低剂量联合化疗对难治性、复发性阵发性睡眠性血红蛋白尿症的疗效观察

Outcome of low dose combined chemotherapy in refractory and relapsed paroxysmal nocturnal hemoglobinuria patients

目的 评价低剂量联合化疗对难治性、复发性阵发性睡眠性血红蛋白尿症 (PNH)的治疗效果 ,探讨进一步提高PNH疗效的方法。方法 回顾分析了 11例难治性、复发性PNH患者采用低剂量联合化疗的疗效。其中 8例患者采用MP方案 [马法兰 2～ 6mg/d ;强的松 0 5mg/ (kg·d) ],另 3例患者分别采用COAP方案 [环磷酰胺2 0 0mg静滴 ,化疗第 2天 ;长春新碱 2mg静滴 ,化疗第 1天 ;阿糖胞苷 10 0mg/d静滴 ,共 5天 ;强的松 0 5mg/ (kg·d)口服 ,共 5天 ]、COA方案 (同COAP方案前 3种药 )、CMAP方案 [环磷酰胺 2 0 0mg静滴 ,隔日 1次 ,共 3次 ;阿糖胞苷 10 0mg/d静滴 ,共 5天 ;6 -巯基嘌呤 10 0mg/d口服 ,共 5天 ;强的松 0 5mg/ (kg·d)口服 ,共 5天 ]方案。所有患者均行 1～ 2疗程化疗。结果 11例患者中 8例有效 ,3例在 1年以内复发。MP方案治疗 8例中 5例有效 ,且 2例达到近期临床缓解 ,1例达到近期临床痊愈。所有患者均无严重的骨髓抑制及毒副反应。结论 低剂量联合化疗可提高PNH的疗效 ,难治性、复发性PNH可选择低剂量联合化疗作为有效的治疗方法。

Objective To evaluate the clinical outcome of low dose combined chemotherapy (LDCC) in refractory and relapsed paroxysmal nocturnal hemoglobinuria (PNH) patients. Methods The effect of LDCC in 11 refractory and relapsed PNH patients was retrospectively analyzed. 8 patients were treated with MP therapy[Mel 2～6mg/d; Prednisone 0 5mg/(kg·d)],and the other 3 patients were treated with COAP therapy[CTX 200mg i.v. drip,the second day;VCR 2mg i.v. drip,the first day;Ara-C 100mg/d i.v. drip,for 5 days;Prednisone 0 5mg/(kg·d) p.o., for 5 days],COA therapy(the same regimens of COAP therapy except for prednisone), CMAP therapy[CTX 200mg i.v. drip,every 2 days for 3 doses;6-MP 100mg/d p.o., for 5 days;Ara-C 100mg/d i.v. drip,for 5 days;Prednisone 0 5mg/(kg·d) p.o. , for 5 days],respectively.All patients were treated for 1～2 courses. Results 8 patients(72 7%) had good effect,of which 3 patients(37 5%) relapsed in 1 year.Of 8 patients treated with MP therapy,5 patients(62 5%) had dramatic responses,and the hemoglobin level had been normal continiously for more than 1 year in 3 patients.There was no bone marrow failure and serious side-effects in all patients. Conclusions LDCC is suitble for refractory and relapsed PNH.