PB1134 - Practical utilisation of Octapharma FVIII concentrates in previously untreated and minimally treated haemophilia A patients entering routine clinical treatment – The Protect-NOW Study

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Susan Halimeh, MD

Professor
Gerinnungszentrum Rhein Ruhr, Duisburg, Germany
Duisburg, Nordrhein-Westfalen, Germany

Co-Author(s)

Johannes Oldenburg

Professor, Chairman and Director
University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany
Bonn, Nordrhein-Westfalen, Germany
Robert Klamroth, MD, PhD

Priv. Doz. Dr. med.
Vivantes Klinikum im Friedrichshain, Clinic for Internal Medicine Angiology and Haemostaseology
Berlin, Berlin, Germany
MM

Mary Mathias

Haemophilia Centre Director
Great Ormond Street Hospital for Children, NHS Foundation Trust
London, England, United Kingdom
GH

Georgina Hall

Consultant paediatric haematologist
Oxford University Hospitals NHS Foundation Trust
Oxford, England, United Kingdom
PM

Pascual Marco Vera

Hematologist
Departmentof Clinical Medicine, Miguel Hernández University, Alicante, Spain
Alicante, Comunidad Valenciana, Spain
MJ

Martina Jansen

Senior Clinical Project Manager
Octapharma Pharmazeutika Produktionsgesellschaft mbH
Vienna, Wien, Austria

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.