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高剂量环磷酰胺治疗难治性自身免疫性溶血性贫血。

High-dose cyclophosphamide for refractory autoimmune hemolytic anemia.

作者信息

Moyo Victor M, Smith Douglas, Brodsky Isadore, Crilley Pamela, Jones Richard J, Brodsky Robert A

机构信息

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

出版信息

Blood. 2002 Jul 15;100(2):704-6. doi: 10.1182/blood-2002-01-0087.

Abstract

High-dose cyclophosphamide, without stem cell rescue, has been used successfully to treat aplastic anemia and other autoimmune disorders. To determine the safety and efficacy of high-dose cyclophosphamide among patients with severe refractory autoimmune hemolytic anemia, we treated 9 patients with cyclophosphamide (50 mg. kg(-1). d(-1) for 4 days) who had failed a median of 3 (range, 1-7) other treatments. The median hemoglobin before treatment was 6.7 g/dL (range, 5-10 g/dL). The median time to reach an absolute neutrophil count of 500/microL or greater was 16 days (range, 12-18 days). Six patients achieved complete remission (normal untransfused hemoglobin for age and sex), and none have relapsed after a median follow-up of 15 months (range, 4-29 months). Three patients achieved and continue in partial remission (hemoglobin at least 10 g/dL without transfusion support). High-dose cyclophosphamide was well tolerated and induced durable remissions in patients with severe refractory autoimmune hemolytic anemia.

摘要

大剂量环磷酰胺,无需干细胞救援,已成功用于治疗再生障碍性贫血和其他自身免疫性疾病。为了确定大剂量环磷酰胺在重度难治性自身免疫性溶血性贫血患者中的安全性和有效性,我们对9例环磷酰胺治疗失败(中位数为3次,范围1 - 7次)其他治疗的患者使用环磷酰胺(50 mg·kg⁻¹·d⁻¹,共4天)进行治疗。治疗前血红蛋白中位数为6.7 g/dL(范围5 - 10 g/dL)。达到绝对中性粒细胞计数500/μL或更高的中位时间为16天(范围12 - 18天)。6例患者实现完全缓解(按年龄和性别计算,未输血时血红蛋白正常),在中位随访15个月(范围4 - 29个月)后无复发。3例患者实现并持续部分缓解(血红蛋白至少10 g/dL,无需输血支持)。大剂量环磷酰胺耐受性良好,可诱导重度难治性自身免疫性溶血性贫血患者实现持久缓解。

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