PB0505 - Long-term joint health outcomes with a recombinant factor VIII Fc from the 48-month prospective, observational A-MORE study: Third interim analysis of up to 24 months

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

JA

Jan Astermark

Professor, Senior Consultant
Department of Translational Medicine, Lund University, Malmö, Sweden
Malmo, Skane Lan, Sweden
Johannes Oldenburg, MD

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

Presenting Author(s)

Rubén Berrueco, PhD, MD

Head of Pediatric Hematology Department
Sant Joan de Déu Hospital, Barcelona, Spain.
Barcelona, Catalonia, Spain

Co-Author(s)

AT

Annarita Tagliaferri

Director
University Hospital of Parma, Regional Reference Center for Inherited Bleeding Disorders
Parma, Emilia-Romagna, Italy
MA

Mikaela Alenäs

Statistician
Sobi
Stockholm, Stockholms Lan, Sweden
MF

Markus Fusser

Global Medical Lead
Sobi, Stockholm, Sweden
Solna, Stockholms Lan, Sweden
SL

Stefan Lethagen

Vice President
Sobi, Stockholm, Sweden
Stockholm, Stockholms Lan, Sweden

Background: Extended half-life (EHL) efmoroctocog alfa (rFVIIIFc) prophylaxis (PPX) has demonstrated improved joint health in persons with hemophilia A (PwHA).

Aims: To report real-world long-term joint health data with rFVIIIFc in PwHA from A-MORE.

Methods: A-MORE (NCT04293523) is an ongoing 48-month prospective, non-interventional study, including PwHA of all ages/severities receiving rFVIIIFc PPX across 14 countries in Europe/the Middle East. This descriptive analysis presents baseline characteristics and 3rd interim data (cut off: 7 July 2023) from the full baseline population, including 12-month retrospective and up to 24-month prospective data.

Results: Overall, 419 PwHA were analyzed (418 males). Median (range) age and on-study follow-up were 22 years (0–83) and 455 days (1–1,065), respectively (Table 1). Within 12 months pre-study, 387/51 PwHA (92.4%/12.2%) received ≥3 months EHL/SHL FVIII PPX.
Mean overall and joint annualized bleed rates (ABRs) were low at baseline and remained low at the 12- and 24-month visits (subset with available data; Figure 1); the proportion with zero bleeds remained stable across these timepoints (63.3%; 66.9%; 64.4%).
At baseline, 12, and 24 months, mean (standard deviation [SD]) weekly injection frequency was 2.2 (0.8), 2.3 (0.7), and 2.5 (0.8), respectively; corresponding mean (SD) weekly prescribed dose was 82 IU/kg (28), 86 IU/kg (39), and 94 IU/kg (47).
At baseline, mean (SD) total Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score and Hemophilia Joint Health Score (HJHS) were 7.4 (9.8; n=88) and 9.2 (15.4; n=103), respectively. At 12 and 24 months, mean (SD) change from baseline was -0.3 (2.0; n=68) and -0.9 (1.9; n=36) for HEAD-US score, and -0.3 (2.7; n=47) and -0.2 (3.4; n=29) for HJHS, respectively.

Conclusion(s): Third interim real-world A-MORE data align with previous analyses, demonstrating that rFVIIIFc PPX offers long-term effective bleed and joint protection in PwHA.
A-MORE was funded by Sobi.