Experts from Children’s National Hospital presented research at the 8th Annual 2024 T1DX-QI (Type 1 Diabetes Exchange Quality Improvement Collaborative) Learning Session held in Chicago, Ill. This session emphasizes delivering equitable, evidence-based and cost-effective diabetes care through integrating clinical science, quality improvement, technology and advocacy.
Oral Presentations:
Assessing Readiness to Transition to Adult Care among Young Adults with T1D by Jody Grundman, M.D.
Improving Microalbuminuria Screening Rates Among Pediatric Diabetes Patients: A Clinic-Wide Initiative by Sarah Lydia Holly, BSN, RN.
Addressing Disparities in Diabetes Care: Implementing SDOH Screening at Diagnosis by Sarah Lydia Holly, BSN, RN.
Developing a Tracking Tool for Insulin Pump Prescriptions Among Children and Adolescents with Type 1 & Type 2 Diabetes by Amanda Perkins, N.P., C.P.N.P., M.P.H.
2024 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.
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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Researchers from Children’s National Hospital participated in the 50th anniversary conference of the International Society for Pediatric and Adolescent Diabetes (ISPAD) in Lisbon, Portugal. As the only global organization dedicated to all forms of childhood diabetes, ISPAD promotes clinical and basic science, research, education and advocacy for children and adolescents with diabetes. The conference provided members worldwide with a platform to exchange scientific and clinical insights on pediatric and adolescent diabetes.
SDOH: The Importance of Screening, Resource Delivery, and Follow-up by Sarah Holly, Lauren DeAnna, Roxanna Velasquez, Jasmine Roberts, Shideh Majidi, M.D.
Investigating Associations among Demographics, Medical Variables, Caregiver Fear of Hypoglycemia, and Sleep Quality among Parents of Children with Type 1 Diabetes (T1D) by Rachel Longendyke, M.D., Christine Wang, John Barber, Randi Streisand, Ph.D.
The participation of Children’s National researchers highlights their commitment to advancing diabetes care and research for children and adolescents. By collaborating with global experts, they continue to contribute collective efforts to improve outcomes for young people living with diabetes worldwide.
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.
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In this webinar, Shideh Majidi, M.D., M.S.C.S., director of the Childhood and Adolescent Diabetes Program at Children’s National Hospital, discussed early antibody screening and use of teplizumab in children.
Key takeaways
Current state of type 1 diabetes: Type 1 Diabetes (T1D) is characterized by absolute insulin deficiency due to pancreatic beta-cell destruction, often diagnosed in childhood but can occur in adults as well. It requires lifelong insulin management.
Increased risk and screening: Individuals with family members who have T1D are at higher risk of developing T1D, but most newly diagnosed cases have no family history. Early screening can help reduce diabetic ketoacidosis (DKA) rates and improve management.
Stages of type 1 diabetes: T1D is classified into three stages: Stage 1 (normal blood sugars with 2 or more autoantibodies), Stage 2 (dysglycemia with 2 or more autoantibodies), and Stage 3 (clinical T1D requiring insulin).
Early screening and follow-up: Screening for diabetes autoantibodies can be done through various programs and labs. Follow-up involves regular monitoring based on the number of antibodies and blood sugar levels. If patients have antibodies or abnormal blood sugars, they can be referred to the T1Delay Program at Children’s National (through an endocrine/diabetes referral).
Teplizumab (T-zield) treatment: T-zield is an FDA-approved treatment for delaying the onset of Stage 3 T1D in individuals aged 8 and older with Stage 2 T1D. It involves a 14-day infusion regimen and has been shown to delay insulin need by a median of 2 years.
Clinical implementation and support: Children’s National offers T-zield treatment through a multi-departmental effort. The program aims to improve both quality of life and clinical outcomes for patients.
More information
For more information on this webinar, as well as access to the presentation slide deck, visit our website.
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Children’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:
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A team of experts at Children’s National Hospital, using the input of teens with Type 1 diabetes from our Children’s National clinics, adapted a six-week virtual group diabetes education program and mindfulness-based intervention. In their new study published in Journal of Pediatric Psychology, the team described how the intervention might work to support teens with Type 1 diabetes experiencing anxiety or depression.
The findings showed that both the mindfulness-based intervention and the diabetes education program were successfully implemented and valued by the participating teens. While the study was limited in scope, focusing primarily on assessing the programs’ acceptance and perceived value among adolescents, preliminary results suggest participation in the programs may also contribute to improvements in mood, diabetes-related distress and glycemic control.
