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Innovations in Review 2024

Pediatrics

For over 100 years, our pediatric experts at Columbia and Weill Cornell Medicine have been caring for children and adolescents with common and complex conditions to help them live longer, healthier, and happier lives. In 2024, physicians and researchers pioneered numerous advances in the field of pediatric care, including the use of novel devices, new chemotherapy approaches, genome sequencing, surgical techniques, and screening practices to improve patients’ physical and mental health.

Pediatrics
Pediatrics

Read more about our top research and key innovations from 2024:

Novel Technology Helps Children with Pulmonary Valve Disease

Intra-Arterial Chemo Advances Treatment of Pediatric Tumors

Reducing Waitlist Mortality for Pediatric Liver Transplant

Understanding Co-Occurrence of Celiac Disease & Eosinophilic Esophagitis

The Connection Between Youth Anxiety Disorders & OCS

Against the Odds: Improving Survival for Children with Pulmonary Vein Stenosis

Cracking the Code: Sequencing the Hodgkin Lymphoma Genome to Uncover New Precision Therapies

A Novel Approach to Pediatric Pulmonary Valve Replacement

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Novel Technology Helps Children with Pulmonary Valve Disease

For pediatric patients with pulmonary valve disease, treatment options are limited, as most mechanical valves are only available in adult sizes and tissue-based valves can wear out quickly. Oliver Barry, MD, a pediatric interventional cardiologist at NewYork‑Presbyterian and Columbia, approached this unique challenge by partnering with Emile Bacha, MD, chief of the Division of Cardiac, Thoracic, and Vascular Surgery at NewYork‑Presbyterian and Columbia, to become one of the sites for the first-in-human trial for the Autus Size-Adjustable Valve. Made of a stainless-steel frame and synthetic leaflets, the Autus Valve can be implanted through a minimally invasive procedure and expanded over time to grow with the child. The study results show that the novel device may be a game changer for children with right-sided congenital heart diseases including double outlet right ventricle, pulmonary atresia with intact ventricular septum, pulmonary stenosis, or Tetralogy of Fallot.

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Autus Size-Adjustable Valve shown at initial and fully expanded sizes. Image courtesy of Autus Valve Technologies, Inc.

Intra-Arterial Chemo Advances Treatment of Pediatric Tumors

Y. Pierre Gobin, MD, director of Interventional Neuroradiology at NewYork‑Presbyterian and Weill Cornell Medicine, first discovered the feasibility of using intra-arterial chemotherapy (IAC) to treat retinoblastoma in 2008, when 95% of children with this form of cancer lost at least one eye. In partnership, Mark Souweidane, MD, director of pediatric neurological surgery at NewYork‑Presbyterian and Weill Cornell Medicine, is building off Dr. Gobin’s work and leading the world’s first clinical trial assessing IAC for two extremely rare pediatric brain tumors—choroid plexus carcinoma and atypical choroid plexus papilloma—which historically have a less than 30%– 40% three-year survival rate. Results from the early clinical trial were promising and suggest that there are opportunities to continue evolving and adopting IAC to treat various cancers.

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A choroid plexus carcinoma presenting with symptoms of hydrocephalus.

Reducing Waitlist Mortality for Pediatric Liver Transplant

Mercedes Martinez, MD, medical director of the Intestinal Transplant Program at the Center for Liver Disease and Abdominal Organ Transplantation at NewYork‑Presbyterian and Columbia, has advocated for increased use of technical variant liver transplantation (TVLT) to reduce the waitlist mortality for children in need of liver transplantation and has been addressing misconceptions about the lifesaving technique. Based on research, when high-volume transplant centers use TVLT to create liver grafts, fewer children on the liver transplant waiting list die before receiving a transplant. Increased adoption of this technique has the potential to create more opportunities for saving more lives.

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Reducing Waitlist Mortality for Pediatric Liver Transplant

Understanding Co-Occurrence of Celiac Disease & Eosinophilic Esophagitis

Arielle Bergman, MD, a pediatric gastroenterologist at NewYork‑Presbyterian and Weill Cornell Medicine, conducted a study to assess the overlap between two conditions with food triggers—celiac disease and eosinophilic esophagitis (EoE). The findings from this study indicated that the two diseases may have pathophysiological overlap and that larger prospective studies are warranted to gain more information about this overlap so that clinicians can update best practices. Based on her research, Dr. Bergman recommended that for all patients with celiac disease, clinicians should obtain biopsies from multiple levels in the esophagus, especially if the patient has a high eosinophil count, in order to ensure the diagnosis and treatment is most effective.

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Understanding Co-Occurrence of Celiac Disease & Eosinophilic Esophagitis

The Connection Between Youth Anxiety Disorders & OCS

Subclinical obsessive and compulsive symptoms (OCS) are highly prevalent among children and adolescents with anxiety disorders. Last year, Kate Fitzgerald, MD, a child psychiatrist at NewYork‑Presbyterian and Columbia, published the results of a study looking at 206 youth—158 with an anxiety disorder and 48 healthy controls—with the goal of identifying the baseline prevalence, comorbidity, and clinical implications that subclinical obsessive and/ or compulsive symptoms has in youth with anxiety disorders. She found that pediatric patients with generalized anxiety disorder were tightly clustered with OCS and had a high risk for later emergence of obsessive-compulsive disorder (OCD) according to self-reported measures. Dr. Fitzgerald has suggested that all youth with generalized anxiety disorder should be assessed for comorbid OCS or OCD. This research has implications for the future of generalized anxiety disorder management as an improved course of care.

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The Connection Between Youth Anxiety Disorders & OCS

Against the Odds: Improving Survival for Children with Pulmonary Vein Stenosis

Pulmonary vein stenosis (PVS) is a rare and challenging condition that narrows the blood vessels around the lungs and typically impacts newborns. Christopher Petit, MD, division chief of pediatric cardiology and co-director of the Children’s Heart Center at NewYork‑Presbyterian and Columbia, has led research in the use of a systemic oral therapy typically used in cancer treatment as well as a hybrid surgical method to treat patients with severe disease. On the Advances in Care podcast, Dr. Petit discusses his career as an innovator for this rare heart disease and his determination to drastically improve survival rates for his patients.

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Advances in Care podcast featuring Dr. Christopher Petit

Cracking the Code: Sequencing the Hodgkin Lymphoma Genome to Uncover New Precision Therapies

For Lisa Roth, MD, director of pediatric oncology at NewYork‑Presbyterian and Weill Cornell Medicine, finding new treatment options for Hodgkin lymphoma has been personal. On the Advances in Care podcast, Dr. Roth discusses her own journey with the very cancer she is dedicating her career to studying, and how she and her team became the first scientists to map the entire Hodgkin lymphoma genome, uncovering new pathways to treat this historically understudied cancer type.

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Advances in care podcast featuring Dr. Lisa Roth

A Novel Approach to Pediatric Pulmonary Valve Replacement

Oliver Barry, MD, a pediatric interventional cardiologist at NewYork‑Presbyterian and Columbia, discusses how the Autus Size-Adjustable Valve is a new option for pediatric patients with pulmonary valve disease that can greatly improve quality of life and outcomes. See how the novel technology functions and how it can grow with the patient through minimally invasive catheter procedures that allow patients to avoid multiple open-heart surgeries.

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The Autus Valve is uniquely suited to pediatric patients as it’s designed to be able to match the patient’s growth, avoiding future open-heart procedures.