Anesthesiology, Perioperative and Pain

2024 with a lightbulb instead of a zero

The best of 2024 from Innovation District

2024 with a lightbulb instead of a zero2024 marked another groundbreaking year for Children’s National Hospital, showcasing remarkable advances across the spectrum of pediatric medicine, research and healthcare innovation. From pioneering surgical procedures to breakthrough artificial intelligence applications, the institution continued to push the boundaries of what’s possible in children’s healthcare. Read on for our list of the most popular articles we published on Innovation District in 2024.

1. Prenatal COVID exposure associated with changes in newborn brain

A study led by researchers at Children’s National Hospital showed that babies born during the COVID-19 pandemic have differences in the size of certain structures in the brain, compared to infants born before the pandemic. The findings suggest that exposure to the coronavirus and being pregnant during the pandemic could play a role in shaping infant brain development.
(3 min. read)

2. Children’s National Hospital again ranked among the best in the nation by U.S. News & World Report

Children’s National Hospital was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.
(2 min. read)

3. Children’s National performs first ever HIFU procedure on patient with cerebral palsy

In January 2023, a team of multidisciplinary doctors performed the first case in the world of using bilateral high intensity focused ultrasound (HIFU) pallidotomy on Jesus, a 22-year-old patient with dyskinetic cerebral palsy. The procedure is part of a clinical trial led by Chima Oluigbo, M.D., pediatric neurosurgeon at Children’s National Hospital.
(3 min. read)

4. Novel ultrasound device gets FDA breakthrough designation with Children’s National support

A novel ultrasound device developed by Bloom Standard received the Food and Drug Administration’s valued breakthrough device designation with the help of Children’s National Hospital. The device that enables autonomous, hands-free ultrasound scans to be performed anywhere, by any user.
(2 min. read)

5. First-of-its-kind pilot study on the impacts of Lyme disease in pregnancy and infant development

Understanding the effects of Lyme disease on the developing fetal brain is essential to ensure timely prenatal and postnatal treatments to protect the fetus and newborn. In response to this need, Children’s National Hospital is leading a pilot study to establish the groundwork needed for a larger study to determine the effect of in utero exposure to Lyme disease on pregnancy and early childhood neurodevelopmental outcomes.
(3 min. read)

6. Earliest hybrid HLHS heart surgery kids thrive 5 years later

Five years ago, Cayden was born 6 weeks early weighing less than four pounds and at risk of dying from her critical congenital heart disease. Today, she’s a happy five-year-old. Early diagnosis of her hypoplastic right ventricle, double inlet left ventricle and critical coarctation of the aorta allowed for the team at Children’s National Hospital to create a careful plan for safe delivery and to offer an innovative hybrid HLHS surgical approach at the hospital within 24 hours after she was born.
(1 min. read)

7. Wayne J. Franklin, M.D., F.A.C.C., named senior vice president of the Children’s National Heart Center

Children’s National Hospital appointed Wayne J. Franklin, M.D., F.A.C.C., as the new senior vice president (SVP) of the Children’s National Heart Center. In this role, Dr. Franklin oversees the full spectrum of heart care services including cardiac imaging and diagnostics, interventional cardiology, electrophysiology, cardiac anesthesia, cardiac surgery and cardiac intensive care.
(2 min. read)

8. Artificial – and accelerated – intelligence: endless applications to expand health equity

By pioneering artificial intelligence (AI) innovation programs at Children’s National Hospital, Marius George Linguraru, D.Phil., M.A., M.Sc., and the AI experts he leads are ensuring patients and families benefit from a coming wave of technological advances. The team is teaching AI to interpret complex data that could otherwise overwhelm clinicians.
(4 min. read)

9. Evidence review: Maternal mental conditions drive climbing death rate in U.S.

Painting a sobering picture, a research team led by Children’s National Hospital culled years of data demonstrating that maternal mental illness is an under-recognized contributor to the death of new mothers. They called for urgent action to address this public health crisis.
(3 min. read)

10. Nathan Kuppermann, M.D., M.P.H., named chief academic officer and chair of Pediatrics

Children’s National Hospital appointed Nathan Kuppermann, M.D., M.P.H., as its new executive vice president, chief academic officer and chair of Pediatrics. In this role, Dr. Kuppermann oversees research, education and innovation for the Children’s National Research Institute as well as academic and administrative leadership in the Department of Pediatrics at George Washington University School of Medicine & Health Services.
(2 min. read)

11. First global clinical trial achieves promising results for hypochondroplasia

Researchers from Children’s National Hospital presented findings from the first clinical trial of the medication vosoritide for children with hypochondroplasia – a rare genetic growth disorder. During the phase 2 trial, researchers found vosoritide increased the growth rate in children with hypochondroplasia, allowing them to grow on average an extra 1.8 cm per year.
(2 min. read)

12. Pioneering research center aims to revolutionize prenatal and neonatal health

Since its establishment in July 2023, the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.
(3 min. read)

Julia Finkel, M.D., measures a patient's pain with the AlgometRx Nociometer, a pain measurement device to better understand and treat pain.

ARPA-H awards $8m to advance device for precision pain measurement

Pain assessment in medicine often relies on imprecise visual rating scales featuring smiling or crying faces, frustrating patients and physicians alike. Children’s National Hospital researchers aim to change this with a new device designed to precisely measure pain, backed by an $8-million award from the Advanced Research Projects Agency for Health (ARPA-H) Sprint for Women’s Health.

The sprint addresses critical unmet challenges in women’s health, champions transformative innovations and tackles health conditions that uniquely or disproportionately affect women. Children’s National researchers – with their collaborators from Johns Hopkins University and Medstar Research Institute – are furthering the development of the AlgometRx Nociometer, a pain measurement device to better understand and treat pain.

Julia Finkel, M.D., pediatric anesthesiologist and director of Pain Medicine Research at the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI) at Children’s National, said she hopes to improve the understanding of gender differences in pain and better treat patients of all ages with analgesics. Children’s National will receive the $8 million over two years through the Sprint for Women’s Health’s launchpad track for later-stage health solutions.

“As physicians, we often feel helpless to understand how our patients are experiencing pain and whether treatments are working,” Dr. Finkel said. “We use the Visual Analog Scale, which fails to classify the pain’s etiology or help guide a specific intervention, often leaving us to treat by trial and error. The Nociometer will change that.”

The patient benefit

Using a nonpainful stimulus, the portable Nociometer uses noninvasive technology and algorithms to analyze pupil dilation and the body’s response to stimulation along select nerves.  In under one minute, the data collected can formulate a patient profile quantifying how the nervous system is processing painful stimuli. This new construct can be used to characterize pain type, intensity and assess the impact of pain relief medications.

Dr. Finkel spent nine years developing the Nociometer at SZI and her spin-out company, AlgometRx, which receives commercialization support from Children’s National Innovation Ventures. She said this transformative platform technology can be applied to any population – including elderly, nonverbal or pediatric patients – and will be instrumental in studying women’s pain experiences.

“Research studies have long shown that pain in women and girls is underestimated and undertreated,” Dr. Finkel said. “Creating a novel technology to quantify pain has tremendous applications in pinpointing and effectively treating pain, potentially altering treatment in nearly any medical setting.”

The big picture

ARPA-H sought solutions within six topics of interest in women’s health and received an unprecedented response of submissions. ARPA-H launched the Sprint for Women’s Health in February, with First Lady Jill Biden announcing the funding as the first major deliverable from the White House Initiative on Women’s Health Research.

