Abstract Number: PB0627
Meeting: ISTH 2021 Congress
Background: Optimal management of previously untreated patients (PUPs) and minimally treated patients (MTPs) with severe haemophilia A (FVIII:C <1%) must address numerous concerns to ensure good long-term outcomes. The efficacy, safety and immunogenicity of Nuwiq®, octanate® and wilate® in PUPs have been investigated in clinical trials; the incidence of high-titre inhibitors was 16%, 8% and 11% with Nuwiq®, octanate® and wilate®, respectively.
Aims: Protect-NOW is an ongoing, non-interventional, prospective and retrospective study. Protect-NOW aims to investigate the effectiveness and overall safety, including immunogenicity, of Nuwiq®, octanate® and wilate® in routine clinical practice for the prevention and treatment of bleeding events in PUPs and MTPs with severe haemophilia A. Optional sub-studies will assess risk factors associated with inhibitor development.
Methods: It is planned to enrol 140 PUPs and MTPs (<5 previous exposure days [EDs] with other FVIII products) of any age and ethnicity for 100 EDs or up to 3 years after ED1. Treatment is at the discretion of the investigator. The primary objectives are effectiveness in the prevention and treatment of bleeding events, as well as overall safety, including inhibitor development. Measurement of inhibitor activity is recommended every 3–4 EDs until ED20 and every 10–12 EDs thereafter. Secondary objectives are to assess treatment utilisation patterns (including dosage and frequency of administration) and effectiveness in surgical prophylaxis.
Results: As of March 2021, 37 patients have been enrolled in the study from 13 sites in Belarus, Canada, France, Germany, Italy, Lithuania, Spain and the United Kingdom, with additional sites to be initiated.
Conclusions: Protect-NOW is collecting clinically relevant information on the outcomes of PUPs and MTPs treated with Octapharma‘s FVIII products in a real-world setting.
To cite this abstract in AMA style:Oldenburg J, Vera PM, Halimeh S, Klamroth R, Lowndes S, Jansen M. PRactical Utilisation of Octapharma FVIII Concentrates in Previously Untreated and Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment with Nuwiq®, octanate® or wilate® – The Protect-NOW Study [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/practical-utilisation-of-octapharma-fviii-concentrates-in-previously-untreated-and-minimally-treated-haemophilia-a-patients-entering-routine-clinical-treatment-with-nuwiq-octanate-or-wila/. Accessed October 19, 2021.
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