Pediatrics
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Efficacy and Safety of Ecopipam for Tourette Syndrome: Phase 3 Randomized Clinical Trial
This phase 3 trial found that ecopipam reduced tic relapse and maintained symptom improvement in children and adolescents with Tourette syndrome, with mainly central nervous system side effects and no major weight or metabolic concerns.
Disrupted MAML1 Phase Separation Emerges as a New Mechanism Linking Notch Failure to Congenital Heart Disease
A Circulation study identifies MAML1 as a candidate congenital heart disease gene and shows that defective phase separation in endocardial cells suppresses Notch signaling, impairing endocardial-to-mesenchymal transition and causing septal
Pilot Study: Busulfan-Based Haploidentical Stem Cell Transplantation Plus GD2 Antibody Therapy for High-Risk Neuroblastoma
A small pilot study found that busulfan-based haploidentical stem cell transplantation followed by dinutuximab beta was feasible and may improve long-term remission in very high-risk neuroblastoma.
Dietary Patterns and Asthma Endotypes in Puerto Rican Youth
In Puerto Rican youth, an unhealthy diet was linked to higher odds of T17-high asthma, suggesting diet may influence asthma through immune pathways beyond allergy-related inflammation.
Spontaneous Preterm Birth as a Sentinel for Cardiovascular Mortality: Insights from a National Registry Study
A large-scale Dutch cohort study reveals that women with a history of spontaneous preterm birth face a significantly elevated risk of cardiovascular mortality, with the highest risk observed in those delivering before 32 weeks of gestation.
Extracorporeal CPR May Improve Survival After Pediatric Out-of-Hospital Cardiac Arrest, but Evidence Remains Early
A Japanese multicenter registry study suggests ECPR may improve 1-month survival and neurologic outcomes in selected children with out-of-hospital cardiac arrest, although estimates were imprecise and residual confounding remains a major li
Extracorporeal Cardiopulmonary Resuscitation for Pediatric Out-of-Hospital Cardiac Arrest
In selected children with out-of-hospital cardiac arrest, extracorporeal CPR was associated with higher one-month survival and better neurologic outcomes than continued conventional CPR, though the study was observational and estimates were
In Transition-Age Childhood-Onset GH Deficiency, Glucagon Test Results Reflect Pituitary Damage More Than Body Mass Index
In 180 adolescents and young adults with childhood-onset GH deficiency, glucagon-stimulated GH secretion was driven mainly by hypothalamic-pituitary disease severity, while BMI had little independent effect after accounting for etiology.
Frenotomy Use in Danish Infants More Than Doubled Over a Decade, With Marked Geographic Variation and Limited Signals of Serious Harm
A nationwide Danish cohort study found rapid growth in infant frenotomy, wide municipal variation, and rare severe complications, raising questions about clinical appropriateness in the setting of limited evidence for benefit.
Nebulized Ciprofloxacin-Dexamethasone Appears Well Tolerated After Pediatric Airway Surgery in a Large Retrospective Cohort
A single-center retrospective study of 399 pediatric airway surgery patients found no significant adverse events clearly linked to postoperative nebulized ciprofloxacin-dexamethasone, supporting short-term safety while leaving efficacy and
A New Steroid Option for Duchenne Muscular Dystrophy? What the Latest Vamorolone Study Means for Families
A new study suggests vamorolone may preserve motor benefits seen with standard steroids in Duchenne muscular dystrophy while better protecting growth, though weight gain remains a concern.
Higher-Fidelity Enhanced Recovery Shortened Stay and Reduced Complications in Children Undergoing GI Surgery, Even Though the Overall Trial Was Neutral
In a stepped-wedge trial across 18 US centers, a pediatric GI surgery enhanced recovery protocol did not improve length of stay overall, but patients receiving at least 13 protocol elements had shorter stays, fewer complications, faster die
Children Born SGA Receiving Growth Hormone Have Similarly Impaired Glucose-Insulin Metabolism as Children With Obesity
Children born SGA receiving growth hormone showed insulin resistance and prediabetes rates similar to obesity, suggesting the need for careful glucose monitoring during and after treatment.
Identifying High-Risk Children Safe for Same-Day Discharge After Tonsillectomy
Children with obesity and severe OSA usually stay overnight after tonsillectomy, but this study found most had no major complications. Oxygen nadir and AHI may help identify very low-risk patients who could be considered for same-day discha
Poverty Exposure in Standard-Risk Pediatric B-ALL Is Linked to Steroid-Resistance Programs and an Immunologically Suppressed Leukemia Microenvironment
Single-cell RNA sequencing suggests that childhood poverty exposure is associated with steroid-resistance signatures in leukemic blasts and adverse immune remodeling at diagnosis in standard-risk B-ALL.
Persistent Challenges in Managing Monocular Deprivation Amblyopia: A Three-Decade Trend Analysis of Infant Unilateral Cataract Outcomes
This review evaluates 30 years of clinical outcomes for monocular congenital cataracts, revealing stagnant visual acuity results, high surgical burdens, and significant rates of secondary glaucoma and nystagmus despite surgical advances.
Web-Based Amblyopia Decision Support Tool
The Amblyopia Navigator offers evidence-based guidance for eye care professionals to improve early detection and treatment of childhood amblyopia, addressing specialist shortages through accessible digital tools.
Glucocorticoid Reduction After Starting Crinecerfont in Pediatric Patients With Classic CAH: Practical Perspectives
New expert recommendations guide glucocorticoid dose reduction in pediatric classic CAH patients using crinecerfont, prioritizing normal growth while minimizing long-term steroid complications.
Pediatric Type 1 Diabetes Care Still Misses LDL Targets Despite Clear Indications for Lipid-Lowering Therapy
Registry data from 55,028 youths with type 1 diabetes show common LDL hypercholesterolemia, infrequent lipid-lowering therapy, and poor attainment of recommended LDL targets.
A New Pediatric VAD Driver Shows High Reliability and Preserves Mobility Without Major Device Malfunctions
In a prospective multicenter evaluation, the EXCOR Active Driver supported pediatric VAD care with no major device malfunctions and excellent short-term survival, while addressing a major unmet need in mobility and day-to-day function.
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