雷帕霉素治疗难治性获得性血友病A1例并文献复习

田长玉; 陈苏宁; 王兆钺

doi:10.13201/j.issn.1004-2806.2016.03.015

雷帕霉素治疗难治性获得性血友病A1例并文献复习

- 1 苏州大学附属第一医院, 江苏省血液研究所;
- 血液学协同创新中心(江苏苏州, 215006)
基金项目:
江苏省科教兴卫工程-临床医学中心(No:ZX201102)

江苏省血液病临床医学研究中心(No:江苏省科技厅生命健康专项-BL2012005)

详细信息

通讯作者: 陈苏宁,E-mail:chensuning@sina.com

中图分类号: R554.1

收稿日期: 2015-11-03

Rapamycin in the treatment of refractory acquired hemophilia A:a case report and literature review

TIAN Changyu CHEN Suning WANG Zhaoyue^1,2,

- Jiangsu Institute of Hematology, the First Affiliated Hospital of Soochow University, Suzhou, 215006, China

More Information

Corresponding author: , E-mail: chensuning@sina.com

Received Date: 03 November 2015

摘要

摘要: 目的: 难治性获得性血友病A(AHA)对激素与常用的免疫抑制剂无反应,出血危险性大,我们试用雷帕霉素治疗1例难治性AHA患者。方法: 用全自动血凝仪测定凝血常规以及相关凝血因子,Bethesda试验检测因子Ⅷ抑制物。结果: 该患者的凝血因子Ⅷ活性仅为1%,因子Ⅷ抑制物效价达87.5 Bethesda单位。该患者对激素与硫唑嘌呤治疗无反应,2次标准剂量的美罗华治疗仅暂时有效,表现为顽固性AHA;改用雷帕霉素(2 mg/d,3个月后减为1 mg/d,疗程6个月)后出血完全停止,凝血指标亦恢复正常,但停药后复发。重新给予雷帕霉素后再次缓解,逐渐减量后停药,随访1年中凝血常规检查维持在正常范围。结论: 雷帕霉素是一种安全、有效的新型免疫抑制剂,本文首次报道1例顽固性AHA患者在对激素与常用的免疫抑制剂无效时改用雷帕霉素后治愈。
- 获得性血友病,难治性 /
- 抗FⅧ的自身抗体 /
- 雷帕霉素
Abstract: Objective: Patients with refractory acquired hemophilia A (AHA) fail to respond to corticosteroids and immunosuppressive agents and have severe bleeding risks.We gave a refractory AHA patient a trial prescription of rapamycin.Method: Coagulation tests as well as activities of related coagulation factors were measured.FⅧ inhibitor was titled with Bethesda method.Result: The plasma FⅧ activity of the patient was only 1%,and FⅧ inhibitor title was 87.5 Bethesda units.He did not respond to initial immunosuppressive therapy,and twice rituximab treatment with standard dosage exerted a short effect,suggesting refractory AHA.We tried to treat him with rapamycin (2 mg/d for 3 months,and then 1 mg/d for other 3 months),his bleeding episodes disappeared with normal hemostasis restored.After a second course of rapamycin,the patient had completely recovered,and remained in good health one year after discontinuation of the drug.Conclusion: Rapamycin is a new safe and effective immunosuppressive agent.It is the first report that a patient was cured with rapamycin who was refractory to standard immunosuppressive treatment.
- refractory acquired hemophilia /
- autoantibody to FⅧ /
- rapamycin

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