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沙利度胺在儿童骨髓移植后难治性慢性移植物抗宿主病治疗中的作用。

The role of thalidomide in the treatment of refractory chronic graft-versus-host disease following bone marrow transplantation in children.

作者信息

Rovelli A, Arrigo C, Nesi F, Balduzzi A, Nicolini B, Locasciulli A, Vassallo E, Miniero R, Uderzo C

机构信息

Clinica Pediatrica dell'Università di Milano, Ospedale San Gerardo, Monza, Italy.

出版信息

Bone Marrow Transplant. 1998 Mar;21(6):577-81. doi: 10.1038/sj.bmt.1701138.

DOI:10.1038/sj.bmt.1701138
PMID:9543061
Abstract

Chronic graft-versus-host disease (cGVHD) is a frequent complication of allogeneic bone marrow transplantation (BMT). Thalidomide was found to have immunosuppressive properties and it has been used in a limited number of children with cGVHD. We report our experience with refractory and/or high-risk cGVHD in 14 children. Six children showed complete clinical response to thalidomide in a median time of 2 months. Four children had partial responses and four failed. Side-effects were usually mild (somnolence, constipation) and only two patients developed sensory peripheral neuropathy. An increased incidence of infectious complications attributable to thalidomide was not observed. Nine out of 10 responding patients are alive 49-111 months post-BMT. Thalidomide can be effective particularly in children with prevailing mucocutaneous cGVHD. All patients should be carefully monitored to detect peripheral neuropathy early.

摘要

慢性移植物抗宿主病(cGVHD)是异基因骨髓移植(BMT)常见的并发症。沙利度胺具有免疫抑制特性,已用于少数患有cGVHD的儿童。我们报告了14例儿童难治性和/或高危cGVHD的治疗经验。6例儿童在中位时间2个月时对沙利度胺表现出完全临床反应。4例儿童有部分反应,4例治疗失败。副作用通常较轻(嗜睡、便秘),仅有2例患者出现感觉性周围神经病变。未观察到沙利度胺所致感染并发症发生率增加。10例有反应的患者中9例在BMT后49 - 111个月存活。沙利度胺可能特别对以皮肤黏膜为主的cGVHD儿童有效。所有患者均应仔细监测以便早期发现周围神经病变。

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