Esteves Pereira Michael, Bocksrucker Christoph, Kremer Hovinga Johanna Anna, Mueller Martin, Daskalakis Michael, Mansouri Taleghani Behrouz, Nagler Michael
Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, and University of Bern, Bern, Switzerland.
Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, and University of Bern, Bern, Switzerland; Department of Clinical Research, University of Bern, Bern, Switzerland.
Transfus Med Rev. 2021 Apr;35(2):125-134. doi: 10.1016/j.tmrv.2021.01.001. Epub 2021 Jan 12.
The treatment of patients with acquired hemophilia is challenging due to life-threatening hemorrhages, delayed response, and adverse effects to immunosuppressive agents. Even though immunoadsorption (IA) rapidly removes autoantibodies against factor VIII, this intervention's effectiveness is still a matter of debate. We aimed to study important outcomes of IA as adjunctive treatment in patients with acquired hemophilia. We performed comprehensive literature searches in MEDLINE and EMBASE databases. Clinical and laboratory data of all patients treated in our institution were additionally included. Literature searching yielded 498 records, of which 10 studies describing 106 patients were finally included. The number of patients varied from 1 to 65, and patients' ages ranged between 14 and 89. Treatment criteria in most patients were (1) failed response to immunosuppressive treatment alone, and/or (2) uncontrollable bleeding episodes, and/or (3) high inhibitor titer. Methodological quality was moderate. The number of IA sessions varied from 1 to 24. Within our institution, 12 patients have been treated since 2002; median age was 76 years (range 34-86); median titer of factor VIII inhibitor was 20 Bethesda units (range 3-214). Pooled estimates, modeling a random-effect binominal distribution incorporating the Freeman-Tukey double arcsine transformation, were 86% in case of factor VIII recovery (95% confidence interval 76%-94%), 95% for reduction of factor VIII inhibitor (83%, 100%), and 7% in case of death (0%, 18%). Our data suggest that IA might be a beneficial adjunctive treatment in patients with high-risk acquired hemophilia, but future studies shall confirm this observation.
获得性血友病患者的治疗具有挑战性，原因在于危及生命的出血、反应延迟以及免疫抑制剂的不良反应。尽管免疫吸附（IA）能迅速清除针对凝血因子VIII的自身抗体，但这种干预措施的有效性仍存在争议。我们旨在研究IA作为获得性血友病患者辅助治疗的重要结果。我们在MEDLINE和EMBASE数据库中进行了全面的文献检索。此外，还纳入了在我们机构接受治疗的所有患者的临床和实验室数据。文献检索共获得498条记录，最终纳入了10项描述106例患者的研究。患者数量从1例到65例不等，年龄在14岁至89岁之间。大多数患者的治疗标准为：（1）单独免疫抑制治疗无效，和/或（2）出血发作无法控制，和/或（3）抑制物滴度高。方法学质量中等。IA治疗次数从1次到24次不等。自2002年以来，我们机构共治疗了12例患者；中位年龄为76岁（范围34 - 86岁）；凝血因子VIII抑制物的中位滴度为20贝塞斯达单位（范围3 - 214）。采用随机效应二项分布并结合Freeman - Tukey双反正弦变换进行汇总估计，凝血因子VIII恢复的比例为86%（95%置信区间76% - 94%），凝血因子VIII抑制物降低的比例为95%（83%，100%），死亡比例为7%（0%，18%）。我们的数据表明，IA可能是高危获得性血友病患者有益的辅助治疗方法，但未来的研究应证实这一观察结果。
