The PEAQ-NOW study: Expanding a successful, prospective, non-interventional study on real-life Haemophilia A treatment with factor VIII concentrates by including other FVIII treatment modalities with enhanced focus on joint health

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Introduction Over the past 10 years, the treatment of haemophilia A (HA) has changed fundamentally, offering haemophilia patients a wider range of treatment options, such as prophylaxis with non-factor preparations or gene therapy. It has also become evident, that joint damage occurs in patients with mild or moderate HA to a significant extent [ 1 ], including female patients formerly classified as asymptomatic carriers. State-of-the-art assessment of real-life treatment with factor VIII (FVIII) concentrates should therefore also include patients treated on-demand (with or without non-factor prophylaxis) with FVIII while assessing joint health parameters along with other haemophilia related treatment parameters. The study GENA-100, which was started 10 years ago to collect data on FVIII prophylactic treatment, has been adapted accordingly. Method PEAQ-NOW (=GENA-100 with amendments) is designed to collect information on FVIII treatment of HA patients of all severities and ages treated with simoctocog alfa and 2 plasma-derived von Willebrand factor containing factor FVIII concentrates from one company. In addition to patients treated prophylactically, patients with Haemophilia A treated on-demand (whether alone or in addition to non-factor prophylaxis) with one of the concentrates are to be included with the last study amendment of GENA-100. The study plans to include at least 100 patients treated on-demand and 100 on prophylaxis. In addition to the Haemophilia Joint Health Score (HJHS), the documentation of HEAD-US (Haemophilia Early Arthropathy Detection by Ultrasound) as well as an extended questionnaire on joint health has been introduced. Thorough documentation of FVIII administrations, bleeding episode and surgical procedures remain the backbone of documentation. In addition to relevant medical history for HA patients, the ISTH-BAT (Bleeding Assessment Tool of the International Society of Thrombosis and Haemostasis) has been introduced, allowing evaluation of the general bleeding tendency between groups. Evaluation will focus on ABR, FVIII requirement and joint health parameters for each treatment group and – where data will allow for meaningful comparisons – for patient groups as well. Results As of October 2025, 88 patients within GENA-100 had received prophylaxis. Results of a current snapshot analysis are reported elsewhere. Conclusion The PEAQ-NOW study is currently the only study that investigates all patient groups in a real-life setting receiving FVIII concentrate on-demand or prophylactically. This will allow for interesting comparisons between treatment modalities and patient groups on a variety of parameters, including joint health. Publication History Article published online: 17 February 2026 © 2026. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

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