Nakao Shinji
Kanazawa University Graduate School of Medical Science.
Nihon Rinsho. 2003 Sep;61(9):1596-600.
Although non-myeloablative stem cell transplantation(NST) was originally developed to induce graft-versus-malignancy effect, the new transplant modality is now being applied for treatment of non-malignant disorders such as aplastic anemia(AA) and paroxysmal nocturnal hemoglobinuria due to its low toxicities. NST may be preferable than conventional transplantation when an AA patient has complications due to hemochromatosis such as heart failure and diabetes mellitus, and severe infections. We treated three aplastic anemia patients complicated by various organ failures, with a conditioning regimen consisting of fludarabine (Flu), cyclophosphamide(CY), and antithymocyte globulin (ATG). Engraftment promptly occurred in all patients without accompanying severe graft-versus-host disease or organ damages. Successful induction of complete chimerism and a low incidence of toxicities warrant a further clinical trial using the Flu + CY + ATG regimen.
尽管非清髓性干细胞移植(NST)最初是为诱导移植物抗恶性肿瘤效应而开发的,但由于其低毒性,这种新的移植方式现在正被应用于治疗诸如再生障碍性贫血(AA)和阵发性夜间血红蛋白尿等非恶性疾病。当AA患者因血色素沉着症出现诸如心力衰竭、糖尿病和严重感染等并发症时,NST可能比传统移植更可取。我们用包含氟达拉滨(Flu)、环磷酰胺(CY)和抗胸腺细胞球蛋白(ATG)的预处理方案治疗了3例并发各种器官衰竭的再生障碍性贫血患者。所有患者均迅速植入,且未伴有严重的移植物抗宿主病或器官损害。成功诱导完全嵌合体以及低毒性发生率证明使用Flu + CY + ATG方案进行进一步临床试验是合理的。