Category Archives: Pediatrics

Pediatrics

PCORI Awards CYSHCNet Researchers $5.5 Million to Study Transitions for Children and Caregivers from Hospital-to … – UNC Health and UNC School of…

Neal dejong, MD, MPH, at the UNC School of Medicine, David Y. Ming, MD, at Duke University School of Medicine and Ryan Coller, MD, at University of Wisconsin School of Medicine and Public Health, received $5.5 million to study hospital-to-home transitions across settings for children and youth with special healthcare needs.

Nearly one in five children, almost 14 million in the United States, have a special healthcare need that may require hospitalization. After a childs hospital stay, the transition back home can be risky, particularly for children with chronic health issues and complex medical conditions. To understand how to best support children and youth with special healthcare needs (CYSHCN), researchers of the CYSHCN National Research Network (CYSHCNet) received $5.5 million for a multi-site studyfunded by the Patient-Centered Outcomes Research Institute (PCORI)to compare two types of hospital-to-home transitional care to optimize outcomes.

The CYSHCNet researchers are led by David Y. Ming, MD, principal investigator, associate professor of pediatrics at Duke University School of Medicine; Neal dejong, MD, MPH, associate professor of pediatrics and member of the UNC Childrens Research Institute at the UNC School of Medicine; and Ryan Coller, MD, associate professor of pediatrics at the University of Wisconsin School of Medicine and Public Health.

Over a one-month post-discharge period, researchers will compare the effectiveness of two transitional care approaches focused doseversusextended dose reducing return visits to the hospital or emergency department and increasing parent-reported confidence. They will also evaluate the impact of each type of hospital-to-home transitional care on specific CYSHCN patient groups, including those with highly complex clinical needs and those from racial/ethnic minority groups or living in rural areas. Lastly, researchers will investigate how and why each approach to hospital-to-home transitional care is effective and for whom they work best.

The study team includes multiple community partners, including parents and caregivers with lived experience, clinicians, policymakers, and health system leaders. A community advisory board, primarily composed of a diverse group of parents/caregivers of CYSHCN, will work closely with the research team throughout the study. This collaborative effort aims to contribute to the equitable improvement of overall care for all CYSHCN transitioning from hospital to home.

About CYSHCNet:

CYSHCNet brings academic researchers and lived experience partners together to study the health care system issues that matter most to CYSHCN and their families. We help CYSHCN and their families by advancing knowledge and implementation of optimal health systems through innovative, rigorous multi-site studies, far reaching dissemination of findings, meaningful partnerships with families, and strong training of emerging CYSHCN investigators. Learn more:https://cyshcnet.org/.

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PCORI Awards CYSHCNet Researchers $5.5 Million to Study Transitions for Children and Caregivers from Hospital-to ... - UNC Health and UNC School of...

Pediatrics

NYC medical school will go tuition-free thanks to $1 billion gift from former pediatrics professor – Anchorage Daily News

By Associated Press

Updated: 1 day ago Published: 1 day ago

The Albert Einstein College of Medicine in New York City. The school will be tuition-free for all students from now on thanks to a $1 billion donation from a former professor, the widow of a Wall Street investor. (Jason Torres/Albert Einstein College of Medicine via AP)

NEW YORK A New York City medical school will be tuition-free for all students from now on thanks to a $1 billion donation from a former professor, the widow of a Wall Street investor.

Ruth Gottesman announced the gift and its purpose to students and faculty at Albert Einstein College of Medicine Monday, bringing some in the audience to tears and others to their feet, cheering. Gottesman, 93, has been affiliated with the college for 55 years and is the chairperson of its board of trustees.

The gift is intended to attract a diverse pool of applicants who otherwise might not have the means to attend. It will also let students graduate without debt that can take decades to repay, college administrators said. Tuition at Einstein is $59,458 per year. The average medical school debt in the U.S. is $202,453, excluding undergraduate debt, according to the Education Data Initiative.

Each year, well over 100 students enter Albert Einstein College of Medicine in their quest for degrees in medicine and science, Gottesman said. They leave as superbly trained scientists and compassionate and knowledgeable physicians, with the expertise to find new ways to prevent diseases and provide the finest health care.

