Solid dosage forms such as fine granules, mini-tablets, and micropellets are gaining popularity worldwide because of their longer shelf lives, lower need for preservatives, and precise dosing compared to liquids. However, international pediatric formulations are often liquids or powders, and it is unclear whether these offer advantages over single-dose packaging, which is used in Japan, for caregiver management, preparation, administration, and cleanup. The acceptability of solid forms that require extra preparation, such as decapsulation, weighing dissolved powders, or taking partial doses, remains uncertain. This study evaluated the acceptability and usability of new dosage forms that involve caregiver preparation. From April 2024 to November 2025, a survey was conducted among parents and caregivers of pediatric patients aged 0–18 years visiting the National Center for Child Health and Development. The survey focused on formulations involving opening capsules, preparing suspensions, and administering partial doses. Four model formulations were selected and evaluated: tacrolimus capsules (A), mycophenolic acid powder (B), valganciclovir dry syrup (C), and desmopressin oral disintegrating tablets (D). Respondents rated management, preparation, administration, and cleanup on an 11-point scale (− 50 to + 50) compared to conventional powders. Descriptive statistics and 95
The European LeukemiaNet has periodically issued guidelines for the diagnosis and management of acute myeloid leukemia (AML) in adults. These consensus recommendations, most recently updated in 2022, incorporate recent advances in genomic testing, disease detection methods, target identification, and response assessment. Whilst similarities exist between AML in children and adults, pediatric AML is frequently characterized by unique cytogenetic and molecular features, which require distinct genetic and immunophenotypic diagnostics, therapeutic approaches, response assessment criteria, and supportive care strategies. To address these specific needs, an international panel of pediatric hematologist-oncologists, biologists, geneticists, and laboratory medicine scientists convened to develop recommendations for the diagnosis and management of AML in children, adolescents, and young adults (hereafter termed pediatric AML) that are discussed in this special report.
To examine the surgical outcomes of surgical aortic valve replacement in the transcatheter aortic valve replacement era and propose a novel patient-specific prognostic model. We randomly divided 772 patients with aortic stenosis who underwent surgical aortic valve replacement in 2016–2021 into two cohorts (derivation, 515; validation, 257). In the derivation cohort, no data were missing for any patients for the candidate predictors including age, sex, body mass index, left ventricular ejection fraction, levels of albumin, hemoglobin, and serum creatinine, presence of chronic atrial fibrillation, and end-stage renal disease requiring hemodialysis. We developed possible scoring models using Cox proportional hazards regression with overall survival as the endpoint and calculated the cross-validated 5-year C-statistics to assess accuracy. The mean patient age was 74.2 years, and 46.9
Very-early-onset inflammatory bowel disease (VEO-IBD), representing cases diagnosed before age 6 years, is increasing in prevalence. Although VEO-IBD often presents as severe, treatment-resistant disease requiring biologic agents, studies showing the effectiveness of biologics, such as ustekinumab (UST) and vedolizumab (VDZ), remain limited. We retrospectively analyzed patients with VEO-IBD treated for at least a year from 13 institutions in Japan, evaluating clinical course including effectiveness of biologics, such as infliximab (IFX), adalimumab (ADL), UST, and VDZ. Patients with monogenic IBD were excluded. Steroid-free clinical remission (SFCR) and treatment persistence were assessed separately for first-line and for second-line or subsequent biologic therapies. We studied 101 VEO-IBD patients (56
