Wang Qianqian, Zhao Xingli, Liu Zixian, Zhao Xiaoli, Zhang Guixin, Yao Jianfeng, Zheng Xiaohui, Zhang Lining, Shen Yuyan, He Yi, Huang Yong, Zhang Rongli, Wei Jialin, Ma Qiaoling, Pang Aiming, Yang Donglin, Jiang Erlie, Feng Sizhou, Zhang Zhuoran, Zhai Weihua, Han Mingzhe
Institute of Hematology & Blood Diseases Hospital, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin, China.
Department of Immuno-Oncology, Beckman Research Institute, City of Hope National Medical Center Duarte, CA, U.S.
Am J Cancer Res. 2020 Apr 1;10(4):1218-1228. eCollection 2020.
To evaluate whether cytoreductive therapy is needed for myelodysplastic syndromes (MDS) patients with excess blasts type 2 (MDS-EB2) and acute myeloid leukemia derived from MDS (MDS-AML) before HLA-matched sibling donor peripheral blood stem cell transplantation (MSD-PBSCT), we retrospectively analyzed 80 cases of MDS-EB2 and MDS-AML patients who received MSD-PBSCT between February 2006 and December 2019 in our hospital. The 3-years overall survival (OS) rate and disease-free survival (DFS) rate were (59.1±5.8)% and (52.5±5.7)%, respectively. The 3-years non-relapse mortality (NRM) rate and relapse rate (RR) were (22.4±0.2)% and (25.4±0.2)%, respectively. Univariate analysis showed that, hematopoietic cell transplantation comorbidity index (HCT-CI) ≥ 2, poor/very poor karyotype and occurrence of grade III-IV acute graft-versus-host disease (aGVHD) are risk factors for OS. Patients received pre-transplant cytoreductive therapy (PCT) and obtained complete remission (CR) had significantly higher OS rate than those who failed to achieve CR (non-CR group) and those who did not receive PCT (non-PCT group) [(80.0±8.3)% (38.1±10.6)% (56.1±9.3)%, =0.010]. PCT significantly increased the OS rate [(62.2±10.0)% (20.0±17.9)%, =0.013] for MDS-AML patients but not for MDS-EB2 patients [(59.2±11.1)% (62.9±10.1)%, =0.991]. Our findings suggest reducing tumor burden by cytoreductive therapy to obtain CR before transplant improves OS. For MDS-AML patients, PCT is beneficial, while for MDS-EB2 patients, PCT is not necessary.
为评估对于伴有2型原始细胞增多的骨髓增生异常综合征（MDS-EB2）以及源自MDS的急性髓系白血病（MDS-AML）患者，在进行人类白细胞抗原（HLA）匹配的同胞供者外周血干细胞移植（MSD-PBSCT）前是否需要进行减瘤治疗，我们回顾性分析了2006年2月至2019年12月间在我院接受MSD-PBSCT的80例MDS-EB2和MDS-AML患者。3年总生存率（OS）和无病生存率（DFS）分别为（59.1±5.8）%和（52.5±5.7）%。3年非复发死亡率（NRM）和复发率（RR）分别为（22.4±0.2）%和（25.4±0.2）%。单因素分析显示，造血细胞移植合并症指数（HCT-CI）≥2、核型差/极差以及发生III-IV级急性移植物抗宿主病（aGVHD）是OS的危险因素。接受移植前减瘤治疗（PCT）并获得完全缓解（CR）的患者，其OS率显著高于未达到CR的患者（非CR组）以及未接受PCT的患者（非PCT组）[（80.0±8.3）% （38.1±10.6）% （56.1±9.3）%，P =0.010]。PCT显著提高了MDS-AML患者的OS率[（62.2±!±10.0）%（20.0±17.9）%，P =0.013]，但对MDS-EB2患者无此作用[（59.2±11.1）%（62.9±10.1）%，P =0.991]。我们的研究结果表明，通过减瘤治疗降低肿瘤负荷以在移植前获得CR可改善OS。对于MDS-AML患者，PCT有益，而对于MDS-EB2患者，PCT并非必要。
