• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Clinical immunology: The official journal of the Clinical Immunology Society >Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).
【24h】

Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).

机译：腺苷脱氨酶缺乏症的管理选择; EBMT卫星讲习班会议记录（2006年3月，汉堡）。

获取原文
获取原文并翻译 | 示例
       
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Adenosine deaminase (ADA) deficiency is a disorder of purine salvage that has its most devastating consequences in the immune system leading to severe combined immunodeficiency (SCID). Management options for ADA SCID include hematopoietic stem cell transplantation, enzyme replacement therapy and gene therapy. Formal data on the outcome following each of the three treatment modalities are limited, and this symposium was held in order to gather together the experience from major centers in Europe and the US. Transplantation for ADA-SCID is highly successful with survival rates of approximately 90% if a matched sibling or matched related donor is available but survival following matched unrelated donor or haploidentical procedures is 63% and 50% respectively with a significant rejectionon-engraftment rate in unconditioned procedures. Successfully transplanted patients demonstrated good immunological recovery with normal cellular and humoral function in the majority of cases. PEG-ADA has been used in over 150 patients worldwide either as an alternative to mismatched transplant or as a stabilizing measure prior to transplant. Overall, approximately two thirds of patients treated with PEG-ADA have survived with the majority of patients showing good clinical improvement. The level of immune recovery long term was less than that seen after transplant and approximately 50% of patients continued to receive immunoglobulin replacement. Gene therapy has been used as an experimental procedure in two centers in Europe. Early results from 9 patients suggest that the treatment is safe and that the majority have shown recovery of cellular immune function. Long-term follow-up of treated patients highlights a significant incidence of non-immunological problems with cognitive, neurological and audiological abnormalities most prominent.
机译：腺苷脱氨酶（ADA）缺乏症是一种嘌呤抢救性疾病，在免疫系统中具有最致命的后果，导致严重的联合免疫缺陷（SCID）。 ADA SCID的管理选项包括造血干细胞移植，酶替代疗法和基因疗法。三种治疗方式中每种治疗方式的结局的正式数据是有限的，举行了这次座谈会是为了收集欧洲和美国主要中心的经验。如果有相配的同胞或相配的相关供体，ADA-SCID的移植将非常成功，存活率约为90％，但相配的不相干供体或单倍程序后的存活率分别为63％和50％，排斥/非移植率很高在无条件的程序中。在大多数情况下，成功移植的患者表现出良好的免疫功能，细胞和体液功能正常。 PEG-ADA已在全球超过150位患者中使用，可作为不匹配移植的替代方法或作为移植前的稳定措施。总体而言，接受PEG-ADA治疗的患者中约有三分之二幸存，大多数患者表现出良好的临床改善。长期的免疫恢复水平低于移植后的水平，大约50％的患者继续接受免疫球蛋白替代治疗。基因治疗已在欧洲的两个中心用作实验方法。 9名患者的早期结果表明该治疗是安全的，并且大多数已显示出细胞免疫功能的恢复。对接受治疗的患者进行的长期随访突显出非免疫问题的发生率很高，其中认知，神经和听觉异常最为突出。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号