University Medical Center Hamburg-Eppendorf, Department of Stem Cell Transplantation, Hamburg, Germany.
Università Tor Vergata, Rome, Italy.
Bone Marrow Transplant. 2024 Aug;59(8):1084-1091. doi: 10.1038/s41409-024-02282-7. Epub 2024 Apr 25.
Short-term outcome of myeloablative (MAC) and reduced intensity (RIC) conditioning in the prospective randomized international EBMT RICMAC study in patients with myelodyplastic syndrome (MDS) was comparable but longer follow up is lacking. Patients with MDS aged 18-65 years were randomized to receive MAC (N = 64) with busulfan/cyclophosphamide or RIC (n = 65) with busulfan/fludarabine followed by stem cell transplantation -(HCT) from HLA matched or mismatched donor. After a median follow-up of 6.2 (0.4-12.5) years, 10-year OS and RFS were 54.0% and 43.9% for RIC and 44.4% and 44.2% for MAC (p = 0.15 and p = 0.78), respectively. Since the first report, 6 patients died on NRM, 4 after RIC, and 2 after MAC. Similarly, 8 patients relapsed (4 in each arm), increasing the number of relapsed patients to 28. The second HCT was performed in 18 pts, 8 in the MAC, and 10 in the RIC arm. In a multivariate analysis, ECOG status and chemotherapy prior to HCT were independent risk factors for OS and RFS, ECOG and low cytogenetic risk for NRM and chemotherapy prior to HCT for RI. Patients with low cytogenetic risk had better OS [p = 0.002], RFS [p = 0.02], and NRM (p = 0.015) after RIC as compared to MAC.
在 prospective randomized international EBMT RICMAC 研究中，接受清髓性（MAC）和减低强度（RIC）预处理条件下造血干细胞移植的骨髓增生异常综合征（MDS）患者的短期结果是可比的，但缺乏长期随访。18-65 岁的 MDS 患者随机分为 MAC 组（n=64），接受马法兰/环磷酰胺预处理；RIC 组（n=65）接受马法兰/氟达拉滨预处理，随后接受 HLA 匹配或不匹配供者的造血干细胞移植（HCT）。中位随访 6.2（0.4-12.5）年后，RIC 和 MAC 组的 10 年 OS 和 RFS 分别为 54.0%和 43.9%，44.4%和 44.2%（p=0.15 和 p=0.78）。自首次报告以来，6 例患者因 NRM 死亡，4 例在 RIC 后，2 例在 MAC 后。同样，8 例患者复发（每臂 4 例），使复发患者人数增加到 28 例。18 例患者接受了第二次 HCT，MAC 组 8 例，RIC 组 10 例。多变量分析显示，ECOG 状态和 HCT 前的化疗是 OS 和 RFS 的独立危险因素，ECOG 和低细胞遗传学风险是 NRM 的独立危险因素，HCT 前的化疗是 RI 的独立危险因素。与 MAC 相比，RIC 组低细胞遗传学风险患者的 OS [p=0.002]、RFS [p=0.02]和 NRM（p=0.015）更好。
