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抗胸腺细胞球蛋白与环孢素用于儿童获得性再生障碍性贫血的治疗

Antithymocyte globulin and cyclosporin in children with acquired aplastic anemia.

作者信息

Chandra Jagdish, Naithani Rahul, Ravi Rakesh, Singh Varinder, Narayan Shashi, Sharma Sunita, Pemde Harish, Dutta A K

机构信息

Kalawati Saran Childrens Hospital, New Delhi, India. jchandra55@ yahoo.co.in

出版信息

Indian J Pediatr. 2008 Mar;75(3):229-33. doi: 10.1007/s12098-008-0050-5.

DOI:10.1007/s12098-008-0050-5
PMID:18376089
Abstract

OBJECTIVE

To assess the responses to ATG and cyclosporin combination in patients of aplastic anemia.

METHODS

Twenty three (17M: 6F) patients of aplastic anemia (11 very severe aplastic anemia (VSAA) and 12 severe aplastic anemia (SAA), were administered antithymocyte globulin and cyclosporin.

RESULTS

The median age of patents was 8 years (range 6-12 years). Three patients died within 2 months of therapy. Twenty children (11 SAA and 9 VSAA) were finally analysed. Six months after the start of treatment, 8/20 (40%) patients responded-2 complete (CR) and 6 partial responses (PR). At the end of 1 year; 2 patients maintained CR and seven patients continued PR (overall responders 45%). The response was better in SAA (54.5%) with 2 CR and 4 PR; than in VSAA (33%) with 3 PR . Eleven (55%) children were alive without response. One patient developed AML 13 months later.

CONCLUSION

We conclude that antithymocyte globulin and cyclosporin combination is an effective treatment for aplastic anemia patients who are ineligible for bone marrow transplantation.

摘要

目的

评估再生障碍性贫血患者对抗胸腺细胞球蛋白(ATG)和环孢素联合治疗的反应。

方法

23例(17例男性:6例女性)再生障碍性贫血患者(11例极重型再生障碍性贫血(VSAA)和12例重型再生障碍性贫血(SAA))接受了抗胸腺细胞球蛋白和环孢素治疗。

结果

患者的中位年龄为8岁(范围6 - 12岁)。3例患者在治疗的2个月内死亡。最终对20名儿童(11例SAA和9例VSAA)进行了分析。治疗开始6个月后,8/20(40%)的患者有反应——2例完全缓解(CR)和6例部分缓解(PR)。在1年结束时;2例患者维持CR,7例患者继续PR(总体缓解率45%)。SAA的反应更好(54.5%),有2例CR和4例PR;高于VSAA(33%),有3例PR。11例(55%)儿童存活但无反应。1例患者在13个月后发生急性髓系白血病(AML)。

结论

我们得出结论,抗胸腺细胞球蛋白和环孢素联合治疗对于不适合进行骨髓移植的再生障碍性贫血患者是一种有效的治疗方法。

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