REI Week 2025 empowers the future in pediatric research and innovation

Children’s National Hospital hosted its fifteenth annual Research, Education and Innovation Week from March 31–April 4, 2025, bringing together clinicians, scientists, educators and innovators from across the institution to celebrate discovery and collaboration. This year’s theme, “Empowering the Future in Pediatric Research and Innovation with Equity, Technology and a Global Reach,” served as a call to action for advancing science that improves child health both locally and around the world.

Each day of the week-long event featured thought-provoking lectures — now available to watch — dynamic panel discussions, interactive workshops and vibrant poster sessions, all highlighting the diverse and interdisciplinary work taking place across Children’s National.

Centering the patient and the planet

REI Week began on Monday with a powerful keynote lecture from Lynn R. Goldman, MD, MS, MPH, Michael and Lori Milken dean of the Milken Institute School of Public Health at the George Washington University. In her talk, “Children: Uniquely vulnerable to climate-related threats,” Dr. Goldman underscored the urgent need to protect children from the environmental hazards of a changing climate and to integrate climate science into pediatric care and advocacy.

At mid-morning, Mary-Anne “Annie” Hartley, MD, PhD, MPH, director of the LiGHT Laboratory at École Polytechnique Fédérale de Lausanne, introduced the “MOOVE” platform — Massive Open Online Validation and Evaluation of clinical LLMs. Her talk demonstrated how artificial intelligence, when rigorously validated, has the potential to transform clinical decision-making and global health equity.

Monday’s final keynote, “Zinc and childhood diarrhea,” was presented by Christopher Duggan, MD, MPH, director of the Division of Nutrition at Harvard Medical School. Dr. Duggan highlighted the global health impact of zinc supplementation in reducing childhood mortality — a reminder that simple, evidence-based interventions can save millions of lives.

In that first day, the first poster session of the week showcased projects in adolescent medicine, global health, infectious diseases, oncology and more. The session reflected the full breadth of research taking place across Children’s National.

Ambroise Wonkam, MD, PhD, professor of genetic medicine at Johns Hopkins University, then delivered Tuesday’s Global Health Keynote Lecture, “Harnessing our common African genomes to improve health and equity globally.” His work affirmed that inclusive genomics is key to building a healthier world.

Later, the Global Health Initiative event and GCAF Faculty Seminar encouraged attendees to pursue collaborative opportunities at home and abroad, reflecting the growing global footprint of Children’s National research programs.

Transforming education and care delivery

On Wednesday, Larrie Greenberg, MD, professor emeritus of pediatrics, kicked off the day with a Grand Rounds keynote on educational transformation: “Shouldn’t teachers be more collaborative with their learners?” He followed with a CAPE workshop exploring the effectiveness of case-based learning.

The Nursing Sponsored Keynote Lecture by Vincent Guilamo-Ramos, PhD, MPH, LCSW, ANP-BC, PMHNP-BC, FAAN, explored “Redesigning the U.S. broken health system.” He offered an urgent and inspiring call to reimagine pediatric care by addressing social determinants of health.

In the Jill Joseph Grand Rounds Lecture, Deena J. Chisolm, PhD, director of the Center for Child Health Equity at Nationwide Children’s Hospital, challenged attendees to move beyond dialogue into action in her talk, “Health equity: A scream to a whisper?,” reminding researchers and clinicians that advocacy and equity must be foundational to care.

The day continued with a poster session spotlighting medical education, neonatology, urology and neuroscience, among other fields.

Posters and pathways to progress

Throughout the week, poster sessions highlighted cutting-edge work across dozens of pediatric disciplines. These sessions gave attendees the opportunity to engage directly with investigators and reflect on the shared mission of discovery across multiple disciplines, including:

Honoring excellence across Children’s National

The REI Week 2025 Awards Ceremony celebrated outstanding contributions in research, mentorship, education and innovation. The winners in each category were:

POSTER SESSION AWARDS

Basic & Translational Research

Faculty:  Benjamin Liu, PhD

“Genetic Conservation and Diversity of SARS-CoV-2 Envelope Gene Across Variants of Concern”

Faculty:  Steve Hui, PhD
“Brain Metabolites in Neonates of Mothers with COVID-19 Infection During Pregnancy”

Faculty: Raj Shekhar, PhD
“StrepApp: Deep Learning-Based Identification of Group A Streptococcal (GAS) Pharyngitis”

Post docs/Fellows/Residents: Dae-young Kim, PhD
“mhGPT: A Lightweight Domain-Specific Language Model for Mental Health Analysis”

Post docs/Fellows/Residents: Leandros Boukas, MD, PhD
“De Novo Variant Identification From Duo Long-Read Sequencing: Improving Equitable Variant Interpretation for Diverse Family Structures”

Staff: Naseem Maghzian
“Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts (ATLANTIC)”

Graduate Students: Abigail Haffey
“Synergistic Integration of TCR and CAR T Cell Platforms for Enhanced Adoptive Immunotherapy in Brain Tumors”

High School/Undergraduate Students: Medha Pappula
“An ADHD Diagnostic Interface Based on EEG Spectrograms and Deep Learning Techniques”

Clinical Research

Faculty: Folasade Ogunlesi, MD
“Poor Air Quality in Sub-Saharan Africa is Associated with Increase Health Care Utilization for Pain in Sickle Cell   Disease Patients”

Faculty: Ayman Saleh, MD
“Growth Parameters and Treatment Approaches in Pediatric ADHD: Examining Differences Across Race”

Post docs/Fellows/Residents: Nicholas Dimenstein, MD, MPH
“Pre-Exposure Prophylaxis (PrEP) Eligibility in the Pediatric Emergency Department”

Staff: Tayla Smith, MPH
“The Public Health Impact of State-Level Abortion and    Firearm Laws on Health Outcomes”

Graduate Students: Natalie Ewing
“Patterns of Bacteriuria and Antimicrobial Resistance in Patients Presenting for Primary Cloacal Repair: Is Assisted Bladder Emptying Associated with Bacteriuria?”

