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Hematology-Oncologyhematopoietic stem cell transplantationposttransplant cyclophosphamide

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

By MedXY|May 4, 2026

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.

newsexposure keratopathyfrontalis suspension

No Clear Winner in Severe Congenital Ptosis Repair: AAO Review Finds Overlapping Efficacy but Distinct Surgical Risks in Children

By MedXY|May 4, 2026

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

Neurologydiagnostic delaypediatric stroke

Pediatric Ischemic Stroke in Switzerland Is Still Diagnosed Too Late for Reperfusion in Most Cases

By MedXY|May 3, 2026

A 24-year Swiss registry study shows modest improvement in pediatric stroke recognition, but most children are still diagnosed beyond thrombolysis and thrombectomy windows.

Neurologyexercise therapyFriedreich's ataxia

Combined Nicotinamide Riboside and Home-Based Exercise Improved Peak VO2 Safely in Friedreich’s Ataxia

By MedXY|May 3, 2026

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.

Diabetes & EndocrinologyBardet-Biedl syndromeciliopathy

Endocrine Profile in Pediatric Bardet-Biedl Syndrome: Insights from a Large Multidisciplinary Cohort

By MedXY|Apr 28, 2026

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.

Hematology-OncologyAMLFLT3-ITD

Allogeneic Stem Cell Transplantation Improves Survival in High-Risk t(6;9) AML, Especially in Younger Patients and Those Achieving CR1

By MedXY|Apr 27, 2026

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 survi

newsaxial length controlDIMS lenses

DSDO Lenses Show Superior Axial Length Control in Pediatric Myopia Management: A Real-World Multicenter Study

By MedXY|Apr 27, 2026

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.

CardiologyHypertrophic Cardiomyopathyleft ventricular hypertrophy

Pediatric Hypertrophic Cardiomyopathy: The High-Risk Profile of Massive Left Ventricular Hypertrophy

By MedXY|Apr 24, 2026

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

Hematology-Oncologygenomic fusion breakpointsmeasurable residual disease

Genomic Fusion Breakpoints: A Breakthrough in Pediatric ALL MRD Monitoring

By MedXY|Apr 24, 2026

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

newsgenotype-phenotype correlations先天性白内障

Whole Genome Sequencing Outperforms Gene Panels in Diagnosing Congenital Cataracts

By MedXY|Apr 24, 2026

Whole genome sequencing (WGS) increases diagnostic yield by 10% compared to gene panels in congenital cataracts, aiding in prognosis, reproductive counseling, and management.

newscohort studyfetal reduction

Fetal Reduction in Triplet Pregnancies Significantly Reduces Neurodevelopmental Risks, Study Finds

By MedXY|Apr 23, 2026

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.

Emergency MedicineComputed tomographyemergency medicine

Observation Reduces CT Use in Pediatric Blunt Abdominal Trauma Without Missing Critical Injuries

By MedXY|Apr 21, 2026

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

Hematology-Oncologyimmune thrombocytopeniapediatric hematology

New Statistical Model Predicts Chronic ITP in Children at Diagnosis

By MedXY|Apr 24, 2026

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.

newsendophthalmitisIRIS Registry

Open Globe Repair Carries the Highest Endophthalmitis Risk, and Pediatric Cataract Surgery Outpaces Adult Surgery in IRIS Registry Data

By MedXY|Apr 21, 2026

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.

Diabetes & Endocrinologydevelopmental milestonesGestational Diabetes

Maternal Diabetes and Early Neurodevelopment: Differential Risks Emerge Across Diabetes Types and Infant Sex

By MedXY|Apr 17, 2026

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

newsAdolescent Depressionmachine learning

Machine Learning Falls Short in Personalizing School-Based Mindfulness for Adolescent Depression Prevention: MYRIAD Trial Insights

By MedXY|Apr 13, 2026

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

$TP53 Alterations Emerge as Critical Adverse Prognostic Factor in Pediatric and Young Adult Patients Treated with CD19-CAR T-Cell Therapy for Relapsed/Refractory B-ALL$

Hematology-OncologyB細胞急性リンパ性白血病CAR T-cell therapy

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

By MedXY|Apr 12, 2026

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

Diabetes & EndocrinologyBurosumabFGF23

Real-World Evidence Confirms Burosumab Effectiveness Across All Ages in X-linked Hypophosphatemia

By MedXY|Apr 6, 2026

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.

Diabetes & EndocrinologyFT3/FT4 ratiopediatric Graves disease

FT3/FT4 Ratio Emerges as a Practical Predictor of Relapse After Antithyroid Drug Withdrawal in Children

By MedXY|Apr 1, 2026

In pediatric autoimmune hyperthyroidism, a higher FT3/FT4 ratio predicted relapse after antithyroid drug withdrawal, especially when orbitopathy was present.

Hematology-OncologyDose-Dense ChemotherapyPediatric Hodgkin Lymphoma

Dose-Dense Stanford V Strategy Safely Spares Radiation in Most Pediatric Patients with Low-Risk Hodgkin Lymphoma

By MedXY|Mar 24, 2026

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

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