Pediatrics
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Unorthodox JAK Inhibitor Dosing in Pediatric Moderate-to-Severe Alopecia Areata: A Comparative Evaluation of Tofacitinib and Baricitinib
This study of 100 pediatric patients with moderate-to-severe alopecia areata demonstrates that unorthodox dosing regimens of tofacitinib and baricitinib provide comparable efficacy and high tolerability over 24 weeks.

Plasma GFAP and NfL Levels Predict Acute Brain Injury and Poor Outcomes in Pediatric ECMO
This multi-center study demonstrates that serial monitoring of plasma GFAP and NfL biomarkers can detect acute brain injury in pediatric ECMO patients before imaging diagnosis, significantly correlating with long-term survival and functiona

Plasma GFAP and NfL as Real-Time Predictors of Brain Injury and Long-Term Disability in Pediatric ECMO
A prospective study demonstrates that rising levels of plasma GFAP and NfL precede neuroimaging-confirmed brain injury in children on ECMO. These biomarkers are also strongly associated with mortality and long-term functional impairment, of

Optimizing Pediatric Outcomes: Evidence-Based Advancements in Respiratory Delivery, Neonatal Support, and Precision Therapeutics
This review synthesizes practice-changing pediatric research, highlighting how device selection in acute wheezing, automated oxygen titration in neonates, and novel siRNA therapies for hypercholesterolemia are redefining clinical standards

Fractional Yellow Fever Vaccination Fails Non-Inferiority in Infants: Why Adult Data Cannot Be Extrapolated to Pediatric Populations
A randomized, double-blind trial in Kenya and Uganda reveals that a 500 IU fractional dose of yellow fever vaccine is not non-inferior to the standard dose in infants, suggesting that lower dose requirements in adults are not generalizable

Cyclic Exclusive Enteral Nutrition: A New Paradigm for Drug-Free Remission in Pediatric Crohn’s Disease
The CD-HOPE trial reveals that cyclic exclusive enteral nutrition (C-EEN) is superior to daily partial enteral nutrition (PEN) for maintaining drug-free remission in children with Crohn’s disease, providing a critical alternative to long-te

Twice-Yearly Inclisiran Successfully Lowers LDL-C in Adolescents with Heterozygous Familial Hypercholesterolaemia: Insights from ORION-16
The ORION-16 trial demonstrates that inclisiran, a twice-yearly siRNA therapy, significantly and safely reduces LDL cholesterol in adolescents with HeFH, offering a potent new tool for early cardiovascular risk management in high-risk pedia

CIFFREO Trial Results: Fordadistrogene Movaparvovec Fails Primary Efficacy Endpoint in Duchenne Muscular Dystrophy
The Phase 3 CIFFREO trial for the gene therapy fordadistrogene movaparvovec failed to meet its primary efficacy endpoint in boys with Duchenne muscular dystrophy, showing no significant functional improvement over placebo and leading to the

Giving Kids a Voice: How Patient-Reported Outcome Measures are Transforming Pediatric Specialty Care
Explore how standardized health questionnaires empower children and parents to share their priorities in medical settings, leading to more personalized and effective treatment plans.

AAV9 Gene Therapy Shows Clinical Promise in Stabilizing Type II GM1 Gangliosidosis
A Phase 1-2 trial of AAV9-GLB1 gene therapy in children with Type II GM1 gangliosidosis demonstrates significant biochemical improvements and stabilization of neurodevelopmental decline, marking a pivotal step toward treating this fatal lys

Kangaroo Care Attenuates Pain and Systemic Inflammatory Markers During Retinopathy of Prematurity Screening
A randomized clinical trial demonstrates that Kangaroo care significantly reduces pain-related stress and systemic inflammatory responses in preterm infants undergoing retinopathy screening, suggesting a simple, non-pharmacological interven

Ultrasound Viscoelastic Imaging: A New Noninvasive Frontier in Pediatric Henoch-Schönlein Purpura Nephritis Diagnosis
This study highlights ultrasound viscoelastic imaging as a high-performance, noninvasive tool for diagnosing Henoch-Schönlein purpura nephritis (HSPN) in children, achieving a combined AUC of 0.95 and providing critical insights into renal

Early Selective Ibuprofen for Patent Ductus Arteriosus Fails to Improve Outcomes in Extremely Preterm Infants: Insights from the Baby-OSCAR Trial
The Baby-OSCAR trial demonstrates that early selective treatment of large patent ductus arteriosus with ibuprofen in extremely preterm infants does not significantly reduce death or bronchopulmonary dysplasia, nor does it improve neurodevel

Prophylactic Acetaminophen Fails to Improve Intact Survival in Preterm Infants: Lessons from the TREOCAPA Trial
The TREOCAPA randomized clinical trial demonstrates that while prophylactic intravenous acetaminophen effectively promotes patent ductus arteriosus closure in extremely preterm infants, it does not significantly increase survival without se

Universal School-Based Mindfulness Fails to Outperform Standard Care for Adolescent Mental Health: Insights from the MYRIAD Trial
A large-scale analysis of the MYRIAD trial indicates that universal school-based mindfulness training does not superiorly reduce depression risk in adolescents. While machine learning identified certain potential responders, the clinical be

Beyond the Daily Pill: The Dawn of Long-Acting HIV Treatment for Adolescents
The IMPAACT 2017/MOCHA study reveals that long-acting injectable cabotegravir and rilpivirine administered every eight weeks is safe and highly effective for adolescents with HIV, potentially transforming the landscape of pediatric HIV care

Beyond End-of-Life: The Rising Global Burden of Chronic Suffering in 10.6 Million Children
A landmark longitudinal study reveals that 10.6 million children experience serious health-related suffering annually, with a critical shift toward survivors requiring long-term palliative support rather than end-of-life care, predominantly

Why Environmental Interventions in Childcare May Not Be Enough: Insights from the NAPSACC UK Trial
The NAPSACC UK cluster RCT found that a one-year environmental intervention in early childhood education settings did not significantly improve total energy consumption or physical activity, highlighting the need for broader policy-level ch

Surgical Fixation Offers No Clinical Benefit and Zero Cost-Effectiveness for Pediatric Medial Epicondyle Fractures: Results from the SCIENCE Trial
The SCIENCE multicentre randomized trial demonstrates that surgical fixation for displaced medial epicondyle fractures in children provides no functional advantage over non-surgical care, carries higher complication risks, and is not a cost

Dolutegravir and Metabolic Health in Children: Five-Year Insights from the ODYSSEY Trial
Ancillary analysis of the ODYSSEY trial demonstrates that dolutegravir-based ART is not associated with excessive weight gain or adiposity in children and adolescents compared to standard care, while offering a superior metabolic profile ov
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