time and followed their medical records to try to identify delayed bleeding. That’s how we set up our methodology.
 

Is It Safe to Discharge Patients With Trauma After 24 Hours?

Dr. Glatter: For the bulk of these patients with negative head CTs, it’s been my practice that when they’re stable and they look fine and there’s no other apparent, distracting painful trauma, injuries and so forth, they’re safe to discharge. 

The secondary outcome in your study is interesting: the need for neurosurgical intervention in terms of those with delayed intracranial hemorrhage.

Dr. Shih: I do believe that it’s certainly not the problem that Menditto described, which is 7%. There are two other prospective studies that have looked at this issue with delayed bleeding on anticoagulants. Both of these also showed a relatively low rate of delayed bleeding, which is between like 0.2% and 1.0%. In our study, it was 0.4%. 

The difference in the studies is that Menditto and colleagues routinely did 24-hour head CTs. They admitted everybody. For these other studies, routine head CT was not part of it. My bet is that there is a rate of delayed bleeding somewhere in between that seen in the Menditto study and that in all the other studies.

However, talking about significant intracranial hemorrhage, ones that perhaps need neurosurgery, I believe most of them are not significant. There’s some number that do occur, but the vast majority of those probably don’t need neurosurgery. We had 14 delayed bleeds out of 6000 patients with head trauma. One of them ended up requiring neurosurgery, so the answer is not zero, but I don’t think it’s 7% either. 

Dr. Glatter: Dr. Shenvi, I want to bring you into the conversation to talk about your experience at UNC, and how you run things in terms of older patients with blunt head trauma on preinjury anticoagulants.

Dr. Shenvi: Thanks, Rob. I remember when this paper came out showing this 7% rate of delayed bleeding and the question was, “Should we be admitting all these people?” Partly just from an overwhelming need for capacity that that would bring, it just wasn’t practical to say, “We’re going to admit every patient with a negative head CT to the hospital and rescan them.” That would be hundreds or thousands of patients each year in any given facility. 

The other thing is that delayed bleeds don’t always happen just in the first 24 hours. It’s not even a matter of bringing patients into observation for 24 hours, watching them, and rescanning them if they have symptoms. It can occur several days out. That never, in almost any institution that I know of, became standard practice. 

The way that it did change my care was to give good return precautions to patients, to make sure they have somebody with them to say, “Hey, sometimes you can have bleeding several days out after a fall, even though your CT scan here today looks perfect,” and to alert them that if they start having severe headaches, vomiting, or other symptoms of intracranial hemorrhage, that they should come back. 

I don’t think it ever became standard practice, and for good reason, because that was one study. The subsequent studies that Richard mentioned, pretty quickly on the heels of that initial one, showed a much lower rate of delayed ICH with the caveats that the methodology was different. 
 

Shift in Anticoagulants

Dr. Shenvi: One other big change from that original study, and now to Richard’s study, is the shift in anticoagulants. Back in the initial study you mentioned, it was all warfarin. We know from other studies looking at warfarin vs the direct oral anticoagulants (DOACs) that DOACs have lower rates of ICH after a head injury, lower rates of need for neurosurgical intervention, and lower rates of discharge to a skilled nursing facility after an intracranial hemorrhage.

Across the board, we know that the DOACs tend to do better. It’s difficult to compare newer studies because it’s a different medication. It did inform my practice to have an awareness of delayed intracranial hemorrhage so that I warn patients more proactively. 

Dr. Glatter: I haven’t seen a patient on warfarin in years. I don’t know if either of you have, but it’s all DOACs now unless there’s some other reason. That shift is quite apparent.

Dr. Shih: The problem with looking at delayed bleeding for DOACs vs warfarin is the numbers were so low. I think we had 13 people, and seven were in the no-anticoagulant group. The numbers are even lower, so it’s hard to say. 

I just wanted to comment on something that Dr. Shenvi said, and I pretty much agree with everything that she said. Anticoagulants and warfarin, and that Menditto study, have a carryover effect. People group DOACs with warfarin similarly. When a patient is brought in, the first thing they talk about with head trauma is, “Oh, they’re on an anticoagulant” or “They’re not on an anticoagulant.” It’s so ingrained.

I believe that, in emergency medicine, we’re pressed for space and time and we’re not as affected by that 24-hour observation. Maybe many of our surgeons will automatically admit those patients. 

I haven’t seen a guideline from the United States, but there are two international guidelines. One is from Austria from 2019, and one is from Scandinavia. Both recommended 24-hour observation if you’re on an anticoagulant.

There is a bit of controversy left over with that. Hopefully, as more and more of information, like in our study, comes out, people will be a little bit more clear about it. I don’t think there’s a need to routinely admit them. 

I do want to mention that the Menditto study had such a massive impact on everybody. They pointed out one subgroup (and it’s such a small number of patients). They had seven cases of delayed bleeding; four or five of them were within that 24 hours, and a couple were diagnosed later over the next couple days.

Of those seven people, four of them had international normalized ratios (INRs) greater than 3. Of those four patients, I’ve heard people talk about this and recommend, “Okay, that’s the subgroup I would admit.” There’s a toss-up with what to do with DOAC because it’s very hard to tell whether there’s an issue, whether there are problems with their dosing, and whatever. 

We actually recently looked at that. We have a much larger sample than four: close to 300 patients who were on warfarin. We looked at patients who had INRs below 3 and above 3, and we didn’t show a difference. We still don’t believe that warfarin is a big issue with delayed bleeding.
 

Should We Be Asking: ‘Are They on Blood Thinners?’

Dr. Shenvi: One of the interesting trends related to warfarin and the DOACs vs no anticoagulant is that as you mentioned, Dr Shih, the first question out of people’s mouths or the first piece of information emergency medical services gives you when they come in with a patient who’s had a head injury is, “Are they on blood thinners or not?”

Yet, the paradigm is shifting to say it’s not actually the blood thinners themselves that are giving older patients the higher risk for bleeding; it’s age and other comorbidities.

Certainly, if you’re on an anticoagulant and you start to bleed, your prognosis is much worse because the bleeding doesn’t stop. In terms of who has a bleeding event, there’s much less impact of anticoagulation than we used to think. That, in part, may be due to the change from warfarin to other medications.

Some of the experts I’ve talked to who have done the research on this have said, “Well, actually, warfarin was more of a marker for being much older and more frail, because it was primarily prescribed to older patients who have significant heart disease, atrial fibrillation, and so on.” It was more a marker for somebody who is at risk for an intracranial hemorrhage. There are many changes that have happened in the past 10 years with medications and also our understanding. 
 

Challenges in Patient Follow-up

Dr. Glatter: That’s a great point. One thing, Rich, I want to ask you about is in terms of your proxy outcome assessment. When you use that at 14 and 60 days with telephone follow-up and then chart review at 60 and 90 days (because, obviously, everyone can’t get another head CT or it’s difficult to follow patients up), did you find that worked out well in your prospective cohort study, in terms of using that as a proxy, so to speak? 

Dr. Shih: I would say to a certain extent. Unfortunately, we don’t have access to the patients to come back to follow up all of them, and there was obviously a large number of patients in our study. 

The next best thing was that we had dedicated research assistants calling all of the patients at 14 days and 60 days. I’ve certainly read research studies where, when they call them, they get 80%-90% follow-up, but we did not achieve that.

I don’t know if people are more inundated with spam phone calls now, or the older people are just afraid of picking up their phone sometimes with all the scams and so forth. I totally understand, but in all honesty, we only had about a 30%-35% follow-up using that follow-up pathway. 

Then the proxy pathway was to look at their charts at 60 and 90 days. Also, we looked at the Florida death registry, which is pretty good, and then finally, we had both Level I trauma centers in the county that we were in participating. It’s standard practice that if you have an intracranial hemorrhage at a non–Level I trauma center, you would be transferred to a Level I trauma center. That’s the protocol. I know that’s not followed 100% of the time, but that’s part of the proxy follow-up. You could criticize the study for not having closer to 90% actual contact, but that’s the best we could do. 

Dr. Glatter: I think that’s admirable. Using that paradigm of what you described certainly allows the reader to understand the difficulty in assessing patients that don’t get follow-up head CT, and hardly anyone does that, as we know.

To your point of having both Level I trauma centers in the county, that makes it pretty secure. If we’re going to do a study encompassing a similar type of regional aspect, it would be similar.

Dr. Shenvi: I think your proxies, to your credit, were as good as you can get. You can never get a 100% follow-up, but you really looked at all the different avenues by which patients might present, either in the death registry or a Level I center. Well done on that aspect.

 

Determining When to Admit Patients for Observation

Dr. Glatter: In terms of admissions: You admit a patient, then you hear back that this patient should not have been admitted because they had a negative head CT, but you put them in anyway in the sense of delayed bleeding happening or not happening.

It’s interesting. Maybe the insurers will start looking at this in some capacity, based on your study, that because it’s so infrequent that you see delayed bleeding, that admitting someone for any reason whatsoever would be declined. Do you see that being an issue? In other words, [do you see] this leading to a pattern in terms of the payers?

Dr. Shih: Certainly, you could interpret it that way, and that would be unfortunate. The [incidence of] delayed bleeding is definitely not zero. That’s the first thing. 

The second thing is that when you’re dealing with an older population, having some sense that they’re not doing well is an important contributor to trying to fully assess what’s going on — whether or not they have a bleed or whether they’re at risk for falling again and then hitting their head and causing a second bleed, and making sure they can do the activities of daily life. There really should be some room for a physician to say, “They just got here, and we don’t know him that well. There’s something that bothers me about this person” and have the ability to watch them for at least another 24 hours. That’s how I feel. 

Dr. Shenvi: In my location, it would be difficult to try to admit somebody purely for observation for delayed bleeding. I think we would get a lot of pushback on that. The reasons I might admit a patient after a fall with a negative head CT, though, are all the things that, Rob, you alluded to earlier — which are, what made them fall in the first place and were they unable to get up? 

I had this happen just this week. A patient who fell couldn’t get off the ground for 12 hours, and so now she’s dehydrated and delirious with slight rhabdomyolysis. Then you’re admitting them either for the sequelae of the fall that are not related to the intracranial hemorrhage, or the fact that they are so debilitated and deconditioned that they cannot take care of themselves. They need physical therapy. Often, we will have physical and occupational therapists come see them in the ED during business hours and help make an assessment of whether they are safe to go home or whether they fall again. That can give more evidence for the need for admission.

Dr. Glatter: To bring artificial intelligence into this discussion, algorithms that are out there that say, “Push a button and the patient’s safe for discharge.” Well, this argues for a clinical gestalt and a human being to make an assessment because you can use these predictive models, which are coming and they’re going to be here soon, and they already are in some sense. Again, we have to use clinical human judgment. 

Dr. Shih: I agree. 
 

Advice for Primary Care Physicians

Dr. Glatter: What return precautions do you discuss with patients who’ve had blunt head trauma that maybe had a head CT, or even didn’t? What are the main things we’re looking for?

Dr. Shenvi: What I usually tell people is if you start to have a worse headache, nausea or vomiting, any weakness in one area of your body, or vision changes, and if there’s a family member or friend there, I’ll say, “If you notice that they’re acting differently or seem confused, come back.”

Dr. Shih: I agree with what she said, and I’m also going to add one thing. The most important part is they are trying to prevent a subsequent fall. We know that when they’ve fallen and they present to the ED, they’re at even higher risk for falling and reinjuring themselves, and that’s a population that’s already at risk.

One of the secondary studies that we published out of this project was looking at follow-up with their primary care physicians, and there were two things that we wanted to address. The first was, how often did they do it? Then, when they did do it, did their primary care physicians try to address and prevent subsequent falls?

Both the answers are actually bad. Amazingly, just over like 60% followed up. 

In some of our subsequent research, because we’re in the midst of a randomized, controlled trial where we do a home visit, when we initially see these individuals that have fallen, they’ll schedule a home visit for us. Then a week or two later, when we schedule the home visit, many of them cancel because they think, Oh, that was a one-off and it’s not going to happen again. Part of the problem is the patients, because many of them believe that they just slipped and fell and it’s not going to happen again, or they’re not prone to it.

The second issue was when patients did go to a primary care physician, we have found that some primary care physicians believe that falling and injuring themselves is just part of the normal aging process. A percentage of them don’t go over assessment for fall risk or even initiate fall prevention treatments or programs. 

I try to take that time to tell them that this is very common in their age group, and believe it or not, a fall from standing is the way people really injure themselves, and there may be ways to prevent subsequent falls and injuries. 

Dr. Glatter: Absolutely. Do you find that their medications are a contributor in some sense? Say they’re antihypertensive, have issues of orthostasis, or a new medication was added in the last week. 

Dr. Shenvi: It’s all of the above. Sometimes it’s one thing, like they just started tamsulosin for their kidney stone, they stood up, they felt lightheaded, and they fell. Usually, it’s multifactorial with some changes in their gait, vision, balance, reflex time, and strength, plus the medications or the need for assistive devices. Maybe they can’t take care of their home as well as they used to and there are things on the floor. It’s really all of the above.
 

‘Harder to Unlearn Something Than to Learn It’

Dr. Glatter: Would either of you like to add any additional points to the discussion or add a few pearls? 

Dr. Shenvi: This just highlights the challenge of how it’s harder to unlearn something than to learn it, where one study that maybe wasn’t quite looking at what we needed to, or practice and prescribing patterns have changed, so it’s no longer really relevant. 

The things that we learned from that, or the fears that we instilled in our minds of, Uh oh, they could go home and have delayed bleeding, are much harder to unlearn, and it takes more studies to unlearn that idea than it did to actually put it into place. 

I’m glad that your team has done this much larger, prospective study and hopefully will reduce the concern about this entity. 

Dr. Shih: I appreciate that segue. It is amazing that, for paramedics and medical students, the first thing out of their mouth is, “Are they on an anticoagulant?”

In terms of the risk of developing an intracranial hemorrhage, I think it’s much less than the weight we’ve put on it before. However, I believe if they have a bleed, the bleeds are worse. It’s kind of a double-edged sword. It’s still an important factor, but it doesn’t come with the Oh my gosh, they’re on an anticoagulant that everybody thinks about.
 

No. 1 Cause of Traumatic Injury Is a Fall from Standing

Dr. Glatter: These are obviously ground-level falls in most patients and not motor vehicle crashes. That’s an important part in the population that you looked at that should be mentioned clearly. 

Dr. Shih: It’s astonishing. I’ve been a program director for over 20 years, and geriatrics is not well taught in the curriculum. It’s astonishing for many of our trainees and emergency physicians in general that the number-one cause for traumatic injury is a fall from standing.

Certainly, we get patients coming in the trauma center like a 95-year-old person who’s on a ladder putting up his Christmas lights. I’m like, oh my God. 

For the vast majority, it’s closer to 90%, but in our study, for the patients we looked at, it was 80% that fall from standing. That’s the mechanism that causes these bleeds and these major injuries. 

Dr. Shenvi: That’s reflective of what we see, so it’s good that that’s what you looked at also. 

Dr. Glatter: Absolutely. Well, thank you both. This has been a very informative discussion. I appreciate your time, and our readers will certainly benefit from your knowledge and expertise. Thank you again.

Dr. Glatter, assistant professor of emergency medicine at Zucker School of Medicine at Hofstra/Northwell in Hempstead, New York, is a medical adviser for this news organization. He disclosed having no relevant financial conflicts. Dr. Shih is professor of emergency medicine at the Charles E. Schmidt College of Medicine at Florida Atlantic University, Boca Raton. His current grant funding and area of research interest involves geriatric emergency department patients with head injury and fall-related injury. He disclosed receiving a research grant from The Florida Medical Malpractice Joint Underwriting Association Grant for Safety of Health Care Services). Dr. Shenvi, associate professor of emergency medicine at the University of North Carolina at Chapel Hill, disclosed ties with the American College of Emergency Physicians, Institute for Healthcare Improvement, AstraZeneca, and CurvaFix.

A version of this article appeared on Medscape.com.

This discussion was recorded on August 2, 2024. This transcript has been edited for clarity. 

Robert D. Glatter, MD: Today, we’ll be discussing the results of a new study published in The Journal of Emergency Medicine, looking at the incidence of delayed intracranial hemorrhage among older patients taking preinjury anticoagulants who present to the emergency department (ED) with blunt head trauma.

Joining me today is the lead author of the study, Dr. Richard Shih, professor of emergency medicine at Florida Atlantic University. Also joining me is Dr. Christina Shenvi, associate professor of emergency medicine at the University of North Carolina (UNC) Chapel Hill, with fellowship training in geriatric emergency medicine. 

Welcome to both of you.

Richard D. Shih, MD: Thanks, Rob. 

Christina L. Shenvi, MD, PhD, MBA: Thanks. Pleasure to be here. 
 

ICH Study Methodology

Dr. Glatter: It’s a pleasure to have you. Rich, this is a great study and targeted toward a population we see daily in the emergency department. I want you to describe your methodology, patient selection, and how you went about organizing your study to look at this important finding of delayed intracranial hemorrhage, especially in those on anticoagulants.

Dr. Shih: This all started for our research team when we first read the 2012 Annals of Emergency Medicine paper. The first author was Vincenzo Menditto, and he looked at a group of patients that had minor head injury, were anticoagulated, and had negative initial head CTs. 

There were about 100 patients, of which about 10 of them did not consent, but they hospitalized all these patients. These were anticoagulated, negative-first head CTs. They hospitalized the patients and then did a routine second CT at about 24 hours. They also followed them for a week, and it turned out a little over 7% of them had delayed head CT. 

We were wondering how many delayed intracranial hemorrhages we had missed because current practice for us was that, if patients had a good physical exam, their head CT was normal, and everything looked good, we would send them home.

Because of that, a number of people across the country wanted to verify those findings from the Menditto study. We tried to design a good study to answer that question. We happen to have a very large geriatric population in Florida, and our ED census is very high for age over 65, at nearly 60%. 

There are two Level I trauma centers in Palm Beach County. We included a second multicenter hospital, and we prospectively enrolled patients. We know the current state of practice is not to routinely do second CTs, so we followed these patients over time and followed their medical records to try to identify delayed bleeding. That’s how we set up our methodology.
 

Is It Safe to Discharge Patients With Trauma After 24 Hours?

Dr. Glatter: For the bulk of these patients with negative head CTs, it’s been my practice that when they’re stable and they look fine and there’s no other apparent, distracting painful trauma, injuries and so forth, they’re safe to discharge. 

The secondary outcome in your study is interesting: the need for neurosurgical intervention in terms of those with delayed intracranial hemorrhage.

Dr. Shih: I do believe that it’s certainly not the problem that Menditto described, which is 7%. There are two other prospective studies that have looked at this issue with delayed bleeding on anticoagulants. Both of these also showed a relatively low rate of delayed bleeding, which is between like 0.2% and 1.0%. In our study, it was 0.4%. 

The difference in the studies is that Menditto and colleagues routinely did 24-hour head CTs. They admitted everybody. For these other studies, routine head CT was not part of it. My bet is that there is a rate of delayed bleeding somewhere in between that seen in the Menditto study and that in all the other studies.

However, talking about significant intracranial hemorrhage, ones that perhaps need neurosurgery, I believe most of them are not significant. There’s some number that do occur, but the vast majority of those probably don’t need neurosurgery. We had 14 delayed bleeds out of 6000 patients with head trauma. One of them ended up requiring neurosurgery, so the answer is not zero, but I don’t think it’s 7% either. 

Dr. Glatter: Dr. Shenvi, I want to bring you into the conversation to talk about your experience at UNC, and how you run things in terms of older patients with blunt head trauma on preinjury anticoagulants.

Dr. Shenvi: Thanks, Rob. I remember when this paper came out showing this 7% rate of delayed bleeding and the question was, “Should we be admitting all these people?” Partly just from an overwhelming need for capacity that that would bring, it just wasn’t practical to say, “We’re going to admit every patient with a negative head CT to the hospital and rescan them.” That would be hundreds or thousands of patients each year in any given facility. 

The other thing is that delayed bleeds don’t always happen just in the first 24 hours. It’s not even a matter of bringing patients into observation for 24 hours, watching them, and rescanning them if they have symptoms. It can occur several days out. That never, in almost any institution that I know of, became standard practice. 

The way that it did change my care was to give good return precautions to patients, to make sure they have somebody with them to say, “Hey, sometimes you can have bleeding several days out after a fall, even though your CT scan here today looks perfect,” and to alert them that if they start having severe headaches, vomiting, or other symptoms of intracranial hemorrhage, that they should come back. 

I don’t think it ever became standard practice, and for good reason, because that was one study. The subsequent studies that Richard mentioned, pretty quickly on the heels of that initial one, showed a much lower rate of delayed ICH with the caveats that the methodology was different. 
 

Shift in Anticoagulants

Dr. Shenvi: One other big change from that original study, and now to Richard’s study, is the shift in anticoagulants. Back in the initial study you mentioned, it was all warfarin. We know from other studies looking at warfarin vs the direct oral anticoagulants (DOACs) that DOACs have lower rates of ICH after a head injury, lower rates of need for neurosurgical intervention, and lower rates of discharge to a skilled nursing facility after an intracranial hemorrhage.

