The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study

Margareth C Ozelo; Cedric Hermans; Manuel Carcao; Benoît Guillet; Joan Gu; Randy Guerra; Leilei Tang; Kate Khair

doi:10.1177/20406207231218624

The effectiveness and safety of octocog alfa in patients with hemophilia A: up to 7-year follow-up of the real-world AHEAD international study

Ther Adv Hematol. 2024 Feb 15:15:20406207231218624. doi: 10.1177/20406207231218624. eCollection 2024.

Authors

Margareth C Ozelo¹, Cedric Hermans², Manuel Carcao³, Benoît Guillet⁴, Joan Gu⁵, Randy Guerra⁶, Leilei Tang⁷, Kate Khair⁸

Affiliations

¹ Hemocentro UNICAMP, University of Campinas, Campinas, São Paulo, Brazil.
² St-Luc University Hospital, Université catholique de Louvain, Louvain, Belgium.
³ Division of Haematology/Oncology, Hospital for Sick Children, Toronto, ON, Canada.
⁴ Haemophilia Treatment Center, Univ Rennes, CHU Rennes, Inserm, EHESP, Irset (Institut de recherche en santé, environnement et travail) - UMR_S 1085, F-35000 Rennes, France.
⁵ Takeda Development Center Americas, Inc., Cambridge, MA, USA.
⁶ Takeda Development Center Americas, Inc., 500 Kendal Street, Cambridge, MA 02142, USA.
⁷ Takeda Pharmaceuticals International AG, Zurich, Switzerland.
⁸ Haemnet, London, UK.

Abstract

Background: Real-world data assessing treatment outcomes in patients with hemophilia A in routine clinical practice are limited.

Objective: To evaluate the effectiveness and safety of octocog alfa in patients with moderate/severe hemophilia A receiving treatment in clinical practice.

Design: The international Antihemophilic Factor Hemophilia A Outcome Database study is an observational, noninterventional, prospective, multicenter study.

Methods: This planned interim data read-out was conducted following 7 years of observation of patients receiving octocog alfa (cut-off, 30 June 2020). The primary endpoint was joint health status, assessed by the Gilbert Score. Secondary endpoints included annualized bleeding rates (ABRs), Hemophilia Joint Health Score (HJHS), health-related quality of life, consumption, and safety. This post hoc analysis stratified data by hemophilia severity at baseline [moderate, factor VIII (FVIII) 1-5%; severe, FVIII <1%].

Results: Of the 711 patients in this analysis, 582 (82%) were receiving prophylaxis with octocog alfa at enrollment, and 498 (70%) had severe disease. Median Gilbert Scores were higher with on-demand therapy versus prophylaxis and scores were comparable in moderate and severe disease. In patients receiving prophylaxis, there was an improvement in HJHS Global Gait Score over 7 years of follow-up overall and in patients with severe disease. ABRs and annualized joint bleeding rates were low across all 7 years. An ABR of zero was reported in 34-56% of prophylaxis patients versus 20-40% in the on-demand group. ABRs were similar in severe and moderate disease. In total, 13/702 (1.9%) patients experienced 18 treatment-related adverse events.

Conclusion: These data demonstrate the long-term effectiveness and safety of octocog alfa in patients with moderate and severe hemophilia A, especially in those receiving prophylaxis. The high number of patients receiving on-demand treatment experiencing zero bleeds could be due to selection bias within the study, with patients with less severe disease more likely to be receiving on-demand treatment.

Trial registration: ClinicalTrials.gov: NCT02078427.

Keywords: bleeding; hemarthrosis; hemophilia A; observational study; recombinant factor VIII.

Associated data

ClinicalTrials.gov/NCT02078427