PB0666 - Clinical outcomes following treatment with octocog alfa in patients with hemophilia A: 9-year interim read-out of the real-world AHEAD international study

Monday, June 26, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Manuel Carcao, MD, FRCPC

Professor
Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Canada
Toronto, Ontario, Canada

Co-Author(s)

Margareth C. Ozelo, MD, PhD (she/her/hers)

Professor
Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil
Campinas, Sao Paulo, Brazil
Cedric Hermans, MD, PhD (he/him/his)

Head Haemostasis and Thrombosis Unit
St-Luc University Hospital
Brussels, Brussels Hoofdstedelijk Gewest, Belgium
Kate Khair, RN, PhD

Director of Research
Haemnet, London, United Kingdom
London, England, United Kingdom
Randy Guerra, MD

Global Medical Lead
Takeda Development Center Americas, Inc., Cambridge, MA, USA
Cambridge, Massachusetts, United States
JG

Joan Gu

Associate Director of Biostatistics
Takeda Development Center Americas, Inc., Cambridge, MA, USA
Cambridge, Massachusetts, United States
Pooja Oberai, MD MS (she/her/hers)

Global Medical Lead
Takeda Development Center Americas, Inc., Cambridge, MA, USA
new york, New York, United States
Renchi Yang, MD (he/him/his)

Professor
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China
Tianjin, Tianjin, China (People's Republic)

Background: The ongoing, international AHEAD study (NCT02078427) evaluates the long term outcomes in patients with severe/moderate hemophilia A (HA) treated with octocog alfa or rurioctocog alfa pegol in routine clinical practice.

Aims: To characterize clinical outcomes in patients with moderate/severe HA receiving standard or pharmacokinetic (PK)-guided prophylaxis or on-demand treatment with octocog alfa.

Methods: This is a 9-year interim read-out (data cutoff: July 15, 2022) of the prospective, noninterventional, multicenter, real-world AHEAD study (104 study centers). Patients have been followed for anywhere between 1-9 years. The primary endpoint is overall Gilbert score (pain, bleeding, and physical examination). Secondary endpoints include annualized bleeding rates (ABR) and annualized joint bleeding rates (AJBRs), quality of life, and safety. Ethics committee approval and informed consent were obtained.

Results: Of 905 patients enrolled, 310 had moderate HA (factor VIII [FVIII] 1–≤5%), 591 had severe HA (FVIII ≤1%), and 4 had no severity grading available. In total, 722 (80.0%) patients received prophylaxis:(standard prophylaxis, n=450 [62.3%]; PK-guided prophylaxis, n=81 [11.2%]; unspecified prophylaxis, n=191 [26.5%]), 164 (18.1%) received on-demand treatment and 18 (2.0%) received immune tolerance induction. Mean ± SD patient age was 19.1 ± 15.7 years, 903 (99.8%) patients were male, and 517 (57.1%) were white. Average Gilbert scores (all joints) (Table 1) were lower in patients receiving prophylaxis compared with on-demand treatment. ABRs (Table 2) were consistently lower, and zero bleed rates (Table 2) were consistently higher, in patients receiving prophylaxis versus on-demand therapy. Adverse events (AEs) were experienced by 465 (51.4%) patients, the majority of which were infections. Serious AEs were reported in 161 (17.8%) patients. A total of 35 AEs (3.9%) were related to octocog alfa, of which 15 were serious AEs.

Conclusion(s): These findings provide evidence for the improved effectiveness of octocog alfa prophylaxis compared with on-demand treatment, as well as demonstrating treatment safety in routine clinical practice.