Validity of assessing inhibitor development in haemophilia PUPs using registry data: the EUHASS project.

摘要: Summary.  Inhibitory antibodies to exogenous FVIII/FIX are a major complication of haemophilia treatment. Up 30% previously untreated patients (PUPs) with severe A develop inhibitors, most likely during the initial 50 exposure days concentrate. In addition classical cohort studies, European monitoring system (EUHASS) has been set up evaluate inhibitor development in PUPs. The present study addresses reliability estimating cumulative incidence this registry. Data from retrospective CANAL study, including 288 PUPs and complete treatment records until 50th FVIII, were used simulate consequences several cross-sectional sampling techniques on estimated inhibitors. Both mathematical calculus computer modelling applied effects sample size introduction new product. For existing concentrates, both longitudinal methods EUHASS method yielded similar estimates cases over 5-year time period: 27.9% (95% CI: 21–36) vs. 29.4% (22–38). newly introduced concentrate, reliable estimate could only be made after 3–4 years, even large datasets. results expected valid. After concentrate can reliably determined an observation period years.