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Maternal pertussis vax effective for infants in most vulnerable months
Maternal pertussis vaccinations, given during pregnancy, prevent an estimated 65% of pertussis infections in infants, new research indicates.
The study, led by Annette K. Regan, PhD, MPH, a perinatal and pediatric infectious disease epidemiologist at Curtin University, Perth, Australia, was published online in Pediatrics.
Dr. Regan – who is also with the University of San Francisco and the University of California, Los Angeles – and colleagues reviewed data on 279,418 infants born to 252,444 mothers in Australia.
There, about 52% of the women in this study received the Tdap vaccine through a maternal pertussis vaccination program.
Duration of effectiveness in infants was one of the main questions the study sought to answer.
The authors wrote that they assessed vaccine effectiveness through 18 months of age. “We observed significant protection against disease until at least 8 months of age, 2 months longer than reported in previous studies.” From 70% to 90% of all pertussis-attributable hospitalizations and death occur in infancy.
Answering the ‘blunting’ question
This study also set out to clarify an important clinical question regarding a potential “blunting” effect in infants. Previous work had suggested that maternal antibodies from the vaccination could interfere with the effectiveness of infants’ DtaP (the version of Tdap for infants) and other vaccines.
Dr. Regan and colleagues found that, “although we observed slightly lower VE [vaccine effectiveness] point estimates for the third dose of infant pertussis vaccine among maternally vaccinated compared with unvaccinated infants (76.5% vs. 92.9%, P = .002), we did not observe higher rates of pertussis infection (hazard ratio, 0.70; 95% confidence interval, 0.61-3.39).
Best time to give mothers the vaccine
Another clinical debate has centered on when to give the mother the vaccine during pregnancy. The authors concluded: “Our findings support the infant health benefits of recommendations to administer a booster dose of pertussis vaccine near 28 weeks of gestational age.”
That 28-week mark was associated with lower risk of infection in infants through 8 months of age, they wrote.
Positive results in the United States
In an invited commentary, Kathryn M. Edwards, MD, with the division of infectious diseases, department of pediatrics, at Vanderbilt University Medical Center, Nashville, Tenn., highlighted similar positive findings for maternal pertussis vaccination in the United States.
The Centers for Disease Control and Prevention did an ecologic study of infant pertussis cases reported between Jan. 1, 2000, and Dec. 31, 2019. Rates were compared for the years before maternal Tdap vaccinations were recommended against the 7-year period after they were implemented.
That study found that in the period before maternal Tdap vaccination, annual pertussis incidence did not change among infants younger than 2 months and increased slightly in infants 6-12 months.
However, during the period after maternal Tdap vaccination had started (2012-2019), pertussis incidence significantly decreased in infants younger than 2 months and was unchanged in infants 6-12 months.
“As with the Australian data, the U.S. data support the overall benefit of the maternal Tdap program and, as with the Australian data, do not suggest that blunting has led to an increase in cases within the first year of life,” Dr. Edwards wrote.
The CDC notes that pertussis cases are rising and outbreaks are happening across the United States.
“On average, about 1,000 infants are hospitalized and typically between 5 and 15 infants die each year in the United States due to pertussis,” the CDC states.
Uptake low despite positive data
Dr. Edwards noted that, despite positive data supporting maternal vaccination to reduce pertussis, uptake rates are low – between 50% and 60% in Australia, the United Kingdom, and the United States. “Active engagement to increase these rates should be implemented.”
Maternal vaccination might also be implemented soon to protect against other diseases including respiratory syncytial virus and group B streptococcal disease after promising study data, she said.
As with pertussis, the potential “blunting” effect will need to be carefully monitored, she said, “as was done in the carefully conducted study of pertussis reported in this issue of Pediatrics.”
One coauthor has received institutional honoraria for participation in advisory groups for Merck Sharpe & Dohme and Pfizer unrelated to this work. Another coauthor was supported by scholarships provided by the Wesfarmers Centre of Vaccines and Infectious Disease at the Telethon Kids Institute. Dr. Edwards reported receiving grants from the CDC and consulting for Bionet, Dynavax, and IBM. She is a member of the data safety and monitoring board for Sanofi, X-4 Pharma, Seqirus, Moderna, Pfizer, Merck, Roche, Novavax, and Brighton Collaboration.
Maternal pertussis vaccinations, given during pregnancy, prevent an estimated 65% of pertussis infections in infants, new research indicates.
The study, led by Annette K. Regan, PhD, MPH, a perinatal and pediatric infectious disease epidemiologist at Curtin University, Perth, Australia, was published online in Pediatrics.
Dr. Regan – who is also with the University of San Francisco and the University of California, Los Angeles – and colleagues reviewed data on 279,418 infants born to 252,444 mothers in Australia.
There, about 52% of the women in this study received the Tdap vaccine through a maternal pertussis vaccination program.
Duration of effectiveness in infants was one of the main questions the study sought to answer.
The authors wrote that they assessed vaccine effectiveness through 18 months of age. “We observed significant protection against disease until at least 8 months of age, 2 months longer than reported in previous studies.” From 70% to 90% of all pertussis-attributable hospitalizations and death occur in infancy.
Answering the ‘blunting’ question
This study also set out to clarify an important clinical question regarding a potential “blunting” effect in infants. Previous work had suggested that maternal antibodies from the vaccination could interfere with the effectiveness of infants’ DtaP (the version of Tdap for infants) and other vaccines.
Dr. Regan and colleagues found that, “although we observed slightly lower VE [vaccine effectiveness] point estimates for the third dose of infant pertussis vaccine among maternally vaccinated compared with unvaccinated infants (76.5% vs. 92.9%, P = .002), we did not observe higher rates of pertussis infection (hazard ratio, 0.70; 95% confidence interval, 0.61-3.39).
Best time to give mothers the vaccine
Another clinical debate has centered on when to give the mother the vaccine during pregnancy. The authors concluded: “Our findings support the infant health benefits of recommendations to administer a booster dose of pertussis vaccine near 28 weeks of gestational age.”
That 28-week mark was associated with lower risk of infection in infants through 8 months of age, they wrote.
Positive results in the United States
In an invited commentary, Kathryn M. Edwards, MD, with the division of infectious diseases, department of pediatrics, at Vanderbilt University Medical Center, Nashville, Tenn., highlighted similar positive findings for maternal pertussis vaccination in the United States.
The Centers for Disease Control and Prevention did an ecologic study of infant pertussis cases reported between Jan. 1, 2000, and Dec. 31, 2019. Rates were compared for the years before maternal Tdap vaccinations were recommended against the 7-year period after they were implemented.
That study found that in the period before maternal Tdap vaccination, annual pertussis incidence did not change among infants younger than 2 months and increased slightly in infants 6-12 months.
However, during the period after maternal Tdap vaccination had started (2012-2019), pertussis incidence significantly decreased in infants younger than 2 months and was unchanged in infants 6-12 months.
“As with the Australian data, the U.S. data support the overall benefit of the maternal Tdap program and, as with the Australian data, do not suggest that blunting has led to an increase in cases within the first year of life,” Dr. Edwards wrote.
The CDC notes that pertussis cases are rising and outbreaks are happening across the United States.
“On average, about 1,000 infants are hospitalized and typically between 5 and 15 infants die each year in the United States due to pertussis,” the CDC states.
Uptake low despite positive data
Dr. Edwards noted that, despite positive data supporting maternal vaccination to reduce pertussis, uptake rates are low – between 50% and 60% in Australia, the United Kingdom, and the United States. “Active engagement to increase these rates should be implemented.”
Maternal vaccination might also be implemented soon to protect against other diseases including respiratory syncytial virus and group B streptococcal disease after promising study data, she said.
As with pertussis, the potential “blunting” effect will need to be carefully monitored, she said, “as was done in the carefully conducted study of pertussis reported in this issue of Pediatrics.”
One coauthor has received institutional honoraria for participation in advisory groups for Merck Sharpe & Dohme and Pfizer unrelated to this work. Another coauthor was supported by scholarships provided by the Wesfarmers Centre of Vaccines and Infectious Disease at the Telethon Kids Institute. Dr. Edwards reported receiving grants from the CDC and consulting for Bionet, Dynavax, and IBM. She is a member of the data safety and monitoring board for Sanofi, X-4 Pharma, Seqirus, Moderna, Pfizer, Merck, Roche, Novavax, and Brighton Collaboration.
Maternal pertussis vaccinations, given during pregnancy, prevent an estimated 65% of pertussis infections in infants, new research indicates.
The study, led by Annette K. Regan, PhD, MPH, a perinatal and pediatric infectious disease epidemiologist at Curtin University, Perth, Australia, was published online in Pediatrics.
Dr. Regan – who is also with the University of San Francisco and the University of California, Los Angeles – and colleagues reviewed data on 279,418 infants born to 252,444 mothers in Australia.
There, about 52% of the women in this study received the Tdap vaccine through a maternal pertussis vaccination program.
Duration of effectiveness in infants was one of the main questions the study sought to answer.
The authors wrote that they assessed vaccine effectiveness through 18 months of age. “We observed significant protection against disease until at least 8 months of age, 2 months longer than reported in previous studies.” From 70% to 90% of all pertussis-attributable hospitalizations and death occur in infancy.
Answering the ‘blunting’ question
This study also set out to clarify an important clinical question regarding a potential “blunting” effect in infants. Previous work had suggested that maternal antibodies from the vaccination could interfere with the effectiveness of infants’ DtaP (the version of Tdap for infants) and other vaccines.
Dr. Regan and colleagues found that, “although we observed slightly lower VE [vaccine effectiveness] point estimates for the third dose of infant pertussis vaccine among maternally vaccinated compared with unvaccinated infants (76.5% vs. 92.9%, P = .002), we did not observe higher rates of pertussis infection (hazard ratio, 0.70; 95% confidence interval, 0.61-3.39).
Best time to give mothers the vaccine
Another clinical debate has centered on when to give the mother the vaccine during pregnancy. The authors concluded: “Our findings support the infant health benefits of recommendations to administer a booster dose of pertussis vaccine near 28 weeks of gestational age.”
That 28-week mark was associated with lower risk of infection in infants through 8 months of age, they wrote.
Positive results in the United States
In an invited commentary, Kathryn M. Edwards, MD, with the division of infectious diseases, department of pediatrics, at Vanderbilt University Medical Center, Nashville, Tenn., highlighted similar positive findings for maternal pertussis vaccination in the United States.
The Centers for Disease Control and Prevention did an ecologic study of infant pertussis cases reported between Jan. 1, 2000, and Dec. 31, 2019. Rates were compared for the years before maternal Tdap vaccinations were recommended against the 7-year period after they were implemented.
That study found that in the period before maternal Tdap vaccination, annual pertussis incidence did not change among infants younger than 2 months and increased slightly in infants 6-12 months.
However, during the period after maternal Tdap vaccination had started (2012-2019), pertussis incidence significantly decreased in infants younger than 2 months and was unchanged in infants 6-12 months.
“As with the Australian data, the U.S. data support the overall benefit of the maternal Tdap program and, as with the Australian data, do not suggest that blunting has led to an increase in cases within the first year of life,” Dr. Edwards wrote.
The CDC notes that pertussis cases are rising and outbreaks are happening across the United States.
“On average, about 1,000 infants are hospitalized and typically between 5 and 15 infants die each year in the United States due to pertussis,” the CDC states.
Uptake low despite positive data
Dr. Edwards noted that, despite positive data supporting maternal vaccination to reduce pertussis, uptake rates are low – between 50% and 60% in Australia, the United Kingdom, and the United States. “Active engagement to increase these rates should be implemented.”
Maternal vaccination might also be implemented soon to protect against other diseases including respiratory syncytial virus and group B streptococcal disease after promising study data, she said.
As with pertussis, the potential “blunting” effect will need to be carefully monitored, she said, “as was done in the carefully conducted study of pertussis reported in this issue of Pediatrics.”
One coauthor has received institutional honoraria for participation in advisory groups for Merck Sharpe & Dohme and Pfizer unrelated to this work. Another coauthor was supported by scholarships provided by the Wesfarmers Centre of Vaccines and Infectious Disease at the Telethon Kids Institute. Dr. Edwards reported receiving grants from the CDC and consulting for Bionet, Dynavax, and IBM. She is a member of the data safety and monitoring board for Sanofi, X-4 Pharma, Seqirus, Moderna, Pfizer, Merck, Roche, Novavax, and Brighton Collaboration.
FROM PEDIATRICS
Should children know the severity of their disease? AAP weighs in with report
When children have a serious illness, some families choose not to disclose the severity to them, reasoning that knowing the extent of the illness may take away their hope. Deciding whether to tell children or adolescents about the seriousness of their disease is a complex judgment and can pose legal, ethical, and moral challenges for parents and care providers.
Default should be inclusion
The American Academy of Pediatrics (AAP) recommends in a new clinical report that the default should be to include children in conversations about their illness in a developmentally appropriate way, to the extent parents are comfortable.
The report, written by Sara Taub, MD and Robert Macauley, MD, MDiv, both in the department of pediatrics at Oregon Health & Science University in Portland, on behalf of the AAP Committee on Bioethics, was published online and appears in the October issue of Pediatrics.
“Rather than taking away hope, as some may fear, this approach of openness may create a space for children to ask their questions, share their concerns, and set goals that are appropriate to the circumstances,” the authors wrote in a press release.
The report offers strategies based on ethical, historical, legal, and cultural considerations when discussing what to share with a child or adolescent.
Some of the AAP’s other recommendations include the following:
- If the parents request nondisclosure, the first response should be seeking to understand why they prefer that stance. The care team members should also explain their position to parents.
- If there is no consensus on disclosure, establishing what each party believes is the minimum information that should be shared is important.
- Additional resources to navigate disagreement may be helpful, such as hospital ethics committees, mediators and patient advocates.
- Conversations with the family should be documented in the medical record.
Children may know more than you think
Dr. Taub said that even very young children may know more about their disease than adults believe.
“Without disclosure,” she said, “as children hear the conversations around them, they glean partial information and may weave together stories that are more frightening than reality.”
Sometimes families and the care team disagree on disclosure and for that scenario, the report offers guidance in finding middle ground.
For instance, when pediatricians feel ethically obligated to share information when parents oppose sharing, “pediatricians can reframe the discussion from whether information should be shared with the patient to what information will be communicated, how, and by whom,” the authors said in a press release.
Should you tell 15-year-old paraplegia is likely?
The authors give a case example of a 15-year-old whose spinal tumor likely will lead to paraplegia within weeks. Very few treatment options are available.
The parents ask the care team to avoid any discussions with the child about prognosis, reasoning that the news will be crushing and it’s better to deal with it if or when it happens.
The care team, however, feels compelled to find out about specific activities important to the child that may no longer be feasible with paraplegia.
The parents cite the child’s love of soccer and desire to see the Statue of Liberty. With that information and keeping the parents’ wishes in mind, the team reframes the conversation with the child in terms of goals, acknowledging that mobility may be more difficult in the future.
That conversation leads the child and the family to discuss moving up the trip to New York they had planned.
Guidance where there has been little
Timothy Joos, MD, MPH, a pediatrician who practices at a community health center in Seattle, who was not part of the recommendation team, said he was glad to see the AAP issue advice on a complex topic for which there is little practical guidance.
The authors’ case examples were “heart-tugging,” he said, and will help pediatricians work through their own scenarios.
Dr. Joos agreed with the overall premise that the default should be sharing the information.
“One of the foundations of medicine is truthfulness and openness and if we depart from that, we really have to have a good reason,” Dr. Joos said.
He said that since lying to patients should be nonnegotiable for any physician, it may help to talk with the parents first before answering an inquisitive patient’s questions and then have all parties gather for a discussion.
The authors note that AAP’s clinical reports are written by medical experts and reflect the latest evidence. The reports go through several rounds of peer review before they can be approved by the AAP board of directors.
The authors and Dr. Joos report no relevant financial relationships.
When children have a serious illness, some families choose not to disclose the severity to them, reasoning that knowing the extent of the illness may take away their hope. Deciding whether to tell children or adolescents about the seriousness of their disease is a complex judgment and can pose legal, ethical, and moral challenges for parents and care providers.
Default should be inclusion
The American Academy of Pediatrics (AAP) recommends in a new clinical report that the default should be to include children in conversations about their illness in a developmentally appropriate way, to the extent parents are comfortable.