A: Many adolescents with Type 1 diabetes experience symptoms of anxiety and depression, yet it can be hard to access care to relieve some of these symptoms. Group-based, brief virtual care may be a way to provide more access to necessary support. Testing whether such programs are desired and useful by teens and their families, and whether they show promise for improving symptoms of depression and anxiety, is an important step in addressing this gap in care.
Q: How does this work move the field forward?
A: This study helped us to use the input of the teens we hope to serve to create and adapt programs that they were enthusiastic about and test them to see if they were helpful. We used these findings to obtain a second grant from the National Institutes of Health to do a larger scale study to see whether these programs help with depression, anxiety, eating, stress and diabetes management.
Q: What’s the goal and how will this work benefit patients?
A: Our ultimate goal is to create easy to access programs which help improve anxiety, depression and diabetes management in teens with Type 1 diabetes. We are trying to ensure that these programs are feasible and accessible and could be translated into our clinics if they prove to be beneficial. We hope to improve the ways we identify teens experiencing anxiety and depression and provide them the option for this brief group-based care in our clinics.
Q: What did you find that excites you and what’s next?
A: We were very excited to find that teens were interested in enrolling in these programs and found them useful. We were also excited to see that our very preliminary data indicates that mindfulness-based interventions may improve depression and even possibly glycemic control.
Children’s National is the first institution that we know of to use teen feedback to adapt a mindfulness-based intervention specific to teens with Type 1 diabetes. Our team is now preparing to embark on a five-year project in collaboration with the Barbara Davis Center in Colorado to evaluate the effectiveness of these programs with a larger group of teens.
The American Diabetes Association 84th Scientific Sessions were held in Orlando, Florida. The meeting presented an opportunity for researchers and health care professionals to exchange knowledge on the latest scientific advances and breakthroughs in diabetes. Participants included:
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A patient with short stature and growth hormone (GH) resistance experienced a growth rate increase of 3.4 cm/year after 12 months of high-dose GH therapy.
In a first-of-its-kind single-patient clinical trial, a patient with short stature and growth hormone (GH) resistance experienced a growth rate increase of 3.4 cm/year after 12 months of high-dose GH therapy as part of a precision medicine approach. The study, led by Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology at Children’s National Hospital, was published in The Journal of Clinical Endocrinology & Metabolism.
What this means
“Many patients with short stature do not get a definitive genetic diagnosis, and even if they do, it takes substantial effort to design a targeted therapeutic trial,” says Dr. Dauber.
This is the first trial of extremely high-dose growth hormone in a single patient with a unique form of growth hormone resistance.
Children’s National leads the way
During an endocrine evaluation for short stature, which included GH stimulation testing demonstrating growth hormone resistance, a next-generation sequencing panel revealed that the patient had an inherited heterozygous frameshift variant in the growth hormone receptor gene (GHR).
The researchers found that the patient’s growth hormone binding protein (GHBP) levels were elevated because of this variant, limiting circulating GH’s ability to bind to the GH receptor. The researchers believed that, with extremely high doses of GH, the mechanism of resistance would be overcome and normal GH signaling would be restored.
“We found that we were able to overcome the patient’s growth hormone resistance, which resulted in substantially improved growth,” says Dr. Dauber. “Our understanding of the underlying pathophysiology of his growth disorder led to our ability to design a precision medicine trial just for this single patient.”
Genetic testing and translational biology can provide insights into the specific underlying mechanism of rare growth disorders, allowing for precision medicine approaches.
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Trial participant Mia Maric is measured by Dr. Andrew Dauber.
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. The results were presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting.
The big picture
Recently approved to increase linear growth and open growth plates in children with achondroplasia, vosoritide is a C-type natriuretic peptide analog that binds its receptor on chondrocytes, leading to increased chondrocyte proliferation and differentiation by inhibiting the ERK1/2-MAPK pathway.
“Vosoritide directly targets the pathway in the growth plate that is affected by the genetic mutation causing hypochondroplasia,” said Andrew Dauber M.D., M.M.Sc., chief of Endocrinology at Children’s National and lead author of the study.
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.
The patient benefit
Ivan Maric’s 11-year-old daughter, Mia, has been participating in the trial for the last 18 months. In 2022, they moved from Croatia to be part of the study.
“This has been life-changing for Mia,” Maric said. “Soon after receiving the initial doses, we immediately noticed growth. Now, she can independently manage everyday tasks such as washing her hair and reaching the sink to wash her hands.”
What’s next
Vosoritide treatment may work as a precision therapy to improve growth in multiple genetic conditions that interact with the ERK1/2-MAPK pathway.