Graduate Students: Manuela Iglesias, MS
“Exploring the Relationship Between Child Opportunity Index and Bayley-III Scores in Young Children”

High School/Undergraduate Students: Nicholas Lohman
“Preliminary Findings: The Efficacy, Feasibility and Acceptability of Group Videoconference Cognitive Behavioral Therapy with Exposure and Response Prevention for Treating Obsessive-Compulsive Disorder Among Children and Young People”

Community-Based Research

Faculty: Sharon Shih, PhD
“Assessing Pediatric Behavioral Health Access in DC using Secret Shopper Methodology”

Post docs/Fellows/Residents: Georgios Sanidas, MD
“Arrested Neuronal Maturation and Development in the Cerebellum of Preterm Infants”

Staff: Sanam Parwani

“Intersectionality of Gender and Sexuality Diversity in Autistic and Non-Autistic Individuals”

Graduate Student: Margaret Dearey
“Assessing the Burden of Period Poverty for Youth and Adolescents in Washington, DC: A Pilot Study”

Quality and Performance Improvement

Faculty: Nichole L. McCollum, MD
“A Quality Improvement Study to Increase   Nurse Initiated Care from Triage and Improve Timeliness to Care”

Post docs/Fellows/Residents:  Hannah Rodriguez, MD
“Reducing Unnecessary Antibiotic Use in a Level IV NICU”

Staff: Amber K. Shojaie, OTD, OTR/L
“Implementing Dynamic Axilla Splints in a Large Burn Patient”

MENTORSHIP AWARDS

Basic Science Research

Conrad Russell Y. Cruz, MD, PhD

Clinical Research

Rana Hamdy, MD, MPH, MSCE

Bench to Bedside Research

Ioannis Koutroulis, MD, PhD, MBA

ELDA ARCE TEACHING SCHOLAR AWARD

Priti D. Bhansali, MD, MEd

Heather Ann Walsh, PhD, RN

SUZANNE FEETHAM NURSING RESEARCH SUPPORT AWARD

Eileen P. Engh, PhD RN 
“Rare Disease Organization Lifecycle” Role in Helping Parents with Everyday Life Information Seeking and Connection (RDO-HELIX)

EXPLORATIONS IN GLOBAL HEALTH PILOT AWARDS

Launchpad Awards

Mi Ran Shin, MD, MPH
“Establishing Interdisciplinary Rehabilitation for Birth and Burn Injuries in Ethiopia”

Susan Harvey, MSN, CPNP-AC
“Implementation of Sickle Cell Pilot Program in Ndhiwa Sub County, Kenya”

Meleah Boyle, PhD, MPH
“Understanding and Addressing Environmental Sustainability to Protect the Health of the Children’s National and Global Communities”

Eiman Abdulrahman, MD
“Research Capacity Building to Improve Pediatric Emergency and Critical Care in Ethiopia”

Pilot Awards

Alexander Andrews, MD
“EEG as a Diagnostic and Prognostic Marker in Severe Pediatric Malaria, Blantyre Malawi”

Daniel Donoho, MD & Timothy Singer, MD
“Feasibility Study of a Novel Artificial Intelligence-Based Educational Platform to Improve Neurosurgical Operative Skills in Tanzania”

Hasan Syed, MD
“Bridging the Gap an Educational Needs Assessment for Pediatric Neurosurgery Training in Pakistan”

Sofia Perazzo, MD & Lamia Soghier, MD, MEd, MBA
“QI Mentorship to Improve Pediatric Screening and Follow-up in Rural Argentina”

Benjamin Liu, PhD
“AI-Empowered Real-Time Sequencing Assay for Rapid Detection of Schistosomiasis in Senegal”

Rae Mittal, MD
“Assessment and Enhancement of Proficiency in Emergency Child Neurology Topics for Post-Graduate Emergency Medicine Trainees in India”

Innovation Day ignites bold thinking

Thursday, REI Week shifted to the Children’s National Research & Innovation Campus for Innovation Day, a celebration of how bold ideas and collaborative culture can accelerate progress in pediatric medicine.

Brandy Salmon, PhD, associate vice president of Innovation and Partnerships at Virginia Tech, opened the day with “The Alchemy of Innovation,” focusing on how institutions can build a culture that fuels transformative partnerships.

A multidisciplinary panel discussion moderated by Nathan Kuppermann, MD, MPH, and Catherine Bollard, MBChB, MD, featured Nehal Mehta, MD, Julia Finkel, MD, Kevin Cleary, PhD, Ioannis Koutroulis, MD, PhD, MBA, Francesca Joseph, MD and Patrick Hanley, PhD, who shared how innovation can be advanced and promoted, especially as a core institutional priority.

A shared vision for the future

REI Week 2025 reaffirmed the values that define Children’s National: a commitment to excellence, collaboration and equity in pediatric research and care. As discoveries continue to emerge from our hospital and our research campuses, the connections built and ideas sparked during this week will help shape the future of pediatric health — locally and globally.

By elevating voices from the bedside to the bench, with the support of the executive sponsors Nathan Kuppermann, MD, MBChB, Catherine Bollard, MBChB, MD, Kerstin Hildebrandt, MSHS, Linda Talley, MS, RN, NE-BC and David Wessel, MD, REI Week demonstrated that we must embrace the community in all aspects of our work. Because we know that there are answers we can only get from the patients that we serve—and we need to be their voice.

Research, Education & Innovation Week will be back next year on April 13-17, 2026.

  • Posters at the REI Week 2025 Monday, March 31 poster session.

    Posters at the REI Week 2025 Monday, March 31 poster session.
  • Panelists discuss innovation during REI Week 2025.

    Panelists discuss innovation during REI Week 2025.
  • Global Health Initiative community engagement event during REI Week 2025.

    Global Health Initiative community engagement event during REI Week 2025.
  • Chris Rees presents his REI Week 2025 lecture.

    Chris Rees presents his REI Week 2025 lecture.
  • Nathan Kuppermann listens to a presenter during the REI Week 2025 Tuesday, April 1, poster session.

    Nathan Kuppermann listens to a presenter during the REI Week 2025 Tuesday, April 1, poster session.
  • Michelle Riley-Brown, Nathan Kuppermann, Catherine Bollard and Naomi Luban on stage during the REI Week 2025 awards ceremony.

    Michelle Riley-Brown, Nathan Kuppermann, Catherine Bollard and Naomi Luban on stage during the REI Week 2025 awards ceremony.
  • Brandy Salmon presents on innovation programs at Virginia Tech during the REI Week 2025 Innovation Day.

    Brandy Salmon presents on innovation programs at Virginia Tech during the REI Week 2025 Innovation Day.
  • Catherine Bollard listens to a presenter during the REI Week 2025 Monday, March 21 poster session.

    Catherine Bollard listens to a presenter during the REI Week 2025 Monday, March 21 poster session.
  • Ambroise Wonkman poses for a picture with Children’s National staff.

    Ambroise Wonkman poses for a picture with Children’s National staff.
  • Tanzeem Choudhury presenting during REI Week 2025.

    Tanzeem Choudhury presenting during REI Week 2025.

U.S. News & World Report voting

U.S. News & World Report badgeChildren’s National is ranked one of the top 10 pediatric hospitals in the nation by U.S. News & World Report. Our faculty and staff are proud of the impact made on the lives of children and families in our community. Your participation in the U.S. News & World Report annual reputational survey validates the quality of care we provide and reflects the mutual respect and trust we share as healthcare professionals.

How to determine your voting eligibility

Voting for the U.S. News & World Report Best Children’s Hospitals rankings can be done only through Doximity.

