Pediatrics news.

The FDA approved Zanvastro (zilganersen) for pediatric and adult patients with Alexander disease. Evidence included a randomized controlled study of 49 patients aged 2 years and older and an open-label substudy of four younger children; reported motor benefits supported approval.

The FDA has issued immediately effective guidance to address a potential supply gap in neonatal starter parenteral nutrition. The temporary policies follow concerns from children’s hospitals about discontinued products from two outsourcing facilities that are permanently shutting down.

A Danish registry-based matched case-control study compared 71,317 children and adolescents diagnosed with ADHD or ASD with 713,170 controls. Associations between prediagnostic characteristics and diagnosis weakened from 2012 to 2022, particularly for socioeconomic and perinatal factors and for ADHD.

A Dutch prospective registry study followed 309 children with moderate to severe atopic dermatitis receiving dupilumab for up to three years. Disease activity remained low, and estimated three-year treatment persistence was 80.1%; ocular surface disease was reported in 36.6% of patients.

The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), the first approved treatment for pediatric Sanfilippo syndrome type A. An open-label, single-arm study reported maintained or improved cognition compared with historical controls; the announcement provided no sample size or numerical effect estimates.

A technical report accompanying the American Academy of Pediatrics’ 2026 UTI guideline explains a revised diagnostic definition for children aged eight days to five years. The panel retained pyuria as a diagnostic criterion and revised inflammation and culture criteria according to age and testing circumstances.

A pragmatic randomized trial involving 1,900 critically ill children found that a permissive mean arterial pressure target reduced vasoactive drug exposure but did not significantly improve the composite of mortality and invasive ventilation duration at 30 days. Kidney replacement therapy use and adverse events did not differ from usual care.

The FDA approved Emcitate (tiratricol) as the first FDA-approved therapy for peripheral thyrotoxicosis in MCT8 deficiency. Evidence came from a randomized, placebo-controlled trial and a longer-term open-label study involving infants through adults; the announcement did not report sample sizes or numerical treatment effects.

An observational study of 34 patients with chronic recurrent multifocal osteomyelitis and 20 controls found neutrophil activation and reduced PSTPIP2 protein levels. Activation correlated with MRI lesion burden; patients receiving anti-TNF therapy had normalized activation and PSTPIP2 levels, although treatment causality cannot be established.

An observational analysis of 422 youth with type 1 diabetes evaluated insulin dose–adjusted HbA1c against stimulated C-peptide within 12 months of diagnosis. The conventional cutoff had 61% sensitivity overall and 45% in participants with overweight or obesity, raising concerns about its use to identify partial remission.

A registry-based longitudinal cohort study included 135 individuals with epidermal differentiation disorders and 26 unaffected siblings. Growth deficits were most evident during the first two years, with persistent low height and weight percentiles at ages 25–48 months among children with SPINK5 variants.

An international modified Delphi consensus provides recommendations for off-label low-dose oral minoxidil in children younger than 12 years. Experts agreed on 84 items covering patient selection, dosing, contraindications, and monitoring; the recommendations reflect expert agreement rather than trial evidence.

All newborns in Sweden will now be screened for metachromatic leukodystrophy following a recommendation from Socialstyrelsen. The programme aims to identify affected children before symptoms and irreversible neurological damage develop, allowing timely consideration of stem cell-based gene therapy.

A pragmatic cluster-randomized trial involving 43 US general hospitals and 11,760 pediatric admissions found that respiratory care pathways improved adherence to evidence-based practices. The intervention also reduced the odds of intensive care transfer, without significant effects on length of stay or 30-day readmission or emergency revisit.

A longitudinal analysis of 895 doxorubicin-treated patients found that dexrazoxane exposure was associated with better preserved left ventricular systolic function. Reduced-function event rates were lower among high-risk survivors, but implications for cardiomyopathy screening require further evaluation.