Acquired haemophilia A in Finland: A nationwide study of incidence, treatment and outcomes

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2024-06-28 DOI:10.1111/hae.15037

Vuokko Nummi, Leena Hiltunen, Timea Szanto, Eira Poikonen, Anna-Elina Lehtinen

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Abstract

Introduction

Acquired haemophilia A (AHA) is a bleeding disorder caused by autoantibody development against factor VIII (FVIII). Studies on AHA have mainly focused on patients treated at specialist centres.

Aim

To determine the incidence, clinical characteristics and outcomes of AHA in an unselected population-based patient cohort from Finland.

Methods

This retrospective observational cohort comprised all cases diagnosed with AHA in Finland between 2006 and 2019. Patients were identified by the two central laboratories performing FVIII antibody testing in Finland, the Finnish Red Cross Blood Service and HUSLAB. Clinical details were collected from all hospitals and healthcare units where patients were treated. This study was performed in conjunction with the AHA in the Nordics study.

Results

The median incidence of AHA was 0.65 per million per year (range 0.19-1.27). Fifty-five patients were identified, with a median age of 76 years and an even sex ratio (51% women). When diagnosed, all had bleeding symptoms with severe bleeds in 92%. First-line immunosuppressive treatment regimens included steroid monotherapy in 31% of cases, steroids and a cytotoxic agent in 51% and a rituximab-based regimen in 16%. Clinical remission was achieved in 71% of cases, and 15% had relapses. Mortality was 13% for bleeds and 9% for treatment-related infections. Overall survival was 64% for 1 year and 56% for 2 years after diagnosis.

Conclusions

In a nationwide population-based cohort study, we discovered a lower incidence of AHA than previously reported. Mortality among patients with AHA was high, calling for the consideration of updated treatment strategies.

Abstract Image

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芬兰后天性血友病 A：关于发病率、治疗和结果的全国性研究。

导言：获得性血友病 A（AHA）是一种由针对因子 VIII（FVIII）的自身抗体引起的出血性疾病。关于AHA的研究主要集中于在专科中心接受治疗的患者。目的：在芬兰一个未经选择的人群患者队列中确定AHA的发病率、临床特征和预后：该回顾性观察队列包括 2006 年至 2019 年期间芬兰确诊为 AHA 的所有病例。患者由芬兰两家进行 FVIII 抗体检测的中央实验室（芬兰红十字血液服务机构和 HUSLAB）确定。从患者接受治疗的所有医院和医疗单位收集临床细节。这项研究与北欧 AHA 研究同时进行：AHA的中位发病率为每年每百万人中有0.65人（范围为0.19-1.27）。共发现 55 名患者，中位年龄为 76 岁，男女比例均衡（女性占 51%）。确诊时，所有患者都有出血症状，92%的患者出血严重。一线免疫抑制治疗方案包括：31%的病例采用类固醇单药治疗，51%的病例采用类固醇和细胞毒药物治疗，16%的病例采用利妥昔单抗治疗方案。71%的病例获得了临床缓解，15%的病例复发。13%的患者因出血而死亡，9%的患者因治疗相关感染而死亡。确诊后1年和2年的总生存率分别为64%和56%：在一项基于全国人口的队列研究中，我们发现 AHA 的发病率低于之前的报道。AHA患者的死亡率很高，因此需要考虑更新治疗策略。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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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.