Rajkumar S Vincent, Mateos María-Victoria, Schaeffer Marcy, Lin Xiwu, Bathija Sacheeta, Gupta-Werner Niodita, Lam Annette, Carson Robin, Dennis Robyn, Kaila Shuchita, Matt Kathryn, Duran Joana, Lonial Sagar
Mayo Clinic, Rochester, MN, USA.
Hospital Universitario de Salamanca, Instituto de Investigación Biomédica de Salamanca (IBSAL), Centro de Investigación del Cancer (IBMCC-USAL, CSIC), Salamanca, Spain.
Blood Cancer J. 2024 Dec 5;14(1):215. doi: 10.1038/s41408-024-01170-z.
This study aimed to provide real-world evidence on progression risk in patients with high-risk smoldering multiple myeloma (SMM). This retrospective, observational study leveraged data from the Flatiron Health database. Eligible patients had SMM and relevant measures to apply Mayo 2018, International Myeloma Working Group (IMWG) 2020, and AQUILA trial risk criteria. Time to progression to active MM (TTP), progression or death (PFS), and death or progression on first-line MM therapy (PFS2) were evaluated using Kaplan-Meier methods and multivariate Cox regression models adjusted for age, Charlson Comorbidity Index, and time from SMM diagnosis to risk classification date. Across the three risk models (Mayo 2018, IMWG 2020, and AQUILA trial), high-risk patients with SMM had 3.0-4.0 times the risk of TTP, 2.1-3.5 times the risk of PFS, and 1.7-3.2 times the risk of PFS2 versus non-high-risk patients (p < 0.001 for all comparisons). Similar results were observed when patients with early treatment, early progression, and/or bone disease were excluded. This study demonstrates that high-risk patients with SMM have worse prognoses than non-high-risk patients, regardless of the criteria used, and highlights a need for early intervention testing.
本研究旨在提供关于高危冒烟型多发性骨髓瘤（SMM）患者疾病进展风险的真实世界证据。这项回顾性观察性研究利用了Flatiron Health数据库中的数据。符合条件的患者患有SMM，并采用了相关指标来应用梅奥2018版、国际骨髓瘤工作组（IMWG）2020版以及AQUILA试验的风险标准。使用Kaplan-Meier方法和多变量Cox回归模型评估进展为活动性骨髓瘤的时间（TTP）、进展或死亡（PFS）以及一线骨髓瘤治疗中的死亡或进展（PFS2），模型针对年龄、Charlson合并症指数以及从SMM诊断到风险分类日期的时间进行了调整。在三种风险模型（梅奥2018版、IMWG 2020版和AQUILA试验）中，与非高危患者相比，高危SMM患者的TTP风险高3.0至4.0倍，PFS风险高2.1至3.5倍，PFS2风险高1.7至3.2倍（所有比较的p值均<0.001）。排除早期治疗、早期进展和/或骨病患者后，观察到了类似结果。本研究表明，无论使用何种标准，高危SMM患者的预后均比非高危患者差，并强调了早期干预检测的必要性。
