环孢素A联合小剂量HA方案治疗难治性阵发性睡眠性血红蛋白尿症的临床疗效观察

Clinical observation of cyclosporine combined with low-dose HA regimen in the treatment of refractory paroxysmal nocturnal hemoglobinuria

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。

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.