Gottesman credited her late husband, David Sandy Gottesman for leaving her with the financial means to make such a donation. David Gottesman built the Wall Street investment house, First Manhattan, and was on the board of Warren Buffetts Berkshire Hathaway. He died in 2022 at age 96.

l feel blessed to be given the great privilege of making this gift to such a worthy cause, Ruth Gottesman said.

The gift is believed to be the largest made to any medical school in the country, according to Montefiore Einstein, the umbrella organization for Albert Einstein College of Medicine and the Montefiore Health System.

I believe we can change health care history when we recognize that access is the path to excellence, said Dr. Philip Ozuah, president and chief executive of Montefiore Einstein.

Gottesman joined Einsteins Childrens Evaluation and Rehabilitation Center in 1968 and developed screening and treatments for learning problems. She started the first-of-its-kind Adult Literacy Program at the center in 1992, and in 1998 was named the founding director of the Emily Fisher Landau Center for the Treatment of Learning Disabilities at CERC. She is clinical professor emerita of pediatrics at Einstein.

Through their foundation, the Gottesman Fund, the family has supported charities in Israel and within the U.S. Jewish community, especially through gifts to schools, universities and New York Citys American Museum of Natural History.

Einstein becomes the second tuition-free medical school in New York. In 2018, New York University School of Medicine announced that it would cover the tuition of all its students.

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NYC medical school will go tuition-free thanks to $1 billion gift from former pediatrics professor - Anchorage Daily News

Pediatrics

Innovative New Pediatric Facility Helps Alleviate Patient Anxiety – Healthcare Construction and Operations News

By HCO Staff

SPRINGFIELD, Ill.For many children, a trip to the doctor can be anxiety-inducing or even downright scary. Pediatricians offices have long tried to combat that fear by creating a softer and more child-friendly environment with toys, games and colorful dcor, but what if the building itself was designed from the ground-up to directly address and distract from childrens anxiety starting from the moment they pull into the parking lot?

This was the concept behind Lawrence Groups design of the new $23 million Springfield Clinic pediatric center in Springfield, Ill. a 41,500-square-foot facility consolidating multiple pediatric practices across Springfield under one roof, including general pediatrics, dermatology, lab and radiology services. The facility officially welcomed its first patients on Oct. 16, 2023. The general contractor on the project was OShea Builders of Springfield, Ill.

The project came to the design team through a longstanding relationship between one of Lawrence Groups business development professionals and the Vice President of Facilities, Real Estate and Construction at Springfield Clinic.

They were looking for something unique and different that still supported their brand but had a new approach, and it had to appeal to a wide range of ages from newborns to young adults in their early twenties, so thats where our expertise in ground-up construction and healthcare and hospitality design came into play, said Lawrence Group Director of Interiors Julie Steffens, NCIDQ.

The building was designed inside out with pediatric patients and their caregivers in mind, employing an on stage (patients) and off stage (staff and providers) model that provides complete separation between these two groups for a seamless patient experience.

Its a trend in healthcare design that a lot of organizations are moving to from that experiential standpoint, Steffens said.

The model was one that Springfield Clinic already had in the works at another facility under construction in Jackson, Ill. Clinic administrators wanted to bring that same methodology and branding to all its clinics. With four pediatric practices across Springfield merging into one space under this new concept, the biggest question was whether it would work for all of them.

From a culture standpoint and operational flow, theres a lot of change that they are going to be going through for the good of the organization itself as well as the patient experience, said Steffens.

Once there was buy-in from the various stakeholders, Lawrence Group proceeded to focus on creating a unique healthcare experience through interior and exterior design with a guiding principle of creating elements of distraction and discovery around every corner to reduce the stress of visiting patients.

The whole idea of this project is about a journey starting with that ride to the doctors office and that connection between a parent and child or a caregiver and a child, said Steffens. Those elements of distraction start when they arrive on site with strategically placed walking paths for safety that have these moments of discovery embedded with footprints and leaves, and exterior sculptures even before they get into the interior.

