PB0685 - A Retrospective Database Analysis Study to Evaluate Disease Burden and Treatment Patterns Among Patients With Hemophilia A in China

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

XS

Xuewen Song

Doctor
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; Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University, Zhengzhou, Henan
Tianjin, Tianjin, China (People's Republic)
WL

Wei Liu

Doctor
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)
FX

Feng Xue

Doctor
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)
YY

Yifan Yang

Doctor
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)

Presenting Author(s)

Shawn X. Sun

Director, Global Evidence and Outcomes
Takeda Development Center Americas, Inc.
Cambridge, Massachusetts, United States

Co-Author(s)

JZ

Jia Zhong

Epidemiologist
Analysis Group, Inc.
Beijing, Beijing, China (People's Republic)
YL

Yi Liu

Doctor
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)
JS

Jun Shi

Doctor
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)
LZ

Lei Zhang

Doctor
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)
EW

Eric Wu

Health Economist
Analysis Group, Inc.
Beijing, Beijing, China (People's Republic)
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 clinical and economic burden of hemophilia A (HA) in China is not well characterized. A greater understanding of this patient population should help to improve quality of care and optimize patient outcomes.

Aims: To describe patient characteristics, treatment patterns, and the clinical and economic burden among patients with HA in China.

Methods: This retrospective database analysis utilized data from the hospital-based National Hemophilia Registry (NHR) and healthcare resource utilization data from the Institute of Hematology and Blood Disease Hospital (IHBDH). Male patients with HA who had been enrolled in the NHR database since January 01, 2015 and had complete patient characteristic information were selected for evaluation of patient characteristics, treatment patterns, and clinical burden (Group 1). Data cutoff: December 31, 2019. Patients with HA who had been enrolled in the NHR database since January 01, 2015, who, between 2017–2019, were continuously enrolled for ≥1 year in a special disease health plan in Tianjin and incurred ≥1 inpatient or outpatient visit at the IHBDH were selected for evaluation of economic burden (Group 2).

Results: Group 1 included 3164 patients. Mean (SD) age at enrollment was 21.5 (15.5) years and 58.9% had severe HA (factor VIII < 1 IU/dl). Mean (SD) time from initial diagnosis to replacement therapy was 2.1 (5.7) years (n=1340). During the study period, 30.1% of patients received prophylaxis; at the index visit, 71.1% were only receiving on-demand treatment (Table 1). In total, 2373/3045 patients (77.9%) experienced joint bleeding over the course of the disease. Annual bleeding rates are presented in Table 1. Group 2 included 118 patients. Mean (SD) total outpatient and hospitalization costs/patient/year were ¥56,736.8 (¥79,517.9) and ¥702.6 (¥1738.4), respectively.

Conclusion(s): These findings highlight the burden of illness and varied disease management experienced by patients with HA in China.