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A single-center retrospective study of 69 pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia reveals that TP53 alterations significantly compromise outcomes following tisagenlecleucel therapy. Pa
A 3-year prospective study demonstrates sustained biochemical and clinical improvements with burosumab in XLH patients across pediatric and adult age groups, including those typically excluded from clinical trials.
In pediatric autoimmune hyperthyroidism, a higher FT3/FT4 ratio predicted relapse after antithyroid drug withdrawal, especially when orbitopathy was present.
The PHC HOD08 study demonstrates that an 8-week dose-dense modified Stanford V regimen achieves high complete response rates, allowing over 75% of low-risk pediatric Hodgkin lymphoma patients to avoid radiotherapy while maintaining an excep
This report examines the role of Intestinal Ultrasound (IUS) in pediatric Acute Severe Ulcerative Colitis (ASUC). Results from 60 patients show that IUS metrics, specifically colonic wall thickness and the Milan Ultrasound Score, can accura
A landmark study using lesion network mapping reveals that cerebral palsy after neonatal stroke is driven by disruptions in extensive functional networks, including the thalamus and cerebellum, rather than just localized motor cortex damage
This article examines the landmark findings from the National Pediatric Prehospital Readiness Project, identifying critical gaps in emergency care for children and highlighting the transformative role of Pediatric Emergency Care Coordinator
The first national assessment of US EMS agencies reveals a critical need for improved pediatric readiness. While equipment scores are high, significant gaps exist in quality improvement and family-centered care, with Pediatric Emergency Car
Recent research highlights a standardized multidisciplinary protocol for managing unilateral vocal fold immobility in infants after cardiothoracic surgery, showing that injection laryngoplasty significantly improves oral feeding advancement
A prospective study of over 700,000 newborns demonstrates that LC-MS/MS significantly improves the positive predictive value (PPV) of CAH screening compared to traditional RIA, identifying specific steroid ratios that offer 100% PPV and red
This review analyzes the significant long-term thyroid toxicity burden in high-risk neuroblastoma survivors, identifying key risk factors such as molecular radiotherapy, tandem myeloablative therapy, and immunotherapy.
A 16-year follow-up of the SVR trial reveals that while shunt types show similar long-term outcomes overall, pre-existing tricuspid regurgitation significantly impacts survival with RVPAS, amid a staggering 87% rate of major morbidity.
This meta-analysis reveals that long-acting growth hormone (LAGH) is associated with a significant increase in BMI SD scores during the first year of treatment and upon switching from daily GH, suggesting a distinct metabolic profile compar
A breakthrough study reveals that specific FBN1 genotypes, particularly in-frame variants in the DNCD region, significantly elevate the risk of early mitral valve surgery in Marfan syndrome patients, paving the way for genotype-guided clini
The ORION-16 trial demonstrates that inclisiran, a twice-yearly siRNA therapy, significantly and safely reduces LDL cholesterol in adolescents with heterozygous familial hypercholesterolaemia, offering a promising solution for long-term adh
A large-scale analysis of 6,026 pediatric heart transplant candidates reveals that status exceptions often prioritize patients with lower mortality risk than those meeting standard criteria, highlighting a significant misalignment in the U.
Researchers have developed a trans-ancestry polygenic risk score (TA-PS) that significantly improves the accuracy of type 1 diabetes prediction across diverse ethnic groups, addressing the long-standing European bias in genetic risk assessm
The Phase 3 CIFFREO trial found that the gene therapy fordadistrogene movaparvovec did not significantly improve motor function in boys with Duchenne muscular dystrophy compared to placebo, leading to the discontinuation of its clinical dev
A UK multicentre study demonstrates that 24-weekly Decapeptyl SR is as effective and well-tolerated as the 12-weekly regimen for central precocious puberty, offering improved patient satisfaction and reduced clinical burden.
The ENDIA cohort study reveals that persistent single ZnT8 autoantibodies detected only via ELISA after age 4 do not significantly increase Type 1 Diabetes risk, whereas earlier detection across multiple assay formats signals higher progres
