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

1Beijing Children’s Hospital, Beijing, Beijing, China (People's Republic), 2The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, China (People's Republic), 3Nanfang Hospital, Nanfang Medical University, Guangzhou, Guangdong, China (People's Republic), 4Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University, Shenzhen, Guangdong, China (People's Republic), 5Shandong Blood Center, Jinan, Shandong, China (People's Republic), 6Shenzhen Children's Hospital, Shenzhen, Guangdong, China (People's Republic), 7Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, Hubei, China (People's Republic), 8Nanjing Drum Tower Hospital, The Affiliated Hospital of Nanjing University Medical School, Nanjing, Jiangsu, China (People's Republic), 9Takeda Development Center Americas, Inc., Cambridge, Massachusetts, United States, 10Takeda Pharmaceuticals International AG, Zürich, Zurich, Switzerland, 11State 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)

Abstract Number: PB0664

Meeting: ISTH 2022 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

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.

Table

Table 1. Clinical Characteristics at Screening

Table

Table 2. Gilbert Score, HJHS, and Bleeding Rates at Screening

To cite this abstract in AMA style:

Wu R, Li Z, Sun J, Du X, Zhang X, Wang Y, Hu Q, Zhou R, Gu J, Guerra R, Tang L, Yang R. Demographic and Baseline Clinical Characteristics of Patients With Hemophilia A in China: Real-world Data From the AHEAD International Study [abstract]. https://abstracts.isth.org/abstract/demographic-and-baseline-clinical-characteristics-of-patients-with-hemophilia-a-in-china-real-world-data-from-the-ahead-international-study/. Accessed October 1, 2023.

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