大剂量环磷酰胺作为重型再生障碍性贫血的挽救治疗方法。

High-dose cyclophosphamide as salvage therapy for severe aplastic anemia.

作者信息

Brodsky Robert A, Chen Allen R, Brodsky Isadore, Jones Richard J

机构信息

The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD 21231, USA.

出版信息

Exp Hematol. 2004 May;32(5):435-40. doi: 10.1016/j.exphem.2004.02.002.

DOI:10.1016/j.exphem.2004.02.002

PMID:15145211

Abstract

OBJECTIVE

The treatment options for patients with aplastic anemia who do not respond to conventional immunosuppression are limited. The aim of this study was to evaluate high-dose cyclophosphamide in patients with refractory severe aplastic anemia (SAA).

MATERIALS AND METHODS

We treated 17 SAA patients with high-dose cyclophosphamide (50 mg/kg/day for 4 consecutive days) who previously did not respond to one or more courses of immunosuppressive therapy. Median age was 31 years (range 6-58); median disease duration was 14 months (range 6-58), and 8 patients met criteria for very severe aplastic anemia (absolute neutrophil count <0.2 x 10(9)/L) at the time of treatment.

RESULTS

At median follow-up of 29 months, 10 patients (59%) are alive. Nine patients (53%) achieved a drug-free remission after high-dose cyclophosphamide; 4 patients achieved a complete remission and 5 patients currently meet criteria for a partial remission but continue to improve. One nonresponder to high-dose cyclophosphamide developed paroxysmal nocturnal hemoglobinuria; another nonresponder developed a myelodysplastic syndrome. In responding patients, median time to 500 neutrophils was 54 days (range 35-119), median time to the last platelet transfusion was 99 days (range 51-751), and median time to the last red cell transfusion was 125 days (range 63-796).

CONCLUSION

High-dose cyclophosphamide shows promise for salvaging patients with refractory SAA.

摘要

目的

对于对传统免疫抑制治疗无反应的再生障碍性贫血患者，治疗选择有限。本研究的目的是评估大剂量环磷酰胺治疗难治性重型再生障碍性贫血（SAA）患者的疗效。

材料与方法

我们对17例SAA患者采用大剂量环磷酰胺治疗（连续4天，50mg/kg/天），这些患者先前对一个或多个疗程的免疫抑制治疗无反应。中位年龄为31岁（范围6 - 58岁）；中位病程为14个月（范围6 - 58个月），8例患者在治疗时符合极重型再生障碍性贫血标准（绝对中性粒细胞计数<0.2×10⁹/L）。

结果

中位随访29个月时，10例患者（59%）存活。9例患者（53%）在大剂量环磷酰胺治疗后实现无药缓解；4例患者实现完全缓解，5例患者目前符合部分缓解标准但仍在持续改善。1例对大剂量环磷酰胺无反应的患者发生了阵发性夜间血红蛋白尿；另1例无反应患者发生了骨髓增生异常综合征。在有反应的患者中，中性粒细胞计数达到500的中位时间为54天（范围35 - 119天），最后一次血小板输注的中位时间为99天（范围51 - 751天），最后一次红细胞输注的中位时间为125天（范围63 - 796天）。