机构:[1]State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, CAMS Center for Stem Cell Medicine, PUMC Department of Stem Cell and Regenerative Medicine, Tianjin, China,[2]Departments of Medicine, Pediatrics and Oncology, University of Calgary, Cumming School of Medicine and Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Medical Centre, Alberta Health Services, Calgary, AB, Canada,[3]Shandong Blood Center, Jinan,[4]First Affiliated Hospital, Chinese University of Science and Technology, Hefei,[5]The Affiliated Hospital of Guizhou Medical University, Guiyang,[6]Beijing Children’s Hospital, Capital Medical University, Beijing,首都医科大学附属北京儿童医院[7]Department of Pediatric Hematology, Tongji Hospital of Tongji Medical College, Huazhong University of Sciences and Technology, Wuhan,华中科技大学同济医学院附属同济医院[8]First Affiliated Hospital of Kunming Medical University, Kunming, Urumchi, China昆明医科大学附属第一医院[9]Xinjiang Uygur Autonomous Region People’s Hospital, Urumchi, China
The development of alloantibodies (inhibitors) against coagulation factor VIII (FVIII) is the most serious complication of FVIII replacement therapy in patients with haemophilia A (HA). We carried out a nationwide study focussing on patients with HA with inhibitors in China to evaluate the condition and management of this population. The study retrospectively analysed patient characteristics, clinical history, manifestation, treatment strategy as well as individual haemophilia care of 493 patients with inhibitors (466 with severe HA and 27 with non-severe HA) registered all over China. The median (interquartile range) age at diagnosis of FVIII inhibitors was 13 (5-28) years in patients with severe HA and 24 (10 center dot 5-39 center dot 5) years in patients with non-severe HA. Most patients (85%) had high-titre inhibitors. Prothrombin complex concentrate and recombinant activated coagulation factor VII were used respectively in 76 center dot 2% and 29 center dot 2% of patients for acute bleeding. Only 22 center dot 3% of patients underwent immune tolerance induction (ITI) treatment, of whom 64 center dot 9% achieved negative inhibitor titre. In patients who did not undergo ITI, the inhibitors turned negative in 17 center dot 7%, and patients with low peak inhibitor titre were more likely to acquire negative titre spontaneously (odds ratio 11 center dot 524, 95% confidence interval 5 center dot 222-25 center dot 432; P = 0 center dot 000). We recorded that 3 center dot 2% of the patients died from haemophilia-related life-threatening bleeding.
基金:
CAMS Innovation Fund for Medical Sciences [2017-I2M-3-018, 2016-I2M-1-002]; Non-profit Central Research Institute Fund of CAMS [2019XK320075]
第一作者机构:[1]State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, CAMS Center for Stem Cell Medicine, PUMC Department of Stem Cell and Regenerative Medicine, Tianjin, China,
通讯作者:
通讯机构:[1]State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, CAMS Center for Stem Cell Medicine, PUMC Department of Stem Cell and Regenerative Medicine, Tianjin, China,[*1]State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, CAMS Center for Stem Cell Medicine, PUMC Department of Stem Cell and Regenerative Medicine, 288 Nanjing Road, Tianjin 300020, PR China.
推荐引用方式(GB/T 7714):
Dou Xueqing,Liu Wei,Poon Man-Chiu,et al.Patients with haemophilia A with inhibitors in China: a national real-world analysis and follow-up[J].BRITISH JOURNAL OF HAEMATOLOGY.2021,192(5):900-908.doi:10.1111/bjh.17322.
APA:
Dou, Xueqing,Liu, Wei,Poon, Man-Chiu,Zhang, Xinsheng,Wu, Jingsheng...&Yang, Renchi.(2021).Patients with haemophilia A with inhibitors in China: a national real-world analysis and follow-up.BRITISH JOURNAL OF HAEMATOLOGY,192,(5)
MLA:
Dou, Xueqing,et al."Patients with haemophilia A with inhibitors in China: a national real-world analysis and follow-up".BRITISH JOURNAL OF HAEMATOLOGY 192..5(2021):900-908