To participate, physicians must:

  • Be board-certified and meet the eligibility criteria for the voting categories.
  • For child and adolescent psychologists, your account must be up to date with your specialty and subspecialty correctly marked.
  • Be a credential-verified member of Doximity (you must have an active and claimed Doximity profile).
  • Have all certifications and board documents currently up-to-date in your Doximity profile.

View the full eligibility criteria

How to claim your Doximity profile to vote

  • You have to claim your profile on Doximity.com to participate in the online survey. If you have not yet claimed your Doximity profile, go to Doximity.com, and click “Find My Profile.”
  • Once your profile has been claimed, you must confirm your email address and board certifications.
  • Verified Doximity members will receive an email inviting them to participate in the U.S. News survey.
  • For more information on how to claim your profile, visit Doximity.com

How to update and verify existing Doximity account information

Your Doximity profile must have up-to-date licenses, certifications and board documents.

  • Once you are logged in, your profile will automatically be in “Edit Mode.” You are able to add new items or edit existing information.
  • Update your Doximity profile and ensure your information is current.

Once registered, users wishing to participate in the online survey should:

  • Watch for an email from Doximity about the annual member survey.
  • Even if you don’t see the email, if you are a registered Doximity user, you can still vote by logging in to Doximity.com with your username and password during the voting period.
  • Once logged in, look for a U.S. News graphic or button on the homepage and click on it.
  • The survey asks users to name the hospitals that provide the best care in your respective specialty, without consideration to location or cost. Pediatric specialists will list 10 hospitals. The order in which you list the hospitals does not matter.

Please note: Children’s National Hospital is listed as “Children’s National Hospital Washington, DC” on the survey.

Visit Doximity’s FAQs if you have issues or questions about registration or claiming your profile.

How to cast your vote

In February 2025 when voting opens, all survey-eligible physicians will receive a notification on the Doximity app for Android or iOS. If you do not use the Doximity app, you will receive an email when voting opens.

  1. Log in to your Doximity account at doximity.com or via the mobile app.
  2. Click the Notifications icon or tap the “Submit your Nominations” button on the homepage. You can also search for “U.S. News Best Hospitals”
  3. Select 10 hospitals in your respective specialty that you believe provide the best care in the United States.
  4. Submit your vote

Having technical issues?

If you have difficulty registering with Doximity or completing the survey, please visit Doximity support for assistance.

Vote

The 2025 U.S. News & World Report Best Children’s Hospitals reputation voting will open in mid-February. Look for your Doximity notification to vote.

Children’s National delivers on the promise in 2024

Children's National Hospital's 2023-2024 Academic Annual Report on a tablet

The Children’s National 2023-2024 Academic Annual Report show on a tablet.

Children’s National Hospital has released its 2023-2024 Academic Annual Report, showcasing a year of transformative progress in pediatric medicine. The report highlights achievements across its research centers, institutes and Innovation Ventures, underscoring the hospital’s role as a leader in advancing child health through innovation and collaboration.

“This year’s report reflects the remarkable progress we have made in advancing the frontiers of pediatric medicine,” said Nathan Kuppermann, MD, MPH, Chief Academic Officer and Chair of Pediatrics. “It highlights groundbreaking work across our research centers, institutes, and Innovation Ventures, showcasing the collaborative spirit that drives our mission forward. These achievements underscore our shared commitment to delivering transformative research and the best possible outcomes for children and families.”

Delivering across centers

The report captures the contributions of each of Children’s National’s research centers, each pushing the boundaries of pediatric healthcare:

  • Center for Cancer & Immunology Research (CCIR): Delivering on the promise of cell and gene therapies, offering innovative treatments for pediatric cancers and immune disorders.
  • Center for Genetic Medicine Research (CGMR): Advancing pediatric genetic medicine through interdisciplinary efforts, addressing complex genetic conditions with cutting-edge science.
  • Center for Neuroscience Research (CNR): A year of growth in scientific excellence, advancing the understanding of brain development and neurological conditions.
  • Center for Prenatal, Neonatal & Maternal Health Research (CPHNMR): Revolutionizing neonatal care with its pioneering infant brain health neuromonitoring program.
  • Center for Translational Research (CTR): Facilitating groundbreaking work by new K awardees and driving translational research to bridge the gap between discovery and clinical care.
  • Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI): Leading the way in advanced research projects in pediatric surgery, pushing technological boundaries to improve outcomes for children worldwide.

Taking the lead in innovation through collaboration

Innovation Ventures at Children’s National is advancing pediatric health security, addressing unique challenges with transformative solutions. Meanwhile, the Children’s National Research & Innovation Campus (CNRIC) continues to thrive as a hub for discovery and collaboration, hosting conferences on topics like artificial intelligence in healthcare, cell and gene therapy, and pediatric epilepsy research.

A vision for the future

The report also highlights Children’s National’s focus on integrating cutting-edge technologies like artificial intelligence into its research and clinical practices, as well as addressing global health challenges such as the effects of climate change on children’s health. These efforts reflect the hospital’s commitment to improving outcomes for children everywhere through innovation, teamwork, and forward-thinking leadership.

The 2023-2024 Academic Annual Report serves as a testament to the dedication and expertise of the Children’s National community, showcasing how collaboration and innovation are shaping the future of pediatric healthcare.

AI for good: Children’s National wins global competitions for measuring brain tumors

Children's National Hospital's winning team for the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

Meet the winners (left to right): Syed M. Anwar, Ph.D., M.S., principal investigator at Children’s National; Daniel Capellan Martin, M.Sc., Polytechnic University of Madrid; Abhijeet Parida, data scientist at Children’s National; and Austin Tapp, Ph.D., postdoctoral research fellow at Children’s National.

Using an award-winning artificial intelligence (AI) algorithm developed at Children’s National Hospital, researchers ranked first in the world in the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge for their approach to identifying different parts of deadly gliomas. The details of their innovative method were recently published on arXiv, a curated research-sharing platform.

“Technology can bridge the gap in healthcare between high- and low-resource countries,” said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI). “By tailoring methods we created at our hospital to fit the needs of specific regions, such as sub-Saharan Africa, our research helps improve medical imaging and diagnosis in challenging environments.”

Dr. Linguraru was the program chair of the International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI) 2024 in Marrakesh, Morocco, the leading global meeting on AI in medical imaging.

Children’s National leads the way

Gliomas are a type of brain tumor with a high death rate and are especially difficult to diagnose in low- and middle-income countries. Given the increased need in Africa, researchers worldwide came together in Morocco to compete over the best way to accurately detect and measure tumors using MRI data and AI.

By applying advanced machine-learning techniques, the researchers adapted tools initially designed for well-resourced settings to work in countries with far fewer.

The study focused on transfer learning, a process in which an AI model is trained in advance on a large number of brain tumor images and then adjusted to work with smaller sets of new data. In this case, the models were adapted to work with local sub-Saharan African data using a strategy to combine different models’ strengths.

When tested, the approach achieved impressive accuracy scores. The Children’s National team, which included a colleague from the Polytechnic University of Madrid, ranked first in the BraTS-Africa 2024 challenge for identifying different parts of gliomas.

