International Center for Transplantation in Thalassemia and Sickle Cell Anemia, Mediterranean Institute of Hematology, Policlinic of the University of Rome Tor Vergata, Viale Oxford, Rome, Italy.
Ann N Y Acad Sci. 2010 Aug;1202:149-54. doi: 10.1111/j.1749-6632.2010.05543.x.
Allogeneic hemopoietic stem cell transplantation (HSCT) represents one of the best cures for thalassemia. Currently, HSCT for thalassemia consists of allogeneic stem cell gene therapy and still awaits autologous genetically modified stem cell transplantation. HSCT for thalassemia has substantially improved over the last two decades, due in large part to improvements in preventive strategies, the effective control of transplant-related complications, and the development of new preparative regimens. A risk classes-based approach to transplantation in thalassemia has led to disease-free survival probability of 87, 85, and 80% in classes 1, 2, and 3 patients, respectively. Adult thalassemia patients, who are higher risk patients for transplant-related toxicity due to an advanced phase of the disease, have a cure rate of 65% with current treatment protocol. Patients who do not have matched family or unrelated donors could benefit from haploidentical mother-to-child transplantation. Overall, the results of this type of transplantation appear encouraging.
同种异体造血干细胞移植（HSCT）是治疗地中海贫血症的最佳方法之一。目前，地中海贫血症的 HSCT 包括异体干细胞基因治疗，仍在等待自体基因修饰干细胞移植。由于预防策略的改进、移植相关并发症的有效控制以及新预处理方案的发展，过去二十年来 HSCT 治疗地中海贫血症取得了显著进展。基于风险分类的地中海贫血症移植方法使 1 类、2 类和 3 类患者的无病生存率分别达到 87%、85%和 80%。由于疾病处于晚期，成年地中海贫血症患者存在更高的移植相关毒性风险，采用目前的治疗方案其治愈率为 65%。没有匹配的家族或无关供体的患者可以从半相合母亲-子女移植中受益。总体而言，这种类型的移植结果令人鼓舞。
