Mutations in FLT3 are present in approximately 30% of patients with AML. The addition of midostaurin (MIDO) to intensive chemotherapy (IC) became standard of care following the RATIFY trial, but comprehensive real-world ...

BACKGROUND: The expanding use of targeted therapies in inflammatory bowel disease has made treatment sequencing increasingly relevant, yet evidence on anti-TNF effectiveness after prior biologics with different mechanisms ...

Data on real-world survival and treatment patterns in large, unselected myelofibrosis (MF) cohorts remain limited. We analysed 1649 patients with MF diagnosed from 2010 onwards and included in the Spanish Myelofibrosis ...

Metabolic dysfunction-associated steatotic liver disease (MASLD) is closely linked to systemic metabolic disturbances, yet the early hepatic mechanisms contributing to disease initiation and progression remain incompletely ...

Real-world comparisons of venetoclax (VEN)-based versus hypomethylating agent (HMA) monotherapy in unfit, newly diagnosed AML remain limited by short follow-up, low genomic testing, and inadequate statistical power. We ...

PURPOSE: To evaluate whether early vascular susceptibility, reflected by first-trimester lipoprotein(a), together with longitudinal renal biomarker trajectories across pregnancy, is associated with the subsequent development ...

RATIONALE: In an 8-week, active-controlled, phase 3 study (445-104), elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in participants ? 12 years of age with cystic fibrosis (CF) and ...

BACKGROUND AND AIMS: Congenital long QT syndrome (LQTS) is a heterogeneous disorder in which genotype and QTc duration modulate the risk of major arrhythmic events (MAEs), but contemporary paediatric outcome data remain ...