“To make the method widely available, the winning algorithm is shared online for others to use and improve upon,” Dr. Linguraru said. “My favorite part of these competitions is how they highlight the way innovation and collaboration can reduce global healthcare inequalities.”

The big picture

Children’s National researchers consistently lead global events using AI and advanced imaging to tackle complex healthcare challenges. In 2023, the team won a global contest to measure pediatric brain tumors at the MICCAI 2023 Conference. This year’s success in the BraTS-Africa challenge builds on this knowledge base and expands its use to adult gliomas.

At the Radiological Society of North America 2024 annual meeting, which drew 50,000 attendees, Zhifan Jiang, Ph.D., a staff scientist in the Precision Medical Imaging Lab at SZI, also won the Cum Laude Award for his scientific poster on applying AI to radiological images to predict severe outcomes for children with brain tumors caused by neurofibromatosis type 1.

“These achievements show how our science is leading the world in using AI for good,” Dr. Linguraru said. “Every day, we’re building on our knowledge of advanced imaging, brain tumors and AI to improve the diagnosis, measurement and treatment of deadly tumors — on a global scale.”

Attendees of the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

Innovative treatment for growth disorders

Dr. Dauber and patient

Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology, with a patient.

Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology, leads a program that brings together comprehensive resources for children with rare genetic growth disorders, including basic science, translational and clinical research.

“Discovery is important, and research gives us many answers,” he says. “But what we do with those answers is what really matters. There’s nothing better than seeing the impact our research is having on an individual patient. It is so gratifying to hear from parents about how their kids are making progress thanks to a protocol we developed.”

Mia thrives with the right approach

Middle schooler Mia loves to dance, practice gymnastics and hang out with friends. She was born in Croatia with hypochondroplasia, a genetic disorder that slows cell growth and causes short stature and limb shortening.

Mia practices her dance moves.

Mia practices her dance moves.

She struggled in kindergarten because she was so small. “Kids can be very mean if you’re different,” says Mia’s dad, Ivan. “For years, we took Mia to different specialists in Europe without getting the help she needed. I researched endocrinologists all over the world. They all pointed us to the United States.” This led Ivan to Children’s National and Dr. Dauber.

Dr. Dauber invited the family to the U.S. to participate in a clinical trial that was to start very soon. “I knew this was our only chance,” said Ivan, who rushed to bring his family to Washington, D.C., for the initial screening appointments. They later returned for more measurements, Mia’s first dose of medicine and a three-month supply to go. The family returns to Children’s National every six months.

In Mia’s first year, she grew more than 3 inches. Her arms also grew longer. “Dr. Dauber is probably the best doctor in the world,” Ivan says. “He is like a friend to Mia and has helped us make sure she has as normal a life as possible. This growth, including in her confidence, has been life-changing for her.” The family relocated to the U.S., and Mia will continue in the trial until she reaches puberty.

“Now, the difference between Mia and other kids is much less,” Ivan says. “She makes new friends more easily and is a happy, happy kid. As for me, I want to cry for how happy I am. Dr. Dauber and Children’s National made it all possible.”

Children’s National in the News: 2024

collage of news logosIn 2024, Children’s National Hospital continued to make remarkable strides across diverse areas of pediatric medicine, from groundbreaking technological innovations to critical health advocacy. The following compilation showcases ten significant stories that demonstrate the breadth and depth of the hospital’s impact, as featured in major national news outlets including NBC Nightly News, CNN, The Washington Post, The New York Times, NPR, The Today Show, Healio, and POLITICO. Delve into our 2024 news highlights for more.

1. World’s smallest pacemaker gives new hope to babies with heart defects

Charles Berul, M.D., and a patient family talk about the pill-sized pacemaker that saved the life of Abby, an infant born with deadly heart defects. (NBC Nightly News)

2. ‘A $10 death trip’: Fentanyl is killing teens. Meet one fighting for his life

Sivabalaji Kaliamurthy, M.D., addiction psychiatrist and director of the Addictions Program, spoke to CNN about the impact of drug addiction on teen health and the lack of resources available to treat opioid use disorder. (CNN)

3. Health panel urges interventions for children and teens with high BMI

Susma Vaidya, M.D., M.P.H., associate medical director of the IDEAL Clinic, shared her concerns about childhood obesity treatment recommendations issued today by a leading panel of independent U.S. health experts. (The Washington Post)

4. An Rx for food? Doctor’s offices offer groceries to those in need

Shideh Majidi, M.D., M.S.C.S., and Emily Frymark, clinical dietitian, spoke about how the food pharmacy, created in partnership with the Capital Area Food Bank, benefits patients with diabetes and other chronic conditions. (The Washington Post)

5. First patient begins newly approved sickle cell gene therapy

Kendric Cromer, a 12-year-old boy being treated at Children’s National Hospital, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. “This is a big effort,” says David Jacobsohn, M.D., ScM, M.B.A. (The New York Times)

6. ‘We created this problem’: A pediatric surgeon on how gun violence affects children

Mikael Petrosyan, M.D., associate chief of General and Thoracic Surgery, discusses the stress medical staff face when treating young victims of gun violence. (NPR)

7. 7th grade boy rings bell after final round of chemotherapy

Landon, an 11-year-old patient, rang the bell at Children’s National Hospital with family, friends, doctors and nurses cheering after finishing his final round of chemotherapy. (The Today Show)

8. Study: One in three adolescents experience ‘period poverty’

Monika Goyal, M.D., M.S.C.E., pediatric emergency medicine specialist and co-director of the Center for Translational Research, emphasized the need for awareness in addressing period poverty in teenagers and young adults. (Healio)

9. The AI assurance labs are coming

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer, participates in a panel discussion covering AI data collection, associated risks, reliance and other topics related to artificial intelligence. (POLITICO)

10. First day of a ‘new life’ for a boy with sickle cell

Children’s National patient Kendric Cromer, 12, became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood. (The New York Times)

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)

Pharmacokinetic and pharmacodynamic analysis of vosoritide in Phase 2 trial

test tubes filled with blood and serum

The research, published in Hormone Research in Paediatrics, examined whether pharmacokinetic (PK) factors or other blood biomarkers correlated with the response to treatment of vosoritide in children with hypochondroplasia.

Researchers from Children’s National Hospital presented new data from the world’s first clinical trial of vosoritide use for growth disorders at the European Society for Pediatric Endocrinology annual meeting. The research, published in Hormone Research in Paediatrics, examined whether pharmacokinetic (PK) factors or other blood biomarkers correlated with the response to treatment of vosoritide in children with hypochondroplasia. The study found that drug exposure ­­— as measured by average PK area under the curve (AUC) — did not correlate with any growth outcome.

