PB1268 - Clinical Outcomes with Octocog Alfa in Patients with Hemophilia A in China: 1-year of Follow-up of the AHEAD International Study

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Co-Author(s)

RW

Runhui Wu

Director and Physician in Chief
Beijing Children’s Hospital, Capital Medical University, Beijing, China
Beijing, Beijing, China (People's Republic)
ZL

Zhenyu Li

Professor
The Affiliated Hospital of Xuzhou Medical University, Xuzhou, China
Xuzhou, Jiangsu, China (People's Republic)
JS

Jing Sun

Physician
Nanfang Hospital, Nanfang Medical University, Guangzhou, China
Guangzhou, Guangdong, China (People's Republic)
XD

Xin Du

Chief Physician
Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University, Shenzhen, China
Shenzhen, Guangdong, China (People's Republic)
XZ

Xinsheng Zhang

Director
Shandong Blood Center, Jinan, China
Jinan, Shandong, China (People's Republic)
YW

Ying Wang

Physician
Shenzhen Children's Hospital, Shenzhen, China
Shenzhen, Guangdong, China (People's Republic)
QH

Qun Hu

Professor
Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, China
Wuhan, Hubei, China (People's Republic)
Rongfu Zhou, PhD，MD

Doctor
Nanjing Drum Tower Hospital, The Affiliated Hospital of Nanjing University Medical School, Nanjing, China
Nanjing, Jiangsu, China (People's Republic)

Presenting Author(s)

JG

Joan Gu

Associate Director of Biostatistics
Takeda Development Center Americas, Inc., Cambridge, MA, USA
Cambridge, Massachusetts, United States

Co-Author(s)

Randy Guerra, MD

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

Co-Author(s)

Pooja Oberai, MD MS (she/her/hers)

Global Medical Lead
Takeda Development Center Americas, Inc., Cambridge, MA, USA
new york, New York, United States

Co-Author(s)

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 international AHEAD study (NCT02078427) is assessing the long-term effectiveness and safety of octocog alfa and rurioctocog alfa pegol in patients with hemophilia A (HA) in real-world clinical practice.

Aims: To report clinical outcomes from 1 year follow-up in patients receiving octocog alfa from the AHEAD China subgroup.

Methods: Data were extracted from the 9th interim read-out (data cutoff: July 15, 2022) from AHEAD, a prospective, non-interventional, multicenter study involving patients with HA. The primary endpoint is joint health, evaluated by Gilbert score. Descriptive data for demographics, annualized bleeding rates (ABRs), annualized joint bleeding rates (AJBRs), and adverse events (AEs) are presented. A post hoc ABR/AJBR analysis using a generalized linear mixed-effects model (GLMM) with the negative binomial distribution was performed. Ethics committee and Human Genetic Resource Administration of China approval, and patient informed consent were obtained. Patient enrollment in China commenced in June 2020.

Results: Of the 168 patients enrolled (all male), 113 patients received prophylaxis, 53 on-demand therapy, 1 immune tolerance induction, and 1 unknown regimen. Mean±SD age (years) was 20.6±12.4 in patients with severe HA (n=80) and 22.6±14.3 in patients with moderate HA (n=85). ABRs/AJBRs were lower with prophylaxis versus on-demand therapy (Table 1). In patients receiving prophylaxis, ABRs/AJBRs were lower and proportion of patients with 0 ABRs/AJBRs were higher at year 1 versus screening. Bleeding rates in the GLMM model were reduced by >99% (Table 2). 39 AEs were experienced by 25 (14.9%) patients and 8 serious AEs by 8 (4.8%) patients (possibly treatment related: anti-FVIII antibody; unlikely to be treatment related: fracture, Henoch-Schonlein purpura; not treatment related: arthropathy, anal fistula, appendicitis, [all n=1]; intracranial hemorrhage, [n=2]).

Conclusion(s): Bleeding rates were lower with octocog alfa prophylaxis versus on-demand therapy, and an exploratory model-based analysis showed significantly reduced ABRs/AJBRs from screening to year 1 with both regimens, irrespective of HA severity.