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Younger children can safely visit HSCT recipients (sometimes)
ORLANDO – For visits to hematopoietic stem cell transplant recipients, the kids are alright – assuming that they’re not sniffling, coughing, or running a fever, that is.
Since their blood and marrow transplant (BMT) unit began allowing child visits to patients in 2018, there have been no increases in either health care–associated infections or average length of stay, and patient satisfaction has markedly improved, reported Mylene R. de Vera, RN, and colleagues from the Greenbaum Cancer Center at the University of Maryland Medical Center in Baltimore.
Although her center had previously had a policy restricting child visitors out of concerns about transmission of infections, “we did a literature search and found that child visitor restriction is really neither evidence based nor recommended by any professional organization,” she said in an interview at the annual Transplantation & Cellular Therapy Meetings.
“At the same time, I also informally surveyed 10 hospitals and BMT centers nationally, and found that 70% of them allow child visitation,” she added at the meeting held by the American Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research.
An informal survey by this reporter found wide variations in practice. For example, Massachusetts General Hospital Cancer Center policy allows only children 12 and older to visit, but other hospitals have somewhat less stringent policies.
“Our policy is that for children under 12, they need to A) be screened, and B) need to get approval from the attending stem-cell physician,” said Reggie E. Duerst, MD, director of the Stem Cell Transplant Program at Ann & Robert H. Lurie Children’s Hospital of Chicago.
Dr. Duerst, who was not involved in the study, said in an interview seeking objective commentary that his center doesn’t especially encourage visits from younger children, on the understanding that, as every parent knows, the odds are higher that children under 12 may be carrying a respiratory virus, compared with adolescents.
He added that visits from children might be beneficial for patients who are more critically ill or at high risk for dying, but again, only if the child has been carefully screened.
At Memorial Sloan Kettering Cancer Center in New York, children are allowed to visit transplant recipients strictly on a case-by-case basis, and only after consultation with and approval from transplant unit staff, an MSKCC spokesman said.
In an interview, Ms. de Vera cited a 2016 study in the American Journal of Critical Care by Mini Jacob, RN, and colleagues from Emory University in Atlanta, who reported that, among family members visiting patients in an intensive care unit with a continuous visitation policy, open visitation was ranked as the second-most important need.
Beginning in 2018, Ms. de Vera and colleagues sought to promote a patient-centered policy by implementing guidelines that would allow safe visitation by children to BMT recipients.
The process included the aforementioned literature search, survey of other centers, and a staff survey, which showed that 70% of staff said they believed that allowing child visitation would support the goal of patient-centered care. The survey also showed, however, that 40% of staff thought that healthy children presented a higher risk of infection to immunocompromised patients, and 50% agreed with the statement that having children on the unit would be disruptive to care.
In August of 2019, all BMT unit staff members took part in an in-service training session designed “to align perception with evidence-based practice.” The training included detailed information and procedures for screening would-be child visitors for potential risks prior to allowing them on the unit.
The one-page screening tool asks about current or recent infections; coughing, sneezing, etc.; and other health-related issues, including whether the child is up to date on vaccinations.
The following month, the BMT unit implemented its new child visitation policy, and by the end of 2019 the unit had logged a total of 71 child visits.
As noted, there were no increases in either health care–associated infections or in the average length of stay, In fact, from April to September 2019 the average length of stay was 13 days, compared with 18 days during the 6-month period in 2018, before child visits were allowed.
In addition, a Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) patient-satisfaction survey showed that the scores on “taking preferences into account” increased to 92% during October through December 2018, compared with 33% from July through September of the same year.
Interestingly, patient-reported satisfaction also fell by half from January to March 2019, when child visits were suspended as part of hospital-wide restrictions during the influenza season.
A survey of staff conducted a year after the child visitation policy was implemented showed a relative 37% increase in reported awareness that the visits promote patient-centered care, a 35% drop in the reported belief that healthy child visitors pose higher risk of infection, and a more than 100% relative decline in the perception that child visitors would upset BMT unit routine.
“I just wanted to prove to my coworkers that child visitation really did not affect length of stay or health care–associated infections, and at the same time may have a positive effect on patient satisfaction,” Ms. de Vera said.
The study was internally supported. All persons interviewed reported no conflicts of interest to disclose.
SOURCE: de Vera MR et al. TCT 2020, Abstract 588.
ORLANDO – For visits to hematopoietic stem cell transplant recipients, the kids are alright – assuming that they’re not sniffling, coughing, or running a fever, that is.
Since their blood and marrow transplant (BMT) unit began allowing child visits to patients in 2018, there have been no increases in either health care–associated infections or average length of stay, and patient satisfaction has markedly improved, reported Mylene R. de Vera, RN, and colleagues from the Greenbaum Cancer Center at the University of Maryland Medical Center in Baltimore.
Although her center had previously had a policy restricting child visitors out of concerns about transmission of infections, “we did a literature search and found that child visitor restriction is really neither evidence based nor recommended by any professional organization,” she said in an interview at the annual Transplantation & Cellular Therapy Meetings.
“At the same time, I also informally surveyed 10 hospitals and BMT centers nationally, and found that 70% of them allow child visitation,” she added at the meeting held by the American Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research.
An informal survey by this reporter found wide variations in practice. For example, Massachusetts General Hospital Cancer Center policy allows only children 12 and older to visit, but other hospitals have somewhat less stringent policies.
“Our policy is that for children under 12, they need to A) be screened, and B) need to get approval from the attending stem-cell physician,” said Reggie E. Duerst, MD, director of the Stem Cell Transplant Program at Ann & Robert H. Lurie Children’s Hospital of Chicago.
Dr. Duerst, who was not involved in the study, said in an interview seeking objective commentary that his center doesn’t especially encourage visits from younger children, on the understanding that, as every parent knows, the odds are higher that children under 12 may be carrying a respiratory virus, compared with adolescents.
He added that visits from children might be beneficial for patients who are more critically ill or at high risk for dying, but again, only if the child has been carefully screened.
At Memorial Sloan Kettering Cancer Center in New York, children are allowed to visit transplant recipients strictly on a case-by-case basis, and only after consultation with and approval from transplant unit staff, an MSKCC spokesman said.
In an interview, Ms. de Vera cited a 2016 study in the American Journal of Critical Care by Mini Jacob, RN, and colleagues from Emory University in Atlanta, who reported that, among family members visiting patients in an intensive care unit with a continuous visitation policy, open visitation was ranked as the second-most important need.
Beginning in 2018, Ms. de Vera and colleagues sought to promote a patient-centered policy by implementing guidelines that would allow safe visitation by children to BMT recipients.
The process included the aforementioned literature search, survey of other centers, and a staff survey, which showed that 70% of staff said they believed that allowing child visitation would support the goal of patient-centered care. The survey also showed, however, that 40% of staff thought that healthy children presented a higher risk of infection to immunocompromised patients, and 50% agreed with the statement that having children on the unit would be disruptive to care.
In August of 2019, all BMT unit staff members took part in an in-service training session designed “to align perception with evidence-based practice.” The training included detailed information and procedures for screening would-be child visitors for potential risks prior to allowing them on the unit.
The one-page screening tool asks about current or recent infections; coughing, sneezing, etc.; and other health-related issues, including whether the child is up to date on vaccinations.
The following month, the BMT unit implemented its new child visitation policy, and by the end of 2019 the unit had logged a total of 71 child visits.
As noted, there were no increases in either health care–associated infections or in the average length of stay, In fact, from April to September 2019 the average length of stay was 13 days, compared with 18 days during the 6-month period in 2018, before child visits were allowed.
In addition, a Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) patient-satisfaction survey showed that the scores on “taking preferences into account” increased to 92% during October through December 2018, compared with 33% from July through September of the same year.
Interestingly, patient-reported satisfaction also fell by half from January to March 2019, when child visits were suspended as part of hospital-wide restrictions during the influenza season.
A survey of staff conducted a year after the child visitation policy was implemented showed a relative 37% increase in reported awareness that the visits promote patient-centered care, a 35% drop in the reported belief that healthy child visitors pose higher risk of infection, and a more than 100% relative decline in the perception that child visitors would upset BMT unit routine.
“I just wanted to prove to my coworkers that child visitation really did not affect length of stay or health care–associated infections, and at the same time may have a positive effect on patient satisfaction,” Ms. de Vera said.
The study was internally supported. All persons interviewed reported no conflicts of interest to disclose.
SOURCE: de Vera MR et al. TCT 2020, Abstract 588.
ORLANDO – For visits to hematopoietic stem cell transplant recipients, the kids are alright – assuming that they’re not sniffling, coughing, or running a fever, that is.
Since their blood and marrow transplant (BMT) unit began allowing child visits to patients in 2018, there have been no increases in either health care–associated infections or average length of stay, and patient satisfaction has markedly improved, reported Mylene R. de Vera, RN, and colleagues from the Greenbaum Cancer Center at the University of Maryland Medical Center in Baltimore.
Although her center had previously had a policy restricting child visitors out of concerns about transmission of infections, “we did a literature search and found that child visitor restriction is really neither evidence based nor recommended by any professional organization,” she said in an interview at the annual Transplantation & Cellular Therapy Meetings.
“At the same time, I also informally surveyed 10 hospitals and BMT centers nationally, and found that 70% of them allow child visitation,” she added at the meeting held by the American Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research.
An informal survey by this reporter found wide variations in practice. For example, Massachusetts General Hospital Cancer Center policy allows only children 12 and older to visit, but other hospitals have somewhat less stringent policies.
“Our policy is that for children under 12, they need to A) be screened, and B) need to get approval from the attending stem-cell physician,” said Reggie E. Duerst, MD, director of the Stem Cell Transplant Program at Ann & Robert H. Lurie Children’s Hospital of Chicago.
Dr. Duerst, who was not involved in the study, said in an interview seeking objective commentary that his center doesn’t especially encourage visits from younger children, on the understanding that, as every parent knows, the odds are higher that children under 12 may be carrying a respiratory virus, compared with adolescents.
He added that visits from children might be beneficial for patients who are more critically ill or at high risk for dying, but again, only if the child has been carefully screened.
At Memorial Sloan Kettering Cancer Center in New York, children are allowed to visit transplant recipients strictly on a case-by-case basis, and only after consultation with and approval from transplant unit staff, an MSKCC spokesman said.
In an interview, Ms. de Vera cited a 2016 study in the American Journal of Critical Care by Mini Jacob, RN, and colleagues from Emory University in Atlanta, who reported that, among family members visiting patients in an intensive care unit with a continuous visitation policy, open visitation was ranked as the second-most important need.
Beginning in 2018, Ms. de Vera and colleagues sought to promote a patient-centered policy by implementing guidelines that would allow safe visitation by children to BMT recipients.
The process included the aforementioned literature search, survey of other centers, and a staff survey, which showed that 70% of staff said they believed that allowing child visitation would support the goal of patient-centered care. The survey also showed, however, that 40% of staff thought that healthy children presented a higher risk of infection to immunocompromised patients, and 50% agreed with the statement that having children on the unit would be disruptive to care.
In August of 2019, all BMT unit staff members took part in an in-service training session designed “to align perception with evidence-based practice.” The training included detailed information and procedures for screening would-be child visitors for potential risks prior to allowing them on the unit.
The one-page screening tool asks about current or recent infections; coughing, sneezing, etc.; and other health-related issues, including whether the child is up to date on vaccinations.
The following month, the BMT unit implemented its new child visitation policy, and by the end of 2019 the unit had logged a total of 71 child visits.
As noted, there were no increases in either health care–associated infections or in the average length of stay, In fact, from April to September 2019 the average length of stay was 13 days, compared with 18 days during the 6-month period in 2018, before child visits were allowed.
In addition, a Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) patient-satisfaction survey showed that the scores on “taking preferences into account” increased to 92% during October through December 2018, compared with 33% from July through September of the same year.
Interestingly, patient-reported satisfaction also fell by half from January to March 2019, when child visits were suspended as part of hospital-wide restrictions during the influenza season.
