Patients with haemophilia A with inhibitors in China: a national real-world analysis and follow-up.

Abstract

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 (10·5-39·5) 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 76·2% and 29·2% of patients for acute bleeding. Only 22·3% of patients underwent immune tolerance induction (ITI) treatment, of whom 64·9% achieved negative inhibitor titre. In patients who did not undergo ITI, the inhibitors turned negative in 17·7%, and patients with low peak inhibitor titre were more likely to acquire negative titre spontaneously (odds ratio 11·524, 95% confidence interval 5·222-25·432; P = 0·000). We recorded that 3·2% of the patients died from haemophilia-related life-threatening bleeding.

• Authors: Xueqing Dou, Wei Liu, Man-Chiu Poon, Xinsheng Zhang, Jingsheng Wu, Xiaojing Zeng, Runhui Wu, Qun Hu, Chengping Li, Xiaomin Wang, Xuewen Song, Lingling Chen, Lei Zhang, Feng Xue, Renchi Yang
• Journal: British journal of haematology (Br J Haematol) Vol. 192 Issue 5 Pg. 900-908 (03 2021) ISSN: 1365-2141 [Electronic] England
• PMID: 33534930 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2021 British Society for Haematology and John Wiley & Sons Ltd.

Chemical References

• Antibodies, Bispecific
• Antibodies, Monoclonal, Humanized
• Blood Coagulation Factors
• Hemostatics
• Isoantibodies
• Recombinant Proteins
• prothrombin complex concentrates
• emicizumab
• Factor VIII
• recombinant FVIIa
• Factor VIIa

Topics

• Adolescent
• Adult
• Aged
• Antibodies, Bispecific (therapeutic use)
• Antibodies, Monoclonal, Humanized (therapeutic use)
• Blood Coagulation Factors (therapeutic use)
• Child
• Child, Preschool
• China (epidemiology)
• Factor VIII (immunology, therapeutic use)
• Factor VIIa (therapeutic use)
• Follow-Up Studies
• Hemophilia A (complications, drug therapy, epidemiology, immunology)
• Hemorrhage (drug therapy, etiology)
• Hemostatics (supply & distribution, therapeutic use)
• Humans
• Isoantibodies (immunology)
• Male
• Middle Aged
• Recombinant Proteins (therapeutic use)
• Registries
• Severity of Illness Index
• Young Adult