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.
本研究旨在为高危冒烟型多发性骨髓瘤（SMM）患者的进展风险提供真实世界证据。这项回顾性观察性研究利用了Flatiron Health数据库的数据。符合入选标准的患者需确诊SMM，并具备应用2018年梅奥标准、2020年国际骨髓瘤工作组（IMWG）标准及AQUILA试验风险标准的相关评估指标。研究通过Kaplan-Meier法和经年龄、查尔森合并症指数、从SMM诊断到风险分类日期时间校正的多变量Cox回归模型，评估了进展为活动性MM的时间、疾病进展或死亡时间以及一线MM治疗期间的死亡或进展时间。在三种风险模型（2018年梅奥标准、2020年IMWG标准和AQUILA试验标准）中，与非高危患者相比，高危SMM患者的疾病进展风险升高3.0-4.0倍，进展或死亡风险升高2.1-3.5倍，一线治疗期间死亡或进展风险升高1.7-3.2倍（所有比较p值均<0.001）。即使在排除早期治疗、早期进展和/或骨病患者后，仍观察到相似结果。本研究证明无论采用何种风险标准，高危SMM患者的预后均劣于非高危患者，并强调了开展早期干预试验的必要性。
肿瘤(癌症)患者之家