Lawrence Group Design Architect Kevin Le used multi-hued terracotta baguettes across the buildings faade, strategically placed to create an immediate visual impression as visitors pull up to and enter the building. The baguettes rise above the roofline on a slope, allowing the sky to peek through.

They wanted to be different and bring the future to this area as opposed to blending with what is there, furthering their mission and their brand identity, said Lawrence Group Architect and Project Manager Ganesh Sathyan. We wanted to continue that feeling going inside, one surprise after another.

Those surprises include large fish tanks, a slide that goes from the second floor to the first and a significant amount of artwork throughout the spaces, both on-stage and off-stage. Lawrence Group worked with St. Louis art consultancy TOC Artwork to curate a variety of both limited-edition and commissioned pieces, including two kinetic art sculptures installed on the exterior.

TOC really listened and tailored the artwork selections to the providers vision. Its pretty unique to Springfield and they did tap into some local artists in the community for a few pieces, said Steffens. The providers were so heavily invested in the art and that was the one piece where they could really personalize their spaces. Many of them use the artwork as a point of conversation to engage the patient and get them talking about something thats familiar to them.

The artwork was carefully selected to tie into the color story created by the interior design team, with six colors used as a means of wayfinding. Each clinic corridor is identified by a unique color, thoughtfully applied so as to not be overwhelming when entering the space. More neutral fixed elements are brightened with pops of color in the artwork, furniture and accents. The design concept also had to support Springfield Clinics brand, so the interior design team worked closely with the clinics marketing and brand experience team to ensure proper execution.

No project is without its challenges, and the biggest one for Springfield Clinic was its mid-pandemic timeline. With work starting early in 2020, the subsequent shutdowns caused substantial schedule shifts. Delays and shortages of materials also required advanced planning. The projects general contractor guided the Lawrence Group team on acquiring certain materials, like metal decking, long before they were needed in order to ensure procurement. Other materials had to be fully substituted due to extensive delays.

The pandemic taught us a lot and we experienced a lot of circumstances that forced us to think differently about how a project gets put together and gets built, Sathyan said. In healthcare, very little for us has been cookie cutter. Every client is different and every solution is different. What we pride ourselves on is the fact that were able to translate the dreams of our clients into reality. The client came to us with a vision to be a premier pediatric facility, and this is the end product.

Throughout the entire design/build process, the Lawrence Group team kept the patient experience as its guiding principles for all decisions and solutions, knowing the profound impact the completed facility would have on the communitys youngest members.

From the beginning, the entire team from the design team to the client side really came together and worked transparently and creatively. The final product is the result of trust, respect and team collaboration, said Steffens.

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Innovative New Pediatric Facility Helps Alleviate Patient Anxiety - Healthcare Construction and Operations News

Pediatrics

Most pediatricians aren’t prepared to handle teen opioid use, study says – WCVB Boston

Even with teen opioid overdose deaths soaring, a new study finds most pediatricians aren't prepared to treat young patients struggling with addiction. The nationwide survey was conducted by researchers at Mass General for Children and Yale School of Medicine. Of those pediatricians surveyed, 48% said they felt prepared to counsel teen patients struggling with opioid use. That's despite the fact nearly all of those surveyed believe it's their responsibility to do so. And, even though 24% said they'd diagnosed Opioid Use Disorder in a young patient, just 5% said they had prescribed any medication considered the standard of care for the disorder. Dr. Scott Hadland, an addiction specialist at Mass General for Children and the lead author on the study, said increasing training is critical when it comes to bridging this gap. That includes starting opioid use education in medical school and continuing it throughout a pediatrician's career, he said. Hadland also stressed that correcting the situation is vital to protect our kids. "It's so important because sometimes the hook, the thing that makes a teenager want to come back and talk to you more is not actually about their drug use; it's about something else," he said. "For example, I might be caring for a teenager who's struggling with acne and also struggling with opioids and they may not be ready to talk to me about their opioid use, but I can help address their acne and that gives me an opportunity to continuously engage them and work at that substance use with them."The survey is published in JAMA Pediatrics.

Even with teen opioid overdose deaths soaring, a new study finds most pediatricians aren't prepared to treat young patients struggling with addiction.

