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一项关于抗胸腺细胞球蛋白(ATG)治疗“低危”骨髓增生异常综合征患者的初步研究。

A pilot study of antithymocyte globulin (ATG) in the treatment of patients with 'low-risk' myelodysplasia.

作者信息

Killick Sally B, Mufti Ghulam, Cavenagh Jamie D, Mijovic Alex, Peacock Janet L, Gordon-Smith Edward C, Bowen David T, Marsh Judith C W

机构信息

Department of Haematology, St George's Hospital, London, UK.

出版信息

Br J Haematol. 2003 Feb;120(4):679-84. doi: 10.1046/j.1365-2141.2003.04136.x.

DOI:10.1046/j.1365-2141.2003.04136.x
PMID:12588356
Abstract

We report 30 'low-risk' patients with myelodysplasia (MDS) (defined as < 10% bone marrow blasts) who were treated with antithymocyte globulin (ATG). In total, 20 patients were evaluable at the study end-point (response to treatment at 6 months). The diagnosis in these 20 patients was refractory anaemia (RA) in 13, RA with excess blasts in four, and RA with ringed sideroblasts in three. Median age was 54.5 years (range, 31-73 years). There were two cases of secondary MDS. The bone marrow was hypocellular in eight cases and cytogenetics were abnormal in four cases. All patients received lymphoglobuline (horse ATG; Sangstat, France) at a dose of 1.5 vials/10 kg/day for 5 d. The treatment was well tolerated. Three patients in the study died (disease progression, invasive aspergillosis and lung carcinoma respectively); 10 out of 20 evaluable patients (50%) responded to treatment and became transfusion independent; eight out of 13 (62%) patients with RA responded. The median duration of response was 15.5 months (2-42+ months) at the time of analysis.

摘要

我们报告了30例接受抗胸腺细胞球蛋白(ATG)治疗的骨髓增生异常综合征(MDS)“低危”患者(定义为骨髓原始细胞<10%)。在研究终点(6个月时的治疗反应)时,共有20例患者可进行评估。这20例患者中,13例诊断为难治性贫血(RA),4例为伴有过多原始细胞的RA,3例为伴有环形铁粒幼细胞的RA。中位年龄为54.5岁(范围31 - 73岁)。有2例继发性MDS。8例患者骨髓细胞减少,4例细胞遗传学异常。所有患者均接受了淋巴细胞球蛋白(马ATG;法国桑斯特公司)治疗,剂量为1.5瓶/10 kg/天,共5天。治疗耐受性良好。研究中有3例患者死亡(分别为疾病进展、侵袭性曲霉病和肺癌);20例可评估患者中有10例(50%)对治疗有反应并不再依赖输血;13例RA患者中有8例(62%)有反应。在分析时,反应的中位持续时间为15.5个月(2 - 42 +个月)。

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