The big picture

Previously published results of the clinical trial of vosoritide in children with hypochondroplasia showed that vosoritide can help kids with hypochondroplasia grow better. For this study, the researchers analyzed PK parameters by non-compartmental methods. Pharmacodynamic markers were measured at day 1, the 6-month and 12-month visits. The PK analysis indicates that drug exposure was correlated to global C-type natriuretic peptide (CNP) activity as measured by urine cyclic guanosine monophosphate (cGMP) concentrations.

Moving the field forward

There are currently no approved therapies for hypochondroplasia. Children’s National is the first site in the world to conduct a trial of a targeted treatment for hypochondroplasia and this is the first study to report PKs of vosoritide in children with hypochondroplasia.

“The results of our study showed that the drug levels achieved are similar to what was achieved in patients with achondroplasia,” says Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology at Children’s National and one of the study authors. “This suggests that the dose we used is a reasonable one, paving the way for a Phase 3 trial of vosoritide in children with hypochondroplasia.”

What’s next

Dr. Dauber emphasized the importance of these findings for the medical community, particularly for those dedicated to pediatric endocrinology. He noted that understanding the nuanced responses among different children is crucial for optimizing future treatments.

“This ongoing commitment to innovative research underscores the relentless pursuit of targeted therapies, bridging gaps and bringing hope to families and patients worldwide,” says Dr. Dauber.

Read the study in Hormone Research in Paediatrics: Phase 2 Trial of Vosoritide Use in patients with Hypochondroplasia: Pharmacokinetic/ Pharmacodynamic analysis from 12 Month Data

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.

Children’s National Hospital the 2024 American Academy of Pediatrics meeting

Children’s National Hospital-affiliated participants will attend this year’s American Academy of Pediatrics National Conference and Exhibition. The meeting will take place in Orlando, Florida from September 27 to October 1. You will find a mini schedule of sessions below.

Date Time Presenter Title
9/27/2024 8:00:00 AM Briony Varda, M.D. H1016: Section on Surgery Program: Day 1
9/27/2024 8:00:00 AM Briony Varda, M.D. H1017: Section on Urology Program: Day 1
9/27/2024 8:00:00 AM Lowell Frank, M.D., FAAP H1014: Section on Cardiology and Cardiac Surgery Program: Day 1
Through the Looking Glass: Using Past Decisions to Inform Current and Future Clinical and Professional Practice
9/27/2024 2:30:00 PM Brian K. Reilly, M.D., FAAP S1208: Sounding the Alarm on Noise: Prevention Is Key
9/27/2024 4:00:00 PM Brandon Kappy, M.D., MPP S1312: Using Lessons From the East Palestine Train Derailment to Prepare for Environmental Disasters
9/28/2024 8:00:00 AM Alicia M. Tucker, M.D. H2013: Section on Obesity Program
9/28/2024 8:00:00 AM Annie L. Andrews, M.D., MSCR H2011: Section on Emergency Medicine Program: Day 2
EmergiQuiz, State of the Section & SOEM Awards Presentations and 2024 PEMPix & Hot Topics in Pediatric Emergency Medicine
9/28/2024 12:00:00 PM Monika Goyal, M.D., M.S.C.E.
Prevalence of Period Poverty in a Pediatric Emergency Department
9/28/2024 3:30:00 PM Allison Markowsky, M.D., MSHS, FAAP S2412: What’s Trending in the Newborn Nursery: Controversies and Evidence
9/28/2024 5:00:00 PM Bernhard L. “Bud” Wiedermann, M.D., M.A., FAAP S2507: Should I Change My Clinical Practice Based on a Hot New Article? Reading Journals With a Critical Eye
9/29/2024 8:30:00 AM Dennis Ren, M.D. H3022: Section on Emergency Medicine Program: Day 3
Inaugural “Babble Royale” Competition & More Hot Topics in PEM; PediSonoFest
9/29/2024 9:00:00 AM Natasha Shur, M.D. S3208: Genetic Testing Boot Camp
9/29/2024 1:00:00 PM Lamia Soghier, M.D., M.Ed., M.B.A., FAAP H3037: Section on International Medical Graduates Program
Opportunities and Paths for Career Development in Academic Pediatrics
9/29/2024 4:00:00 PM Margaret Rush, M.D., MSHS I3304: Emergencies in the Technology-Dependent Medically Complex Children: What Every Pediatrician Should Know
9/29/2024 5:00:00 PM Anjna Melwani, M.D. S3501: Emergencies in the Technology-Dependent Medically Complex Children: What Every Pediatrician Should Know
9/30/2024 2:00:00 PM Rana F. Hamdy, M.D., M.P.H., MSCE S4302: Diagnosis and Treatment of Community-Acquired Pneumonia
10/1/2024 9:00:00 AM Nathaniel S. Beers, M.D., M.P.A., FAAP S5207: Eliminating Suspension, Expulsion, and Other Harsh Discipline Practices in Schools and Early Education

 

Around the world

Our Global Health Initiative launched in 2016 with the goal of eliminating pediatric health disparities around the world. We aim to address the most pressing pediatric health issues through better care for medically underserved populations. This leadership helps us achieve our mission of caring for all children. A broad range of education and research projects improves health outcomes. They also offer enriching opportunities for experienced faculty and emerging leaders to advance clinical excellence.

Healing hearts in Uganda

Dr. Craig Sable in Uganda

Dr. Craig Sable and team train partners in Uganda.

Craig Sable, M.D., interim chief of Cardiology, improves care for young people with rheumatic heart disease (RHD) in Uganda. Donors, including the Karp Family Foundation, Huron Philanthropies, Zachary Blumenfeld Fund and the Wood family, make this possible. RHD affects 50 million people, mostly children, worldwide. It claims 400,000 lives each year.

Dr. Sable and Ugandan partners completed important research showing that early RHD detection, coupled with monthly penicillin treatment, can protect the heart. They are working on practical solutions, such as a new portable device with artificial intelligence (AI) that can easily screen for RHD.

In 2023, Dr. Sable led two missions in Uganda where he and his team did surgeries and special tests for 18 children with RHD. They also taught local doctors new skills to help more kids on their own.

Plastic surgery and reconstructive care in Kenya and Nepal

Each year our Craniofacial & Pediatric Plastic Surgery team, under the leadership of Johnston Family Professor of Pediatric Plastic Surgery and Chief of Pediatric Plastic Surgery Gary Rogers, M.D., J.D., LL.M., M.B.A., M.P.H., provides opportunities for fellows to participate in surgical missions.

In 2024, Perry Bradford, M.D., traveled to the Moi Teaching Hospital in Eldoret, Kenya where she provided patients with burn, pressure wound and cleft reconstruction. She built community connections with the local plastic surgeons and educated registrars and medical students. “This gave me firsthand experience working in a community with limited resources and forced me to be more creative,” Dr. Bradford says. “The experience inspired me to examine what it means to have consistent access to advanced tools and equipment.”