The nationwide survey was conducted by researchers at Mass General for Children and Yale School of Medicine.

Of those pediatricians surveyed, 48% said they felt prepared to counsel teen patients struggling with opioid use. That's despite the fact nearly all of those surveyed believe it's their responsibility to do so.

And, even though 24% said they'd diagnosed Opioid Use Disorder in a young patient, just 5% said they had prescribed any medication considered the standard of care for the disorder.

Dr. Scott Hadland, an addiction specialist at Mass General for Children and the lead author on the study, said increasing training is critical when it comes to bridging this gap. That includes starting opioid use education in medical school and continuing it throughout a pediatrician's career, he said.

Hadland also stressed that correcting the situation is vital to protect our kids.

"It's so important because sometimes the hook, the thing that makes a teenager want to come back and talk to you more is not actually about their drug use; it's about something else," he said. "For example, I might be caring for a teenager who's struggling with acne and also struggling with opioids and they may not be ready to talk to me about their opioid use, but I can help address their acne and that gives me an opportunity to continuously engage them and work at that substance use with them."

The survey is published in JAMA Pediatrics.

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Most pediatricians aren't prepared to handle teen opioid use, study says - WCVB Boston

Pediatrics

As Medicaid Shrinks, Clinics for the Poor Are Trying to Survive – The New York Times

Appointment cancellations and financial distress have become a constant at Bethesda Pediatrics, a nonprofit medical clinic in East Texas that is heavily dependent on Medicaid, the health insurance program for the poor.

On a recent Monday, the mother of a toddler who had a primary care appointment broke down in tears after learning the child had just lost Medicaid coverage, wondering how she could pay the bill.

Another mother told Dr. Danny Price, the clinics lead pediatrician, that she was afraid to get her child a flu shot because of the $8 fee she would have to pay now that the child had been dropped from Medicaid.

A child with depression did not show up, most likely, Dr. Price presumed, because of having lost Medicaid coverage.

The uncertainty and panic at the clinic, tucked inconspicuously in a poor residential pocket of Tyler, Texas, highlight a little-examined consequence of the vast trimming of the Medicaid rolls since a policy that barred states from kicking anyone out of the program during the pandemic ended last spring. The loss of coverage has not only affected families, but is also threatening the financial stability of vital components of the American safety net.

Medicaid payments are the lifeblood of our health centers and their ability to serve, said Dr. Kyu Rhee, the president and chief executive of the National Association of Community Health Centers, which treat roughly one in 11 people in the United States and rely on Medicaid and federal grants to provide a financial cushion for the uncompensated care they give uninsured patients.

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As Medicaid Shrinks, Clinics for the Poor Are Trying to Survive - The New York Times

Pediatrics

Many children hospitalized with COVID continue to face challenges. – Baltimore Sun

A new study by the Kennedy Krieger Institute in Baltimore and 11 other medical sites found that up to a third of children who were hospitalized because of COVID-19 experienced persistent symptoms one to two years after they were released.

The research, published earlier this month in Frontiers in Pediatrics, analyzed the post-hospital quality of life for children previously diagnosed with COVID-19 or multisystem inflammatory syndrome in children a rare condition associated with COVID where body parts like the heart, lungs, kidneys, brain, skin and eyes become inflamed.

Researchers found that 30% of the families participating in the study reported that their child had not fully recovered from COVID or the syndrome also known by its initials as MIS-C.

Of the children who hadnt recovered following their hospital discharge, the majority 87% had neurological symptoms, Kennedy Krieger said in a news release this week. About three in five children continued to struggle with headaches and just under half experienced weakness.

These numbers speak volumes about the lasting effects that COVID has on some children, said Dr. Beth Slomine, co-director at Kennedy Kriegers Brain Injury Clinical Research Center and one of the studys co-authors.

Our findings highlight the urgent need for better monitoring systems and care strategies to address the long-term effects that the pandemic made on pediatric health, said Slomine, who is also Kennedy Kriegers assistant vice president of psychology.

The study included 79 children from 12 medical centers across the country and lasted from January 2020 to July 2021 during the coronavirus pandemic. It is part of a global research effort to better understand how often people hospitalized with COVID experience neurological problems once they are released.

