Real-World Treatment Patterns and Long-Term Clinical Outcomes in Thai Patients With Acquired Haemophilia A.

IF 3 2区医学 Q2 HEMATOLOGY Haemophilia Pub Date : 2025-03-18 DOI:10.1111/hae.70028

Nonthakorn Hantrakun, Piangrawee Niprapan, Pakinee Tuntivate, Nuttanun Wongsarikan, Lalita Norasetthada, Adisak Tantiworawit, Ekarat Rattarittamrong, Chatree Chai-Adisaksopha, Thanawat Rattanathammethee, Sasinee Hantrakool, Pokpong Piriyakhuntorn, Teerachat Punnachet

{"title":"Real-World Treatment Patterns and Long-Term Clinical Outcomes in Thai Patients With Acquired Haemophilia A.","authors":"Nonthakorn Hantrakun, Piangrawee Niprapan, Pakinee Tuntivate, Nuttanun Wongsarikan, Lalita Norasetthada, Adisak Tantiworawit, Ekarat Rattarittamrong, Chatree Chai-Adisaksopha, Thanawat Rattanathammethee, Sasinee Hantrakool, Pokpong Piriyakhuntorn, Teerachat Punnachet","doi":"10.1111/hae.70028","DOIUrl":null,"url":null,"abstract":"Introduction: Data regarding long-term clinical outcomes in Asian patients with acquired haemophilia A (AHA) was limited.Aim: This study aimed to evaluate the effectiveness of current treatments and their outcomes in a real-world setting among Thai patients with AHA.Methods: This was a retrospective cohort study conducted at a university-based hospital. Patients' characteristics, treatment patterns and disease outcomes were collected. Univariate and multivariate Gray's competing risk analyses were used to examine the factors related to the time to disease response.Results: From 2009 to 2022, 69 AHA patients with a median age of 68 years (range 36-97) were enrolled. The majority of cases were characterised by the absence of an underlying aetiology (82.6%) and presented as major bleeding (71.0%). As first-line treatment, 79.7% were treated with steroid monotherapy, and 13.0% received a combination of steroid and rituximab. Thirty-one patients (44.9%) received at least one dose of haemostatic agents. After a median time to follow-up of 24.9 months (interquartile range 1.6-78.5), 41 patients (59.4%) attained first disease remission. Factor VIII below 1 IU/dL and the combination of steroid and rituximab were associated with time to disease remission, with subdistribution hazard ratio of 0.3 (95% confidence interval [CI], 0.1-0.7) and 5.2 (95% CI, 2.0-13.4), respectively. The most common complication in this cohort was infection (40.6%).Conclusion: The combination of steroid and rituximab demonstrated efficacy in the management of AHA. In addition, infectious complications were a significant concern when treating AHA patients.","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0000,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Haemophilia","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/hae.70028","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Introduction: Data regarding long-term clinical outcomes in Asian patients with acquired haemophilia A (AHA) was limited.

Aim: This study aimed to evaluate the effectiveness of current treatments and their outcomes in a real-world setting among Thai patients with AHA.

Methods: This was a retrospective cohort study conducted at a university-based hospital. Patients' characteristics, treatment patterns and disease outcomes were collected. Univariate and multivariate Gray's competing risk analyses were used to examine the factors related to the time to disease response.

Results: From 2009 to 2022, 69 AHA patients with a median age of 68 years (range 36-97) were enrolled. The majority of cases were characterised by the absence of an underlying aetiology (82.6%) and presented as major bleeding (71.0%). As first-line treatment, 79.7% were treated with steroid monotherapy, and 13.0% received a combination of steroid and rituximab. Thirty-one patients (44.9%) received at least one dose of haemostatic agents. After a median time to follow-up of 24.9 months (interquartile range 1.6-78.5), 41 patients (59.4%) attained first disease remission. Factor VIII below 1 IU/dL and the combination of steroid and rituximab were associated with time to disease remission, with subdistribution hazard ratio of 0.3 (95% confidence interval [CI], 0.1-0.7) and 5.2 (95% CI, 2.0-13.4), respectively. The most common complication in this cohort was infection (40.6%).

Conclusion: The combination of steroid and rituximab demonstrated efficacy in the management of AHA. In addition, infectious complications were a significant concern when treating AHA patients.

查看原文

微信好友朋友圈 QQ好友复制链接

本刊更多论文

泰国后天性血友病 A 患者的实际治疗模式和长期临床疗效。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文去求助

来源期刊

Haemophilia 医学-血液学

CiteScore

6.50

自引率

28.20%

发文量

226

审稿时长

3-6 weeks

期刊介绍： Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include: clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI replacement therapy for clotting factor deficiencies component therapy in the developing world transfusion transmitted disease haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics nursing laboratory diagnosis carrier detection psycho-social concerns economic issues audit inherited platelet disorders.