Evaluation of allogeneic cell transplantation in acute myeloid leukemia I (ETAL I)

异基因造血干细胞移植在急性髓系白血病患者中的作用仍然是一个有争议的问题。尽管大多数研究者都同意在高危AML患者中异基因SCT介导的生存获益，但迄今为止还没有针对标准风险患者（标准风险由核型定义）进行的前瞻性随机试验专门解决这个问题。由于HLA匹配的无关供者的可用性增加了潜在合适供者的患者数量，因此，现在首次可以将具有前瞻性供者的患者随机分配在首次CR的选择性异基因SCT与仅在复发情况下进行常规缓解后治疗和移植之间。为此，570名60岁以下的AML患者必须在三年的研究期内进行预登记。70%的患者可能会找到匹配的同胞或无关供体，允许考虑399例患者。主要在随机化前约有10%的患者脱落（剩余360例患者），随机化后约有10例患者脱落，假设350例患者在首次CR时随机接受同种异体SCT与常规治疗，仅在复发时接受移植，将准备进行最终分析。这350例病例将允许检测到随机化后4年同种异体SCT和常规缓解后治疗之间总生存率的真实差异约为15%（60% vs. 45%）。

The role of allogeneic stem cell transplantation in patients with acute myeloid leukemia is still a matter of debate. Whereas most investigators agree on the survival benefit mediated by allogeneic SCT in high-risk AML patients, no prospective randomized trial has so far been performed specifically addressing this question in standard-risk patients, with standard risk as defined by karyotype. Since the availability of HLA-matched unrelated donors increases the number of patients with a potentially suitable donor, it is now for the first time possible to randomize patients with a prospective donor between an elective allogeneic SCT in first CR versus conventional postremission therapy and transplantation only in case of relapse.The current trial would therefore specifically help to define the role of allogeneic SCT in AML patients in first CR. For this purpose, 570 patients with AML below the age of 60 will have to be pre-registered over a three years study period. A matched sibling or unrelated donor will probably be identified for 70% of all patients, allowing the consideration of 399 patients. After about 10% drop-outs mainly before randomisation (remaining 360 patients) and a drop out of about 10 patients after randomization, it is assumed that 350 patients randomized between allogeneic SCT in first CR versus conventional therapy and transplantation only in case of relapse will be ready for final analyses. The 350 cases would allow the detection of a true difference in the probability of overall survival between allogeneic SCT and conventional postremission therapy of about 15% (60% vs. 45%) four years after randomisation.