Across the board, we know that the DOACs tend to do better. It’s difficult to compare newer studies because it’s a different medication. It did inform my practice to have an awareness of delayed intracranial hemorrhage so that I warn patients more proactively. 

Dr. Glatter: I haven’t seen a patient on warfarin in years. I don’t know if either of you have, but it’s all DOACs now unless there’s some other reason. That shift is quite apparent.

Dr. Shih: The problem with looking at delayed bleeding for DOACs vs warfarin is the numbers were so low. I think we had 13 people, and seven were in the no-anticoagulant group. The numbers are even lower, so it’s hard to say. 

I just wanted to comment on something that Dr. Shenvi said, and I pretty much agree with everything that she said. Anticoagulants and warfarin, and that Menditto study, have a carryover effect. People group DOACs with warfarin similarly. When a patient is brought in, the first thing they talk about with head trauma is, “Oh, they’re on an anticoagulant” or “They’re not on an anticoagulant.” It’s so ingrained.

I believe that, in emergency medicine, we’re pressed for space and time and we’re not as affected by that 24-hour observation. Maybe many of our surgeons will automatically admit those patients. 

I haven’t seen a guideline from the United States, but there are two international guidelines. One is from Austria from 2019, and one is from Scandinavia. Both recommended 24-hour observation if you’re on an anticoagulant.

There is a bit of controversy left over with that. Hopefully, as more and more of information, like in our study, comes out, people will be a little bit more clear about it. I don’t think there’s a need to routinely admit them. 

I do want to mention that the Menditto study had such a massive impact on everybody. They pointed out one subgroup (and it’s such a small number of patients). They had seven cases of delayed bleeding; four or five of them were within that 24 hours, and a couple were diagnosed later over the next couple days.

Of those seven people, four of them had international normalized ratios (INRs) greater than 3. Of those four patients, I’ve heard people talk about this and recommend, “Okay, that’s the subgroup I would admit.” There’s a toss-up with what to do with DOAC because it’s very hard to tell whether there’s an issue, whether there are problems with their dosing, and whatever. 

We actually recently looked at that. We have a much larger sample than four: close to 300 patients who were on warfarin. We looked at patients who had INRs below 3 and above 3, and we didn’t show a difference. We still don’t believe that warfarin is a big issue with delayed bleeding.
 

Should We Be Asking: ‘Are They on Blood Thinners?’

Dr. Shenvi: One of the interesting trends related to warfarin and the DOACs vs no anticoagulant is that as you mentioned, Dr Shih, the first question out of people’s mouths or the first piece of information emergency medical services gives you when they come in with a patient who’s had a head injury is, “Are they on blood thinners or not?”

Yet, the paradigm is shifting to say it’s not actually the blood thinners themselves that are giving older patients the higher risk for bleeding; it’s age and other comorbidities.

Certainly, if you’re on an anticoagulant and you start to bleed, your prognosis is much worse because the bleeding doesn’t stop. In terms of who has a bleeding event, there’s much less impact of anticoagulation than we used to think. That, in part, may be due to the change from warfarin to other medications.

Some of the experts I’ve talked to who have done the research on this have said, “Well, actually, warfarin was more of a marker for being much older and more frail, because it was primarily prescribed to older patients who have significant heart disease, atrial fibrillation, and so on.” It was more a marker for somebody who is at risk for an intracranial hemorrhage. There are many changes that have happened in the past 10 years with medications and also our understanding. 
 

Challenges in Patient Follow-up

Dr. Glatter: That’s a great point. One thing, Rich, I want to ask you about is in terms of your proxy outcome assessment. When you use that at 14 and 60 days with telephone follow-up and then chart review at 60 and 90 days (because, obviously, everyone can’t get another head CT or it’s difficult to follow patients up), did you find that worked out well in your prospective cohort study, in terms of using that as a proxy, so to speak? 

Dr. Shih: I would say to a certain extent. Unfortunately, we don’t have access to the patients to come back to follow up all of them, and there was obviously a large number of patients in our study. 

The next best thing was that we had dedicated research assistants calling all of the patients at 14 days and 60 days. I’ve certainly read research studies where, when they call them, they get 80%-90% follow-up, but we did not achieve that.

I don’t know if people are more inundated with spam phone calls now, or the older people are just afraid of picking up their phone sometimes with all the scams and so forth. I totally understand, but in all honesty, we only had about a 30%-35% follow-up using that follow-up pathway. 

Then the proxy pathway was to look at their charts at 60 and 90 days. Also, we looked at the Florida death registry, which is pretty good, and then finally, we had both Level I trauma centers in the county that we were in participating. It’s standard practice that if you have an intracranial hemorrhage at a non–Level I trauma center, you would be transferred to a Level I trauma center. That’s the protocol. I know that’s not followed 100% of the time, but that’s part of the proxy follow-up. You could criticize the study for not having closer to 90% actual contact, but that’s the best we could do. 

Dr. Glatter: I think that’s admirable. Using that paradigm of what you described certainly allows the reader to understand the difficulty in assessing patients that don’t get follow-up head CT, and hardly anyone does that, as we know.

To your point of having both Level I trauma centers in the county, that makes it pretty secure. If we’re going to do a study encompassing a similar type of regional aspect, it would be similar.

Dr. Shenvi: I think your proxies, to your credit, were as good as you can get. You can never get a 100% follow-up, but you really looked at all the different avenues by which patients might present, either in the death registry or a Level I center. Well done on that aspect.

 

Determining When to Admit Patients for Observation

Dr. Glatter: In terms of admissions: You admit a patient, then you hear back that this patient should not have been admitted because they had a negative head CT, but you put them in anyway in the sense of delayed bleeding happening or not happening.

It’s interesting. Maybe the insurers will start looking at this in some capacity, based on your study, that because it’s so infrequent that you see delayed bleeding, that admitting someone for any reason whatsoever would be declined. Do you see that being an issue? In other words, [do you see] this leading to a pattern in terms of the payers?

Dr. Shih: Certainly, you could interpret it that way, and that would be unfortunate. The [incidence of] delayed bleeding is definitely not zero. That’s the first thing. 

The second thing is that when you’re dealing with an older population, having some sense that they’re not doing well is an important contributor to trying to fully assess what’s going on — whether or not they have a bleed or whether they’re at risk for falling again and then hitting their head and causing a second bleed, and making sure they can do the activities of daily life. There really should be some room for a physician to say, “They just got here, and we don’t know him that well. There’s something that bothers me about this person” and have the ability to watch them for at least another 24 hours. That’s how I feel. 

Dr. Shenvi: In my location, it would be difficult to try to admit somebody purely for observation for delayed bleeding. I think we would get a lot of pushback on that. The reasons I might admit a patient after a fall with a negative head CT, though, are all the things that, Rob, you alluded to earlier — which are, what made them fall in the first place and were they unable to get up? 

I had this happen just this week. A patient who fell couldn’t get off the ground for 12 hours, and so now she’s dehydrated and delirious with slight rhabdomyolysis. Then you’re admitting them either for the sequelae of the fall that are not related to the intracranial hemorrhage, or the fact that they are so debilitated and deconditioned that they cannot take care of themselves. They need physical therapy. Often, we will have physical and occupational therapists come see them in the ED during business hours and help make an assessment of whether they are safe to go home or whether they fall again. That can give more evidence for the need for admission.

Dr. Glatter: To bring artificial intelligence into this discussion, algorithms that are out there that say, “Push a button and the patient’s safe for discharge.” Well, this argues for a clinical gestalt and a human being to make an assessment because you can use these predictive models, which are coming and they’re going to be here soon, and they already are in some sense. Again, we have to use clinical human judgment. 

Dr. Shih: I agree. 
 

Advice for Primary Care Physicians

Dr. Glatter: What return precautions do you discuss with patients who’ve had blunt head trauma that maybe had a head CT, or even didn’t? What are the main things we’re looking for?

Dr. Shenvi: What I usually tell people is if you start to have a worse headache, nausea or vomiting, any weakness in one area of your body, or vision changes, and if there’s a family member or friend there, I’ll say, “If you notice that they’re acting differently or seem confused, come back.”

Dr. Shih: I agree with what she said, and I’m also going to add one thing. The most important part is they are trying to prevent a subsequent fall. We know that when they’ve fallen and they present to the ED, they’re at even higher risk for falling and reinjuring themselves, and that’s a population that’s already at risk.

One of the secondary studies that we published out of this project was looking at follow-up with their primary care physicians, and there were two things that we wanted to address. The first was, how often did they do it? Then, when they did do it, did their primary care physicians try to address and prevent subsequent falls?

Both the answers are actually bad. Amazingly, just over like 60% followed up. 

In some of our subsequent research, because we’re in the midst of a randomized, controlled trial where we do a home visit, when we initially see these individuals that have fallen, they’ll schedule a home visit for us. Then a week or two later, when we schedule the home visit, many of them cancel because they think, Oh, that was a one-off and it’s not going to happen again. Part of the problem is the patients, because many of them believe that they just slipped and fell and it’s not going to happen again, or they’re not prone to it.

The second issue was when patients did go to a primary care physician, we have found that some primary care physicians believe that falling and injuring themselves is just part of the normal aging process. A percentage of them don’t go over assessment for fall risk or even initiate fall prevention treatments or programs. 

I try to take that time to tell them that this is very common in their age group, and believe it or not, a fall from standing is the way people really injure themselves, and there may be ways to prevent subsequent falls and injuries. 

Dr. Glatter: Absolutely. Do you find that their medications are a contributor in some sense? Say they’re antihypertensive, have issues of orthostasis, or a new medication was added in the last week. 

Dr. Shenvi: It’s all of the above. Sometimes it’s one thing, like they just started tamsulosin for their kidney stone, they stood up, they felt lightheaded, and they fell. Usually, it’s multifactorial with some changes in their gait, vision, balance, reflex time, and strength, plus the medications or the need for assistive devices. Maybe they can’t take care of their home as well as they used to and there are things on the floor. It’s really all of the above.
 

‘Harder to Unlearn Something Than to Learn It’

Dr. Glatter: Would either of you like to add any additional points to the discussion or add a few pearls? 

Dr. Shenvi: This just highlights the challenge of how it’s harder to unlearn something than to learn it, where one study that maybe wasn’t quite looking at what we needed to, or practice and prescribing patterns have changed, so it’s no longer really relevant. 

The things that we learned from that, or the fears that we instilled in our minds of, Uh oh, they could go home and have delayed bleeding, are much harder to unlearn, and it takes more studies to unlearn that idea than it did to actually put it into place. 

I’m glad that your team has done this much larger, prospective study and hopefully will reduce the concern about this entity. 

Dr. Shih: I appreciate that segue. It is amazing that, for paramedics and medical students, the first thing out of their mouth is, “Are they on an anticoagulant?”

In terms of the risk of developing an intracranial hemorrhage, I think it’s much less than the weight we’ve put on it before. However, I believe if they have a bleed, the bleeds are worse. It’s kind of a double-edged sword. It’s still an important factor, but it doesn’t come with the Oh my gosh, they’re on an anticoagulant that everybody thinks about.
 

No. 1 Cause of Traumatic Injury Is a Fall from Standing

Dr. Glatter: These are obviously ground-level falls in most patients and not motor vehicle crashes. That’s an important part in the population that you looked at that should be mentioned clearly. 

Dr. Shih: It’s astonishing. I’ve been a program director for over 20 years, and geriatrics is not well taught in the curriculum. It’s astonishing for many of our trainees and emergency physicians in general that the number-one cause for traumatic injury is a fall from standing.

Certainly, we get patients coming in the trauma center like a 95-year-old person who’s on a ladder putting up his Christmas lights. I’m like, oh my God. 

For the vast majority, it’s closer to 90%, but in our study, for the patients we looked at, it was 80% that fall from standing. That’s the mechanism that causes these bleeds and these major injuries. 

Dr. Shenvi: That’s reflective of what we see, so it’s good that that’s what you looked at also. 

Dr. Glatter: Absolutely. Well, thank you both. This has been a very informative discussion. I appreciate your time, and our readers will certainly benefit from your knowledge and expertise. Thank you again.

Dr. Glatter, assistant professor of emergency medicine at Zucker School of Medicine at Hofstra/Northwell in Hempstead, New York, is a medical adviser for this news organization. He disclosed having no relevant financial conflicts. Dr. Shih is professor of emergency medicine at the Charles E. Schmidt College of Medicine at Florida Atlantic University, Boca Raton. His current grant funding and area of research interest involves geriatric emergency department patients with head injury and fall-related injury. He disclosed receiving a research grant from The Florida Medical Malpractice Joint Underwriting Association Grant for Safety of Health Care Services). Dr. Shenvi, associate professor of emergency medicine at the University of North Carolina at Chapel Hill, disclosed ties with the American College of Emergency Physicians, Institute for Healthcare Improvement, AstraZeneca, and CurvaFix.

A version of this article appeared on Medscape.com.

This discussion was recorded on August 2, 2024. This transcript has been edited for clarity. 

Robert D. Glatter, MD: Today, we’ll be discussing the results of a new study published in The Journal of Emergency Medicine, looking at the incidence of delayed intracranial hemorrhage among older patients taking preinjury anticoagulants who present to the emergency department (ED) with blunt head trauma.

Joining me today is the lead author of the study, Dr. Richard Shih, professor of emergency medicine at Florida Atlantic University. Also joining me is Dr. Christina Shenvi, associate professor of emergency medicine at the University of North Carolina (UNC) Chapel Hill, with fellowship training in geriatric emergency medicine. 

Welcome to both of you.

Richard D. Shih, MD: Thanks, Rob. 

Christina L. Shenvi, MD, PhD, MBA: Thanks. Pleasure to be here. 
 

ICH Study Methodology

Dr. Glatter: It’s a pleasure to have you. Rich, this is a great study and targeted toward a population we see daily in the emergency department. I want you to describe your methodology, patient selection, and how you went about organizing your study to look at this important finding of delayed intracranial hemorrhage, especially in those on anticoagulants.

Dr. Shih: This all started for our research team when we first read the 2012 Annals of Emergency Medicine paper. The first author was Vincenzo Menditto, and he looked at a group of patients that had minor head injury, were anticoagulated, and had negative initial head CTs. 

There were about 100 patients, of which about 10 of them did not consent, but they hospitalized all these patients. These were anticoagulated, negative-first head CTs. They hospitalized the patients and then did a routine second CT at about 24 hours. They also followed them for a week, and it turned out a little over 7% of them had delayed head CT. 

We were wondering how many delayed intracranial hemorrhages we had missed because current practice for us was that, if patients had a good physical exam, their head CT was normal, and everything looked good, we would send them home.

Because of that, a number of people across the country wanted to verify those findings from the Menditto study. We tried to design a good study to answer that question. We happen to have a very large geriatric population in Florida, and our ED census is very high for age over 65, at nearly 60%. 

There are two Level I trauma centers in Palm Beach County. We included a second multicenter hospital, and we prospectively enrolled patients. We know the current state of practice is not to routinely do second CTs, so we followed these patients over time and followed their medical records to try to identify delayed bleeding. That’s how we set up our methodology.
 

Is It Safe to Discharge Patients With Trauma After 24 Hours?

Dr. Glatter: For the bulk of these patients with negative head CTs, it’s been my practice that when they’re stable and they look fine and there’s no other apparent, distracting painful trauma, injuries and so forth, they’re safe to discharge. 

The secondary outcome in your study is interesting: the need for neurosurgical intervention in terms of those with delayed intracranial hemorrhage.

Dr. Shih: I do believe that it’s certainly not the problem that Menditto described, which is 7%. There are two other prospective studies that have looked at this issue with delayed bleeding on anticoagulants. Both of these also showed a relatively low rate of delayed bleeding, which is between like 0.2% and 1.0%. In our study, it was 0.4%. 

The difference in the studies is that Menditto and colleagues routinely did 24-hour head CTs. They admitted everybody. For these other studies, routine head CT was not part of it. My bet is that there is a rate of delayed bleeding somewhere in between that seen in the Menditto study and that in all the other studies.

However, talking about significant intracranial hemorrhage, ones that perhaps need neurosurgery, I believe most of them are not significant. There’s some number that do occur, but the vast majority of those probably don’t need neurosurgery. We had 14 delayed bleeds out of 6000 patients with head trauma. One of them ended up requiring neurosurgery, so the answer is not zero, but I don’t think it’s 7% either. 

Dr. Glatter: Dr. Shenvi, I want to bring you into the conversation to talk about your experience at UNC, and how you run things in terms of older patients with blunt head trauma on preinjury anticoagulants.

Dr. Shenvi: Thanks, Rob. I remember when this paper came out showing this 7% rate of delayed bleeding and the question was, “Should we be admitting all these people?” Partly just from an overwhelming need for capacity that that would bring, it just wasn’t practical to say, “We’re going to admit every patient with a negative head CT to the hospital and rescan them.” That would be hundreds or thousands of patients each year in any given facility. 

The other thing is that delayed bleeds don’t always happen just in the first 24 hours. It’s not even a matter of bringing patients into observation for 24 hours, watching them, and rescanning them if they have symptoms. It can occur several days out. That never, in almost any institution that I know of, became standard practice. 

The way that it did change my care was to give good return precautions to patients, to make sure they have somebody with them to say, “Hey, sometimes you can have bleeding several days out after a fall, even though your CT scan here today looks perfect,” and to alert them that if they start having severe headaches, vomiting, or other symptoms of intracranial hemorrhage, that they should come back. 

I don’t think it ever became standard practice, and for good reason, because that was one study. The subsequent studies that Richard mentioned, pretty quickly on the heels of that initial one, showed a much lower rate of delayed ICH with the caveats that the methodology was different. 
 

Shift in Anticoagulants

Dr. Shenvi: One other big change from that original study, and now to Richard’s study, is the shift in anticoagulants. Back in the initial study you mentioned, it was all warfarin. We know from other studies looking at warfarin vs the direct oral anticoagulants (DOACs) that DOACs have lower rates of ICH after a head injury, lower rates of need for neurosurgical intervention, and lower rates of discharge to a skilled nursing facility after an intracranial hemorrhage.

Across the board, we know that the DOACs tend to do better. It’s difficult to compare newer studies because it’s a different medication. It did inform my practice to have an awareness of delayed intracranial hemorrhage so that I warn patients more proactively. 

Dr. Glatter: I haven’t seen a patient on warfarin in years. I don’t know if either of you have, but it’s all DOACs now unless there’s some other reason. That shift is quite apparent.

Dr. Shih: The problem with looking at delayed bleeding for DOACs vs warfarin is the numbers were so low. I think we had 13 people, and seven were in the no-anticoagulant group. The numbers are even lower, so it’s hard to say. 

I just wanted to comment on something that Dr. Shenvi said, and I pretty much agree with everything that she said. Anticoagulants and warfarin, and that Menditto study, have a carryover effect. People group DOACs with warfarin similarly. When a patient is brought in, the first thing they talk about with head trauma is, “Oh, they’re on an anticoagulant” or “They’re not on an anticoagulant.” It’s so ingrained.

I believe that, in emergency medicine, we’re pressed for space and time and we’re not as affected by that 24-hour observation. Maybe many of our surgeons will automatically admit those patients. 

I haven’t seen a guideline from the United States, but there are two international guidelines. One is from Austria from 2019, and one is from Scandinavia. Both recommended 24-hour observation if you’re on an anticoagulant.

There is a bit of controversy left over with that. Hopefully, as more and more of information, like in our study, comes out, people will be a little bit more clear about it. I don’t think there’s a need to routinely admit them. 

I do want to mention that the Menditto study had such a massive impact on everybody. They pointed out one subgroup (and it’s such a small number of patients). They had seven cases of delayed bleeding; four or five of them were within that 24 hours, and a couple were diagnosed later over the next couple days.

Of those seven people, four of them had international normalized ratios (INRs) greater than 3. Of those four patients, I’ve heard people talk about this and recommend, “Okay, that’s the subgroup I would admit.” There’s a toss-up with what to do with DOAC because it’s very hard to tell whether there’s an issue, whether there are problems with their dosing, and whatever. 

We actually recently looked at that. We have a much larger sample than four: close to 300 patients who were on warfarin. We looked at patients who had INRs below 3 and above 3, and we didn’t show a difference. We still don’t believe that warfarin is a big issue with delayed bleeding.
 

Should We Be Asking: ‘Are They on Blood Thinners?’

Dr. Shenvi: One of the interesting trends related to warfarin and the DOACs vs no anticoagulant is that as you mentioned, Dr Shih, the first question out of people’s mouths or the first piece of information emergency medical services gives you when they come in with a patient who’s had a head injury is, “Are they on blood thinners or not?”

Yet, the paradigm is shifting to say it’s not actually the blood thinners themselves that are giving older patients the higher risk for bleeding; it’s age and other comorbidities.

Certainly, if you’re on an anticoagulant and you start to bleed, your prognosis is much worse because the bleeding doesn’t stop. In terms of who has a bleeding event, there’s much less impact of anticoagulation than we used to think. That, in part, may be due to the change from warfarin to other medications.

