Retreatment with rabbit anti-thymocyte globulin and ciclosporin for patients with relapsed or refractory severe aplastic anaemia
Retreatment with rabbit anti-thymocyte globulin and ciclosporin for patients with relapsed or refractory severe aplastic anaemia
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DOI：
10.1111/j.1365-2141.2006.06098.x
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发表时间：
2006-06-01
影响因子：
6.5
通讯作者：
Young, NS
中科院分区：
文献类型：
--
作者：
Scheinberg, P;Nunez, O;Young, NS
严重再生障碍性贫血（SAA）患者没有匹配的同胞供体，马抗胸腺细胞球蛋白（h-ATG）/环孢素（CsA）治疗失败的管理是不确定的。基于ATG的免疫抑制的重复过程经常被采用;在儿童和越来越多的成人中，替代供体造血干细胞移植是一种选择。我们分析了在过去5年中在我们机构治疗的43例患者中使用兔ATG（r-ATG）/CsA再治疗的成功率; 22例对基于h-ATG/CsA的方案难治（20例成人; 2例儿童），21例（17例成人; 4例儿童）在基于h-ATG/CsA的方案后复发。h-ATG难治性患者的总体缓解率为30%，h-ATG后复发的患者为65%。对r-ATG有反应的患者的1000天生存率为90%，而无反应者为65%。6例患者发生了克隆性血液病; 2例为应答者，2例为非应答者，2例在r-ATG治疗后3个月评估应答前发生了演变。13例患者死亡; 3例为应答者，6例为非应答者，4例患者在评估应答时3个月前死亡。在我们的研究中，难治性患者的缓解率低于先前报道的水平。
The management of patients with severe aplastic anaemia (SAA) who do not have a matched sibling donor and fail a course of horse anti-thymocyte globulin (h-ATG)/ciclosporin (CsA) is uncertain. Repeated courses of ATG-based immunosuppression are often employed; in children and increasingly in adults, alternative donor haematopoietic stem cell transplantation is an option. We analysed the success rate of retreatment with rabbit ATG (r-ATG)/CsA in 43 patients treated at our institution in the last 5 years; 22 were refractory (20 adults; two children) to h-ATG/CsA-based regimens and 21 (17 adults; four children) had relapsed after h-ATG/CsA-based regimens. The overall response rate was 30% in patients who were refractory to h-ATG and 65% in patients who had relapsed following h-ATG. The 1000-d survival in patients who responded to r-ATG was 90% compared with 65% in non-responders. Six patients developed a clonal haematological disorder; two were responders, two were non-responders and in two the evolution occurred before the response could be assessed at 3 months following r-ATG. Thirteen patients died; three were responders, six were non-responders and four patients died prior to 3 months when response was assessed. In our study, the response rate in refractory patients was inferior to what has been previously reported.