The report, written by Sara Taub, MD and Robert Macauley, MD, MDiv, both in the department of pediatrics at Oregon Health & Science University in Portland, on behalf of the AAP Committee on Bioethics, was published online and appears in the October issue of Pediatrics.
“Rather than taking away hope, as some may fear, this approach of openness may create a space for children to ask their questions, share their concerns, and set goals that are appropriate to the circumstances,” the authors wrote in a press release.
The report offers strategies based on ethical, historical, legal, and cultural considerations when discussing what to share with a child or adolescent.
Some of the AAP’s other recommendations include the following:
- If the parents request nondisclosure, the first response should be seeking to understand why they prefer that stance. The care team members should also explain their position to parents.
- If there is no consensus on disclosure, establishing what each party believes is the minimum information that should be shared is important.
- Additional resources to navigate disagreement may be helpful, such as hospital ethics committees, mediators and patient advocates.
- Conversations with the family should be documented in the medical record.
Children may know more than you think
Dr. Taub said that even very young children may know more about their disease than adults believe.
“Without disclosure,” she said, “as children hear the conversations around them, they glean partial information and may weave together stories that are more frightening than reality.”
Sometimes families and the care team disagree on disclosure and for that scenario, the report offers guidance in finding middle ground.
For instance, when pediatricians feel ethically obligated to share information when parents oppose sharing, “pediatricians can reframe the discussion from whether information should be shared with the patient to what information will be communicated, how, and by whom,” the authors said in a press release.
Should you tell 15-year-old paraplegia is likely?
The authors give a case example of a 15-year-old whose spinal tumor likely will lead to paraplegia within weeks. Very few treatment options are available.
The parents ask the care team to avoid any discussions with the child about prognosis, reasoning that the news will be crushing and it’s better to deal with it if or when it happens.
The care team, however, feels compelled to find out about specific activities important to the child that may no longer be feasible with paraplegia.
The parents cite the child’s love of soccer and desire to see the Statue of Liberty. With that information and keeping the parents’ wishes in mind, the team reframes the conversation with the child in terms of goals, acknowledging that mobility may be more difficult in the future.
That conversation leads the child and the family to discuss moving up the trip to New York they had planned.
Guidance where there has been little
Timothy Joos, MD, MPH, a pediatrician who practices at a community health center in Seattle, who was not part of the recommendation team, said he was glad to see the AAP issue advice on a complex topic for which there is little practical guidance.
The authors’ case examples were “heart-tugging,” he said, and will help pediatricians work through their own scenarios.
Dr. Joos agreed with the overall premise that the default should be sharing the information.
“One of the foundations of medicine is truthfulness and openness and if we depart from that, we really have to have a good reason,” Dr. Joos said.
He said that since lying to patients should be nonnegotiable for any physician, it may help to talk with the parents first before answering an inquisitive patient’s questions and then have all parties gather for a discussion.
The authors note that AAP’s clinical reports are written by medical experts and reflect the latest evidence. The reports go through several rounds of peer review before they can be approved by the AAP board of directors.
The authors and Dr. Joos report no relevant financial relationships.
When children have a serious illness, some families choose not to disclose the severity to them, reasoning that knowing the extent of the illness may take away their hope. Deciding whether to tell children or adolescents about the seriousness of their disease is a complex judgment and can pose legal, ethical, and moral challenges for parents and care providers.
Default should be inclusion
The American Academy of Pediatrics (AAP) recommends in a new clinical report that the default should be to include children in conversations about their illness in a developmentally appropriate way, to the extent parents are comfortable.
The report, written by Sara Taub, MD and Robert Macauley, MD, MDiv, both in the department of pediatrics at Oregon Health & Science University in Portland, on behalf of the AAP Committee on Bioethics, was published online and appears in the October issue of Pediatrics.
“Rather than taking away hope, as some may fear, this approach of openness may create a space for children to ask their questions, share their concerns, and set goals that are appropriate to the circumstances,” the authors wrote in a press release.
The report offers strategies based on ethical, historical, legal, and cultural considerations when discussing what to share with a child or adolescent.
Some of the AAP’s other recommendations include the following:
- If the parents request nondisclosure, the first response should be seeking to understand why they prefer that stance. The care team members should also explain their position to parents.
- If there is no consensus on disclosure, establishing what each party believes is the minimum information that should be shared is important.
- Additional resources to navigate disagreement may be helpful, such as hospital ethics committees, mediators and patient advocates.
- Conversations with the family should be documented in the medical record.
Children may know more than you think
Dr. Taub said that even very young children may know more about their disease than adults believe.
“Without disclosure,” she said, “as children hear the conversations around them, they glean partial information and may weave together stories that are more frightening than reality.”
Sometimes families and the care team disagree on disclosure and for that scenario, the report offers guidance in finding middle ground.
For instance, when pediatricians feel ethically obligated to share information when parents oppose sharing, “pediatricians can reframe the discussion from whether information should be shared with the patient to what information will be communicated, how, and by whom,” the authors said in a press release.
Should you tell 15-year-old paraplegia is likely?
The authors give a case example of a 15-year-old whose spinal tumor likely will lead to paraplegia within weeks. Very few treatment options are available.
The parents ask the care team to avoid any discussions with the child about prognosis, reasoning that the news will be crushing and it’s better to deal with it if or when it happens.
The care team, however, feels compelled to find out about specific activities important to the child that may no longer be feasible with paraplegia.
The parents cite the child’s love of soccer and desire to see the Statue of Liberty. With that information and keeping the parents’ wishes in mind, the team reframes the conversation with the child in terms of goals, acknowledging that mobility may be more difficult in the future.
That conversation leads the child and the family to discuss moving up the trip to New York they had planned.
Guidance where there has been little
Timothy Joos, MD, MPH, a pediatrician who practices at a community health center in Seattle, who was not part of the recommendation team, said he was glad to see the AAP issue advice on a complex topic for which there is little practical guidance.
The authors’ case examples were “heart-tugging,” he said, and will help pediatricians work through their own scenarios.
Dr. Joos agreed with the overall premise that the default should be sharing the information.
“One of the foundations of medicine is truthfulness and openness and if we depart from that, we really have to have a good reason,” Dr. Joos said.
He said that since lying to patients should be nonnegotiable for any physician, it may help to talk with the parents first before answering an inquisitive patient’s questions and then have all parties gather for a discussion.
The authors note that AAP’s clinical reports are written by medical experts and reflect the latest evidence. The reports go through several rounds of peer review before they can be approved by the AAP board of directors.
The authors and Dr. Joos report no relevant financial relationships.
From Pediatrics
Creatine may improve key long COVID symptoms: Small study
Taking creatine as a supplement for 6 months appears to significantly improve clinical features of post–COVID-19 fatigue syndrome (PVFS or long COVID), a small randomized, placebo-controlled, double-blinded study suggests.
Researchers, led by Jelena Slankamenac, with Applied Bioenergetics Lab, Faculty of Sport and PE, University of Novi Sad, Serbia, published their findings in Food, Science & Nutrition .
“This is the first human study known to the authors that evaluated the efficacy and safety of supplemental creatine for fatigue, tissue bioenergetics, and patient-reported outcomes in patients with post–COVID-19 fatigue syndrome,” the authors write.
They say the findings may be attributed to creatine’s “energy-replenishing and neuroprotective activity.”
Significant reductions in symptoms
Researchers randomized the 12 participants into two groups of 6 each. The creatine group received 4 g creatine monohydrate per day, while the placebo group received the same amount of inulin.
At 3 months, dietary creatine supplements produced a significant reduction in fatigue, compared with baseline values ( P = .04) and significantly improved scores for several long COVID–related symptoms, including loss of taste, breathing difficulties, body aches, headache, and difficulties concentrating) ( P < .05), the researchers report.
Intervention effect sizes were assessed by Cohen statistics, with a d of at least 0.8 indicating a large effect.
Among highlights of the results were that patients reported a significant 77.8% drop in scores for concentration difficulties at the 3-month follow-up (Cohen’s effect, d = 1.19) and no concentration difficulties at the 6-month follow-up (Cohen’s effect, d = 2.46).
Total creatine levels increased in several locations across the brain (as much as 33% for right parietal white matter). No changes in tissue creatine levels were found in the placebo group during the trial.
“Since PVFS is characterized by impaired tissue bioenergetics ..., supplemental creatine might be an effective dietary intervention to uphold brain creatine in post–COVID-19 fatigue syndrome,” the authors write.
The authors add that creatine supplements for long COVID patients could benefit organs beyond the brain as participants saw “a significant drop in lung and body pain after the intervention.”
Unanswered questions
Some experts said the results should be interpreted with caution.
“This research paper is very interesting,” says Nisha Viswanathan, MD, director of the long COVID program at University of California, Los Angeles, “but the limited number of patients makes the results difficult to generalize.”
Dr. Viswanathan, who was not part of the study, pointed out that the patients included in this study had a recent COVID infection (under 3 months).
“Acute COVID infection can take up to 3 months to resolve,” she says. “We define patients with long COVID as those with symptoms lasting greater than 3 months. Therefore, these patients could have had improvements in their fatigue due to the natural course of the illness rather than creatine supplementation.”
Alba Azola, MD, assistant professor in the department of physical medicine and rehabilitation at Johns Hopkins University, Baltimore, said she also was troubled by the window of 3 months for recent COVID infection.
She said she would like to see results for patients who have ongoing symptoms for at least 6 months after infection, especially given creatine supplements’ history in research.
Creatine supplements for other conditions, such as fibromyalgia and chronic fatigue syndrome, have been tested for nearly 2 decades, she pointed out, with conflicting findings, something the authors acknowledge in the paper.
“I think it’s premature to say (creatine) is the key,” she says. She added that the small sample size is important to consider given the heterogeneity of patients with long COVID.
That said, Dr. Azola says, she applauds all efforts to find treatments for long COVID, especially randomized, controlled studies like this one.
No major side effects
No major side effects were reported for either intervention, except for transient mild nausea reported by one patient after taking creatine.
Compliance with the intervention was 90.6% ± 3.5% in the creatine group and 95.3% ± 5.0% in the control group (P = .04).
Participants were eligible for inclusion if they were 18-65 years old, had a positive COVID test within the last 3 months (documented by a valid polymerase chain reaction [PCR] or antigen test performed in a COVID-19–certified lab); had moderate to severe fatigue; and at least one additional COVID-related symptom, including loss of taste or smell, breathing trouble, lung pain, body aches, headaches, or difficulties concentrating.
The authors acknowledge that they selected a sample of young to middle-aged adults experiencing moderate long COVID symptoms, and it’s unknown whether creatine is equally effective in other PVFS populations, such as elderly people, children, or patients with less or more severe disease.
Senior author Dr. Sergei Ostojic serves as a member of the Scientific Advisory Board on creatine in health and medicine (AlzChem LLC). He co-owns a patent for “Supplements Based on Liquid Creatine” at the European Patent Office. He has received research support related to creatine during the past 36 months from the Serbian Ministry of Education, Science, and Technological Development; Provincial Secretariat for Higher Education and Scientific Research; Alzchem GmbH; ThermoLife International; and Hueston Hennigan LLP. He does not own stocks and shares in any organization. Other authors declare no known relevant financial interests. Dr. Viswanathan and Dr. Azola report no relevant financial relationships.
Taking creatine as a supplement for 6 months appears to significantly improve clinical features of post–COVID-19 fatigue syndrome (PVFS or long COVID), a small randomized, placebo-controlled, double-blinded study suggests.
Researchers, led by Jelena Slankamenac, with Applied Bioenergetics Lab, Faculty of Sport and PE, University of Novi Sad, Serbia, published their findings in Food, Science & Nutrition .
“This is the first human study known to the authors that evaluated the efficacy and safety of supplemental creatine for fatigue, tissue bioenergetics, and patient-reported outcomes in patients with post–COVID-19 fatigue syndrome,” the authors write.
They say the findings may be attributed to creatine’s “energy-replenishing and neuroprotective activity.”
Significant reductions in symptoms
Researchers randomized the 12 participants into two groups of 6 each. The creatine group received 4 g creatine monohydrate per day, while the placebo group received the same amount of inulin.
At 3 months, dietary creatine supplements produced a significant reduction in fatigue, compared with baseline values ( P = .04) and significantly improved scores for several long COVID–related symptoms, including loss of taste, breathing difficulties, body aches, headache, and difficulties concentrating) ( P < .05), the researchers report.
Intervention effect sizes were assessed by Cohen statistics, with a d of at least 0.8 indicating a large effect.
Among highlights of the results were that patients reported a significant 77.8% drop in scores for concentration difficulties at the 3-month follow-up (Cohen’s effect, d = 1.19) and no concentration difficulties at the 6-month follow-up (Cohen’s effect, d = 2.46).
Total creatine levels increased in several locations across the brain (as much as 33% for right parietal white matter). No changes in tissue creatine levels were found in the placebo group during the trial.
“Since PVFS is characterized by impaired tissue bioenergetics ..., supplemental creatine might be an effective dietary intervention to uphold brain creatine in post–COVID-19 fatigue syndrome,” the authors write.
The authors add that creatine supplements for long COVID patients could benefit organs beyond the brain as participants saw “a significant drop in lung and body pain after the intervention.”
Unanswered questions
Some experts said the results should be interpreted with caution.
“This research paper is very interesting,” says Nisha Viswanathan, MD, director of the long COVID program at University of California, Los Angeles, “but the limited number of patients makes the results difficult to generalize.”
Dr. Viswanathan, who was not part of the study, pointed out that the patients included in this study had a recent COVID infection (under 3 months).
“Acute COVID infection can take up to 3 months to resolve,” she says. “We define patients with long COVID as those with symptoms lasting greater than 3 months. Therefore, these patients could have had improvements in their fatigue due to the natural course of the illness rather than creatine supplementation.”
Alba Azola, MD, assistant professor in the department of physical medicine and rehabilitation at Johns Hopkins University, Baltimore, said she also was troubled by the window of 3 months for recent COVID infection.
She said she would like to see results for patients who have ongoing symptoms for at least 6 months after infection, especially given creatine supplements’ history in research.
Creatine supplements for other conditions, such as fibromyalgia and chronic fatigue syndrome, have been tested for nearly 2 decades, she pointed out, with conflicting findings, something the authors acknowledge in the paper.
“I think it’s premature to say (creatine) is the key,” she says. She added that the small sample size is important to consider given the heterogeneity of patients with long COVID.
That said, Dr. Azola says, she applauds all efforts to find treatments for long COVID, especially randomized, controlled studies like this one.
No major side effects
No major side effects were reported for either intervention, except for transient mild nausea reported by one patient after taking creatine.
Compliance with the intervention was 90.6% ± 3.5% in the creatine group and 95.3% ± 5.0% in the control group (P = .04).
Participants were eligible for inclusion if they were 18-65 years old, had a positive COVID test within the last 3 months (documented by a valid polymerase chain reaction [PCR] or antigen test performed in a COVID-19–certified lab); had moderate to severe fatigue; and at least one additional COVID-related symptom, including loss of taste or smell, breathing trouble, lung pain, body aches, headaches, or difficulties concentrating.
The authors acknowledge that they selected a sample of young to middle-aged adults experiencing moderate long COVID symptoms, and it’s unknown whether creatine is equally effective in other PVFS populations, such as elderly people, children, or patients with less or more severe disease.
Senior author Dr. Sergei Ostojic serves as a member of the Scientific Advisory Board on creatine in health and medicine (AlzChem LLC). He co-owns a patent for “Supplements Based on Liquid Creatine” at the European Patent Office. He has received research support related to creatine during the past 36 months from the Serbian Ministry of Education, Science, and Technological Development; Provincial Secretariat for Higher Education and Scientific Research; Alzchem GmbH; ThermoLife International; and Hueston Hennigan LLP. He does not own stocks and shares in any organization. Other authors declare no known relevant financial interests. Dr. Viswanathan and Dr. Azola report no relevant financial relationships.
Taking creatine as a supplement for 6 months appears to significantly improve clinical features of post–COVID-19 fatigue syndrome (PVFS or long COVID), a small randomized, placebo-controlled, double-blinded study suggests.
Researchers, led by Jelena Slankamenac, with Applied Bioenergetics Lab, Faculty of Sport and PE, University of Novi Sad, Serbia, published their findings in Food, Science & Nutrition .