Other important findings from the study included that of the children who had not recovered from COVID, 40% had at least one return emergency visit and 24% were readmitted to the hospital. These children also had increased signs of inflammation when they were admitted to the hospital, compared to those whose parents reported them being recovered.

Researchers also noted that children in the not recovered category were more likely to be from families who had reported changes in their routines, stress, food access and access to mental health treatment during the pandemic.

More research is needed to better understand the relationship between pandemic-related social stress and prolonged symptoms, Slomine said in the news release.

Also in the news release, Dr. Ericka Fink lead author of the study and a critical care pediatrician at the UPMC Childrens Hospital of Pittsburgh said the next step for researchers is to dig deeper into understanding factors that could help predict which patients are at risk for persistent symptoms.

In the future, she said, scientists may be able to develop a screening tool to help doctors identify those children more likely to require longitudinal, multidisciplinary care.

Most children diagnosed with COVID and long COVID a collection of ongoing, returning or new symptoms experienced after an initial COVID infection dont require hospitalization, according to Kennedy Kriegers news release. However, Slomine emphasized the importance of equipping families with information about the risks associated with the illness so they can make the best decisions for their children.

As we continue to learn about COVID-19, education and awareness remains at the forefront at Kennedy Krieger and around the world, Dr. Slomine said. Collaborative endeavors such as these are pivotal in guiding our research and ensuring the best care possible for children affected by the virus.

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Many children hospitalized with COVID continue to face challenges. - Baltimore Sun

Pediatrics

Exposure to neighborhood violence linked to unmet health needs and increased care utilization in children – News-Medical.Net

A new collaborative study between Boston Medical Center, Brigham and Women's Hospital, Boston Children's Hospital, Hennepin Healthcare Research Institute, University of Pennsylvania, and Children's Hospital of Philadelphia finds exposure to neighborhood violence among children was associated with unmet health needs and increased acute care utilization. Published in the American Journal of Preventive Medicine and based on nationally representative data on violence exposure and gold standard access to care measures from the National Health Interview Survey, this study shows that evidence-based interventions to improve access to care in communities impacted by violence are needed to mitigate long-term physical and mental health consequences for children.

Millions of children in the U.S. are exposed to violence in their homes or communities. Research has shown that children exposed to violence have worse school performance in childhood, increased rates of substance use disorder in adolescence, increased rates of anxiety, depression, and post-traumatic stress disorder in adulthood, and increased risk of developing chronic medical conditions throughout their lives. Exposure to violence also deepens child health inequities, particularly in marginalized communities disproportionately exposed to violence due to systemic racism across generations.

This study helps examine violence exposure at the population level as both a direct driver of health inequities and as a consequence of fundamental causes like racism, poverty, and other structural risk conditions. The researchers found that, even after controlling for the effects of other important factors like family income and insurance status, children exposed to neighborhood violence face unmet physical and mental health care needs, cost-related barriers, decreased access to prescription drugs, increased urgent care and emergency department utilization, and decreased access to preventive care, mental health care, and medications.

The study also identified an association between exposure to neighborhood violence and mental health symptoms, including increased rates of depression and anxiety, which was consistent with multiple previous studies. Researchers also found that children exposed to violence have higher rates of delayed and forgone mental health care despite experiencing more mental health symptoms. Prior research shows how early access to care can mitigate the mental health consequences of violence exposure; improving access to high-quality, affordable mental health care services remains critical in communities impacted by neighborhood violence.

Researchers call attention to built-in environment reforms like neighborhood greening and cleanup, poverty alleviation interventions like the expanded Child Tax Credit, insurance coverage protections like continuous Medicaid enrollment, and hospital-based violence prevention programs as opportunities to take action.

Our findings highlight the profound impacts of limited access to care in communities affected by violence. We also identify specific opportunities for evidence-based clinician, health system, and policy actions that can reduce the incidence of neighborhood violence and mitigate its health consequences."