A survey of staff conducted a year after the child visitation policy was implemented showed a relative 37% increase in reported awareness that the visits promote patient-centered care, a 35% drop in the reported belief that healthy child visitors pose higher risk of infection, and a more than 100% relative decline in the perception that child visitors would upset BMT unit routine.
“I just wanted to prove to my coworkers that child visitation really did not affect length of stay or health care–associated infections, and at the same time may have a positive effect on patient satisfaction,” Ms. de Vera said.
The study was internally supported. All persons interviewed reported no conflicts of interest to disclose.
SOURCE: de Vera MR et al. TCT 2020, Abstract 588.
REPORTING FROM TCT 2020
Under the influence
I don’t know how successful you have been at getting your adolescent patients to follow your suggestions, but I would guess that my batting average was in the low 100s. Even when I try stepping off my soapbox to involve the patient in a nonjudgmental dialogue, my successes pale in comparison to my failures.
Just looking at our national statistics for obesity, it’s pretty obvious that we are all doing a pretty rotten job of modifying our patients behaviors. You could point to a few encouraging numbers but they are few and far between. You could claim correctly that by the time a child reaches preschool, the die is already cast, throw up your arms, and not even raise the subject of diet with your overweight teenage patients.
A recent article in the journal Appetite hints at a group of strategies for molding patient behavior that so far have gotten very little attention from physicians (“Do perceived norms of social media users eating habits and preferences predict our own food consumption and BMI?” Appetite. 2020 Jan 18. doi: 10.1016/j.appet.2020.104611). Researchers at the department of psychology at Ashton University in Birmingham, England, surveyed more than 350 college-age students asking them about the dietary preference of their Facebook contacts and their own dietary habits. What the investigators found was that respondents who perceived their peers ate a healthy diet ate a healthier diet. Conversely, if the respondents thought their social media contacts ate junk food, they reported eating more of an unhealthy diet themselves.
In other words, it appears that, through social media, we have the potential to influence the eating habits of our patients’ peers. Before we get too excited, it should be pointed out that this study from England wasn’t of a long enough duration to demonstrate an effect on body mass index. And another study of 176 children recently published in Pediatrics found that while influencer marketing of unhealthy foods increased children’s immediate food intake, the equivalent marketing of healthy foods had no effect (“Social influencer marketing and children’s food intake: A randomized trial.” Pediatrics. 2019 Apr 1. doi: 10.1542/peds.2018-2554).
Not being terribly aware of the whos, whats, and wheres of influencers, I did a little bit of Internet searching at the Influencer Marketing hub and learned that influencers comes in all shapes and sizes, from “nanoinfluencers” who have acknowledged expertise and a very small Internet following numbering as few as a hundred to “megainfluencers” who have more than a million followers and might charge large entities a million dollars for a single post. The influencer’s content could appear as a blog, a YouTube video, a podcast, or simply a social media post.
The field of influencer marketing is new and growing exponentially. This initiative could come in the form of an office dedicated to Influencer Marketing created by the American Academy of Pediatrics. That group could search for megainfluencers who might be funded by the academy. But it also could develop a handbook for individual practitioners and groups to help them identify nano- and micro- (1,000-40,000 followers) influencers in their own practices.
You probably don’t ask your patients about their social media habits other than to caution them about time management. Maybe it’s time to dig a little deeper. You may find that you have a potent influencer hidden in your practice. She or he might just be willing to spread a good word or two for you.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].
I don’t know how successful you have been at getting your adolescent patients to follow your suggestions, but I would guess that my batting average was in the low 100s. Even when I try stepping off my soapbox to involve the patient in a nonjudgmental dialogue, my successes pale in comparison to my failures.
Just looking at our national statistics for obesity, it’s pretty obvious that we are all doing a pretty rotten job of modifying our patients behaviors. You could point to a few encouraging numbers but they are few and far between. You could claim correctly that by the time a child reaches preschool, the die is already cast, throw up your arms, and not even raise the subject of diet with your overweight teenage patients.
A recent article in the journal Appetite hints at a group of strategies for molding patient behavior that so far have gotten very little attention from physicians (“Do perceived norms of social media users eating habits and preferences predict our own food consumption and BMI?” Appetite. 2020 Jan 18. doi: 10.1016/j.appet.2020.104611). Researchers at the department of psychology at Ashton University in Birmingham, England, surveyed more than 350 college-age students asking them about the dietary preference of their Facebook contacts and their own dietary habits. What the investigators found was that respondents who perceived their peers ate a healthy diet ate a healthier diet. Conversely, if the respondents thought their social media contacts ate junk food, they reported eating more of an unhealthy diet themselves.
In other words, it appears that, through social media, we have the potential to influence the eating habits of our patients’ peers. Before we get too excited, it should be pointed out that this study from England wasn’t of a long enough duration to demonstrate an effect on body mass index. And another study of 176 children recently published in Pediatrics found that while influencer marketing of unhealthy foods increased children’s immediate food intake, the equivalent marketing of healthy foods had no effect (“Social influencer marketing and children’s food intake: A randomized trial.” Pediatrics. 2019 Apr 1. doi: 10.1542/peds.2018-2554).
Not being terribly aware of the whos, whats, and wheres of influencers, I did a little bit of Internet searching at the Influencer Marketing hub and learned that influencers comes in all shapes and sizes, from “nanoinfluencers” who have acknowledged expertise and a very small Internet following numbering as few as a hundred to “megainfluencers” who have more than a million followers and might charge large entities a million dollars for a single post. The influencer’s content could appear as a blog, a YouTube video, a podcast, or simply a social media post.
The field of influencer marketing is new and growing exponentially. This initiative could come in the form of an office dedicated to Influencer Marketing created by the American Academy of Pediatrics. That group could search for megainfluencers who might be funded by the academy. But it also could develop a handbook for individual practitioners and groups to help them identify nano- and micro- (1,000-40,000 followers) influencers in their own practices.
You probably don’t ask your patients about their social media habits other than to caution them about time management. Maybe it’s time to dig a little deeper. You may find that you have a potent influencer hidden in your practice. She or he might just be willing to spread a good word or two for you.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].
I don’t know how successful you have been at getting your adolescent patients to follow your suggestions, but I would guess that my batting average was in the low 100s. Even when I try stepping off my soapbox to involve the patient in a nonjudgmental dialogue, my successes pale in comparison to my failures.
Just looking at our national statistics for obesity, it’s pretty obvious that we are all doing a pretty rotten job of modifying our patients behaviors. You could point to a few encouraging numbers but they are few and far between. You could claim correctly that by the time a child reaches preschool, the die is already cast, throw up your arms, and not even raise the subject of diet with your overweight teenage patients.
A recent article in the journal Appetite hints at a group of strategies for molding patient behavior that so far have gotten very little attention from physicians (“Do perceived norms of social media users eating habits and preferences predict our own food consumption and BMI?” Appetite. 2020 Jan 18. doi: 10.1016/j.appet.2020.104611). Researchers at the department of psychology at Ashton University in Birmingham, England, surveyed more than 350 college-age students asking them about the dietary preference of their Facebook contacts and their own dietary habits. What the investigators found was that respondents who perceived their peers ate a healthy diet ate a healthier diet. Conversely, if the respondents thought their social media contacts ate junk food, they reported eating more of an unhealthy diet themselves.
In other words, it appears that, through social media, we have the potential to influence the eating habits of our patients’ peers. Before we get too excited, it should be pointed out that this study from England wasn’t of a long enough duration to demonstrate an effect on body mass index. And another study of 176 children recently published in Pediatrics found that while influencer marketing of unhealthy foods increased children’s immediate food intake, the equivalent marketing of healthy foods had no effect (“Social influencer marketing and children’s food intake: A randomized trial.” Pediatrics. 2019 Apr 1. doi: 10.1542/peds.2018-2554).
Not being terribly aware of the whos, whats, and wheres of influencers, I did a little bit of Internet searching at the Influencer Marketing hub and learned that influencers comes in all shapes and sizes, from “nanoinfluencers” who have acknowledged expertise and a very small Internet following numbering as few as a hundred to “megainfluencers” who have more than a million followers and might charge large entities a million dollars for a single post. The influencer’s content could appear as a blog, a YouTube video, a podcast, or simply a social media post.
The field of influencer marketing is new and growing exponentially. This initiative could come in the form of an office dedicated to Influencer Marketing created by the American Academy of Pediatrics. That group could search for megainfluencers who might be funded by the academy. But it also could develop a handbook for individual practitioners and groups to help them identify nano- and micro- (1,000-40,000 followers) influencers in their own practices.
You probably don’t ask your patients about their social media habits other than to caution them about time management. Maybe it’s time to dig a little deeper. You may find that you have a potent influencer hidden in your practice. She or he might just be willing to spread a good word or two for you.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].
Probably okay to skip SIBO breath test before starting rifaximin in IBS-D
MAUI, HAWAII – , according to IBS expert Lin Chang, MD, a professor and vice chief of the division of digestive diseases at the University of California, Los Angeles.
Rifaximin is an antibiotic that works in the intestines but is not absorbed by the body, and is approved for irritable bowel syndrome with diarrhea (IBS-D). It’s a second-line option for moderate illness when diet, OTC medications, and other easier options don’t do the trick. Endpoints were met by about 10% more patients on rifaximin than placebo in randomized trials, perhaps by resetting gut microbiota.
“It was a small therapeutic gain, but it was statistically significant,” Dr. Chang said at the Gastroenterology Updates, IBD, Liver Disease Conference. In her own practice, she said it sometimes helps even with bloating and abdominal distension, two of the most vexing problems in IBS-D.
However, symptoms come back after 3-6 months, and prices approach $2,000 for the 2-week IBS-D course. Insurance doesn’t always fully cover it, and the Food and Drug Administration has capped treatments at three.
That’s left providers wondering what to do when people ask for rifaximin after seeing it advertised and eager for some way to predict if it will work or not. Some clinicians have turned to a breath test for small-intestinal bacterial overgrowth (SIBO) before prescribing rifaximin because there is evidence that SIBO contributes to IBS-D, but others have not. It’s a contentious issue in IBS medicine, Dr. Chang said.
A recent open-label study funded by rifaximin maker Salix Pharmaceuticals made a case for testing. Among 93 IBS-D patients, 60% with a positive breath test at baseline had reduced pain, diarrhea, and other symptoms after 550 mg three times daily for 2 weeks, versus 26% with a negative test (Am J Gastroenterol. 2019 Dec;114[12]:1886-93).
Even so, when asked after her IBS presentation, Dr. Chang said she doesn’t breath test because it doesn’t make sense. Even with positive results, “you don’t really know if they have SIBO or not. Sometimes healthy controls have a positive test, and some people with IBS who end up responding to rifaximin have a negative breath test. It’s up to you, [but] it doesn’t correlate” with outcomes, she said.
Indeed, a letter in response to the study made a strong case that nine of the subjects should have been counted as having a negative, not positive, breath test, and if they had, the difference in outcome would have disappeared (Am J Gastroenterol. 2020 Mar 2. doi: 10.14309/ajg.0000000000000569).
Dr. Chang has opted for an empiric approach. “I just treat patients who meet the criteria used in the clinical trials,” such as moderate abdominal pain and bloating, she said (for example, N Engl J Med. 2011 Jan 6;364[1]:22-32).
And she doesn’t push if people can’t afford rifaximin. Some turn to other antibiotics, like metronidazole (Flagyl) or ciprofloxacin (Cipro), which are absorbed in the GI tract, but Dr. Chang said symptoms will recur after a few months regardless of the antibiotic. “You don’t want all these young women to be given antibiotics over and over again. Their symptoms are just going to come back, and you are going to have to learn how to treat them anyway,” she said.
Instead, she moves on to other options when patients are at the point where they need prescription pharmaceuticals. One of her top choices is the tricyclic antidepressant amitriptyline. It helps with the pain; the anticholinergic effects counter the diarrhea; and the sedative effects help patients who have a hard time sleeping. Sleep problems make IBS-D symptoms worse, Dr. Chang said.
MAUI, HAWAII – , according to IBS expert Lin Chang, MD, a professor and vice chief of the division of digestive diseases at the University of California, Los Angeles.