The ARPA-H Sprint for Women’s Health is conducted in collaboration with the Investor Catalyst Hub of ARPANET-H, the agency’s nationwide health innovation network that connects people, innovators and institutions to accelerate better health outcomes for everyone. Children’s National is a spoke member of the Investor Catalyst Hub.

Children’s National will work with an ARPA-H program manager and the Investor Catalyst Hub over two years to develop their proposed solution, receiving milestone-based payments aligned to research activities and performance objectives.

The ARPA-H launchpad program accelerates transformative health solutions’ path to impact by providing funding and market transition support. As a launchpad performer, Dr. Finkel will also work with an entrepreneur-in-residence and participate in Launchpad Accelerator, which includes a customized curriculum, virtual events, and in-person workshops to support the transition from research to commercialization.

2024-25 US News Badges

Children’s National again ranked among the best in the nation by U.S. News & World Report

2024-25 US News BadgesChildren’s National Hospital in Washington, D.C., was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

This year, U.S. News ended ordinal rankings on its Honor Roll. Instead of assigning a numerical rank from 1 to 10, all hospitals on the Honor Roll will be recognized as having attained the highest standards of care in the nation.

In addition, Children’s National tied for #1 pediatric hospital in the Mid-Atlantic region, which includes New York, New Jersey, Delaware, Pennsylvania, the District of Columbia, West Virginia and Virginia. It’s also best in the Mid-Atlantic in Neonatology.

For the fourteenth straight year, Children’s National ranked in 10 specialty services. New this year, U.S. News included behavioral health as a service line in the rankings. Since it’s the first year, there are no ordinal rankings for behavioral health, but the Children’s National program was named one of the top 50 programs in the country.

“In my first year here, I witnessed what makes Children’s National so special — our commitment to collaboration, empowering one another, and charting a bold path forward for pediatric care,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “I’m proud U.S. News again recognized Children’s National as one of the top in the nation and the highest-ranked pediatric hospital in D.C., Maryland and Virginia. Together, we’ll continue to push the boundaries of care, research and innovation to make a difference for those who matter most — the kids.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

“For nearly two decades, U.S. News has published Best Children’s Hospitals to empower the parents and caregivers of children with complex medical needs,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals appearing on the U.S. News Honor Roll have a track record of delivering unparalleled specialized care.”

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other four specialties ranked among the top 50 are Behavioral Health, Cardiology and Heart Surgery, Pulmonology and Lung Surgery, and Urology.

Monika Goyal, M.D., MSCE

Q&A with Dr. Goyal: Trailblazing equity work leads to election to ASCI

Monika Goyal, M.D., MSCE, associate division chief of Emergency Medicine and Trauma Services

Monika Goyal, M.D., MSCE, associate division chief of Emergency Medicine and Trauma Services, is joining The American Society for Clinical Investigation (ASCI), one of a small cadre of pediatric emergency medicine physicians elected to the premier medical honor society for physician-scientists. Dr. Goyal considers her new honor a pivotal opportunity to represent pediatric emergency medicine, which is often underrepresented in research.

Interim Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., nominated Dr. Goyal to ASCI because of her nationally renowned work as an equity science scholar, having published more than 130 peer-reviewed manuscripts and securing more than $25 million in funding from the National Institutes of Health (NIH). “From the very beginning of her career, Dr. Goyal has been a trailblazer as it relates to her research interests,” said Dr. Bollard, also an ASCI member.

Dr. Goyal serves as the inaugural chair for Women in Science and Medicine and associate division chief for Academic Affairs and Research at Children’s National Hospital. Her academic work has focused on disparities in the emergency setting, where she has studied the impacts of gun violence, pain management, sexual health and more. In 2023, the journal Pediatrics named a Children’s National manuscript on gun violence one of the 12 most significant papers in its 75-year history. Dr. Goyal was the first author and remains tremendously proud of the impact science can make on society.

Q: How did you find yourself investigating the science of gun laws?

A: A half-dozen years ago, three other pediatric emergency medicine physicians and I had been caring for countless numbers of children who had been victims of gun violence and felt motivated to act. I started SAFER at Children’s National – Safer through Advocacy, Firearm, Education and Research – which is now an institution-wide initiative to address gun violence within our community and beyond. More than 50 individuals at Children’s National are now active in our organization.

We’ve been able to publish a lot of research in this area, including our national study looking at the association between the strictness of gun laws with firearm-related deaths in children. We found that children are more likely to die from gun violence in states with less strict gun laws. It’s not surprising, yet the recognition by the American Academy of Pediatrics demonstrates the importance of using science to understand this, and it shows how far we’ve come in the medical community. Until recently, this issue has been under-recognized and under-supported, despite it being a massive public health crisis for our children and our country.

Q: Given the pace of emergency medicine, how did you find your way to research?

A: Early in my career, much of my work focused on adolescent sexual health. I kept finding that there was racial bias in terms of who we considered to be at risk for sexually transmitted infections. This finding then motivated me to investigate and understand whether racial bias impacted other aspects of care delivery across various clinical conditions in the Emergency Department.

I started by looking at whether differences existed in pain management based on a patient’s race. In evaluating data on children nationwide who are diagnosed with appendicitis, we found that Black children are less likely to receive appropriate pain management compared to white children, even after we adjusted for pain score and illness severity. We have found similar themes with respect to pain management among children diagnosed with fractures.

Q: What can be done?

A: We are grateful to have been awarded additional NIH funding to develop and test interventions to mitigate inequities. Currently, we are studying the impact of audit and feedback through the provision of what we are calling ‘equity report cards’ and clinical decision support embedded in the electronic health record to improve racial, ethnic and language equity in pain management.

Hundreds of studies have demonstrated that this is an issue at hospitals across the country. I am proud of Children’s National for having the humility for this self-reflection and the courage to do something about it. Our work here is helping to inform efforts across the country, and I am proud of our institution’s leadership in advancing health equity through community-informed, evidence-based interventions.

Boy lying in a hospital bed, surrounded by medical equipment

Black, Hispanic children at greater risk for complications during hospitalization

Boy lying in a hospital bed, surrounded by medical equipment

The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure.

Evaluating more than 5 million pediatric hospital stays nationwide, researchers found children who are Black, Hispanic or insured with Medicaid face a greater risk of health events after surgeries than white patients, according to a new study published in the journal Pediatrics.

“We looked at the data, and we calculated the risks,” said Kavita Parikh, M.D., MSHS, medical director of Quality & Safety Research, research director of the Division of Hospital Medicine and first author on the multi-institute study. “Despite decades of focus on eliminating medical errors, we know that children continue to suffer substantial harms in hospital settings, and our study highlights where children who are Black, Hispanic or insured with Medicaid are at the greatest risk.”

The big picture

The study analyzed data from more than 5.2 million hospitalizations collected by the 2019 Kids’ Inpatient Database, a national repository of data on hospital stays. It includes a 10% sample of newborns and an 80% sample of other pediatric discharges from 4,000 U.S. hospitals. More than 80% of patients were younger than 1 year of age.

The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure. Patients who are Hispanic experienced the greatest disparity in postoperative respiratory failure, a complication that can limit breathing and ventilation.

Plausible factors cited include structural racism in the U.S. healthcare system, clinician bias, insufficient cultural responsiveness, communication barriers and limited access to high-quality healthcare.

What’s ahead

The study – “Disparities in Racial, Ethnic, and Payor Groups for Pediatric Safety Events in U.S. Hospitals” – is foundational in understanding what is happening among pediatric patients. Dr. Parikh said that researchers now must conduct further studies into these alarming disparities and qualitative work to understand drivers, with the action-oriented goal of developing interventions to improve patient safety in the hospital for all children.

