Abstract Number: PB1062
Meeting: ISTH 2020 Congress
Background: There is still a need to systematically analyze the prophylactic treatment of Haemophilia A (HA) in real life routine clinical practice since clinical trials are subjected to certain restrictions.
Aims: The prospective, observational study NIS-Previq is designed to collect information on prophylactic treatment of HA patients of all severities and ages treated with simoctocog alfa and 2 plasma derived von-Willebrand factor containing FVIII concentrates from one company.
Methods: Patients with HA of all ages receiving prophylaxis and general compliance to their therapy regimen 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), health related quality of life (SF-36) and a PK-assessment including dosing simulation for potential adaptation of therapy schedules with the full PK approach or the population-based PK using WAPPS-Hemo (www.wapps-hemo.org).
Results: As of January 2020, 7 German Haemophilia Centres enrolled 41 prophylactically treated patients, including 1 previously untreated patient and 1 with 15 previous exposure days. Total observation time was 834.2 months / 69.5 patient years with a mean of 20.35 months per patient (IQR: 7.2 – 27.5). In total, 31.6 Mio units FVIII were given, mainly for prophylactic purposes (88% of injections). The median ABR for spontaneous bleeds was 0 (IQR 0.9 – 3.3, for treated bleeds 2.0 (IQR 0.0 – 1.3). A traumatic cause was indicated for 64 % of bleeds. The efficacy of treated bleeds was rated “excellent/good” in 95 % of cases. 20 surgical procedures were performed in 12 patients.
No inhibitor formation or any other adverse drug reaction occurred during the observation period.
Conclusions: The snapshot on the study data so far confirm the good tolerability and efficacy of FVIII-based prophylaxis with 3 different FVIII concentrates.
|Presence of at least 1 target joint||3||3||2||10||5||1|
|Previous Inhibitor activity||0||3||0||3||0||0|
|Non-severe Haemophilia A (>1%FVIII)||0||0||2||0||1||1|
[Number of patients by age group and selected demographic information at study entry (n patients)]
To cite this abstract in AMA style:Halimeh S, Behnisch W, Escuriola-Ettingshausen C, Feddern J, Huth-Kühne A, Oldenburg J, Scholz U, Seeger S, Wenning S, Klamroth R. A Snapshot of a Prospective, Non-interventional Study to Evaluate Routine Practice Prophylactic Treatment Schedules – NIS-Previq [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/a-snapshot-of-a-prospective-non-interventional-study-to-evaluate-routine-practice-prophylactic-treatment-schedules-nis-previq/. Accessed May 6, 2021.
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