Evidence map›Paper›PMID 40465201›Full record

ArticleAdvances in therapy2025

Disease Burden and Treatment Patterns Amongst Patients with Haemophilia A in China: A Retrospective Database Analysis Study.

Xuewen Song, Feng Xue, Lingling Chen, Donghui Yuan, Yifan Yang, Huiyuan Li, Lei Zhang, Shawn X Sun, Jia Zhong, Eric Wu and 1 more

Abstract read
In one paragraph

Article in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

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Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. The therapeutic landscape of inherited bleeding disorders in China.Research and practice in thrombosis and haemostasis · 2026
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors.

Xuewen SongState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China.
Feng XueState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China.
Lingling ChenState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China.
Donghui YuanState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China.
Yifan YangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China.
Huiyuan LiState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China.
Lei ZhangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China.
Shawn X SunTakeda Development Center Americas, Inc, Cambridge, MA, USA.
Jia ZhongAnalysis Group Inc., Beijing, China.
Eric WuAnalysis Group Inc., Beijing, China.
Renchi YangState Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Tianjin, 300020, China. rcyang@ihcams.ac.cn.

Funding

CAMS Innovation Fund for Medical Sciences 2021-I2M-1-003CAMS Innovation Fund for Medical Sciences 2022-I2M-C&T-A-015National Key Research and Development Program of China 2023YFC2507802
6 · The paper itself

Abstract

introductionDespite concerted efforts of healthcare agencies, haemophilia treatment coverage remains inadequate in China. This real-world study was conducted to understand patient characteristics, clinical and economic burden, and treatment patterns amongst patients with haemophilia A in China with the aim of improving patient outcomes and quality of life.

methodsTwo data sources, namely the National Haemophilia Registry and Institute of Haematology and Blood Diseases Hospital, were used to analyse the disease burden, treatment patterns and economic burden of haemophilia A in China. The economic burden was assessed from 2017 to 2019.

resultsOverall, 3164 male patients with haemophilia A (mean age 21.5 years) were analysed. Almost half (48.3%) of the patients were children or adolescents; amongst them, 13.5% were enrolled from Guangdong and 10.7% from Hebei. Furthermore, 58.9% of the patients had severe haemophilia A. The most common sites for the first bleeding and bleeding during disease course were skin/mucous membrane (40.0%) and joints (77.9%), respectively. Plasma-derived factor VIII constituted the most frequently used therapy (45.8% of patients) and only 30.1% of patients used a prophylactic treatment. The hospitalisation rate increased from 8% in 2017 to 21% in 2019, whereas 63% to 75% of patients had outpatient visits during each year. The total annual cost of treating haemophilia (hospitalisation plus outpatient) including the cost of FVIII replacement per patient was 57,439.4 CNY.

conclusionSuboptimal disease management and low utilisation of prophylaxis are the main challenges for patients with haemophilia A in China. Therefore, continued efforts to improve disease awareness and treatment accessibility are required.

Indexed as

Cost of IllnessFactor VIIIHemophilia AAdolescentAdultChildChild, PreschoolChinaDatabases, FactualHemorrhageHospitalizationHumansInfantMaleMiddle AgedQuality of LifeFactor VIIIBleeding rateDisease burdenFactor VIII proteinHaemophilia AHospitalisationOutpatient costsTreatment patterns

Identifiers

PMID40465201
PMCPMC12313731

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