In 2022, a group traveled to Nepal to provide care. Some patients arrived after days of travel by yak or buffalo. One child with a burn injury recovered use of her hand. The team educated local providers to deliver life-changing treatments unavailable in Nepal.

Dr. Tesfaye Zelleke in Ethiopia

Dr. Tesfaye Zelleke, left, and team in Ethopia.

Elevating epilepsy care in Ethiopia

Neurologist Tesfaye Zelleke, M.D., and partners in Ethiopia are seeking to improve the lives of children with epilepsy. The BAND Foundation provides support. Ethiopia has a population of about 120 million yet only a handful of pediatric neurologists.

Dr. Zelleke’s team trained nonspecialist providers to diagnose and treat children in the primary care setting. They also launched a mobile epilepsy clinic to provide community care and build the capacity of local clinicians. In collaboration with advocacy groups, the team educates the public about epilepsy with a goal of reducing stigma.

New hope in Norway

In 2023, our Division of Colorectal & Pelvic Reconstruction shared its expertise with clinicians at Oslo University Hospital, Rikshospitalet, in Norway. This effort was a key first step in Oslo becoming the first dedicated colorectal center in Scandinavia.

Marc Levitt, M.D., and team members performed complex surgeries otherwise unavailable for waiting patients. They led an academic conference. They held clinics to educate nurses, reviewed patient records and made care recommendations. Specialized care enabled a young patient with significant bowel difficulties to recover function and lead a normal life.

The team will travel to South Africa, the Czech Republic and Spain in 2024. Donors, including The Dune Road Foundation and Deanna and Howard Bayless, make this work possible.

Improving outcomes for babies in the Congo

AI can be a valuable tool for diagnosing genetic conditions. It detects unique facial patterns that clinicians without genetics training can miss. However, existing facial analysis software struggles in nonwhite populations.

A team led by Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor of Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation, is working to improve the newborn diagnosis rate worldwide. They are testing smartphone software in the Democratic Republic of Congo. Diverse newborn data improves AI’s ability to detect a variety of genetic conditions in more children. Early detection, diagnosis and informed care lead to better health outcomes.

Nephrology care for kids in Jamaica

Dr. Moxey-Mims and team in Jamaica

Jennifer Carver and Dr. Marva Moxey-Mims, center, with staff at Bustamante Children’s Hospital.

Marva Moxey-Mims, M.D., chief of Nephrology, is bringing care to children with kidney disease in Jamaica, with a goal of improving health equity. An International Pediatric Nephrology Association grant helped make it possible.

On a recent trip, Dr. Moxey-Mims and a small team — including Jennifer Carver, RN, CNN, lead peritoneal dialysis nurse at Children’s National, and three pediatric nephrologists from Jamaica — trained nearly 30 nurses from Jamaican hospitals. Nurses received hands-on dialysis education to improve their clinical skills. The team also worked to educate the community in disease awareness and prevention.

Read more stories like this one in the latest issue of Believe magazine.

Pilot program improves well-being of families during advanced care planning


Children with life-limiting rare diseases and their caregivers face tremendous stress and anxiety about the heart-breaking decisions before them. A new intervention – designed at Children’s National Hospital to support the palliative needs of these families – improved their spiritual and emotional well-being, according to new research published in the journal, Pediatrics.

Called FACE Rare (FAmily CEntered Pediatric Advance Care Planning Intervention for Rare Diseases), the counseling tools were found to be safe, effective and increased feelings of peace among families in this underserved population.

“Seventy-four percent of the families in that intervention group reported feelings of sadness, yet 100% reported our pilot intervention was a worthwhile experience,” said Maureen Lyon, Ph.D., a clinical psychologist and principal investigator at the Center for Translational Research at Children’s National. “If you’re talking about the possibility that the worst thing in the world would happen to you – that your child might die, and what you would want for them – the families found that our intervention helped. They had a place to process their feelings and consider what would be important to their child.”

The big picture

A rare disease is defined as any condition affecting fewer than 200,000 people in the United States. In pediatrics, these diseases often require constant caregiving and require families to face the cruel reality that the diagnosis may be life-limiting. In such cases, clinical teams often decide that conversations about advanced care planning (ACP) are needed.

The pilot-phase, randomized trial enrolled patients from Children’s National between 2021 and 2023. Research nurses underwent two days of training to be certified in the new intervention. Families assigned to the new approach received three, weekly 60-minute sessions and were evaluated using evidence-based assessment tools, including the Carer Support Needs Assessment Tool/Action Plan and the Next Steps: Respecting Choices Pediatric ACP Conversation. Measures of anxiety and spiritualization were tracked, and families returned after three months for follow-up.

Patients had a range of diagnoses that put tremendous strain on the caregivers, including complex digestive disorders, white-matter diseases and rare forms of epilepsy. Yet the caregivers expressed similar challenges: having time for themselves during the day, knowing what to expect in the future for their child’s care, and managing financial, legal and work issues.

What we found

Reinforcing the need to improve engagement and support for these families, the investigative team found that those who received the FACE Rare intervention reported higher levels of spiritual meaning and peace than those who received “treatment as usual.” All families living below the poverty line reported greater anxiety, and noteworthy to the team, black caregivers were less likely to report caregiver distress than non-black caregivers.

Dr. Lyon said future research needs to understand better how families respond to the challenges of rare diseases and unique social determinants of health that can change the approach to care.

“In some cultures, and families, having strong feelings is discouraged,” Dr. Lyon said. “We want to give families and caregivers space to have these emotions and think about what would be important for their child, if the worst were to happen. They appreciated that they participated in the program and had the space to consider these difficult questions.”

Read the full study, “Advance Care Planning for Children with Rare Diseases: A Pilot RCT,” in Pediatrics.

Novel cell therapy treatments offer promise to immune-compromised children

teenager receiving an intravenous infusion

In a first-of-its-kind clinical trial, researchers found that intravenous therapies made from virus-specific T-cells (VST) can effectively treat immunocompromised pediatric patients, far surpassing the current standard of care, according to new research published in Nature Communications.

More than 60% of patients in the phase 2 clinical trial led by investigators from Children’s National Hospital and Huntsman Cancer Institute responded to the innovative VST therapy. This new treatment uses blood from healthy donors to manufacture a highly specialized immune therapy that, when given to immune-compromised patients, prompts their immune system to fight off potentially life-threatening viruses, including cytomegalovirus, Epstein-Barr and adenovirus. Without this therapy, estimates suggest that less than 30% of patients would recover, using standard protocols.

“A vast majority of our patients not only responded to the therapy, but they were able to come off their antivirals, which come with extensive side effects,” said Michael Keller, M.D., the paper’s first author and the Translational Research Laboratory director at the Children’s National Cell Enhancement and Technologies for Immunotherapy (CETI) program. “This promising data suggests hope for patients with rare immune-compromising diseases that leave them vulnerable to so much in the world.”