“This study provides a proof of principle that this medicine will work for these children and supports further research in this area,” said Dr. Dauber. “I was excited to see how well tolerated the medication was and how some patients had excellent responses.”
This clinical trial funded by BioMarin is the first-of-its-kind to treat children with genetic short stature who do not have achondroplasia. Other growth-related conditions included in this phase 2 trial were Noonan syndrome, NPR2 mutations and Aggrecan mutations.
“In many circumstances, access to care for precocious puberty has the same issue that we have with access to healthcare in general. Many of these children may not get care in a timely manner.”
Roopa Kanakatti Shankar, M.D., M.S., pediatric endocrinologist, spoke to NBC News about the uptick in early puberty and the importance of access to care for those who need treatment. Read her interview with NBC News.
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Weighing approximately 50 pounds, the food packages are rich in protein, fiber and healthy fats, and include fruits and vegetables.
Social Determinants of Health are an essential focus in providing equitable diabetes care. Food and nutrition are integral parts of Type 1 diabetes (T1D) and Type 2 diabetes (T2D) management. Food insecurity increases the risk for T2D and is associated with higher A1C levels and hospitalization in those with diabetes.
The big picture
A study published in the American Diabetes Association Journal Diabetes assessed food insecurity among families with diabetes during their clinical visits. As of June 2023, 62% of the participants screened positive for food insecurity. In response, Children’s National Hospital partnered with the Capital Area Food Bank to create a food pharmacy within the diabetes clinic that supplies an average of 117 families per month with additional groceries to support those affected by diabetes.
What does this mean?
These packages are designed to sustain a family of four for three days. Weighing approximately 50 pounds, pantry essentials are rich in protein, fiber and healthy fats, and include fruits and vegetables. Additionally, the packages come with recipe cards that offer helpful suggestions for replacing or supplementing items, enabling individuals to obtain nutritious food beyond their diabetes appointments.
“We hope that these findings and future research will help bring to light the importance of food security, in addition to medication, in managing chronic illness,” said Alexis Richardson, M.S., R.D., L.D.N., C.D.C.E.S., food pharmacy founder and diabetes educator at Children’s National. “This research also opens up the possibilities for other studies on the effect food insecurity and Type 2 diabetes.”
What’s been the hold-up in the field?
Providing 50 pounds per set to over 100 families each month requires a substantial area for a food pharmacy. The storage facility must adhere to food safety standards and comply with Department of Health regulations. Additionally, all staff members are required to be ServSafe certified. Shortages in staffing and funding pose significant challenges. Clear procedures are needed for tasks such as placing orders, receiving food deliveries, managing loading docks and handling the refrigeration and shelving of bags.
Over the years, Children’s National has engaged with numerous programs, sharing their experiences and challenges to offer guidance and assist other institutions in establishing their own food pharmacies.
The iLet Bionic Pancreas was recently cleared by the U.S. Food and Drug Administration (FDA) and is now commercially available.
In 2021, Children’s National Hospital participated in a multi-center clinical trial to test the efficacy of the iLet Bionic Pancreas — a device that automatically regulates blood sugar levels in patients with Type 1 diabetes. The iLet Bionic Pancreas was recently cleared by the U.S. Food and Drug Administration (FDA) and is now commercially available.
“The bionic pancreas serves as an additional resource empowering patients to effectively manage their Type 1 diabetes with confidence,” says Fran Cogen, M.D., CDCES, director emerita of the Childhood and Adolescent Diabetes Program at Children’s National. “It requires minimal input from the patient.”
During initialization of the pump, patients will no longer need to enter carbohydrate amounts, just their weight. After the patient indicates whether they are having a usual meal, smaller than usual meal, or larger than usual meal, the device’s algorithms will adjust insulin doses automatically.
“Patients will not need to add correction insulin,” says Dr. Cogen. “There are 3 algorithms – one to adjust background insulin, one to adjust insulin needed to cover carbohydrates and one to adjust insulin needed to correct high blood sugars. The background insulin dosing will also be adjusted if the blood sugars decrease or become low.”
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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.
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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Advanced 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.
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
https://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/2023-with-lightbulb-CNRI-feature.png385685Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2023-12-27 10:55:522023-12-28 11:49:10The best of 2023 from Innovation District
“Select patients will have the opportunity to participate in research studies focused on cutting-edge genomic investigations into their growth disorders,” says Youn Hee Jee, M.D., M.Med., endocrinologist.
