Nationwide Children's Hospital

Searching the Druggable Genome using Large Language Models

Summary: The druggable genome encompasses the genes that are known or predicted to interact with drugs. The Drug-Gene Interaction Database (DGIdb) provides an integrated resource for discovering and contextualizing these interactions, supporting a broad range of research and clin...

Paediatric therapeutic development workshop on osteosarcoma

The fourth Paediatric Therapeutic Development Workshop focused on osteosarcoma, the most common primary bone cancer in children and young adults. Current treatment of osteosarcoma comprises surgery and chemotherapy. Outcome has shown very little improvement over the last four dec...

MIR17HG Expression Is Transcriptionally Regulated by PAX3::FOXO1 and MYCN and is Necessary for Oncogenic Activity in Fusion-Positive Rhabdomyosarcoma

Alveolar rhabdomyosarcoma (RMS), an aggressive pediatric soft tissue cancer, is driven by the oncogenic fusion transcription factor PAX3::FOXO1 (P3F) or PAX7::FOXO1. In a subset of fusion-positive (FP)-RMS cases, amplification of the MIR17HG locus leads to overexpression of the m...

Variation in In-Hospital Emergency Transfer Rates Across Pediatric Hospitals

BACKGROUND: Clinical deterioration with late escalation, termed emergency transfer (ET), is a key safety metric. However, national benchmarking is limited by variable definitions. We sought to estimate pooled ET rates and assess variation across pediatric hospitals. METHODS: We p...

The Technology Dependence Index (TDI): Development and Validation in Neonatal and Pediatric Populations

The survival of infants and children with complex medical conditions continues to increase, underscoring the need to quantify technology dependence more effectively. Existing measures emphasize mortality risk and fail to capture the burden of ongoing technological support. To add...

Non-functional capillary modulates the honeymoon period in congenital diaphragmatic hernia

Congenital diaphragmatic hernia (CDH) results in lung hypoplasia and vascular remodeling with persistent pulmonary hypertension with significant mortality and morbidity. While only the analysis of the ‘’functional’’ gas-exchange capillary with diverging findings has been publishe...

Bridging Perspectives: Rebalancing Learner–Educator Dynamics in Modern Medical Education

Rebecca Scherzer, MD, Nancy Liao, MD, Shauna Schord, MD; Bridging Perspectives: Rebalancing Learner–Educator Dynamics in Modern Medical Education, ATS Scho

Management of Infantile Epileptic Spasms Syndrome: A survey of US pediatric hospitals

Abstract Objective To evaluate the management practices of Infantile Epileptic Spasms Syndrome (IESS) across tertiary pediatric hospitals in the United States using a survey‐based approach. Methods A 21‐question survey focused on management setting, work‐up, follow‐up and treatme...

HeartMate 3 in Pediatric and Complex Populations: Physiologic Rationale, Patient Selection, and Management Strategies

The HeartMate 3 (HM3) left ventricular assist device (LVAD), initially designed for adults, is increasingly utilized in children, small adults, and those with congenital heart disease (CHD), necessitating expanded knowledge and collaborative efforts. This review highlights key ph...

A compendium of next-generation patient-derived models for diverse cancers

Abstract The development of new therapeutics and the validation of pathogenetic cancer mechanisms require representative laboratory models 1,2 . However, existing collections represent only a fraction of the diversity observed in human cancer 2–4 . Recent technologies have enable...

Effect of time to palliative epilepsy surgery on outcomes

Abstract Objective Epilepsy duration is a modifiable risk factor in the outcome of definitive epilepsy surgery; however, an analogous effect in palliative procedures has not been shown. We reviewed the Pediatric Epilepsy Surgery Database data for an association between epilepsy d...

Optimizing Neurodevelopment in Infants With Severe Bronchopulmonary Dysplasia

Bronchopulmonary dysplasia (BPD) is associated with multiple morbidities, including neurodevelopmental impairment. This association is multifactorial and further complicated by prolonged hospital stays during a critical period of rapid brain growth. Despite the well-established l...