The study brings together experts from the Pediatric Transplantation and Cell Therapy Consortium (PTCTC) and the Primary Immune Deficiency Treatment Consortium (PIDTC) to create the first multi-center, pediatric-consortium trial of adoptive T-cell therapies for viruses. It also represents one of the first to include critically ill patients, who are often excluded from research.

Children’s National leads the way

Working alongside Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research (CCIR), Dr. Keller and the CCIR team helped build an internationally recognized program, pioneering therapies to prevent complications from viral infections in immunocompromised patients. This includes patients with congenital immune deficiency and others who have undergone bone marrow transplantation for malignancies or non-malignant conditions, such as sickle cell disease.

While doctors can treat some immune-compromised patients for infections with standard antivirals, a small fraction don’t respond. Children’s National is one of a handful of hospitals in the country that has options. Over the last several decades, researchers have found ways to develop VST therapies made from banked T-cells, a more advanced application of how donated red blood cells are used to treat anemia.

In 2017, Drs. Keller and Bollard started collaborating with Michael Pulsipher, M.D.—now with Intermountain Primary Children’s Hospital and the Huntsman Cancer Institute at the University of Utah—to create a multi-institute clinical study. They combined the expertise at Children’s National in producing and banking cell therapy products with the community built around the PTCTC. Ultimately, they launched a clinical trial that was open to 35 centers in the U.S., enrolling 51 patients at 22 hospitals from 2018-2022.

“We wanted to prove that this potentially life-saving therapy could be given safely at regional pediatric centers that had never been able to use this approach before,” said Dr. Pulsipher, who served as the study’s co-principal investigator with Dr. Keller. “We united top experts in this area from the PTCTC and PIDTC and successfully treated some of the most challenging patients ever treated with this approach. Our findings helped define who can benefit the most from this therapy, paving the way for commercial development.”

The Good Manufacturing Practices (GMP) laboratory at Children’s National, led by Patrick Hanley, Ph.D., provided suitable VST therapies for 75 of 77 patients who requested to join the study. Clinical responses were achieved in 62% of patients who underwent stem cell transplants and in 73% of patients who were treated with VST and evaluated one month after their infusion. The paper laid out risks and clinical factors impacting outcomes when third-party donors are used to manufacture the VST therapies.

What’s ahead

Given that researchers are only beginning to develop cell therapies, work remains to understand the many ways they interact with the immune system. In a separate paper also recently published in Nature Communications, members of the multi-institute team documented a case of an infant with severe combined immune deficiency, who faced extremely rare side effects when the VST treatment interfered with her donor bone marrow graft. The case led the team to work with the Food and Drug Administration to identify criteria for VST donors enrolled in this study to mitigate complications.

In the decade ahead, Dr. Bollard sees promise in cell therapies for patients with cancer, immune deficiencies after transplant and dozens of other disorders, including genetic and autoimmune diseases. “Future studies will continue to look at ways to optimize the manufacturing, the administration and the long-term outcomes for these therapies—and to enhance the lifelong impact on our patients,” she said. “When we pair human ingenuity with the power of technology, I see tremendous potential.”

Acknowledgments: This study was funded with a nearly $5 million grant from the California Institute of Regenerative Medicine and was run through the operations center at the Children’s Hospital of Los Angeles, where Dr. Pulsipher was formerly on faculty.

Inequity complicates care for children with tracheostomies

child with a tracheostomy tube

Study finds healthcare inequities complicate care of children with tracheostomies.

Approximately 4,000 children in the United States annually undergo tracheostomies, a lifesaving procedure for severe respiratory compromise or upper-airway obstruction. While children with tracheostomies are living longer because of technological advances, their caretakers encounter a variety of issues once at-home care begins, as reported in a study published in Qualitative Health Research.

The study identified lingering problems from the lack of equitable access to appropriate support for patient families and caregivers. Jules Sherman, M.F.A., director of the Biodesign Program at Children’s National Innovation Ventures, led the research in collaboration with Kyle L. Bower, Ph.D., a researcher and instructor at The University of Georgia, and Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National.

The big picture

Interview subjects were 11 primary caregivers of children ages 16 months to 11 years who had a tracheostomy and were supported by multiple lifesaving devices at home such as a ventilator, humidifier and other required medical supplies.

Subjects were questioned about influences and relationships they believed impacted the trajectory of care given to children with tracheostomies. Three themes emerged:

  • Insufficient peer support: Caregivers struggled to find groups who shared similar experiences to help them navigate the uncertainty surrounding their child’s medical care, and content on social media peer groups sometimes heightened their sense of worry.
  • Ineffective trach-care training:
    • For families — Caregivers said the trach-care training they received at their respective hospitals did not reflect real-time situations at home, resulting in high levels of anxiety, multiple home emergency situations and routine calls to 911.
    • Home health nurses — Caregivers shared instances of poor decision-making by at-home nurses in emergency situations, lack of communication with primary caregivers and lack of respect for their input.
  • Challenges facing integrated care: The absence of cohesion between medical professionals, durable medical equipment (DME) companies and insurance providers creates many challenges for caregivers. Due to the excessive cost of lifesaving medical devices and negative interactions by caregivers with insurance companies and DMEs, some caregivers reported turning to the black market to purchase needed medical supplies.

Why it matters

In the United States, 60 percent of children with tracheostomies are readmitted to the hospital for tracheostomy-associated respiratory infection within the first six months of the surgery. This study highlights systemic failures and inequities within the healthcare system, beginning at the hospital and following affected families into their homes, indicating that improvements in the support system, training and healthcare coverage are sorely needed.

The need for a better tracheostomy solution was introduced to Children’s National’s Innovation Ventures by two hospital physicians who are otolaryngologists, Habib Zalzal, M.D. and Rahul Shah, M.D., M.B.A. Followed by an initial market study and design by design firm Archimedic, study author, Sherman, continues working with clinicians at Children’s National and engineers at the University of Oregon and University of Maryland to enhance the prototype developed at Children’s National. The device, called as Trach Sense, is designed to detect accidental decannulation and obstruction events, which are leading causes of tracheostomy-related complications.

This study was supported by the Food and Drug Administration grants P50FD006430 and P50FD007965. The contents are those of the authors and do not necessarily represent the official views of, nor an endorsement, by the FDA, Department of Health and Human Services or the U.S. government.

Pioneering research center aims to revolutionize prenatal and neonatal health

  • Posters at the REI Week 2025 Monday, March 31 poster session.

    Posters at the REI Week 2025 Monday, March 31 poster session.
  • Panelists discuss innovation during REI Week 2025.

    Panelists discuss innovation during REI Week 2025.
  • Global Health Initiative community engagement event during REI Week 2025.

    Global Health Initiative community engagement event during REI Week 2025.
  • Chris Rees presents his REI Week 2025 lecture.

    Chris Rees presents his REI Week 2025 lecture.
  • Nathan Kuppermann listens to a presenter during the REI Week 2025 Tuesday, April 1, poster session.