Some of the experts I’ve talked to who have done the research on this have said, “Well, actually, warfarin was more of a marker for being much older and more frail, because it was primarily prescribed to older patients who have significant heart disease, atrial fibrillation, and so on.” It was more a marker for somebody who is at risk for an intracranial hemorrhage. There are many changes that have happened in the past 10 years with medications and also our understanding. 
 

Challenges in Patient Follow-up

Dr. Glatter: That’s a great point. One thing, Rich, I want to ask you about is in terms of your proxy outcome assessment. When you use that at 14 and 60 days with telephone follow-up and then chart review at 60 and 90 days (because, obviously, everyone can’t get another head CT or it’s difficult to follow patients up), did you find that worked out well in your prospective cohort study, in terms of using that as a proxy, so to speak? 

Dr. Shih: I would say to a certain extent. Unfortunately, we don’t have access to the patients to come back to follow up all of them, and there was obviously a large number of patients in our study. 

The next best thing was that we had dedicated research assistants calling all of the patients at 14 days and 60 days. I’ve certainly read research studies where, when they call them, they get 80%-90% follow-up, but we did not achieve that.

I don’t know if people are more inundated with spam phone calls now, or the older people are just afraid of picking up their phone sometimes with all the scams and so forth. I totally understand, but in all honesty, we only had about a 30%-35% follow-up using that follow-up pathway. 

Then the proxy pathway was to look at their charts at 60 and 90 days. Also, we looked at the Florida death registry, which is pretty good, and then finally, we had both Level I trauma centers in the county that we were in participating. It’s standard practice that if you have an intracranial hemorrhage at a non–Level I trauma center, you would be transferred to a Level I trauma center. That’s the protocol. I know that’s not followed 100% of the time, but that’s part of the proxy follow-up. You could criticize the study for not having closer to 90% actual contact, but that’s the best we could do. 

Dr. Glatter: I think that’s admirable. Using that paradigm of what you described certainly allows the reader to understand the difficulty in assessing patients that don’t get follow-up head CT, and hardly anyone does that, as we know.

To your point of having both Level I trauma centers in the county, that makes it pretty secure. If we’re going to do a study encompassing a similar type of regional aspect, it would be similar.

Dr. Shenvi: I think your proxies, to your credit, were as good as you can get. You can never get a 100% follow-up, but you really looked at all the different avenues by which patients might present, either in the death registry or a Level I center. Well done on that aspect.

 

Determining When to Admit Patients for Observation

Dr. Glatter: In terms of admissions: You admit a patient, then you hear back that this patient should not have been admitted because they had a negative head CT, but you put them in anyway in the sense of delayed bleeding happening or not happening.

It’s interesting. Maybe the insurers will start looking at this in some capacity, based on your study, that because it’s so infrequent that you see delayed bleeding, that admitting someone for any reason whatsoever would be declined. Do you see that being an issue? In other words, [do you see] this leading to a pattern in terms of the payers?

Dr. Shih: Certainly, you could interpret it that way, and that would be unfortunate. The [incidence of] delayed bleeding is definitely not zero. That’s the first thing. 

The second thing is that when you’re dealing with an older population, having some sense that they’re not doing well is an important contributor to trying to fully assess what’s going on — whether or not they have a bleed or whether they’re at risk for falling again and then hitting their head and causing a second bleed, and making sure they can do the activities of daily life. There really should be some room for a physician to say, “They just got here, and we don’t know him that well. There’s something that bothers me about this person” and have the ability to watch them for at least another 24 hours. That’s how I feel. 

Dr. Shenvi: In my location, it would be difficult to try to admit somebody purely for observation for delayed bleeding. I think we would get a lot of pushback on that. The reasons I might admit a patient after a fall with a negative head CT, though, are all the things that, Rob, you alluded to earlier — which are, what made them fall in the first place and were they unable to get up? 

I had this happen just this week. A patient who fell couldn’t get off the ground for 12 hours, and so now she’s dehydrated and delirious with slight rhabdomyolysis. Then you’re admitting them either for the sequelae of the fall that are not related to the intracranial hemorrhage, or the fact that they are so debilitated and deconditioned that they cannot take care of themselves. They need physical therapy. Often, we will have physical and occupational therapists come see them in the ED during business hours and help make an assessment of whether they are safe to go home or whether they fall again. That can give more evidence for the need for admission.

Dr. Glatter: To bring artificial intelligence into this discussion, algorithms that are out there that say, “Push a button and the patient’s safe for discharge.” Well, this argues for a clinical gestalt and a human being to make an assessment because you can use these predictive models, which are coming and they’re going to be here soon, and they already are in some sense. Again, we have to use clinical human judgment. 

Dr. Shih: I agree. 
 

Advice for Primary Care Physicians

Dr. Glatter: What return precautions do you discuss with patients who’ve had blunt head trauma that maybe had a head CT, or even didn’t? What are the main things we’re looking for?

Dr. Shenvi: What I usually tell people is if you start to have a worse headache, nausea or vomiting, any weakness in one area of your body, or vision changes, and if there’s a family member or friend there, I’ll say, “If you notice that they’re acting differently or seem confused, come back.”

Dr. Shih: I agree with what she said, and I’m also going to add one thing. The most important part is they are trying to prevent a subsequent fall. We know that when they’ve fallen and they present to the ED, they’re at even higher risk for falling and reinjuring themselves, and that’s a population that’s already at risk.

One of the secondary studies that we published out of this project was looking at follow-up with their primary care physicians, and there were two things that we wanted to address. The first was, how often did they do it? Then, when they did do it, did their primary care physicians try to address and prevent subsequent falls?

Both the answers are actually bad. Amazingly, just over like 60% followed up. 

In some of our subsequent research, because we’re in the midst of a randomized, controlled trial where we do a home visit, when we initially see these individuals that have fallen, they’ll schedule a home visit for us. Then a week or two later, when we schedule the home visit, many of them cancel because they think, Oh, that was a one-off and it’s not going to happen again. Part of the problem is the patients, because many of them believe that they just slipped and fell and it’s not going to happen again, or they’re not prone to it.

The second issue was when patients did go to a primary care physician, we have found that some primary care physicians believe that falling and injuring themselves is just part of the normal aging process. A percentage of them don’t go over assessment for fall risk or even initiate fall prevention treatments or programs. 

I try to take that time to tell them that this is very common in their age group, and believe it or not, a fall from standing is the way people really injure themselves, and there may be ways to prevent subsequent falls and injuries. 

Dr. Glatter: Absolutely. Do you find that their medications are a contributor in some sense? Say they’re antihypertensive, have issues of orthostasis, or a new medication was added in the last week. 

Dr. Shenvi: It’s all of the above. Sometimes it’s one thing, like they just started tamsulosin for their kidney stone, they stood up, they felt lightheaded, and they fell. Usually, it’s multifactorial with some changes in their gait, vision, balance, reflex time, and strength, plus the medications or the need for assistive devices. Maybe they can’t take care of their home as well as they used to and there are things on the floor. It’s really all of the above.
 

‘Harder to Unlearn Something Than to Learn It’

Dr. Glatter: Would either of you like to add any additional points to the discussion or add a few pearls? 

Dr. Shenvi: This just highlights the challenge of how it’s harder to unlearn something than to learn it, where one study that maybe wasn’t quite looking at what we needed to, or practice and prescribing patterns have changed, so it’s no longer really relevant. 

The things that we learned from that, or the fears that we instilled in our minds of, Uh oh, they could go home and have delayed bleeding, are much harder to unlearn, and it takes more studies to unlearn that idea than it did to actually put it into place. 

I’m glad that your team has done this much larger, prospective study and hopefully will reduce the concern about this entity. 

Dr. Shih: I appreciate that segue. It is amazing that, for paramedics and medical students, the first thing out of their mouth is, “Are they on an anticoagulant?”

In terms of the risk of developing an intracranial hemorrhage, I think it’s much less than the weight we’ve put on it before. However, I believe if they have a bleed, the bleeds are worse. It’s kind of a double-edged sword. It’s still an important factor, but it doesn’t come with the Oh my gosh, they’re on an anticoagulant that everybody thinks about.
 

No. 1 Cause of Traumatic Injury Is a Fall from Standing

Dr. Glatter: These are obviously ground-level falls in most patients and not motor vehicle crashes. That’s an important part in the population that you looked at that should be mentioned clearly. 

Dr. Shih: It’s astonishing. I’ve been a program director for over 20 years, and geriatrics is not well taught in the curriculum. It’s astonishing for many of our trainees and emergency physicians in general that the number-one cause for traumatic injury is a fall from standing.

Certainly, we get patients coming in the trauma center like a 95-year-old person who’s on a ladder putting up his Christmas lights. I’m like, oh my God. 

For the vast majority, it’s closer to 90%, but in our study, for the patients we looked at, it was 80% that fall from standing. That’s the mechanism that causes these bleeds and these major injuries. 

Dr. Shenvi: That’s reflective of what we see, so it’s good that that’s what you looked at also. 

Dr. Glatter: Absolutely. Well, thank you both. This has been a very informative discussion. I appreciate your time, and our readers will certainly benefit from your knowledge and expertise. Thank you again.

Dr. Glatter, assistant professor of emergency medicine at Zucker School of Medicine at Hofstra/Northwell in Hempstead, New York, is a medical adviser for this news organization. He disclosed having no relevant financial conflicts. Dr. Shih is professor of emergency medicine at the Charles E. Schmidt College of Medicine at Florida Atlantic University, Boca Raton. His current grant funding and area of research interest involves geriatric emergency department patients with head injury and fall-related injury. He disclosed receiving a research grant from The Florida Medical Malpractice Joint Underwriting Association Grant for Safety of Health Care Services). Dr. Shenvi, associate professor of emergency medicine at the University of North Carolina at Chapel Hill, disclosed ties with the American College of Emergency Physicians, Institute for Healthcare Improvement, AstraZeneca, and CurvaFix.

A version of this article appeared on Medscape.com.

TOPLINE:

Mobile images may be reliable for assessing cutaneous neurofibroma (cNF) features in patients with neurofibromatosis type 1 (NF1), according to a crowd-sourced registry study that also suggests correlations between cNF burden and quality of life (QoL), particularly the impact of facial severity on emotional well-being.

METHODOLOGY:

• To learn more about the association of cNFs with QoL, pain, and itch in patients with this rare disease, researchers enrolled 1016 individuals aged 40 years and older with NF1 who had at least one cNF, from May 2021 to December 2023, after reaching out to patient-led or NF1 advocacy organizations in 13 countries, including the United States.
• Participants provided demographic data, detailed photographs, and saliva samples for genetic sequencing, with 583 participants (mean age, 51.7 years; 65.9% women) submitting high-quality photographs from seven body regions at the time of the study analysis.
• A subset of 50 participants also underwent whole-body imaging.
• Four researchers independently rated the photographs for various cNF features, including general severity, number, size, facial severity, and subtypes.

TAKEAWAY:

• Based on evaluations by NF1 specialists, the agreement between mobile and whole-body images was “substantial” (74%-88% agreement) for the number of cNFs, general severity, and facial severity. Agreement between self-reported numbers of cNFs and investigator-rated numbers based on photographs was “minimal to fair.”
• Female sex, the number of cNFs, severity of cNFs on the face, and globular cNFs were associated with worse QoL (based on Skindex scores); severity of cNFs on the face had the strongest impact on overall QoL (P < .001).
• An increasing number of cNFs and worsening facial severity were strongly correlated with higher emotion subdomain scores.
• A higher number of cNFs, more severe cNFs on the face, and larger cNFs were all slightly associated with increased itch and pain (P < .01).

IN PRACTICE:

“To develop effective therapeutics, meaningful clinical outcomes that are tied with improvement in QoL for persons with NF1 must be clearly defined,” the authors wrote. The results of this study, they added, “suggested the benefit of this crowd-sourced resource by identifying the features of cNFs with the greatest association with QoL and symptoms of pain and itch in persons with NF1, highlighting new intervention strategies and features to target to most improve QoL in NF1.”

SOURCE:

The study was led by Michelle Jade Lin, BS, Stanford University School of Medicine, Redwood City, California, and was published online in JAMA Dermatology.

LIMITATIONS:

The study included only a small number of individuals from racial and ethnic minority groups and did not capture ethnicity information, which could have provided further insights into disease impact across different demographics.

DISCLOSURES:

This study was supported by Johns Hopkins University, Baltimore, and the Bloomberg Family Foundation. Ms. Lin reported support from the Stanford Medical Scholars Research Program. Three authors reported personal fees or grants outside this work. Other authors reported no competing interests.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

Mobile images may be reliable for assessing cutaneous neurofibroma (cNF) features in patients with neurofibromatosis type 1 (NF1), according to a crowd-sourced registry study that also suggests correlations between cNF burden and quality of life (QoL), particularly the impact of facial severity on emotional well-being.

METHODOLOGY:

• To learn more about the association of cNFs with QoL, pain, and itch in patients with this rare disease, researchers enrolled 1016 individuals aged 40 years and older with NF1 who had at least one cNF, from May 2021 to December 2023, after reaching out to patient-led or NF1 advocacy organizations in 13 countries, including the United States.
• Participants provided demographic data, detailed photographs, and saliva samples for genetic sequencing, with 583 participants (mean age, 51.7 years; 65.9% women) submitting high-quality photographs from seven body regions at the time of the study analysis.
• A subset of 50 participants also underwent whole-body imaging.
• Four researchers independently rated the photographs for various cNF features, including general severity, number, size, facial severity, and subtypes.

TAKEAWAY:

• Based on evaluations by NF1 specialists, the agreement between mobile and whole-body images was “substantial” (74%-88% agreement) for the number of cNFs, general severity, and facial severity. Agreement between self-reported numbers of cNFs and investigator-rated numbers based on photographs was “minimal to fair.”
• Female sex, the number of cNFs, severity of cNFs on the face, and globular cNFs were associated with worse QoL (based on Skindex scores); severity of cNFs on the face had the strongest impact on overall QoL (P < .001).
• An increasing number of cNFs and worsening facial severity were strongly correlated with higher emotion subdomain scores.
• A higher number of cNFs, more severe cNFs on the face, and larger cNFs were all slightly associated with increased itch and pain (P < .01).

IN PRACTICE:

“To develop effective therapeutics, meaningful clinical outcomes that are tied with improvement in QoL for persons with NF1 must be clearly defined,” the authors wrote. The results of this study, they added, “suggested the benefit of this crowd-sourced resource by identifying the features of cNFs with the greatest association with QoL and symptoms of pain and itch in persons with NF1, highlighting new intervention strategies and features to target to most improve QoL in NF1.”

SOURCE:

The study was led by Michelle Jade Lin, BS, Stanford University School of Medicine, Redwood City, California, and was published online in JAMA Dermatology.

LIMITATIONS:

The study included only a small number of individuals from racial and ethnic minority groups and did not capture ethnicity information, which could have provided further insights into disease impact across different demographics.

DISCLOSURES:

This study was supported by Johns Hopkins University, Baltimore, and the Bloomberg Family Foundation. Ms. Lin reported support from the Stanford Medical Scholars Research Program. Three authors reported personal fees or grants outside this work. Other authors reported no competing interests.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE:

Mobile images may be reliable for assessing cutaneous neurofibroma (cNF) features in patients with neurofibromatosis type 1 (NF1), according to a crowd-sourced registry study that also suggests correlations between cNF burden and quality of life (QoL), particularly the impact of facial severity on emotional well-being.

METHODOLOGY:

• To learn more about the association of cNFs with QoL, pain, and itch in patients with this rare disease, researchers enrolled 1016 individuals aged 40 years and older with NF1 who had at least one cNF, from May 2021 to December 2023, after reaching out to patient-led or NF1 advocacy organizations in 13 countries, including the United States.
• Participants provided demographic data, detailed photographs, and saliva samples for genetic sequencing, with 583 participants (mean age, 51.7 years; 65.9% women) submitting high-quality photographs from seven body regions at the time of the study analysis.
• A subset of 50 participants also underwent whole-body imaging.
• Four researchers independently rated the photographs for various cNF features, including general severity, number, size, facial severity, and subtypes.

TAKEAWAY:

• Based on evaluations by NF1 specialists, the agreement between mobile and whole-body images was “substantial” (74%-88% agreement) for the number of cNFs, general severity, and facial severity. Agreement between self-reported numbers of cNFs and investigator-rated numbers based on photographs was “minimal to fair.”
• Female sex, the number of cNFs, severity of cNFs on the face, and globular cNFs were associated with worse QoL (based on Skindex scores); severity of cNFs on the face had the strongest impact on overall QoL (P < .001).
• An increasing number of cNFs and worsening facial severity were strongly correlated with higher emotion subdomain scores.
• A higher number of cNFs, more severe cNFs on the face, and larger cNFs were all slightly associated with increased itch and pain (P < .01).

IN PRACTICE:

“To develop effective therapeutics, meaningful clinical outcomes that are tied with improvement in QoL for persons with NF1 must be clearly defined,” the authors wrote. The results of this study, they added, “suggested the benefit of this crowd-sourced resource by identifying the features of cNFs with the greatest association with QoL and symptoms of pain and itch in persons with NF1, highlighting new intervention strategies and features to target to most improve QoL in NF1.”

SOURCE:

The study was led by Michelle Jade Lin, BS, Stanford University School of Medicine, Redwood City, California, and was published online in JAMA Dermatology.

LIMITATIONS:

The study included only a small number of individuals from racial and ethnic minority groups and did not capture ethnicity information, which could have provided further insights into disease impact across different demographics.

DISCLOSURES:

This study was supported by Johns Hopkins University, Baltimore, and the Bloomberg Family Foundation. Ms. Lin reported support from the Stanford Medical Scholars Research Program. Three authors reported personal fees or grants outside this work. Other authors reported no competing interests.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

The ABYSS trial found that interruption of beta-blocker therapy in patients after myocardial infarction (MI) was not noninferior to continuing the drugs. 

I will argue why I think it is okay to stop beta-blockers after MI — despite this conclusion. The results of ABYSS are, in fact, similar to REDUCE-AMI, which compared beta-blocker use or nonuse immediately after MI, and found no difference in a composite endpoint of death or MI. 

Translation of the ABYSS trial results to patient care is a case where we must look past the paper’s abstract and conclusions. The key problem is the authors’ choice of primary endpoint, which obscures the correct clinical answer.
 

The ABYSS Trial

ABYSS investigators randomly assigned nearly 3700 patients who had MI and were prescribed a beta-blocker to either continue (control arm) or stop (active arm) the drug at 1 year. 

Patients had to have a left ventricular ejection fraction (LVEF) at least 40%; the median was 60%. 

The composite primary endpoint included death, MI, stroke, or hospitalization for any cardiovascular reason. ABYSS authors chose a noninferiority design. The assumption must have been that the interruption arm offered an easier option for patients — eg, fewer pills. 

Over 3 years, a primary endpoint occurred in 23.8% of the interruption group vs 21.1% in the continuation group. 

In ABYSS, the noninferiority margin was set at a 3% absolute risk increase. The 2.7% absolute risk increase had an upper bound of the 95% CI (worst case) of 5.5% leading to the not-noninferior conclusion (5.5% exceeds the noninferiority margins). 

More simply stated, the primary outcome event rate was higher in the interruption arm. 
 

Does This Mean we Should Continue Beta-Blockers in Post-MI Patients?

This led some to conclude that we should continue beta-blockers. I disagree. To properly interpret the ABYSS trial, you must consider trial procedures, components of the primary endpoint, and then compare ABYSS with REDUCE-AMI. 

It’s also reasonable to have extremely pessimistic prior beliefs about post-MI beta-blockade because the evidence establishing benefit comes from trials conducted before urgent revascularization became the standard therapy. 

ABYSS was a pragmatic open-label trial. The core problem with this design is that one of the components of the primary outcome (hospitalization for cardiovascular reasons) requires clinical judgment — and is therefore susceptible to bias, particularly in an open-label trial. 

This becomes apparent when we look at the components of the primary outcome in the two arms of the trial (interrupt vs continue): 

• For death, the rates were 4.1 and 4.0%
• For MI, the rates were 2.5 and 2.4%
• For stroke, the rates were 1.0% in both arms
• For CV hospitalization, the rates were 18.9% vs 16.6%

The higher rate CV hospitalization alone drove the results of ABYSS. Death, MI, and stroke rates were nearly identical. 

The most common reason for admission to the hospital in this category was for angiography. In fact, the rate of angiography was 2.3% higher in the interruption arm — identical to the rate increase in the CV hospitalization component of the primary endpoint. 

The results of ABYSS, therefore, were driven by higher rates of angiography in the interrupt arm. 

You need not imply malfeasance to speculate that patients who had their beta-blocker stopped might be treated differently regarding hospital admissions or angiography than those who stayed on beta-blockers. Researchers from Imperial College London called such a bias in unblinded trials “subtraction anxiety and faith healing.”

Had the ABYSS investigators chosen the simpler, less bias-prone endpoints of death, MI, or stroke, their results would have been the same as REDUCE-AMI. 
 