“This is the first human study known to the authors that evaluated the efficacy and safety of supplemental creatine for fatigue, tissue bioenergetics, and patient-reported outcomes in patients with post–COVID-19 fatigue syndrome,” the authors write.
They say the findings may be attributed to creatine’s “energy-replenishing and neuroprotective activity.”
Significant reductions in symptoms
Researchers randomized the 12 participants into two groups of 6 each. The creatine group received 4 g creatine monohydrate per day, while the placebo group received the same amount of inulin.
At 3 months, dietary creatine supplements produced a significant reduction in fatigue, compared with baseline values ( P = .04) and significantly improved scores for several long COVID–related symptoms, including loss of taste, breathing difficulties, body aches, headache, and difficulties concentrating) ( P < .05), the researchers report.
Intervention effect sizes were assessed by Cohen statistics, with a d of at least 0.8 indicating a large effect.
Among highlights of the results were that patients reported a significant 77.8% drop in scores for concentration difficulties at the 3-month follow-up (Cohen’s effect, d = 1.19) and no concentration difficulties at the 6-month follow-up (Cohen’s effect, d = 2.46).
Total creatine levels increased in several locations across the brain (as much as 33% for right parietal white matter). No changes in tissue creatine levels were found in the placebo group during the trial.
“Since PVFS is characterized by impaired tissue bioenergetics ..., supplemental creatine might be an effective dietary intervention to uphold brain creatine in post–COVID-19 fatigue syndrome,” the authors write.
The authors add that creatine supplements for long COVID patients could benefit organs beyond the brain as participants saw “a significant drop in lung and body pain after the intervention.”
Unanswered questions
Some experts said the results should be interpreted with caution.
“This research paper is very interesting,” says Nisha Viswanathan, MD, director of the long COVID program at University of California, Los Angeles, “but the limited number of patients makes the results difficult to generalize.”
Dr. Viswanathan, who was not part of the study, pointed out that the patients included in this study had a recent COVID infection (under 3 months).
“Acute COVID infection can take up to 3 months to resolve,” she says. “We define patients with long COVID as those with symptoms lasting greater than 3 months. Therefore, these patients could have had improvements in their fatigue due to the natural course of the illness rather than creatine supplementation.”
Alba Azola, MD, assistant professor in the department of physical medicine and rehabilitation at Johns Hopkins University, Baltimore, said she also was troubled by the window of 3 months for recent COVID infection.
She said she would like to see results for patients who have ongoing symptoms for at least 6 months after infection, especially given creatine supplements’ history in research.
Creatine supplements for other conditions, such as fibromyalgia and chronic fatigue syndrome, have been tested for nearly 2 decades, she pointed out, with conflicting findings, something the authors acknowledge in the paper.
“I think it’s premature to say (creatine) is the key,” she says. She added that the small sample size is important to consider given the heterogeneity of patients with long COVID.
That said, Dr. Azola says, she applauds all efforts to find treatments for long COVID, especially randomized, controlled studies like this one.
No major side effects
No major side effects were reported for either intervention, except for transient mild nausea reported by one patient after taking creatine.
Compliance with the intervention was 90.6% ± 3.5% in the creatine group and 95.3% ± 5.0% in the control group (P = .04).
Participants were eligible for inclusion if they were 18-65 years old, had a positive COVID test within the last 3 months (documented by a valid polymerase chain reaction [PCR] or antigen test performed in a COVID-19–certified lab); had moderate to severe fatigue; and at least one additional COVID-related symptom, including loss of taste or smell, breathing trouble, lung pain, body aches, headaches, or difficulties concentrating.
The authors acknowledge that they selected a sample of young to middle-aged adults experiencing moderate long COVID symptoms, and it’s unknown whether creatine is equally effective in other PVFS populations, such as elderly people, children, or patients with less or more severe disease.
Senior author Dr. Sergei Ostojic serves as a member of the Scientific Advisory Board on creatine in health and medicine (AlzChem LLC). He co-owns a patent for “Supplements Based on Liquid Creatine” at the European Patent Office. He has received research support related to creatine during the past 36 months from the Serbian Ministry of Education, Science, and Technological Development; Provincial Secretariat for Higher Education and Scientific Research; Alzchem GmbH; ThermoLife International; and Hueston Hennigan LLP. He does not own stocks and shares in any organization. Other authors declare no known relevant financial interests. Dr. Viswanathan and Dr. Azola report no relevant financial relationships.
FROM FOOD, SCIENCE & NUTRITION
Minimally invasive surfactant shows some benefit in infants’ first 2 years
Results of the OPTIMIST follow-up study were published online in JAMA.
Researchers, led by Peter A. Dargaville, MD, department of paediatrics, Royal Hobart (Australia) Hospital, found that MIST, which involves administering surfactant via a thin catheter, compared with sham treatment, did not reduce the incidence of death or neurodevelopmental disability (NDD) by 2 years of age.
However, infants who received MIST had lower rates of poor respiratory outcomes during those first 2 years of life.
Study spanned 11 countries
The study was conducted in 33 tertiary neonatal intensive care units (NICUs) in 11 countries, including Australia, Canada, Israel, New Zealand, Qatar, Singapore, Slovenia, the Netherlands, Turkey, the United Kingdom, and the United States.
It included 486 infants 25-28 weeks old supported with CPAP; 453 had follow-up data available and data on the key secondary outcome were available for 434 infants.
The sham treatment consisted of only transient repositioning without airway instruments. Treating clinicians, outcome assessors, and parents were blinded to group status.
No significant difference in deaths, NDD
Death or NDD occurred in 36.3% of the patients in the MIST group and 36.1% in the control group (risk difference, 0%; 95% confidence interval, −7.6% to 7.7%; relative risk, 1.0; 95% confidence interval, 0.81-1.24).
Secondary respiratory outcomes were better in the MIST group:
- Hospitalization with respiratory illness occurred in 25.1% in the MIST group versus 38.2% in the control group (RR, 0.66; 95% CI, 0.54-0.81).
- Parent-reported wheezing or breathing difficulty occurred in 40.6% in the MIST group versus 53.6% in controls (RR, 0.76; 95% CI, 0.63-0.90).
- Asthma diagnosed by a physician was reported in 4.4% and 11.9% of MIST and control-group infants, respectively.
- Reported use of inhaled relievers (beta2 agonists) was 23.9% in the MIST group versus 38.7% in controls.
The previous study of early outcomes of deaths or bronchopulmonary dysplasia (BPD; chronic lung injury in preterm infants) was published by the same group of researchers in 2021.
Important benefit for respiratory health
Suhas G. Kallapur, MD, chief of the divisions of neonatology and developmental biology at University of California, Los Angeles, who was not part of either study, said: “This is one of the largest studies to date examining whether the MIST procedure for surfactant is beneficial in preterm babies born at 25-28 weeks’ gestation.”
Overall, when considering the 2021 and 2023 studies together, it appears that the MIST therapy has important benefits for respiratory health during a NICU stay and in early infancy, even though the primary outcome of death or NDD was not different between the treatment and control groups, Dr. Kallapur said.
“The slight (nonsignificant) increase in deaths in the MIST group was confined to the more immature babies – 25-26 weeks’ gestation at birth,” he pointed out. “In the bigger and more mature babies – 27-28 week gestation infants – the benefits of MIST therapy occurred without any increase in mortality, suggesting that this group of babies may be the group that stands to benefit most from this therapy.”
Dr. Kallapur said new data in the developmental origins of health and disease “now show that the trajectory of respiratory health in infancy is an important determinant of respiratory health into adulthood and older age.”
Therefore, the finding of benefit to respiratory health is particularly important, he said.
He noted that MIST or similar therapy is already in use in many NICUs throughout the world and that those already using it will likely feel vindicated by this study.
“Neonatologists who were on the sidelines will likely see these results – especially childhood respiratory outcomes – as a reason to initiate this procedure in all but the most immature preterm infants,” Dr. Kallapur says.
Dr. Dargaville reports personal fees from AbbVie and Chiesi Farmaceutici and provision of surfactant at reduced cost and support for conference travel from Chiesi Farmaceutici during the conduct of the study; in addition, Dr. Dargaville has been issued a patent for a catheter design. One coauthor reports grants from Chiesi Farmaceutici during the conduct of the study. Another coauthor reports serving as chief investigator for OPTI-SURF, an observational study on United Kingdom neonatal surfactant use in respiratory distress syndrome funded by Chiesi UK outside the submitted work. A third coauthor reports personal fees from Chiesi Farmaceutici outside the submitted work. This study was funded by grants from the Royal Hobart Hospital Research Foundation and the Australian National Health and Medical Research Council. Exogenous surfactant was provided at reduced cost by Chiesi Farmaceutici. Dr. Kallapur has no relevant financial relationships.
Results of the OPTIMIST follow-up study were published online in JAMA.
Researchers, led by Peter A. Dargaville, MD, department of paediatrics, Royal Hobart (Australia) Hospital, found that MIST, which involves administering surfactant via a thin catheter, compared with sham treatment, did not reduce the incidence of death or neurodevelopmental disability (NDD) by 2 years of age.
However, infants who received MIST had lower rates of poor respiratory outcomes during those first 2 years of life.
Study spanned 11 countries
The study was conducted in 33 tertiary neonatal intensive care units (NICUs) in 11 countries, including Australia, Canada, Israel, New Zealand, Qatar, Singapore, Slovenia, the Netherlands, Turkey, the United Kingdom, and the United States.
It included 486 infants 25-28 weeks old supported with CPAP; 453 had follow-up data available and data on the key secondary outcome were available for 434 infants.
The sham treatment consisted of only transient repositioning without airway instruments. Treating clinicians, outcome assessors, and parents were blinded to group status.
No significant difference in deaths, NDD
Death or NDD occurred in 36.3% of the patients in the MIST group and 36.1% in the control group (risk difference, 0%; 95% confidence interval, −7.6% to 7.7%; relative risk, 1.0; 95% confidence interval, 0.81-1.24).
Secondary respiratory outcomes were better in the MIST group:
- Hospitalization with respiratory illness occurred in 25.1% in the MIST group versus 38.2% in the control group (RR, 0.66; 95% CI, 0.54-0.81).
- Parent-reported wheezing or breathing difficulty occurred in 40.6% in the MIST group versus 53.6% in controls (RR, 0.76; 95% CI, 0.63-0.90).
- Asthma diagnosed by a physician was reported in 4.4% and 11.9% of MIST and control-group infants, respectively.
- Reported use of inhaled relievers (beta2 agonists) was 23.9% in the MIST group versus 38.7% in controls.
The previous study of early outcomes of deaths or bronchopulmonary dysplasia (BPD; chronic lung injury in preterm infants) was published by the same group of researchers in 2021.
Important benefit for respiratory health
Suhas G. Kallapur, MD, chief of the divisions of neonatology and developmental biology at University of California, Los Angeles, who was not part of either study, said: “This is one of the largest studies to date examining whether the MIST procedure for surfactant is beneficial in preterm babies born at 25-28 weeks’ gestation.”
Overall, when considering the 2021 and 2023 studies together, it appears that the MIST therapy has important benefits for respiratory health during a NICU stay and in early infancy, even though the primary outcome of death or NDD was not different between the treatment and control groups, Dr. Kallapur said.
“The slight (nonsignificant) increase in deaths in the MIST group was confined to the more immature babies – 25-26 weeks’ gestation at birth,” he pointed out. “In the bigger and more mature babies – 27-28 week gestation infants – the benefits of MIST therapy occurred without any increase in mortality, suggesting that this group of babies may be the group that stands to benefit most from this therapy.”
Dr. Kallapur said new data in the developmental origins of health and disease “now show that the trajectory of respiratory health in infancy is an important determinant of respiratory health into adulthood and older age.”
Therefore, the finding of benefit to respiratory health is particularly important, he said.
He noted that MIST or similar therapy is already in use in many NICUs throughout the world and that those already using it will likely feel vindicated by this study.
“Neonatologists who were on the sidelines will likely see these results – especially childhood respiratory outcomes – as a reason to initiate this procedure in all but the most immature preterm infants,” Dr. Kallapur says.
Dr. Dargaville reports personal fees from AbbVie and Chiesi Farmaceutici and provision of surfactant at reduced cost and support for conference travel from Chiesi Farmaceutici during the conduct of the study; in addition, Dr. Dargaville has been issued a patent for a catheter design. One coauthor reports grants from Chiesi Farmaceutici during the conduct of the study. Another coauthor reports serving as chief investigator for OPTI-SURF, an observational study on United Kingdom neonatal surfactant use in respiratory distress syndrome funded by Chiesi UK outside the submitted work. A third coauthor reports personal fees from Chiesi Farmaceutici outside the submitted work. This study was funded by grants from the Royal Hobart Hospital Research Foundation and the Australian National Health and Medical Research Council. Exogenous surfactant was provided at reduced cost by Chiesi Farmaceutici. Dr. Kallapur has no relevant financial relationships.
Results of the OPTIMIST follow-up study were published online in JAMA.
Researchers, led by Peter A. Dargaville, MD, department of paediatrics, Royal Hobart (Australia) Hospital, found that MIST, which involves administering surfactant via a thin catheter, compared with sham treatment, did not reduce the incidence of death or neurodevelopmental disability (NDD) by 2 years of age.
However, infants who received MIST had lower rates of poor respiratory outcomes during those first 2 years of life.
Study spanned 11 countries
The study was conducted in 33 tertiary neonatal intensive care units (NICUs) in 11 countries, including Australia, Canada, Israel, New Zealand, Qatar, Singapore, Slovenia, the Netherlands, Turkey, the United Kingdom, and the United States.
It included 486 infants 25-28 weeks old supported with CPAP; 453 had follow-up data available and data on the key secondary outcome were available for 434 infants.
The sham treatment consisted of only transient repositioning without airway instruments. Treating clinicians, outcome assessors, and parents were blinded to group status.
No significant difference in deaths, NDD
Death or NDD occurred in 36.3% of the patients in the MIST group and 36.1% in the control group (risk difference, 0%; 95% confidence interval, −7.6% to 7.7%; relative risk, 1.0; 95% confidence interval, 0.81-1.24).
Secondary respiratory outcomes were better in the MIST group:
- Hospitalization with respiratory illness occurred in 25.1% in the MIST group versus 38.2% in the control group (RR, 0.66; 95% CI, 0.54-0.81).
- Parent-reported wheezing or breathing difficulty occurred in 40.6% in the MIST group versus 53.6% in controls (RR, 0.76; 95% CI, 0.63-0.90).
- Asthma diagnosed by a physician was reported in 4.4% and 11.9% of MIST and control-group infants, respectively.
- Reported use of inhaled relievers (beta2 agonists) was 23.9% in the MIST group versus 38.7% in controls.
The previous study of early outcomes of deaths or bronchopulmonary dysplasia (BPD; chronic lung injury in preterm infants) was published by the same group of researchers in 2021.
Important benefit for respiratory health
Suhas G. Kallapur, MD, chief of the divisions of neonatology and developmental biology at University of California, Los Angeles, who was not part of either study, said: “This is one of the largest studies to date examining whether the MIST procedure for surfactant is beneficial in preterm babies born at 25-28 weeks’ gestation.”
Overall, when considering the 2021 and 2023 studies together, it appears that the MIST therapy has important benefits for respiratory health during a NICU stay and in early infancy, even though the primary outcome of death or NDD was not different between the treatment and control groups, Dr. Kallapur said.
“The slight (nonsignificant) increase in deaths in the MIST group was confined to the more immature babies – 25-26 weeks’ gestation at birth,” he pointed out. “In the bigger and more mature babies – 27-28 week gestation infants – the benefits of MIST therapy occurred without any increase in mortality, suggesting that this group of babies may be the group that stands to benefit most from this therapy.”
Dr. Kallapur said new data in the developmental origins of health and disease “now show that the trajectory of respiratory health in infancy is an important determinant of respiratory health into adulthood and older age.”
Therefore, the finding of benefit to respiratory health is particularly important, he said.
He noted that MIST or similar therapy is already in use in many NICUs throughout the world and that those already using it will likely feel vindicated by this study.
“Neonatologists who were on the sidelines will likely see these results – especially childhood respiratory outcomes – as a reason to initiate this procedure in all but the most immature preterm infants,” Dr. Kallapur says.