Rohan Khazanchi, MD, MPH, lead author,resident in the Harvard Internal Medicine-Pediatrics Residency Program at Brigham & Women's Hospital, Boston Children's Hospital, and Boston Medical Center

"To improve health outcomes for the millions of children in the United States affected by neighborhood violence, we have to invest in their families and communities," said senior author Aditi Vasan, MD, MSHP, a pediatrician at Children's Hospital of Philadelphia and Assistant Professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine. "Our findings underscore the need for both upstream policy interventions, like the expanded Child Tax Credit, which would provide essential economic support for families in low-income communities impacted by violence, and downstream health system interventions focused on improving access to high-quality, trauma-informed care for these children and their families."

Source:

Journal reference:

Khazanchi, R.,et al.(2024) Health Care Access and Use Among U.S. Children Exposed to Neighborhood Violence.American Journalof Preventive Medicine.doi.org/10.1016/j.amepre.2024.01.009.

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Exposure to neighborhood violence linked to unmet health needs and increased care utilization in children - News-Medical.Net

Pediatrics

OUtMATCH: Omalizumab Proves Utility in Multiple Food Allergies for Pediatric, Adult Patients – MD Magazine

R. Sharon Chinthrajah, MD Credit: Stanford Medicine

The management of food allergies has undergone a revolution in recent years, with the field propelled into the future by the development of clinical programs and pipeline items, including oral immunotherapy and epicutaneous immunotherapies.

Now, in early 2024, the field welcomed what could be the most significant breakthrough in its history, with the February 16, 2024 US FDA approval of omalizumab (Xolair) for the treatment of multiple food allergies.1

The basis of this approval, the OUtMATCH trial, was presented in full at the2024 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting.2,3

Im excited that we have a promising new treatment for multifood allergic patients. This new approach showed really great responses for many of the foods that trigger their allergies, said senior investigatorSharon Chinthrajah, MD, associate professor of medicine and of pediatrics, and the acting director of theSean N. Parker Center for Allergy and Asthma Researchat Stanford Medicine.3

Named the Omalizumab as Monotherapy and as Adjunct Therapy to Multi-Allergen Oral Immunotherapy (OIT) in Food Allergic Children and Adults (OUtMATCH) trial, the study was designed as a double-blind, randomized, placebo-controlled trial with the intent of comparing omalizumab, a monoclonal anti-IgE antibody, against placebo therapy in management of multiple foods allergies. Conducted at 10 sites across the US, the trial screened 462 people aged 1 to 55 years with a history of allergy to peanut and at least 2 other foods from a protocol-specified list, including cashew, milk, egg, walnut, wheat, and hazelnut.2

Of the 462 who underwent screening, 180 were randomized in a 2:1 ratio to receive either omalizumab or placebo administered subcutaneously, with the dose based on weight and IgE levels, every 2 to 4 weeks for 16 to 20 weeks. For inclusion in the trial, participants were required to have a reaction to a food challenge of 100 mg or less of peanut protein and 300 mg or less of 2 of the other aforementioned foods. At the conclusion of the treatment period, the challenges were repeated.2

The primary outcome of interest for the trial was consumption of at least a single dose of at least 600 mg of peanut protein without dose-limiting symptoms at the completion of the first stage of the trial. The trial also included 3 key secondary outcomes of interest: the consumption of cashew, of milk, and of egg in single doses of at least 1000 mg each without dose-limiting symptoms.2

The overall study cohort had a median age of 7.0 years and 56% of the participants were boys. Investigators highlighted the trial population is representative of patients with multiple food allergies except for a lower percentage of Hispanic participants than in the general population. Investigators noted the analysis population included 177 individuals aged 1 to 17 years.2

Upon analysis, the primary outcome was achieved among 67% (79 of 118) of patients receiving omalizumab. In contrast, this occurred among just 7% (4 of 59) patients receiving placebo therapy (P < .001). Analysis off secondary outcomes of interest revealed a similar trend, with a greater rate of patients in the omalizumab group able to consume at least 1000 mg of cashew (41% v 3%), milk (66% vs 10%), and egg (68% vs 0) than their counterparts receiving placebo (P for all < .001).2

