Pediatrics
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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.
Reduced-Intensity Thiotepa Plus Post-Transplant Cyclophosphamide Delivers Near-Universal Engraftment and Minimal GVHD in Matched Sibling Transplantation for Adolescents and Adults With Sickle Cell Disease
A prospective multicenter study reports excellent survival, no graft failure, and very low GVHD with a thiotepa-based reduced-intensity matched sibling transplant platform for severe sickle cell disease.
No Clear Winner in Severe Congenital Ptosis Repair: AAO Review Finds Overlapping Efficacy but Distinct Surgical Risks in Children
An American Academy of Ophthalmology review found that frontalis flap, frontalis suspension, and maximal levator resection show overlapping success and exposure rates in severe congenital ptosis, with surgical choice often guided more by co
Pediatric Ischemic Stroke in Switzerland Is Still Diagnosed Too Late for Reperfusion in Most Cases
A 24-year Swiss registry study shows modest improvement in pediatric stroke recognition, but most children are still diagnosed beyond thrombolysis and thrombectomy windows.
Combined Nicotinamide Riboside and Home-Based Exercise Improved Peak VO2 Safely in Friedreich’s Ataxia
In a phase 2 factorial trial, 12 weeks of nicotinamide riboside plus individualized exercise improved cardiopulmonary fitness in Friedreich’s ataxia, with mild-to-moderate adverse events and no signal of major safety concerns.
Endocrine Profile in Pediatric Bardet-Biedl Syndrome: Insights from a Large Multidisciplinary Cohort
A retrospective study of 135 pediatric BBS patients reveals high prevalence of obesity (77.6%) and short stature (21.1%), with low rates of hypogonadism, hypothyroidism, and glucose metabolism disorders compared to adult populations.
Allogeneic Stem Cell Transplantation Improves Survival in High-Risk t(6;9) AML, Especially in Younger Patients and Those Achieving CR1
A large EBMT study shows allo-HSCT provides favorable 2-year survival (65.7%) in high-risk t(6;9) AML, with superior outcomes in CR1 and pediatric/AYA patients. FLT3-ITD positivity triples relapse risk but doesn’t impact overall survival.
DSDO Lenses Show Superior Axial Length Control in Pediatric Myopia Management: A Real-World Multicenter Study
DSDO lenses demonstrated significant efficacy in slowing axial elongation in myopic and non-myopic children, with pronounced effects in younger age groups and low myopia subgroups during the first 6 months of intervention.
Pediatric Hypertrophic Cardiomyopathy: The High-Risk Profile of Massive Left Ventricular Hypertrophy
A multiregistry analysis reveals that massive LVH in pediatric HCM is linked to early diagnosis, sarcomeric mutations, and heightened risks of arrhythmias, heart failure, and mortality. Nearly 25% of patients show significant LVH regression
Genomic Fusion Breakpoints: A Breakthrough in Pediatric ALL MRD Monitoring
A novel genomic fusion breakpoint (GFB)-based MRD monitoring approach demonstrates superior sensitivity and specificity in pediatric ALL, addressing limitations of current IG/TCR-based methods, particularly in ETV6::RUNX1- and MEF2D-rearran
Whole Genome Sequencing Outperforms Gene Panels in Diagnosing Congenital Cataracts
Whole genome sequencing (WGS) increases diagnostic yield by 10% compared to gene panels in congenital cataracts, aiding in prognosis, reproductive counseling, and management.
Fetal Reduction in Triplet Pregnancies Significantly Reduces Neurodevelopmental Risks, Study Finds
A nationwide cohort study reveals that fetal reduction from triplets to twins lowers the long-term risk of neurodevelopmental disorders in surviving children compared to non-reduced pregnancies.
Observation Reduces CT Use in Pediatric Blunt Abdominal Trauma Without Missing Critical Injuries
A multicenter study shows that observation with deferred CT decision-making significantly reduces CT use in children with blunt abdominal trauma, particularly in cases with intermediate clinician suspicion, without increasing missed injurie
New Statistical Model Predicts Chronic ITP in Children at Diagnosis
A novel statistical model predicts chronic immune thrombocytopenia (ITP) in children at diagnosis, using age, sex, immunoglobulin levels, and lab values, offering early clinical guidance.
Open Globe Repair Carries the Highest Endophthalmitis Risk, and Pediatric Cataract Surgery Outpaces Adult Surgery in IRIS Registry Data
An IRIS Registry analysis of 17.5 million procedures found endophthalmitis was rare overall but highest after open globe repair, with pediatric cataract surgery carrying more risk than adult surgery.
Maternal Diabetes and Early Neurodevelopment: Differential Risks Emerge Across Diabetes Types and Infant Sex
A nationwide cohort study of 466,462 infants reveals that maternal pregestational diabetes significantly increases the risk of delayed developmental milestones, with type 1 diabetes conferring the highest risk and female offspring showing p
Machine Learning Falls Short in Personalizing School-Based Mindfulness for Adolescent Depression Prevention: MYRIAD Trial Insights
Secondary analysis of the MYRIAD trial reveals that while machine learning models can identify adolescents who may benefit from school-based mindfulness training, the clinical relevance of such predictions remains minimal. Both causal fores
TP53 Alterations Emerge as Critical Adverse Prognostic Factor in Pediatric and Young Adult Patients Treated with CD19-CAR T-Cell Therapy for Relapsed/Refractory B-ALL
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
Real-World Evidence Confirms Burosumab Effectiveness Across All Ages in X-linked Hypophosphatemia
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.
FT3/FT4 Ratio Emerges as a Practical Predictor of Relapse After Antithyroid Drug Withdrawal in Children
In pediatric autoimmune hyperthyroidism, a higher FT3/FT4 ratio predicted relapse after antithyroid drug withdrawal, especially when orbitopathy was present.
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