Baseline Data from a 24-month Prospective, Non-interventional, Multicentre Study in Germany to Evaluate the Real-world Usage and Effectiveness of rFVIIIFc and rFIXFc in Patients with Haemophilia A or B (PREVENT)

Scientific Research Question: Recombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) have been approved on the basis of their pivotal phase 3 trials in children, adolescents and adults with haemophilia A or B, respectively. The ongoing PREVENT study (NCT03055611) is a non-interventional study with the objective to provide data on the real-world usage and effectiveness of prophylaxis with rFVIIIFc and rFIXFc in Germany over a 24-month prospective period. Here the aim is to report on the current status of the study and present baseline characteristics and retrospective clinical data.

Methodology: The prospective, multicentre, non-interventional PREVENT study aims to recruit and collect data from at least 100 patients with haemophilia A and B in Germany. At enrolment, baseline characteristics and retrospective data are collected. The retrospective data includes factor usage and bleeding events during the 12-month period before the first injection with rFVIIIFc/rFIXFc and if applicable, during the period after switch to rFVIIIFc/rFIXFc until enrolment.

Findings: The study is ongoing and as of 25 September 2018, 77 haemophilia A and 23 haemophilia B patients have been enrolled at 20 centres. Preliminary baseline data: Of the 100 patients recruited, 61 are adults and 39 are children. Ninety-two have severe, 7 have moderate and 1 has mild haemophilia. The rationale for initiating treatment with rFVIIIFc or rFIXFc, as provided by treating physician at baseline, was to reduce injection frequency while maintaining protection from bleeds for 70 patients, to improve protection from bleeds for 27 patients and to improve protection to increase physical activity level for 2 patients (data missing for 1 patient). Further patient and disease characteristics at enrolment will be presented including age distribution, presence of target joints, history of inhibitors, history of prophylaxis, as well as bleeding events, prescribed injection frequency and dose before enrolment.

Conclusions: The baseline data from the ongoing PREVENT study provide insights into the demographics and clinical characteristics, as well as the rationale for initiating prophylaxis with rFVIIIFc and rFIXFc, in patients with haemophilia A and B in Germany.

No conflict of interest has been declared by the author(s).