Reduced intensity allogeneic stem cell transplantation for systemic primary amyloidosis refractory to high‐dose melphalan
Reduced intensity allogeneic stem cell transplantation for systemic primary amyloidosis refractory to high‐dose melphalan
复制标题
降低强度同种异体干细胞移植治疗高剂量马法兰难治性系统性原发性淀粉样变性
DOI：
--
复制
发表时间：
2004
影响因子：
3.1
通讯作者：
T. Ueda
中科院分区：
文献类型：
--
作者：
Y. Kawai;K. Kinoshita;H. Arai;A. Kuwata;Y. Fukuoka;M. Yamaoka;S. Imamura;H. Tsutani;T. Ueda
完全消除浆细胞病变是一个合理的治疗目标，因为阻断前体蛋白的供应是淀粉样蛋白沉积主要消退的必要条件。高剂量美法兰联合自体干细胞移植可诱导完全血液学反应（HR）并恢复器官功能障碍。然而，这种治疗的HR率很少超过40%。我们在这里描述了已知的第一例成功的低强度异基因干细胞移植（RIST）患者，该患者患有原发性淀粉样变性合并肾病综合征，但没有心脏病，通过大剂量美伐兰与自体干细胞移植仅获得部分HR。RIST可能是可行的，并且能够在可接受的毒性下实现肾病综合征的完全HR恢复。
Complete elimination of the plasma cell dyscrasia is a rational therapeutic goal, as intercepting supply of precursor protein is a necessary condition for a major regression of amyloid deposits. High‐dose melphalan with autologous stem cell transplantation has shown the ability to induce complete hematological response (HR) along with recovery of organ dysfunction. However, the rate of HR with this treatment rarely exceeds 40%. We describe here the first known case of successful reduced intensity allogeneic stem cell transplantation (RIST) for a patient with primary amyloidosis complicated with nephrotic syndrome but without cardiac disease, who had obtained only partial HR by high‐dose melphalan with autologous stem cell transplantation. RIST may be feasible and be capable of achieving complete HR along with recovery from nephrotic syndrome with acceptable toxicity.
影响因子：
20.3
作者：
McSweeney, PA;Niederwieser, D;Storb, RF
通讯作者：
Storb, RF
影响因子：
20.3
作者：
Giralt, S;Estey, E;Champlin, R
通讯作者：
Champlin, R