Dr. Dargaville reports personal fees from AbbVie and Chiesi Farmaceutici and provision of surfactant at reduced cost and support for conference travel from Chiesi Farmaceutici during the conduct of the study; in addition, Dr. Dargaville has been issued a patent for a catheter design. One coauthor reports grants from Chiesi Farmaceutici during the conduct of the study. Another coauthor reports serving as chief investigator for OPTI-SURF, an observational study on United Kingdom neonatal surfactant use in respiratory distress syndrome funded by Chiesi UK outside the submitted work. A third coauthor reports personal fees from Chiesi Farmaceutici outside the submitted work. This study was funded by grants from the Royal Hobart Hospital Research Foundation and the Australian National Health and Medical Research Council. Exogenous surfactant was provided at reduced cost by Chiesi Farmaceutici. Dr. Kallapur has no relevant financial relationships.
FROM JAMA
Hydroxychloroquine blood level ‘sweet spot’ may maximize efficacy in lupus
A blood-level reference range of 750-1,200 ng/mL of hydroxychloroquine (HCQ) has been linked with 71% lower odds of active lupus, new research suggests.
Researchers, led by Shivani Garg, MD, assistant professor of rheumatology at the University of Wisconsin–Madison, also found that maintaining levels within that range lowered the odds for flares by 26% over 9 months of follow-up.
The findings, published in Arthritis Care & Research, could help clinicians personalize HCQ doses to maximize efficacy for each patient.
HCQ levels in whole blood and the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) were measured during a baseline visit and again during a routine follow-up visit.
Among 158 baseline patient visits, 19% of the patients had active lupus. Researchers longitudinally followed 42 patients using convenience sampling, and among those patients, 7 (17%) had flares at the follow-up visit.
Michelle Petri, MD, MPH, director of the Johns Hopkins Lupus Center in Baltimore, called the findings that suggest upper and lower efficacy and safety boundaries “very important.”
The findings highlight that guidelines for dosing don’t match efficacy needs, said Dr. Petri, who was not involved with the study.
“HCQ dosing has been under threat by guidelines insisting that the dose should be < 5 mg/kg even though this does not correlate with efficacy,” she said. “Basically, if we dose too low, the patient loses efficacy. If we dose too high, the risk of retinopathy increases, so this paper hones down the sweet spot.”
A 2014 study identified a higher eye toxicity risk with HCQ doses > 5 mg/kg per day, and the American Academy of Ophthalmology followed with guidelines for HCQ retinopathy screening that recommended reducing HCQ to ≤ 5 mg/kg per day.
Dr. Petri said that the range Dr. Garg and colleagues identified corroborates findings in one of her team’s studies.
That paper showed that thrombotic events dropped by 69% in patients with average HCQ blood levels ≥ 1,068 ng/mL vs. those with levels < 648 ng/mL (relative risk, 0.31; 95% confidence interval, 0.11-0.86; P = .024).
Dr. Garg and colleagues write that current lupus treatment guidelines do not universally recommend blood level monitoring for HCQ “as different cut-points have been used to define therapeutic HCQ blood levels and an effective range of HCQ levels with upper and lower bounds for efficacy has not been extensively examined.”
When to start checking levels
Blood levels of HCQ can be checked for any patient, although 1-3 months after starting the medication may be best to get steady levels, Dr. Garg told this news organization.
Dr. Petri said that she recommends HCQ whole blood levels be checked routinely for maximum dosing efficacy “but also to identify patients who are missing so many doses that they are subtherapeutic.”
She noted that nonadherence is a major issue among patients with systemic lupus erythematosus, especially among those who are younger and newly diagnosed.
Dr. Garg and Dr. Petri both said that insurance does not automatically cover the costs of checking HCQ levels in the blood, which has been a consistent frustration in the field.
“Having more data validates the reason to do it,” Dr. Garg said.
She added that “HCQ blood levels are still not done routinely in all patients, and at times the test needs to be sent to outside laboratories.”
Importance for patients with CKD
Many patient factors can affect how the body absorbs HCQ, Dr. Garg said, so finding the right level that is safe and maximizes benefit individually is important.
The findings are particularly important for patients with chronic kidney disease (CKD) of stage 3 or higher, Dr. Garg said.
The authors write that because kidneys clear more than half of all HCQ, impaired kidney function could boost HCQ blood levels, risking toxicity.
“Our study found a sixfold higher odds of having supratherapeutic HCQ blood levels in patients with CKD stage ≥ 3,” they write.
Dr. Garg added that if blood levels cannot be analyzed in all patients, they could be prioritized in patients with CKD stage 3 or above because these patients are at “higher risk of being underdosed with arbitrary reductions in HCQ doses and carry higher risk of toxicity if HCQ doses are not adjusted.”
More research will uncover other high-risk groups who would benefit most from close monitoring of HCQ blood levels, she said.
The study was supported by an award from the University of Wisconsin–Madison, and by an award to the institution from the National Institutes of Health National Center for Advancing Translational Sciences. Dr. Garg and coauthors as well as Dr. Petri report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
A blood-level reference range of 750-1,200 ng/mL of hydroxychloroquine (HCQ) has been linked with 71% lower odds of active lupus, new research suggests.
Researchers, led by Shivani Garg, MD, assistant professor of rheumatology at the University of Wisconsin–Madison, also found that maintaining levels within that range lowered the odds for flares by 26% over 9 months of follow-up.
The findings, published in Arthritis Care & Research, could help clinicians personalize HCQ doses to maximize efficacy for each patient.
HCQ levels in whole blood and the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) were measured during a baseline visit and again during a routine follow-up visit.
Among 158 baseline patient visits, 19% of the patients had active lupus. Researchers longitudinally followed 42 patients using convenience sampling, and among those patients, 7 (17%) had flares at the follow-up visit.
Michelle Petri, MD, MPH, director of the Johns Hopkins Lupus Center in Baltimore, called the findings that suggest upper and lower efficacy and safety boundaries “very important.”
The findings highlight that guidelines for dosing don’t match efficacy needs, said Dr. Petri, who was not involved with the study.
“HCQ dosing has been under threat by guidelines insisting that the dose should be < 5 mg/kg even though this does not correlate with efficacy,” she said. “Basically, if we dose too low, the patient loses efficacy. If we dose too high, the risk of retinopathy increases, so this paper hones down the sweet spot.”
A 2014 study identified a higher eye toxicity risk with HCQ doses > 5 mg/kg per day, and the American Academy of Ophthalmology followed with guidelines for HCQ retinopathy screening that recommended reducing HCQ to ≤ 5 mg/kg per day.
Dr. Petri said that the range Dr. Garg and colleagues identified corroborates findings in one of her team’s studies.
That paper showed that thrombotic events dropped by 69% in patients with average HCQ blood levels ≥ 1,068 ng/mL vs. those with levels < 648 ng/mL (relative risk, 0.31; 95% confidence interval, 0.11-0.86; P = .024).
Dr. Garg and colleagues write that current lupus treatment guidelines do not universally recommend blood level monitoring for HCQ “as different cut-points have been used to define therapeutic HCQ blood levels and an effective range of HCQ levels with upper and lower bounds for efficacy has not been extensively examined.”
When to start checking levels
Blood levels of HCQ can be checked for any patient, although 1-3 months after starting the medication may be best to get steady levels, Dr. Garg told this news organization.
Dr. Petri said that she recommends HCQ whole blood levels be checked routinely for maximum dosing efficacy “but also to identify patients who are missing so many doses that they are subtherapeutic.”
She noted that nonadherence is a major issue among patients with systemic lupus erythematosus, especially among those who are younger and newly diagnosed.
Dr. Garg and Dr. Petri both said that insurance does not automatically cover the costs of checking HCQ levels in the blood, which has been a consistent frustration in the field.
“Having more data validates the reason to do it,” Dr. Garg said.
She added that “HCQ blood levels are still not done routinely in all patients, and at times the test needs to be sent to outside laboratories.”
Importance for patients with CKD
Many patient factors can affect how the body absorbs HCQ, Dr. Garg said, so finding the right level that is safe and maximizes benefit individually is important.
The findings are particularly important for patients with chronic kidney disease (CKD) of stage 3 or higher, Dr. Garg said.
The authors write that because kidneys clear more than half of all HCQ, impaired kidney function could boost HCQ blood levels, risking toxicity.
“Our study found a sixfold higher odds of having supratherapeutic HCQ blood levels in patients with CKD stage ≥ 3,” they write.
Dr. Garg added that if blood levels cannot be analyzed in all patients, they could be prioritized in patients with CKD stage 3 or above because these patients are at “higher risk of being underdosed with arbitrary reductions in HCQ doses and carry higher risk of toxicity if HCQ doses are not adjusted.”
More research will uncover other high-risk groups who would benefit most from close monitoring of HCQ blood levels, she said.
The study was supported by an award from the University of Wisconsin–Madison, and by an award to the institution from the National Institutes of Health National Center for Advancing Translational Sciences. Dr. Garg and coauthors as well as Dr. Petri report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
A blood-level reference range of 750-1,200 ng/mL of hydroxychloroquine (HCQ) has been linked with 71% lower odds of active lupus, new research suggests.
Researchers, led by Shivani Garg, MD, assistant professor of rheumatology at the University of Wisconsin–Madison, also found that maintaining levels within that range lowered the odds for flares by 26% over 9 months of follow-up.
The findings, published in Arthritis Care & Research, could help clinicians personalize HCQ doses to maximize efficacy for each patient.
HCQ levels in whole blood and the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) were measured during a baseline visit and again during a routine follow-up visit.
Among 158 baseline patient visits, 19% of the patients had active lupus. Researchers longitudinally followed 42 patients using convenience sampling, and among those patients, 7 (17%) had flares at the follow-up visit.
Michelle Petri, MD, MPH, director of the Johns Hopkins Lupus Center in Baltimore, called the findings that suggest upper and lower efficacy and safety boundaries “very important.”
The findings highlight that guidelines for dosing don’t match efficacy needs, said Dr. Petri, who was not involved with the study.
“HCQ dosing has been under threat by guidelines insisting that the dose should be < 5 mg/kg even though this does not correlate with efficacy,” she said. “Basically, if we dose too low, the patient loses efficacy. If we dose too high, the risk of retinopathy increases, so this paper hones down the sweet spot.”
A 2014 study identified a higher eye toxicity risk with HCQ doses > 5 mg/kg per day, and the American Academy of Ophthalmology followed with guidelines for HCQ retinopathy screening that recommended reducing HCQ to ≤ 5 mg/kg per day.
Dr. Petri said that the range Dr. Garg and colleagues identified corroborates findings in one of her team’s studies.
That paper showed that thrombotic events dropped by 69% in patients with average HCQ blood levels ≥ 1,068 ng/mL vs. those with levels < 648 ng/mL (relative risk, 0.31; 95% confidence interval, 0.11-0.86; P = .024).
Dr. Garg and colleagues write that current lupus treatment guidelines do not universally recommend blood level monitoring for HCQ “as different cut-points have been used to define therapeutic HCQ blood levels and an effective range of HCQ levels with upper and lower bounds for efficacy has not been extensively examined.”
When to start checking levels
Blood levels of HCQ can be checked for any patient, although 1-3 months after starting the medication may be best to get steady levels, Dr. Garg told this news organization.
Dr. Petri said that she recommends HCQ whole blood levels be checked routinely for maximum dosing efficacy “but also to identify patients who are missing so many doses that they are subtherapeutic.”
She noted that nonadherence is a major issue among patients with systemic lupus erythematosus, especially among those who are younger and newly diagnosed.
Dr. Garg and Dr. Petri both said that insurance does not automatically cover the costs of checking HCQ levels in the blood, which has been a consistent frustration in the field.
“Having more data validates the reason to do it,” Dr. Garg said.
She added that “HCQ blood levels are still not done routinely in all patients, and at times the test needs to be sent to outside laboratories.”
Importance for patients with CKD
Many patient factors can affect how the body absorbs HCQ, Dr. Garg said, so finding the right level that is safe and maximizes benefit individually is important.
The findings are particularly important for patients with chronic kidney disease (CKD) of stage 3 or higher, Dr. Garg said.
The authors write that because kidneys clear more than half of all HCQ, impaired kidney function could boost HCQ blood levels, risking toxicity.
“Our study found a sixfold higher odds of having supratherapeutic HCQ blood levels in patients with CKD stage ≥ 3,” they write.
Dr. Garg added that if blood levels cannot be analyzed in all patients, they could be prioritized in patients with CKD stage 3 or above because these patients are at “higher risk of being underdosed with arbitrary reductions in HCQ doses and carry higher risk of toxicity if HCQ doses are not adjusted.”
More research will uncover other high-risk groups who would benefit most from close monitoring of HCQ blood levels, she said.
The study was supported by an award from the University of Wisconsin–Madison, and by an award to the institution from the National Institutes of Health National Center for Advancing Translational Sciences. Dr. Garg and coauthors as well as Dr. Petri report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
FROM ARTHRITIS CARE & RESEARCH
Sepsis too often neglected in hospitals
according to a recent survey by the Centers for Disease Control and Prevention.
For the hospitals that do have sepsis teams, only 55% of them report that their team leaders get dedicated time to manage their sepsis programs.
“One in three people who dies in a hospital has sepsis during that hospitalization,” CDC Director Mandy Cohen, MD, MPH, noted in a statement. “That’s why CDC is calling on all U.S. hospitals to have a sepsis program and raise the bar on sepsis care by incorporating seven core elements.”
The sepsis seven
- Leadership: Dedicating the necessary human, financial, and information technology resources.
- Accountability: Appointing a leader responsible for program outcomes and setting concrete goals.
- Multiprofessional: Engaging key partners throughout the organization.
- Action: Implementing structures and processes to improve the identification, management, and recovery from sepsis.
- Tracking: Measuring sepsis epidemiology, outcomes, and progress toward program goals and the impact of sepsis initiatives.
- Reporting: Providing usable information on sepsis treatment and outcomes to relevant partners.
- Education: Providing sepsis education to health care professionals during onboarding and annually.
Craig Weinert, MD, MPH, a pulmonologist and critical care physician and professor of medicine at the University of Minnesota, Minneapolis, says the point the CDC is making with the announcement is that when these sepsis programs have been implemented at hospitals, they have been successful at reducing mortality. And now, the agency is urging all hospitals to implement them and support them properly.
“It’s not asking hospitals to develop new, innovative kinds of sepsis programs. This is not about new drugs or new antibiotics or new devices,” Dr. Weinert says. “This is about having hospitals dedicate organizational resources to implementing sepsis programs.”
The CDC’s announcement is aimed toward hospital administrators, Dr. Weinert adds. The agency is making the case that sepsis needs more funding in hospitals that either don’t have the programs or aren’t supporting them with dedicated resources.
There’s another message as well, Dr. Weinert says.
“COVID basically obliterated sepsis programs for two and a half years,” he says. Now the CDC is saying it’s time to divert staff back to sepsis care.
Stepping up sepsis care
Raymund Dantes, MD, assistant professor of medicine at Emory University, Atlanta, one of the developers of the core elements, says this is like a recipe for sepsis care.
Dr. Dantes compares the instructions for hospitals with getting a good restaurant up and running. And in the restaurant business, “you need more than the recipes. You need a leader or manager to ensure you have the right people working together, with the right supplies, getting the right feedback on their work to continuously improve,” he explains.
Dr. Dantes, who is also the physician lead for the Emory Healthcare Sepsis Program, says the approach is meant to be flexible to the size of the hospital, population served, and available resources.
He points out that a well-run sepsis program at a 25-bed rural hospital will look very different from the program at a 1,000-bed tertiary care hospital.
Some hospitals, Dr. Dantes says, will be starting from scratch when getting a sepsis program, and for those hospitals, the developers included a “Getting Started” section as part of the detailed, 29-page full report.
In September, Sepsis Awareness Month, the CDC will provide educational information to health care professionals, patients, families, and caregivers about preventing infections that can lead to sepsis through its ongoing Get Ahead of Sepsis campaign.
A version of this article first appeared on Medscape.com.
according to a recent survey by the Centers for Disease Control and Prevention.
For the hospitals that do have sepsis teams, only 55% of them report that their team leaders get dedicated time to manage their sepsis programs.
“One in three people who dies in a hospital has sepsis during that hospitalization,” CDC Director Mandy Cohen, MD, MPH, noted in a statement. “That’s why CDC is calling on all U.S. hospitals to have a sepsis program and raise the bar on sepsis care by incorporating seven core elements.”
