含硼替佐米方案的诱导治疗序贯自体造血干细胞移植治疗初诊时合并髓外病变的多发性骨髓瘤的疗效分析

Efficacy analysis of bortezomib-based induction followed with autologous hematopoietic stem cell transplantation in newly diagnosed multiple myeloma with extramedullary disease

目的:分析含硼替佐米诱导治疗序贯自体造血干细胞移植(ASCT)的治疗方案对初诊时合并髓外病变(EMD)的多发性骨髓瘤(MM)患者预后的影响。方法:回顾性分析244例初诊MM患者的临床资料,主要研究终点为总生存(OS)和无进展生存(PFS)。结果:入选的244例初诊患者中合并EMD者66例(27.0%)。244例患者按治疗方案分为3组:传统化疗组59例,持续新药治疗组87例,新药序贯ASCT组98例。传统化疗组中伴有EMD患者的中位PFS显著低于不伴EMD患者(7.433∶25.400个月,P=0.024),持续新药治疗组中伴有EMD患者的中位PFS与不伴EMD患者比较,差异无统计学意义(15.133∶27.100个月,P=0.269),硼替佐米序贯ASCT组中伴有EMD患者的中位PFS与不伴EMD患者比较,差异亦无统计学意义(46.667∶44.867个月,P=0.743)。在EMD患者中,硼替佐米序贯ASCT组与传统化疗组及持续新药治疗组相比,均可显著提高患者的中位PFS(P<0.05)。传统化疗组中伴有EMD患者的中位OS显著低于不伴EMD患者(21.033∶34.667个月,P=0.043),持续新药治疗组中伴有EMD患者的中位OS显著低于不伴EMD患者(10.400∶44.300个月,P=0.004),而在硼替佐米序贯ASCT组中伴有EMD与不伴EMD患者间OS比较,差异无统计学意义(P=0.325)。在EMD患者中,硼替佐米序贯ASCT组与传统化疗组及持续新药治疗组相比,均可显著提高患者的中位OS(P<0.005)。结论:含硼替佐米诱导治疗序贯ASCT的治疗方案可显著提高初诊伴EMD的MM患者的生存,克服EMD的不良预后影响。

Objective：To determine the effect of bortezomib-based induction followed with autologous hematopoietic stem cell transplantation（ASCT）on the outcomes of newly-diagnosed multiple myeloma（MM）patients with extramedullary disease（EMD）.Method：A total of 244newly-diagnosed and untreated symptomatic MM patients were retrospectively analyzed.The primary endpoints were overall survival（OS）and progression free survival（PFS）.Result：Sixty-six（27.0%）of the study population had EMD at diagnosis.The subjects were divided into three groups according to treatment strategies：conventional chemotherapy（n=59）,novel agents-based regimens without ASCT（n=87）,and bortezomib-based induction followed with ASCT（n=98）.In the conventional chemotherapy group,PFS of patients with EMD was significantly shorter than that of patients without EMD（7.433 vs.25.400 months,P=0.024）.However,in the group treated with novel agents and the group treated with bortezomib-based induction followed with ASCT,there was no significant difference in the median PFS between EMD patients and those without EMD.Bortezomib-based induction followed with ASCT improved PFS in EMD patients as compared to those treated with conventional chemotherapy or novel agents（P〈0.05）.In the conventional treatment group and novel drugs without ASCT treatment group,the median OS of patients with EMD was significantly shorter than those without EMD（21.033 vs.34.667 months,P=0.043;10.400 vs.44.300 months,P=0.004）.While in the group treated with bortezomib-based induction followed with ASCT,there was no significant difference in median OS between the patients with and without EMD（P=0.325）.Bortezomib-based induction followed with ASCT improved OS in EMD patients as compared to those treated with conventional chemotherapy or novel agents only（P〈0.005）.Conclusion：Bortezomib-based induction followed with ASCT significantly improves survival of newly diagnosed MM with EMD,and overcomes the negative prognostic impact of EMD on MM.