PB1255 - An update on real-world use of rVIII-SingleChain in patients with haemophilia A in Germany: Interim results from a prospective, non-interventional study

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Martin Olivieri (he/him/his)

Head of the Paediatric Thrombosis and Haemostasis Unit
Paediatric Thrombosis and Haemostasis Unit, Paediatric Haemophilia Center, Dr. von Hauner Children's Hospital, LMU Clinic, Munich, Germany
Munich, Bayern, Germany

Co-Author(s)

SJ

Sabrina Juranek

Specialist in paediatrics and youth medicine; Central Emergency Room; Hemostaseology
Paediatric Thrombosis and Haemostasis Unit, Dr. von Hauner Children´s Hospital LMU Munich, Germany
Munich, Bayern, Germany
FL

Florian Langer

Medical Specialist in Internal Medicine
Universitätsklinikum Eppendorf, II. Medizinische Klinik und Poliklinik, Hubertus Wald Tumorzentrum, Universitäres Cancer Center Hamburg (UCCH), Hamburg, Germany
Hamburg, Hamburg, Germany
Carmen Escuriola-Ettingshausen, MD

Physician
Haemophilia Centre Rhine-Main, Mörfelden-Walldorf, Germany
Mörfelden-Walldorf, Hessen, Germany
TL

Thomas Linhoff

Medical Affairs Manager Hemophilia & VWD
CSL Behring, Hattersheim, Germany.
Hattersheim, Hessen, Germany
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

Background: Previous studies have shown that rVIII-SingleChain is efficacious and well tolerated in patients with haemophilia A; however, long-term studies are needed to confirm real-world safety/effectiveness in routine clinical practice.

Aims: To evaluate the real-world safety and efficacy of rVIII-SingleChain in patients with haemophilia A in Germany.

Methods: Data are reported from a sub-analysis of patients from Germany included in a pan-European prospective, non-interventional study of patients with haemophilia A receiving rVIII-SingleChain prophylaxis or on-demand treatment. Patients were monitored for up to 3 years, and annualised bleeding rates (ABR), annualised spontaneous bleeding rates (AsBR), infusion frequency, dosage and haemostatic effectiveness were recorded.

Results: At the interim data cut-off on 14 January 2022, 43 patients with a mean (standard deviation [SD]) age of 32.7 (16.9) years were enrolled in the study over a mean (SD) observation period of 14.0 (5.9) months. Patient characteristics and treatment data are provided in Table 1. The median ABR and AsBR across all prophylaxis dosing regimens were both 0.0, and 1.1 and 0.0, respectively, in patients receiving on-demand treatment. Most of the bleeding episodes reported were of mild/moderate severity in both the prophylaxis and on-demand groups (53.3% and 63.2%, respectively). Overall haemostatic effectiveness was rated as excellent (n=15, 53.6%) or good (n=13, 46.4%) in patients with available data from at least one assessment (n=28). In total, 12 adverse events were reported in 6 patients, including one event of gastritis that was considered serious.

Conclusion(s): This interim analysis confirms the safety and efficacy of rVIII-SingleChain for routine prophylaxis and on-demand treatment in patients with haemophilia A. Data collection is ongoing to assess long-term effectiveness and tolerability.