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Practical utilisation of Octapharma FVIII concentrates in previously untreated and minimally treated haemophilia A patients entering routine clinical treatment – The Protect-NOW Study

1Gerinnungszentrum Rhein-Ruhr, Duisburg, Nordrhein-Westfalen, Germany, 2University Clinic Bonn, Bonn, Nordrhein-Westfalen, Germany, 3Vivantes Klinikum im Friedrichshain, Berlin, Berlin, Germany, 4Great Ormond Street Hospital for Children, NHS Foundation Trust, London, England, United Kingdom, 5Children’s Hospital Oxford, Oxford Haemophilia and Thrombosis Comprehensive Care Centre, Oxford University Hospital NHS Foundation Trust, Oxford, England, United Kingdom, 6Departamento de Medicina Clínica. Universidad Miguel Hernández, Jefe de Servicio de Hematología y Hemoterapia, Hospital General Universitario de Alicante, Alicante, Comunidad Valenciana, Spain, 7Octapharma Pharmazeutika Produktionsgesellschaft m.b.H, Vienna, Salzburg, Austria

Abstract Number: PB1134

Meeting: ISTH 2022 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: For previously untreated patients (PUPs) and minimally treated patients (MTPs) with severe haemophilia A it is important to balance the need to minimise the risk of inhibitor development while providing all-around bleed prevention. The efficacy, safety and immunogenicity of Octapharma FVIII products in PUPs have been demonstrated in prospective clinical trials. The incidence of high-titre inhibitors was 16%, 8% and 11% in trials with Nuwiq®, octanate® and wilate®, respectively.

Aims: The aim of the ongoing, non-interventional, prospective and retrospective Protect-NOW study (NCT03695978) is to investigate the effectiveness, safety, immunogenicity and utilisation of Nuwiq®, octanate® and wilate® in routine clinical practice in PUPs and MTPs with severe haemophilia A. Optional sub-studies will assess risk factors associated with inhibitor development.

Methods: 140 PUPs and MTPs ( < 5 previous exposure days [EDs] with other FVIII products) of any age and ethnicity will be followed for 100 EDs or up to 3 years after ED1. The primary outcome measures are the annualised rate of breakthrough bleeds to assess prophylactic efficacy and the incidence of adverse drug reactions to assess safety. Measurement of inhibitor activity is recommended every 3–4 EDs until ED20 and every 10–12 EDs thereafter. Optional sub-studies include F8 gene analysis, measurement of non-neutralising anti-FVIII antibodies, and epitope mapping. Patients on concomitant emicizumab prophylaxis may be included in the study.

Results: As of January 2022, 51 patients have been enrolled in the study from 29 sites in 14 countries. Recruitment is ongoing, and additional sites are being initiated.

Conclusion(s): Protect-NOW is collecting real-world data on the effectiveness and safety of Octapharma FVIII products in routine clinical practice in PUPs and MTPs.

To cite this abstract in AMA style:

Halimeh S, Oldenburg J, Klamroth R, Mathias M, Hall G, Marco Vera P, Jansen M. Practical utilisation of Octapharma FVIII concentrates in previously untreated and minimally treated haemophilia A patients entering routine clinical treatment – The Protect-NOW Study [abstract]. https://abstracts.isth.org/abstract/practical-utilisation-of-octapharma-fviii-concentrates-in-previously-untreated-and-minimally-treated-haemophilia-a-patients-entering-routine-clinical-treatment-the-protect-now-study/. Accessed October 1, 2023.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/practical-utilisation-of-octapharma-fviii-concentrates-in-previously-untreated-and-minimally-treated-haemophilia-a-patients-entering-routine-clinical-treatment-the-protect-now-study/