Rifaximin is an antibiotic that works in the intestines but is not absorbed by the body, and is approved for irritable bowel syndrome with diarrhea (IBS-D). It’s a second-line option for moderate illness when diet, OTC medications, and other easier options don’t do the trick. Endpoints were met by about 10% more patients on rifaximin than placebo in randomized trials, perhaps by resetting gut microbiota.
“It was a small therapeutic gain, but it was statistically significant,” Dr. Chang said at the Gastroenterology Updates, IBD, Liver Disease Conference. In her own practice, she said it sometimes helps even with bloating and abdominal distension, two of the most vexing problems in IBS-D.
However, symptoms come back after 3-6 months, and prices approach $2,000 for the 2-week IBS-D course. Insurance doesn’t always fully cover it, and the Food and Drug Administration has capped treatments at three.
That’s left providers wondering what to do when people ask for rifaximin after seeing it advertised and eager for some way to predict if it will work or not. Some clinicians have turned to a breath test for small-intestinal bacterial overgrowth (SIBO) before prescribing rifaximin because there is evidence that SIBO contributes to IBS-D, but others have not. It’s a contentious issue in IBS medicine, Dr. Chang said.
A recent open-label study funded by rifaximin maker Salix Pharmaceuticals made a case for testing. Among 93 IBS-D patients, 60% with a positive breath test at baseline had reduced pain, diarrhea, and other symptoms after 550 mg three times daily for 2 weeks, versus 26% with a negative test (Am J Gastroenterol. 2019 Dec;114[12]:1886-93).
Even so, when asked after her IBS presentation, Dr. Chang said she doesn’t breath test because it doesn’t make sense. Even with positive results, “you don’t really know if they have SIBO or not. Sometimes healthy controls have a positive test, and some people with IBS who end up responding to rifaximin have a negative breath test. It’s up to you, [but] it doesn’t correlate” with outcomes, she said.
Indeed, a letter in response to the study made a strong case that nine of the subjects should have been counted as having a negative, not positive, breath test, and if they had, the difference in outcome would have disappeared (Am J Gastroenterol. 2020 Mar 2. doi: 10.14309/ajg.0000000000000569).
Dr. Chang has opted for an empiric approach. “I just treat patients who meet the criteria used in the clinical trials,” such as moderate abdominal pain and bloating, she said (for example, N Engl J Med. 2011 Jan 6;364[1]:22-32).
And she doesn’t push if people can’t afford rifaximin. Some turn to other antibiotics, like metronidazole (Flagyl) or ciprofloxacin (Cipro), which are absorbed in the GI tract, but Dr. Chang said symptoms will recur after a few months regardless of the antibiotic. “You don’t want all these young women to be given antibiotics over and over again. Their symptoms are just going to come back, and you are going to have to learn how to treat them anyway,” she said.
Instead, she moves on to other options when patients are at the point where they need prescription pharmaceuticals. One of her top choices is the tricyclic antidepressant amitriptyline. It helps with the pain; the anticholinergic effects counter the diarrhea; and the sedative effects help patients who have a hard time sleeping. Sleep problems make IBS-D symptoms worse, Dr. Chang said.
MAUI, HAWAII – , according to IBS expert Lin Chang, MD, a professor and vice chief of the division of digestive diseases at the University of California, Los Angeles.
Rifaximin is an antibiotic that works in the intestines but is not absorbed by the body, and is approved for irritable bowel syndrome with diarrhea (IBS-D). It’s a second-line option for moderate illness when diet, OTC medications, and other easier options don’t do the trick. Endpoints were met by about 10% more patients on rifaximin than placebo in randomized trials, perhaps by resetting gut microbiota.
“It was a small therapeutic gain, but it was statistically significant,” Dr. Chang said at the Gastroenterology Updates, IBD, Liver Disease Conference. In her own practice, she said it sometimes helps even with bloating and abdominal distension, two of the most vexing problems in IBS-D.
However, symptoms come back after 3-6 months, and prices approach $2,000 for the 2-week IBS-D course. Insurance doesn’t always fully cover it, and the Food and Drug Administration has capped treatments at three.
That’s left providers wondering what to do when people ask for rifaximin after seeing it advertised and eager for some way to predict if it will work or not. Some clinicians have turned to a breath test for small-intestinal bacterial overgrowth (SIBO) before prescribing rifaximin because there is evidence that SIBO contributes to IBS-D, but others have not. It’s a contentious issue in IBS medicine, Dr. Chang said.
A recent open-label study funded by rifaximin maker Salix Pharmaceuticals made a case for testing. Among 93 IBS-D patients, 60% with a positive breath test at baseline had reduced pain, diarrhea, and other symptoms after 550 mg three times daily for 2 weeks, versus 26% with a negative test (Am J Gastroenterol. 2019 Dec;114[12]:1886-93).
Even so, when asked after her IBS presentation, Dr. Chang said she doesn’t breath test because it doesn’t make sense. Even with positive results, “you don’t really know if they have SIBO or not. Sometimes healthy controls have a positive test, and some people with IBS who end up responding to rifaximin have a negative breath test. It’s up to you, [but] it doesn’t correlate” with outcomes, she said.
Indeed, a letter in response to the study made a strong case that nine of the subjects should have been counted as having a negative, not positive, breath test, and if they had, the difference in outcome would have disappeared (Am J Gastroenterol. 2020 Mar 2. doi: 10.14309/ajg.0000000000000569).
Dr. Chang has opted for an empiric approach. “I just treat patients who meet the criteria used in the clinical trials,” such as moderate abdominal pain and bloating, she said (for example, N Engl J Med. 2011 Jan 6;364[1]:22-32).
And she doesn’t push if people can’t afford rifaximin. Some turn to other antibiotics, like metronidazole (Flagyl) or ciprofloxacin (Cipro), which are absorbed in the GI tract, but Dr. Chang said symptoms will recur after a few months regardless of the antibiotic. “You don’t want all these young women to be given antibiotics over and over again. Their symptoms are just going to come back, and you are going to have to learn how to treat them anyway,” she said.
Instead, she moves on to other options when patients are at the point where they need prescription pharmaceuticals. One of her top choices is the tricyclic antidepressant amitriptyline. It helps with the pain; the anticholinergic effects counter the diarrhea; and the sedative effects help patients who have a hard time sleeping. Sleep problems make IBS-D symptoms worse, Dr. Chang said.
REPORTING FROM GUILD 2020
FDA cancels or postpones meetings amid COVID-19 concerns
Officials at the Food and Drug Administration’s Center for Drug Evaluation and Research are taking the precautionary step of canceling or postponing advisory committee meetings and limiting staff travel in an effort to help curb the spread of the COVID-19.
“The outbreak of respiratory illness caused by a novel coronavirus, COVID-19, that started in China is spreading to other countries, including the United States,” CDER Director Janet Woodcock, MD, said in a memo to CDER staff. “As a precaution, FDA is canceling foreign official agency travel and limiting domestic travel to mission critical only, effective immediately and through April.”
Additionally, the memo notes that “CDER-organized external meetings, conferences, and workshops will be postponed or canceled from March 10 through April.”
“To mitigate the impact on our work, I encourage you to hold meetings with external stakeholders through teleconference, when possible,” she wrote.
Thus far, only a few CDER events on the FDA’s meeting webpage are listed as being canceled or postponed. Some of the affected meetings include a March 10 public meeting on patient-focused drug development for stimulant-use disorder, a March 11 meeting of the Nonprescription Drug Advisory Committee, and a March 30 public meeting on patient-focused drug development for vitiligo, all of which are postponed until further notice. The Center for Biologics Evaluation and Research also has postponed until further notice its U.S.–Japan Cellular and Gene Therapy Conference, originally scheduled for March 12.
Dr. Woodcock also noted in the memo that in relation to inspections, “we plan to use technology and established agreements with our foreign counterparts to minimize disruptions to the drug supply chain and to applications under review, so that Americans can continue to get their medications.”
Officials at the Food and Drug Administration’s Center for Drug Evaluation and Research are taking the precautionary step of canceling or postponing advisory committee meetings and limiting staff travel in an effort to help curb the spread of the COVID-19.
“The outbreak of respiratory illness caused by a novel coronavirus, COVID-19, that started in China is spreading to other countries, including the United States,” CDER Director Janet Woodcock, MD, said in a memo to CDER staff. “As a precaution, FDA is canceling foreign official agency travel and limiting domestic travel to mission critical only, effective immediately and through April.”
Additionally, the memo notes that “CDER-organized external meetings, conferences, and workshops will be postponed or canceled from March 10 through April.”
“To mitigate the impact on our work, I encourage you to hold meetings with external stakeholders through teleconference, when possible,” she wrote.
Thus far, only a few CDER events on the FDA’s meeting webpage are listed as being canceled or postponed. Some of the affected meetings include a March 10 public meeting on patient-focused drug development for stimulant-use disorder, a March 11 meeting of the Nonprescription Drug Advisory Committee, and a March 30 public meeting on patient-focused drug development for vitiligo, all of which are postponed until further notice. The Center for Biologics Evaluation and Research also has postponed until further notice its U.S.–Japan Cellular and Gene Therapy Conference, originally scheduled for March 12.
Dr. Woodcock also noted in the memo that in relation to inspections, “we plan to use technology and established agreements with our foreign counterparts to minimize disruptions to the drug supply chain and to applications under review, so that Americans can continue to get their medications.”
Officials at the Food and Drug Administration’s Center for Drug Evaluation and Research are taking the precautionary step of canceling or postponing advisory committee meetings and limiting staff travel in an effort to help curb the spread of the COVID-19.
“The outbreak of respiratory illness caused by a novel coronavirus, COVID-19, that started in China is spreading to other countries, including the United States,” CDER Director Janet Woodcock, MD, said in a memo to CDER staff. “As a precaution, FDA is canceling foreign official agency travel and limiting domestic travel to mission critical only, effective immediately and through April.”
Additionally, the memo notes that “CDER-organized external meetings, conferences, and workshops will be postponed or canceled from March 10 through April.”
“To mitigate the impact on our work, I encourage you to hold meetings with external stakeholders through teleconference, when possible,” she wrote.
Thus far, only a few CDER events on the FDA’s meeting webpage are listed as being canceled or postponed. Some of the affected meetings include a March 10 public meeting on patient-focused drug development for stimulant-use disorder, a March 11 meeting of the Nonprescription Drug Advisory Committee, and a March 30 public meeting on patient-focused drug development for vitiligo, all of which are postponed until further notice. The Center for Biologics Evaluation and Research also has postponed until further notice its U.S.–Japan Cellular and Gene Therapy Conference, originally scheduled for March 12.
Dr. Woodcock also noted in the memo that in relation to inspections, “we plan to use technology and established agreements with our foreign counterparts to minimize disruptions to the drug supply chain and to applications under review, so that Americans can continue to get their medications.”
Botox: A new option for endometriosis pain?
NATIONAL HARBOR, MD. – Botulinum toxin injection into the vagina appears to relieve pain associated with endometriosis by relaxing the pelvic floor muscles, new research suggests.
In a randomized study, women with surgically diagnosed endometriosis who had chronic pelvic pain despite optimal surgical and hormonal treatment had less pain after injection vs their counterparts who received placebo.
This result suggests pelvic floor spasm may be an important factor in endometriosis-associated pelvic pain, the investigators note.
“Botulinum toxin injection offers an alternative approach for women with pelvic pain,” lead author Barbara Illowsky Karp, MD, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Md., told Medscape Medical News.
“We focused on endometriosis, but there are reasons to think it may be effective for pelvic pain from other causes if there is spasm of the muscle,” said Karp, a neurologist who has used botulinum toxin therapeutically since it was first developed in the 1980s.
She noted that it is unknown whether the toxin will work in women who do not have actual spasm, “but the effect on spasm is not the sole effect of toxin,” as demonstrated by its use in other pain conditions.
“It seems to have a direct effect on the pain pathways in the nervous system as well,” Karp added.