My Final Two Conclusions

I would conclude that interruption of beta-blockers at 1 year vs continuation in post-MI patients did not lead to an increase in death, MI, or stroke. 

ABYSS, therefore, is consistent with REDUCE-AMI. Taken together, along with the pessimistic priors, these are important findings because they allow us to stop a medicine and reduce the work of being a patient. 

My second conclusion concerns ways of knowing in medicine. I’ve long felt that randomized controlled trials (RCTs) are the best way to sort out causation. This idea led me to the believe that medicine should have more RCTs rather than follow expert opinion or therapeutic fashion. 

I’ve now modified my love of RCTs — a little. The ABYSS trial is yet another example of the need to be super careful with their design.

Something as seemingly simple as choosing what to measure can alter the way clinicians interpret and use the data. 

So, let’s have (slightly) more trials, but we should be really careful in their design. Slow and careful is the best way to practice medicine. And it’s surely the best way to do research as well.

Dr. Mandrola, clinical electrophysiologist, Baptist Medical Associates, Louisville, Kentucky, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

The ABYSS trial found that interruption of beta-blocker therapy in patients after myocardial infarction (MI) was not noninferior to continuing the drugs. 

I will argue why I think it is okay to stop beta-blockers after MI — despite this conclusion. The results of ABYSS are, in fact, similar to REDUCE-AMI, which compared beta-blocker use or nonuse immediately after MI, and found no difference in a composite endpoint of death or MI. 

Translation of the ABYSS trial results to patient care is a case where we must look past the paper’s abstract and conclusions. The key problem is the authors’ choice of primary endpoint, which obscures the correct clinical answer.
 

The ABYSS Trial

ABYSS investigators randomly assigned nearly 3700 patients who had MI and were prescribed a beta-blocker to either continue (control arm) or stop (active arm) the drug at 1 year. 

Patients had to have a left ventricular ejection fraction (LVEF) at least 40%; the median was 60%. 

The composite primary endpoint included death, MI, stroke, or hospitalization for any cardiovascular reason. ABYSS authors chose a noninferiority design. The assumption must have been that the interruption arm offered an easier option for patients — eg, fewer pills. 

Over 3 years, a primary endpoint occurred in 23.8% of the interruption group vs 21.1% in the continuation group. 

In ABYSS, the noninferiority margin was set at a 3% absolute risk increase. The 2.7% absolute risk increase had an upper bound of the 95% CI (worst case) of 5.5% leading to the not-noninferior conclusion (5.5% exceeds the noninferiority margins). 

More simply stated, the primary outcome event rate was higher in the interruption arm. 
 

Does This Mean we Should Continue Beta-Blockers in Post-MI Patients?

This led some to conclude that we should continue beta-blockers. I disagree. To properly interpret the ABYSS trial, you must consider trial procedures, components of the primary endpoint, and then compare ABYSS with REDUCE-AMI. 

It’s also reasonable to have extremely pessimistic prior beliefs about post-MI beta-blockade because the evidence establishing benefit comes from trials conducted before urgent revascularization became the standard therapy. 

ABYSS was a pragmatic open-label trial. The core problem with this design is that one of the components of the primary outcome (hospitalization for cardiovascular reasons) requires clinical judgment — and is therefore susceptible to bias, particularly in an open-label trial. 

This becomes apparent when we look at the components of the primary outcome in the two arms of the trial (interrupt vs continue): 

• For death, the rates were 4.1 and 4.0%
• For MI, the rates were 2.5 and 2.4%
• For stroke, the rates were 1.0% in both arms
• For CV hospitalization, the rates were 18.9% vs 16.6%

The higher rate CV hospitalization alone drove the results of ABYSS. Death, MI, and stroke rates were nearly identical. 

The most common reason for admission to the hospital in this category was for angiography. In fact, the rate of angiography was 2.3% higher in the interruption arm — identical to the rate increase in the CV hospitalization component of the primary endpoint. 

The results of ABYSS, therefore, were driven by higher rates of angiography in the interrupt arm. 

You need not imply malfeasance to speculate that patients who had their beta-blocker stopped might be treated differently regarding hospital admissions or angiography than those who stayed on beta-blockers. Researchers from Imperial College London called such a bias in unblinded trials “subtraction anxiety and faith healing.”

Had the ABYSS investigators chosen the simpler, less bias-prone endpoints of death, MI, or stroke, their results would have been the same as REDUCE-AMI. 
 

My Final Two Conclusions

I would conclude that interruption of beta-blockers at 1 year vs continuation in post-MI patients did not lead to an increase in death, MI, or stroke. 

ABYSS, therefore, is consistent with REDUCE-AMI. Taken together, along with the pessimistic priors, these are important findings because they allow us to stop a medicine and reduce the work of being a patient. 

My second conclusion concerns ways of knowing in medicine. I’ve long felt that randomized controlled trials (RCTs) are the best way to sort out causation. This idea led me to the believe that medicine should have more RCTs rather than follow expert opinion or therapeutic fashion. 

I’ve now modified my love of RCTs — a little. The ABYSS trial is yet another example of the need to be super careful with their design.

Something as seemingly simple as choosing what to measure can alter the way clinicians interpret and use the data. 

So, let’s have (slightly) more trials, but we should be really careful in their design. Slow and careful is the best way to practice medicine. And it’s surely the best way to do research as well.

Dr. Mandrola, clinical electrophysiologist, Baptist Medical Associates, Louisville, Kentucky, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

The ABYSS trial found that interruption of beta-blocker therapy in patients after myocardial infarction (MI) was not noninferior to continuing the drugs. 

I will argue why I think it is okay to stop beta-blockers after MI — despite this conclusion. The results of ABYSS are, in fact, similar to REDUCE-AMI, which compared beta-blocker use or nonuse immediately after MI, and found no difference in a composite endpoint of death or MI. 

Translation of the ABYSS trial results to patient care is a case where we must look past the paper’s abstract and conclusions. The key problem is the authors’ choice of primary endpoint, which obscures the correct clinical answer.
 

The ABYSS Trial

ABYSS investigators randomly assigned nearly 3700 patients who had MI and were prescribed a beta-blocker to either continue (control arm) or stop (active arm) the drug at 1 year. 

Patients had to have a left ventricular ejection fraction (LVEF) at least 40%; the median was 60%. 

The composite primary endpoint included death, MI, stroke, or hospitalization for any cardiovascular reason. ABYSS authors chose a noninferiority design. The assumption must have been that the interruption arm offered an easier option for patients — eg, fewer pills. 

Over 3 years, a primary endpoint occurred in 23.8% of the interruption group vs 21.1% in the continuation group. 

In ABYSS, the noninferiority margin was set at a 3% absolute risk increase. The 2.7% absolute risk increase had an upper bound of the 95% CI (worst case) of 5.5% leading to the not-noninferior conclusion (5.5% exceeds the noninferiority margins). 

More simply stated, the primary outcome event rate was higher in the interruption arm. 
 

Does This Mean we Should Continue Beta-Blockers in Post-MI Patients?

This led some to conclude that we should continue beta-blockers. I disagree. To properly interpret the ABYSS trial, you must consider trial procedures, components of the primary endpoint, and then compare ABYSS with REDUCE-AMI. 

It’s also reasonable to have extremely pessimistic prior beliefs about post-MI beta-blockade because the evidence establishing benefit comes from trials conducted before urgent revascularization became the standard therapy. 

ABYSS was a pragmatic open-label trial. The core problem with this design is that one of the components of the primary outcome (hospitalization for cardiovascular reasons) requires clinical judgment — and is therefore susceptible to bias, particularly in an open-label trial. 

This becomes apparent when we look at the components of the primary outcome in the two arms of the trial (interrupt vs continue): 

• For death, the rates were 4.1 and 4.0%
• For MI, the rates were 2.5 and 2.4%
• For stroke, the rates were 1.0% in both arms
• For CV hospitalization, the rates were 18.9% vs 16.6%

The higher rate CV hospitalization alone drove the results of ABYSS. Death, MI, and stroke rates were nearly identical. 

The most common reason for admission to the hospital in this category was for angiography. In fact, the rate of angiography was 2.3% higher in the interruption arm — identical to the rate increase in the CV hospitalization component of the primary endpoint. 

The results of ABYSS, therefore, were driven by higher rates of angiography in the interrupt arm. 

You need not imply malfeasance to speculate that patients who had their beta-blocker stopped might be treated differently regarding hospital admissions or angiography than those who stayed on beta-blockers. Researchers from Imperial College London called such a bias in unblinded trials “subtraction anxiety and faith healing.”

Had the ABYSS investigators chosen the simpler, less bias-prone endpoints of death, MI, or stroke, their results would have been the same as REDUCE-AMI. 
 

My Final Two Conclusions

I would conclude that interruption of beta-blockers at 1 year vs continuation in post-MI patients did not lead to an increase in death, MI, or stroke. 

ABYSS, therefore, is consistent with REDUCE-AMI. Taken together, along with the pessimistic priors, these are important findings because they allow us to stop a medicine and reduce the work of being a patient. 

My second conclusion concerns ways of knowing in medicine. I’ve long felt that randomized controlled trials (RCTs) are the best way to sort out causation. This idea led me to the believe that medicine should have more RCTs rather than follow expert opinion or therapeutic fashion. 

I’ve now modified my love of RCTs — a little. The ABYSS trial is yet another example of the need to be super careful with their design.

Something as seemingly simple as choosing what to measure can alter the way clinicians interpret and use the data. 

So, let’s have (slightly) more trials, but we should be really careful in their design. Slow and careful is the best way to practice medicine. And it’s surely the best way to do research as well.

Dr. Mandrola, clinical electrophysiologist, Baptist Medical Associates, Louisville, Kentucky, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

LONDON — It may not be advisable for patients with a history of myocardial infarction and preserved left ventricular function to discontinue long-term beta-blocker therapy, warn investigators.

In the randomized ABYSS trial, although there was no difference in death, MI, or stroke between patients who discontinued and those who continued taking beta-blockers, those who stopped taking the drugs had a higher rate of cardiovascular hospitalization.

Discontinuation was also associated with an increase in blood pressure and heart rate, without any improvement in quality of life.

“We thought we would be able to withdraw beta-blockers safely and that this would result in improved quality of life, but our trial results suggest this is not the case,” said lead investigator Johanne Silvain, MD, PhD, from Pitié-Salpêtrière University Hospital in Paris, who presented the ABYSS findings here at the European Society of Cardiology (ESC) Congress. 

The results, which were simultaneously published online in The New England Journal of Medicine, call into question current guidelines, which suggest that beta-blockers may be discontinued after 1 year in certain patient groups.

Beta-blockers have long been considered the standard of care for patients after MI, but trials showing the benefit of these drugs were conducted before the modern era of myocardial reperfusion and pharmacotherapy, which have led to sharp decreases in the risk for heart failure and for death after MI, Dr. Silvain explained. 

This has led to questions about the add-on benefits of lifelong beta-blocker treatment for patients with MI and a preserved left ventricular ejection fraction and no other primary indication for beta-blocker therapy.
 

The ABYSS Trial

To explore this issue, the open-label, non-inferiority ABYSS trial randomly assigned 3698 patients with a history of MI to the discontinuation or continuation of beta-blocker treatment. All study participants had a left ventricular ejection fraction of at least 40%, were receiving long-term beta-blocker treatment, and had experienced no cardiovascular event in the previous 6 months. 

At a median follow-up of 3 years, the primary endpoint — a composite of death, MI, stroke, and hospitalization for cardiovascular reasons — occurred more often in the discontinuation group than in the continuation group (23.8% vs 21.1%; hazard ratio, 1.16; 95% CI, 1.01-1.33). This did not meet the criteria for non-inferiority of discontinuation, compared with continuation, of beta-blocker therapy (P for non-inferiority = .44).

The difference in event rates between the two groups was driven by cardiovascular hospitalizations, which occurred more often in the discontinuation group than in the continuation group (18.9% vs 16.6%).

Other key results showed that there was no difference in quality of life between the two groups.

However, 6 months after randomization, there were increases in blood pressure and heart rate in the discontinuation group. Systolic blood pressure increased by 3.7 mm Hg and diastolic blood pressure increased by 3.9 mm Hg. Resting heart rate increased by 9.8 beats per minute.

“We were not able to show the non-inferiority of stopping beta-blockers in terms of cardiovascular events, [but we] showed a safety signal with this strategy of an increase in blood pressure and heart rate, with no improvement in quality of life,” Dr. Sylvain said.

“While recent guidelines suggest it may be reasonable to stop beta-blockers in this population, after these results, I will not be stopping these drugs if they are being well tolerated,” he said.

Sylvain said he was surprised that there was not an improvement in quality of life in the group that discontinued beta-blockers. “We are always told that beta-blockers have many side effects, so we expected to see an improvement in quality of life in the patients who stopped these drugs.”

One possible reason for the lack of improvement in quality of life is that the trial participants had been taking beta-blockers for several years. “We may have, therefore, selected patients who tolerate these drugs quite well. Those who had tolerance issues had probably already stopped taking them,” he explained.

In addition, the patient population had relatively high quality-of-life scores at baseline. “They were well treated and the therapies they were taking were well tolerated, so maybe it is difficult to improve quality of life further,” he said.
 

The REDUCE-AMI Trial

The ABYSS results appear at first to differ from results from the recent REDUCE-AMI trial, which failed to show the superiority of beta-blocker therapy, compared with no beta-blocker therapy, in acute MI patients with preserved ejection fraction.

But the REDUCE-AMI primary endpoint was a composite of death from any cause or new myocardial infarction; it did not include cardiovascular hospitalization, which was the main driver of the difference in outcomes in the ABYSS study, Dr. Sylvain pointed out.

“We showed an increase in coronary cases of hospitalization with stopping beta-blockers, and you have to remember that beta-blockers were developed to reduce coronary disease,” he said.
 

‘Slightly Inconclusive’

Jane Armitage, MBBS, University of Oxford, England, the ABYSS discussant for the ESC HOTLINE session, pointed out some limitations of the study, which led her to report that the result was “slightly inconclusive.” 

The open-label design may have allowed some bias regarding the cardiovascular hospitalization endpoint, she said.

“The decision whether to admit a patient to [the] hospital is somewhat subjective and could be influenced by a physician’s knowledge of treatment allocation. That is why, ideally, we prefer blinded trials. I think there are questions there,” she explained.

She also questioned whether the non-inferiority margin could have been increased, given the higher-than-expected event rate.

More data on this issue will come from several trials that are currently ongoing, Dr. Armitage said.

The ABYSS and REDUCE-AMI trials together suggest that it is safe, with respect to serious cardiac events, to stop beta-blocker treatment in MI patients with preserved ejection fraction, writes Tomas Jernberg, MD, PhD, from the Karolinska Institute in Stockholm, Sweden, in an accompanying editorial.

However, “because of the anti-ischemic effects of beta-blockers, an interruption may increase the risk of recurrent angina and the need for rehospitalization,” he adds.

“It is prudent to wait for the results of additional ongoing trials of beta-blockers involving patients with MI and a preserved left ventricular ejection fraction before definitively updating guidelines,” Dr. Jernberg concludes.

The ABYSS trial was funded by the French Ministry of Health and the ACTION Study Group. Dr. Sylvain, Dr. Armitage, and Dr. Jernberg report no relevant financial relationships.

A version of this article appeared on Medscape.com.

LONDON — It may not be advisable for patients with a history of myocardial infarction and preserved left ventricular function to discontinue long-term beta-blocker therapy, warn investigators.

In the randomized ABYSS trial, although there was no difference in death, MI, or stroke between patients who discontinued and those who continued taking beta-blockers, those who stopped taking the drugs had a higher rate of cardiovascular hospitalization.

Discontinuation was also associated with an increase in blood pressure and heart rate, without any improvement in quality of life.

“We thought we would be able to withdraw beta-blockers safely and that this would result in improved quality of life, but our trial results suggest this is not the case,” said lead investigator Johanne Silvain, MD, PhD, from Pitié-Salpêtrière University Hospital in Paris, who presented the ABYSS findings here at the European Society of Cardiology (ESC) Congress. 

The results, which were simultaneously published online in The New England Journal of Medicine, call into question current guidelines, which suggest that beta-blockers may be discontinued after 1 year in certain patient groups.

Beta-blockers have long been considered the standard of care for patients after MI, but trials showing the benefit of these drugs were conducted before the modern era of myocardial reperfusion and pharmacotherapy, which have led to sharp decreases in the risk for heart failure and for death after MI, Dr. Silvain explained. 

This has led to questions about the add-on benefits of lifelong beta-blocker treatment for patients with MI and a preserved left ventricular ejection fraction and no other primary indication for beta-blocker therapy.
 

The ABYSS Trial

To explore this issue, the open-label, non-inferiority ABYSS trial randomly assigned 3698 patients with a history of MI to the discontinuation or continuation of beta-blocker treatment. All study participants had a left ventricular ejection fraction of at least 40%, were receiving long-term beta-blocker treatment, and had experienced no cardiovascular event in the previous 6 months. 

At a median follow-up of 3 years, the primary endpoint — a composite of death, MI, stroke, and hospitalization for cardiovascular reasons — occurred more often in the discontinuation group than in the continuation group (23.8% vs 21.1%; hazard ratio, 1.16; 95% CI, 1.01-1.33). This did not meet the criteria for non-inferiority of discontinuation, compared with continuation, of beta-blocker therapy (P for non-inferiority = .44).

The difference in event rates between the two groups was driven by cardiovascular hospitalizations, which occurred more often in the discontinuation group than in the continuation group (18.9% vs 16.6%).

Other key results showed that there was no difference in quality of life between the two groups.

However, 6 months after randomization, there were increases in blood pressure and heart rate in the discontinuation group. Systolic blood pressure increased by 3.7 mm Hg and diastolic blood pressure increased by 3.9 mm Hg. Resting heart rate increased by 9.8 beats per minute.

“We were not able to show the non-inferiority of stopping beta-blockers in terms of cardiovascular events, [but we] showed a safety signal with this strategy of an increase in blood pressure and heart rate, with no improvement in quality of life,” Dr. Sylvain said.

“While recent guidelines suggest it may be reasonable to stop beta-blockers in this population, after these results, I will not be stopping these drugs if they are being well tolerated,” he said.

Sylvain said he was surprised that there was not an improvement in quality of life in the group that discontinued beta-blockers. “We are always told that beta-blockers have many side effects, so we expected to see an improvement in quality of life in the patients who stopped these drugs.”

One possible reason for the lack of improvement in quality of life is that the trial participants had been taking beta-blockers for several years. “We may have, therefore, selected patients who tolerate these drugs quite well. Those who had tolerance issues had probably already stopped taking them,” he explained.

In addition, the patient population had relatively high quality-of-life scores at baseline. “They were well treated and the therapies they were taking were well tolerated, so maybe it is difficult to improve quality of life further,” he said.
 

The REDUCE-AMI Trial

The ABYSS results appear at first to differ from results from the recent REDUCE-AMI trial, which failed to show the superiority of beta-blocker therapy, compared with no beta-blocker therapy, in acute MI patients with preserved ejection fraction.

But the REDUCE-AMI primary endpoint was a composite of death from any cause or new myocardial infarction; it did not include cardiovascular hospitalization, which was the main driver of the difference in outcomes in the ABYSS study, Dr. Sylvain pointed out.

“We showed an increase in coronary cases of hospitalization with stopping beta-blockers, and you have to remember that beta-blockers were developed to reduce coronary disease,” he said.
 

‘Slightly Inconclusive’

Jane Armitage, MBBS, University of Oxford, England, the ABYSS discussant for the ESC HOTLINE session, pointed out some limitations of the study, which led her to report that the result was “slightly inconclusive.” 

The open-label design may have allowed some bias regarding the cardiovascular hospitalization endpoint, she said.

“The decision whether to admit a patient to [the] hospital is somewhat subjective and could be influenced by a physician’s knowledge of treatment allocation. That is why, ideally, we prefer blinded trials. I think there are questions there,” she explained.

She also questioned whether the non-inferiority margin could have been increased, given the higher-than-expected event rate.

More data on this issue will come from several trials that are currently ongoing, Dr. Armitage said.

The ABYSS and REDUCE-AMI trials together suggest that it is safe, with respect to serious cardiac events, to stop beta-blocker treatment in MI patients with preserved ejection fraction, writes Tomas Jernberg, MD, PhD, from the Karolinska Institute in Stockholm, Sweden, in an accompanying editorial.

However, “because of the anti-ischemic effects of beta-blockers, an interruption may increase the risk of recurrent angina and the need for rehospitalization,” he adds.

“It is prudent to wait for the results of additional ongoing trials of beta-blockers involving patients with MI and a preserved left ventricular ejection fraction before definitively updating guidelines,” Dr. Jernberg concludes.

The ABYSS trial was funded by the French Ministry of Health and the ACTION Study Group. Dr. Sylvain, Dr. Armitage, and Dr. Jernberg report no relevant financial relationships.

A version of this article appeared on Medscape.com.

LONDON — It may not be advisable for patients with a history of myocardial infarction and preserved left ventricular function to discontinue long-term beta-blocker therapy, warn investigators.

