Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study

Carmen Escuriola Ettingshausen; Cedric Hermans; Pål A Holme; Ana R Cid; Kate Khair; Johannes Oldenburg; Claude Négrier; Jaco Botha; Aurelia Lelli; Jerzy Windyga

doi:10.1177/20406207231184323

Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study

Ther Adv Hematol. 2023 Jul 24:14:20406207231184323. doi: 10.1177/20406207231184323. eCollection 2023.

Authors

Carmen Escuriola Ettingshausen¹, Cedric Hermans², Pål A Holme³, Ana R Cid⁴, Kate Khair⁵, Johannes Oldenburg⁶, Claude Négrier⁷, Jaco Botha⁸, Aurelia Lelli^{8

9}, Jerzy Windyga¹⁰

Affiliations

¹ Hämophilie-Zentrum Rhein Main - HZRM GmbH, Mörfelden-Walldorf, Germany.
² Hemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, UCLouvain, Brussels, Belgium.
³ Department of Haematology, Oslo University Hospital and Institute for Clinical Medicine, University of Oslo, Oslo, Norway.
⁴ Unidad de Hemostasia y Trombosis, Hospital Universitario y Politécnico La Fe, València, Spain.
⁵ Centre for Outcomes and Experience Research in Children's Health, Illness and Disability (ORCHID), Great Ormond Street Hospital for Children, London, UK.
⁶ Institute of Experimental Hematology and Transfusion Medicine, Bonn University Clinic, Bonn, Germany.
⁷ University Claude Bernard Lyon 1, Lyon, France.
⁸ Takeda Pharmaceuticals International AG, Zürich, Switzerland.
⁹ R&D, Plasma Derived Therapy, Takeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Glattpark-Opfikon, Zürich, Switzerland.
¹⁰ Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland.

Abstract

Background: The bypassing agent, activated prothrombin complex concentrate [aPCC, FEIBA (factor VIII inhibitor bypass activity); Baxalta US Inc, a Takeda company, Lexington, MA, USA], is indicated for the treatment of bleeding episodes, perioperative management, and routine prophylaxis in patients with hemophilia A or B with inhibitors. In certain countries, aPCC is also indicated for the treatment of bleeding episodes and perioperative management in patients with acquired hemophilia A.

Objectives: To describe long-term, real-world effectiveness, safety, and quality-of-life outcomes for patients with congenital hemophilia A or B and high-responding inhibitors receiving aPCC treatment in routine clinical practice.

Design: FEIBA Global Outcome (FEIBA GO; EUPAS6691) was a prospective, observational study.

Methods: Investigators determined the treatment regimen and clinical monitoring frequency. The planned patient observation period was 4 years. Data are from the safety analysis set (patients who received ⩾1 aPCC infusion).

Results: Overall, 50 patients received either aPCC prophylaxis (n = 37) or on-demand therapy (n = 13) at screening [hemophilia A, n = 49; hemophilia B, n = 1; median (range) age, 16.5 [2-71] years). Mean ± standard deviation overall annualized bleeding rate and annualized joint bleeding rate for patients receiving prophylaxis were 6.82 ± 11.52 and 3.77 ± 5.71, respectively, and for patients receiving on-demand therapy were 10.94 ± 11.27 and 6.94 ± 7.39, respectively. Overall, 177 and 31 adverse events (AEs) were reported in 28 of 40 and 10 of 13 patients receiving prophylaxis or on-demand therapy, respectively. Two serious AEs were considered possibly related to aPCC: acute myocardial infarction due to coronary artery embolism in one patient receiving prophylaxis. No thrombotic microangiopathy was reported. No AEs resulted in death.

Conclusion: This study demonstrated the long-term, real-world effectiveness and consistent safety profile of aPCC as on-demand therapy and prophylactic treatment in patients with hemophilia and high-responding inhibitors.

Trial registry: FEIBA Global Outcome Study; EUPAS6691 https://www.encepp.eu/encepp/viewResource.htm?id=32774.

Keywords: factor VIII inhibitor bypassing activity; hemophilia A; hemophilia B; inhibitors; observational study.