A snapshot analysis of a prospective, non-interventional study to evaluate real-life prophylactic treatment schedules of factor VIII concentrates

Introduction There is still a need to systematically document the prophylactic treatment of Haemophilia A (HA) in real-life clinical practice. Hence dosing and other elements of HA treatment are documented in a non-interventional fashion. The primary objective of this non-interventional study (NIS) is the assessment of the influence of the weekly prophylactic FVIII dose on annualized bleeding rates (ABR).

It further aims to analyze all aspects of prophylactic treatment with Simoctocog alfa (Nuwiq) and the plasma-derived FVIII concentrates Wilate and Octanate.

Method Patients with HA of all ages receiving prophylaxis and having a good compliance are eligible to be enrolled after informed consent is given. All details of bleeding episodes and factor VIII administrations are recorded. 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 2022, 53 patients were included with ages at study entry ranging between 1 and 91 years. 26 patients below age 12 were enrolled. The median observation time was 31.5 months. In total, 447 episodes were documented as bleeds (57.3 % mild, 33.3 % moderate, 6.9 % of severe intensity). 75 % of episodes had a traumatic cause. 97 % of rated episodes received an “excellent/good“ assessment of treatment. Mean ABR was 2.94 for all bleeds, 0.7 for spontaneous bleeds and 2.01 for traumatic bleeds. These reflects the high proportion of children in the study with a large inter-patient variability, however. 35 surgeries in 18 patients were performed during the observation time. No inhibitor formation or any other adverse drug reactions were observed ([Tab. 1]).

Tab. 1 Patient Status at Study Entry.

Conclusion This snapshot analysis with a long observation time of a median of 31.5 months delivered important data on the ABR in real-life treatment of haemophilia A. It further confirms the outstanding tolerability and efficacy of the concentrates investigated.

Publikationsverlauf

Artikel online veröffentlicht:
20. Februar 2023