Clinical observation of cyclosporine combined with low-dose HA regimen in the treatment of refractory paroxysmal nocturnal hemoglobinuria
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摘要: 目的:观察环孢素A联合小剂量HA方案(高三尖杉酯碱+阿糖胞苷)治疗难治性阵发性睡眠性血红蛋白尿症(PNH)的临床疗效及毒副反应。方法:采用环孢素A联合小剂量HA方案治疗15例难治性PNH,分析患者的临床疗效及不良反应。结果:4个疗程后,15例难治性PNH的疗效为14例(93.33%)有效,其中2例(13.33%)达到近期临床痊愈,4例(26.67%)达到近期临床缓解,5例(33.33%)达到近期明显进步,3例(20.00%)达到近期进步;另外1例(6.67%)无效。所有患者均无严重的骨髓抑制及毒副反应。结论:环孢素A联合小剂量HA方案对难治性PNH的治疗安全、有效,毒副反应可耐受。
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关键词:
- 环孢素A /
- 小剂量HA方案 /
- 难治性阵发性睡眠性血红蛋白尿症
Abstract: Objective:To observe the clinical efficacy and adverse events of cyclosporine A combined with low-dose HA regimen(homoharringtonine + cytarabine) in the treatment of refractory paroxysmal nocturnal hemoglobinuria(PNH).Method:Fifteen patients with refractory PNH were treated with cyclosporine A combined with low-dose HA regimen.We analyzed the clinical efficacy and adverse events of patients.Result:After 4 courses of treatment,15 patients with refractory PNH were evaluated.A total of 14 cases(93.33%) were effective,of which 2 cases(13.33%) achieved clinical recovery in the near future,4 cases(26.67%) achieved recent clinical remission,5 cases(33.33%) achieved significant progress in the near future,and 3 cases(20.00%) achieved recent progress.One case(6.67%) was invalid.All patients had no severe myelosuppression and adverse events.Conclusion:Cyclosporine A combined with low-dose HA regimen is safe and effective in the treatment of refractory PNH,and the toxicity can be tolerated. -
[1] 张立平,赵亚玲,付建珠,等.阵发性睡眠性血红蛋白尿继发自身免疫性溶血性贫血1例[J].临床血液学杂志,2015,28(7):633-634.
[2] Jeffrey J,Robert A,Brodsky MD,et al.Paroxysmal nocturnal hemoglobinuria from bench to bedside[J].Clin Transl Sci,2011,4:219-224.
[3] 胡彬,岳青芳,陈烨,等.阵发性睡眠性血红蛋白尿患者并发大脑上矢状窦血栓形成1例并文献复习[J].临床血液学杂志,2014,27(5):412-413,417.
[4] Luzzatto L.Recent advances in the pathogenesis and treatment of paroxysmal nocturnal hemoglobinuria[J].F1000Res,2016,5:pii:F1000 Faculty Rev-209.
[5] Luzzatto L,Gianfaldoni G,Notaro R.Management of paroxysmal nocturnal haemoglobinuria:a personal view[J].Br J Haematol,2011,153:709-720.
[6] Santarone S,Bacigalupo A,Risitano AM,et al.Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria:long-term results of a retrospective study on behalf of the Gruppo Italiano Trapianto Midollo Osseo (GITMO)[J].Haematologica,2010,95:983-988.
[7] Peffault de Latour R,Schrezenmeier H,Bacigalupo A,et al.Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria[J].Haematologica,2012,97:1666-1672.
[8] Griffin M,Kulasekararaj A,Gandhi S,et al.Concurrent treatment of aplastic anemia/paroxysmal nocturnal hemoglobinuria syndrome with immunosuppressive therapy and eculizumab:a UK experience[J].Haematologica,2018,103:e345-e347.
[9] Parker CJ.Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy[J].Hematology Am SocHematol Educ Program,2011,2011:21-29.
[10] Kelly RJ,Hill A,Arnold LM,et al.Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria:sustained efficacy and improved survival[J].Blood,2011,117:6786-6792.
[11] Hillmen P,Elebute M,Kelly R,et al.Long-term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria[J].Am J Hematol,2010,85:553-559.
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