Analysis of safety outcomes in the trial revealed the incidence and severity of adverse events were similar between the study arms, except for injection site-related events, which were more common among the omalizumab group. Of note, a single serious adverse event occurred in a 1-year-old participant within the omalizumab group. In this child, liver enzyme levels became elevated during the first stage of the trial, but, after discontinuing treatment and a complete evaluation, investigators concluded that omalizumab was unlikely to be the cause.2

Chinthrajah noted, although this breakthrough adds a much-needed tool to the armamentarium of clinicians, the results of the trial provoke additional questions surrounding optimal management as well as the pathophysiology of food allergies.3

We have a lot of unanswered questions: How long do patients need to take this drug? Have we permanently changed the immune system? What factors predict which people will have the strongest response? Chinthrajah added.3We dont know yet.

References:

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OUtMATCH: Omalizumab Proves Utility in Multiple Food Allergies for Pediatric, Adult Patients - MD Magazine

Pediatrics

Neffy pharmacokinetic profiles compared to epinephrine injections – Contemporary Pediatrics

Sarina Tanimoto, MD, MBA, chief medical officer, ARS Pharma | Image Credit: ARS Pharma

Neffy (epinephrine nasal spray; ARS Pharma) is a potential needle-free option to treat type 1 allergic reactions, including anaphylaxis in adults and children weighing more than 66 lbs.1

ARS Pharmaceuticals recently announced topline data for repeat doses of neffy, compared to repeat doses of epinephrine intramuscular (IM) injections with and without nasal allergen challenge (NAC) conditions in response to the FDA's request of a pharmacokinetic (PK) and pharmacodynamic (PD) study issued in a September 2023 Complete Response Letter (CRL).1

Sarina Tanimoto, MD, MBA, chief medical officer, ARS Pharma, explains data from the study "Pediatric doses of neffy (intranasal nasal spray) demonstrate pharmacokinetic profiles that are equivalent to epinephrine injections products," in the Q+A interview below.

Data will be presented at the 2024 American Academy of Allergy, Asthma, & Immunology Annual Meeting (AAAAI) in Washington DC from February 23 to February 26, 2024.

Contemporary Pediatrics:

Can you explain how this study was organized, and what methods were used to compare pharmacokinetic profiles between neffy and epinephrine injection products?

Sarina Tanimoto, MD, MBA:

This study wasorganized asa single-arm, open-label [Pharmacokinetic-Pharmacodynamic] (PK/PD) study dosingneffy0.65 mg,1 mg or 2 mg (depending on subject weight) with PK/PD modeling to bridge to the data collected with injection in adults.A total of 80 total [individuals] were enrolled during the pediatric development of neffy. The 42 [individuals] presented in this AAAAI 2024 poster represents the data for the 2 doses (1 mg and 2 mg) dosed in the intended population for approval 1 mg for 15-30 kg [individuals], and 2 mg for 30 kg + [individuals].

[The] age of [participants] ranged from 4 years to 17 years old. Blood samples were collected over 120 minutes to determine how much epinephrine was absorbed after administration ofneffyand PD measures included blood pressure and heart rate which are directly impacted by epinephrine exposure.

The reason for the single-arm, open-label design was that FDA agreed it was not practical to conduct a randomized, crossover PK/PD study in children thatincluded injection as a comparator due to a variety of factors including the intense study conduct with respect to blood draws and duration, and general difficulty enrolling children wiling to be injected with epinephrine.FDA requested 10 [individuals] per dose group, but Europe requested 20 [individuals] per dose group, which is why this study has 21 [individuals] per dose group (for each 1 mg and 2 mg).

Separately from this EPI-10 study, to compare these data to injection, integrated PK analyses, including pharmacological base absorption model (PBAM) and a population pharmacokinetics assessments (POP PK) model werealsoused to extrapolate results from 0.3 mg IM injection and 0.3 mg EpiPen in our adult studies to pediatric subjects. These analyses demonstrated thatneffyachieved exposures in pediatrics that are comparable to or slightly greater than that observed withinjection inadults.

Contemporary Pediatrics:

Can you explain the make-up of the study?

Tanimoto:

EPI 10 included a total of 80 doses of neffy. Twelve [individuals] with body weight 15-30 kg received 0.65 mgneffyand 21 [individuals] with body weight 15-30 kg received 1 mgneffy. In the higher weight group of 30 kg or greater, 26 subjects received a 1 mg dose ofneffyand 21 subjects received a 2 mg dose ofneffy.