The sepsis seven
- Leadership: Dedicating the necessary human, financial, and information technology resources.
- Accountability: Appointing a leader responsible for program outcomes and setting concrete goals.
- Multiprofessional: Engaging key partners throughout the organization.
- Action: Implementing structures and processes to improve the identification, management, and recovery from sepsis.
- Tracking: Measuring sepsis epidemiology, outcomes, and progress toward program goals and the impact of sepsis initiatives.
- Reporting: Providing usable information on sepsis treatment and outcomes to relevant partners.
- Education: Providing sepsis education to health care professionals during onboarding and annually.
Craig Weinert, MD, MPH, a pulmonologist and critical care physician and professor of medicine at the University of Minnesota, Minneapolis, says the point the CDC is making with the announcement is that when these sepsis programs have been implemented at hospitals, they have been successful at reducing mortality. And now, the agency is urging all hospitals to implement them and support them properly.
“It’s not asking hospitals to develop new, innovative kinds of sepsis programs. This is not about new drugs or new antibiotics or new devices,” Dr. Weinert says. “This is about having hospitals dedicate organizational resources to implementing sepsis programs.”
The CDC’s announcement is aimed toward hospital administrators, Dr. Weinert adds. The agency is making the case that sepsis needs more funding in hospitals that either don’t have the programs or aren’t supporting them with dedicated resources.
There’s another message as well, Dr. Weinert says.
“COVID basically obliterated sepsis programs for two and a half years,” he says. Now the CDC is saying it’s time to divert staff back to sepsis care.
Stepping up sepsis care
Raymund Dantes, MD, assistant professor of medicine at Emory University, Atlanta, one of the developers of the core elements, says this is like a recipe for sepsis care.
Dr. Dantes compares the instructions for hospitals with getting a good restaurant up and running. And in the restaurant business, “you need more than the recipes. You need a leader or manager to ensure you have the right people working together, with the right supplies, getting the right feedback on their work to continuously improve,” he explains.
Dr. Dantes, who is also the physician lead for the Emory Healthcare Sepsis Program, says the approach is meant to be flexible to the size of the hospital, population served, and available resources.
He points out that a well-run sepsis program at a 25-bed rural hospital will look very different from the program at a 1,000-bed tertiary care hospital.
Some hospitals, Dr. Dantes says, will be starting from scratch when getting a sepsis program, and for those hospitals, the developers included a “Getting Started” section as part of the detailed, 29-page full report.
In September, Sepsis Awareness Month, the CDC will provide educational information to health care professionals, patients, families, and caregivers about preventing infections that can lead to sepsis through its ongoing Get Ahead of Sepsis campaign.
A version of this article first appeared on Medscape.com.
according to a recent survey by the Centers for Disease Control and Prevention.
For the hospitals that do have sepsis teams, only 55% of them report that their team leaders get dedicated time to manage their sepsis programs.
“One in three people who dies in a hospital has sepsis during that hospitalization,” CDC Director Mandy Cohen, MD, MPH, noted in a statement. “That’s why CDC is calling on all U.S. hospitals to have a sepsis program and raise the bar on sepsis care by incorporating seven core elements.”
The sepsis seven
- Leadership: Dedicating the necessary human, financial, and information technology resources.
- Accountability: Appointing a leader responsible for program outcomes and setting concrete goals.
- Multiprofessional: Engaging key partners throughout the organization.
- Action: Implementing structures and processes to improve the identification, management, and recovery from sepsis.
- Tracking: Measuring sepsis epidemiology, outcomes, and progress toward program goals and the impact of sepsis initiatives.
- Reporting: Providing usable information on sepsis treatment and outcomes to relevant partners.
- Education: Providing sepsis education to health care professionals during onboarding and annually.
Craig Weinert, MD, MPH, a pulmonologist and critical care physician and professor of medicine at the University of Minnesota, Minneapolis, says the point the CDC is making with the announcement is that when these sepsis programs have been implemented at hospitals, they have been successful at reducing mortality. And now, the agency is urging all hospitals to implement them and support them properly.
“It’s not asking hospitals to develop new, innovative kinds of sepsis programs. This is not about new drugs or new antibiotics or new devices,” Dr. Weinert says. “This is about having hospitals dedicate organizational resources to implementing sepsis programs.”
The CDC’s announcement is aimed toward hospital administrators, Dr. Weinert adds. The agency is making the case that sepsis needs more funding in hospitals that either don’t have the programs or aren’t supporting them with dedicated resources.
There’s another message as well, Dr. Weinert says.
“COVID basically obliterated sepsis programs for two and a half years,” he says. Now the CDC is saying it’s time to divert staff back to sepsis care.
Stepping up sepsis care
Raymund Dantes, MD, assistant professor of medicine at Emory University, Atlanta, one of the developers of the core elements, says this is like a recipe for sepsis care.
Dr. Dantes compares the instructions for hospitals with getting a good restaurant up and running. And in the restaurant business, “you need more than the recipes. You need a leader or manager to ensure you have the right people working together, with the right supplies, getting the right feedback on their work to continuously improve,” he explains.
Dr. Dantes, who is also the physician lead for the Emory Healthcare Sepsis Program, says the approach is meant to be flexible to the size of the hospital, population served, and available resources.
He points out that a well-run sepsis program at a 25-bed rural hospital will look very different from the program at a 1,000-bed tertiary care hospital.
Some hospitals, Dr. Dantes says, will be starting from scratch when getting a sepsis program, and for those hospitals, the developers included a “Getting Started” section as part of the detailed, 29-page full report.
In September, Sepsis Awareness Month, the CDC will provide educational information to health care professionals, patients, families, and caregivers about preventing infections that can lead to sepsis through its ongoing Get Ahead of Sepsis campaign.
A version of this article first appeared on Medscape.com.
Ruxolitinib for vitiligo: Experts share experiences from first year
.
The Food and Drug Administration approved the cream formulation of ruxolitinib (Opzelura), a JAK inhibitor, for repigmentation of nonsegmental vitiligo in July 2022 for people aged 12 years and older.
Raj Chovatiya, MD, PhD, assistant professor of dermatology at Northwestern University, Chicago, said that he likes to use ruxolitinib cream in combination with other treatments.
“In the real world with vitiligo patients, we’re oftentimes doing combinatorial therapy anyway. So phototherapy, specifically, narrow-band UVB, is something that we have a lot of clinical evidence for over the years, and it’s a modality that can combine with topical steroids and topical calcineurin inhibitors.”
He said trials to study combinations will yield better guidance on optimal use of ruxolitinib cream. “In general, vitiligo patients can really benefit from phototherapy,” he said in an interview. (Labeling recommends against combination with other JAK inhibitors, biologics, or potent immunosuppressants, such as azathioprine or cyclosporine.)
This first year has shown that ruxolitinib is an effective option, but counseling patients to expect slow improvement is important so that patients stick with it, he noted.
Documenting what treatments patients with vitiligo have used before is important, he said, as is counseling patients that ruxolitinib is approved only for use on up to 10% of a person’s body surface area. (Product labeling recommends that a thin layer be applied twice a day to affected areas up to 10% of body surface area.)
Ruxolitinib has brought a “louder voice” to vitiligo and has opened up options for patients with the disease, Dr. Chovatiya said. “Having the ability to topically treat people who have very extensive disease really gives us a lot more flexibility than we have had before.”
Good experiences with payers at safety-net hospital
Candrice R. Heath, MD, assistant professor of dermatology at Temple University, Philadelphia, said that real-world experience with topical ruxolitinib will be more evident after its been on the market for 18-24 months.
Dr. Heath said she, too, encourages use of narrow-band UVB phototherapy in conjunction with the treatment.
From an insurance reimbursement standpoint, she said that she is glad that there have been fewer hurdles in getting ruxolitinib to patients than she has experienced with other medications.
In her safety-net hospital, she told this news organization, she sees patients with many types of insurance, but most have Medicaid. “So, I’m always expecting the step therapies, denials, pushbacks, etc.,” she said. But the path has been smoother for ruxolitinib coverage, she noted.
Her colleagues are committed to documenting everything the patient has tried, she added, and that helps with prior authorization.
Dr. Heath said that pointing out to insurers that ruxolitinib is the only approved treatment for repigmentation helps facilitate coverage.
“The science is advancing, and I’m happy to be practicing during a time when we actually have something approved for vitiligo,” she said. But she pointed out that phototherapy often is not covered for vitiligo, “which is horrible, when it is readily approved for psoriasis and atopic dermatitis.”
To document progress, Dr. Heath said that she always takes photographs of her patients with vitiligo because “the pictures remind us how far we have come.”
Data spotlight success in adolescents
Data from two trials give a clinical picture of the drug’s safety and efficacy in younger patients.
Adolescents had particularly good results in the first year with ruxolitinib, according to pooled phase 3 data from TRuE-V1 and TRuE-V2, this news organization reported.
The findings, presented at the 25th World Congress of Dermatology in Singapore, indicate that more than half of the participants achieved at least a 50% improvement from baseline in the total Vitiligo Area Scoring Index (T-VASI50) at 52 weeks.
The percentages of young patients aged 12-17 years taking twice-daily ruxolitinib who achieved T-VASI 50 at weeks 12, 24, and 52 were 11.5%, 26.9%, and 57.7%, respectively. The corresponding percentages for all in the study population were 10.7%, 22.7%, and 44.4%, respectively.
At the meeting, the presenter, Julien Seneschal, MD, PhD, professor of dermatology and head of the vitiligo and pigmentary disorders clinic at the University of Bordeaux, France, said, “This suggests that younger patients can respond better to the treatment.” He noted, however, that there were few adolescents in the studies.
New excitement in the field
Daniel Gutierrez, MD, assistant professor of dermatology at New York University, said the treatment has brought new excitement to the field.
“Patients with vitiligo are very motivated to treat their disease,” he said, because it typically is on the face and other highly visual areas, which can affect their overall perception of self.
Previously, he noted in an interview, the only FDA-approved treatment was monobenzone, but that was for depigmentation rather than repigmentation.
Otherwise, treatments were being used off label, and patients were receiving compounded formulations that often weren’t covered by insurance and often had shorter shelf life.
He said that he still occasionally gets denials from payers who consider vitiligo a cosmetic condition.
“I’ve had more luck with insurance, at least in the New York State area.” He added that sometimes payers require use of a topical calcineurin inhibitor for about 12 weeks before they will cover ruxolitinib.
Dr. Gutierrez also recommends using phototherapy with topical ruxolitinib “because they work on slightly different pathways.”
When he starts patients on a new therapy such as ruxolitinib, he asks them to come back in 3 months, and often by then, progress is evident. Facial areas show the most response, he said, while hands and feet are less likely to show significant improvement.
He said that it’s important for physicians and patients to know that improvements can take weeks or months to be noticeable. “I tell patients not to give up,” he added.
Showing the patients pictures from the current appointment and comparing them with pictures from previous appointments can help them better understand their progress, he said.
Lead investigator adds observations
David Rosmarin, MD, chair of the department of dermatology at Indiana University, Indianapolis, was the lead investigator of the pivotal TruE-V1 and TruE-V2 trials for vitiligo. In that role, he has been treating vitiligo patients with topical ruxolitinib since 2015.
In an interview, he said that many patients “don’t hit their optimal results at 3 months, 6 months, even the year mark. With continued use, many can see continued benefit.”
Other patients, he said, don’t respond within the first 6 months but with continued use may eventually respond, he said.
“Unfortunately, we have no way of knowing, based on clinical characteristics or baseline demographics, whether a patient will be a delayed responder or not or an early responder,” Dr. Rosmarin added.
He provided several observations about people who have stopped taking the medication.
“When people stop,” he said, “some maintain their response, but some start to depigment again. Again, we have no way of predicting who will be in which category.”
He said that once patients have hit their desired response, he usually advises them to taper down to maybe twice a week or to stop treatment, but if they see any recurrence, they should start reusing the medicine.
“We have some patients who have gone 6 or 7 years now before they had a recurrence, but others may start to depigment again in 2 to 3 months,” Dr. Rosmarin said.
As for phototherapy, he said, the combination with topical ruxolitinib is being studied.
“We think the combination is synergistic and better than either alone, but we’re still waiting for data to prove that,” he said.
In his practice, he offers patients the option either to use just ruxolitinib cream or the combination early on. Many patients, because of convenience, say they’ll first try the cream to see if that works.
“The challenge with light [therapy] is that it can be very inconvenient,” he said. Patients have to live close to a phototherapy unit to receive therapy 2-3 times a week or have a phototherapy product in their home.
Next in the pipeline
Experts say the progress doesn’t stop with ruxolitinib cream. Current trials of several medications show there’s more to come for patients with vitiligo.
Dr. Chovatiya said that next up may be oral ritlecitinib (Litfulo), a JAK inhibitor that was approved for severe alopecia areata in June for people aged 12 years and older. Phase 2 results have been published for its use with vitiligo.
“This would be an oral medication that may be able to help people with much more extensive disease as far as vitiligo goes,” he said, adding that he expects approval for a vitiligo indication within a few years.
He pointed out that longer-term safety data will be available because it is already on the market for alopecia.
Upadacitinib (Rinvoq), an oral JAK inhibitor, is approved for atopic dermatitis but is being studied for vitiligo as well, he noted. “I’m very excited to see what that holds for patients as well,” Dr. Chovatiya said.
Dr. Gutierrez said that he is excited about oral JAK inhibitors but sees potential in finding new ways to transplant melanocytes into areas where there are none.
The pigmentation field has seen new energy since last year’s approval, he said, particularly among people of color.
“We have new options for vitiligo that were lacking compared with other conditions, such as atopic dermatitis and psoriasis,” he said. “Hopefully, there will be more promising breakthroughs.”
Dr. Rosmarin is the chief investigator for the pivotal trials that led to FDA approval of ruxolitinib. He disclosed ties with AbbVie, Abcuro, AltruBio, Amgen, Arena, Boehringer Ingelheim, Bristol-Meyers Squibb, Celgene, Concert, CSL Behring, Dermavant, Dermira, Galderma, Incyte, Janssen, Kyowa Kirin, Lilly, Merck, Novartis, Pfizer, Regeneron, Revolo Biotherapeutics, Sanofi, Sun Pharmaceuticals, UCB, and Viela Bio. Dr. Chovatiya disclosed ties with AbbVie, Arcutis, Arena, Argenx, Beiersdorf, Bristol-Myers Squibb, Dermavant, Eli Lilly, EPI Health, Incyte, LEO Pharma, L’Oréal, National Eczema Association, Pfizer, Regeneron, Sanofi, and UCB. Dr. Heath and Dr. Gutierrez report no relevant financial relationships.
A version of this article appeared on Medscape.com.
.
The Food and Drug Administration approved the cream formulation of ruxolitinib (Opzelura), a JAK inhibitor, for repigmentation of nonsegmental vitiligo in July 2022 for people aged 12 years and older.
Raj Chovatiya, MD, PhD, assistant professor of dermatology at Northwestern University, Chicago, said that he likes to use ruxolitinib cream in combination with other treatments.
“In the real world with vitiligo patients, we’re oftentimes doing combinatorial therapy anyway. So phototherapy, specifically, narrow-band UVB, is something that we have a lot of clinical evidence for over the years, and it’s a modality that can combine with topical steroids and topical calcineurin inhibitors.”
He said trials to study combinations will yield better guidance on optimal use of ruxolitinib cream. “In general, vitiligo patients can really benefit from phototherapy,” he said in an interview. (Labeling recommends against combination with other JAK inhibitors, biologics, or potent immunosuppressants, such as azathioprine or cyclosporine.)
This first year has shown that ruxolitinib is an effective option, but counseling patients to expect slow improvement is important so that patients stick with it, he noted.
Documenting what treatments patients with vitiligo have used before is important, he said, as is counseling patients that ruxolitinib is approved only for use on up to 10% of a person’s body surface area. (Product labeling recommends that a thin layer be applied twice a day to affected areas up to 10% of body surface area.)
Ruxolitinib has brought a “louder voice” to vitiligo and has opened up options for patients with the disease, Dr. Chovatiya said. “Having the ability to topically treat people who have very extensive disease really gives us a lot more flexibility than we have had before.”
Good experiences with payers at safety-net hospital
Candrice R. Heath, MD, assistant professor of dermatology at Temple University, Philadelphia, said that real-world experience with topical ruxolitinib will be more evident after its been on the market for 18-24 months.
