低剂量联合化疗治疗复发难治的再生障碍性贫血-阵发性睡眠性血红蛋白尿综合征的疗效  

Efficacy of Low Dose Combined Chemotherapy for Patients with Relapsed and Refractory Aplastic Anemia-Paroxysmal Nocturnal Hemoglobinuria Syndrome

作　　者：林莺 张荣东 陈仁利 LIN Ying;ZHANG Rong-Dong;CHEN Ren-Li(Department of Hematology,Ningde Hospital Affiliated to Fujian Medical University,Ningde 352100,Fujian Province,China)

机构地区：[1]福建医科大学附属宁德市医院血液科

出　　处：《中国实验血液学杂志》2019年第4期1215-1219,共5页Journal of Experimental Hematology

基　　金：宁德市科技局指导性项目(20170119);市级临床重点专科建设项目(2120899)

摘　　要：目的:评价低剂量MP方案与HA方案治疗复发难治的再生障碍性贫血-阵发性睡眠性血红蛋白尿(AAPNH)综合征患者的治疗效果,探讨低剂量联合化疗在治疗AA-PNH综合征方面的优势。方法:回顾分析9例复发难治的AA-PNH综合征患者临床特征及采用低剂量联合化疗的疗效,其中5例予MP方案(马法兰2mg/(m^2·d),泼尼松0.5mg/(kg·d),口服,d1-7),4例予HA方案(高三尖杉酯碱2mg/d,阿糖胞苷100mg/d,静滴,d1-5),比较不同患者治疗前后PNH克隆、激素用量及溶血指标的变化,同时比较疾病复发、输血依赖及不良反应等情况。每名患者均行1-2个疗程化疗。结果:9例患者中7例患者治疗有效,其激素用量较化疗前明显减少,胆红素水平显著低于化疗前,化疗后贫血有所缓解(P<0.05)。7例有效患者中1年内复发1例,且有效者中3例PNH克隆负荷转阴,5例患者1年内均未再输血,所有病人均未发生严重骨髓抑制。结论:低剂量联合化疗方案对AA-PNH患者具有较好的疗效和安全性,且患者对其耐受性较好,可作为复发难治的AA-PNH综合征的治疗选择。Objective:To evaluate the clinical efficacy of low dose combined chemotherapy ( LDCC) for patients with relapsed and refractory aplastic anemia-paroxysmal nocturnal hemoglobinuria( AA-PNH) syndrome,and to analyze the advantages of LDCC in the treatment of AA-PNH syndrome.Methods:The clinical characteristics and the curative effect of LDCC in 9 patients with relapsed and refractory AA-PNH syndrome were retrospectively analyzed.Five patients were treated with MP therapy[melphalan 2 mg /( m^2·d);prednisone 0.5 mg /( kg·d)],and the other 4 patients were treated with HA therapy( HHT 2 mg /d iv drip,for 5 days;Ara-C 100 mg /d iv drip,for 5 days).The changes of PNH clone,dosage of corticosteroid,hemolysis and the relapse of disease,hematological parameters and adverse reactions were compared before and after therapy.All patients were treated for 1 - 2 courses.Results:Seven out of 9 patients responded well,the dosage of corticosteroid and the bilirubin concentration decreased significantly and anemia was relieved in 7 patients ( P < 0.05).One patient relapsed in one year.PNH clone of 3 patients turned negative.Five patients did not rely on blood transfusion in 1 year.There was no bone marrow failure to be found in all patients. Conclusion:The LDCC has better efficacy and safety in the treatment of patients with AA-PNH syndrome,moreover, the patients is more tolerant to LDCC,thus the LDCC may be a selection for treatment of patients with relapsed and refractory AA-PNH syndrome.

关 键 词：低剂量 MP方案 HA方案 AA-PNH综合征 疗效 

分 类 号：R556.5[医药卫生—血液循环系统疾病] R556.6[医药卫生—内科学]