Thabet Ahmad F, Faisal Mostafa
Clinical Hematology Unit, Department of Internal Medicine, Faculty of Medicine, Assiut University, Assiut, Egypt.
Indian J Hematol Blood Transfus. 2014 Dec;30(4):313-8. doi: 10.1007/s12288-013-0290-z. Epub 2013 Aug 15.
Treatment of steroid refractory autoimmune hemolytic anemia (AIHA) is challenging especially with no evidence based consensus guide lines and limited resources. The aim of this study was to evaluate the efficacy of pulse cyclophosphamide therapy in patients with severe refractory warm AIHA. The prospective study was designed to evaluate the efficacy of pulse cyclophosphamide-1 g/month for four consecutive months-in 17 patients (10 males and 7 females) with severe refractory warm AIHA [13 primary AIHA and 4 (females) secondary to SLE], all studied patients failed to respond to high dose of steroid therapy ± azathioprine ± intravenous immunoglobulin ± oral cyclophosphamide. Mean hemoglobin level, reticulocytic count and direct antiglobulin test were assessed before and after cyclophosphamide treatment every month. After the 4th cycle of cyclophosphamide (82 %, 14 patients) achieved partial response while the remaining (17 %, 3 patients) showed no response, while after 6 months follow up 47 % (8 patients) show complete response, while 53 % (9 patients) showed partial response. The mean hemoglobin levels were significantly increased after the 1st, 2nd, 3rd and 4th months of pulse cyclophosphamide therapy when compared to before treatment (P < 0.01, P < 0.001, P < 0.001 and P < 0.001) respectively, and the mean reticulocyte (%) were significantly decreased after the 2nd, 3rd and 4th months (P < 0.05, P < 0.01 and P < 0.001) respectively. We conclude that pulse cyclophosphamide therapy is well tolerated and induces good response in patients with severe refractory warm AIHA.
治疗类固醇难治性自身免疫性溶血性贫血(AIHA)具有挑战性,尤其是在缺乏基于证据的共识指南且资源有限的情况下。本研究的目的是评估脉冲环磷酰胺疗法对重度难治性温抗体型AIHA患者的疗效。这项前瞻性研究旨在评估连续四个月每月使用1g脉冲环磷酰胺对17例重度难治性温抗体型AIHA患者(10例男性和7例女性)的疗效[13例原发性AIHA和4例(女性)继发于系统性红斑狼疮(SLE)],所有研究患者对高剂量类固醇疗法±硫唑嘌呤±静脉注射免疫球蛋白±口服环磷酰胺均无反应。在环磷酰胺治疗前后每月评估平均血红蛋白水平、网织红细胞计数和直接抗球蛋白试验。在第4个环磷酰胺周期后,14例患者(82%)获得部分缓解,其余3例患者(17%)无反应,而在6个月的随访后,47%(8例患者)完全缓解,53%(9例患者)部分缓解。与治疗前相比,在脉冲环磷酰胺治疗的第1、2、3和4个月后,平均血红蛋白水平显著升高(分别为P < 0.01、P < 0.001、P < 0.001和P < 0.001),在第2、3和4个月后,平均网织红细胞(%)显著降低(分别为P < 0.05、P < 0.01和P < 0.001)。我们得出结论,脉冲环磷酰胺疗法耐受性良好,对重度难治性温抗体型AIHA患者诱导出良好反应。