The study findings were presented here at the American Academy of Pain Medicine (AAPM) 2020 Annual Meeting.
Less pain
The investigators randomly assigned 29 women between the ages of 18 and 50 years to receive injections with 100 U onabotulinumtoxinA (n = 15) or saline placebo (n = 14).
All of the women had endometriosis with chronic pelvic pain lasting at least 3 months (mean time, 6 years) and confirmed pelvic floor spasm as a main pain generator.
One month after treatment, participants were asked if they had improvement in their pain.
“Our primary outcome was just simply asking the women if they felt better or not, because we were blinded as to what treatment they received. One month is typically when the toxin reaches its maximal effect,” Karp said.
At 1 month, 11 women in the placebo group reported that they had no benefit, compared with only 4 women in the botulinum toxin group (P = .027).
The botulinum toxin group reported a greater degree of benefit compared with the placebo group (P = .030) and greater percent of improvement (P = .034).
Neither group reported substantial changes in pain rating on the visual analog scale. However, a definite pain score is often difficult to measure in women with chronic pelvic pain, coinvestigator Pamela Stratton, MD, a gynecologist in Bethesda, said.
“Some women report their pain as a 2, some as an 8. Also, women may have a lot of pain one day and not have that much pain another day, and how do you measure that? This is why we have not focused solely on the pain score but instead wanted the women to tell us if they were improved or not,” Stratton told Medscape Medical News.
Disability worsened considerably in the placebo group, but remained consistent in the botulinum toxin group. Five patients in the botulinum toxin group were able to reduce pain medication compared with one patient in the placebo group.
“Compelling” findings but early days
Commenting on the findings for Medscape Medical News, Ann E. Hansen, MD, Chronic Pain Wellness Center, Phoenix VA Health Care System, Arizona, noted that this “preliminary study” showed some benefit for a complex and challenging-to-treat syndrome.
“Injection of botulinum toxin prevents local muscle contraction, thus effectively relieving a variety of neuromuscular conditions such as torticollis; spasticity; pain syndromes such as headache and migraine; and some neurologic disorders, for instance, overactive bladder,” said Hansen, who was not involved with the research.
“Using botulinum toxin injection to target pelvic floor muscle spasm, a known pain generator in women suffering from chronic pelvic pain, makes sense. Future studies will be helpful in elucidating optimal treatment protocols for this debilitating condition,” she added.
Also commenting for Medscape Medical News, Kathryn T. Hall, PhD, MPH, Brigham & Women’s Hospital, Boston, Massachusetts, called the results “quite compelling” although, “it’s still early days.”
“It remains to be seen if the treatment effect will endure or if side effects will emerge. Hopefully all will go well,” Hall said.
The study was funded by an unrestricted grant from the National Institutes of Health. Allergan provided the botulinum toxin that was used in the study. Karp, Stratton, Hansen, and Hall have disclosed no relevant financial relationships.
This article first appeared on Medscape.com.
NATIONAL HARBOR, MD. – Botulinum toxin injection into the vagina appears to relieve pain associated with endometriosis by relaxing the pelvic floor muscles, new research suggests.
In a randomized study, women with surgically diagnosed endometriosis who had chronic pelvic pain despite optimal surgical and hormonal treatment had less pain after injection vs their counterparts who received placebo.
This result suggests pelvic floor spasm may be an important factor in endometriosis-associated pelvic pain, the investigators note.
“Botulinum toxin injection offers an alternative approach for women with pelvic pain,” lead author Barbara Illowsky Karp, MD, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Md., told Medscape Medical News.
“We focused on endometriosis, but there are reasons to think it may be effective for pelvic pain from other causes if there is spasm of the muscle,” said Karp, a neurologist who has used botulinum toxin therapeutically since it was first developed in the 1980s.
She noted that it is unknown whether the toxin will work in women who do not have actual spasm, “but the effect on spasm is not the sole effect of toxin,” as demonstrated by its use in other pain conditions.
“It seems to have a direct effect on the pain pathways in the nervous system as well,” Karp added.
The study findings were presented here at the American Academy of Pain Medicine (AAPM) 2020 Annual Meeting.
Less pain
The investigators randomly assigned 29 women between the ages of 18 and 50 years to receive injections with 100 U onabotulinumtoxinA (n = 15) or saline placebo (n = 14).
All of the women had endometriosis with chronic pelvic pain lasting at least 3 months (mean time, 6 years) and confirmed pelvic floor spasm as a main pain generator.
One month after treatment, participants were asked if they had improvement in their pain.
“Our primary outcome was just simply asking the women if they felt better or not, because we were blinded as to what treatment they received. One month is typically when the toxin reaches its maximal effect,” Karp said.
At 1 month, 11 women in the placebo group reported that they had no benefit, compared with only 4 women in the botulinum toxin group (P = .027).
The botulinum toxin group reported a greater degree of benefit compared with the placebo group (P = .030) and greater percent of improvement (P = .034).
Neither group reported substantial changes in pain rating on the visual analog scale. However, a definite pain score is often difficult to measure in women with chronic pelvic pain, coinvestigator Pamela Stratton, MD, a gynecologist in Bethesda, said.
“Some women report their pain as a 2, some as an 8. Also, women may have a lot of pain one day and not have that much pain another day, and how do you measure that? This is why we have not focused solely on the pain score but instead wanted the women to tell us if they were improved or not,” Stratton told Medscape Medical News.
Disability worsened considerably in the placebo group, but remained consistent in the botulinum toxin group. Five patients in the botulinum toxin group were able to reduce pain medication compared with one patient in the placebo group.
“Compelling” findings but early days
Commenting on the findings for Medscape Medical News, Ann E. Hansen, MD, Chronic Pain Wellness Center, Phoenix VA Health Care System, Arizona, noted that this “preliminary study” showed some benefit for a complex and challenging-to-treat syndrome.
“Injection of botulinum toxin prevents local muscle contraction, thus effectively relieving a variety of neuromuscular conditions such as torticollis; spasticity; pain syndromes such as headache and migraine; and some neurologic disorders, for instance, overactive bladder,” said Hansen, who was not involved with the research.
“Using botulinum toxin injection to target pelvic floor muscle spasm, a known pain generator in women suffering from chronic pelvic pain, makes sense. Future studies will be helpful in elucidating optimal treatment protocols for this debilitating condition,” she added.
Also commenting for Medscape Medical News, Kathryn T. Hall, PhD, MPH, Brigham & Women’s Hospital, Boston, Massachusetts, called the results “quite compelling” although, “it’s still early days.”
“It remains to be seen if the treatment effect will endure or if side effects will emerge. Hopefully all will go well,” Hall said.
The study was funded by an unrestricted grant from the National Institutes of Health. Allergan provided the botulinum toxin that was used in the study. Karp, Stratton, Hansen, and Hall have disclosed no relevant financial relationships.
This article first appeared on Medscape.com.
NATIONAL HARBOR, MD. – Botulinum toxin injection into the vagina appears to relieve pain associated with endometriosis by relaxing the pelvic floor muscles, new research suggests.
In a randomized study, women with surgically diagnosed endometriosis who had chronic pelvic pain despite optimal surgical and hormonal treatment had less pain after injection vs their counterparts who received placebo.
This result suggests pelvic floor spasm may be an important factor in endometriosis-associated pelvic pain, the investigators note.
“Botulinum toxin injection offers an alternative approach for women with pelvic pain,” lead author Barbara Illowsky Karp, MD, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Md., told Medscape Medical News.
“We focused on endometriosis, but there are reasons to think it may be effective for pelvic pain from other causes if there is spasm of the muscle,” said Karp, a neurologist who has used botulinum toxin therapeutically since it was first developed in the 1980s.
She noted that it is unknown whether the toxin will work in women who do not have actual spasm, “but the effect on spasm is not the sole effect of toxin,” as demonstrated by its use in other pain conditions.
“It seems to have a direct effect on the pain pathways in the nervous system as well,” Karp added.
The study findings were presented here at the American Academy of Pain Medicine (AAPM) 2020 Annual Meeting.
Less pain
The investigators randomly assigned 29 women between the ages of 18 and 50 years to receive injections with 100 U onabotulinumtoxinA (n = 15) or saline placebo (n = 14).
All of the women had endometriosis with chronic pelvic pain lasting at least 3 months (mean time, 6 years) and confirmed pelvic floor spasm as a main pain generator.
One month after treatment, participants were asked if they had improvement in their pain.
“Our primary outcome was just simply asking the women if they felt better or not, because we were blinded as to what treatment they received. One month is typically when the toxin reaches its maximal effect,” Karp said.
At 1 month, 11 women in the placebo group reported that they had no benefit, compared with only 4 women in the botulinum toxin group (P = .027).
The botulinum toxin group reported a greater degree of benefit compared with the placebo group (P = .030) and greater percent of improvement (P = .034).
Neither group reported substantial changes in pain rating on the visual analog scale. However, a definite pain score is often difficult to measure in women with chronic pelvic pain, coinvestigator Pamela Stratton, MD, a gynecologist in Bethesda, said.
“Some women report their pain as a 2, some as an 8. Also, women may have a lot of pain one day and not have that much pain another day, and how do you measure that? This is why we have not focused solely on the pain score but instead wanted the women to tell us if they were improved or not,” Stratton told Medscape Medical News.
Disability worsened considerably in the placebo group, but remained consistent in the botulinum toxin group. Five patients in the botulinum toxin group were able to reduce pain medication compared with one patient in the placebo group.
“Compelling” findings but early days
Commenting on the findings for Medscape Medical News, Ann E. Hansen, MD, Chronic Pain Wellness Center, Phoenix VA Health Care System, Arizona, noted that this “preliminary study” showed some benefit for a complex and challenging-to-treat syndrome.
“Injection of botulinum toxin prevents local muscle contraction, thus effectively relieving a variety of neuromuscular conditions such as torticollis; spasticity; pain syndromes such as headache and migraine; and some neurologic disorders, for instance, overactive bladder,” said Hansen, who was not involved with the research.
“Using botulinum toxin injection to target pelvic floor muscle spasm, a known pain generator in women suffering from chronic pelvic pain, makes sense. Future studies will be helpful in elucidating optimal treatment protocols for this debilitating condition,” she added.
Also commenting for Medscape Medical News, Kathryn T. Hall, PhD, MPH, Brigham & Women’s Hospital, Boston, Massachusetts, called the results “quite compelling” although, “it’s still early days.”
“It remains to be seen if the treatment effect will endure or if side effects will emerge. Hopefully all will go well,” Hall said.
The study was funded by an unrestricted grant from the National Institutes of Health. Allergan provided the botulinum toxin that was used in the study. Karp, Stratton, Hansen, and Hall have disclosed no relevant financial relationships.
This article first appeared on Medscape.com.
Age does not appear to affect efficacy of siponimod in secondary progressive MS
WEST PALM BEACH, FLA. – (MS), according to data presented at the meeting held by the Americas Committee for Treatment and Research in Multiple Sclerosis. Siponimod’s tolerability also does not vary with age, the researchers said.
Although many effective treatments for relapsing-remitting MS are available, patients still have a high risk of developing secondary progressive MS. Increasing age is associated with disability accumulation, regardless of disease duration. This accumulation of disability could affect treatment outcomes.
Siponimod is an approved treatment for adults with relapsing forms of MS, including clinically isolated syndrome and active secondary progressive MS. Compared with placebo, siponimod significantly reduced the risk of confirmed disability progression in the phase 3 EXPAND trial.
Efficacy was similar between age groups
Le Hua, MD, a neurologist at the Cleveland Clinic Lou Ruvo Center for Brain Health in Las Vegas, and colleagues sought to evaluate the safety and efficacy of siponimod by age in patients with active secondary progressive MS who participated in the EXPAND study. The investigators defined active disease as a relapse in the 2 years before screening or one or more T1 gadolinium–enhancing lesions at baseline. Time to 3- and 6-month confirmed disability progression, defined using Expanded Disability Status Scale (EDSS) scores, were the efficacy endpoints of this analysis. Dr. Hua and colleagues also examined adverse events, serious adverse events, and adverse events leading to treatment discontinuation.
