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.