Dr. Heath said she, too, encourages use of narrow-band UVB phototherapy in conjunction with the treatment.
From an insurance reimbursement standpoint, she said that she is glad that there have been fewer hurdles in getting ruxolitinib to patients than she has experienced with other medications.
In her safety-net hospital, she told this news organization, she sees patients with many types of insurance, but most have Medicaid. “So, I’m always expecting the step therapies, denials, pushbacks, etc.,” she said. But the path has been smoother for ruxolitinib coverage, she noted.
Her colleagues are committed to documenting everything the patient has tried, she added, and that helps with prior authorization.
Dr. Heath said that pointing out to insurers that ruxolitinib is the only approved treatment for repigmentation helps facilitate coverage.
“The science is advancing, and I’m happy to be practicing during a time when we actually have something approved for vitiligo,” she said. But she pointed out that phototherapy often is not covered for vitiligo, “which is horrible, when it is readily approved for psoriasis and atopic dermatitis.”
To document progress, Dr. Heath said that she always takes photographs of her patients with vitiligo because “the pictures remind us how far we have come.”
Data spotlight success in adolescents
Data from two trials give a clinical picture of the drug’s safety and efficacy in younger patients.
Adolescents had particularly good results in the first year with ruxolitinib, according to pooled phase 3 data from TRuE-V1 and TRuE-V2, this news organization reported.
The findings, presented at the 25th World Congress of Dermatology in Singapore, indicate that more than half of the participants achieved at least a 50% improvement from baseline in the total Vitiligo Area Scoring Index (T-VASI50) at 52 weeks.
The percentages of young patients aged 12-17 years taking twice-daily ruxolitinib who achieved T-VASI 50 at weeks 12, 24, and 52 were 11.5%, 26.9%, and 57.7%, respectively. The corresponding percentages for all in the study population were 10.7%, 22.7%, and 44.4%, respectively.
At the meeting, the presenter, Julien Seneschal, MD, PhD, professor of dermatology and head of the vitiligo and pigmentary disorders clinic at the University of Bordeaux, France, said, “This suggests that younger patients can respond better to the treatment.” He noted, however, that there were few adolescents in the studies.
New excitement in the field
Daniel Gutierrez, MD, assistant professor of dermatology at New York University, said the treatment has brought new excitement to the field.
“Patients with vitiligo are very motivated to treat their disease,” he said, because it typically is on the face and other highly visual areas, which can affect their overall perception of self.
Previously, he noted in an interview, the only FDA-approved treatment was monobenzone, but that was for depigmentation rather than repigmentation.
Otherwise, treatments were being used off label, and patients were receiving compounded formulations that often weren’t covered by insurance and often had shorter shelf life.
He said that he still occasionally gets denials from payers who consider vitiligo a cosmetic condition.
“I’ve had more luck with insurance, at least in the New York State area.” He added that sometimes payers require use of a topical calcineurin inhibitor for about 12 weeks before they will cover ruxolitinib.
Dr. Gutierrez also recommends using phototherapy with topical ruxolitinib “because they work on slightly different pathways.”
When he starts patients on a new therapy such as ruxolitinib, he asks them to come back in 3 months, and often by then, progress is evident. Facial areas show the most response, he said, while hands and feet are less likely to show significant improvement.
He said that it’s important for physicians and patients to know that improvements can take weeks or months to be noticeable. “I tell patients not to give up,” he added.
Showing the patients pictures from the current appointment and comparing them with pictures from previous appointments can help them better understand their progress, he said.
Lead investigator adds observations
David Rosmarin, MD, chair of the department of dermatology at Indiana University, Indianapolis, was the lead investigator of the pivotal TruE-V1 and TruE-V2 trials for vitiligo. In that role, he has been treating vitiligo patients with topical ruxolitinib since 2015.
In an interview, he said that many patients “don’t hit their optimal results at 3 months, 6 months, even the year mark. With continued use, many can see continued benefit.”
Other patients, he said, don’t respond within the first 6 months but with continued use may eventually respond, he said.
“Unfortunately, we have no way of knowing, based on clinical characteristics or baseline demographics, whether a patient will be a delayed responder or not or an early responder,” Dr. Rosmarin added.
He provided several observations about people who have stopped taking the medication.
“When people stop,” he said, “some maintain their response, but some start to depigment again. Again, we have no way of predicting who will be in which category.”
He said that once patients have hit their desired response, he usually advises them to taper down to maybe twice a week or to stop treatment, but if they see any recurrence, they should start reusing the medicine.
“We have some patients who have gone 6 or 7 years now before they had a recurrence, but others may start to depigment again in 2 to 3 months,” Dr. Rosmarin said.
As for phototherapy, he said, the combination with topical ruxolitinib is being studied.
“We think the combination is synergistic and better than either alone, but we’re still waiting for data to prove that,” he said.
In his practice, he offers patients the option either to use just ruxolitinib cream or the combination early on. Many patients, because of convenience, say they’ll first try the cream to see if that works.
“The challenge with light [therapy] is that it can be very inconvenient,” he said. Patients have to live close to a phototherapy unit to receive therapy 2-3 times a week or have a phototherapy product in their home.
Next in the pipeline
Experts say the progress doesn’t stop with ruxolitinib cream. Current trials of several medications show there’s more to come for patients with vitiligo.
Dr. Chovatiya said that next up may be oral ritlecitinib (Litfulo), a JAK inhibitor that was approved for severe alopecia areata in June for people aged 12 years and older. Phase 2 results have been published for its use with vitiligo.
“This would be an oral medication that may be able to help people with much more extensive disease as far as vitiligo goes,” he said, adding that he expects approval for a vitiligo indication within a few years.
He pointed out that longer-term safety data will be available because it is already on the market for alopecia.
Upadacitinib (Rinvoq), an oral JAK inhibitor, is approved for atopic dermatitis but is being studied for vitiligo as well, he noted. “I’m very excited to see what that holds for patients as well,” Dr. Chovatiya said.
Dr. Gutierrez said that he is excited about oral JAK inhibitors but sees potential in finding new ways to transplant melanocytes into areas where there are none.
The pigmentation field has seen new energy since last year’s approval, he said, particularly among people of color.
“We have new options for vitiligo that were lacking compared with other conditions, such as atopic dermatitis and psoriasis,” he said. “Hopefully, there will be more promising breakthroughs.”
Dr. Rosmarin is the chief investigator for the pivotal trials that led to FDA approval of ruxolitinib. He disclosed ties with AbbVie, Abcuro, AltruBio, Amgen, Arena, Boehringer Ingelheim, Bristol-Meyers Squibb, Celgene, Concert, CSL Behring, Dermavant, Dermira, Galderma, Incyte, Janssen, Kyowa Kirin, Lilly, Merck, Novartis, Pfizer, Regeneron, Revolo Biotherapeutics, Sanofi, Sun Pharmaceuticals, UCB, and Viela Bio. Dr. Chovatiya disclosed ties with AbbVie, Arcutis, Arena, Argenx, Beiersdorf, Bristol-Myers Squibb, Dermavant, Eli Lilly, EPI Health, Incyte, LEO Pharma, L’Oréal, National Eczema Association, Pfizer, Regeneron, Sanofi, and UCB. Dr. Heath and Dr. Gutierrez report no relevant financial relationships.
A version of this article appeared on Medscape.com.
.
The Food and Drug Administration approved the cream formulation of ruxolitinib (Opzelura), a JAK inhibitor, for repigmentation of nonsegmental vitiligo in July 2022 for people aged 12 years and older.
Raj Chovatiya, MD, PhD, assistant professor of dermatology at Northwestern University, Chicago, said that he likes to use ruxolitinib cream in combination with other treatments.
“In the real world with vitiligo patients, we’re oftentimes doing combinatorial therapy anyway. So phototherapy, specifically, narrow-band UVB, is something that we have a lot of clinical evidence for over the years, and it’s a modality that can combine with topical steroids and topical calcineurin inhibitors.”
He said trials to study combinations will yield better guidance on optimal use of ruxolitinib cream. “In general, vitiligo patients can really benefit from phototherapy,” he said in an interview. (Labeling recommends against combination with other JAK inhibitors, biologics, or potent immunosuppressants, such as azathioprine or cyclosporine.)
This first year has shown that ruxolitinib is an effective option, but counseling patients to expect slow improvement is important so that patients stick with it, he noted.
Documenting what treatments patients with vitiligo have used before is important, he said, as is counseling patients that ruxolitinib is approved only for use on up to 10% of a person’s body surface area. (Product labeling recommends that a thin layer be applied twice a day to affected areas up to 10% of body surface area.)
Ruxolitinib has brought a “louder voice” to vitiligo and has opened up options for patients with the disease, Dr. Chovatiya said. “Having the ability to topically treat people who have very extensive disease really gives us a lot more flexibility than we have had before.”
Good experiences with payers at safety-net hospital
Candrice R. Heath, MD, assistant professor of dermatology at Temple University, Philadelphia, said that real-world experience with topical ruxolitinib will be more evident after its been on the market for 18-24 months.
Dr. Heath said she, too, encourages use of narrow-band UVB phototherapy in conjunction with the treatment.
From an insurance reimbursement standpoint, she said that she is glad that there have been fewer hurdles in getting ruxolitinib to patients than she has experienced with other medications.
In her safety-net hospital, she told this news organization, she sees patients with many types of insurance, but most have Medicaid. “So, I’m always expecting the step therapies, denials, pushbacks, etc.,” she said. But the path has been smoother for ruxolitinib coverage, she noted.
Her colleagues are committed to documenting everything the patient has tried, she added, and that helps with prior authorization.
Dr. Heath said that pointing out to insurers that ruxolitinib is the only approved treatment for repigmentation helps facilitate coverage.
“The science is advancing, and I’m happy to be practicing during a time when we actually have something approved for vitiligo,” she said. But she pointed out that phototherapy often is not covered for vitiligo, “which is horrible, when it is readily approved for psoriasis and atopic dermatitis.”
To document progress, Dr. Heath said that she always takes photographs of her patients with vitiligo because “the pictures remind us how far we have come.”
Data spotlight success in adolescents
Data from two trials give a clinical picture of the drug’s safety and efficacy in younger patients.
Adolescents had particularly good results in the first year with ruxolitinib, according to pooled phase 3 data from TRuE-V1 and TRuE-V2, this news organization reported.
The findings, presented at the 25th World Congress of Dermatology in Singapore, indicate that more than half of the participants achieved at least a 50% improvement from baseline in the total Vitiligo Area Scoring Index (T-VASI50) at 52 weeks.
The percentages of young patients aged 12-17 years taking twice-daily ruxolitinib who achieved T-VASI 50 at weeks 12, 24, and 52 were 11.5%, 26.9%, and 57.7%, respectively. The corresponding percentages for all in the study population were 10.7%, 22.7%, and 44.4%, respectively.
At the meeting, the presenter, Julien Seneschal, MD, PhD, professor of dermatology and head of the vitiligo and pigmentary disorders clinic at the University of Bordeaux, France, said, “This suggests that younger patients can respond better to the treatment.” He noted, however, that there were few adolescents in the studies.
New excitement in the field
Daniel Gutierrez, MD, assistant professor of dermatology at New York University, said the treatment has brought new excitement to the field.
“Patients with vitiligo are very motivated to treat their disease,” he said, because it typically is on the face and other highly visual areas, which can affect their overall perception of self.
Previously, he noted in an interview, the only FDA-approved treatment was monobenzone, but that was for depigmentation rather than repigmentation.
Otherwise, treatments were being used off label, and patients were receiving compounded formulations that often weren’t covered by insurance and often had shorter shelf life.
He said that he still occasionally gets denials from payers who consider vitiligo a cosmetic condition.
“I’ve had more luck with insurance, at least in the New York State area.” He added that sometimes payers require use of a topical calcineurin inhibitor for about 12 weeks before they will cover ruxolitinib.
Dr. Gutierrez also recommends using phototherapy with topical ruxolitinib “because they work on slightly different pathways.”
When he starts patients on a new therapy such as ruxolitinib, he asks them to come back in 3 months, and often by then, progress is evident. Facial areas show the most response, he said, while hands and feet are less likely to show significant improvement.
He said that it’s important for physicians and patients to know that improvements can take weeks or months to be noticeable. “I tell patients not to give up,” he added.
Showing the patients pictures from the current appointment and comparing them with pictures from previous appointments can help them better understand their progress, he said.
Lead investigator adds observations
David Rosmarin, MD, chair of the department of dermatology at Indiana University, Indianapolis, was the lead investigator of the pivotal TruE-V1 and TruE-V2 trials for vitiligo. In that role, he has been treating vitiligo patients with topical ruxolitinib since 2015.
In an interview, he said that many patients “don’t hit their optimal results at 3 months, 6 months, even the year mark. With continued use, many can see continued benefit.”
Other patients, he said, don’t respond within the first 6 months but with continued use may eventually respond, he said.
“Unfortunately, we have no way of knowing, based on clinical characteristics or baseline demographics, whether a patient will be a delayed responder or not or an early responder,” Dr. Rosmarin added.
He provided several observations about people who have stopped taking the medication.
“When people stop,” he said, “some maintain their response, but some start to depigment again. Again, we have no way of predicting who will be in which category.”
He said that once patients have hit their desired response, he usually advises them to taper down to maybe twice a week or to stop treatment, but if they see any recurrence, they should start reusing the medicine.
“We have some patients who have gone 6 or 7 years now before they had a recurrence, but others may start to depigment again in 2 to 3 months,” Dr. Rosmarin said.
As for phototherapy, he said, the combination with topical ruxolitinib is being studied.
“We think the combination is synergistic and better than either alone, but we’re still waiting for data to prove that,” he said.
In his practice, he offers patients the option either to use just ruxolitinib cream or the combination early on. Many patients, because of convenience, say they’ll first try the cream to see if that works.
“The challenge with light [therapy] is that it can be very inconvenient,” he said. Patients have to live close to a phototherapy unit to receive therapy 2-3 times a week or have a phototherapy product in their home.
Next in the pipeline
Experts say the progress doesn’t stop with ruxolitinib cream. Current trials of several medications show there’s more to come for patients with vitiligo.
Dr. Chovatiya said that next up may be oral ritlecitinib (Litfulo), a JAK inhibitor that was approved for severe alopecia areata in June for people aged 12 years and older. Phase 2 results have been published for its use with vitiligo.
“This would be an oral medication that may be able to help people with much more extensive disease as far as vitiligo goes,” he said, adding that he expects approval for a vitiligo indication within a few years.
He pointed out that longer-term safety data will be available because it is already on the market for alopecia.
Upadacitinib (Rinvoq), an oral JAK inhibitor, is approved for atopic dermatitis but is being studied for vitiligo as well, he noted. “I’m very excited to see what that holds for patients as well,” Dr. Chovatiya said.
Dr. Gutierrez said that he is excited about oral JAK inhibitors but sees potential in finding new ways to transplant melanocytes into areas where there are none.
The pigmentation field has seen new energy since last year’s approval, he said, particularly among people of color.
“We have new options for vitiligo that were lacking compared with other conditions, such as atopic dermatitis and psoriasis,” he said. “Hopefully, there will be more promising breakthroughs.”
Dr. Rosmarin is the chief investigator for the pivotal trials that led to FDA approval of ruxolitinib. He disclosed ties with AbbVie, Abcuro, AltruBio, Amgen, Arena, Boehringer Ingelheim, Bristol-Meyers Squibb, Celgene, Concert, CSL Behring, Dermavant, Dermira, Galderma, Incyte, Janssen, Kyowa Kirin, Lilly, Merck, Novartis, Pfizer, Regeneron, Revolo Biotherapeutics, Sanofi, Sun Pharmaceuticals, UCB, and Viela Bio. Dr. Chovatiya disclosed ties with AbbVie, Arcutis, Arena, Argenx, Beiersdorf, Bristol-Myers Squibb, Dermavant, Eli Lilly, EPI Health, Incyte, LEO Pharma, L’Oréal, National Eczema Association, Pfizer, Regeneron, Sanofi, and UCB. Dr. Heath and Dr. Gutierrez report no relevant financial relationships.
A version of this article appeared on Medscape.com.
Making one key connection may increase HPV vax uptake
The understanding that human papillomavirus (HPV) causes oropharyngeal squamous cell carcinoma (OPSCC) has been linked with increased likelihood of adults having been vaccinated for HPV, new research indicates.