In the randomized ABYSS trial, although there was no difference in death, MI, or stroke between patients who discontinued and those who continued taking beta-blockers, those who stopped taking the drugs had a higher rate of cardiovascular hospitalization.

Discontinuation was also associated with an increase in blood pressure and heart rate, without any improvement in quality of life.

“We thought we would be able to withdraw beta-blockers safely and that this would result in improved quality of life, but our trial results suggest this is not the case,” said lead investigator Johanne Silvain, MD, PhD, from Pitié-Salpêtrière University Hospital in Paris, who presented the ABYSS findings here at the European Society of Cardiology (ESC) Congress. 

The results, which were simultaneously published online in The New England Journal of Medicine, call into question current guidelines, which suggest that beta-blockers may be discontinued after 1 year in certain patient groups.

Beta-blockers have long been considered the standard of care for patients after MI, but trials showing the benefit of these drugs were conducted before the modern era of myocardial reperfusion and pharmacotherapy, which have led to sharp decreases in the risk for heart failure and for death after MI, Dr. Silvain explained. 

This has led to questions about the add-on benefits of lifelong beta-blocker treatment for patients with MI and a preserved left ventricular ejection fraction and no other primary indication for beta-blocker therapy.
 

The ABYSS Trial

To explore this issue, the open-label, non-inferiority ABYSS trial randomly assigned 3698 patients with a history of MI to the discontinuation or continuation of beta-blocker treatment. All study participants had a left ventricular ejection fraction of at least 40%, were receiving long-term beta-blocker treatment, and had experienced no cardiovascular event in the previous 6 months. 

At a median follow-up of 3 years, the primary endpoint — a composite of death, MI, stroke, and hospitalization for cardiovascular reasons — occurred more often in the discontinuation group than in the continuation group (23.8% vs 21.1%; hazard ratio, 1.16; 95% CI, 1.01-1.33). This did not meet the criteria for non-inferiority of discontinuation, compared with continuation, of beta-blocker therapy (P for non-inferiority = .44).

The difference in event rates between the two groups was driven by cardiovascular hospitalizations, which occurred more often in the discontinuation group than in the continuation group (18.9% vs 16.6%).

Other key results showed that there was no difference in quality of life between the two groups.

However, 6 months after randomization, there were increases in blood pressure and heart rate in the discontinuation group. Systolic blood pressure increased by 3.7 mm Hg and diastolic blood pressure increased by 3.9 mm Hg. Resting heart rate increased by 9.8 beats per minute.

“We were not able to show the non-inferiority of stopping beta-blockers in terms of cardiovascular events, [but we] showed a safety signal with this strategy of an increase in blood pressure and heart rate, with no improvement in quality of life,” Dr. Sylvain said.

“While recent guidelines suggest it may be reasonable to stop beta-blockers in this population, after these results, I will not be stopping these drugs if they are being well tolerated,” he said.

Sylvain said he was surprised that there was not an improvement in quality of life in the group that discontinued beta-blockers. “We are always told that beta-blockers have many side effects, so we expected to see an improvement in quality of life in the patients who stopped these drugs.”

One possible reason for the lack of improvement in quality of life is that the trial participants had been taking beta-blockers for several years. “We may have, therefore, selected patients who tolerate these drugs quite well. Those who had tolerance issues had probably already stopped taking them,” he explained.

In addition, the patient population had relatively high quality-of-life scores at baseline. “They were well treated and the therapies they were taking were well tolerated, so maybe it is difficult to improve quality of life further,” he said.
 

The REDUCE-AMI Trial

The ABYSS results appear at first to differ from results from the recent REDUCE-AMI trial, which failed to show the superiority of beta-blocker therapy, compared with no beta-blocker therapy, in acute MI patients with preserved ejection fraction.

But the REDUCE-AMI primary endpoint was a composite of death from any cause or new myocardial infarction; it did not include cardiovascular hospitalization, which was the main driver of the difference in outcomes in the ABYSS study, Dr. Sylvain pointed out.

“We showed an increase in coronary cases of hospitalization with stopping beta-blockers, and you have to remember that beta-blockers were developed to reduce coronary disease,” he said.
 

‘Slightly Inconclusive’

Jane Armitage, MBBS, University of Oxford, England, the ABYSS discussant for the ESC HOTLINE session, pointed out some limitations of the study, which led her to report that the result was “slightly inconclusive.” 

The open-label design may have allowed some bias regarding the cardiovascular hospitalization endpoint, she said.

“The decision whether to admit a patient to [the] hospital is somewhat subjective and could be influenced by a physician’s knowledge of treatment allocation. That is why, ideally, we prefer blinded trials. I think there are questions there,” she explained.

She also questioned whether the non-inferiority margin could have been increased, given the higher-than-expected event rate.

More data on this issue will come from several trials that are currently ongoing, Dr. Armitage said.

The ABYSS and REDUCE-AMI trials together suggest that it is safe, with respect to serious cardiac events, to stop beta-blocker treatment in MI patients with preserved ejection fraction, writes Tomas Jernberg, MD, PhD, from the Karolinska Institute in Stockholm, Sweden, in an accompanying editorial.

However, “because of the anti-ischemic effects of beta-blockers, an interruption may increase the risk of recurrent angina and the need for rehospitalization,” he adds.

“It is prudent to wait for the results of additional ongoing trials of beta-blockers involving patients with MI and a preserved left ventricular ejection fraction before definitively updating guidelines,” Dr. Jernberg concludes.

The ABYSS trial was funded by the French Ministry of Health and the ACTION Study Group. Dr. Sylvain, Dr. Armitage, and Dr. Jernberg report no relevant financial relationships.

A version of this article appeared on Medscape.com.

A medical student’s life is an endless cycle of classes, exams, clinical rotations, and residency preparation. While students typically have little free time, some still manage to build a mega social media presence. On TikTok and Instagram, among other sites, they share medical school experiences and lessons learned in the classroom and advocate for causes such as increased diversity and gender rights in the medical field.

This news organization caught up with a few social media influencers with a large online following to learn how medical students can effectively use social media to build a professional brand and network. Most of the students interviewed said that their social media platforms offered an opportunity to educate others about significant medical developments, feel part of a community with a like-minded audience, and network with doctors who may lead them to a future residency or career path.

Many med students said that they built their large audiences by creating a platform for people of their ethnic background, nationality, race, gender, or simply what others weren’t already talking about. They said they saw a niche in social media that was missing or others hadn’t tackled in the same way.

When Joel Bervell began med school in 2020, he questioned some of the lessons he learned about how race is used in medical practice, which didn’t make sense to him. So, he began his own research. He had about 2000 followers on Instagram at the time.

Mr. Bervell read a new study about pulse oximeters and how they often produce misleading readings on patients with dark skin.

He wondered why he hadn’t learned this in medical school, so he posted it on TikTok. Within 24 hours, about 500,000 people viewed it. Most of the comments were from doctors, nurses, and physician assistants who said they weren’t aware of the disparity.

While his initial posts detailed his journey to medical school and a day-in-the-life of a medical student, he transitioned to posts primarily about race, health equity, and what he perceives as racial bias in medicine.

Now, the fourth-year Ghanaian-American student at the Elson S. Floyd College of Medicine at Washington State University Spokane has close to 1.2 million followers on Instagram and TikTok combined. He frequently visits the White House to advise on social media’s influence on healthcare and has appeared on the Kelly Clarkson Show, Good Morning America, CNN, and ABC, among others.

He said he also uses social media to translate complex medical information for a general audience, many of whom access health information online so they can manage their own healthcare. He sees his social media work as an extension of his medical education, allowing him to delve deeper into subjects and report on them as if he were publishing research in a medical journal.

“When I came to medical school, yes, I wanted to be a doctor. But I also wanted to impact people.” Social media allows him to educate many more people than individual patients, the 29-year-old told this news organization.
 

Inspiring Minorities

Tabhata Paulet, 27, started her TikTok presence as a premed student in 2021. She aimed to provide free resources to help low-income, first-generation Latinx students like herself study for standardized exams.

“I always looked online for guidance and resources, and the medical influencers did not share a similar background. So, I shared my story and what I had to do as a first-generation and first person in my family to become a physician. I did not have access to the same resources as my peers,” said Ms. Paulet, who was born in Peru and came to New Jersey as a child.

Students who are Hispanic, Latinx, or of Spanish origin made up 6.8% of total medical school enrollment in 2023-2024, up slightly from 6.7% in 2022-2023, according to the Association of American Medical Colleges (AAMC).

Ms. Paulet’s online presence grew when she began documenting her experiences as a first-year medical student, bridging the language barrier for Spanish-speaking patients so they could understand their diagnosis and treatment. She often posts about health disparity and barriers to care for underserved communities.

Most of her nearly 22,000 followers are Hispanic, said the now fourth-year student at Rutgers New Jersey Medical School in Newark, New Jersey. “I talk a lot about my interesting Spanish-speaking patients ... and how sometimes speaking their native language truly makes a difference in their care.”

She believes that she serves an important role in social media. “It can be very inspirational for those who come after you [in med school] to see someone from a similar culture and upbringing.”
 

Creating a Community

It was during a therapy session 4 years ago that Jeremy “JP” Scott decided to share Instagram posts about his experiences as a nontraditional medical student. The 37-year-old was studying at Ross University School of Medicine in Barbados and was feeling lonely as an international medical student training to be a doctor as a second career.

Before starting med school, Mr. Scott was an adjunct professor and lab supervisor at the University of Hartford Biology Department, West Hartford, Connecticut, and then a research assistant and lab manager at the Wistar Institute in Philadelphia.

Although he wanted to follow his mother’s path to becoming a doctor, it was more difficult than he envisioned, said the fourth-year student who completed clinical rotations in the United States and is now applying for residencies.

“I talked about how medical school is not what it appears to be ... There are a lot of challenges we are going through,” especially as people of color, he said.

Mr. Scott believes social media helps people feel included and less alone. He said many of his followers are med students and physicians.

His posts often focus on LGBTQIA+ pride and being a minority as a Black man in medicine.

“The pandemic spurred a lot of us. We had a racial reckoning in our country at the time. It inspired us to talk as Black creators and Black medical students.”

Black or African American medical students made up 8.5% of total med school enrollment in 2023-2024, a slight increase from 2022 to 2023, according to AAMC figures. Black men represented 7% of total enrollment in 2023-2024, while Black women represented 9.8%.

After only a handful of online posts in which Mr. Scott candidly discussed his mental health struggles and relationships, he attracted the attention of several medical apparel companies, including the popular FIGS scrubs. He’s now an ambassador for the company, which supports him and his content.

“My association with FIGS has helped attract a wider online audience, increasing my presence.” Today, he has 14,000 Instagram followers. “It opened up so many opportunities,” Mr. Scott said. One example is working with the national LGBTQIA+ community.

“The goal was never to be a social media influencer, to gain sponsorships or photo opportunities,” he said.

“My job, first, is as a medical student. Everything else is second. I am not trying to be a professional social media personality. I’m trying to be an actual physician.” He also tries to separate JP “social media” from Jeremy, the medical student.

“On Instagram, anyone can pull it up and see what you’re doing. The last thing I want is for them to think that I’m not serious about what I’m doing, that I’m not here to learn and become a doctor.”
 

Benefits and Drawbacks

Ms. Paulet said her social media following helped her connect with leaders in the Latinx medical community, including an obstetrics anesthesiologist, her intended specialty. “I don’t think I’d be able to do that without a social media platform.”

Her online activity also propelled her from regional to national leadership in the Latino Medical Student Association (LMSA). She now also runs their Instagram page, which has 14,000 followers.

Mr. Bervell believes social media is a great way to network. He’s connected with people he wouldn’t have met otherwise, including physicians. “I think it will help me get into a residency,” he said. “It allows people to know who you are ... They will be able to tell in a few videos the type of doctor I want to be.”

On the other hand, Mr. Bervell is aware of the negative impacts of social media on mental health. “You can get lost in social media.” For that reason, he often tries to disconnect. “I can go days without my phone.”

Posting on social media can be time-consuming, Mr. Bervell admitted. He said he spent about 2 hours a day researching, editing, and posting on TikTok when he first started building his following. Now, he spends about 2-3 hours a week creating videos. “I don’t post every day anymore. I don’t have the time.”

When she started building her TikTok presence, Ms. Paulet said she devoted 15 hours a week to the endeavor, but now she spends 10-12 hours a week posting online, including on LMSA’s Instagram page. “Whenever you are done with an exam or have a study break, this is something fun to do.” She also says you never know who you’re going to inspire when you put yourself out there.

“Talk about your journey, rotations, or your experience in your first or second year of medical school. Talk about milestones like board exams.”
 

Word to the Wise

Some students may be concerned that their posts might affect a potential residency program. But the medical students interviewed say they want to find programs that align with their values and accept them for who they are.

Mr. Scott said he’s not worried about someone not liking him because of who he is. “I am Black and openly gay. If it’s a problem, I don’t need to work with you or your institution.”

Mr. Bervell stressed that medical students should stay professional online. “I reach 5-10 million people a month, and I have to think: Would I want them to see this? You have to know at all times that someone is watching. I’m very careful about how I post. I script out every video.”

Mr. Scott agreed. He advises those interested in becoming medical influencers to know what they can’t post online. For example, to ensure safety and privacy, Mr. Scott doesn’t take photos in the hospital, show his medical badge, or post patient information. “You want to be respectful of your future medical profession,” he said.

“If it’s something my mother would be ashamed of, I don’t need to post about it.”
 

A version of this article first appeared on Medscape.com.

A medical student’s life is an endless cycle of classes, exams, clinical rotations, and residency preparation. While students typically have little free time, some still manage to build a mega social media presence. On TikTok and Instagram, among other sites, they share medical school experiences and lessons learned in the classroom and advocate for causes such as increased diversity and gender rights in the medical field.

This news organization caught up with a few social media influencers with a large online following to learn how medical students can effectively use social media to build a professional brand and network. Most of the students interviewed said that their social media platforms offered an opportunity to educate others about significant medical developments, feel part of a community with a like-minded audience, and network with doctors who may lead them to a future residency or career path.

Many med students said that they built their large audiences by creating a platform for people of their ethnic background, nationality, race, gender, or simply what others weren’t already talking about. They said they saw a niche in social media that was missing or others hadn’t tackled in the same way.

When Joel Bervell began med school in 2020, he questioned some of the lessons he learned about how race is used in medical practice, which didn’t make sense to him. So, he began his own research. He had about 2000 followers on Instagram at the time.

Mr. Bervell read a new study about pulse oximeters and how they often produce misleading readings on patients with dark skin.

He wondered why he hadn’t learned this in medical school, so he posted it on TikTok. Within 24 hours, about 500,000 people viewed it. Most of the comments were from doctors, nurses, and physician assistants who said they weren’t aware of the disparity.

While his initial posts detailed his journey to medical school and a day-in-the-life of a medical student, he transitioned to posts primarily about race, health equity, and what he perceives as racial bias in medicine.

Now, the fourth-year Ghanaian-American student at the Elson S. Floyd College of Medicine at Washington State University Spokane has close to 1.2 million followers on Instagram and TikTok combined. He frequently visits the White House to advise on social media’s influence on healthcare and has appeared on the Kelly Clarkson Show, Good Morning America, CNN, and ABC, among others.

He said he also uses social media to translate complex medical information for a general audience, many of whom access health information online so they can manage their own healthcare. He sees his social media work as an extension of his medical education, allowing him to delve deeper into subjects and report on them as if he were publishing research in a medical journal.

“When I came to medical school, yes, I wanted to be a doctor. But I also wanted to impact people.” Social media allows him to educate many more people than individual patients, the 29-year-old told this news organization.
 

Inspiring Minorities

Tabhata Paulet, 27, started her TikTok presence as a premed student in 2021. She aimed to provide free resources to help low-income, first-generation Latinx students like herself study for standardized exams.

“I always looked online for guidance and resources, and the medical influencers did not share a similar background. So, I shared my story and what I had to do as a first-generation and first person in my family to become a physician. I did not have access to the same resources as my peers,” said Ms. Paulet, who was born in Peru and came to New Jersey as a child.

Students who are Hispanic, Latinx, or of Spanish origin made up 6.8% of total medical school enrollment in 2023-2024, up slightly from 6.7% in 2022-2023, according to the Association of American Medical Colleges (AAMC).

Ms. Paulet’s online presence grew when she began documenting her experiences as a first-year medical student, bridging the language barrier for Spanish-speaking patients so they could understand their diagnosis and treatment. She often posts about health disparity and barriers to care for underserved communities.

Most of her nearly 22,000 followers are Hispanic, said the now fourth-year student at Rutgers New Jersey Medical School in Newark, New Jersey. “I talk a lot about my interesting Spanish-speaking patients ... and how sometimes speaking their native language truly makes a difference in their care.”

She believes that she serves an important role in social media. “It can be very inspirational for those who come after you [in med school] to see someone from a similar culture and upbringing.”
 

Creating a Community

It was during a therapy session 4 years ago that Jeremy “JP” Scott decided to share Instagram posts about his experiences as a nontraditional medical student. The 37-year-old was studying at Ross University School of Medicine in Barbados and was feeling lonely as an international medical student training to be a doctor as a second career.

Before starting med school, Mr. Scott was an adjunct professor and lab supervisor at the University of Hartford Biology Department, West Hartford, Connecticut, and then a research assistant and lab manager at the Wistar Institute in Philadelphia.

Although he wanted to follow his mother’s path to becoming a doctor, it was more difficult than he envisioned, said the fourth-year student who completed clinical rotations in the United States and is now applying for residencies.

“I talked about how medical school is not what it appears to be ... There are a lot of challenges we are going through,” especially as people of color, he said.

Mr. Scott believes social media helps people feel included and less alone. He said many of his followers are med students and physicians.

His posts often focus on LGBTQIA+ pride and being a minority as a Black man in medicine.

“The pandemic spurred a lot of us. We had a racial reckoning in our country at the time. It inspired us to talk as Black creators and Black medical students.”

Black or African American medical students made up 8.5% of total med school enrollment in 2023-2024, a slight increase from 2022 to 2023, according to AAMC figures. Black men represented 7% of total enrollment in 2023-2024, while Black women represented 9.8%.

After only a handful of online posts in which Mr. Scott candidly discussed his mental health struggles and relationships, he attracted the attention of several medical apparel companies, including the popular FIGS scrubs. He’s now an ambassador for the company, which supports him and his content.

“My association with FIGS has helped attract a wider online audience, increasing my presence.” Today, he has 14,000 Instagram followers. “It opened up so many opportunities,” Mr. Scott said. One example is working with the national LGBTQIA+ community.

“The goal was never to be a social media influencer, to gain sponsorships or photo opportunities,” he said.

“My job, first, is as a medical student. Everything else is second. I am not trying to be a professional social media personality. I’m trying to be an actual physician.” He also tries to separate JP “social media” from Jeremy, the medical student.

“On Instagram, anyone can pull it up and see what you’re doing. The last thing I want is for them to think that I’m not serious about what I’m doing, that I’m not here to learn and become a doctor.”
 

Benefits and Drawbacks

Ms. Paulet said her social media following helped her connect with leaders in the Latinx medical community, including an obstetrics anesthesiologist, her intended specialty. “I don’t think I’d be able to do that without a social media platform.”

Her online activity also propelled her from regional to national leadership in the Latino Medical Student Association (LMSA). She now also runs their Instagram page, which has 14,000 followers.

Mr. Bervell believes social media is a great way to network. He’s connected with people he wouldn’t have met otherwise, including physicians. “I think it will help me get into a residency,” he said. “It allows people to know who you are ... They will be able to tell in a few videos the type of doctor I want to be.”

On the other hand, Mr. Bervell is aware of the negative impacts of social media on mental health. “You can get lost in social media.” For that reason, he often tries to disconnect. “I can go days without my phone.”

Posting on social media can be time-consuming, Mr. Bervell admitted. He said he spent about 2 hours a day researching, editing, and posting on TikTok when he first started building his following. Now, he spends about 2-3 hours a week creating videos. “I don’t post every day anymore. I don’t have the time.”

When she started building her TikTok presence, Ms. Paulet said she devoted 15 hours a week to the endeavor, but now she spends 10-12 hours a week posting online, including on LMSA’s Instagram page. “Whenever you are done with an exam or have a study break, this is something fun to do.” She also says you never know who you’re going to inspire when you put yourself out there.

“Talk about your journey, rotations, or your experience in your first or second year of medical school. Talk about milestones like board exams.”
 

Word to the Wise

Some students may be concerned that their posts might affect a potential residency program. But the medical students interviewed say they want to find programs that align with their values and accept them for who they are.

Mr. Scott said he’s not worried about someone not liking him because of who he is. “I am Black and openly gay. If it’s a problem, I don’t need to work with you or your institution.”

Mr. Bervell stressed that medical students should stay professional online. “I reach 5-10 million people a month, and I have to think: Would I want them to see this? You have to know at all times that someone is watching. I’m very careful about how I post. I script out every video.”