“We’re dedicated to unraveling the mysteries that families have long sought answers to,” says Andrew Dauber, M.M.Sc., M.D., chief of Endocrinology at Children’s National Hospital. “There are numerous endocrine and genetic conditions with the potential to impact a child’s growth. That’s why we’ve assembled a team of leading endocrinologists and geneticists to create a new Growth Specialty Clinic and address these issues with a fresh perspective.”
This team, combined with the expertise of the hospital’s translational scientists, is making significant progress in identifying the causes of a variety of growth disorders and developing innovative treatments. And at the core of this work, Dr. Dauber says, is a recognition of the unique impact endocrine disorders have on each individual child.
Dr. Shur emphasizes the commitment to providing answers for these families. “This collaborative effort goes beyond diagnosis; it opens doors to potential treatment options.”
The Growth Specialty Clinic is for children with severe undiagnosed growth disorders that are suspected to have a genetic etiology and children with rare genetic diagnoses who would benefit from the expertise of practitioners more familiar with those disorders. It is also closely linked to the Center for Genetic Medicine Research.
“Select patients will have the opportunity to participate in research studies focused on cutting-edge genomic investigations into their growth disorders,” says Youn Hee Jee, M.D., M.Med., endocrinologist.
In one case, Dr. Jee identified a new genetic cause of an overgrowth syndrome. Rare genetic conditions known as generalized overgrowth syndromes manifest as excessive body growth during fetal life and/or childhood, frequently resulting in tall stature. She is investigating the mechanisms that promote healthy bone growth.
Additionally, Dr. Jee identified a new genetic cause of short stature. Her research showed that the identified genetic cause impairs the recycling of essential proteins for growth, expanding our knowledge of human growth.
Moving the field forward
“We’re taking innovative approaches to treatment by leveraging our insights into the genetic origin of each patient’s growth disorder,” says Dr. Dauber.
In the brief time since the clinic’s launch, several new diagnoses and treatment pathways have already been offered. In one single-patient study, researchers were able to successfully overcome the patient’s growth hormone resistance using a targeted approach, and the patient has shown significant catch-up growth after one year of treatment.
Children’s National is also at the forefront of other groundbreaking research, launching novel clinical trials that are advancing the field of endocrinology:
Vosoritide clinical trial: Children’s National has the first clinical trial in the world testing Vosoritide in children with certain genetic causes of short stature. Researchers have enrolled approximately 50 subjects with exciting preliminary results for patients with Noonan syndrome, Aggrecan gene mutations and NPR2 gene mutations. All 24 hypochondroplasia patients have completed the 18-month trial. Dr. Dauber intends to present results at the 2024 American College of Medical Genetics meeting in Toronto.
Hypochondroplasia study: Children’s National is the first site to launch BioMarin’s new natural history study for children with hypochondroplasia which will also be a lead into their future Phase 3 trial.
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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.
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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.”
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There will be over 20 Children’s National Hospital-affiliated participants at this year’s American Academy of Pediatrics National Conference and Exhibition. The meeting will take place in Washington, D.C., from October 20 – October 24. We have compiled their sessions into a mini schedule below.
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Children with hypochondroplasia have low parent-reported quality of life (QOL) scores, according to findings from researchers at Children’s National Hospital.
Children with hypochondroplasia have low parent-reported quality of life (QOL) scores, according to findings from researchers at Children’s National Hospital. The data, presented as part of a poster presentation at the Pediatric Endocrine Society (PES) annual meeting, also found older age and shorter height may further exacerbate effects on QOL.
Hypochondroplasia is a skeletal dysplasia that has an estimated prevalence of 1 in 15,000-40,000 births and is characterized by short stature and disproportionately short arms, legs, hands and feet.
Participants of the study were 13 prepubertal boys ages 3-11 and 13 prepubertal girls ages 3-10, all with height z-scores < -2.25 SD and genetically proven hypochondroplasia.
Moving the field forward
Effective medications for growth in patients with hypochondroplasia are limited. Children’s National is participating in an ongoing study of a new drug, vosoritide, used to treat children in this population and will compare pre- and post-intervention QOL scores.
How will this work benefit patients?
Understanding QOL of children with hypochondroplasia will help clinicians provide better care and support.
“Knowing how a medication affects QOL will help guide counseling about use of this medication,” says Nicole Rangos, M.D., pediatric resident at Children’s National and lead author of the study.
Dr. Rangos received the Human Growth Foundation Award for best growth-related abstract at the PES annual meeting in May 2023. The award was established in 2007 to encourage fellows training in pediatric endocrinology to pursue clinical and bench investigations that may lead to a better understanding of human growth.
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