“We brought together leaders in pediatric medicine, health policy and public health to analyze this data, and we are committed to taking the next steps to improve outcomes for pediatric patients,” Dr. Parikh said. “It will take more patient-centered work and research, resources and multifaceted strategies to resolve these worrying disparities for our pediatric patients nationwide.”

collage of news outlet logos

Children’s National in the News: 2023

collage of news outlet logos
Explore some of the notable medical advancements and stories of bravery that defined 2023, showcasing the steadfast commitment of healthcare professionals at Children’s National Hospital and the resilient spirit of the children they support. Delve into our 2023 news highlights for more.

1. COVID during pregnancy dramatically increases the risk of complications and maternal death, large new study finds

According to a study published in British Medical Journal Global Health, women who get COVID during pregnancy are nearly eight times more likely to die and face a significantly elevated risk of ICU admission and pneumonia. Sarah Mulkey, M.D., prenatal-neonatologist neurologist, discussed findings based on her work with pregnant women and their babies.
(Fortune)

2. Rest isn’t necessarily best for concussion recovery in children, study says

A study led by Christopher Vaughan, Psy.D., pediatric neuropsychologist, suggests that — despite what many people may presume — getting kids back to school quickly is the best way to boost their chance for a rapid recovery after a concussion.
(CNN)

3. Pediatric hospital beds are in high demand for ailing children. Here’s why

David Wessel, M.D., executive vice president, chief medical officer and physician-in-chief, explained that one reason parents were still having trouble getting their children beds in a pediatric hospital or a pediatric unit after the fall 2022 respiratory surge is that pediatric hospitals are paid less by insurance.
(CNN)

4. Anisha Abraham details impact of social media use on children: ‘True mental health crisis’

Anisha Abraham, M.D., M.P.H., chief of the Division of Adolescent and Young Adult Medicine, joined America’s Newsroom to discuss the impact social media access has had on children’s mental health.
(FOX News)

5. Saving Antonio: Can a renowned hospital keep a boy from being shot again?

After 13-year-old Antonio was nearly killed outside his mom’s apartment, Children’s National Hospital went beyond treating his bullet wounds. Read how our Youth Violence Intervention Program team supported him and his family during his recovery.
(The Washington Post)

6. Formerly conjoined twins reunite with doctors who separated them

Erin and Jade Buckles underwent a successful separation at Children’s National Hospital. Nearly 20 years later they returned to meet with some of the medical staff who helped make it happen.
(Good Morning America)

7. Asthma mortality rates differ by location, race/ethnicity, age

Shilpa Patel, M.D., M.P.H., medical director of the Children’s National IMPACT DC Asthma Clinic, weighed in on a letter published in Annals of Allergy, Asthma & Immunology, asserting that the disparities in mortality due to asthma in the United States vary based on whether they occurred in a hospital, ethnicity or race and age of the patient.
(Healio)

8. How one Afghan family made the perilous journey across the U.S.-Mexico border

After one family embarked on a perilous journey from Afghanistan through Mexico to the U.S.-Mexico border, they eventually secured entry to the U.S. where Karen Smith, M.D., medical director of Global Services, aided the family’s transition and provided their daughter with necessary immediate medical treatment.
(NPR)

9. When a child is shot, doctors must heal more than just bullet holes

With the number of young people shot by guns on the rise in the U.S., providers and staff at Children’s National Hospital are trying to break the cycle of violence. But it’s not just the physical wounds though that need treating: young victims may also need help getting back on the right track — whether that means enrolling in school, finding a new group of friends or getting a job.
(BBC News)

10. This 6-year-old is a pioneer in the quest to treat a deadly brain tumor

Callie, a 6-year-old diagnosed with diffuse intrinsic pontine glioma, was treated with low-intensity focused ultrasound (LIFU) at Children’s National Hospital and is the second child in the world to receive this treatment for a brain tumor. LIFU is an emerging technology that experts like Hasan Syed, M.D., and Adrianna Fonseca, M.D., are trialing to treat this fatal childhood brain tumor.
(The Washington Post)

11. F.D.A. approves sickle cell treatments, including one that uses CRISPR

The FDA approved a new genetic therapy, giving people with sickle cell disease new opportunities to eliminate their symptoms. David Jacobsohn, M.B.A., M.D., confirmed that Children’s National Hospital is one of the authorized treatment centers and talked about giving priority to the sickest patients if they are on Vertex’s list.
(The New York Times)

12. 6-year-old fulfils wish to dance in the Nutcracker

After the potential need for open-heart surgery threatened Caroline’s Nutcracker performance, Manan Desai, M.D., a cardiac surgeon, figured out a less invasive procedure to help reduce her recovery time so she could perform in time for the holidays.
(Good Morning America)

MRI scan of human lumbar spine

Improving outcomes of infant spinal anesthesia through new intervention program

MRI scan of human lumbar spine

New intervention program could help improve outcomes of infant spinal anesthesia.

Spinal anesthesia has a long history as an effective and safe technique to avoid general anesthesia (GA) in infants undergoing surgery. However, adverse effects of GA on infants — children younger than 1 year of age — include respiratory complications, delayed postanesthesia care unit (PACU) discharge and unplanned hospital admissions. In addition, while some reassuring studies have demonstrated the safety of anesthetic agents on the developing brain, the data are still inconclusive, particularly for patients with longer, more frequent anesthetic exposures.

The big picture

In a recent study published in the journal of Pediatric Quality and Safety, Sophie Rose Pestieau, M.D., anesthesiologist at Children’s National Hospital and senior author of the study, and other experts, created a healthcare improvement program aimed to increase the percentage of successful spinal placements as the primary anesthetic in infants undergoing circumcision, open orchidopexy or hernia repair from 11% to 50% by the end of 2019 and sustain that rate for 6 months.

What it means

The interdisciplinary team created a key driver diagram and implemented the following interventions: education of nurses, surgeons and patient families; focused anesthesiologist training on the infant spinal procedure; premedication; availability of supplies; and surgical schedule optimization.

The team collected data retrospectively by reviewing electronic medical records. The primary outcome was the percentage of infants undergoing circumcision, open orchidopexy or hernia repair who received a successful spinal as the primary anesthetic. The team tracked this measure and evaluated using a statistical process control chart.

“We were able to move the needle and successfully offer this technique to many more infants as we educated families on the benefits of spinal anesthesia, as well as other surgical colleagues and the perioperative nursing staff,” Dr. Pestieau said.

Why it matters

Before this quality improvement project, 11% of the circumcisions, open orchidopexies and inguinal hernia repairs were performed in infants using spinal anesthesia as the primary anesthetic. Between August 1, 2018, and February 29, 2020, 470 infants underwent circumcision, open orchidopexy or inguinal hernia repair (235 preintervention and 235 postintervention). Of these infants, 132 underwent a successful spinal anesthetic (26 preintervention and 106 postintervention). Following the interventions, spinal placements increased from 11% to 45%, and the success rate of infant spinal anesthesia increased from 73% to 89%.

This quality improvement project successfully increased the percentage of patients receiving spinal anesthesia for specific surgical procedures by increasing the number of patients who underwent successful spinal anesthesia placement.

“We continue to grow the program by expanding the use of spinal anesthesia to other surgical procedures,” Dr. Pestieau concluded.

