【摘要】 目的 分析巨灶型多发性骨髓瘤(MFMM)患者的临床特征、治疗方案及预后情况，为MFMM的临床管理和治疗提供重要依据。方法 采用病例对照研究设计，以2019年1月—2024年12月就诊于西安交通大学第二附属医院的MFMM患者为病例组，以诊断时间相近、诱导治疗方案相似的新诊断经典MM患者为对照组进行1∶2匹配，收集社会人口学信息、临床特征、实验室检查结果、遗传学特征、治疗方案、疗效及预后情况，采用非参数检验、卡方检验进行组间差异比较，采用Kaplan-Meier法及log-rank检验进行预后比较。结果 纳入MFMM病例组共27例、经典MM对照组54例。MFMM病例组初诊中位年龄显著低于经典MM对照组(Z=2.328,P=0.020)。所有MFMM患者均为ISSⅠ、Ⅱ期，对照组中ISSⅠ期6例(11.1%)、ISSⅡ期23例(42.6%),MFMM病例组的ISS分期与经典MM对照组存在显著差异(χ~2=33.770,P<0.001)。mSMART3.0分层，MFMM病例组的标危比例显著高于经典MM对照组(χ~2=10.408,P=0.005)。MFMM病例组与经典MM对照组的诱导治疗方案、自体移植率相似，达到最佳疗效(CR)比例无显著差异(59.3%vs 46.3%,P=0.067)。MFMM患者中，诱导治疗方案为PIs为主、ImiDs为主、PIs+ImiDs的患者之间的疗效差异无统计学意义(χ~2=8.735,P=0.068)。截至2024年12月，MFMM病例组的无进展生存期(PFS)(P=0.017)、总生存期(OS)(P=0.041)均显著优于经典MM对照组。结论 MFMM是一种相对惰性的多发性骨髓瘤亚型，患者初诊年龄较轻，肿瘤负荷较低，在新药时代下，不同诱导方案对MFMM患者均敏感，预后较好。更多还原

【Abstract】 Objective To analyze the clinical characteristics, treatment regimens, and prognosis of patients with macrofocal multiple myeloma(MFMM), providing critical evidence for clinical management and treatment of MFMM. Methods In this retrospective case-control study, 27 MFMM patients diagnosed at the Second Affiliated Hospital of Xi’an Jiaotong University(January 2019 to December 2024) were compared with 54 matched classical MM controls(1:2 ratio) sharing comparable induction regimens and diagnostic timelines. Comprehensive datasets encompassing demographics, clinicopathological profiles, cytogenetic abnormalities, treatment responses, and survival outcomes were analyzed. Nonparametric tests and chi-square tests were used for intergroup comparisons, and Kaplan-Meier analysis and log-rank tests were applied for survival analysis. Results The study included 27 MFMM cases and 54 classical MM controls. The median age at diagnosis in the MFMM group was significantly younger than in the classical MM group(Z=2.328, P=0.020). All MFMM patients were classified as ISS stage Ⅰ or Ⅱ, whereas the control group included 6 ISS stage Ⅰ(11.1%) and 23 ISS stage Ⅱ(42.6%) cases, with significant differences in ISS staging between the two groups(χ~2=33.770, P<0.001). Stratification by mSMART3.0 revealed a significantly higher proportion of standard-risk patients in the MFMM group(χ~2=10.408, P=0.005). Induction regimens and autologous stem cell transplantation rates were similar between the two groups, with no significant difference in complete response(CR) rates(59.3% vs. 46.3%, P=0.067). Among MFMM patients, no statistically significant differences in efficacy were observed across proteasome inhibitor(PI), immunomodulatory drug(IMiD), or PI+IMiD(χ~2=8.735, P=0.068). By December 2024, MFMM patients demonstrated significantly longer progression-free survival(PFS)(P=0.017) and overall survival(OS)(P=0.041) compared to classical MM controls.Conclusion MFMM represents a relatively indolent MM subtype characterized by younger age at diagnosis, lower tumor burden, and favorable prognosis in the era of novel therapies. Diverse induction regimens show comparable efficacy in MFMM patients.更多还原