Haemophilia A Prophylaxis with FVIII in Real-life: Snapshot Analysis on the Observational Study “Prevention of Bleeding in Haemophilia A by Prophylactic Treatment with Octanate^®, Wilate^® or Nuwiq^® - NIS Previq”

Scientific Research Question: The primary objective of the NIS-Previq study is the assessment of the influence of the weekly prophylactic FVIII dose on annualized bleeding rates (ABR). It further documents the prophylactic treatment regimen with Nuwiq, Octanate or Wilate in haemophilia A patients as it is done in routine clinical practice. With the possibility to evaluate the elimination rates of FVIII of patients, the practicability of PK-based for individualization of prophylactic schedules can be investigated.

Methodology: Patients with Haemophilia A of all ages receiving prophylaxis - defined as at least 1 injection per week planned for at least the following 6 months after inclusion - and general compliance are eligible to be enrolled after informed consent has been given. All details of bleeding episodes (date, reason, location, duration, intensity, efficacy) and factor VIII administrations (concentrate, dose, date, time, reason) including weekly distribution of prophylactic injections and changes in treatment schedules are recorded. Optional study elements comprise initial and regular assessment of joint scores (HJHS), health related quality of life (SF-36) and a PK-assessment including dosing simulation for potential adaptation of therapy schedules with either the full PK approach or the population-based PK using WAPPS-hemo.

Findings: Out of 23 patients included since January 2015, 18 are treated with Nuwiq, 4 with Octanate and one with Wilate. The median observation time is 27 months. At study entry, 7 patients were children (< 12 years), 7 patients were adolescents/young adults (13-23 years) and 9 patients were adults (26-59 years).

All patients are on home treatment. 20/23 showed good or very good compliance in documenting their injections. The frequency of follow-up visits varies between 4 times and once per year.

The general physical stress in most of the patients is documented to be “light”. The time of injections during the day varies considerably in some patients: only about 1/3 of all injections are performed in the morning.

No inhibitor formation or any other adverse drug reactions were observed.

Conclusions: The snapshot analysis on the study data so far confirm the good tolerability and efficacy of Nuwiq, Octanate and Wilate. Such study allows to further evaluate the real-life application of prescribed prophylactic schedules.

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