    Nathan Kuppermann listens to a presenter during the REI Week 2025 Tuesday, April 1, poster session.
  • Michelle Riley-Brown, Nathan Kuppermann, Catherine Bollard and Naomi Luban on stage during the REI Week 2025 awards ceremony.

    Michelle Riley-Brown, Nathan Kuppermann, Catherine Bollard and Naomi Luban on stage during the REI Week 2025 awards ceremony.
  • Brandy Salmon presents on innovation programs at Virginia Tech during the REI Week 2025 Innovation Day.

    Brandy Salmon presents on innovation programs at Virginia Tech during the REI Week 2025 Innovation Day.
  • Catherine Bollard listens to a presenter during the REI Week 2025 Monday, March 21 poster session.

    Catherine Bollard listens to a presenter during the REI Week 2025 Monday, March 21 poster session.
  • Ambroise Wonkman poses for a picture with Children’s National staff.

    Ambroise Wonkman poses for a picture with Children’s National staff.
  • Tanzeem Choudhury presenting during REI Week 2025.

    Tanzeem Choudhury presenting during REI Week 2025.

Catherine Limperopoulos, Ph.D., was drawn to understanding the developing brain, examining how early adverse environments for a mother can impact the baby at birth and extend throughout its entire lifetime. She has widened her lens – and expanded her team – to create the new Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital.

“Despite the obvious connection between mothers and babies, we know that conventional medicine often addresses the two beings separately. We want to change that,” said Dr. Limperopoulos, who also directs the Developing Brain Institute. “Given the current trajectory of medicine toward precision care and advanced imaging, we thought this was the right moment to channel our talent and resources into understanding this delicate and highly dynamic relationship.”

Moving the field forward

Since its establishment in July 2023, the new research center has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.

Dr. Limperopoulos has been at the forefront of groundbreaking research, directing attention to the consequences of maternal stress on the unborn baby and the placenta. In addition, under the guidance of Kevin Cook, Ph.D., investigators published a pivotal study on the correlation between pain experienced by premature infants in the Neonatal Intensive Care Unit and the associated risks of autism and developmental delays.

Another area of research has focused on understanding the impact of congenital heart disease (CHD) on prenatal brain development, given the altered blood flow to the brain caused by these conditions during this period of rapid development. Led by Josepheen De Asis-Cruz, M.D., Ph.D., a research team uncovered variations in the functional connectivity of the brains of infants with CHD. In parallel, Nickie Andescavage, M.D., and her team employed advanced imaging techniques to identify potential biomarkers in infants with CHD, holding promise for guiding improved diagnostics and postnatal care. Separately, she is investigating the impact of COVID-19 on fetal brain development.

In the months ahead, the team plans to concentrate its efforts on these areas and several others, including the impact of infectious disease, social determinants of health and protecting developing brains from the negative impacts of maternal stress, pre-eclampsia and other conditions prevalent among expectant mothers.

Assembling a team

Given its robust research plan and opportunities for collaboration, the center pulled together expertise from across the hospital’s faculty and has attracted new talent from across the country, including several prominent faculty members:

  • Katherine L. Wisner, M.S., M.D., has accumulated extensive knowledge on the impact of maternal stress on babies throughout her career, and her deep background in psychiatry made her a natural addition to the center. While Dr. Wisner conducts research into the urgent need to prioritize maternal mental health, she will also be treating mothers as part of the DC Mother-Baby Wellness Initiative — a novel program based at Children’s National that allows mothers to more seamlessly get care for themselves and participate in mother-infant play groups timed to align with their clinical appointments.
  • Catherine J. Stoodley, B.S., M.S., D.Phil., brings extensive research into the role of the cerebellum in cognitive development. Dr. Stoodley uses clinical studies, neuroimaging, neuromodulation and behavioral testing to investigate the functional anatomy of the part of the brain responsible for cognition.
  • Katherine M. Ottolini, M.D., attending neonatologist, is developing NICU THRIVE – a research program studying the effects of tailored nutrition on the developing newborn brain, including the impact of fortifying human milk with protein, fat and carbohydrates. With a grant from the Gerber Foundation, Dr. Ottolini is working to understand how personalized fortification for high-risk babies could help them grow.

Early accolades

The new center brings together award-winning talent. This includes Yao Wu, Ph.D., who recently earned the American Heart Association’s Outstanding Research in Pediatric Cardiology award for her groundbreaking work in CHD, particularly for her research on the role of altered placental function and neurodevelopmental outcomes in toddlers with CHD. Dr. Wu became the third Children’s National faculty member to earn the distinction, joining an honor roll that includes Dr. Limperopoulos and David Wessel, M.D., executive vice president and chief medical officer.

Interim Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., said the cross-disciplinary collaboration now underway at the new center has the potential to make a dramatic impact on the field of neonatology and early child development. “This group epitomizes the Team Science approach that we work tirelessly to foster at Children’s National,” Dr. Bollard said. “Given their energetic start, we know these scientists and physicians are poised to tackle some of the toughest questions in maternal-fetal medicine and beyond, which will improve outcomes for our most fragile patients.”

New hemimegalencephaly procedure is all about teamwork

Children’s National experts pioneered a novel approach of inducing strokes to stop seizures and improve neurodevelopmental outcomes in newborns under three months old with hemimegalencephaly (HME). The procedure, called an endovascular embolic hemispherectomy, can be safely used to provide definitive treatment of HME-related epilepsy in neonates and young infants. Monica Pearl, M.D., neurointerventional radiologist, and Panagiotis Kratimenos, M.D., Ph.D., neonatologist, discuss why having a multidisciplinary team skilled at this procedure is the reason we’re the only center in the world capable of providing this treatment.

The history behind the novel hemimegalencephaly procedure

Traditionally, when a baby is diagnosed with hemimegalencephaly (HME), doctors turn to a hemispherectomy at 3 months of age, which involves surgically removing half of a baby’s brain. At Children’s National Hospital, our doctors pioneered the endovascular embolic hemispherectomy, an approach using induced controlled strokes to eliminate the affected part of the brain, halting seizures. Monica Pearl, M.D., neurointerventional radiologist, and Tammy Tsuchida, M.D., Ph.D., neonatal neurologist, talk about this life-changing procedure.

Inducing strokes to better treat babies with hemimegalencephaly

When a family from Texas received a shocking diagnosis for their newborn daughter, they knew there was one place they needed to go – Children’s National Hospital in Washington, D.C. At birth, Angelique was diagnosed with a rare and devastating condition known as hemimegalencephaly (HME) which causes uncontrollable and frequent seizures. Monica Pearl, M.D., neurointerventional radiologist, and the team at Children’s National have pioneered an approach to treat HME, where they induce controlled strokes to eliminate the affected part of the brain, halting seizures in their tracks. They’re the only team in the world doing this work. Angelique’s parents knew the clock was ticking — every day they waited meant irreversible damage to their daughter’s developing brain.

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)

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)