自体造血干细胞移植治疗多发性骨髓瘤53例的疗效评价及预后因素分析

Efficacy and prognostic factors of autologous hematopoietic stem cell transplantation for multiple myeloma: clinical analysis of 53 cases

目的评估自体造血干细胞移植(autologous hematopoietic stem cell transplantation, auto-HSCT)对多发性骨髓瘤(multiple myeloma, MM)患者的疗效及其影响预后的因素。方法回顾性分析2010-2019年在本院诱导化疗序贯auto-HSCT的53例MM患者资料,观察其治疗效果,分析影响其预后的因素。结果 53例患者均获得造血重建,未出现移植相关死亡病例。患者移植后有效缓解率(CR+VGPR)为84.9%,显著高于移植前的52.8%(P<0.001)。46例单次移植患者,移植前后有效缓解率分别为52.2%和84.8%,差异有统计学意义(P=0.001);7例连续双次移植患者,移植前后有效缓解率分别为57.1%和85.7%,差异无统计学意义(P=0.559)。单次移植和连续双次移植的PFS和OS比较,差异均无统计学意义(P>0.05)。年龄≥50岁、R-ISS分期Ⅲ期、确诊到移植时间≥180 d、危险分层高危、移植前和移植后疾病状态在部分缓解(partial remission, PR)及其以下、移植前和移植后微小残留病灶(minimal residual disease, MRD)检测阳性是预后不良的影响因素。结论 Auto-HSCT治疗MM患者安全有效,能提高缓解率,改善预后;单次移植较连续双次移植效果好。患者R-ISS分期、危险分层、移植前和移植后疾病状态以及MRD与预后密切相关。

Objective To evaluate the efficacy of autologous hematopoietic stem cell transplantation(auto-HSCT) on multiple myeloma(MM) and to investigate the factors affecting the prognosis. Methods The clinical data of 53 MM patients treated with auto-HSCT in our hospital from January 2010 to December 2019 were collected and retrospectively analyzed. The therapeutic effect was observed and the prognostic factors were analyzed. Results Hematopoietic reconstruction was achieved in all 53 patients, and no transplant-related death occurred. As complete remission(CR) and very good partial remission(VGPR) were defined as effective response, the rate was 84.9% after transplantation, significantly higher than that before treatment(52.8%)(P<0.001). The effective remission rates were 52.2% and 84.8%, respectively, before and after treatment in the 46 patients with single transplantation(P=0.001). However, the rates were 57.1% and 85.7% respectively for the 7 patients with consecutive double transplantation(P=0.559). There were no significant differences in progression-free survival(PFS) and overall survival(OS) between the single and continuous double transplantation patients(P>0.05). Influential factors for poor prognosis were identified as: age≥50 years, stage Ⅲ by revised international staging system(R-ISS), time from diagnosis to transplantation ≥180 d, high-risk stratification, partial remission(PR) or worse before and after transplantation, as well as positive results of minimal residual disease(MRD) test before and after treatment. Conclusion Auto-HSCT is safe and effective in MM patients, and can significantly improve the remission rate and prognosis. Single transplantation is better than continuous double treatment in the effectiveness. R-ISS stage, risk stratification, disease status before and after transplantation, and result of MRD test are closely associated with the prognosis of the patients.