In a study published online in JAMA Otolaryngology–Head and Neck Surgery, most of the 288 adults surveyed with validated questions were not aware that HPV causes OPSCC and had not been told of the relationship by their health care provider.
Researchers found that when participants knew about the relationship between HPV infection and OPSCC they were more than three times as likely to be vaccinated (odds ratio, 3.7; 95% confidence interval, 1.8-7.6) as those without the knowledge.
The survey was paired with a novel point-of-care adult vaccination program within an otolaryngology clinic.
“Targeted education aimed at unvaccinated adults establishing the relationship between HPV infection and OPSCC, paired with point-of-care vaccination, may be an innovative strategy for increasing HPV vaccination rates in adults,” write the authors, led by Jacob C. Bloom, MD, with the department of otolaryngology–head and neck surgery at Boston Medical Center.
Current HPV vaccination recommendations include three parts:
- Routine vaccination at age 11 or 12 years
- Catch-up vaccination at ages 13-26 years if not adequately vaccinated
- Shared clinical decision-making in adults aged 27-45 years if the vaccine series has not been completed.
Despite proven efficacy and safety of the HPV vaccine, vaccination rates are low for adults. Although 75% of adolescents aged 13-17 years have initiated the HPV vaccine, recent studies show only 16% of U.S. men aged 18-21 years have received at least 1 dose of the HPV vaccine, the authors write.
Christiana Zhang, MD, with the division of internal medicine at Johns Hopkins University in Baltimore, who was not part of the study, said she was not surprised by the lack of knowledge about the HPV-OPSCC link.
Patients are often counseled on the relationship between HPV and genital warts or anogenital cancers like cervical cancer, she says, but there is less patient education surrounding the relationship between HPV and oropharyngeal cancers.
She says she does counsel patients on the link with OPSCC, but not all providers do and provider knowledge in general surrounding HPV is low.
“Research has shown that knowledge and confidence among health care providers surrounding HPV vaccination is generally low, and this corresponds with a low vaccination recommendation rate,” she says.
She adds, “Patient education on HPV infection and its relationship with OPSCC, paired with point-of-care vaccination for qualifying patients, is a great approach.”
But the education needs to go beyond patients, she says.
“Given the important role that health care providers play in vaccine uptake, I think further efforts are needed to educate providers on HPV vaccination as well,” she says.
The study included patients aged 18-45 years who sought routine outpatient care at the otolaryngology clinic at Boston Medical Center from Sept. 1, 2020, to May 19, 2021.
Limitations of this study include studying a population from a single otolaryngology clinic in an urban, academic medical center. The population was more racially and ethnically diverse than the U.S. population with 60.3% identifying as racial and ethnic minorities. Gender and educational levels were also not reflective of U.S. demographics as half (50.8%) of the participants had a college degree or higher and 58.3% were women.
Dr. Bloom reports grants from the American Head and Neck Cancer Society during the conduct of the study. Coauthor Dr. Faden reports personal fees from Merck, Neotic, Focus, BMS, Chrystalis Biomedical Advisors, and Guidepoint; receiving nonfinancial support from BostonGene and Predicine; and receiving grants from Calico outside the submitted work. Dr. Zhang reports no relevant financial relationships.
The understanding that human papillomavirus (HPV) causes oropharyngeal squamous cell carcinoma (OPSCC) has been linked with increased likelihood of adults having been vaccinated for HPV, new research indicates.
In a study published online in JAMA Otolaryngology–Head and Neck Surgery, most of the 288 adults surveyed with validated questions were not aware that HPV causes OPSCC and had not been told of the relationship by their health care provider.
Researchers found that when participants knew about the relationship between HPV infection and OPSCC they were more than three times as likely to be vaccinated (odds ratio, 3.7; 95% confidence interval, 1.8-7.6) as those without the knowledge.
The survey was paired with a novel point-of-care adult vaccination program within an otolaryngology clinic.
“Targeted education aimed at unvaccinated adults establishing the relationship between HPV infection and OPSCC, paired with point-of-care vaccination, may be an innovative strategy for increasing HPV vaccination rates in adults,” write the authors, led by Jacob C. Bloom, MD, with the department of otolaryngology–head and neck surgery at Boston Medical Center.
Current HPV vaccination recommendations include three parts:
- Routine vaccination at age 11 or 12 years
- Catch-up vaccination at ages 13-26 years if not adequately vaccinated
- Shared clinical decision-making in adults aged 27-45 years if the vaccine series has not been completed.
Despite proven efficacy and safety of the HPV vaccine, vaccination rates are low for adults. Although 75% of adolescents aged 13-17 years have initiated the HPV vaccine, recent studies show only 16% of U.S. men aged 18-21 years have received at least 1 dose of the HPV vaccine, the authors write.
Christiana Zhang, MD, with the division of internal medicine at Johns Hopkins University in Baltimore, who was not part of the study, said she was not surprised by the lack of knowledge about the HPV-OPSCC link.
Patients are often counseled on the relationship between HPV and genital warts or anogenital cancers like cervical cancer, she says, but there is less patient education surrounding the relationship between HPV and oropharyngeal cancers.
She says she does counsel patients on the link with OPSCC, but not all providers do and provider knowledge in general surrounding HPV is low.
“Research has shown that knowledge and confidence among health care providers surrounding HPV vaccination is generally low, and this corresponds with a low vaccination recommendation rate,” she says.
She adds, “Patient education on HPV infection and its relationship with OPSCC, paired with point-of-care vaccination for qualifying patients, is a great approach.”
But the education needs to go beyond patients, she says.
“Given the important role that health care providers play in vaccine uptake, I think further efforts are needed to educate providers on HPV vaccination as well,” she says.
The study included patients aged 18-45 years who sought routine outpatient care at the otolaryngology clinic at Boston Medical Center from Sept. 1, 2020, to May 19, 2021.
Limitations of this study include studying a population from a single otolaryngology clinic in an urban, academic medical center. The population was more racially and ethnically diverse than the U.S. population with 60.3% identifying as racial and ethnic minorities. Gender and educational levels were also not reflective of U.S. demographics as half (50.8%) of the participants had a college degree or higher and 58.3% were women.
Dr. Bloom reports grants from the American Head and Neck Cancer Society during the conduct of the study. Coauthor Dr. Faden reports personal fees from Merck, Neotic, Focus, BMS, Chrystalis Biomedical Advisors, and Guidepoint; receiving nonfinancial support from BostonGene and Predicine; and receiving grants from Calico outside the submitted work. Dr. Zhang reports no relevant financial relationships.
The understanding that human papillomavirus (HPV) causes oropharyngeal squamous cell carcinoma (OPSCC) has been linked with increased likelihood of adults having been vaccinated for HPV, new research indicates.
In a study published online in JAMA Otolaryngology–Head and Neck Surgery, most of the 288 adults surveyed with validated questions were not aware that HPV causes OPSCC and had not been told of the relationship by their health care provider.
Researchers found that when participants knew about the relationship between HPV infection and OPSCC they were more than three times as likely to be vaccinated (odds ratio, 3.7; 95% confidence interval, 1.8-7.6) as those without the knowledge.
The survey was paired with a novel point-of-care adult vaccination program within an otolaryngology clinic.
“Targeted education aimed at unvaccinated adults establishing the relationship between HPV infection and OPSCC, paired with point-of-care vaccination, may be an innovative strategy for increasing HPV vaccination rates in adults,” write the authors, led by Jacob C. Bloom, MD, with the department of otolaryngology–head and neck surgery at Boston Medical Center.
Current HPV vaccination recommendations include three parts:
- Routine vaccination at age 11 or 12 years
- Catch-up vaccination at ages 13-26 years if not adequately vaccinated
- Shared clinical decision-making in adults aged 27-45 years if the vaccine series has not been completed.
Despite proven efficacy and safety of the HPV vaccine, vaccination rates are low for adults. Although 75% of adolescents aged 13-17 years have initiated the HPV vaccine, recent studies show only 16% of U.S. men aged 18-21 years have received at least 1 dose of the HPV vaccine, the authors write.
Christiana Zhang, MD, with the division of internal medicine at Johns Hopkins University in Baltimore, who was not part of the study, said she was not surprised by the lack of knowledge about the HPV-OPSCC link.
Patients are often counseled on the relationship between HPV and genital warts or anogenital cancers like cervical cancer, she says, but there is less patient education surrounding the relationship between HPV and oropharyngeal cancers.
She says she does counsel patients on the link with OPSCC, but not all providers do and provider knowledge in general surrounding HPV is low.
“Research has shown that knowledge and confidence among health care providers surrounding HPV vaccination is generally low, and this corresponds with a low vaccination recommendation rate,” she says.
She adds, “Patient education on HPV infection and its relationship with OPSCC, paired with point-of-care vaccination for qualifying patients, is a great approach.”
But the education needs to go beyond patients, she says.
“Given the important role that health care providers play in vaccine uptake, I think further efforts are needed to educate providers on HPV vaccination as well,” she says.
The study included patients aged 18-45 years who sought routine outpatient care at the otolaryngology clinic at Boston Medical Center from Sept. 1, 2020, to May 19, 2021.
Limitations of this study include studying a population from a single otolaryngology clinic in an urban, academic medical center. The population was more racially and ethnically diverse than the U.S. population with 60.3% identifying as racial and ethnic minorities. Gender and educational levels were also not reflective of U.S. demographics as half (50.8%) of the participants had a college degree or higher and 58.3% were women.
Dr. Bloom reports grants from the American Head and Neck Cancer Society during the conduct of the study. Coauthor Dr. Faden reports personal fees from Merck, Neotic, Focus, BMS, Chrystalis Biomedical Advisors, and Guidepoint; receiving nonfinancial support from BostonGene and Predicine; and receiving grants from Calico outside the submitted work. Dr. Zhang reports no relevant financial relationships.
FROM JAMA OTOLARYNGOLOGY–HEAD AND NECK SURGERY
AAP’s hearing test clinical update is the first since 2009
The update is the first since 2009.
The AAP’s clinical report was published online in Pediatrics.
Charles Bower, MD, with the department of otolaryngology at Arkansas Children’s Hospital in Little Rock, led the research team representing AAP’s Committee on Practice and Ambulatory Medicine, section on otolaryngology and head and neck surgery.
The report details how primary care clinicians can detect changes in hearing status by age.
Eliminating terms such as ‘failed’ or ‘impairment’
A key change in this report is that it no longer uses terms such as “loss,” “failed,” or “impairment,” “to reflect that children who are deaf or hard of hearing (D/HH) are equal, healthy, and whole,” the authors wrote.
The report’s recommendations are based on the literature and engagement with deaf and hard of hearing professionals and partner organizations, such as the National Association of the Deaf, working with the AAP Early Hearing Detection and Intervention program.
Birth to 5 a critical time
The authors noted that early medical support for hearing is especially important between birth and 5 years of age. That span is a critical time for brain and language development.
Parents and caregivers are often the first to notice a child’s inattention or erratic responses to sound, they wrote, and it’s important to address these concerns with a pediatrician even if the child has passed a newborn hearing test after birth.
Among recommendations in the update:
- All children should have an objective, evidence-based risk assessment for changes in hearing.
- Children at all ages should have prompt screening if there is clinical or caregiver concern about hearing.
- A child who screens positive for atypical hearing in one or both ears should be referred to an audiologist for diagnostic consultation and testing.
- Because standard testing for children with developmental or behavioral health conditions may be impossible or inaccurate, referral may be more appropriate to audiology for electrophysiological hearing testing using auditory brainstem response (ABR) with sedation.
- To prevent false negatives and to avoid delays in identification, access to language, and support, screening tests should not be repeated more than once before referral to audiology.
Additional recommendations
The report authors pointed out that genetic causes may affect hearing and may show up beyond the newborn period.
They wrote that congenital cytomegalovirus (cCMV) infection is the most common infectious cause of childhood sensorineural hearing change and accounts for 25% of deaf and hard of hearing children at age 4.
Meningitis and otitis media also are leading causes of a change in hearing.
Judith E.C. Lieu, MD, MSPH, professor, program director and vice-chair for education in the department of otolaryngology and head and neck surgery at Washington University in St. Louis, who was not part of the research team, said screening recommendations have not changed much in the update, but she highlighted some points.
She noted that tympanometry is not listed as a method of hearing screening in primary care.
“I agree that tympanogram is not a hearing screening. It is an adjunct to look at middle ear function, but that doesn’t necessarily mean it looks for hearing,” she said.
Dr. Lieu says she does take issue with the stated length of one of the tests in the paper. She said she is concerned that the pure-tone audiometry test for ages 4 through adolescence is listed as taking 30 minutes in a primary care setting. She said she worries that pediatricians will be put off by reading that it is a 30-minute test.
“Honestly, in my experience, it doesn’t take 30 minutes. Maybe 10 minutes,” she said. “I don’t know any pediatrician who could devote 30 minutes to one screening test.”
Development milestones have been adjusted
Also different in these recommendations are the developmental and speech milestones updated according to the most recent AAP information, Dr. Lieu said. Though the new milestones don’t change by much, they are important to note, she said, such as updated guidance on when to be concerned about speech delay.
She said she wished the guidance included more about hearing loss in older children.
The report authors stated that about 1 to 3 per 1,000 children have atypical hearing at birth and similar numbers become deaf or hard of hearing later in childhood.
But Dr. Lieu says that statistic may give the wrong impression about frequency of atypical hearing.
“Hearing loss increases during childhood,” she pointed out. “By the time they hit about age 18, about 15% of kids have some kind of hearing loss.”
“I don’t think it’s made clear to pediatricians that this is not 1 or 2 in a thousand children – this happens much more frequently,” she said.
The report authors and Dr. Lieu report no relevant financial relationships.
The update is the first since 2009.
The AAP’s clinical report was published online in Pediatrics.
Charles Bower, MD, with the department of otolaryngology at Arkansas Children’s Hospital in Little Rock, led the research team representing AAP’s Committee on Practice and Ambulatory Medicine, section on otolaryngology and head and neck surgery.
The report details how primary care clinicians can detect changes in hearing status by age.
Eliminating terms such as ‘failed’ or ‘impairment’
A key change in this report is that it no longer uses terms such as “loss,” “failed,” or “impairment,” “to reflect that children who are deaf or hard of hearing (D/HH) are equal, healthy, and whole,” the authors wrote.
The report’s recommendations are based on the literature and engagement with deaf and hard of hearing professionals and partner organizations, such as the National Association of the Deaf, working with the AAP Early Hearing Detection and Intervention program.
Birth to 5 a critical time
The authors noted that early medical support for hearing is especially important between birth and 5 years of age. That span is a critical time for brain and language development.
Parents and caregivers are often the first to notice a child’s inattention or erratic responses to sound, they wrote, and it’s important to address these concerns with a pediatrician even if the child has passed a newborn hearing test after birth.
Among recommendations in the update:
- All children should have an objective, evidence-based risk assessment for changes in hearing.
- Children at all ages should have prompt screening if there is clinical or caregiver concern about hearing.
- A child who screens positive for atypical hearing in one or both ears should be referred to an audiologist for diagnostic consultation and testing.
- Because standard testing for children with developmental or behavioral health conditions may be impossible or inaccurate, referral may be more appropriate to audiology for electrophysiological hearing testing using auditory brainstem response (ABR) with sedation.
- To prevent false negatives and to avoid delays in identification, access to language, and support, screening tests should not be repeated more than once before referral to audiology.
Additional recommendations
The report authors pointed out that genetic causes may affect hearing and may show up beyond the newborn period.
They wrote that congenital cytomegalovirus (cCMV) infection is the most common infectious cause of childhood sensorineural hearing change and accounts for 25% of deaf and hard of hearing children at age 4.
Meningitis and otitis media also are leading causes of a change in hearing.
Judith E.C. Lieu, MD, MSPH, professor, program director and vice-chair for education in the department of otolaryngology and head and neck surgery at Washington University in St. Louis, who was not part of the research team, said screening recommendations have not changed much in the update, but she highlighted some points.
She noted that tympanometry is not listed as a method of hearing screening in primary care.
“I agree that tympanogram is not a hearing screening. It is an adjunct to look at middle ear function, but that doesn’t necessarily mean it looks for hearing,” she said.