Mr. Scott agreed. He advises those interested in becoming medical influencers to know what they can’t post online. For example, to ensure safety and privacy, Mr. Scott doesn’t take photos in the hospital, show his medical badge, or post patient information. “You want to be respectful of your future medical profession,” he said.

“If it’s something my mother would be ashamed of, I don’t need to post about it.”
 

A version of this article first appeared on Medscape.com.

A medical student’s life is an endless cycle of classes, exams, clinical rotations, and residency preparation. While students typically have little free time, some still manage to build a mega social media presence. On TikTok and Instagram, among other sites, they share medical school experiences and lessons learned in the classroom and advocate for causes such as increased diversity and gender rights in the medical field.

This news organization caught up with a few social media influencers with a large online following to learn how medical students can effectively use social media to build a professional brand and network. Most of the students interviewed said that their social media platforms offered an opportunity to educate others about significant medical developments, feel part of a community with a like-minded audience, and network with doctors who may lead them to a future residency or career path.

Many med students said that they built their large audiences by creating a platform for people of their ethnic background, nationality, race, gender, or simply what others weren’t already talking about. They said they saw a niche in social media that was missing or others hadn’t tackled in the same way.

When Joel Bervell began med school in 2020, he questioned some of the lessons he learned about how race is used in medical practice, which didn’t make sense to him. So, he began his own research. He had about 2000 followers on Instagram at the time.

Mr. Bervell read a new study about pulse oximeters and how they often produce misleading readings on patients with dark skin.

He wondered why he hadn’t learned this in medical school, so he posted it on TikTok. Within 24 hours, about 500,000 people viewed it. Most of the comments were from doctors, nurses, and physician assistants who said they weren’t aware of the disparity.

While his initial posts detailed his journey to medical school and a day-in-the-life of a medical student, he transitioned to posts primarily about race, health equity, and what he perceives as racial bias in medicine.

Now, the fourth-year Ghanaian-American student at the Elson S. Floyd College of Medicine at Washington State University Spokane has close to 1.2 million followers on Instagram and TikTok combined. He frequently visits the White House to advise on social media’s influence on healthcare and has appeared on the Kelly Clarkson Show, Good Morning America, CNN, and ABC, among others.

He said he also uses social media to translate complex medical information for a general audience, many of whom access health information online so they can manage their own healthcare. He sees his social media work as an extension of his medical education, allowing him to delve deeper into subjects and report on them as if he were publishing research in a medical journal.

“When I came to medical school, yes, I wanted to be a doctor. But I also wanted to impact people.” Social media allows him to educate many more people than individual patients, the 29-year-old told this news organization.
 

Inspiring Minorities

Tabhata Paulet, 27, started her TikTok presence as a premed student in 2021. She aimed to provide free resources to help low-income, first-generation Latinx students like herself study for standardized exams.

“I always looked online for guidance and resources, and the medical influencers did not share a similar background. So, I shared my story and what I had to do as a first-generation and first person in my family to become a physician. I did not have access to the same resources as my peers,” said Ms. Paulet, who was born in Peru and came to New Jersey as a child.

Students who are Hispanic, Latinx, or of Spanish origin made up 6.8% of total medical school enrollment in 2023-2024, up slightly from 6.7% in 2022-2023, according to the Association of American Medical Colleges (AAMC).

Ms. Paulet’s online presence grew when she began documenting her experiences as a first-year medical student, bridging the language barrier for Spanish-speaking patients so they could understand their diagnosis and treatment. She often posts about health disparity and barriers to care for underserved communities.

Most of her nearly 22,000 followers are Hispanic, said the now fourth-year student at Rutgers New Jersey Medical School in Newark, New Jersey. “I talk a lot about my interesting Spanish-speaking patients ... and how sometimes speaking their native language truly makes a difference in their care.”

She believes that she serves an important role in social media. “It can be very inspirational for those who come after you [in med school] to see someone from a similar culture and upbringing.”
 

Creating a Community

It was during a therapy session 4 years ago that Jeremy “JP” Scott decided to share Instagram posts about his experiences as a nontraditional medical student. The 37-year-old was studying at Ross University School of Medicine in Barbados and was feeling lonely as an international medical student training to be a doctor as a second career.

Before starting med school, Mr. Scott was an adjunct professor and lab supervisor at the University of Hartford Biology Department, West Hartford, Connecticut, and then a research assistant and lab manager at the Wistar Institute in Philadelphia.

Although he wanted to follow his mother’s path to becoming a doctor, it was more difficult than he envisioned, said the fourth-year student who completed clinical rotations in the United States and is now applying for residencies.

“I talked about how medical school is not what it appears to be ... There are a lot of challenges we are going through,” especially as people of color, he said.

Mr. Scott believes social media helps people feel included and less alone. He said many of his followers are med students and physicians.

His posts often focus on LGBTQIA+ pride and being a minority as a Black man in medicine.

“The pandemic spurred a lot of us. We had a racial reckoning in our country at the time. It inspired us to talk as Black creators and Black medical students.”

Black or African American medical students made up 8.5% of total med school enrollment in 2023-2024, a slight increase from 2022 to 2023, according to AAMC figures. Black men represented 7% of total enrollment in 2023-2024, while Black women represented 9.8%.

After only a handful of online posts in which Mr. Scott candidly discussed his mental health struggles and relationships, he attracted the attention of several medical apparel companies, including the popular FIGS scrubs. He’s now an ambassador for the company, which supports him and his content.

“My association with FIGS has helped attract a wider online audience, increasing my presence.” Today, he has 14,000 Instagram followers. “It opened up so many opportunities,” Mr. Scott said. One example is working with the national LGBTQIA+ community.

“The goal was never to be a social media influencer, to gain sponsorships or photo opportunities,” he said.

“My job, first, is as a medical student. Everything else is second. I am not trying to be a professional social media personality. I’m trying to be an actual physician.” He also tries to separate JP “social media” from Jeremy, the medical student.

“On Instagram, anyone can pull it up and see what you’re doing. The last thing I want is for them to think that I’m not serious about what I’m doing, that I’m not here to learn and become a doctor.”
 

Benefits and Drawbacks

Ms. Paulet said her social media following helped her connect with leaders in the Latinx medical community, including an obstetrics anesthesiologist, her intended specialty. “I don’t think I’d be able to do that without a social media platform.”

Her online activity also propelled her from regional to national leadership in the Latino Medical Student Association (LMSA). She now also runs their Instagram page, which has 14,000 followers.

Mr. Bervell believes social media is a great way to network. He’s connected with people he wouldn’t have met otherwise, including physicians. “I think it will help me get into a residency,” he said. “It allows people to know who you are ... They will be able to tell in a few videos the type of doctor I want to be.”

On the other hand, Mr. Bervell is aware of the negative impacts of social media on mental health. “You can get lost in social media.” For that reason, he often tries to disconnect. “I can go days without my phone.”

Posting on social media can be time-consuming, Mr. Bervell admitted. He said he spent about 2 hours a day researching, editing, and posting on TikTok when he first started building his following. Now, he spends about 2-3 hours a week creating videos. “I don’t post every day anymore. I don’t have the time.”

When she started building her TikTok presence, Ms. Paulet said she devoted 15 hours a week to the endeavor, but now she spends 10-12 hours a week posting online, including on LMSA’s Instagram page. “Whenever you are done with an exam or have a study break, this is something fun to do.” She also says you never know who you’re going to inspire when you put yourself out there.

“Talk about your journey, rotations, or your experience in your first or second year of medical school. Talk about milestones like board exams.”
 

Word to the Wise

Some students may be concerned that their posts might affect a potential residency program. But the medical students interviewed say they want to find programs that align with their values and accept them for who they are.

Mr. Scott said he’s not worried about someone not liking him because of who he is. “I am Black and openly gay. If it’s a problem, I don’t need to work with you or your institution.”

Mr. Bervell stressed that medical students should stay professional online. “I reach 5-10 million people a month, and I have to think: Would I want them to see this? You have to know at all times that someone is watching. I’m very careful about how I post. I script out every video.”

Mr. Scott agreed. He advises those interested in becoming medical influencers to know what they can’t post online. For example, to ensure safety and privacy, Mr. Scott doesn’t take photos in the hospital, show his medical badge, or post patient information. “You want to be respectful of your future medical profession,” he said.

“If it’s something my mother would be ashamed of, I don’t need to post about it.”
 

A version of this article first appeared on Medscape.com.

Earlier in 2024 the American Headache Society issued a position statement that CGRP (calcitonin gene-related peptide) agents are a first-line option for migraine prevention.

No Shinola, Sherlock.

Any of us working frontline neurology have figured that out, including me. And I was, honestly, pretty skeptical of them when they hit the pharmacy shelves. But these days, to quote The Monkees (and Neil Diamond), “I’m a Believer.”

Unfortunately, things don’t quite work out that way. Just because a drug is clearly successful doesn’t make it practical to use first line. Most insurances won’t even let family doctors prescribe them, so they have to send patients to a neurologist (which I’m not complaining about).

Then me and my neuro-brethren have to jump through hoops because of their cost. One month of any of these drugs costs the same as a few years (or more) of generic Topamax, Nortriptyline, Nadolol, etc. Granted, I shouldn’t complain about that, either. If everyone with migraines was getting them it would drive up insurance premiums across the board — including mine.

So, after patients have tried and failed at least two to four other options (depending on their plan) I can usually get a CGRP covered. This involves filling out some forms online and submitting them ... then waiting.

Even if the drug is approved, and successful, that’s still not the end of the story. Depending on the plan I have to get them reauthorized anywhere from every 3 to 12 months. There’s also the chance that in December I’ll get a letter saying the drug won’t be covered starting January, and to try one of the recommended alternatives, like generic Topamax, Nortriptyline, Nadolol, etc. Wash, rinse, repeat.

Having celebrities like Lady Gaga pushing them doesn’t help. The commercials never mention that getting the medication isn’t as easy as “ask your doctor.” Nor does it point out that Lady Gaga won’t have an issue with a CGRP agent’s price tag of $800-$1000 per month, while most of her fans need that money for rent and groceries.

The guidelines, in essence, are useful, but only apply to a perfect world where drug cost doesn’t matter. We aren’t in one. I’m not knocking the pharmaceutical companies — research and development take A LOT of money, and every drug that comes to market has to pay not only for itself, but for several others that failed. Innovation isn’t cheap.

That doesn’t make it any easier to explain to patients, who see ads, or news blurbs on Facebook, or whatever. I just wish the advertisements would have more transparency about how the pricing works.

After all, regardless of how good an automobile may be, don’t car ads show an MSRP?

Dr. Block has a solo neurology practice in Scottsdale, Arizona.

Earlier in 2024 the American Headache Society issued a position statement that CGRP (calcitonin gene-related peptide) agents are a first-line option for migraine prevention.

No Shinola, Sherlock.

Any of us working frontline neurology have figured that out, including me. And I was, honestly, pretty skeptical of them when they hit the pharmacy shelves. But these days, to quote The Monkees (and Neil Diamond), “I’m a Believer.”

Unfortunately, things don’t quite work out that way. Just because a drug is clearly successful doesn’t make it practical to use first line. Most insurances won’t even let family doctors prescribe them, so they have to send patients to a neurologist (which I’m not complaining about).

Then me and my neuro-brethren have to jump through hoops because of their cost. One month of any of these drugs costs the same as a few years (or more) of generic Topamax, Nortriptyline, Nadolol, etc. Granted, I shouldn’t complain about that, either. If everyone with migraines was getting them it would drive up insurance premiums across the board — including mine.

So, after patients have tried and failed at least two to four other options (depending on their plan) I can usually get a CGRP covered. This involves filling out some forms online and submitting them ... then waiting.

Even if the drug is approved, and successful, that’s still not the end of the story. Depending on the plan I have to get them reauthorized anywhere from every 3 to 12 months. There’s also the chance that in December I’ll get a letter saying the drug won’t be covered starting January, and to try one of the recommended alternatives, like generic Topamax, Nortriptyline, Nadolol, etc. Wash, rinse, repeat.

Having celebrities like Lady Gaga pushing them doesn’t help. The commercials never mention that getting the medication isn’t as easy as “ask your doctor.” Nor does it point out that Lady Gaga won’t have an issue with a CGRP agent’s price tag of $800-$1000 per month, while most of her fans need that money for rent and groceries.

The guidelines, in essence, are useful, but only apply to a perfect world where drug cost doesn’t matter. We aren’t in one. I’m not knocking the pharmaceutical companies — research and development take A LOT of money, and every drug that comes to market has to pay not only for itself, but for several others that failed. Innovation isn’t cheap.

That doesn’t make it any easier to explain to patients, who see ads, or news blurbs on Facebook, or whatever. I just wish the advertisements would have more transparency about how the pricing works.

After all, regardless of how good an automobile may be, don’t car ads show an MSRP?

Dr. Block has a solo neurology practice in Scottsdale, Arizona.

Earlier in 2024 the American Headache Society issued a position statement that CGRP (calcitonin gene-related peptide) agents are a first-line option for migraine prevention.

No Shinola, Sherlock.

Any of us working frontline neurology have figured that out, including me. And I was, honestly, pretty skeptical of them when they hit the pharmacy shelves. But these days, to quote The Monkees (and Neil Diamond), “I’m a Believer.”

Unfortunately, things don’t quite work out that way. Just because a drug is clearly successful doesn’t make it practical to use first line. Most insurances won’t even let family doctors prescribe them, so they have to send patients to a neurologist (which I’m not complaining about).

Then me and my neuro-brethren have to jump through hoops because of their cost. One month of any of these drugs costs the same as a few years (or more) of generic Topamax, Nortriptyline, Nadolol, etc. Granted, I shouldn’t complain about that, either. If everyone with migraines was getting them it would drive up insurance premiums across the board — including mine.

So, after patients have tried and failed at least two to four other options (depending on their plan) I can usually get a CGRP covered. This involves filling out some forms online and submitting them ... then waiting.

Even if the drug is approved, and successful, that’s still not the end of the story. Depending on the plan I have to get them reauthorized anywhere from every 3 to 12 months. There’s also the chance that in December I’ll get a letter saying the drug won’t be covered starting January, and to try one of the recommended alternatives, like generic Topamax, Nortriptyline, Nadolol, etc. Wash, rinse, repeat.

Having celebrities like Lady Gaga pushing them doesn’t help. The commercials never mention that getting the medication isn’t as easy as “ask your doctor.” Nor does it point out that Lady Gaga won’t have an issue with a CGRP agent’s price tag of $800-$1000 per month, while most of her fans need that money for rent and groceries.

The guidelines, in essence, are useful, but only apply to a perfect world where drug cost doesn’t matter. We aren’t in one. I’m not knocking the pharmaceutical companies — research and development take A LOT of money, and every drug that comes to market has to pay not only for itself, but for several others that failed. Innovation isn’t cheap.

That doesn’t make it any easier to explain to patients, who see ads, or news blurbs on Facebook, or whatever. I just wish the advertisements would have more transparency about how the pricing works.

After all, regardless of how good an automobile may be, don’t car ads show an MSRP?

Dr. Block has a solo neurology practice in Scottsdale, Arizona.

Advocacy groups for patients with neurologic disorders have become a common feature in the landscape of drug and device development and federal research funding allocation.

On Capitol Hill, advocates have racked up some impressive legislative wins that aim to set a federal agenda for developing new medications.

At the Food and Drug Administration (FDA), advocacy groups played a significant role in several recent high-profile and controversial approvals for drugs for Alzheimer’s disease, Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS).

Such gains suggest these groups are growing in power. But with these wins come questions about whether large advocacy organizations — some of which receive significant industry funding — wield too much influence.

“You need to think very carefully about how you open these processes up to greater patient involvement,” Matthew S. McCoy, PhD, assistant professor of medical ethics and health policy at the University of Pennsylvania, Philadelphia, told this news organization. It’s important not to “end up with a situation where it’s the best-connected, the most well-resourced, the most-savvy patient organizations that are able to exercise outsize influence.”

Just because a group has deep pockets does not mean that its priorities align with the disease burden. And not every patient population is represented by a professionalized patient advocacy organization, Dr. McCoy noted. “There is the potential for the rich to get richer.”
 

A Seat at the Table

Long ago, the FDA and the National Institutes of Health (NIH) began giving patients a seat at the table, in part because of the path blazed by AIDS activists in the late 1980s and early 1990s, said Dr. McCoy.

Patient advocacy is often visible during FDA advisory committee meetings. The agency usually allows an hour, sometimes more, for members of the public to express support or concerns about the product being reviewed. Patients and caregivers — often aided by advocacy organizations — also submit hundreds, sometimes thousands, of letters before a product review.

The Alzheimer’s Association spent years advocating for approval of the anti-amyloid agent aducanumab (Aduhelm, Biogen/Eisai). In 2020, the organization urged patients and caregivers to submit written and oral testimony to the FDA advisory panel that was reviewing the drug. Despite patients’ pleas, the panel ultimately declined to support the drug’s approval, citing safety concerns and limited evidence of efficacy.

As controversy swirled around the medication — which had the potential for life-threatening brain swelling — advocates continued to apply pressure. Going against the expert panel’s recommendation, in June 2021, the FDA granted accelerated approval prompting three of the panelists to resign in protest.

Aducanumab’s initial price — $56,000 a year — was seen as a major threat to the viability of Medicare. Still, the Alzheimer’s Association stood behind the decision to approve the drug. But by early 2024, Biogen/Eisai said they would stop selling aducanumab, citing other priorities.

Once again patient advocates showed up in March 2022 when the FDA advisers were reviewing Amylyx Pharmaceuticals’ ALS drug Relyvrio (sodium phenylbutyrate and taurursodiol). Trials had showed limited efficacy, but patients testified they would accept greater risk for a chance to be treated with the drug. The committee ultimately voted against approval; 6 months later, the FDA approved Relyvrio anyway.

In April 2024, Amylyx removed Relyvrio from the market following phase 3 trial results that showed no difference between the treatment and placebo.

The drug manufacturer Sarepta Therapeutics, which develops treatments for genetic conditions such as DMD, has a history of working with — and funding — patient advocacy groups. The company encourages nonprofits to apply for grants or sponsorship on its website. At a 2016 advisory committee, when Sarepta was seeking approval of its first DMD therapy eteplirsen (Exondys 51), 52 speakers, most from patient advocacy groups, pleaded for the drug’s approval. When the panel voted no, Sarepta mobilized families to pressure the agency. Exondys was eventually approved.

In June, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, unilaterally gave final expanded approval to Sarepta Therapeutics’ gene therapy Elevidys for DMD. Dr. Marks overrode his own FDA reviewers, who said the product lacked substantial evidence of efficacy. He acknowledged the drug had not met its primary endpoint but said he found secondary and exploratory endpoints “compelling” and cited an unmet medical need.

In an opinion piece in The Washington Post, Aaron Kesselheim, MD, JD, MPH, the director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, Boston, Massachusetts, and a former member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, questioned the approval stating that it undermined both public trust and manufacturers’ incentives to do the hard work of proving effectiveness.
 

Patient Voices the ‘Secret Sauce’

Drugmakers aren’t alone in seeing the value of having patients speak directly to government entities. When the Michael J. Fox Foundation wanted to gather cosponsors for the National Plan to End Parkinson’s Act, which President Joe Biden signed into law in July, it recruited and trained patients and caregivers for congressional meetings, said Ted Thompson, senior vice president of public policy at the foundation.

Having those individuals “making the personal case for how this disease affects their families ... was really the secret sauce,” in garnering a large number of cosponsors and getting legislation signed into law within 2 years of its introduction, Mr. Thompson told this news organization.

ALS advocacy groups launched a similar campaign to secure passage of the Accelerating Access to Critical Therapies for ALS Act in 2021.

Both pieces of legislation seek to set a federal agenda for developing new therapies in neurodegenerative diseases, in part by directing the FDA and NIH to fund research, engage patients more directly, and form public-private partnerships and councils to spur innovation.

But some said patient advocates are still coming far too late to the party.

“By the time you hear from patient groups at the meetings at the FDA, often the best opportunities for their input are long past,” Leah Zoe Gibson Rand, DPhil, a research scientist with the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, told this news organization. There should be more focus on the patient perspective earlier in drug development and trial design.

“There are some things that the patient voice could uniquely tell the agency,” said Holly Fernandez Lynch, JD, associate professor of medical ethics and health policy at the University of Pennsylvania. Patients can give insight on what it means to live with a disease, what symptoms are particularly burdensome, and which endpoints matter.

But, she said, “listening to the patient voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try.” Individuals “who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.”

If the FDA allows drugs on the market just because patients are willing to try, “5 or 10 years down the road, it’s not at all clear that we would end up with drugs that are better, or drugs that work, or drugs that we know anything more about,” said Dr. Lynch.
 

Does Taking Industry Money Equal Conflicts of Interest?

Many patient advocacy organizations receive funding from drug companies, medical device makers, or other industry sources, but they aren’t always transparent about how much or from which companies, according to studies.

The Alzheimer’s Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug.

