PB0632 - Quality of life 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)

Kate Khair, RN, PhD

Director of Research
Haemnet, London, United Kingdom
London, England, United Kingdom

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
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)
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
Manuel Carcao, MD, FRCPC

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

Background: Hemophilia A (HA) significantly impacts patients’ quality of life (QoL). The international AHEAD study (NCT02078427) is evaluating long-term outcomes in patients with severe/moderate HA treated with octocog alfa or rurioctocog alfa pegol in routine clinical practice.

Aims: To assess QoL 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 AHEAD study (104 study centers). The primary endpoint is overall Gilbert score (pain, bleeding, and physical examination). Secondary endpoints include annualized bleeding rates, safety, and QoL (assessed by Short-Form 12 version 2 [SF-12v2] and EuroQol-5 dimensions [EQ-5D] questionnaires in adults). This analysis assesses QoL scores over the first 5 years. Ethics committee approval and informed consent were obtained.

Results: 905 patients were enrolled. Mean ± SD SF-12v2 physical functioning (T-score) ranged from 47.5 ± 10.5 to 49.7 ± 9.8 in patients receiving prophylaxis (n=78-218, years 1-5) versus 42.5 ± 11.5 to 46.3 ± 9.6 in those treated on-demand (n=10-49, years 1-5). SF-12v2 physical functioning and general health T-scores stratified by prophylactic treatment and HA severity are shown in Table 1. Mean ± SD EQ-5D visual analog scale (VAS) scores were higher in patients receiving prophylaxis (79.9 ± 16.5-82.5 ± 14.5 [n=78-214]) compared with on-demand treatment (67.9 ± 17.4-77.8 ± 13.2 [n=10-48]). Of patients receiving prophylaxis (years 1-5), 59.6%-62.2% reported no problems walking and 44.5%-53.9% experienced no pain or discomfort, compared with 41.3%-61.2% and 30.0%-34.7%, respectively, of those treated on-demand (years 1-5). EQ-5D VAS scores stratified by prophylactic treatment and hemophilia severity are shown in Table 2.

Conclusion(s): These findings demonstrate improved QoL for patients receiving prophylaxis, who experienced less physical disability and pain compared with those receiving on-demand treatment. QoL scores were similar in patients receiving standard and PK-guided prophylaxis.