Dr. Lieu says she does take issue with the stated length of one of the tests in the paper. She said she is concerned that the pure-tone audiometry test for ages 4 through adolescence is listed as taking 30 minutes in a primary care setting. She said she worries that pediatricians will be put off by reading that it is a 30-minute test.
“Honestly, in my experience, it doesn’t take 30 minutes. Maybe 10 minutes,” she said. “I don’t know any pediatrician who could devote 30 minutes to one screening test.”
Development milestones have been adjusted
Also different in these recommendations are the developmental and speech milestones updated according to the most recent AAP information, Dr. Lieu said. Though the new milestones don’t change by much, they are important to note, she said, such as updated guidance on when to be concerned about speech delay.
She said she wished the guidance included more about hearing loss in older children.
The report authors stated that about 1 to 3 per 1,000 children have atypical hearing at birth and similar numbers become deaf or hard of hearing later in childhood.
But Dr. Lieu says that statistic may give the wrong impression about frequency of atypical hearing.
“Hearing loss increases during childhood,” she pointed out. “By the time they hit about age 18, about 15% of kids have some kind of hearing loss.”
“I don’t think it’s made clear to pediatricians that this is not 1 or 2 in a thousand children – this happens much more frequently,” she said.
The report authors and Dr. Lieu report no relevant financial relationships.
The update is the first since 2009.
The AAP’s clinical report was published online in Pediatrics.
Charles Bower, MD, with the department of otolaryngology at Arkansas Children’s Hospital in Little Rock, led the research team representing AAP’s Committee on Practice and Ambulatory Medicine, section on otolaryngology and head and neck surgery.
The report details how primary care clinicians can detect changes in hearing status by age.
Eliminating terms such as ‘failed’ or ‘impairment’
A key change in this report is that it no longer uses terms such as “loss,” “failed,” or “impairment,” “to reflect that children who are deaf or hard of hearing (D/HH) are equal, healthy, and whole,” the authors wrote.
The report’s recommendations are based on the literature and engagement with deaf and hard of hearing professionals and partner organizations, such as the National Association of the Deaf, working with the AAP Early Hearing Detection and Intervention program.
Birth to 5 a critical time
The authors noted that early medical support for hearing is especially important between birth and 5 years of age. That span is a critical time for brain and language development.
Parents and caregivers are often the first to notice a child’s inattention or erratic responses to sound, they wrote, and it’s important to address these concerns with a pediatrician even if the child has passed a newborn hearing test after birth.
Among recommendations in the update:
- All children should have an objective, evidence-based risk assessment for changes in hearing.
- Children at all ages should have prompt screening if there is clinical or caregiver concern about hearing.
- A child who screens positive for atypical hearing in one or both ears should be referred to an audiologist for diagnostic consultation and testing.
- Because standard testing for children with developmental or behavioral health conditions may be impossible or inaccurate, referral may be more appropriate to audiology for electrophysiological hearing testing using auditory brainstem response (ABR) with sedation.
- To prevent false negatives and to avoid delays in identification, access to language, and support, screening tests should not be repeated more than once before referral to audiology.
Additional recommendations
The report authors pointed out that genetic causes may affect hearing and may show up beyond the newborn period.
They wrote that congenital cytomegalovirus (cCMV) infection is the most common infectious cause of childhood sensorineural hearing change and accounts for 25% of deaf and hard of hearing children at age 4.
Meningitis and otitis media also are leading causes of a change in hearing.
Judith E.C. Lieu, MD, MSPH, professor, program director and vice-chair for education in the department of otolaryngology and head and neck surgery at Washington University in St. Louis, who was not part of the research team, said screening recommendations have not changed much in the update, but she highlighted some points.
She noted that tympanometry is not listed as a method of hearing screening in primary care.
“I agree that tympanogram is not a hearing screening. It is an adjunct to look at middle ear function, but that doesn’t necessarily mean it looks for hearing,” she said.
Dr. Lieu says she does take issue with the stated length of one of the tests in the paper. She said she is concerned that the pure-tone audiometry test for ages 4 through adolescence is listed as taking 30 minutes in a primary care setting. She said she worries that pediatricians will be put off by reading that it is a 30-minute test.
“Honestly, in my experience, it doesn’t take 30 minutes. Maybe 10 minutes,” she said. “I don’t know any pediatrician who could devote 30 minutes to one screening test.”
Development milestones have been adjusted
Also different in these recommendations are the developmental and speech milestones updated according to the most recent AAP information, Dr. Lieu said. Though the new milestones don’t change by much, they are important to note, she said, such as updated guidance on when to be concerned about speech delay.
She said she wished the guidance included more about hearing loss in older children.
The report authors stated that about 1 to 3 per 1,000 children have atypical hearing at birth and similar numbers become deaf or hard of hearing later in childhood.
But Dr. Lieu says that statistic may give the wrong impression about frequency of atypical hearing.
“Hearing loss increases during childhood,” she pointed out. “By the time they hit about age 18, about 15% of kids have some kind of hearing loss.”
“I don’t think it’s made clear to pediatricians that this is not 1 or 2 in a thousand children – this happens much more frequently,” she said.
The report authors and Dr. Lieu report no relevant financial relationships.
FROM PEDIATRICS
New RSV shot is a monoclonal antibody, not a vaccine
For the first time in the fall of 2023, families will be offered season-long protection for infants and some children against respiratory syncytial virus (RSV).
The Food and Drug Administration in July approved a prevention called nirsevimab (Beyfortus, AstraZeneca/Sanofi) and it is expected to be widely rolled out in the coming weeks as the RSV season begins.
And monoclonal antibodies are often used for treatment rather than prevention.
Adding to potential confusion is the fact the Centers for Disease Control and Prevention has included nirsevimab in the Vaccines for Children program, which covers the costs for uninsured kids and makes it more accessible.
Nirsevimab is approved for infants (up to 8 months old) born during or entering their first RSV season, and in children up to 2 years of age who are still vulnerable to severe RSV through their second season.
It’s recommended that all infants get one injection in their first 8 months for prevention instead of the previous monthly shots used to help prevent kids at high risk from getting severe RSV.
If monoclonal antibodies can be used for preventing disease in infants, could they become a viable vaccine alternative for adults?
Specialists say no.
That’s partly because of the difference in body size. Although an injection is an option for a newborn, pediatricians suggest, it would take far too much of the treatment to work as a shot for adults.
Ruth Karron, MD, an expert in pediatric infectious diseases at Johns Hopkins Medicine, Baltimore, said that, while vaccines come in small amounts and activate immune cells, monoclonal antibodies are more like a drug, with the dose based on weight.
“You’d have to give it intravenously,” for larger doses, she explained, which has never been studied before and would also be very expensive. “It really couldn’t be an option for adults.”
What’s the difference between vaccines and antibodies?
Monoclonal antibodies are proteins made in a lab to mimic the immune system’s ability to fight pathogens such as viruses.
Dr. Karron explained that a wide variety of monoclonal antibodies have long been used to treat diseases such as cancers and autoimmune disease. In recent years, the antibodies have been used to treat COVID.
Monoclonal antibodies have also been used to treat RSV in children, but the effects don’t last long – they confer passive immunity and “when it’s gone, it’s gone,” Dr. Karron said.
That means kids at high risk for severe RSV have had to get monthly injections.
But with nirsevimab, the mutated antibodies stay in circulation longer so they can last 5 or 6 months, enough to cover the RSV season, Dr. Karron explained. “It’s highly, highly effective.”
Vaccines train the body
“The idea with vaccines is that you engage the individual’s immune system. You teach it to make antibodies,” Dr. Karron said. Conversely, “you give an antibody and it’s good for as long as the antibody lasts. It’s not teaching your body anything.”
Frank Esper, MD, a pediatric infectious disease specialist at Cleveland Clinic Children’s Hospital, said monoclonal antibody protection for RSV is particularly welcome. “We’ve been trying to make an RSV vaccine since the 1960s and have done nothing but fail miserably.”
“The best thing is always a vaccine,” Dr. Esper said, explaining that vaccines teach the body to make its own antibodies and confer long-term protection and are “probably more efficacious than anything that’s ever manmade.
“But since we’ve really not done very well for pediatric RSV vaccines, nirsevimab is certainly something I’m looking forward to,” he said.
Fast-acting monoclonal antibodies
An advantage for monoclonal antibodies is that they start working almost immediately.
Children can get sick with RSV in the first few months of life so the speed of the monoclonal antibodies to begin protection is important, Dr. Esper said, adding that RSV “is the worst during the first year of life.”
The peak age for babies getting infected enough to require hospitalization is about 2 months, he said.
By 14 months, he said, kids’ immune systems and airways have matured enough “that it’s not nearly as bad.”
To get protection from a vaccine, he added, “usually takes 2-4 weeks from the time you get your shot to the time you see some benefit. With an antibody, you’re bypassing the processing that the body has to do, and it goes straight to ‘protection’ mode,” Dr. Esper said. “You get protected pretty much as soon as you get the antibody.”
A version of this article first appeared on Medscape.com.
For the first time in the fall of 2023, families will be offered season-long protection for infants and some children against respiratory syncytial virus (RSV).
The Food and Drug Administration in July approved a prevention called nirsevimab (Beyfortus, AstraZeneca/Sanofi) and it is expected to be widely rolled out in the coming weeks as the RSV season begins.
And monoclonal antibodies are often used for treatment rather than prevention.
Adding to potential confusion is the fact the Centers for Disease Control and Prevention has included nirsevimab in the Vaccines for Children program, which covers the costs for uninsured kids and makes it more accessible.
Nirsevimab is approved for infants (up to 8 months old) born during or entering their first RSV season, and in children up to 2 years of age who are still vulnerable to severe RSV through their second season.
It’s recommended that all infants get one injection in their first 8 months for prevention instead of the previous monthly shots used to help prevent kids at high risk from getting severe RSV.
If monoclonal antibodies can be used for preventing disease in infants, could they become a viable vaccine alternative for adults?
Specialists say no.
That’s partly because of the difference in body size. Although an injection is an option for a newborn, pediatricians suggest, it would take far too much of the treatment to work as a shot for adults.
Ruth Karron, MD, an expert in pediatric infectious diseases at Johns Hopkins Medicine, Baltimore, said that, while vaccines come in small amounts and activate immune cells, monoclonal antibodies are more like a drug, with the dose based on weight.
“You’d have to give it intravenously,” for larger doses, she explained, which has never been studied before and would also be very expensive. “It really couldn’t be an option for adults.”
What’s the difference between vaccines and antibodies?
Monoclonal antibodies are proteins made in a lab to mimic the immune system’s ability to fight pathogens such as viruses.
Dr. Karron explained that a wide variety of monoclonal antibodies have long been used to treat diseases such as cancers and autoimmune disease. In recent years, the antibodies have been used to treat COVID.
Monoclonal antibodies have also been used to treat RSV in children, but the effects don’t last long – they confer passive immunity and “when it’s gone, it’s gone,” Dr. Karron said.
That means kids at high risk for severe RSV have had to get monthly injections.
But with nirsevimab, the mutated antibodies stay in circulation longer so they can last 5 or 6 months, enough to cover the RSV season, Dr. Karron explained. “It’s highly, highly effective.”
Vaccines train the body
“The idea with vaccines is that you engage the individual’s immune system. You teach it to make antibodies,” Dr. Karron said. Conversely, “you give an antibody and it’s good for as long as the antibody lasts. It’s not teaching your body anything.”
Frank Esper, MD, a pediatric infectious disease specialist at Cleveland Clinic Children’s Hospital, said monoclonal antibody protection for RSV is particularly welcome. “We’ve been trying to make an RSV vaccine since the 1960s and have done nothing but fail miserably.”
“The best thing is always a vaccine,” Dr. Esper said, explaining that vaccines teach the body to make its own antibodies and confer long-term protection and are “probably more efficacious than anything that’s ever manmade.
“But since we’ve really not done very well for pediatric RSV vaccines, nirsevimab is certainly something I’m looking forward to,” he said.
Fast-acting monoclonal antibodies
An advantage for monoclonal antibodies is that they start working almost immediately.
Children can get sick with RSV in the first few months of life so the speed of the monoclonal antibodies to begin protection is important, Dr. Esper said, adding that RSV “is the worst during the first year of life.”
The peak age for babies getting infected enough to require hospitalization is about 2 months, he said.
By 14 months, he said, kids’ immune systems and airways have matured enough “that it’s not nearly as bad.”
To get protection from a vaccine, he added, “usually takes 2-4 weeks from the time you get your shot to the time you see some benefit. With an antibody, you’re bypassing the processing that the body has to do, and it goes straight to ‘protection’ mode,” Dr. Esper said. “You get protected pretty much as soon as you get the antibody.”
A version of this article first appeared on Medscape.com.
For the first time in the fall of 2023, families will be offered season-long protection for infants and some children against respiratory syncytial virus (RSV).
The Food and Drug Administration in July approved a prevention called nirsevimab (Beyfortus, AstraZeneca/Sanofi) and it is expected to be widely rolled out in the coming weeks as the RSV season begins.
And monoclonal antibodies are often used for treatment rather than prevention.
Adding to potential confusion is the fact the Centers for Disease Control and Prevention has included nirsevimab in the Vaccines for Children program, which covers the costs for uninsured kids and makes it more accessible.
Nirsevimab is approved for infants (up to 8 months old) born during or entering their first RSV season, and in children up to 2 years of age who are still vulnerable to severe RSV through their second season.
It’s recommended that all infants get one injection in their first 8 months for prevention instead of the previous monthly shots used to help prevent kids at high risk from getting severe RSV.
If monoclonal antibodies can be used for preventing disease in infants, could they become a viable vaccine alternative for adults?
Specialists say no.
That’s partly because of the difference in body size. Although an injection is an option for a newborn, pediatricians suggest, it would take far too much of the treatment to work as a shot for adults.
Ruth Karron, MD, an expert in pediatric infectious diseases at Johns Hopkins Medicine, Baltimore, said that, while vaccines come in small amounts and activate immune cells, monoclonal antibodies are more like a drug, with the dose based on weight.
“You’d have to give it intravenously,” for larger doses, she explained, which has never been studied before and would also be very expensive. “It really couldn’t be an option for adults.”
What’s the difference between vaccines and antibodies?
Monoclonal antibodies are proteins made in a lab to mimic the immune system’s ability to fight pathogens such as viruses.
Dr. Karron explained that a wide variety of monoclonal antibodies have long been used to treat diseases such as cancers and autoimmune disease. In recent years, the antibodies have been used to treat COVID.
Monoclonal antibodies have also been used to treat RSV in children, but the effects don’t last long – they confer passive immunity and “when it’s gone, it’s gone,” Dr. Karron said.
That means kids at high risk for severe RSV have had to get monthly injections.
But with nirsevimab, the mutated antibodies stay in circulation longer so they can last 5 or 6 months, enough to cover the RSV season, Dr. Karron explained. “It’s highly, highly effective.”
Vaccines train the body
“The idea with vaccines is that you engage the individual’s immune system. You teach it to make antibodies,” Dr. Karron said. Conversely, “you give an antibody and it’s good for as long as the antibody lasts. It’s not teaching your body anything.”
Frank Esper, MD, a pediatric infectious disease specialist at Cleveland Clinic Children’s Hospital, said monoclonal antibody protection for RSV is particularly welcome. “We’ve been trying to make an RSV vaccine since the 1960s and have done nothing but fail miserably.”
“The best thing is always a vaccine,” Dr. Esper said, explaining that vaccines teach the body to make its own antibodies and confer long-term protection and are “probably more efficacious than anything that’s ever manmade.
“But since we’ve really not done very well for pediatric RSV vaccines, nirsevimab is certainly something I’m looking forward to,” he said.
Fast-acting monoclonal antibodies
An advantage for monoclonal antibodies is that they start working almost immediately.
Children can get sick with RSV in the first few months of life so the speed of the monoclonal antibodies to begin protection is important, Dr. Esper said, adding that RSV “is the worst during the first year of life.”
The peak age for babies getting infected enough to require hospitalization is about 2 months, he said.
By 14 months, he said, kids’ immune systems and airways have matured enough “that it’s not nearly as bad.”
To get protection from a vaccine, he added, “usually takes 2-4 weeks from the time you get your shot to the time you see some benefit. With an antibody, you’re bypassing the processing that the body has to do, and it goes straight to ‘protection’ mode,” Dr. Esper said. “You get protected pretty much as soon as you get the antibody.”
A version of this article first appeared on Medscape.com.