It is not uncommon for individuals who speak in favor of a product’s approval to receive money for transportation and/or lodging from the drug’s manufacturer. In 2018, Dr. McCoy and colleagues reported in JAMA Internal Medicine that, between 2009 and 2017, a quarter of the speakers at the Anesthetic and Analgesic Drug Products Advisory Committee had conflicts of interest (COIs), mostly from industry, and that they were not disclosed in approximately 20% of the instances.

In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, Dr. McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions. Of these, only 12% had published policies for managing institutional COIs.

Dr. McCoy emphasized the industry’s reliance on partnering with patient groups, particularly during FDA advisory committee meetings. “The sponsors wouldn’t be paying for patients to show up and give these testimonies if they didn’t think it made a difference. The audience isn’t just panel members; it’s also agency officials and maybe elected officials as well.”

“The Fox Foundation, with a $300 million-plus budget, gets about $5-$6 million a year from industry,” said Mr. Thompson. The money is earmarked for the organization’s Parkinson’s Disease Education Consortium; none goes toward advocacy. And, “the foundation has never specifically endorsed a product or device.”

When organizations that receive industry funding back a particular product, “it does appear to be [a conflict], and whether it is an actual one or not, appearances sometimes are all that matter,” said Mr. Thompson.

Dr. Lynch said accepting industry money “is a really significant conflict.” While advocates might need that money to fund advocacy efforts or make grants to advance research priorities, the acceptance might hinder willingness to demand evidence or to complain about a product’s price tag. “You don’t want to bite the hand that feeds you, right?”

Both Dr. McCoy and Dr. Lynch said patient groups — and individual patients — should at a minimum disclose industry funding, especially when speaking at an advisory committee.

Federal agencies and members of Congress actively seek patient input when considering legislation and funding priorities. But the individuals testifying at an advisory committee aren’t likely to represent all patients, and there’s a danger that they are just the loudest voices, said Dr. McCoy.

“We need to think more carefully about how we actually understand the preferences of a big, diverse patient population,” he said.

Dr. Lynch agreed.

Within the ALS community, “a lot of people who take different perspectives than some of those that are the leading voices get shouted down, and their voices get drowned out, and they get attacked on social media,” she said.

The group may be at the table, “but they’re just one voice at the table,” she said.

Dr. McCoy reported that his wife works for the Leukemia & Lymphoma Society, a patient advocacy organization. Dr. Rand reported no relevant financial relationships. Dr. Lynch received funding from Arnold Ventures and the Greenwall Foundation for work related to the FDA and patient advocacy.

A version of this article first appeared on Medscape.com.

Advocacy groups for patients with neurologic disorders have become a common feature in the landscape of drug and device development and federal research funding allocation.

On Capitol Hill, advocates have racked up some impressive legislative wins that aim to set a federal agenda for developing new medications.

At the Food and Drug Administration (FDA), advocacy groups played a significant role in several recent high-profile and controversial approvals for drugs for Alzheimer’s disease, Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS).

Such gains suggest these groups are growing in power. But with these wins come questions about whether large advocacy organizations — some of which receive significant industry funding — wield too much influence.

“You need to think very carefully about how you open these processes up to greater patient involvement,” Matthew S. McCoy, PhD, assistant professor of medical ethics and health policy at the University of Pennsylvania, Philadelphia, told this news organization. It’s important not to “end up with a situation where it’s the best-connected, the most well-resourced, the most-savvy patient organizations that are able to exercise outsize influence.”

Just because a group has deep pockets does not mean that its priorities align with the disease burden. And not every patient population is represented by a professionalized patient advocacy organization, Dr. McCoy noted. “There is the potential for the rich to get richer.”
 

A Seat at the Table

Long ago, the FDA and the National Institutes of Health (NIH) began giving patients a seat at the table, in part because of the path blazed by AIDS activists in the late 1980s and early 1990s, said Dr. McCoy.

Patient advocacy is often visible during FDA advisory committee meetings. The agency usually allows an hour, sometimes more, for members of the public to express support or concerns about the product being reviewed. Patients and caregivers — often aided by advocacy organizations — also submit hundreds, sometimes thousands, of letters before a product review.

The Alzheimer’s Association spent years advocating for approval of the anti-amyloid agent aducanumab (Aduhelm, Biogen/Eisai). In 2020, the organization urged patients and caregivers to submit written and oral testimony to the FDA advisory panel that was reviewing the drug. Despite patients’ pleas, the panel ultimately declined to support the drug’s approval, citing safety concerns and limited evidence of efficacy.

As controversy swirled around the medication — which had the potential for life-threatening brain swelling — advocates continued to apply pressure. Going against the expert panel’s recommendation, in June 2021, the FDA granted accelerated approval prompting three of the panelists to resign in protest.

Aducanumab’s initial price — $56,000 a year — was seen as a major threat to the viability of Medicare. Still, the Alzheimer’s Association stood behind the decision to approve the drug. But by early 2024, Biogen/Eisai said they would stop selling aducanumab, citing other priorities.

Once again patient advocates showed up in March 2022 when the FDA advisers were reviewing Amylyx Pharmaceuticals’ ALS drug Relyvrio (sodium phenylbutyrate and taurursodiol). Trials had showed limited efficacy, but patients testified they would accept greater risk for a chance to be treated with the drug. The committee ultimately voted against approval; 6 months later, the FDA approved Relyvrio anyway.

In April 2024, Amylyx removed Relyvrio from the market following phase 3 trial results that showed no difference between the treatment and placebo.

The drug manufacturer Sarepta Therapeutics, which develops treatments for genetic conditions such as DMD, has a history of working with — and funding — patient advocacy groups. The company encourages nonprofits to apply for grants or sponsorship on its website. At a 2016 advisory committee, when Sarepta was seeking approval of its first DMD therapy eteplirsen (Exondys 51), 52 speakers, most from patient advocacy groups, pleaded for the drug’s approval. When the panel voted no, Sarepta mobilized families to pressure the agency. Exondys was eventually approved.

In June, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, unilaterally gave final expanded approval to Sarepta Therapeutics’ gene therapy Elevidys for DMD. Dr. Marks overrode his own FDA reviewers, who said the product lacked substantial evidence of efficacy. He acknowledged the drug had not met its primary endpoint but said he found secondary and exploratory endpoints “compelling” and cited an unmet medical need.

In an opinion piece in The Washington Post, Aaron Kesselheim, MD, JD, MPH, the director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, Boston, Massachusetts, and a former member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, questioned the approval stating that it undermined both public trust and manufacturers’ incentives to do the hard work of proving effectiveness.
 

Patient Voices the ‘Secret Sauce’

Drugmakers aren’t alone in seeing the value of having patients speak directly to government entities. When the Michael J. Fox Foundation wanted to gather cosponsors for the National Plan to End Parkinson’s Act, which President Joe Biden signed into law in July, it recruited and trained patients and caregivers for congressional meetings, said Ted Thompson, senior vice president of public policy at the foundation.

Having those individuals “making the personal case for how this disease affects their families ... was really the secret sauce,” in garnering a large number of cosponsors and getting legislation signed into law within 2 years of its introduction, Mr. Thompson told this news organization.

ALS advocacy groups launched a similar campaign to secure passage of the Accelerating Access to Critical Therapies for ALS Act in 2021.

Both pieces of legislation seek to set a federal agenda for developing new therapies in neurodegenerative diseases, in part by directing the FDA and NIH to fund research, engage patients more directly, and form public-private partnerships and councils to spur innovation.

But some said patient advocates are still coming far too late to the party.

“By the time you hear from patient groups at the meetings at the FDA, often the best opportunities for their input are long past,” Leah Zoe Gibson Rand, DPhil, a research scientist with the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, told this news organization. There should be more focus on the patient perspective earlier in drug development and trial design.

“There are some things that the patient voice could uniquely tell the agency,” said Holly Fernandez Lynch, JD, associate professor of medical ethics and health policy at the University of Pennsylvania. Patients can give insight on what it means to live with a disease, what symptoms are particularly burdensome, and which endpoints matter.

But, she said, “listening to the patient voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try.” Individuals “who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.”

If the FDA allows drugs on the market just because patients are willing to try, “5 or 10 years down the road, it’s not at all clear that we would end up with drugs that are better, or drugs that work, or drugs that we know anything more about,” said Dr. Lynch.
 

Does Taking Industry Money Equal Conflicts of Interest?

Many patient advocacy organizations receive funding from drug companies, medical device makers, or other industry sources, but they aren’t always transparent about how much or from which companies, according to studies.

The Alzheimer’s Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug.

It is not uncommon for individuals who speak in favor of a product’s approval to receive money for transportation and/or lodging from the drug’s manufacturer. In 2018, Dr. McCoy and colleagues reported in JAMA Internal Medicine that, between 2009 and 2017, a quarter of the speakers at the Anesthetic and Analgesic Drug Products Advisory Committee had conflicts of interest (COIs), mostly from industry, and that they were not disclosed in approximately 20% of the instances.

In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, Dr. McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions. Of these, only 12% had published policies for managing institutional COIs.

Dr. McCoy emphasized the industry’s reliance on partnering with patient groups, particularly during FDA advisory committee meetings. “The sponsors wouldn’t be paying for patients to show up and give these testimonies if they didn’t think it made a difference. The audience isn’t just panel members; it’s also agency officials and maybe elected officials as well.”

“The Fox Foundation, with a $300 million-plus budget, gets about $5-$6 million a year from industry,” said Mr. Thompson. The money is earmarked for the organization’s Parkinson’s Disease Education Consortium; none goes toward advocacy. And, “the foundation has never specifically endorsed a product or device.”

When organizations that receive industry funding back a particular product, “it does appear to be [a conflict], and whether it is an actual one or not, appearances sometimes are all that matter,” said Mr. Thompson.

Dr. Lynch said accepting industry money “is a really significant conflict.” While advocates might need that money to fund advocacy efforts or make grants to advance research priorities, the acceptance might hinder willingness to demand evidence or to complain about a product’s price tag. “You don’t want to bite the hand that feeds you, right?”

Both Dr. McCoy and Dr. Lynch said patient groups — and individual patients — should at a minimum disclose industry funding, especially when speaking at an advisory committee.

Federal agencies and members of Congress actively seek patient input when considering legislation and funding priorities. But the individuals testifying at an advisory committee aren’t likely to represent all patients, and there’s a danger that they are just the loudest voices, said Dr. McCoy.

“We need to think more carefully about how we actually understand the preferences of a big, diverse patient population,” he said.

Dr. Lynch agreed.

Within the ALS community, “a lot of people who take different perspectives than some of those that are the leading voices get shouted down, and their voices get drowned out, and they get attacked on social media,” she said.

The group may be at the table, “but they’re just one voice at the table,” she said.

Dr. McCoy reported that his wife works for the Leukemia & Lymphoma Society, a patient advocacy organization. Dr. Rand reported no relevant financial relationships. Dr. Lynch received funding from Arnold Ventures and the Greenwall Foundation for work related to the FDA and patient advocacy.

A version of this article first appeared on Medscape.com.

Advocacy groups for patients with neurologic disorders have become a common feature in the landscape of drug and device development and federal research funding allocation.

On Capitol Hill, advocates have racked up some impressive legislative wins that aim to set a federal agenda for developing new medications.

At the Food and Drug Administration (FDA), advocacy groups played a significant role in several recent high-profile and controversial approvals for drugs for Alzheimer’s disease, Duchenne muscular dystrophy (DMD), and amyotrophic lateral sclerosis (ALS).

Such gains suggest these groups are growing in power. But with these wins come questions about whether large advocacy organizations — some of which receive significant industry funding — wield too much influence.

“You need to think very carefully about how you open these processes up to greater patient involvement,” Matthew S. McCoy, PhD, assistant professor of medical ethics and health policy at the University of Pennsylvania, Philadelphia, told this news organization. It’s important not to “end up with a situation where it’s the best-connected, the most well-resourced, the most-savvy patient organizations that are able to exercise outsize influence.”

Just because a group has deep pockets does not mean that its priorities align with the disease burden. And not every patient population is represented by a professionalized patient advocacy organization, Dr. McCoy noted. “There is the potential for the rich to get richer.”
 

A Seat at the Table

Long ago, the FDA and the National Institutes of Health (NIH) began giving patients a seat at the table, in part because of the path blazed by AIDS activists in the late 1980s and early 1990s, said Dr. McCoy.

Patient advocacy is often visible during FDA advisory committee meetings. The agency usually allows an hour, sometimes more, for members of the public to express support or concerns about the product being reviewed. Patients and caregivers — often aided by advocacy organizations — also submit hundreds, sometimes thousands, of letters before a product review.

The Alzheimer’s Association spent years advocating for approval of the anti-amyloid agent aducanumab (Aduhelm, Biogen/Eisai). In 2020, the organization urged patients and caregivers to submit written and oral testimony to the FDA advisory panel that was reviewing the drug. Despite patients’ pleas, the panel ultimately declined to support the drug’s approval, citing safety concerns and limited evidence of efficacy.

As controversy swirled around the medication — which had the potential for life-threatening brain swelling — advocates continued to apply pressure. Going against the expert panel’s recommendation, in June 2021, the FDA granted accelerated approval prompting three of the panelists to resign in protest.

Aducanumab’s initial price — $56,000 a year — was seen as a major threat to the viability of Medicare. Still, the Alzheimer’s Association stood behind the decision to approve the drug. But by early 2024, Biogen/Eisai said they would stop selling aducanumab, citing other priorities.

Once again patient advocates showed up in March 2022 when the FDA advisers were reviewing Amylyx Pharmaceuticals’ ALS drug Relyvrio (sodium phenylbutyrate and taurursodiol). Trials had showed limited efficacy, but patients testified they would accept greater risk for a chance to be treated with the drug. The committee ultimately voted against approval; 6 months later, the FDA approved Relyvrio anyway.

In April 2024, Amylyx removed Relyvrio from the market following phase 3 trial results that showed no difference between the treatment and placebo.

The drug manufacturer Sarepta Therapeutics, which develops treatments for genetic conditions such as DMD, has a history of working with — and funding — patient advocacy groups. The company encourages nonprofits to apply for grants or sponsorship on its website. At a 2016 advisory committee, when Sarepta was seeking approval of its first DMD therapy eteplirsen (Exondys 51), 52 speakers, most from patient advocacy groups, pleaded for the drug’s approval. When the panel voted no, Sarepta mobilized families to pressure the agency. Exondys was eventually approved.

In June, Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, unilaterally gave final expanded approval to Sarepta Therapeutics’ gene therapy Elevidys for DMD. Dr. Marks overrode his own FDA reviewers, who said the product lacked substantial evidence of efficacy. He acknowledged the drug had not met its primary endpoint but said he found secondary and exploratory endpoints “compelling” and cited an unmet medical need.

In an opinion piece in The Washington Post, Aaron Kesselheim, MD, JD, MPH, the director of the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, Boston, Massachusetts, and a former member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, questioned the approval stating that it undermined both public trust and manufacturers’ incentives to do the hard work of proving effectiveness.
 

Patient Voices the ‘Secret Sauce’

Drugmakers aren’t alone in seeing the value of having patients speak directly to government entities. When the Michael J. Fox Foundation wanted to gather cosponsors for the National Plan to End Parkinson’s Act, which President Joe Biden signed into law in July, it recruited and trained patients and caregivers for congressional meetings, said Ted Thompson, senior vice president of public policy at the foundation.

Having those individuals “making the personal case for how this disease affects their families ... was really the secret sauce,” in garnering a large number of cosponsors and getting legislation signed into law within 2 years of its introduction, Mr. Thompson told this news organization.

ALS advocacy groups launched a similar campaign to secure passage of the Accelerating Access to Critical Therapies for ALS Act in 2021.

Both pieces of legislation seek to set a federal agenda for developing new therapies in neurodegenerative diseases, in part by directing the FDA and NIH to fund research, engage patients more directly, and form public-private partnerships and councils to spur innovation.

But some said patient advocates are still coming far too late to the party.

“By the time you hear from patient groups at the meetings at the FDA, often the best opportunities for their input are long past,” Leah Zoe Gibson Rand, DPhil, a research scientist with the Program on Regulation, Therapeutics, and Law at Brigham and Women’s Hospital, told this news organization. There should be more focus on the patient perspective earlier in drug development and trial design.

“There are some things that the patient voice could uniquely tell the agency,” said Holly Fernandez Lynch, JD, associate professor of medical ethics and health policy at the University of Pennsylvania. Patients can give insight on what it means to live with a disease, what symptoms are particularly burdensome, and which endpoints matter.

But, she said, “listening to the patient voice cannot mean that FDA just steps aside and lets anything on the market that patients are willing to try.” Individuals “who lack good treatment options have a very good reason to want to try things that haven’t yet been proven.”

If the FDA allows drugs on the market just because patients are willing to try, “5 or 10 years down the road, it’s not at all clear that we would end up with drugs that are better, or drugs that work, or drugs that we know anything more about,” said Dr. Lynch.
 

Does Taking Industry Money Equal Conflicts of Interest?

Many patient advocacy organizations receive funding from drug companies, medical device makers, or other industry sources, but they aren’t always transparent about how much or from which companies, according to studies.

The Alzheimer’s Association continued to push for the approval of aducanumab, even as the group received millions of dollars from the drugmakers. The association was accused of failing to disclose the potential conflict. It still lobbied for approval, even after the FDA advisers in 2020 voted against the drug.

It is not uncommon for individuals who speak in favor of a product’s approval to receive money for transportation and/or lodging from the drug’s manufacturer. In 2018, Dr. McCoy and colleagues reported in JAMA Internal Medicine that, between 2009 and 2017, a quarter of the speakers at the Anesthetic and Analgesic Drug Products Advisory Committee had conflicts of interest (COIs), mostly from industry, and that they were not disclosed in approximately 20% of the instances.

In a 2017 study of 104 large patient advocacy organizations published in The New England Journal of Medicine, Dr. McCoy and colleagues reported that 83% had received funds from industry. At least 39% had a current or former industry executive on the governing board, and 12% had a current or former industry executive in a board leadership position. Of the 104, 38 were focused on cancer and 13 on neurologic conditions. Of these, only 12% had published policies for managing institutional COIs.

Dr. McCoy emphasized the industry’s reliance on partnering with patient groups, particularly during FDA advisory committee meetings. “The sponsors wouldn’t be paying for patients to show up and give these testimonies if they didn’t think it made a difference. The audience isn’t just panel members; it’s also agency officials and maybe elected officials as well.”

“The Fox Foundation, with a $300 million-plus budget, gets about $5-$6 million a year from industry,” said Mr. Thompson. The money is earmarked for the organization’s Parkinson’s Disease Education Consortium; none goes toward advocacy. And, “the foundation has never specifically endorsed a product or device.”

When organizations that receive industry funding back a particular product, “it does appear to be [a conflict], and whether it is an actual one or not, appearances sometimes are all that matter,” said Mr. Thompson.

Dr. Lynch said accepting industry money “is a really significant conflict.” While advocates might need that money to fund advocacy efforts or make grants to advance research priorities, the acceptance might hinder willingness to demand evidence or to complain about a product’s price tag. “You don’t want to bite the hand that feeds you, right?”

Both Dr. McCoy and Dr. Lynch said patient groups — and individual patients — should at a minimum disclose industry funding, especially when speaking at an advisory committee.

Federal agencies and members of Congress actively seek patient input when considering legislation and funding priorities. But the individuals testifying at an advisory committee aren’t likely to represent all patients, and there’s a danger that they are just the loudest voices, said Dr. McCoy.

“We need to think more carefully about how we actually understand the preferences of a big, diverse patient population,” he said.

Dr. Lynch agreed.

Within the ALS community, “a lot of people who take different perspectives than some of those that are the leading voices get shouted down, and their voices get drowned out, and they get attacked on social media,” she said.

The group may be at the table, “but they’re just one voice at the table,” she said.

Dr. McCoy reported that his wife works for the Leukemia & Lymphoma Society, a patient advocacy organization. Dr. Rand reported no relevant financial relationships. Dr. Lynch received funding from Arnold Ventures and the Greenwall Foundation for work related to the FDA and patient advocacy.

A version of this article first appeared on Medscape.com.

Repeated 1-minute bursts of high-intensity interval training (HIIT) are more effective than conventional moderate, continuous exercise for improving aerobic fitness after stroke, according to a multicenter randomized controlled trial.

“We hoped that we would see improvements in cardiovascular fitness after HIIT and anticipated that these improvements would be greater than in the moderate-intensity group, but we were pleasantly surprised by the degree of improvement we observed,” said Ada Tang, PT, PhD, associate professor of health sciences at McMaster University in Hamilton, Ontario, Canada. “The improvements seen in the HIIT group were twofold higher than in the other group.”

The results were published in Stroke.
 

Clinically Meaningful

Researchers compared the effects of 12 weeks of short-interval HIIT with those of moderate-intensity continuous training (MICT) on peak oxygen uptake (VO₂peak), cardiovascular risk factors, and mobility outcomes after stroke.

They randomly assigned participants to receive 3 days per week of HIIT or traditional moderate exercise sessions for 12 weeks. Participants’ mean age was 65 years, and 39% were women. They enrolled at a mean age of 1.8 years after sustaining a mild stroke.

