Meyer Children's Hospital

Early Introduction and Prolonged Full‐Dose Anakinra Reduce Recurrence Risk After Discontinuation in Recurrent Pericarditis: An International Multicenter Cohort

Background Anakinra is effective in recurrent pericarditis, yet optimal treatment duration and strategies for safe drug discontinuation remain uncertain. Identifying predictors of sustained remission after discontinuation is important for clinical management. Methods We retrospec...

Emerging therapeutic strategies and clinical trials in severe asthma management: current evidence and future directions

Introduction Biologic therapies have transformed the management of severe asthma, particularly in type 2-high disease, but important unmet needs remain. Incomplete responses, uncertainty in treatment positioning, and the lack of effective options for non-type 2 or mixed inflammat...

Training the Next Generation of Future Surgical Residents: A Scoping Review and Proposal for an Undergraduate Curriculum From the Italian Polispecialistic Society of Young Surgeons

AIM: The transition from medical school to surgical residency is marked by a persistent gap between theoretical instruction and clinical readiness. In Italy, variability in training programs across centers creates persistent gaps in residents’ medical education. Scientific societ...

Vitamin D status and adiposity in children with low-energy fractures: a prospective case–control study

Fractures in children are influenced by both skeletal biology and mechanical factors. While alterations in vitamin D and other metabolic markers have been proposed as contributors to fracture risk, their role in predicting fractures in otherwise healthy pediatric populations rema...

An international multicenter analysis of the outcome of pediatric first variceal hemorrhage

Background & Aims: Management of portal hypertension in children including primary prophylaxis of variceal bleeding is controversial due to limited understanding of mortality risk after first variceal hemorrhage (1VH). This international multicenter study was designed to advance...

Persistent Post-Burn Pruritus Following Antihistamine Initiation: A Retrospective Longitudinal Study at a Single Italian Pediatric Burn Center

Background/Objectives: An operationally defined unchanged itch-score sequence may indicate persistent symptoms rather than symptom resolution. We described pruritus following antihistamine initiation in children with burns and examined how its apparent stabilization depended on t...

Intraoperative neurophysiology monitoring in pediatric epilepsy surgery: a case series from a tertiary Italian center

Objectives To characterize intraoperative neurophysiological monitoring in pediatric epilepsy surgery in a single tertiary center. Methods We reviewed intraoperative neurophysiological monitoring procedures during pediatric epilepsy surgeries at Meyer Children’s Hospital, Florenc...

Postpandemic Trends in Pediatric Respiratory Virus Hospitalizations: A Multicenter Study, 2019–2025

ABSTRACT Background The COVID‐19 pandemic profoundly altered the circulation of respiratory viruses in children, changing the epidemiology of acute lower respiratory tract infections. In the postpandemic period, respiratory pathogens showed marked temporal variability. The 2024–2...

Lithium chloride in vitro treatment shows potential to rescue the neuronal phenotype caused by WDFY3 haploinsufficiency

We provide a comprehensive phenotypic characterization of loss-of-function (LoF) variants in WDFY3 based on the largest cohort reported to date (n = 32). Our findings define a monogenic disorder marked by neuropsychiatric features (including autism and ADHD), mild to moderate neu...

Standards for practical management of drug hypersensitivity reactions to intravenous drugs and proactive early allergy involvement before referral: A WAO Statement

Hypersensitivity reactions to intravenous therapies—including antibiotics, chemotherapeutics, biologics, anesthesia agents, and contrast media—pose significant challenges in acute care settings. These reactions often occur outside the purview of allergy specialists, leading to de...

Epidemiology, impact on oncologic treatments, and management of community-acquired respiratory virus (CARV) infections in pediatric onco-hematology patients: a survey among Italian pediatric oncology hematology centres (AIEOP)

Abstract A survey was conducted across Italian Pediatric Onco‑Hematology centres to assess epidemiology of community‑acquired respiratory virus (CARV) infections, chemotherapy management, Oseltamivir use and vaccination policies. Most reported infections were mild and managed out...

Treatment and Outcomes of Kawasaki Disease Over 40 Years

BACKGROUND Kawasaki disease (KD) is the leading cause of acquired heart disease in children in developed countries. Long-term prognosis depends on coronary sequelae, yet Western longitudinal data integrating acute-phase features, treatment, and long-term coronary outcomes remain...

Oropharyngeal Kingella kingae Detection and Spondylodiscitis in Early Childhood—A Causal or Casual Association?

BACKGROUND: Pediatric spondylodiscitis is a rare condition, primarily affecting children aged 6-48 months. Kingella kingae is the leading pathogen in this age group, often presenting with mild or atypical features, which delay diagnosis. METHODS: We retrospectively reviewed pedia...

Defining fever-related discomfort in children: an international Delphi consensus

Fever is one of the most common symptoms in childhood and a frequent reason for medical consultation. Current guidelines recommend treating fever only when discomfort is present. However, the concept of fever-related discomfort remains poorly defined and inconsistently applied in...

Aficamten for Symptomatic Nonobstructive Hypertrophic Cardiomyopathy

BACKGROUND: Nonobstructive hypertrophic cardiomyopathy (HCM) is a common condition that is associated with substantial morbidity and no proven medical therapy. Whether treatment with aficamten, a cardiac myosin inhibitor, can benefit patients with this condition is unknown. METHO...

Global Efficacy of Aficamten in Nonobstructive Hypertrophic Cardiomyopathy: Results From ACACIA-HCM

BACKGROUND: Nonobstructive hypertrophic cardiomyopathy (nHCM) is associated with significant morbidity with no approved treatment. Aficamten is a cardiac myosin inhibitor targeting excess contractility and diastolic impairment, the predominant mechanism responsible for adverse ou...

A Novel Constitutional TUBB Variant Associated With Familial Malformations of Cortical Development

ABSTRACT Most pathogenic tubulin variants arise de novo in sporadic patients, causing severe brain malformations and significant neurodevelopmental impairment. The resulting reproductive disadvantage typically prevents these mutations from being transmitted to offspring. While a...

Correlation of polygenic risk score and clinical phenotype in patients with genetic generalized epilepsy

Abstract Objective The polygenic risk score (PRS) for individuals with genetic generalized epilepsy (GGE) quantifies the common risk variants in genes identified in genome‐wide association studies. We hypothesized that the phenotype of GGE patients differs based on their GGE PRS....

Efficacy and safety of fenfluramine in Dravet syndrome: The impact of patient clinical characteristics

Abstract Objective To assess the efficacy and safety of fenfluramine in patients with Dravet syndrome (DS) stratified by age, number of previously attempted antiseizure medications (ASMs), and SCN1A pathogenic variant status. Methods In this post hoc analysis, data from three ran...

Cross-cultural adaptation and validation of the Localised Scleroderma Quality of Life Instrument (LoSQI): an international study

Juvenile localised scleroderma (jLS) is a rare disease which impacts negatively on health-related quality of life (HRQoL). We aimed to undertake the cross-cultural adaptation and validation of a novel disease-specific HRQoL measure, the Localised Scleroderma Quality of Life Instr...