Wang Q Q, Liu Z X, Zhao X L, Zhang G X, Yao J F, Zheng X H, Zhang L N, Shen Y Y, Zhao X L, He Y, Huang Y, Zhang R L, Wei J L, Ma Q L, Pang A M, Yang D L, Zhai W H, Jiang E L, Feng S Z, Han M Z
Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Tianjin 300020, China.
Zhonghua Xue Ye Xue Za Zhi. 2020 Feb 14;41(2):132-137. doi: 10.3760/cma.j.issn.0253-2727.2020.02.009.
To evaluate the outcomes of myelodysplastic syndromes (MDS) patients who received HLA-matched sibling donor allogeneic peripheral blood stem cell transplantation (MSD-PBSCT) . The clinical data of 138 MDS patients received MSD-PBSCT from Sep. 2005 to Dec. 2017 were retrospectively analyzed, and the overall survival (OS) rate, disease-free survival (DFS) rate, relapse rate (RR) , non-relapse mortality (NRM) rate and the related risk factors were explored. ①After a median follow-up of 1 050 (range 4 to 4 988) days, the 3-year OS and DFS rates were (66.6±4.1) % and (63.3±4.1) %, respectively. The 3-year cumulative incidence of RR and NRM rates were (13.9±0.1) % and (22.2±0.1) %, respectively. ②Univariate analysis showed that patients with grade Ⅲ-Ⅳ acute graft-versus-host disease (aGVHD) or hematopoietic cell transplantation comorbidity index (HCT-CI) ≥2 points or patients in very high-risk group of the Revised International Prognostic Scoring System (IPSS-R) had significantly decreased OS[ (42.9±13.2) % (72.9±4.2) %, (2)=8.620, =0.003; (53.3±7.6) % (72.6±4.7) %, (2)=6.681, =0.010; (53.8±6.8) % (76.6±6.2) % (73.3±7.7) %, (2)=6.337, =0.042]. For MDS patients with excess blasts-2 (MDS-EB2) and acute myeloid leukemia patients derived from MDS (MDS-AML) , pre-transplant chemotherapy or hypomethylating agents (HMA) therapy could not improve the OS rate[ (60.4±7.8) % (59.2±9.6) %, (2)=0.042, =0.838]. ③Multivariate analysis indicated that the HCT-CI was an independent risk factor for OS and DFS (=0.012, =2.108, 95% 1.174-3.785; =0.008, =2.128, 95% 1.219-3.712) . HCT-CI was better than the IPSS-R in predicting the outcomes after transplantation. The occurrence of grade Ⅲ-Ⅳ aGVHD is a poor prognostic factor for OS. For patients of MDS-EB2 and MDS-AML, immediate transplantation was recommended instead of receiving pre-transplant chemotherapy or HMA therapy.
评估接受人类白细胞抗原（HLA）匹配的同胞供体异基因外周血干细胞移植（MSD - PBSCT）的骨髓增生异常综合征（MDS）患者的预后情况。回顾性分析了2005年9月至2017年12月期间138例接受MSD - PBSCT的MDS患者的临床资料，探讨总生存（OS）率、无病生存（DFS）率、复发率（RR）、非复发死亡率（NRM）率及相关危险因素。①中位随访1050（范围4至4988）天，3年OS率和DFS率分别为（66.6±4.1）%和（63.3±4.1）%。3年RR累积发生率和NRM率分别为（13.9±0.1）%和（22.2±0.1）%。②单因素分析显示，Ⅲ - Ⅳ级急性移植物抗宿主病（aGVHD）患者、造血细胞移植合并症指数（HCT - CI）≥2分的患者或修订国际预后评分系统（IPSS - R）极高危组患者的OS显著降低[（42.9±13.2）%对（72.9±4.2）%，χ² = 8.620，P = 0.003；（53.3±7.6）%对（72.6±4.7）%，χ² = 6.681，P = 0.010；（53.8±6.8）%对（76.6±6.2）%对（73.3±7.7）%，χ² = 6.337，P = 0.042]。对于伴有过多原始细胞2型（MDS - EB2）的MDS患者及源自MDS的急性髓系白血病（MDS - AML）患者，移植前化疗或去甲基化药物（HMA）治疗并不能提高OS率[（60.4±7.8）%对（59.2±9.6）%，χ² = 0.042，P = 0.838]。③多因素分析表明，HCT - CI是OS和DFS的独立危险因素（P = 0.012，β = 2.108，95%CI 1.174 - 3.785；P = 0.008，β = 2.128，95%CI 1.219 - 3.712）。在预测移植后预后方面，HCT - CI优于IPSS - R。Ⅲ - Ⅳ级aGVHD的发生是OS的不良预后因素。对于MDS - EB2和MDS - AML患者，建议立即移植，而非接受移植前化疗或HMA治疗。