Of the 1,651 patients included in EXPAND, 779 had active secondary progressive MS. Dr. Hua and colleagues categorized this group as younger than 45 years (306 patients) or 45 years or older (473 patients). In the overall EXPAND population, siponimod reduced the risk of 3-month confirmed disability progression by 21% and the risk of 6-month confirmed disability progression by 26%, compared with placebo. Among patients younger than 45 years, siponimod reduced the risks of 3- and 6-month confirmed disability progression by 32% and 40%, respectively. Among patients aged 45 years or older, siponimod reduced the risks of 3- and 6-month confirmed disability progression by 31% and 33%, respectively.
Siponimod’s safety profile was similar between age groups. Among participants younger than 45 years, the rate of any adverse event was 82.6% for siponimod and 82.8% for placebo. In patients age 45 years or older, the rate of any adverse event was 89.8% for siponimod and 75.9% for placebo. The rate of serious adverse events was lower for siponimod than for placebo in both age groups. Among patients younger than 45 years, the rate was 12.7% for siponimod and 15.1% for placebo. Among patients age 45 years or older, the rate was 18.2% for siponimod and 19.4% for placebo. The rate of adverse events leading to discontinuation, however, was slightly higher among older patients. For younger patients, the rate was 3.3% for siponimod and 4.3% for placebo. For older patients, the rate was 7.6% for siponimod and 7.1% for placebo.
Real-world data will provide clearer answers
Rates of adverse events, serious adverse events, and adverse events leading to discontinuation were all higher in patients aged 45 or older. “This was not a surprising finding,” said Dr. Hua. “The differences appeared small, and the study was not powered to detect if these differences were significant. ... In general, older patients are more likely to have more cardiac events and infections than younger patients.”
Few studies have analyzed the efficacy and safety of MS therapy by age, said Dr. Hua. In 2015, Matell et al. evaluated the effectiveness of natalizumab in patients over age 50 years. In that study, a greater number of older patients, compared with younger patients, stopped treatment because of lack of effectiveness. Studies of other disease-modifying therapies (DMTs) have included subgroup analyses based on age, “but subgroup analyses are limited in the ability to make any strong conclusions regarding significant differences in safety,” said Dr. Hua. “A recent meta-analysis of published clinical trials of all DMTs performed by Weideman et al. indicated that the efficacy of DMTs appears to wane after age 53 years. However, there are limitations in interpreting this data, as they weren’t able to evaluate the raw data. And in most clinical trials, the age cutoff is usually 55 years, which limits generalizations. Real-world data analyses are needed to truly understand efficacy and safety of DMT in older patients.”
Novartis funded the study. Dr. Hua received fees from Novartis, Biogen, Celgene, EMD Serono, Genentech, and Genzyme.
SOURCE: Hua L et al. ACTRIMS FORUM 2020, Abstract P029.
WEST PALM BEACH, FLA. – (MS), according to data presented at the meeting held by the Americas Committee for Treatment and Research in Multiple Sclerosis. Siponimod’s tolerability also does not vary with age, the researchers said.
Although many effective treatments for relapsing-remitting MS are available, patients still have a high risk of developing secondary progressive MS. Increasing age is associated with disability accumulation, regardless of disease duration. This accumulation of disability could affect treatment outcomes.
Siponimod is an approved treatment for adults with relapsing forms of MS, including clinically isolated syndrome and active secondary progressive MS. Compared with placebo, siponimod significantly reduced the risk of confirmed disability progression in the phase 3 EXPAND trial.
Efficacy was similar between age groups
Le Hua, MD, a neurologist at the Cleveland Clinic Lou Ruvo Center for Brain Health in Las Vegas, and colleagues sought to evaluate the safety and efficacy of siponimod by age in patients with active secondary progressive MS who participated in the EXPAND study. The investigators defined active disease as a relapse in the 2 years before screening or one or more T1 gadolinium–enhancing lesions at baseline. Time to 3- and 6-month confirmed disability progression, defined using Expanded Disability Status Scale (EDSS) scores, were the efficacy endpoints of this analysis. Dr. Hua and colleagues also examined adverse events, serious adverse events, and adverse events leading to treatment discontinuation.
Of the 1,651 patients included in EXPAND, 779 had active secondary progressive MS. Dr. Hua and colleagues categorized this group as younger than 45 years (306 patients) or 45 years or older (473 patients). In the overall EXPAND population, siponimod reduced the risk of 3-month confirmed disability progression by 21% and the risk of 6-month confirmed disability progression by 26%, compared with placebo. Among patients younger than 45 years, siponimod reduced the risks of 3- and 6-month confirmed disability progression by 32% and 40%, respectively. Among patients aged 45 years or older, siponimod reduced the risks of 3- and 6-month confirmed disability progression by 31% and 33%, respectively.
Siponimod’s safety profile was similar between age groups. Among participants younger than 45 years, the rate of any adverse event was 82.6% for siponimod and 82.8% for placebo. In patients age 45 years or older, the rate of any adverse event was 89.8% for siponimod and 75.9% for placebo. The rate of serious adverse events was lower for siponimod than for placebo in both age groups. Among patients younger than 45 years, the rate was 12.7% for siponimod and 15.1% for placebo. Among patients age 45 years or older, the rate was 18.2% for siponimod and 19.4% for placebo. The rate of adverse events leading to discontinuation, however, was slightly higher among older patients. For younger patients, the rate was 3.3% for siponimod and 4.3% for placebo. For older patients, the rate was 7.6% for siponimod and 7.1% for placebo.
Real-world data will provide clearer answers
Rates of adverse events, serious adverse events, and adverse events leading to discontinuation were all higher in patients aged 45 or older. “This was not a surprising finding,” said Dr. Hua. “The differences appeared small, and the study was not powered to detect if these differences were significant. ... In general, older patients are more likely to have more cardiac events and infections than younger patients.”
Few studies have analyzed the efficacy and safety of MS therapy by age, said Dr. Hua. In 2015, Matell et al. evaluated the effectiveness of natalizumab in patients over age 50 years. In that study, a greater number of older patients, compared with younger patients, stopped treatment because of lack of effectiveness. Studies of other disease-modifying therapies (DMTs) have included subgroup analyses based on age, “but subgroup analyses are limited in the ability to make any strong conclusions regarding significant differences in safety,” said Dr. Hua. “A recent meta-analysis of published clinical trials of all DMTs performed by Weideman et al. indicated that the efficacy of DMTs appears to wane after age 53 years. However, there are limitations in interpreting this data, as they weren’t able to evaluate the raw data. And in most clinical trials, the age cutoff is usually 55 years, which limits generalizations. Real-world data analyses are needed to truly understand efficacy and safety of DMT in older patients.”
Novartis funded the study. Dr. Hua received fees from Novartis, Biogen, Celgene, EMD Serono, Genentech, and Genzyme.
SOURCE: Hua L et al. ACTRIMS FORUM 2020, Abstract P029.
WEST PALM BEACH, FLA. – (MS), according to data presented at the meeting held by the Americas Committee for Treatment and Research in Multiple Sclerosis. Siponimod’s tolerability also does not vary with age, the researchers said.
Although many effective treatments for relapsing-remitting MS are available, patients still have a high risk of developing secondary progressive MS. Increasing age is associated with disability accumulation, regardless of disease duration. This accumulation of disability could affect treatment outcomes.
Siponimod is an approved treatment for adults with relapsing forms of MS, including clinically isolated syndrome and active secondary progressive MS. Compared with placebo, siponimod significantly reduced the risk of confirmed disability progression in the phase 3 EXPAND trial.
Efficacy was similar between age groups
Le Hua, MD, a neurologist at the Cleveland Clinic Lou Ruvo Center for Brain Health in Las Vegas, and colleagues sought to evaluate the safety and efficacy of siponimod by age in patients with active secondary progressive MS who participated in the EXPAND study. The investigators defined active disease as a relapse in the 2 years before screening or one or more T1 gadolinium–enhancing lesions at baseline. Time to 3- and 6-month confirmed disability progression, defined using Expanded Disability Status Scale (EDSS) scores, were the efficacy endpoints of this analysis. Dr. Hua and colleagues also examined adverse events, serious adverse events, and adverse events leading to treatment discontinuation.
Of the 1,651 patients included in EXPAND, 779 had active secondary progressive MS. Dr. Hua and colleagues categorized this group as younger than 45 years (306 patients) or 45 years or older (473 patients). In the overall EXPAND population, siponimod reduced the risk of 3-month confirmed disability progression by 21% and the risk of 6-month confirmed disability progression by 26%, compared with placebo. Among patients younger than 45 years, siponimod reduced the risks of 3- and 6-month confirmed disability progression by 32% and 40%, respectively. Among patients aged 45 years or older, siponimod reduced the risks of 3- and 6-month confirmed disability progression by 31% and 33%, respectively.
Siponimod’s safety profile was similar between age groups. Among participants younger than 45 years, the rate of any adverse event was 82.6% for siponimod and 82.8% for placebo. In patients age 45 years or older, the rate of any adverse event was 89.8% for siponimod and 75.9% for placebo. The rate of serious adverse events was lower for siponimod than for placebo in both age groups. Among patients younger than 45 years, the rate was 12.7% for siponimod and 15.1% for placebo. Among patients age 45 years or older, the rate was 18.2% for siponimod and 19.4% for placebo. The rate of adverse events leading to discontinuation, however, was slightly higher among older patients. For younger patients, the rate was 3.3% for siponimod and 4.3% for placebo. For older patients, the rate was 7.6% for siponimod and 7.1% for placebo.
Real-world data will provide clearer answers
Rates of adverse events, serious adverse events, and adverse events leading to discontinuation were all higher in patients aged 45 or older. “This was not a surprising finding,” said Dr. Hua. “The differences appeared small, and the study was not powered to detect if these differences were significant. ... In general, older patients are more likely to have more cardiac events and infections than younger patients.”
Few studies have analyzed the efficacy and safety of MS therapy by age, said Dr. Hua. In 2015, Matell et al. evaluated the effectiveness of natalizumab in patients over age 50 years. In that study, a greater number of older patients, compared with younger patients, stopped treatment because of lack of effectiveness. Studies of other disease-modifying therapies (DMTs) have included subgroup analyses based on age, “but subgroup analyses are limited in the ability to make any strong conclusions regarding significant differences in safety,” said Dr. Hua. “A recent meta-analysis of published clinical trials of all DMTs performed by Weideman et al. indicated that the efficacy of DMTs appears to wane after age 53 years. However, there are limitations in interpreting this data, as they weren’t able to evaluate the raw data. And in most clinical trials, the age cutoff is usually 55 years, which limits generalizations. Real-world data analyses are needed to truly understand efficacy and safety of DMT in older patients.”
Novartis funded the study. Dr. Hua received fees from Novartis, Biogen, Celgene, EMD Serono, Genentech, and Genzyme.
SOURCE: Hua L et al. ACTRIMS FORUM 2020, Abstract P029.
REPORTING FROM ACTRIMS FORUM 2020
Drug reduces oral mucositis without affecting tumor control
A small molecule can provide a “clinically meaningful reduction” in severe oral mucositis without affecting tumor control in head and neck cancer, according to an investigator from a phase 2 trial.
The molecule, GC4419 (avasopasem manganese), is designed to convert superoxide to hydrogen peroxide and oxygen, thereby protecting normal tissue from damage associated with radiotherapy.
Investigators tested GC4419 in a phase 2 trial of patients with locally advanced oral cavity or oropharynx cancer who received intensity-modulated radiotherapy plus cisplatin. Initial results from this trial were published in December (J Clin Oncol. 2019 Dec 1;37[34]:3256-65).
Carryn M. Anderson, MD, of University of Iowa Hospitals & Clinics in Iowa City, presented updated results at the Multidisciplinary Head and Neck Cancer Symposium, sponsored by the American Society for Radiation Oncology.
The trial (NCT02508389) enrolled 223 patients scheduled to be treated with intensity-modulated radiotherapy (60-72 Gy or greater than 50 Gy to more than two oral sites) plus cisplatin (weekly or every 3 weeks).