The project aimed to increase the number of urological procedures successfully performed under spinal anesthesia in children less than 1 year of age. The interventions increased the number of patients who underwent spinal anesthesia placement. They also increased the success rate of spinal anesthesia placements. Further quality improvement efforts may increase the number of infants considered for infant spinal anesthesia as primary anesthesia for their procedure, beyond circumcision, open orchidopexy and inguinal hernia repair.

2023 with a lightbulb

The best of 2023 from Innovation District

2023 with a lightbulbAdvanced MRI visualization techniques to follow blood flow in the hearts of cardiac patients. Gene therapy for pediatric patients with Duchenne muscular dystrophy. 3D-printed casts for treating clubfoot. These were among the most popular articles we published on Innovation District in 2023. Read on for our full list.

1. Advanced MRI hopes to improve outcomes for Fontan cardiac patients

Cardiac imaging specialists and cardiac surgeons at Children’s National Hospital are applying advanced magnetic resonance imaging visualization techniques to understand the intricacies of blood flow within the heart chambers of children with single ventricle heart defects like hypoplastic left heart syndrome. The data allows surgeons to make critical corrections to the atrioventricular valve before a child undergoes the single ventricle procedure known as the Fontan.
(3 min. read)

2. Children’s National gives first commercial dose of new FDA-approved gene therapy for Duchenne muscular dystrophy

Children’s National Hospital became the first pediatric hospital to administer a commercial dose of Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of pediatric patients with Duchenne muscular dystrophy (DMD). Elevidys is a one-time intravenous gene therapy that aims to delay or halt the progression of DMD by delivering a modified, functional version of dystrophin to muscle cells.
(2 min. read)

3. New model to treat Becker Muscular Dystrophy

Researchers at Children’s National Hospital developed a pre-clinical model to test drugs and therapies for Becker Muscular Dystrophy (BMD), a debilitating neuromuscular disease that is growing in numbers and lacks treatment options. The work provides scientists with a much-needed method to identify, develop and de-risk drugs for patients with BMD.
(2 min. read)

4. First infants in the U.S. with specially modified pacemakers show excellent early outcomes

In 2022, five newborns with life-threatening congenital heart disease affecting their heart rhythms were the first in the United States to receive a novel modified pacemaker generator to stabilize their heart rhythms within days of birth. Two of the five cases were cared for at Children’s National Hospital. In a follow-up article, the team at Children’s National shared that “early post-operative performance of this device has been excellent.”
(2 min. read)

5. AI: The “single greatest tool” for improving access to pediatric healthcare

Experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere came together to discuss how pediatric specialties can use AI to provide medical care to kids more efficiently, more quickly and more effectively at the inaugural symposium on AI in Pediatric Health and Rare Diseases, hosted by Children’s National Hospital and the Fralin Biomedical Research Institute at Virginia Tech.
(3 min. read)

6. AAP names Children’s National gun violence study one of the most influential articles ever published

The American Academy of Pediatrics (AAP) named a 2019 study led by clinician-researchers at Children’s National Hospital one of the 12 most influential Pediatric Emergency Medicine articles ever published in the journal Pediatrics. The findings showed that states with stricter gun laws and laws requiring universal background checks for gun purchases had lower firearm-related pediatric mortality rates but that more investigation was needed to better understand the impact of firearm legislation on pediatric mortality.
(2 min. read)

7. Why a colorectal transition program matters

Children’s National Hospital recently welcomed pediatric and adult colorectal surgeon Erin Teeple, M.D., to the Division of Colorectal and Pelvic Reconstruction. Dr. Teeple is the only person in the United States who is board-certified as both a pediatric surgeon and adult colorectal surgeon, uniquely positioning her to care for people with both acquired and congenital colorectal disease and help them transition from pediatric care to adult caregivers.
(3 min. read)

8. First-of-its-kind holistic program for managing pain in sickle cell disease

The sickle cell team at Children’s National Hospital received a grant from the Founders Auxiliary Board to launch a first-of-its-kind, personalized holistic transformative program for the management of pain in sickle cell disease. The clinic uses an inter-disciplinary approach of hematology, psychology, psychiatry, anesthesiology/pain medicine, acupuncture, mindfulness, relaxation and aromatherapy services.
(3 min read)

9. Recommendations for management of positive monosomy X on cell-free DNA screening

Non-invasive prenatal testing using cell-free DNA (cfDNA) is currently offered to all pregnant women regardless of the fetal risk. In a study published in the American Journal of Obstetrics and Gynecology, researchers from Children’s National Hospital provided context and expert recommendations for maternal and fetal evaluation and management when cfDNA screening is positive for monosomy X or Turner Syndrome.
(2 min. read)

10. Innovation in clubfoot management using 3D anatomical mapping

While clubfoot is relatively common and the treatment is highly successful, the weekly visits required for Ponseti casting can be a significant burden on families. Researchers at Children’s National Hospital are looking for a way to relieve that burden with a new study that could eliminate the weekly visits with a series of 3D-printed casts that families can switch out at home.
(1 min. read)

11. Gender Self-Report seeks to capture the gender spectrum for broad research applications

A new validated self-report tool provides researchers with a way to characterize the gender of research participants beyond their binary designated sex at birth. The multi-dimensional Gender Self-Report, developed using a community-driven approach and then scientifically validated, was outlined in a peer-reviewed article in the American Psychologist, a journal of the American Psychological Association.
(2 min. read)

12. Cardiovascular and bone diseases in chronic kidney disease

In a study published by Advances in Chronic Kidney Disease, a team at Children’s National Hospital reviewed cardiovascular and bone diseases in chronic kidney disease and end-stage kidney disease patients with a focus on pediatric issues and concerns.
(1 min. read)

ARPA-H logo

Children’s National selected as member of ARPA-H Investor Catalyst Hub spoke network

ARPA-H logoThe hospital will advocate for the unique needs of children as part of nationwide network working to accelerate transformative health solutions.

Children’s National Hospital was selected as a spoke for the Investor Catalyst Hub, a regional hub of ARPANET-H, a nationwide health innovation network launched by the Advanced Research Projects Agency for Health (ARPA-H).

The Investor Catalyst Hub seeks to accelerate the commercialization of groundbreaking and accessible biomedical solutions. It uses an innovative hub-and-spoke model designed to reach a wide range of nonprofit organizations and Minority-Serving Institutions, with the aim of delivering scalable healthcare outcomes for all Americans.

“The needs of children often differ significantly from those of adults. This partnership reflects our commitment to advancing pediatric healthcare through innovation and making sure we’re addressing those needs effectively,” said Kolaleh Eskandanian, Ph.D., M.B.A., vice president and chief innovation officer at Children’s National. “Leveraging the strength of this hub-and-spoke model, we anticipate delivering transformative solutions to enhance the health and well-being of the patients and families we serve.”

Children’s National joins a dynamic nationwide network of organizations aligned to ARPA-H’s overarching mission to improve health outcomes through the following research focus areas: health science futures, proactive health, scalable solutions and resilient systems. Investor Catalyst Hub spokes represent a broad spectrum of expertise, geographic diversity and community perspectives.

“Our spoke network embodies a rich and representative range of perspectives and expertise,” said Mark Marino, vice president of Growth Strategy and Development for VentureWell and project director for the Investor Catalyst Hub. “Our spokes comprise a richly diverse network that will be instrumental in ensuring that equitable health solutions reach communities across every state and tribal nation.”

As an Investor Catalyst Hub spoke, Children’s National gains access to potential funding and flexible contracting for faster award execution compared to traditional government contracts. Spoke membership also offers opportunities to provide input on ARPA-H challenge areas and priorities, along with access to valuable networking opportunities and a robust resource library.

