PB0664 - Demographic and Baseline Clinical Characteristics of Patients With Hemophilia A in China: Real-world Data From the AHEAD International Study

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

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, Jiangsu, China (People's Republic)
JS

Jing Sun

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

Xin Du

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

Xinsheng Zhang

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

Ying Wang

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

Qun Hu

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

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

Joan Gu

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

Presenting Author(s)

Randy Guerra, MD

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

Co-Author(s)

LT

Leilei Tang

Global Medical Lead
Takeda Pharmaceuticals International AG
Zürich, Zurich, Switzerland
RY

Renchi Yang

Director
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Hematological Disorders, Institute of Hematology & Blood Diseases Hospital, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin
Tianjin, Tianjin, China (People's Republic)

Background: The international AHEAD study (NCT02078427, started in 2011) is assessing the long-term effectiveness and safety of octocog alfa and rurioctocog alfa pegol in patients with hemophilia in real-world clinical practice.

Aims: To report baseline demographic and clinical characteristics of patients from the AHEAD China subgroup.

Methods: AHEAD is a prospective, non-interventional, multicenter study conducted in 22 countries. Patients of any age with hemophilia A are eligible for inclusion. The primary endpoint is joint health outcomes evaluated by Gilbert score (pain, bleeding, physical exam). Secondary endpoints include annualized bleeding rates (ABRs), annualized joint bleeding rates (AJBRs), Hemophilia Joint Health Score (HJHS), and safety. Ethics committee approval and informed consent were obtained. Patient enrollment in China commenced in June 2020. Data were extracted from the 8th interim read-out (data cutoff: July 01, 2021).

Results: The AHEAD China subgroup included 141 patients (mean [SD] age, 20.3 [12.0] years) who received treatment with octocog alfa: 95 (67.4%) received prophylaxis (mean [SD] age, 17.2 [11.3] years) and 46 (32.6%) received on-demand treatment (mean [SD] age, 26.8 [10.9] years). All patients were male and of Asian ethnicity. Hemophilia A severity, history of FVIII inhibitor detection, and number of target joints at baseline are shown in Table 1. At screening, 72 prophylaxis patients (75.8%) and 39 on-demand patients (84.8%) had experienced bleeding events, which occurred up to 12 months prior to screening. Mean ABRs and AJBRs at baseline were numerically lower in patients receiving prophylaxis compared with on-demand treatment (Table 2). Gilbert scores and HJHS: Global Gait Scores at baseline were consistent across treatment groups (Table 2).

Conclusion(s): These real-world data from the AHEAD China subgroup demonstrate lower bleeding rates in patients receiving prophylaxis compared with on-demand treatment at baseline. These data will provide a basis for future analyses observing the clinical outcomes of patients and standard of care in China.