The patients were randomized to receive 30 mg of GC4419 (n = 73), 90 mg of GC4419 (n = 76), or placebo (n = 74) intravenously over 60 minutes prior to each radiotherapy fraction. Baseline patient and tumor characteristics were well balanced among the treatment arms.
Efficacy and safety
The study’s primary endpoint was the duration of severe oral mucositis. When compared with placebo, the 90-mg dose of GC4419 conferred a 92% reduction in median days of severe oral mucositis (P = .024).
The 90-mg dose of GC4419 also reduced the incidence of severe oral mucositis by 34% (P = .009) and the rate of grade 4 oral mucositis by 47% (P = .045) when compared with placebo.
“GC4419, particularly the 90-mg dosage, provides a clinically meaningful reduction in severe oral mucositis duration, incidence, and severity,” Dr. Anderson noted.
“Safety was comparable across study arms,” she added. “The most frequent adverse events were those already expected with cisplatin and radiation, and those were not worsened in any way by the addition of this drug.”
Adverse events expected with GC4419 were “mild and transient,” according to Dr. Anderson. These events included syncope, hypotension/orthostasis, and oral/facial paresthesia.
Grade 3 syncope occurred in 4% of patients in the placebo arm, 4% in the 30-mg arm, and 6% in the 90-mg arm. Grade 3 hypotension/orthostasis occurred in 6%, 4%, and 4%, respectively. Grade 1 oral/facial paresthesia occurred in 15%, 10%, and 19%, respectively.
Long-term outcomes
“The 1- and 2-year tumor control is similar with utilization of [GC4419], and this is consistent with the drug’s known mechanism and previous animal models,” Dr. Anderson said.
At 1 and 2 years, there were no significant differences between the treatment arms with regard to locoregional control, distant metastasis, progression-free survival, or overall survival.
The 1-year progression-free survival was 82% in the placebo arm, 86% in the 30-mg arm, and 80% in the 90-mg arm. The 1-year overall survival was 93%, 91%, and 88%, respectively.
The 2-year progression-free survival was 77% in the placebo arm, 76% in the 30-mg arm, and 77% in the 90-mg arm. The 1-year overall survival was 87%, 85%, and 86%, respectively.
From trials to practice
Despite the favorable results of this trial, meeting attendees expressed concerns that the administration of GC4419 may not be feasible in real-life because it is labor- and resource-intensive.
“Methodologically, the study was excellent,” said attendee Shahid Iqbal, MBBS, a consultant clinical oncologist at Newcastle upon Tyne Hospitals NHS Foundation Trust in the United Kingdom, who was not involved in this trial.
Dr. Iqbal noted that this placebo-controlled trial had a “very reasonable number of patients in each arm,” the 90-mg dose of GC4419 was “effective,” and the drug had “no adverse impact on survival.”
“However, in my personal opinion, this intravenous infusion on a daily basis is not feasible in real life,” Dr. Iqbal said. “I cannot see this becoming a standard of care in National Health Service UK. This is simply not cost-effective at all.”
Although she didn’t address costs, Dr. Anderson did acknowledge that administering GC4419 is labor intensive.
“But we are hopeful that, if this drug ultimately makes it to [Food and Drug Administration] approval, the efficacy benefits we are showing will make the work flow changes worthwhile,” she said. “We certainly have shown that, in this study, 44 institutions could make that happen. At the University of Iowa, we found it easiest to institute an infusion space within the department of radiation oncology rather than relying on our medical oncology colleagues to let us borrow a chair.”
GC4419 is now under investigation in a phase 3 trial (NCT03689712) and has received fast track and breakthrough therapy designations from the FDA.
The phase 2 trial was sponsored by Galera Therapeutics. Dr. Anderson is an uncompensated research adviser to the company. Dr. Iqbal has no relevant conflicts of interest.
SOURCE: Anderson CM et al. Head and Neck Cancer Symposium. Abstract LBA 2.
A small molecule can provide a “clinically meaningful reduction” in severe oral mucositis without affecting tumor control in head and neck cancer, according to an investigator from a phase 2 trial.
The molecule, GC4419 (avasopasem manganese), is designed to convert superoxide to hydrogen peroxide and oxygen, thereby protecting normal tissue from damage associated with radiotherapy.
Investigators tested GC4419 in a phase 2 trial of patients with locally advanced oral cavity or oropharynx cancer who received intensity-modulated radiotherapy plus cisplatin. Initial results from this trial were published in December (J Clin Oncol. 2019 Dec 1;37[34]:3256-65).
Carryn M. Anderson, MD, of University of Iowa Hospitals & Clinics in Iowa City, presented updated results at the Multidisciplinary Head and Neck Cancer Symposium, sponsored by the American Society for Radiation Oncology.
The trial (NCT02508389) enrolled 223 patients scheduled to be treated with intensity-modulated radiotherapy (60-72 Gy or greater than 50 Gy to more than two oral sites) plus cisplatin (weekly or every 3 weeks).
The patients were randomized to receive 30 mg of GC4419 (n = 73), 90 mg of GC4419 (n = 76), or placebo (n = 74) intravenously over 60 minutes prior to each radiotherapy fraction. Baseline patient and tumor characteristics were well balanced among the treatment arms.
Efficacy and safety
The study’s primary endpoint was the duration of severe oral mucositis. When compared with placebo, the 90-mg dose of GC4419 conferred a 92% reduction in median days of severe oral mucositis (P = .024).
The 90-mg dose of GC4419 also reduced the incidence of severe oral mucositis by 34% (P = .009) and the rate of grade 4 oral mucositis by 47% (P = .045) when compared with placebo.
“GC4419, particularly the 90-mg dosage, provides a clinically meaningful reduction in severe oral mucositis duration, incidence, and severity,” Dr. Anderson noted.
“Safety was comparable across study arms,” she added. “The most frequent adverse events were those already expected with cisplatin and radiation, and those were not worsened in any way by the addition of this drug.”
Adverse events expected with GC4419 were “mild and transient,” according to Dr. Anderson. These events included syncope, hypotension/orthostasis, and oral/facial paresthesia.
Grade 3 syncope occurred in 4% of patients in the placebo arm, 4% in the 30-mg arm, and 6% in the 90-mg arm. Grade 3 hypotension/orthostasis occurred in 6%, 4%, and 4%, respectively. Grade 1 oral/facial paresthesia occurred in 15%, 10%, and 19%, respectively.
Long-term outcomes
“The 1- and 2-year tumor control is similar with utilization of [GC4419], and this is consistent with the drug’s known mechanism and previous animal models,” Dr. Anderson said.
At 1 and 2 years, there were no significant differences between the treatment arms with regard to locoregional control, distant metastasis, progression-free survival, or overall survival.
The 1-year progression-free survival was 82% in the placebo arm, 86% in the 30-mg arm, and 80% in the 90-mg arm. The 1-year overall survival was 93%, 91%, and 88%, respectively.
The 2-year progression-free survival was 77% in the placebo arm, 76% in the 30-mg arm, and 77% in the 90-mg arm. The 1-year overall survival was 87%, 85%, and 86%, respectively.
From trials to practice
Despite the favorable results of this trial, meeting attendees expressed concerns that the administration of GC4419 may not be feasible in real-life because it is labor- and resource-intensive.
“Methodologically, the study was excellent,” said attendee Shahid Iqbal, MBBS, a consultant clinical oncologist at Newcastle upon Tyne Hospitals NHS Foundation Trust in the United Kingdom, who was not involved in this trial.
Dr. Iqbal noted that this placebo-controlled trial had a “very reasonable number of patients in each arm,” the 90-mg dose of GC4419 was “effective,” and the drug had “no adverse impact on survival.”
“However, in my personal opinion, this intravenous infusion on a daily basis is not feasible in real life,” Dr. Iqbal said. “I cannot see this becoming a standard of care in National Health Service UK. This is simply not cost-effective at all.”
Although she didn’t address costs, Dr. Anderson did acknowledge that administering GC4419 is labor intensive.
“But we are hopeful that, if this drug ultimately makes it to [Food and Drug Administration] approval, the efficacy benefits we are showing will make the work flow changes worthwhile,” she said. “We certainly have shown that, in this study, 44 institutions could make that happen. At the University of Iowa, we found it easiest to institute an infusion space within the department of radiation oncology rather than relying on our medical oncology colleagues to let us borrow a chair.”
GC4419 is now under investigation in a phase 3 trial (NCT03689712) and has received fast track and breakthrough therapy designations from the FDA.
The phase 2 trial was sponsored by Galera Therapeutics. Dr. Anderson is an uncompensated research adviser to the company. Dr. Iqbal has no relevant conflicts of interest.
SOURCE: Anderson CM et al. Head and Neck Cancer Symposium. Abstract LBA 2.
A small molecule can provide a “clinically meaningful reduction” in severe oral mucositis without affecting tumor control in head and neck cancer, according to an investigator from a phase 2 trial.
The molecule, GC4419 (avasopasem manganese), is designed to convert superoxide to hydrogen peroxide and oxygen, thereby protecting normal tissue from damage associated with radiotherapy.
Investigators tested GC4419 in a phase 2 trial of patients with locally advanced oral cavity or oropharynx cancer who received intensity-modulated radiotherapy plus cisplatin. Initial results from this trial were published in December (J Clin Oncol. 2019 Dec 1;37[34]:3256-65).
Carryn M. Anderson, MD, of University of Iowa Hospitals & Clinics in Iowa City, presented updated results at the Multidisciplinary Head and Neck Cancer Symposium, sponsored by the American Society for Radiation Oncology.
The trial (NCT02508389) enrolled 223 patients scheduled to be treated with intensity-modulated radiotherapy (60-72 Gy or greater than 50 Gy to more than two oral sites) plus cisplatin (weekly or every 3 weeks).
The patients were randomized to receive 30 mg of GC4419 (n = 73), 90 mg of GC4419 (n = 76), or placebo (n = 74) intravenously over 60 minutes prior to each radiotherapy fraction. Baseline patient and tumor characteristics were well balanced among the treatment arms.
Efficacy and safety
The study’s primary endpoint was the duration of severe oral mucositis. When compared with placebo, the 90-mg dose of GC4419 conferred a 92% reduction in median days of severe oral mucositis (P = .024).
The 90-mg dose of GC4419 also reduced the incidence of severe oral mucositis by 34% (P = .009) and the rate of grade 4 oral mucositis by 47% (P = .045) when compared with placebo.
“GC4419, particularly the 90-mg dosage, provides a clinically meaningful reduction in severe oral mucositis duration, incidence, and severity,” Dr. Anderson noted.
“Safety was comparable across study arms,” she added. “The most frequent adverse events were those already expected with cisplatin and radiation, and those were not worsened in any way by the addition of this drug.”
Adverse events expected with GC4419 were “mild and transient,” according to Dr. Anderson. These events included syncope, hypotension/orthostasis, and oral/facial paresthesia.
Grade 3 syncope occurred in 4% of patients in the placebo arm, 4% in the 30-mg arm, and 6% in the 90-mg arm. Grade 3 hypotension/orthostasis occurred in 6%, 4%, and 4%, respectively. Grade 1 oral/facial paresthesia occurred in 15%, 10%, and 19%, respectively.
Long-term outcomes
“The 1- and 2-year tumor control is similar with utilization of [GC4419], and this is consistent with the drug’s known mechanism and previous animal models,” Dr. Anderson said.
At 1 and 2 years, there were no significant differences between the treatment arms with regard to locoregional control, distant metastasis, progression-free survival, or overall survival.
The 1-year progression-free survival was 82% in the placebo arm, 86% in the 30-mg arm, and 80% in the 90-mg arm. The 1-year overall survival was 93%, 91%, and 88%, respectively.
The 2-year progression-free survival was 77% in the placebo arm, 76% in the 30-mg arm, and 77% in the 90-mg arm. The 1-year overall survival was 87%, 85%, and 86%, respectively.
From trials to practice
Despite the favorable results of this trial, meeting attendees expressed concerns that the administration of GC4419 may not be feasible in real-life because it is labor- and resource-intensive.
