Ten years of FVIII prophylaxis: a snapshot analysis of a prospective, non-interventional study to evaluate real-life Haemophilia A treatment – GENA-100

Résumé fourni par la source

Introduction The prospective, observational study GENA-100 is designed to collect information on prophylactic treatment of Haemophilia A (HA) patients of all severities and ages treated with simoctocog alfa and 2 plasma-derived von Willebrand factor containing factor VIII (FVIII) concentrates from one company. The primary objective is to assess the influence of weekly FVIII dose on the annualized bleeding rate (ABR). Other objectives include the description of standard and pharmacokinetic (PK)-guided prophylactic schedules, joint score, efficacy and safety outcomes. Considering the fundamental expansion of treatment options for HA patients, the study was amended after 10 years to open inclusion for patients receiving on-demand therapy, with or without non-factor treatment. The new study outline will be reported elsewhere. Method Patients with Haemophilia A of all ages receiving prophylaxis and having a good compliance were eligible for enrolment upon providing informed consent.Detailed documentation of FVIII administrations, bleeding episodes, and surgical procedures is ensured throughout the entire follow-up period, which – unlike in clinical trials – is not individually restricted, thereby allowing for more comprehensive and longitudinal data collection. Optional study elements comprise assessment of joint scores (HJHS) and a pharmacokinetic (PK)-assessment including dosing simulation for potential adaptation of therapy schedules with a population-based PK using WAPPS-Hemo. Results As of October 2025, 96 patients were included with ages at study entry ranging between 1 and 91 years. In total, 119 million units of FVIII were administered on 42,321 exposure days (43,291 injections) to 88 patients being treated with one of the FVIII concentrates (see [ Fig. 1 ]). 64% of the patients were children or adolescents at time of inclusion (see [ Fig. 2 ]). In total, 790 bleeding episodes were documented of which 694 were treated with FVIII outside the prophylactic schedule. Bleeding events were classified as mild in 69.1% of cases, moderate in 27.0%, severe in 3.5%, and of unknown severity in 0.4%. 98.3% of evaluated episodes were rated with an “excellent/good“ efficacy. Mean ABR was 2.7 for treated episodes. Two thirds of bleeding episodes had a traumatic cause, reflecting the high proportion of children in the study. No inhibitor formation or any other adverse drug reactions were observed despite the considerable observation time of 258 patient years on prophylaxis with more than 40,000 FVIII injections. Fig. 1 Patients treated with concentrates (n) Fig. 2 Patient status at study entry Conclusion This snapshot analysis with a long observation time of a median of 31.5 months delivers important data on the ABR in real-life treatment of Haemophilia A. It further confirms the outstanding tolerability and efficacy of the concentrates investigated with no adverse drug reaction occurring within 10 years of study observation. Prophylaxis with FVIII concentrates remains a safe and effective treatment option for Haemophilia A patients. Publication History Article published online: 17 February 2026 © 2026. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.