摘要目的 评价异基因造血干细胞移植前诱导治疗对高危组骨髓增生异常综合征(MDS)患者移植预后的影响.方法 收集2002年11月至2012年12月苏州大学附属第一医院接受异基因造血干细胞移植的49例MDS患者的临床资料,按照国际预后评分系统(IPSS)积分、骨髓原始细胞比例等因素进行比较,评估移植前诱导治疗对其生存、复发和移植相关死亡率(TRM)等预后的影响.结果 移植后中位随访时间24.4(6.2～72.0)个月,死亡17例,复发2例.5年总体生存(OS)率、无病生存(DFS)率、复发率和TRM分别为59.9％、59.2％、10.5％和31.8％.高危诱导治疗组(n=17)、高危支持治疗组(n=6)和低危组(n=26)患者间OS、DFS率差异有统计学意义(72.1％比16.7％比68.1％,P=0.028；72.1％比16.7％比67.9％,P=0.030)；而无论诱导治疗是否获得骨髓缓解,高危诱导治疗组的OS、DFS均与低危组相当(P=0.526、0.504),高危诱导治疗组OS、DFS明显优于高危支持治疗组(均P =0.020).高危诱导治疗组中获得缓解者OS、DFS均高于未缓解者(均为100％比46.7％,P=0.049).高危组诱导治疗后骨髓原始细胞负荷明显降低(P=0.010),但累积TRM并未高于支持治疗组(28.9％比33.6％,P=0.612).结论 MDS异基因造血干细胞移植前诱导治疗可降低肿瘤负荷,改善高危组移植后结果,而TRM并未增高.

abstractsObjective To explore the impact of prior-to-transplantation induction therapy (IT) on patient outcome after allogeneic hematopoietic stem-cell transplantation (Allo-HSCT) for higher-risk myelodysplastic syndromes (MDS).Methods A total of 49 consecutive patients underwent Allo-HSCT for MDS between November 2002 and December 2012.Twenty-six lower-risk MDS cases received supportive therapy (ST).And 17/23 cases of higher-risk MDS received IT prior to transplantation while anothers 6 only with ST.Their survival,relapse rate and incidence of transplantation-related mortality (TRM) were retrospectively analyzed according to International Prognostic Scoring System (IPSS) scores and marrow blast count.Results The 5-year cumulative overall survival (OS),disease-free survival (DFS),relapse rate and incidence of transplantation related mortality (TRM) were 59.9％,59.2％,10.5％ and 31.8％ during a median follow-up period of 24.4 (6.2-72.0) months.The OS and DFS of higher-risk group with IT,ST and lower-risk group were different (72.1％ vs 16.7％ vs 68.1％,P =0.028; 72.1％ vs 16.7％ vs 67.9％,P =0.030).And the OS and DFS of higher-risk group with IT were similar to those of lower-risk group (P =0.526,0.504).For the higher-risk group,the patients on IT had improved survival than those on ST in terms of OS and DFS (both P =0.020).Moreover,the OS and DFS of remission group were higher than non-remission group in patients on IT (both 100％ vs 46.7％,P =0.049).The number of marrow blasts significantly decreased after IT (P =0.010) without increased TRM (28.9％ vs 33.6％,P =0.612).Conclusion Induction therapy prior to Allo-HSCT for MDS may reduce clone burden and improve the outcomes of higher-risk MDS without increased TRM.