“Methodologically, the study was excellent,” said attendee Shahid Iqbal, MBBS, a consultant clinical oncologist at Newcastle upon Tyne Hospitals NHS Foundation Trust in the United Kingdom, who was not involved in this trial.
Dr. Iqbal noted that this placebo-controlled trial had a “very reasonable number of patients in each arm,” the 90-mg dose of GC4419 was “effective,” and the drug had “no adverse impact on survival.”
“However, in my personal opinion, this intravenous infusion on a daily basis is not feasible in real life,” Dr. Iqbal said. “I cannot see this becoming a standard of care in National Health Service UK. This is simply not cost-effective at all.”
Although she didn’t address costs, Dr. Anderson did acknowledge that administering GC4419 is labor intensive.
“But we are hopeful that, if this drug ultimately makes it to [Food and Drug Administration] approval, the efficacy benefits we are showing will make the work flow changes worthwhile,” she said. “We certainly have shown that, in this study, 44 institutions could make that happen. At the University of Iowa, we found it easiest to institute an infusion space within the department of radiation oncology rather than relying on our medical oncology colleagues to let us borrow a chair.”
GC4419 is now under investigation in a phase 3 trial (NCT03689712) and has received fast track and breakthrough therapy designations from the FDA.
The phase 2 trial was sponsored by Galera Therapeutics. Dr. Anderson is an uncompensated research adviser to the company. Dr. Iqbal has no relevant conflicts of interest.
SOURCE: Anderson CM et al. Head and Neck Cancer Symposium. Abstract LBA 2.
REPORTING FROM HEAD AND NECK CANCERS SYMPOSIUM 2020
Know the 15% rule in scleroderma
MAUI, HAWAII – The 15% rule in scleroderma is a handy tool that raises awareness of the disease’s associated prevalence of various severe organ complications so clinicians can screen appropriately, Janet Pope, MD, said at the 2020 Rheumatology Winter Clinical Symposium.
Dr. Pope and colleagues in the Canadian Scleroderma Research Group developed the 15% rule because they recognized that scleroderma is rare enough that most physicians practicing outside of a few specialized centers don’t see many affected patients. The systemic autoimmune disease is marked by numerous possible expressions of vascular inflammation and malfunction, fibrosis, and autoimmunity in different organ systems.
“A lot of clinicians do not know how common this stuff is,” according to Dr. Pope, professor of medicine at the University of Western Ontario and head of the division of rheumatology at St. Joseph’s Health Center in London, Ont.
Basically, the 15% rule holds that, at any given time, a patient with scleroderma has roughly a 15% chance – or one in six – of having any of an extensive array of severe organ complications. That means a 15% chance of having prevalent clinically significant pulmonary hypertension as defined by a systolic pulmonary artery pressure of 45 mm Hg or more on Doppler echocardiography, a 15% likelihood of interstitial lung disease or clinically significant pulmonary fibrosis as suggested by a forced vital capacity less than 70% of predicted, a 15% prevalence of Sjögren’s syndrome, a 15% likelihood of having pulmonary artery hypertension upon right heart catheterization, a 15% chance of inflammatory arthritis, and a one-in-six chance of having a myopathy or myositis. Also, diastolic dysfunction, 15%. Ditto symptomatic arrhythmias.
“It’s a good little rule of thumb,” Dr. Pope commented.
The odds of having a current digital ulcer on any given day? Again, about 15%. In addition, scleroderma patients have a 15% lifetime risk of developing a complicated digital ulcer requiring hospitalization and/or amputation, she continued.
And while the prevalence of scleroderma renal crisis in the overall population with scleroderma is low, at 3%, in the subgroup with diffuse cutaneous systemic sclerosis, it climbs to 12%-15%.
Every rule has its exceptions. The 15% rule doesn’t apply to Raynaud’s phenomenon, which is present in nearly all patients with scleroderma, nor to gastroesophageal reflux disease or dysphagia, present in roughly 80% of patients.
Dr. Pope and coinvestigators developed the 15% rule pertaining to the prevalence of serious organ complications in scleroderma by conducting a systematic review of 69 published studies, each including a minimum of 50 scleroderma patients. The detailed results of the systematic review have been published.
Dr. Pope reported receiving research grants from and/or serving as a consultant to more than a dozen pharmaceutical companies.
MAUI, HAWAII – The 15% rule in scleroderma is a handy tool that raises awareness of the disease’s associated prevalence of various severe organ complications so clinicians can screen appropriately, Janet Pope, MD, said at the 2020 Rheumatology Winter Clinical Symposium.
Dr. Pope and colleagues in the Canadian Scleroderma Research Group developed the 15% rule because they recognized that scleroderma is rare enough that most physicians practicing outside of a few specialized centers don’t see many affected patients. The systemic autoimmune disease is marked by numerous possible expressions of vascular inflammation and malfunction, fibrosis, and autoimmunity in different organ systems.
“A lot of clinicians do not know how common this stuff is,” according to Dr. Pope, professor of medicine at the University of Western Ontario and head of the division of rheumatology at St. Joseph’s Health Center in London, Ont.
Basically, the 15% rule holds that, at any given time, a patient with scleroderma has roughly a 15% chance – or one in six – of having any of an extensive array of severe organ complications. That means a 15% chance of having prevalent clinically significant pulmonary hypertension as defined by a systolic pulmonary artery pressure of 45 mm Hg or more on Doppler echocardiography, a 15% likelihood of interstitial lung disease or clinically significant pulmonary fibrosis as suggested by a forced vital capacity less than 70% of predicted, a 15% prevalence of Sjögren’s syndrome, a 15% likelihood of having pulmonary artery hypertension upon right heart catheterization, a 15% chance of inflammatory arthritis, and a one-in-six chance of having a myopathy or myositis. Also, diastolic dysfunction, 15%. Ditto symptomatic arrhythmias.
“It’s a good little rule of thumb,” Dr. Pope commented.
The odds of having a current digital ulcer on any given day? Again, about 15%. In addition, scleroderma patients have a 15% lifetime risk of developing a complicated digital ulcer requiring hospitalization and/or amputation, she continued.
And while the prevalence of scleroderma renal crisis in the overall population with scleroderma is low, at 3%, in the subgroup with diffuse cutaneous systemic sclerosis, it climbs to 12%-15%.
Every rule has its exceptions. The 15% rule doesn’t apply to Raynaud’s phenomenon, which is present in nearly all patients with scleroderma, nor to gastroesophageal reflux disease or dysphagia, present in roughly 80% of patients.
Dr. Pope and coinvestigators developed the 15% rule pertaining to the prevalence of serious organ complications in scleroderma by conducting a systematic review of 69 published studies, each including a minimum of 50 scleroderma patients. The detailed results of the systematic review have been published.
Dr. Pope reported receiving research grants from and/or serving as a consultant to more than a dozen pharmaceutical companies.
MAUI, HAWAII – The 15% rule in scleroderma is a handy tool that raises awareness of the disease’s associated prevalence of various severe organ complications so clinicians can screen appropriately, Janet Pope, MD, said at the 2020 Rheumatology Winter Clinical Symposium.
Dr. Pope and colleagues in the Canadian Scleroderma Research Group developed the 15% rule because they recognized that scleroderma is rare enough that most physicians practicing outside of a few specialized centers don’t see many affected patients. The systemic autoimmune disease is marked by numerous possible expressions of vascular inflammation and malfunction, fibrosis, and autoimmunity in different organ systems.
“A lot of clinicians do not know how common this stuff is,” according to Dr. Pope, professor of medicine at the University of Western Ontario and head of the division of rheumatology at St. Joseph’s Health Center in London, Ont.
Basically, the 15% rule holds that, at any given time, a patient with scleroderma has roughly a 15% chance – or one in six – of having any of an extensive array of severe organ complications. That means a 15% chance of having prevalent clinically significant pulmonary hypertension as defined by a systolic pulmonary artery pressure of 45 mm Hg or more on Doppler echocardiography, a 15% likelihood of interstitial lung disease or clinically significant pulmonary fibrosis as suggested by a forced vital capacity less than 70% of predicted, a 15% prevalence of Sjögren’s syndrome, a 15% likelihood of having pulmonary artery hypertension upon right heart catheterization, a 15% chance of inflammatory arthritis, and a one-in-six chance of having a myopathy or myositis. Also, diastolic dysfunction, 15%. Ditto symptomatic arrhythmias.
“It’s a good little rule of thumb,” Dr. Pope commented.
The odds of having a current digital ulcer on any given day? Again, about 15%. In addition, scleroderma patients have a 15% lifetime risk of developing a complicated digital ulcer requiring hospitalization and/or amputation, she continued.
And while the prevalence of scleroderma renal crisis in the overall population with scleroderma is low, at 3%, in the subgroup with diffuse cutaneous systemic sclerosis, it climbs to 12%-15%.
Every rule has its exceptions. The 15% rule doesn’t apply to Raynaud’s phenomenon, which is present in nearly all patients with scleroderma, nor to gastroesophageal reflux disease or dysphagia, present in roughly 80% of patients.
Dr. Pope and coinvestigators developed the 15% rule pertaining to the prevalence of serious organ complications in scleroderma by conducting a systematic review of 69 published studies, each including a minimum of 50 scleroderma patients. The detailed results of the systematic review have been published.
Dr. Pope reported receiving research grants from and/or serving as a consultant to more than a dozen pharmaceutical companies.
REPORTING FROM RWCS 2020
Internal Medicine 2020 canceled
The American College of Physicians has recently joined the list of medical-specialty organizations to have canceled an upcoming meeting because of the ongoing COVID-19 (coronavirus disease) outbreak.
according to a statement from the organization.
“ACP’s decision is based on recent reports from the World Health Organization (WHO) and the Centers for Disease Control and Prevention (CDC) of rapidly escalating concerns about the Coronavirus Disease 2019 (COVID-19), and in recognition of the vital role of internal medicine physicians in diagnosing, managing, and caring for their patients and communities on the front lines,” according to the announcement.
The ACP is offering a refund to those who have already registered to attend the meeting.
The organization has included responses on its website to a number of frequently asked questions related to the cancellation. One response notes that ACP is offering paid registrants an opportunity to apply their meeting registration credit toward a 30-hour CME package, “which will be made available as soon as possible.” This package, named ACP CME 30, “will comprise curated, online lectures originally scheduled for live presentation at Internal Medicine Meeting 2020.”
The American College of Physicians has recently joined the list of medical-specialty organizations to have canceled an upcoming meeting because of the ongoing COVID-19 (coronavirus disease) outbreak.
according to a statement from the organization.
“ACP’s decision is based on recent reports from the World Health Organization (WHO) and the Centers for Disease Control and Prevention (CDC) of rapidly escalating concerns about the Coronavirus Disease 2019 (COVID-19), and in recognition of the vital role of internal medicine physicians in diagnosing, managing, and caring for their patients and communities on the front lines,” according to the announcement.
The ACP is offering a refund to those who have already registered to attend the meeting.
The organization has included responses on its website to a number of frequently asked questions related to the cancellation. One response notes that ACP is offering paid registrants an opportunity to apply their meeting registration credit toward a 30-hour CME package, “which will be made available as soon as possible.” This package, named ACP CME 30, “will comprise curated, online lectures originally scheduled for live presentation at Internal Medicine Meeting 2020.”
The American College of Physicians has recently joined the list of medical-specialty organizations to have canceled an upcoming meeting because of the ongoing COVID-19 (coronavirus disease) outbreak.
according to a statement from the organization.
“ACP’s decision is based on recent reports from the World Health Organization (WHO) and the Centers for Disease Control and Prevention (CDC) of rapidly escalating concerns about the Coronavirus Disease 2019 (COVID-19), and in recognition of the vital role of internal medicine physicians in diagnosing, managing, and caring for their patients and communities on the front lines,” according to the announcement.
The ACP is offering a refund to those who have already registered to attend the meeting.
