Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, China.
Department of Hematology, The First People's Hospital of Chenzhou, Chenzhou, China.
Int J Cancer. 2021 Sep 1;149(5):1109-1120. doi: 10.1002/ijc.33608. Epub 2021 May 28.
Whether patients with myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) benefit from pretransplant cytoreductive therapy remains controversial. Our study compared the outcomes of upfront transplantation with those of pretransplant cytoreductive therapy in the patients who received transplantation and those who dropped out due to cytoreductive therapy-related adverse effects. Patients with MDS-EB-1 or MDS-EB-2 were enrolled and divided into three groups based on therapy pretransplantation: upfront transplantation (upfront, n = 54), induction chemotherapy (CT, n = 66) and hypomethylating agents (HMA, n = 37) alone. One hundred fifty-seven patients were enrolled and 124 received allo-HSCT, with 5.6%, 28.8% and 29.7% of drop-out rate of transplantation in upfront, CT and HMA groups (P = .030), respectively. Overall therapy-related mortality (TRM), cytoreductive therapy and transplant-related mortality was 13.0%, 32.4% and 28.4% (P = .028), and 5-year overall survival (OS) was 73.6%, 43.4% and 46.9% (P = .033). Multivariate analysis showed that CT and HMA were risk factors for TRM and OS, and transplantation was a protective factor for OS. In transplant patients, 3-year cumulative incidence of relapse was 10.6%, 20.4% and 20.3% (P = .033), 5-year TRM was 14.5%, 20.0% and 17.6% (P = .651), OS was 77.3%, 64.3% and 68.8% (P = .047) and DFS was 74.0%, 63.0% and 65.8% (P = .042). Multivariate analysis showed that CT was a risk factor for DFS, while CT, HMA and poor karyotype were risk factors for relapse. Results suggested that pretransplant cytoreductive therapy was not associated with better outcomes in the patients who had undergone transplantation. Therefore, upfront transplantation may be preferable for MDS patients.
对于接受异基因造血干细胞移植（allo-HSCT）的骨髓增生异常综合征（MDS）患者，移植前细胞减灭治疗是否获益仍存在争议。本研究比较了接受移植和因细胞减灭治疗相关不良反应而退出的患者中，直接移植与移植前细胞减灭治疗的结果。纳入 MDS-EB-1 或 MDS-EB-2 患者，并根据移植前治疗分为三组：直接移植（ upfront，n=54）、诱导化疗（CT，n=66）和低甲基化药物（HMA，n=37）。共纳入 157 例患者，其中 124 例接受 allo-HSCT，直接移植、CT 和 HMA 组的移植脱落率分别为 5.6%、28.8%和 29.7%（P=0.030）。总治疗相关死亡率（TRM）、细胞减灭治疗和移植相关死亡率分别为 13.0%、32.4%和 28.4%（P=0.028），5 年总生存率（OS）分别为 73.6%、43.4%和 46.9%（P=0.033）。多因素分析显示，CT 和 HMA 是 TRM 和 OS 的危险因素，而移植是 OS 的保护因素。在移植患者中，3 年累积复发率分别为 10.6%、20.4%和 20.3%（P=0.033），5 年 TRM 分别为 14.5%、20.0%和 17.6%（P=0.651），OS 分别为 77.3%、64.3%和 68.8%（P=0.047），DFS 分别为 74.0%、63.0%和 65.8%（P=0.042）。多因素分析显示，CT 是 DFS 的危险因素，而 CT、HMA 和不良核型是复发的危险因素。结果表明，移植前细胞减灭治疗与移植患者的结局改善无关。因此，对于 MDS 患者，直接移植可能是更好的选择。