Alliance for Pediatric Device Innovation consortium members

Children’s National awarded nearly $7.5 million by FDA to lead pediatric device innovation consortium

Alliance for Pediatric Device Innovation consortium membersChildren’s National Hospital was awarded nearly $7.5 million in a five-year grant to continue its leadership of an FDA-funded pediatric device consortium. Building upon a decade of previous consortium leadership, the new consortium is Alliance for Pediatric Device Innovation (APDI) and features a new and expanded roster of partners that reflects its added focus on providing pediatric innovators with expert support on evidence generation, including the use of real-world evidence (RWE), for pediatric device development.

Collaborating for success

With the goal of helping more pediatric medical devices complete the journey to commercialization, APDI is led by Children’s National, with Kolaleh Eskandanian, Ph.D., M.B.A., vice president and chief innovation officer, serving as program director and principal investigator, and Julia Finkel, M.D., pediatric anesthesiologist and director of Pain Medicine Research and Development in the Sheikh Zayed Institute for Pediatric Surgical Innovation, serving as principal investigator.

Consortium members include Johns Hopkins University, CIMIT at Mass General Brigham, Tufts Medical Center, Medstar Health Research Institute and MedTech Color. Publicly traded OrthoPediatrics Corp., which exclusively focuses on advancing pediatric orthopedics, is serving as APDI’s strategic advisor and role model for device innovators whose primary focus is children.

Why we’re excited

Consortium initiatives got underway quickly with the announcement of a special MedTech Color edition of the “Make Your Medical Device Pitch for Kids!”™ competition that focuses on African American and Hispanic innovators. Interested innovators can find details and apply at MedTech Color Pitch Competition. The competition was announced at the recent MedTech Color networking breakfast on Oct. 10,2023 at The MedTech Conference powered by AdvaMed.

“We all benefit from greater equity and inclusion among pediatric MedTech founders, decision-makers, investigators and developers in more effectively addressing the needs of the entire pediatric population,” said Eskandanian. “We need the expertise and insights of innovators from diverse backgrounds, and we want to provide these talented individuals with more opportunities to present their work and share their perspectives on pediatric device development.”

Additional details

APDI is one of five FDA-funded consortia created to provide a platform of services, expertise and funding to help pediatric innovators bring medical devices to the market that specifically address the needs of children.

 

Patient and doctor demoing Rare-CAP technology

M.D. in your pocket: New platform allows rare disease patients to carry medical advice everywhere

When someone has a rare disease, a trip to the emergency room can be a daunting experience: Patients and their caregivers must share the particulars of their illness or injury, with the added burden of downloading a non-specialist on the details of a rare diagnosis that may change treatment decisions.

Innovators at Children’s National Hospital and Vanderbilt University Medical Center, supported by Takeda, are trying to simplify that experience using a new web-based platform called the Rare Disease Clinical Activity Protocols, or Rare-CAP. This revolutionary collection of medical information allows patients to carry the latest research-based guidance about their rare disorders in their phones, providing a simple QR code that can open a trove of considerations for any medical provider to evaluate as they work through treatment options for someone with an underlying rare disease.

“No one should worry about what happens when they need medical help, especially patients with rare diseases,” said Debra Regier, M.D., division chief of Genetics and Metabolism at Children’s National and Rare-CAP’s lead medical advisor. “We built this new tool because I have watched as my patient-families have wound up in an emergency room — after all, kids get sprains or fractures — but they don’t have the expertise of a rare disease specialist with them. My hope is that they’re going to pull out their phones and access Rare-CAP, which will explain their rare disease to a new provider who can provide more thoughtful and meaningful care.”

The big picture

A rare disease is defined as any disorder that affects less than 200,000 people in the United States. Some 30 million Americans are believed to be living with one of the 7,000 known rare disorders tracked by the National Organization of Rare Diseases (NORD). Led by Dr. Regier, the Rare Disease Institute at Children’s National is one of 40 NORD centers for excellence in the country that provide care, guidance and leadership for the wide array of disorders that make up the rare disease community.

While a key goal of Rare-CAP is to bolster patient self-advocacy, the platform will also allow medical providers to proactively search for protocols on rare diseases when they know they need specialized advice from experts at Children’s National, a network of tertiary care centers and patient organizations.

As a leading values-based, R&D-driven biopharmaceutical company, Takeda has committed $3.85 million to the project to help activate meaningful change and empower a brighter future for rare disease communities, providing a unique understanding of the struggle that patients and caregivers face when they need care.

“Our team, alongside the medical and rare disease community, saw the need for a single portal to collect standardized care protocols, and we are thrilled to see this innovative tool come to life,” said Tom Koutsavlis, M.D., head of U.S. Medical Affairs at Takeda. “People with rare diseases and their caregivers need faster access to authoritative medical information that providers anywhere can act on, this will lead to improving the standard of care, accelerating time to diagnosis and breaking down barriers to increase equitable access.”

The patient benefit

The creators of Rare-CAP imagined its use in a wide range of settings, including emergency rooms, surgical suites, dental offices, urgent care offices and school clinics. The platform will eventually profile thousands of rare diseases and lay out the implications for care, while also creating a dynamic conversation among users who can offer updates based on real-world experience and changes in medical guidance.

“Our patients are unique, and so is this tool,” Dr. Regier said. “As we roll out Rare-CAP, we believe it is just the beginning of the conversation to expand the platform and see its power for the patient and provider grow, with each entry and each new rare disease that’s added to the conversation.”

neonate

Documentation of pain assessment after pain management interventions in the NICU

neonate

Timely assessment of pain in neonates continues to be difficult for staff due to high acuity, workload and staffing shortages.

Neonates may experience more than 300 painful procedures and surgeries throughout their hospitalization. A proactive assessment of pain allows the medical team to detect pain early and adequately manage it. At Children’s National, the Neonatal Intensive Care Unit (NICU) team wants to minimize the impact of pain, particularly in preterm nonverbal infants as it has short- and long-term adverse neurodevelopment effects. Smitha Israel, BSN, RN, clinical program coordinator in the Children’s National neonatology department, created a team to increase compliance in pain reassessments and documentation in the NICU using technology to speed up reporting. That compliance increased from 50% to 75% and has been sustained for over two years.

What’s been the hold-up in the field?

Timely assessment of pain in neonates continues to be difficult for staff due to high acuity, workload and staffing shortages. To combat this, Israel’s group partnered with experts from the triggers team to speed up the reporting of pain assessments and to standardize the assessments when administering a pharmacological intervention.

How will this work benefit patients?

This work improves patient care in the NICU by improving pain management and reducing those possible adverse effects in a neonate’s development that pain may contribute to.

How is this work unique?

This study at Children’s National is the first study of pain assessment in the NICU that has shown improvement beyond published rates and has been sustained for six months after implementation.

Read more here.

 

U.S. News Badges

Children’s National Hospital ranked #5 in the nation on U.S. News & World Report’s Best Children’s Hospitals Honor Roll

U.S. News BadgesChildren’s National Hospital in Washington, D.C., was ranked #5 in the nation on the U.S. News & World Report 2023-24 Best Children’s Hospitals annual rankings. This marks the seventh straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

For the thirteenth straight year, Children’s National also ranked in all 10 specialty services, with eight specialties ranked in the top 10 nationally. In addition, the hospital was ranked best in the Mid-Atlantic for neonatology, cancer, neurology and neurosurgery.

