Introduction: Haemophilia management and patients' quality of life significantly improved.
However, data on current patients', caregivers' and clinicians' satisfaction and limitations of …

Background Knowledge about the risk for bleeding in patients with hemophilia (PWH) would
be relevant for patients, stakeholders, and policy makers. Objectives To perform a systematic …

Background Previous studies have reported marked interindividual variation in factor VIII
(FVIII) clearance in patients with hemophilia (PWH) and proposed a number of factors that …

Background Little is known on how sports participation affects bleeding risk in hemophilia.
This study aimed to examine associations between sports participation, factor VIII (FVIII) …

Background Treatment choices for individual patients with an inborn bleeding disorder are
increasingly challenging due to increasing options and rising costs for society. We have …

Introduction Considering the advances in haemophilia management and treatment
observed in the last decades, a new set of value‐based outcome indicators is needed to …

Clinical guidelines and expert groups recommend the use of pharmacokinetic (PK)-guided
dosing of factor replacement therapy for the treatment of bleeding disorders, especially for …

Background Efanesoctocog alfa is a new class of factor (F) VIII replacement therapy
designed to provide high sustained factor levels for longer by overcoming the von …

Background Physical activity is a crucial part of an active lifestyle for haemophiliac children.
However, the fear of bleeds has been identified as barriers to participating physical activity …

Background Pharmacokinetic (PK)-guided dosing is used to individualize factor (F) VIII and
FIX replacement therapy. Objectives This study investigates the reliability and feasibility of …