PB0557 - Efficacy and safety of rVIII-SingleChain in people with hemophilia A: Interim results from a non-interventional study

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Johannes Oldenburg, MD

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

Co-Author(s)

SJ

Sabrina Juranek

Specialist in paediatrics and adolescent medicine
Dr. von Haunersches Children Hospital, Ludwig-Maximilians-Universität, Munich, Germany
Munich, Bayern, Germany
OK

Olga Katsarou

Head of the Blood Transfusion Unit, Laiko hospital, Greece
Laiko Hospital, Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Athens, Greece
Athens, Attiki, Greece
SB

Soraya Benchikh El Fegoun

Global Medical Director, Global Medical Affairs Hematology TA
CSL Behring, Bern, Switzerland
Bern, Bern, Switzerland
Ingrid Pabinger

Professor
Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna
Vienna, Wien, Austria

Background: Clinical trial data have shown that rVIII‑SingleChain, a recombinant factor VIII, is efficacious and well-tolerated in people with hemophilia A (HA).

Aims: To assess the long-term effectiveness and safety of rVIII-SingleChain in routine clinical practice in Europe.

Methods: In a non-interventional study, people with HA received rVIII-SingleChain in routine clinical practice and were followed up for up to three years. The key primary endpoint was annualized bleeding rate (ABR). The severity of the bleeds was evaluated by the investigators.

Results: In the third data-cut, 72 patients (46 with severe HA), with a mean (standard deviation [SD]) age of 31.2 (16.05) years from Germany (n=43), Greece (n=12), Austria (n=9), Belgium (n=4), Czech Republic (n=2), and Hungary (n=2) were enrolled. Participants had been followed up for a mean (SD) period of 27.50 (7.83) months; 13 participants have completed the study. Thirteen participants discontinued (none due to lack of effectiveness of rVIII-SingleChain. The mean ABR and AsBR (both n=56) for participants on prophylaxis were 1.21 and 0.80, respectively. In the on-demand group, ABR and AsBR (both n=12) were 2.01 and 0.24, respectively. Most bleeds reported across both treatment groups were mild (n=71/164 for the prophylaxis group; n=40/61 for the on-demand); 20/164 and zero severe bleeds were reported in the prophylaxis and on-demand groups, respectively. Overall, hemostatic effectiveness was excellent (n=42/63; 48.8%) or good (n=33/63; 38.4%) for patients with at least one assessment of bleeding. Twenty-six adverse events were reported in 16 patients; none were related to rVIII-SingleChain and no inhibitor formation was observed.

Conclusion(s): Interim results from this non-interventional study confirmed the effectiveness and favorable safety profile of rVIII-SingleChain in prophylaxis and on-demand treatment in routine patient care.