The organization has included responses on its website to a number of frequently asked questions related to the cancellation. One response notes that ACP is offering paid registrants an opportunity to apply their meeting registration credit toward a 30-hour CME package, “which will be made available as soon as possible.” This package, named ACP CME 30, “will comprise curated, online lectures originally scheduled for live presentation at Internal Medicine Meeting 2020.”
American Headache Society updates guideline on neuroimaging for migraine
Migraine with atypical features may require neuroimaging, according to the guideline. These include an unusual aura; change in clinical features; a first or worst migraine; a migraine that presents with brainstem aura, confusion, or motor manifestation; migraine accompaniments in later life; headaches that are side-locked or posttraumatic; and aura that presents without headache.
Assessing the evidence
The recommendation to avoid MRI or CT in otherwise neurologically normal patients with migraine carried a grade A recommendation from the American Headache Society, while the specific considerations for neuroimaging was based on consensus and carried a grade C recommendation, according to lead author Randolph W. Evans, MD, of the department of neurology at Baylor College of Medicine in Houston, and colleagues.
The recommendations, published in the journal Headache (2020 Feb;60(2):318-36), came from a systematic review of 23 studies of adults at least 18 years old who underwent MRI or CT during outpatient treatment for migraine between 1973 and 2018. Ten studies looked at CT neuroimaging in patients with migraine, nine studies examined MRI neuroimaging alone in patients with migraine, and four studies contained adults with headache or migraine who underwent either MRI or CT. The majority of studies analyzed were retrospective or cross-sectional in nature, while four studies were prospective observational studies.
Dr. Evans and colleagues noted that neuroimaging for patients with suspected migraine is ordered for a variety of reasons, such as excluding conditions that aren’t migraine, diagnostic certainty, cognitive bias, practice workflow, medicolegal concerns, addressing patient and family anxiety, and addressing clinician anxiety. Neuroimaging also can be costly, they said, adding up to an estimated $1 billion annually according to one study, and can lead to additional testing from findings that may not be clinically significant.
Good advice, with caveats
In an interview, Alan M. Rapoport, MD, editor-in-chief of Neurology Reviews, said that while he generally does not like broad guideline recommendations, the recommendation made by the American Headache Society to avoid neuroimaging in patients with a normal neurological examination without any atypical features and red flags “takes most of the important factors into consideration and will work almost all the time.” The recommendation made by consensus for specific considerations of neuroimaging was issued by top headache specialists in the United States who reviewed the data, and it is unlikely a patient with a migraine as diagnosed by the International Classification of Headache Disorders with a normal neurological examination would have a significant abnormality that would appear with imaging, Dr. Rapoport said.
“If everyone caring for migraine patients knew these recommendations, and used them unless the patients fit the exclusions mentioned, we would have more efficient clinical practice and save lots of money on unnecessary scanning,” he said.
However, Dr. Rapoport, clinical professor of neurology at the University of California, Los Angeles, founder of the New England Center for Headache, and past president of The International Headache Society, said that not all clinicians will be convinced by the American Headache Society’s recommendations.
“Various third parties often jump on society recommendations or guidelines and prevent smart clinicians from doing what they need to do when they want to disregard the recommendation or guideline,” he explained. “More importantly, if a physician feels the need to think out of the box and image a patient without a clear reason, and the patient cannot pay for the scan when a medical insurance company refuses to authorize it, there can be a bad result if the patient does not get the study.”
Dr. Rapoport noted that the guideline does not address situations where neuroimaging may not pick up conditions that lead to migraine, such as a subarachnoid or subdural hemorrhage, reversible cerebral vasoconstriction syndrome, or early aspects of low cerebrospinal fluid pressure syndrome. Anxiety on the part of the patient or the clinician is another area that can be addressed by future research, he said.
“If the clinician does a good job of explaining the odds of anything significant being found with a typical migraine history and normal examination, and the patient says [they] need an MRI with contrast to be sure, it will be difficult to dissuade them,” said Dr. Rapoport. “If you don’t order one, they will find a way to get one. If it is abnormal, you could be in trouble. Also, if the clinician has no good reason to do a scan but has anxiety about what is being missed, it will probably get done.”
There was no funding source for the guidelines. The authors reported personal and institutional relationships in the form of advisory board memberships, investigator appointments, speakers bureau positions, research support, and consultancies for a variety of pharmaceutical companies, agencies, institutions, publishers, and other organizations.
Migraine with atypical features may require neuroimaging, according to the guideline. These include an unusual aura; change in clinical features; a first or worst migraine; a migraine that presents with brainstem aura, confusion, or motor manifestation; migraine accompaniments in later life; headaches that are side-locked or posttraumatic; and aura that presents without headache.
Assessing the evidence
The recommendation to avoid MRI or CT in otherwise neurologically normal patients with migraine carried a grade A recommendation from the American Headache Society, while the specific considerations for neuroimaging was based on consensus and carried a grade C recommendation, according to lead author Randolph W. Evans, MD, of the department of neurology at Baylor College of Medicine in Houston, and colleagues.
The recommendations, published in the journal Headache (2020 Feb;60(2):318-36), came from a systematic review of 23 studies of adults at least 18 years old who underwent MRI or CT during outpatient treatment for migraine between 1973 and 2018. Ten studies looked at CT neuroimaging in patients with migraine, nine studies examined MRI neuroimaging alone in patients with migraine, and four studies contained adults with headache or migraine who underwent either MRI or CT. The majority of studies analyzed were retrospective or cross-sectional in nature, while four studies were prospective observational studies.
Dr. Evans and colleagues noted that neuroimaging for patients with suspected migraine is ordered for a variety of reasons, such as excluding conditions that aren’t migraine, diagnostic certainty, cognitive bias, practice workflow, medicolegal concerns, addressing patient and family anxiety, and addressing clinician anxiety. Neuroimaging also can be costly, they said, adding up to an estimated $1 billion annually according to one study, and can lead to additional testing from findings that may not be clinically significant.
Good advice, with caveats
In an interview, Alan M. Rapoport, MD, editor-in-chief of Neurology Reviews, said that while he generally does not like broad guideline recommendations, the recommendation made by the American Headache Society to avoid neuroimaging in patients with a normal neurological examination without any atypical features and red flags “takes most of the important factors into consideration and will work almost all the time.” The recommendation made by consensus for specific considerations of neuroimaging was issued by top headache specialists in the United States who reviewed the data, and it is unlikely a patient with a migraine as diagnosed by the International Classification of Headache Disorders with a normal neurological examination would have a significant abnormality that would appear with imaging, Dr. Rapoport said.
“If everyone caring for migraine patients knew these recommendations, and used them unless the patients fit the exclusions mentioned, we would have more efficient clinical practice and save lots of money on unnecessary scanning,” he said.
However, Dr. Rapoport, clinical professor of neurology at the University of California, Los Angeles, founder of the New England Center for Headache, and past president of The International Headache Society, said that not all clinicians will be convinced by the American Headache Society’s recommendations.
“Various third parties often jump on society recommendations or guidelines and prevent smart clinicians from doing what they need to do when they want to disregard the recommendation or guideline,” he explained. “More importantly, if a physician feels the need to think out of the box and image a patient without a clear reason, and the patient cannot pay for the scan when a medical insurance company refuses to authorize it, there can be a bad result if the patient does not get the study.”
Dr. Rapoport noted that the guideline does not address situations where neuroimaging may not pick up conditions that lead to migraine, such as a subarachnoid or subdural hemorrhage, reversible cerebral vasoconstriction syndrome, or early aspects of low cerebrospinal fluid pressure syndrome. Anxiety on the part of the patient or the clinician is another area that can be addressed by future research, he said.
“If the clinician does a good job of explaining the odds of anything significant being found with a typical migraine history and normal examination, and the patient says [they] need an MRI with contrast to be sure, it will be difficult to dissuade them,” said Dr. Rapoport. “If you don’t order one, they will find a way to get one. If it is abnormal, you could be in trouble. Also, if the clinician has no good reason to do a scan but has anxiety about what is being missed, it will probably get done.”
There was no funding source for the guidelines. The authors reported personal and institutional relationships in the form of advisory board memberships, investigator appointments, speakers bureau positions, research support, and consultancies for a variety of pharmaceutical companies, agencies, institutions, publishers, and other organizations.
Migraine with atypical features may require neuroimaging, according to the guideline. These include an unusual aura; change in clinical features; a first or worst migraine; a migraine that presents with brainstem aura, confusion, or motor manifestation; migraine accompaniments in later life; headaches that are side-locked or posttraumatic; and aura that presents without headache.
Assessing the evidence
The recommendation to avoid MRI or CT in otherwise neurologically normal patients with migraine carried a grade A recommendation from the American Headache Society, while the specific considerations for neuroimaging was based on consensus and carried a grade C recommendation, according to lead author Randolph W. Evans, MD, of the department of neurology at Baylor College of Medicine in Houston, and colleagues.
The recommendations, published in the journal Headache (2020 Feb;60(2):318-36), came from a systematic review of 23 studies of adults at least 18 years old who underwent MRI or CT during outpatient treatment for migraine between 1973 and 2018. Ten studies looked at CT neuroimaging in patients with migraine, nine studies examined MRI neuroimaging alone in patients with migraine, and four studies contained adults with headache or migraine who underwent either MRI or CT. The majority of studies analyzed were retrospective or cross-sectional in nature, while four studies were prospective observational studies.
Dr. Evans and colleagues noted that neuroimaging for patients with suspected migraine is ordered for a variety of reasons, such as excluding conditions that aren’t migraine, diagnostic certainty, cognitive bias, practice workflow, medicolegal concerns, addressing patient and family anxiety, and addressing clinician anxiety. Neuroimaging also can be costly, they said, adding up to an estimated $1 billion annually according to one study, and can lead to additional testing from findings that may not be clinically significant.
Good advice, with caveats
In an interview, Alan M. Rapoport, MD, editor-in-chief of Neurology Reviews, said that while he generally does not like broad guideline recommendations, the recommendation made by the American Headache Society to avoid neuroimaging in patients with a normal neurological examination without any atypical features and red flags “takes most of the important factors into consideration and will work almost all the time.” The recommendation made by consensus for specific considerations of neuroimaging was issued by top headache specialists in the United States who reviewed the data, and it is unlikely a patient with a migraine as diagnosed by the International Classification of Headache Disorders with a normal neurological examination would have a significant abnormality that would appear with imaging, Dr. Rapoport said.
“If everyone caring for migraine patients knew these recommendations, and used them unless the patients fit the exclusions mentioned, we would have more efficient clinical practice and save lots of money on unnecessary scanning,” he said.
However, Dr. Rapoport, clinical professor of neurology at the University of California, Los Angeles, founder of the New England Center for Headache, and past president of The International Headache Society, said that not all clinicians will be convinced by the American Headache Society’s recommendations.
“Various third parties often jump on society recommendations or guidelines and prevent smart clinicians from doing what they need to do when they want to disregard the recommendation or guideline,” he explained. “More importantly, if a physician feels the need to think out of the box and image a patient without a clear reason, and the patient cannot pay for the scan when a medical insurance company refuses to authorize it, there can be a bad result if the patient does not get the study.”
Dr. Rapoport noted that the guideline does not address situations where neuroimaging may not pick up conditions that lead to migraine, such as a subarachnoid or subdural hemorrhage, reversible cerebral vasoconstriction syndrome, or early aspects of low cerebrospinal fluid pressure syndrome. Anxiety on the part of the patient or the clinician is another area that can be addressed by future research, he said.
“If the clinician does a good job of explaining the odds of anything significant being found with a typical migraine history and normal examination, and the patient says [they] need an MRI with contrast to be sure, it will be difficult to dissuade them,” said Dr. Rapoport. “If you don’t order one, they will find a way to get one. If it is abnormal, you could be in trouble. Also, if the clinician has no good reason to do a scan but has anxiety about what is being missed, it will probably get done.”
There was no funding source for the guidelines. The authors reported personal and institutional relationships in the form of advisory board memberships, investigator appointments, speakers bureau positions, research support, and consultancies for a variety of pharmaceutical companies, agencies, institutions, publishers, and other organizations.
FROM HEADACHE