PB0624 - A-SURE: Intra-patient Comparison of Prophylactic Effectiveness of a Recombinant Factor VIII Fc Fusion Protein Versus Standard Half-life Factor VIII in Hemophilia A

Monday, June 26, 2023

18:30 – 19:30 ET

Room: Exhibition

Co-Author(s)

Johannes Oldenburg, MD (he/him/his)

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 (she/her/hers)

Director
IRCCS Ca' Granda Maggiore Hospital Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milano, Italy
Milano, Lombardia, Italy
AL

Anna-Elina Lehtinen

Adjunct Professor, Head of the Unit
Coagulation Disorders Unit, Department of Hematology, Helsinki University Hospital Comprehensive Cancer Center and University of Helsinki, Helsinki, Finland
Helsinki, Uusimaa, Finland

Presenting Author(s)

Ingrid Pabinger, MD

Co-Head of the Thrombosis and Haemostasis Research Group
Clinical Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria
Vienna, Wien, Austria

Co-Author(s)

EN

Eveline Nüesch

Statistical Science Director
Sobi, Basel, Switzerland
Basel, Basel-Stadt, Switzerland
HM

Håkan Malmström

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

Aletta Falk

Director Medical Affairs Haemophilia
Sobi, Stockholm, Sweden
Stockholm, Stockholms Lan, Sweden
SL

Stefan Lethagen

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

Background: Significantly lower annualized bleeding rate (ABR), injection frequency, and factor consumption with efmoroctocog alfa (recombinant factor VIII [FVIII] Fc fusion protein [rFVIIIFc]) were reported in the head-to-head comparison with standard half-life (SHL) FVIII products in the observational A-SURE study in patients with hemophilia A (HA) [1].

Aims: To report intra-patient analyses of real-world effectiveness of prophylaxis with rFVIIIFc versus previous SHL FVIII in patients switching from SHL FVIII to rFVIIIFc.

Methods: A-SURE (NCT02976753), a 24-month prospective, non-interventional, European multicenter study in HA, compared rFVIIIFc with SHL FVIII; ≤12 months retrospective data before enrollment were also assessed. Patients included in these post-hoc analyses had received SHL FVIII for ≥3 months followed by rFVIIIFc for ≥3 months. For all annualized endpoints, ≥3 months documented treatment was required. The analyses performed describe treatment outcomes in patients switching prophylaxis to rFVIIIFc from SHL FVIII.

Results: The analyses included 131/356 A-SURE patients (median [range] age, 20 [1–76] years). rFVIIIFc prophylaxis was initiated before enrollment (n=82), at enrollment (n=27), or after enrollment (n=22). Median (interquartile range) treatment duration (months) on previous SHL FVIII was 10.4 (7.1, 12.0), and 24.8 (19.6, 27.7) on rFVIIIFc. Mean (95% confidence interval [CI]) change after switch from SHL FVIII to rFVIIIFc in ABR and annualized joint bleeding rate were −1.9 (−3.0, −0.8) and −1.2 (−2.0, −0.5), respectively. Mean (95% CI) change for weekly injection frequency and weekly factor consumption (international unit/kg) were −0.8 (−1.0, −0.7) and −5.7 (−10.7, −0.6), respectively (Figure 1).

Conclusion(s): Switching to rFVIIIFc from SHL FVIII prophylaxis reduces ABRs, injection frequency, and factor consumption, which is consistent with previously reported primary outcomes of A-SURE.

1. Oldenburg J, et al. Res Pract Thromb Haemost 2022;6(S1):e12787.

The A-SURE study (NCT02976753) was funded by Sobi.