Outcome of acquired haemophilia in France: the prospective SACHA (Surveillance des Auto antiCorps au cours de l'Hemophilie Acquise) registry
Outcome of acquired haemophilia in France: the prospective SACHA (Surveillance des Auto antiCorps au cours de l'Hemophilie Acquise) registry
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DOI：
10.1111/hae.12138
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发表时间：
2013-07-01
期刊：
影响因子：
3.9
通讯作者：
Levesque, H.
中科院分区：
文献类型：
--
作者：
Borg, J. Y.;Guillet, B.;Levesque, H.
获得性血友病A（AHA）虽然非常罕见，但可导致严重出血，这可能是致命的。自身抗体发展的根本原因尚未完全了解。治疗目标是控制出血和根除自身抗体。在我们研究的时候，对于AHA的标准治疗策略还没有达成共识。既往数据主要为回顾性或来自单中心队列。我们对法国AHA患者进行了一项前瞻性、对照、基于登记的研究。前瞻性法国登记研究（Surveillance des Auto antiCorps Au cours de l 'Hemophilie Acquise [SACHA]）收集了82例患者的患病率、临床病程、疾病相关性和止血治疗和自身抗体根除结局的数据，并进行了1年随访。与早期研究相似，老年人AHA的患病率较高，三分之二的患者年龄> 70岁。大约一半的AHA病例与基础疾病有关，最常见的是年轻和老年患者的自身免疫性疾病和癌症。46%的患者最初接受了止血治疗。22/27例（81%）rFVIIa治疗患者和所有6例接受pd-aPCC的患者的初始出血完全消退或改善。大多数患者（94%）接受免疫抑制剂治疗，61%（36/59）的患者在3个月时完全缓解，98%（50/51）的患者在1年时完全缓解。总体死亡率为33%：仅3例患者继发于出血，但10例患者继发于败血症。止血剂在控制AHA患者的出血方面是有效的。免疫抑制治疗应早期使用，但要谨慎，尤其是老年患者。
Although extremely rare, acquired haemophilia A (AHA) can cause severe bleeding, which may be fatal. The underlying causes of autoantibody development are not fully understood. Treatment goals are bleeding control and autoantibody eradication. At the time of our study, there was no consensus on a standard treatment strategy for AHA. Previous data were mainly retrospective or from single-centre cohorts. We conducted a prospective, controlled, registry-based study of patients with AHA in France. The prospective French registry (Surveillance des Auto antiCorps au cours de l'Hemophilie Acquise [SACHA]) collected data on prevalence, clinical course, disease associations and outcomes for haemostatic treatment and autoantibody eradication in 82 patients with a 1-year follow-up. Similar to earlier studies, the prevalence of AHA was higher in the elderly, with two thirds of patients aged >70years. Around half of AHA cases were associated with underlying disease, most commonly autoimmune disease and cancer in younger and older patients respectively. Haemostatic treatment was initially administered to 46% of patients. Complete resolution or improvement of initial bleeding occurred in 22/27 (81%) rFVIIa-treated patients and in all six cases receiving pd-aPCC. The majority of patients (94%) received immunosuppressive therapy, with complete remission at 3months in 61% (36/59) and in 98% (50/51) at 1year. Overall mortality was 33%: secondary to bleeding in only three patients but to sepsis in 10. Bypassing agents were effective at controlling bleeding in patients with AHA. Immunosuppressive therapy should be used early but with caution, particularly in elderly patients.