“Even from a team that is now a fixture on the list of the very best children’s hospitals in the nation, these results are phenomenal,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “It takes a ton of dedication and sacrifice to provide the best care anywhere and I could not be prouder of the team. Their commitment to excellence is in their DNA and will continue long after I retire as CEO later this month.”

“Congratulations to the entire Children’s National team on these truly incredible results. They leave me further humbled by the opportunity to lead this exceptional organization and contribute to its continued success,” said Michelle Riley-Brown, MHA, FACHE, who becomes the new president and CEO of Children’s National on July 1. “I am deeply committed to fostering a culture of collaboration, empowering our talented teams and charting a bold path forward to provide best in class pediatric care. Our focus will always remain on the kids.”

“I am incredibly proud of Kurt and the entire team. These rankings help families know that when they come to Children’s National, they’re receiving the best care available in the country,” said Horacio Rozanski, chair of the board of directors of Children’s National. “I’m confident that the organization’s next leader, Michelle Riley-Brown, will continue to ensure Children’s National is always a destination for excellent care.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

“For 17 years, U.S. News has provided information to help parents of sick children and their doctors find the best children’s hospital to treat their illness or condition,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals that are on the Honor Roll transcend in providing exceptional specialized care.”

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The eight Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other two specialties ranked among the top 50 were cardiology and heart surgery, and urology.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

Abstract Happy 2022 New Year greeting card with light bulbA clinical trial testing a new drug to increase growth in children with short stature. The first ever high-intensity focused ultrasound procedure on a pediatric patient with neurofibromatosis. A low dose gene therapy vector that restores the ability of injured muscle fibers to repair. These were among the most popular articles we published on Innovation District in 2022. Read on for our full top 10 list.

1. Vosoritide shows promise for children with certain genetic growth disorders

Preliminary results from a phase II clinical trial at Children’s National Hospital showed that a new drug, vosoritide, can increase growth in children with certain growth disorders. This was the first clinical trial in the world testing vosoritide in children with certain genetic causes of short stature.
(2 min. read)

2. Children’s National uses HIFU to perform first ever non-invasive brain tumor procedure

Children’s National Hospital successfully performed the first ever high-intensity focused ultrasound (HIFU) non-invasive procedure on a pediatric patient with neurofibromatosis. This was the youngest patient to undergo HIFU treatment in the world.
(3 min. read)

3. Gene therapy offers potential long-term treatment for limb-girdle muscular dystrophy 2B

Using a single injection of a low dose gene therapy vector, researchers at Children’s National restored the ability of injured muscle fibers to repair in a way that reduced muscle degeneration and enhanced the functioning of the diseased muscle.
(3 min. read)

4. Catherine Bollard, M.D., M.B.Ch.B., selected to lead global Cancer Grand Challenges team

A world-class team of researchers co-led by Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National, was selected to receive a $25m Cancer Grand Challenges award to tackle solid tumors in children.
(4 min. read)

5. New telehealth command center redefines hospital care

Children’s National opened a new telehealth command center that uses cutting-edge technology to keep continuous watch over children with critical heart disease. The center offers improved collaborative communication to better help predict and prevent major events, like cardiac arrest.
(2 min. read)

6. Monika Goyal, M.D., recognized as the first endowed chair of Women in Science and Health

Children’s National named Monika Goyal, M.D., M.S.C.E., associate chief of Emergency Medicine, as the first endowed chair of Women in Science and Health (WISH) for her outstanding contributions in biomedical research.
(2 min. read)

7. Brain tumor team performs first ever LIFU procedure on pediatric DIPG patient

A team at Children’s National performed the first treatment with sonodynamic therapy utilizing low intensity focused ultrasound (LIFU) and 5-aminolevulinic acid (5-ALA) medication on a pediatric patient. The treatment was done noninvasively through an intact skull.
(3 min. read)

8. COVID-19’s impact on pregnant women and their babies

In an editorial, Roberta L. DeBiasi, M.D., M.S., provided a comprehensive review of what is known about the harmful effects of SARS-CoV-2 infection in pregnant women themselves, the effects on their newborns, the negative impact on the placenta and what still is unknown amid the rapidly evolving field.
(2 min. read)

9. Staged surgical hybrid strategy changes outcome for baby born with HLHS

Doctors at Children’s National used a staged, hybrid cardiac surgical strategy to care for a patient who was born with hypoplastic left heart syndrome (HLHS) at 28-weeks-old. Hybrid heart procedures blend traditional surgery and a minimally invasive interventional, or catheter-based, procedure.
(4 min. read)

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

In a review article in Seminars in Pediatric Surgery, Marc Levitt, M.D., chief of the Division of Colorectal and Pelvic Reconstruction at Children’s National, discussed the history of pediatric colorectal and pelvic reconstructive surgery and described the key advances that have improved patients’ lives.
(11 min. read)

Drs. Sophie Pestieau and Andrew Waberski

Children’s National receives sustainability award for reducing anesthetic gases

Drs. Sophie Pestieau and Andrew Waberski

Drs. Sophie Pestieau and Andrew Waberski.

The District of Columbia Department of Energy & Environment (DOEE) has awarded Children’s National Hospital with a 2022 District Sustainability Award for its successful work around reducing anesthetic gases that contribute to ozone depletion and greenhouse warming.

The big picture

Current data suggests the U.S. healthcare sector contributes 10% of the nation’s greenhouse effect. Volatile inhaled anesthetic gases are often used in the operating room (OR) during procedures that require anesthesia. Most of the organic anesthetic gases remain in the atmosphere for a long time, where they have the potential to act as greenhouse gases.

  • “In perspective, one hour of anesthesia with the volatile anesthetic Desflurane is equivalent to the greenhouse gas emissions from driving 190 miles,” said Andrew Waberski, M.D., pediatric cardiac anesthesiologist, at Children’s National.

The Children’s National Sustainability Council recognized that gas flows can be safely reduced to levels that provide both economical and health benefits to patients as well helping hospitals “go green.” By doing so, hospitals can decrease the amount of inhaled anesthetics used, which decreases the Global Warming Potential (GWP), and also reduce costs.

Why we’re excited

After assessing the impact of anesthetic gases, the Children’s National anesthesia team proposed several interventions to deliver safe and sustainable anesthesia to children. After implementing low-flow anesthesia techniques and reducing Desflurane usage, Children’s National has reduced its GWP of volatile anesthetics by 80%.

What they’re saying

  • “Thank you to the DOEE for recognizing the sustainability efforts we made in the Anesthesia Division at Children’s National,” said Dr. Waberski. “We are now preventing the emission of approximately 725 tons of CO2 per year. We thank our staff members, faculty and providers for helping to implement these changes and contributing to our sustainability efforts.”
  • “I became passionate about sustainability in healthcare once I became a parent, wanting a healthy environment and healthy future not only for my children to grow in, but for all children,” said Sophie Pestieau, M.D., vice chair of Clinical Affairs, Division of Anesthesiology, Pain and Perioperative Medicine. “As a physician with a duty to ‘first do no harm,’ I see an opportunity to directly address the industry’s growing environmental footprint and assist in the transition to greener healthcare.”
  • “Our Sustainability Council is focused on the hospital’s mission of helping kids grow up stronger, and we pursue this by creating healthy environments. Our projects are successful at reducing waste, saving financial resources and generating quality improvement,” said Rusty Siedschlag, manager of Sustainability at Children’s National.

In September 2021, 200 medical journals named climate change the number one threat to global public health. Children’s National joined the Biden Administration for a White House event on June 30, 2022, with the U.S. Department of Health and Human Services, where our team and industry colleagues pledged meaningful action to decarbonize the healthcare sector and make healthcare facilities more resilient to the effects of climate change. Children’s National has committed to meet the Biden administration’s climate goal of reducing emissions by 50% by 2030 and achieving net zero emissions by 2050.

