OC 27.4 - Recombinant factor VIII Fc showed better prophylactic effectiveness compared to standard half-life factor VIII in haemophilia A - results from A-SURE, a 24-month prospective, non-interventional study

Sunday, July 10, 2022

3:30 PM – 3:45 PM

Location: ExCel Center, ICC Capital Suite Room 2-4

Presentation Format: In-Person

Presenting 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

Co-Author(s)

CH

Charles Hay

Clinical Professor of Haemostasis and Thrombosis, Consultant Haematologist
University Department of Haematology, Manchester Royal Infirmary, Manchester, United Kingdom
Manchester, England, United Kingdom
Flora Peyvandi, MD, PhD

Professor of Internal Medicine; Director of the Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
Milan, Lombardia, Italy
AT

Annarita Tagliaferri

Dr
Regional Reference Center for Inherited Bleeding Disorders, University Hospital of Parma
Parma, Emilia-Romagna, Italy
Pål Andrè Holme

Professor
1Department of Haematology, Oslo University Hospital 2Research Institute of Internal Medicine, Oslo University Hospital 3Institute of Clinical Medicine, University of Oslo, Oslo, Norway
Oslo, Oslo, Norway
María Teresa Alvarez-Román, MD, PhD

Head of the Thrombosis and Haemostasis Unit
La Paz University Hospital, Madrid, Spain
Madrid, Madrid, Spain
CB

Christine Biron

MD
Haemophilia Treatment Centre, University Hospital, Montpellier, France
Montpellier, Languedoc-Roussillon, France
HM

Håkan Malmström

Director, Head of Observational Research
Sobi, Stockholm, Sweden
Stockholm, Stockholms Lan, Sweden
SL

Stefan Lethagen

Vice President Medical & Clinical Sciences
Sobi, Stockholm, Sweden
Höllviken, Skane Lan, Sweden

Background: Recombinant factor VIII Fc (rFVIIIFc) was the first approved extended half-life (EHL) FVIII product for people with haemophilia A (PwHA). Efficacy and safety were established in phase 3 studies and their extension.

Aims: To report direct comparative real-world effectiveness of rFVIIIFc prophylaxis vs. a matched treatment group on standard half-life (SHL) FVIII.

Methods: A-SURE (NCT02976753) was a 24-month prospective, non-interventional, European multicentre study in PwHA comparing rFVIIIFc with SHL FVIII prophylaxis (with ethics committee approvals and informed consents). Considering different aims for prophylactic regimens, three primary endpoints were defined, including annualised bleeding rate (ABR), injection frequency, and factor consumption. Each included patient on rFVIIIFc was matched by age and last prescribed weekly SHL FVIII dose prior to baseline at local site level to a patient on SHL FVIII. To reduce potential effects of confounding, propensity scores were estimated based on patient characteristics at baseline and adjusted for in the statistical analysis. All primary endpoints were analysed with Generalized Linear Mixed Models, including the ABR endpoint for which poor model fit ruled out the use of the predefined negative binomial regression model.

Results: 356 PwHA were enrolled and eligible, 186 in the rFVIIIFc group and 170 in the SHL FVIII group.
All primary endpoints i.e. mean ABR, annualized injection frequency and annualized factor consumption, were significantly lower in the rFVIIIFc group compared to the SHL FVIII group during the 24-month prospective period (Table 1).
rFVIIIFc was well tolerated, consistent with the established safety profile. No inhibitors occurred, including in patients with a previous inhibitor history.

Conclusion(s): The 24-month prospective non-interventional A-SURE study with a solid comparative design including a matched control group, demonstrated significantly lower ABR, injection frequency and factor consumption with rFVIIIFc compared to SHL FVIII. Thus, rFVIIIFc shows beneficial effects on disease and treatment burden compared to SHL FVIII in a real-world setting.