IPSS-R及WPSS移植前评估对异基因造血干细胞移植治疗骨髓增生异常综合征患者预后意义的研究

The prognostic significance of pretransplantation evaluation of IPSS-R and WPSS in patients with myelodysplastic syndrome undergoing allogeneic hematopoietic stem cell transplantation

目的探究不同时间进行修订版国际预后积分系统(IPSS-R)及WHO预后积分系统(WPSS)评分对异基因造血干细胞移植(allo-HSCT)治疗骨髓增生异常综合征(MDS)患者的预后评估价值。方法回顾性分析2016年7月至2019年6月于苏州大学附属第一医院行allo-HSCT的184例MDS患者临床资料,在初诊及移植前两个时间点进行IPSS-R及WPSS评分,比较两者预后评估价值,分析影响移植预后的危险因素。结果中位随访21.9(0.5~47.5)个月,移植后2年总体生存(OS)、无进展生存(PFS)率分别为(75.1±3.4)%、(71.6±3.6)%,2年复发(RR)率和无复发死亡率(NRM)分别为(11.9±0.1)%、(16.5±0.1)%。初诊时IPSS-R≤3.5分与>3.5分组OS、PFS差异无统计学意义(P=0.409;P=0.724),WPSS≤2分与>2分组OS、PFS差异无统计学意义(P=0.462;P=0.726)。移植前除外10例患者转化为急性髓系白血病(AML),174例患者可进行IPSS-R及WPSS评估,其中IPSS-R≤3.5分组OS、PFS率显著优于>3.5分组[OS:(88.6±4.1)%对(65.8±5.3)%,P=0.003;PFS:(87.6±4.2)%对(60.5±5.8)%,P=0.002],WPSS≤2分与>2分组间OS、PFS差异无统计学意义(P=0.584;P=0.565)。此外,移植前IPSS-R较初诊时相比改善组OS、PFS率显著优于未改善组[OS:(83.8±4.6)%对(69.3±5.8)%,P=0.027;PFS:(82.8±4.4)%对(64.0±7.2)%,P=0.006],多因素分析发现移植前TP53突变(P=0.047,HR=2.460,95%CI 1.014~5.971)和移植前IPSS-R>3.5分(P=0.021,HR=2.510,95%CI 1.151~5.476)是影响OS的独立不良预后因素,移植前细胞遗传学差及极差(P=0.008,HR=2.765,95%CI 1.305~5.856)和移植前IPSS-R>3.5分(P=0.017,HR=2.457,95%CI 1.175~5.141)是影响PFS的独立不良预后因素。结论移植前进行IPSS-R评分对行allo-HSCT的MDS患者的预后评估有重要价值,移植前IPSS-R较初诊时改善的患者有着更好的预后。

Objective This study aimed to explore the prognostic value of the revised international prognostic scoring system(IPSS-R)and the WHO prognostic scoring system(WPSS)in patients with myelodysplastic syndrome(MDS)undergoing allogeneic hematopoietic stem cell transplantation(allo-HSCT).Methods The clinical data of 184 patients with MDS who received allo-HSCT from July 2016 to June 2019 were retrospectively analyzed.IPSS-R and WPSS were performed at diagnosis and before transplantation.The prognostic values of IPSS-R and WPSS and potential risk factors were explored.Results With a median follow-up of 21.9(0.5-47.5)months,the two-year overall survival(OS)and progression-free survival(PFS)rates were(75.1±3.4)% and(71.6±3.6)%,respectively.The two-year cumulative relapse rate and nonrelapse mortality rate were(11.9±0.1)% and(16.5±0.1)%,respectively.There were no significant differences in OS and PFS between the IPSS-R≤3.5 and>3.5 groups at diagnosis(P=0.409;P=0.724).No significant differences in OS and PFS between the WPSS≤2 and>2 groups(P=0.426;P=0.726)were observed as well.When the patients were reevaluated before transplantation,the OS and PFS of the IPSS-R≤3.5 group were significantly better than>3.5 group[OS:(88.6±4.1)%vs(65.8±5.3)%,P=0.003;PFS:(87.6±4.2)%vs(60.5±5.8)%,P=0.002].However,there were no significant differences in OS and PFS among the WPSS≤2 and>2 groups(P=0.584;P=0.565).In addition,the OS and PFS of the improved group based on IPSS-R were significantly better than those of the unimproved group before transplantation[OS:(83.8±4.6)%vs(69.3±5.8)%,P=0.027;PFS:(82.8±4.4)%vs.(64.0±7.2)%,P=0.006].Multivariate analysis indicated that a pretransplant IPSS-R of>3.5(P=0.021,HR=2.510,95%CI 1.151-5.476)and TP53 mutation(P=0.047,HR=2.460,95%CI 1.014-5.971)were independent risk factors for OS,whereas a pretransplant IPSS-R of>3.5(P=0.017,HR=2.457,95%CI 1.175-5.141)and pretransplant cytogenetic poor and very poor(P=0.008,HR=2.765,95%CI 1.305-5.856)were independent risk factors for PFS.Conclusion A pretransplantation evaluation of IPSS-R could help determine the prognosis of patients with MDS undergoing allo-HSCT.In addition,patients with improved IPSS-R scores before undergoing allo-HSCT had a better prognosis.