Drs. Kane and Petrosyan

POEM procedure is safe and effective for children with esophageal achalasia

Drs. Kane and Petrosyan

Drs. Petrosyan and Kane combined perform more POEM procedures for children than any other pediatric surgeons in the United States.

Peroral endoscopic myotomy (POEM) is a safe and effective procedure to treat pediatric achalasia according to a single-center outcomes study in the Journal of Pediatric Surgery.

Authors Timothy Kane, M.D., chief of General and Thoracic Surgery at Children’s National Hospital, and Mikael Petrosyan, M.D., MBA, associate chief of that division, together perform more of these procedures than any other pediatric surgeons in the United States.

Their experience with POEM shows that it is as safe and effective as the current standard of care for pediatric achalasia, which is a procedure called the laparoscopic Heller myotomy (LHM). Even better, previous research in adults and now in pediatric patients, has shown that those who undergo POEM as an alternative to LHM report less pain and often require shorter hospital stays after surgery.

Why it matters

POEM has been an option for adults with achalasia for many years, but not for children because it requires technical skill and expertise not readily available everywhere. More studies of young patients with successful outcomes following POEM procedures can help make the case for training more pediatric surgeons to learn this approach, and help this alternative method become an additional surgical option for children with achalasia.

The hold-up in the field

Achalasia is a rare condition in adults (1/100,000) and even less common in children, occurring in only 0.1 per 100,000 patients with an estimated prevalence of 10 per 100,000. The rarity of achalasia in children compared with adults makes collecting enough statistically significant evidence about how best to treat them difficult, more so than for other more common pediatric surgical conditions.

Children’s National Hospital leads the way

Children’s National Hospital is one of the only children’s hospitals in the country to offer the option of POEM for treatment of these conditions in children — and Drs. Kane and Petrosyan combined perform more of these procedures than any other pediatric surgeons in the United States.

The surgeons at Children’s National offer POEM as a primary intervention for children with esophageal achalasia and are also applying the same approach for pediatric gastroparesis as well.

Children’s National Hospital is one of only 12 children’s hospitals in the country, and the only hospital in the Washington, D.C., region, to be verified as a Level 1 Children’s Surgery Center by the American College of Surgeons (ACS) Children’s Surgery Verification Quality Improvement Program. This distinction recognizes surgery centers whose quality improvement programs have measurably improved pediatric surgical quality, prevented complications, reduced costs and saved lives.

Bottom line

Given their reported outcomes so far, the authors believe that in the long term this approach may replace the current pediatric standard of care, the LHM. More research is needed to make this case, however, including long-term follow-up studies of the patients who have undergone the procedure so far.

You can read the full study, “Per Oral Endoscopic Myotomy (POEM) for Pediatric Achalasia: Institutional Experience and Outcomes,” in the Journal of Pediatric Surgery.

little boy in hospital bed

IV acetaminophen administration reduces duration of opioid use

little boy in hospital bed

The study led by Children’s National Hospital experts further suggested that administering IV acetaminophen prior to IV opioid should be considered earlier in multimodal pain regimens because it may reduce the overall use of IV opioids.

A new study published in JAMA Network Open used a diverse, national pediatric inpatient sample, which showed that intravenous (IV) acetaminophen can effectively reduce IV opioid requirements by 15.5% compared to IV opioids use alone. The study led by Children’s National Hospital experts further suggested that administering IV acetaminophen prior to IV opioid should be considered earlier in multimodal pain regimens because it may reduce the overall use of IV opioids.

“The information shared through this research study has the potential to reduce inpatient pediatric IV opioid utilization and therefore reduce opioid related complications such as addiction, withdrawal, respiratory depression and delayed gut motility,” said Anita Patel, M.D., critical care specialist at Children’s National.

The multidisciplinary team of clinicians, data scientists and statisticians came together under the overall guidance of Murray Pollack, M.D., M.B.A., professor of pediatrics at Children’s National and senior author, coupled with the unique access to the Health Facts database that made this study possible. This is the first assessment of the opioid sparing association of IV acetaminophen in a general, real-world pediatric inpatient population.

“This study will help us reduce the hospital use of opioids in infants, children and adolescents,” said Dr. Pollack. “Reducing opioid use is especially important for patients needing prolonged pain relief and will help care-givers minimize the risks of opioids including addiction and withdrawal.”

Non-opioid analgesic medications have yet to be effectively adopted with the goal of minimizing opioid medications to hospitalized pediatric inpatients. Studies with a sufficient sample size have also been difficult to perform in pediatrics to study non-opioid medications on a large scale until now.

“This work was a necessary first step in what I plan on being my lifelong goal of optimizing pediatric pain while minimizing the adverse effects related to many opioid derived pain medications,” said Dr. Patel.

Patel et al. performed a comparative effectiveness research of data collected from 274 U.S. hospitals between January 2011 and June 2016 with 893,293 hospitalized children who received IV acetaminophen prior to IV opioids. These were associated with a significant 15.5% reduction in total IV opioid duration when compared to patients who received IV opioids alone.

Patel plans on applying the skills and knowledge gained through this research to address how we can minimize the opioid related side effect of Iatrogenic withdrawal in critically ill children.

pill bottles and pills

Racial and ethnic disparities in ED opioid prescriptions have decreased

pill bottles and pills

Whereas in 2012, there were clear racial and ethnic differences in opioid prescription rates. By 2019, those differences were no longer statistically significant overall or within sites.

As the provision of opioid prescriptions declined over time, previously marked racial and ethnic disparities in opioid prescription rates at the time of Emergency Department (ED) discharge also attenuated, according to new findings led by Monika Goyal, M.D., M.S.C.E., associate division chief of Emergency Medicine and Trauma Services at Children’s National Hospital, and others.

The research, published in Pediatrics, investigated whether racial and ethnic differences in the delivery of outpatient opioid prescriptions for children discharged from the ED with long-bone fractures diminished over time.

“In 2012 compared to 2019, although rates of opioid prescribing were higher across all racial/ethnic groups, they were highest for NH-white youth,” said Dr. Goyal. “It’s reassuring to see that as rates of opioid prescribing declined over time, such racial and ethnic differences have attenuated.”

Whereas in 2012, there were clear racial and ethnic differences in opioid prescription rates. By 2019, those differences were no longer statistically significant overall or within sites.

However, as clinicians prescribed fewer opioids, sites continued to have moderate racial and ethnic variability in opioid prescribing rates for non-Hispanic white, non-Hispanic Black and Hispanic children.

The authors caution that the reduction of opioid prescription is possibly attributed to the response against the opioid epidemic.  There is also a need for more studies that seek to identify optimal outpatient pain management for children with fractures and ensure suitable post-discharge pain control for all children, regardless of race and/or ethnicity.

The retrospective cross-sectional study sampled children 4-18 years with long bone fractures using the Pediatric Emergency Care Applied Research Network (PECARN) Registry, an electronic health record registry of four geographically diverse pediatric EDs, from January 1, 2012, to December 31, 2019.

Patient race and ethnicity were categorized as non-Hispanic (NH) white, NH-Black, Hispanic or other. The investigators performed bivariable and multivariable logistic regression to measure the association between patient race and ethnicity and outpatient prescription. Out of the 42,803 ED eligible visits to analyze, 6,441 received an opioid prescription at ED discharge. This data showed that disparities when prescribing an opioid decreased over a 7-year period.