Thedata presented in this poster from the study included a total of 42 pediatric [individuals] that received the commercial doses ofneffywhich will be 1 mg for children 15 to <30 kg and 2 mg for children 30 kg or greater.

Half the [individuals] (n = 21) were 15-30 kg in weight, while the other half (n = 21) were 30kg+ in weight. Age range of subjects in the study is 4 to 17 years.

Contemporary Pediatrics:

Can you highlight the benefits of neffy compared to epinephrine injections, of course acknowledging there is no needle. What other benefits are there for neffy regarding the pediatric indication?

Tanimoto:

The literature indicates that parents are fearful and anxious to inject their own children with an epinephrine autoinjector, which causes them to hesitate and potentially allow an allergic reaction to progress to a more serious and potentially life-threatening event.

Parents also worry whether caretakers, guardians or teachers will be willing or know how to use the autoinjector device when the parent is not present given their complexity and risk of injury due to the needle. There are more than 3,500 accidental self-injections in the [United States] annually with epinephrine autoinjectors according to National Poison Center data.

Furthermore, pediatric patients are particularly fearful of being injected or self-injecting. There are very routinereports of pediatric patients resisting injection, and twisting and jerking, which led to lacerations and injuries due to the needle.

Neffy, as an alternative to injection, addresses all these concerns in pediatric patients by removing the fear and anxiety associated with the needle as neither children nor untrained adults can harm the child or themselves withneffy.

Autoinjectors are also large and bulky, which makes it less likely for children to carry the device; there is some social stigma that has been reported with carrying the device around, especially among adolescents. Literature indicates that only 45% of pediatric subjects carry one epinephrine autoinjector with them, and only 22% of pediatric subjects carry 2 devices, as recommended by treatment guidelines.2

Neffyis easy for children to carry as it is much smaller than autoinjectors, fitting in a pocket in a convenient carrying case that can fit 2 devices as guidelines recommend (since a second dose is required in ~10% of events) just like a wireless earbuds case.

Many pediatric patients and their caregivers do not correctly use their EpiPen and other autoinjector devices. In one published study, only 32% of pediatric subjects correctly demonstrated use of the epinephrine autoinjector.3 In another randomized controlled study, only 43% of mothers were able to use an epinephrine autoinjector after training.4

Neffyis also easier to use than an autoinjector as 100% of children were able to useneffy in formal human factors studies after training, and even 100% of adult lay people (e.g. third-party caregivers) who have no knowledge of anaphylaxis were able to useneffywithout any training. Therefore, both children and their caregivers may more easily be able to useneffyconfidently at the first sign of an allergic reaction without anxiety of fear, or risk of injury from the needle.

References:

1. Fitch, J. Neffy epinephrine nasal spray demonstrates positive data in repeat dosing study. Contemporary Pediatrics. February 21, 2024. Accessed February 23, 2024. https://www.contemporarypediatrics.com/view/neffy-epinephrine-nasal-spray-demonstrates-positive-data-repeat-dosing-study

2. Warren CM, Zaslavsky JM, Kan K, Spergel JM, Gupta RS. Epinephrine auto-injector carriage and use practices among US children, adolescents, and adults.Ann Allergy Asthma Immunol. 2018;121(4):479-489.e2. doi:10.1016/j.anai.2018.06.010

3. Sicherer SH, Forman JA, Noone SA. Use assessment of self-administered epinephrine among food-allergic children and pediatricians.Pediatrics. 2000;105(2):359-362. doi:10.1542/peds.105.2.359

4.UmasuntharT,ProcktorA,HodesM,SmithJG,GoreC,CoxHE,MarrsT, HannaH,PhillipsK,PintoC,TurnerPJ,WarnerJO,BoyleRJ.Patients' ability to treat anaphylaxis using adrenaline autoinjectors: a randomized controlled trial.Allergy2015;70:855863.

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Neffy pharmacokinetic profiles compared to epinephrine injections - Contemporary Pediatrics