Objective: To evaluate the efficacy and long-term outcome of a combined protocol for multiple myeloma (MM) , including induction therapy, autologous hematopoietic stem cell transplantation (ASCT) and consolidation and maintenance therapy. Methods: Clinical records of 144 patients with MM from January 1, 2005 to February 1, 2016 were retrospectively analyzed. Results: The overall response rate (ORR) after ASCT was 100.0%, in which the complete remission (CR) was 64.1% and the best treatment response rate of superior to PR was 89.4%. During a median follow-up of 47 months, patients with an overall survival (OS) and progression free survival (PFS) was 120.9 and 56.9 months respectively. 5y-OS (73.7±4.7) %, 7y-OS (60.5±6.3) %; 3y-PFS (69.2±4.2) %, 5y-PFS (47.8±5.3) %. The median OS and PFS between the first line transplantation group and salvage transplantation group were 120.9 months vs 50.1 months and 60.2 months vs 16.7 months (all P=0.000). In 127 patients with R-ISS staging, the median survival of Ⅰ, Ⅱ, Ⅲ stage was 120.9 months (n=43) , 88.4 months (n=64) , 35.6 months (n=20) , respectively (P=0.000). For subgroup analysis of survival in early and late ASCT, the median OS of patients with R-ISS stage Ⅲ (35.6 months vs 15.8 months, P=0.031) and the median PFS of two groups (phase Ⅰ: 72.1 months vs 18.9 months, P=0.000; Ⅱ: 53.4 months vs 16.7 months, P=0.012; Ⅲ: 28.5 months vs 5.9 months, P=0.001) were different. Multivariate analysis showed that only R-ISS and the degree of remission before transplantation had impact on OS (HR=8.486, 95% CI 2.549-28.255, P=0.003) and PFS (HR=2.412, 95% CI 1.364-4.266, P=0.002) , respectively. Conclusion: The combined protocol containing ASCT is effective for MM patients, improving remission rate and remission depth, prolonging PFS and OS. First line transplantation could significantly prolong the OS and PFS as compared with salvage transplantation. R-ISS and pre-transplantation remission depth are prognostic factors for survival.
目的： 评价诱导治疗联合自体周血造血干细胞移植（ASCT）治疗、移植后巩固维持治疗的整体方案治疗多发性骨髓瘤（MM）患者的有效性和长期随访结果。 方法： 回顾性分析2005年1月1日至2016年2月1日接受整体方案治疗的144例MM患者资料，总结其长期随访结果，分析移植前疗效、移植后缓解深度，以及早期移植、晚期移植、不同预后分期系统等因素对患者生存的影响。 结果： 144例患者中获得部分缓解（PR）以上治疗反应率为89.4%[完全缓解（CR）率64.1%]。中位随访47（6~121）个月，患者中位总体生存（OS）和无进展生存（PFS）时间分别为120.9和56.9个月；早期和晚期移植组患者的中位OS分别为120.9和50.1个月，中位PFS时间分别为60.2和16.7个月（P值均<0.001）。127例可进行修订的国际分期系统（R-ISS）分期患者中，Ⅰ期（43例）、Ⅱ期（64例）、Ⅲ期（20例）患者的中位OS时间分别为120.9、88.4、35.6个月，组间差异有统计学意义（P=0.000）。对早期和晚期移植组患者进行亚组生存分析，R-ISS Ⅲ期患者中位OS时间差异有统计学意义（35.6个月对15.8个月，P=0.031）；两组患者各期中位PFS时间差异均有统计学意义（Ⅰ期：72.1个月对18.9个月；Ⅱ期：53.4个月对16.7个月；Ⅲ期：28.5对5.9个月，P值分别为0.000、0.012、0.001）。多因素分析结果显示R-ISS Ⅲ期是影响患者OS的危险因素（HR=8.486，95%CI 2.549~28.255，P=0.003），移植后未获得CR是影响患者PFS的危险因素（HR=2.412，95%CI 1.364~4.266，P=0.002）。 结论： 以新药为基础的化疗联合ASCT的整体治疗方案对适合移植的MM患者是有效的，可进一步提高缓解率和缓解深度，延长患者的PFS和OS时间。早期较晚期移植可明显延长患者的OS和PFS时间。R-ISS分期和移植前疗效是影响患者生存的预后因素。.
Keywords: Hematopoietic stem cell transplantation; Multiple myeloma; Prognosis.