Drs. Packer and van den Acker at the Pediatric Device Innovators Forum

Pediatric Device Innovators Forum explores state of focused ultrasound

For children living with pediatric tumors, less invasive and less painful treatment with no radiation exposure was not always possible. In recent years, the development of technologies like Magnetic resonance guided high intensity focused ultrasound (MR-HIFU) and Low intensity transcranial focused ultrasound (LIFU) is helping to reverse that trend.

This topic was the focus of the recent Pediatric Device Innovators Forum (PDIF) hosted by the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) in partnership with the U.S. Food and Drug Administration’s (FDA) Pediatric Device Consortia (PDC) grant program. A collaboration between Children’s National Hospital and University of Maryland Fischell Institute for Biomedical Devices, NCC-PDI is one of five PDCs funded by the FDA to support pediatric device innovators in bringing more medical devices to market for children.

The discussion, moderated by Kolaleh Eskandanian, Ph.D., MBA, PMP, vice president and chief innovation officer at Children’s National and principal investigator of NCC-PDI, explored the use of focused ultrasound’s noninvasive therapeutic technology for two pediatric indications, Osteoid Osteoma (OO) and Diffuse Intrinsic Pontine Glioma (DIPG), and the ways it can increase the quality of life for pediatric patients while also decreasing the cost of care.

The discussion also examined the most common barriers preventing more widespread implementation of focused ultrasound technology, specifically small sample size for evidence generation, lack of funding opportunities and reimbursement issues that can make or break a technology’s chances at reaching the patients that need it.

Karun Sharma, M.D., director of Interventional Radiology at Children’s National, emphasized the potential for focused ultrasound to treat localized pain relief and treat other diseases that, like OO, do not have any other therapeutic alternative

“At Children’s National, we use MR-HIFU to focus an ultrasound beam into lesions, usually tumors of the bone and soft tissues, to heat and destroy the harmful tissue in that region, eliminating the need for incisions,” says Sharma. “In 2015, Children’s National doctors became the first in the U.S. to use MR-HIFU to treat pediatric osteoid osteoma (OO), a painful, but benign, bone tumor that commonly occurs in children and young adults. The trial demonstrated early success in establishing the safety and feasibility of noninvasive MR-HIFU in children as an alternative to current, more invasive approaches to treat these tumors.”

In November 2020, the FDA approved this MR-HIFU system to treat OO in pediatric patients.

Roger Packer, M.D., senior vice president of the Center for Neuroscience and Behavioral Medicine at Children’s National, also discussed how focused ultrasound, specifically LIFU, has also proven to be an attractive modality for its ability to non-invasively, focally and temporarily disrupt the blood brain barrier (BBB) to allow therapies to reach tumors that, until recently, would have been considered unreachable without severe intervention.

“This presents an opportunity in pediatric care to treat conditions like Diffuse Intrinsic Pontine Glioma (DIPG), a highly aggressive brain tumor that typically causes death and morbidity,” says Packer.

Packer is planning a clinical trial protocol to investigate the safety and efficacy of LIFU for this pediatric indication.

The forum also featured insight from Jessica Foley, M.D., chief scientific officer, Focused Ultrasound Foundation; Arjun Desai, M.D., chief strategic innovation officer, Insighttec; Arun Menawat, M.D., chairman and CEO, Profound Medical; Francesca Joseph, M.D., Children’s National; Johannes N. van den Anker, M.D., Ph.D., vice chair of Experimental Therapeutics, Children’s National; Gordon Schatz, president, Schatz Reimbursement Strategies; Mary Daymont, vice president of Revenue Cycle and Care Management, Children’s National; and Michael Anderson, MD, MBA, FAAP, FCCM, FAARC, senior advisor to US Department of Health and Human Services (HHS/ASPR) and Children’s National.

Anthony Sandler, M.D., senior vice president and surgeon-in-chief of the Joseph E. Robert Jr. Center for Surgical Care and director of the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital, and Sally Allain, regional head of Johnson & Johnson Innovation, JLABS @ Washington, DC, opened the forum by reinforcing both organizations’ commitment to improving pediatric health.

In September 2020, the Focused Ultrasound Foundation designated Children’s National Hospital as the first global pediatric Center of Excellence for using this technology to help patients with specific types of childhood tumors. As a designated COE, Children’s National has the necessary infrastructure to support the ongoing use of this technology, especially for carrying out future pediatric clinical trials. This infrastructure includes an ethics committee familiar with focused ultrasound, a robust clinical trials research support team, a data review committee for ongoing safety monitoring and annual safety reviews, and a scientific review committee for protocol evaluation.

The Pediatric Device Innovators Forum is a recurring collaborative educational experience designed by the FDA-supported pediatric device consortia to connect and foster synergy among innovators across the technology development ecosystem interested in pediatric medical device development. Each forum is hosted by one of the five consortia. This hybrid event took place at the new Children’s National Research and Innovation Campus, the first-of-its-kind focused on pediatric health care innovation, on the former Walter Reed Army Medical Center campus in Washington, D.C.

To view the latest edition of the forum, visit the NCC-PDI website.

Panelists at the Pediatric Device Innovators Forum

The recent Pediatric Device Innovators Forum (PDIF) exploring the state of focused ultrasound was held at the new Children’s National Research and Innovation Campus, a first-of-its-kind focused on pediatric health care innovation.

morphine vial and needle

Replacing morphine with methadone in the NICU

morphine vial and needle

A synthetic analgesic drug, known as methadone, may serve as a better alternative for newborns in the neonatal intensive care unit (NICU) suffering from opioid withdrawal syndrome, according to a commentary published in Pediatric Research.

A synthetic analgesic drug, known as methadone, may serve as a better alternative for newborns in the neonatal intensive care unit (NICU) suffering from opioid withdrawal syndrome, according to a commentary published in Pediatric Research. Some existing literature suggests that methadone may also address painful stimuli that hinders neurodevelopment throughout adulthood, added Johannes van den Anker, M.D., Ph.D., division chief of Clinical Pharmacology at Children’s National Hospital.

The commentary was selected as the Editor’s Focus in Pediatric Research for the June edition, signaling the scientific community as noteworthy to further explore methadone’s potential as an alternative for pharmacologic treatments instead of morphine.

“It is important to define the pharmacokinetics and pharmacodynamics of methadone to treat pain in neonates in intensive care before replacing morphine with methadone. Pre-clinical research shows that the use of methadone might have fewer side effects than morphine,” said Dr. van den Anker. “If this is also the case in the human neonate, then a shift from morphine to methadone might be beneficial. However, first, we need to define what the safe and effective dose of methadone will be for this purpose.”

While there is a need to better understand how newborns and preemies metabolize methadone, there is existing knowledge that this drug minimizes pain. The commentary, too, raises the question for clinicians to possibly consider methadone as a better option to avoid long-term negative neurodevelopmental consequences — such as hypersensitivity to re-injury in later life — usually associated with pain.

The current but limited data out there still provides “exciting and stimulating” information about the possible use of methadone for the treatment of neonatal pain in the NICU, according to Dr. van den Anker. He believes that, in the future, methadone could also serve as mechanism-based analgesia in newborns experiencing pain.

“There needs to be a collaboration between neonatal medicine specialists, pharmacometricians and developmental pharmacologists to assure not only the generation of evidence-based data to determine these optimal dosing regimens, but also to facilitate the implementation of this new knowledge into daily clinical care in neonatal intensive care units across the globe,” added Dr. van den Anker.