A total of 42 participants were randomized to HIIT and 40 to MICT. There were no significant differences between the groups at baseline, and both groups exercised on adaptive recumbent steppers, which are suitable for stroke survivors with varying abilities.

The short-interval HIIT protocol involved 10 1-minute intervals of high-intensity exercise, interspersed with nine 1-minute low-intensity intervals, for a total of 19 minutes. HIIT intervals targeted 80% heart rate reserve (HRR) and progressed by 10% every 4 weeks up to 100% HRR. The low-intensity intervals targeted 30% HRR.

The traditional MICT protocol for stroke rehabilitation targeted 40% HRR for 20 minutes and progressed by 10% HRR and 5 minutes every 4 weeks, up to 60% HRR for 30 minutes.

The HIIT group’s cardiorespiratory fitness levels (VO₂peak) improved twice as much as those of the MICT group: 3.5 mL of oxygen consumed in 1 minute per kg of body weight (mL/kg/min) compared with 1.8 mL/kg/min.

Of note, changes in VO₂peak from baseline remained above the clinically important threshold of 1.0 mL/kg/min at 8-week follow-up in the HIIT group (1.71 mL/kg/min) but not in the MICT group (0.67 mL/kg/min).

Both groups increased their 6-minute walk test distances by 8.8 m at 12 weeks and by 18.5 m at 20 weeks. No between-group differences were found for cardiovascular risk or mobility outcomes, and no adverse events occurred in either group.

On average, the HIIT group spent 36% of total training time exercising at intensities above 80% HRR throughout the intervention, while the MICT group spent 42% of time at intensities of 40%-59% HRR.

The study was limited by a small sample size of high-functioning individuals who sustained a mild stroke. Enrollment was halted for 2 years due to the COVID-19 lockdowns, limiting the study’s statistical power.

Nevertheless, the authors concluded, “Given that a lack of time is a significant barrier to the implementation of aerobic exercise in stroke clinical practice, our findings suggest that short-interval HIIT may be an effective alternative to traditional MICT for improving VO₂peak after stroke, with potential clinically meaningful benefits sustained in the short-term.”

“Our findings show that a short HIIT protocol is possible in people with stroke, which is exciting to see,” said Tang. “But there are different factors that clinicians should consider before recommending this training for their patients, such as their health status and their physical status. Stroke rehabilitation specialists, including stroke physical therapists, can advise on how to proceed to ensure the safety and effectiveness of HIIT.”
 

Selected Patients May Benefit

“Broad implementation of this intervention may be premature without further research,” said Ryan Glatt, CPT, senior brain health coach and director of the FitBrain Program at Pacific Neuroscience Institute in Santa Monica, California. “The study focused on relatively high-functioning stroke survivors, which raises questions about the applicability of the results to those with more severe impairments.” Mr. Glatt did not participate in the research.

“Additional studies are needed to confirm whether these findings are applicable to more diverse and severely affected populations and to assess the long-term sustainability of the benefits observed,” he said. “Also, the lack of significant improvements in other critical outcomes, such as mobility, suggests limitations in the broader application of HIIT for stroke rehabilitation.”

“While HIIT shows potential, it should be approached with caution,” Mr. Glatt continued. “It may benefit select patients, but replacing traditional exercise protocols with HIIT should not be done in all cases. More robust evidence and careful consideration of individual patient needs are essential.”

This study was funded by an operating grant from the Canadian Institutes of Health Research. Dr. Tang reported grants from the Canadian Institutes of Health Research, the Physiotherapy Foundation of Canada, and the Heart and Stroke Foundation of Canada. Mr. Glatt declared no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

Repeated 1-minute bursts of high-intensity interval training (HIIT) are more effective than conventional moderate, continuous exercise for improving aerobic fitness after stroke, according to a multicenter randomized controlled trial.

“We hoped that we would see improvements in cardiovascular fitness after HIIT and anticipated that these improvements would be greater than in the moderate-intensity group, but we were pleasantly surprised by the degree of improvement we observed,” said Ada Tang, PT, PhD, associate professor of health sciences at McMaster University in Hamilton, Ontario, Canada. “The improvements seen in the HIIT group were twofold higher than in the other group.”

The results were published in Stroke.
 

Clinically Meaningful

Researchers compared the effects of 12 weeks of short-interval HIIT with those of moderate-intensity continuous training (MICT) on peak oxygen uptake (VO₂peak), cardiovascular risk factors, and mobility outcomes after stroke.

They randomly assigned participants to receive 3 days per week of HIIT or traditional moderate exercise sessions for 12 weeks. Participants’ mean age was 65 years, and 39% were women. They enrolled at a mean age of 1.8 years after sustaining a mild stroke.

A total of 42 participants were randomized to HIIT and 40 to MICT. There were no significant differences between the groups at baseline, and both groups exercised on adaptive recumbent steppers, which are suitable for stroke survivors with varying abilities.

The short-interval HIIT protocol involved 10 1-minute intervals of high-intensity exercise, interspersed with nine 1-minute low-intensity intervals, for a total of 19 minutes. HIIT intervals targeted 80% heart rate reserve (HRR) and progressed by 10% every 4 weeks up to 100% HRR. The low-intensity intervals targeted 30% HRR.

The traditional MICT protocol for stroke rehabilitation targeted 40% HRR for 20 minutes and progressed by 10% HRR and 5 minutes every 4 weeks, up to 60% HRR for 30 minutes.

The HIIT group’s cardiorespiratory fitness levels (VO₂peak) improved twice as much as those of the MICT group: 3.5 mL of oxygen consumed in 1 minute per kg of body weight (mL/kg/min) compared with 1.8 mL/kg/min.

Of note, changes in VO₂peak from baseline remained above the clinically important threshold of 1.0 mL/kg/min at 8-week follow-up in the HIIT group (1.71 mL/kg/min) but not in the MICT group (0.67 mL/kg/min).

Both groups increased their 6-minute walk test distances by 8.8 m at 12 weeks and by 18.5 m at 20 weeks. No between-group differences were found for cardiovascular risk or mobility outcomes, and no adverse events occurred in either group.

On average, the HIIT group spent 36% of total training time exercising at intensities above 80% HRR throughout the intervention, while the MICT group spent 42% of time at intensities of 40%-59% HRR.

The study was limited by a small sample size of high-functioning individuals who sustained a mild stroke. Enrollment was halted for 2 years due to the COVID-19 lockdowns, limiting the study’s statistical power.

Nevertheless, the authors concluded, “Given that a lack of time is a significant barrier to the implementation of aerobic exercise in stroke clinical practice, our findings suggest that short-interval HIIT may be an effective alternative to traditional MICT for improving VO₂peak after stroke, with potential clinically meaningful benefits sustained in the short-term.”

“Our findings show that a short HIIT protocol is possible in people with stroke, which is exciting to see,” said Tang. “But there are different factors that clinicians should consider before recommending this training for their patients, such as their health status and their physical status. Stroke rehabilitation specialists, including stroke physical therapists, can advise on how to proceed to ensure the safety and effectiveness of HIIT.”
 

Selected Patients May Benefit

“Broad implementation of this intervention may be premature without further research,” said Ryan Glatt, CPT, senior brain health coach and director of the FitBrain Program at Pacific Neuroscience Institute in Santa Monica, California. “The study focused on relatively high-functioning stroke survivors, which raises questions about the applicability of the results to those with more severe impairments.” Mr. Glatt did not participate in the research.

“Additional studies are needed to confirm whether these findings are applicable to more diverse and severely affected populations and to assess the long-term sustainability of the benefits observed,” he said. “Also, the lack of significant improvements in other critical outcomes, such as mobility, suggests limitations in the broader application of HIIT for stroke rehabilitation.”

“While HIIT shows potential, it should be approached with caution,” Mr. Glatt continued. “It may benefit select patients, but replacing traditional exercise protocols with HIIT should not be done in all cases. More robust evidence and careful consideration of individual patient needs are essential.”

This study was funded by an operating grant from the Canadian Institutes of Health Research. Dr. Tang reported grants from the Canadian Institutes of Health Research, the Physiotherapy Foundation of Canada, and the Heart and Stroke Foundation of Canada. Mr. Glatt declared no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

Repeated 1-minute bursts of high-intensity interval training (HIIT) are more effective than conventional moderate, continuous exercise for improving aerobic fitness after stroke, according to a multicenter randomized controlled trial.

“We hoped that we would see improvements in cardiovascular fitness after HIIT and anticipated that these improvements would be greater than in the moderate-intensity group, but we were pleasantly surprised by the degree of improvement we observed,” said Ada Tang, PT, PhD, associate professor of health sciences at McMaster University in Hamilton, Ontario, Canada. “The improvements seen in the HIIT group were twofold higher than in the other group.”

The results were published in Stroke.
 

Clinically Meaningful

Researchers compared the effects of 12 weeks of short-interval HIIT with those of moderate-intensity continuous training (MICT) on peak oxygen uptake (VO₂peak), cardiovascular risk factors, and mobility outcomes after stroke.

They randomly assigned participants to receive 3 days per week of HIIT or traditional moderate exercise sessions for 12 weeks. Participants’ mean age was 65 years, and 39% were women. They enrolled at a mean age of 1.8 years after sustaining a mild stroke.

A total of 42 participants were randomized to HIIT and 40 to MICT. There were no significant differences between the groups at baseline, and both groups exercised on adaptive recumbent steppers, which are suitable for stroke survivors with varying abilities.

The short-interval HIIT protocol involved 10 1-minute intervals of high-intensity exercise, interspersed with nine 1-minute low-intensity intervals, for a total of 19 minutes. HIIT intervals targeted 80% heart rate reserve (HRR) and progressed by 10% every 4 weeks up to 100% HRR. The low-intensity intervals targeted 30% HRR.

The traditional MICT protocol for stroke rehabilitation targeted 40% HRR for 20 minutes and progressed by 10% HRR and 5 minutes every 4 weeks, up to 60% HRR for 30 minutes.

The HIIT group’s cardiorespiratory fitness levels (VO₂peak) improved twice as much as those of the MICT group: 3.5 mL of oxygen consumed in 1 minute per kg of body weight (mL/kg/min) compared with 1.8 mL/kg/min.

Of note, changes in VO₂peak from baseline remained above the clinically important threshold of 1.0 mL/kg/min at 8-week follow-up in the HIIT group (1.71 mL/kg/min) but not in the MICT group (0.67 mL/kg/min).

Both groups increased their 6-minute walk test distances by 8.8 m at 12 weeks and by 18.5 m at 20 weeks. No between-group differences were found for cardiovascular risk or mobility outcomes, and no adverse events occurred in either group.

On average, the HIIT group spent 36% of total training time exercising at intensities above 80% HRR throughout the intervention, while the MICT group spent 42% of time at intensities of 40%-59% HRR.

The study was limited by a small sample size of high-functioning individuals who sustained a mild stroke. Enrollment was halted for 2 years due to the COVID-19 lockdowns, limiting the study’s statistical power.

Nevertheless, the authors concluded, “Given that a lack of time is a significant barrier to the implementation of aerobic exercise in stroke clinical practice, our findings suggest that short-interval HIIT may be an effective alternative to traditional MICT for improving VO₂peak after stroke, with potential clinically meaningful benefits sustained in the short-term.”

“Our findings show that a short HIIT protocol is possible in people with stroke, which is exciting to see,” said Tang. “But there are different factors that clinicians should consider before recommending this training for their patients, such as their health status and their physical status. Stroke rehabilitation specialists, including stroke physical therapists, can advise on how to proceed to ensure the safety and effectiveness of HIIT.”
 

Selected Patients May Benefit

“Broad implementation of this intervention may be premature without further research,” said Ryan Glatt, CPT, senior brain health coach and director of the FitBrain Program at Pacific Neuroscience Institute in Santa Monica, California. “The study focused on relatively high-functioning stroke survivors, which raises questions about the applicability of the results to those with more severe impairments.” Mr. Glatt did not participate in the research.

“Additional studies are needed to confirm whether these findings are applicable to more diverse and severely affected populations and to assess the long-term sustainability of the benefits observed,” he said. “Also, the lack of significant improvements in other critical outcomes, such as mobility, suggests limitations in the broader application of HIIT for stroke rehabilitation.”

“While HIIT shows potential, it should be approached with caution,” Mr. Glatt continued. “It may benefit select patients, but replacing traditional exercise protocols with HIIT should not be done in all cases. More robust evidence and careful consideration of individual patient needs are essential.”

This study was funded by an operating grant from the Canadian Institutes of Health Research. Dr. Tang reported grants from the Canadian Institutes of Health Research, the Physiotherapy Foundation of Canada, and the Heart and Stroke Foundation of Canada. Mr. Glatt declared no relevant financial relationships.
 

A version of this article appeared on Medscape.com.

A single night of sleep deprivation had a significant impact on human blood serum, based on new data from an analysis of nearly 500 proteins. Compromised sleep has demonstrated negative effects on cardiovascular, immune, and neuronal systems, and previous studies have shown human serum proteome changes after a simulation of night shift work, wrote Alvhild Alette Bjørkum, MD, of Western Norway University of Applied Sciences, Bergen, and colleagues.

In a pilot study published in Sleep Advances, the researchers recruited eight healthy adult women aged 22-57 years with no history of neurologic or psychiatric problems to participate in a study of the effect of compromised sleep on protein profiles, with implications for effects on cells, tissues, and organ systems. Each of the participants served as their own controls, and blood samples were taken after 6 hours of sleep at night, and again after 6 hours of sleep deprivation the following night.

The researchers identified analyzed 494 proteins using mass spectrometry. Of these, 66 were differentially expressed after 6 hours of sleep deprivation. The top enriched biologic processes of these significantly changed proteins were protein activation cascade, platelet degranulation, blood coagulation, and hemostasis.

Further analysis using gene ontology showed changes in response to sleep deprivation in biologic process, molecular function, and immune system process categories, including specific associations related to wound healing, cholesterol transport, high-density lipoprotein particle receptor binding, and granulocyte chemotaxis.

The findings were limited by several factors including the small sample size, inclusion only of adult females, and the use of data from only 1 night of sleep deprivation, the researchers noted. However, the results support previous studies showing a negative impact of sleep deprivation on biologic functions, they said.

“Our study was able to reveal another set of human serum proteins that were altered by sleep deprivation and could connect similar biological processes to sleep deprivation that have been identified before with slightly different methods,” the researchers concluded. The study findings add to the knowledge base for the protein profiling of sleep deprivation, which may inform the development of tools to manage lack of sleep and mistimed sleep, particularly in shift workers.
 

Too Soon for Clinical Implications

“The adverse impact of poor sleep across many organ systems is gaining recognition, but the mechanisms underlying sleep-related pathology are not well understood,” Evan L. Brittain, MD, of Vanderbilt University, Nashville, Tennessee, said in an interview. “Studies like this begin to shed light on the mechanisms by which poor or reduced sleep affects specific bodily functions,” added Dr. Brittain, who was not involved in the study.

“The effects of other acute physiologic stressor such as exercise on the circulating proteome are well described. In that regard, it is not surprising that a brief episode of sleep deprivation would lead to detectable changes in the circulation,” Dr. Brittain said.

However, the specific changes reported in this study are difficult to interpret because of methodological and analytical concerns, particularly the small sample size, lack of an external validation cohort, and absence of appropriate statistical adjustments in the results, Dr. Brittain noted. These limitations prevent consideration of clinical implications without further study.

The study received no outside funding. Neither the researchers nor Dr. Brittain disclosed any conflicts of interest.

A version of this article first appeared on Medscape.com.

A single night of sleep deprivation had a significant impact on human blood serum, based on new data from an analysis of nearly 500 proteins. Compromised sleep has demonstrated negative effects on cardiovascular, immune, and neuronal systems, and previous studies have shown human serum proteome changes after a simulation of night shift work, wrote Alvhild Alette Bjørkum, MD, of Western Norway University of Applied Sciences, Bergen, and colleagues.

In a pilot study published in Sleep Advances, the researchers recruited eight healthy adult women aged 22-57 years with no history of neurologic or psychiatric problems to participate in a study of the effect of compromised sleep on protein profiles, with implications for effects on cells, tissues, and organ systems. Each of the participants served as their own controls, and blood samples were taken after 6 hours of sleep at night, and again after 6 hours of sleep deprivation the following night.

The researchers identified analyzed 494 proteins using mass spectrometry. Of these, 66 were differentially expressed after 6 hours of sleep deprivation. The top enriched biologic processes of these significantly changed proteins were protein activation cascade, platelet degranulation, blood coagulation, and hemostasis.

Further analysis using gene ontology showed changes in response to sleep deprivation in biologic process, molecular function, and immune system process categories, including specific associations related to wound healing, cholesterol transport, high-density lipoprotein particle receptor binding, and granulocyte chemotaxis.

The findings were limited by several factors including the small sample size, inclusion only of adult females, and the use of data from only 1 night of sleep deprivation, the researchers noted. However, the results support previous studies showing a negative impact of sleep deprivation on biologic functions, they said.

“Our study was able to reveal another set of human serum proteins that were altered by sleep deprivation and could connect similar biological processes to sleep deprivation that have been identified before with slightly different methods,” the researchers concluded. The study findings add to the knowledge base for the protein profiling of sleep deprivation, which may inform the development of tools to manage lack of sleep and mistimed sleep, particularly in shift workers.
 

Too Soon for Clinical Implications

“The adverse impact of poor sleep across many organ systems is gaining recognition, but the mechanisms underlying sleep-related pathology are not well understood,” Evan L. Brittain, MD, of Vanderbilt University, Nashville, Tennessee, said in an interview. “Studies like this begin to shed light on the mechanisms by which poor or reduced sleep affects specific bodily functions,” added Dr. Brittain, who was not involved in the study.

“The effects of other acute physiologic stressor such as exercise on the circulating proteome are well described. In that regard, it is not surprising that a brief episode of sleep deprivation would lead to detectable changes in the circulation,” Dr. Brittain said.

However, the specific changes reported in this study are difficult to interpret because of methodological and analytical concerns, particularly the small sample size, lack of an external validation cohort, and absence of appropriate statistical adjustments in the results, Dr. Brittain noted. These limitations prevent consideration of clinical implications without further study.

The study received no outside funding. Neither the researchers nor Dr. Brittain disclosed any conflicts of interest.

A version of this article first appeared on Medscape.com.

A single night of sleep deprivation had a significant impact on human blood serum, based on new data from an analysis of nearly 500 proteins. Compromised sleep has demonstrated negative effects on cardiovascular, immune, and neuronal systems, and previous studies have shown human serum proteome changes after a simulation of night shift work, wrote Alvhild Alette Bjørkum, MD, of Western Norway University of Applied Sciences, Bergen, and colleagues.

In a pilot study published in Sleep Advances, the researchers recruited eight healthy adult women aged 22-57 years with no history of neurologic or psychiatric problems to participate in a study of the effect of compromised sleep on protein profiles, with implications for effects on cells, tissues, and organ systems. Each of the participants served as their own controls, and blood samples were taken after 6 hours of sleep at night, and again after 6 hours of sleep deprivation the following night.

The researchers identified analyzed 494 proteins using mass spectrometry. Of these, 66 were differentially expressed after 6 hours of sleep deprivation. The top enriched biologic processes of these significantly changed proteins were protein activation cascade, platelet degranulation, blood coagulation, and hemostasis.

Further analysis using gene ontology showed changes in response to sleep deprivation in biologic process, molecular function, and immune system process categories, including specific associations related to wound healing, cholesterol transport, high-density lipoprotein particle receptor binding, and granulocyte chemotaxis.

The findings were limited by several factors including the small sample size, inclusion only of adult females, and the use of data from only 1 night of sleep deprivation, the researchers noted. However, the results support previous studies showing a negative impact of sleep deprivation on biologic functions, they said.

“Our study was able to reveal another set of human serum proteins that were altered by sleep deprivation and could connect similar biological processes to sleep deprivation that have been identified before with slightly different methods,” the researchers concluded. The study findings add to the knowledge base for the protein profiling of sleep deprivation, which may inform the development of tools to manage lack of sleep and mistimed sleep, particularly in shift workers.
 

Too Soon for Clinical Implications

“The adverse impact of poor sleep across many organ systems is gaining recognition, but the mechanisms underlying sleep-related pathology are not well understood,” Evan L. Brittain, MD, of Vanderbilt University, Nashville, Tennessee, said in an interview. “Studies like this begin to shed light on the mechanisms by which poor or reduced sleep affects specific bodily functions,” added Dr. Brittain, who was not involved in the study.

“The effects of other acute physiologic stressor such as exercise on the circulating proteome are well described. In that regard, it is not surprising that a brief episode of sleep deprivation would lead to detectable changes in the circulation,” Dr. Brittain said.

However, the specific changes reported in this study are difficult to interpret because of methodological and analytical concerns, particularly the small sample size, lack of an external validation cohort, and absence of appropriate statistical adjustments in the results, Dr. Brittain noted. These limitations prevent consideration of clinical implications without further study.

The study received no outside funding. Neither the researchers nor Dr. Brittain disclosed any conflicts of interest.

A version of this article first appeared on Medscape.com.