University of Wisconsin Carbone Cancer Center, 600 Highland Ave, Madison, WI, 3792, USA.
Curr Hematol Malig Rep. 2021 Apr;16(2):172-182. doi: 10.1007/s11899-021-00623-7. Epub 2021 May 13.
Smoldering multiple myeloma (SMM) is defined as an asymptomatic clonal proliferation of pre-malignant plasma cells and an increased risk of progression to multiple myeloma (MM) relative to monoclonal gammopathy of undetermined significance. Whether patients with SMM should be treated prior to development of symptomatic disease is fiercely debated and is a highly active area of research.
The ECOG E3A06 study demonstrated that early treatment with lenalidomide significantly reduced the risk of progression to MM compared to observation in patients with high risk SMM. The IMWG recently validated a risk stratification model to include cytogenetics and a personalized risk calculator for individual patients. Beyond this, molecular genomic aberrations and immunological phenomena that promote progression from asymptomatic disease to MM have been recently characterized and may help to more precisely identify patients who are most suitable for early intervention. As highly effective and tolerable therapies for plasma cell disorders evolve, the field is approaching a paradigm shift that involves the adoption of intervention for patients with SMM who are at high risk for progression to symptomatic myeloma in order to prevent morbidity and mortality. This review highlights our current understanding of the biology of patients with SMM, clarifies the rationale for early intervention, and summarizes early results of various treatment strategies for patients with high-risk smoldering myeloma.
冒烟型多发性骨髓瘤（SMM）定义为恶性前浆细胞的克隆性增生，与意义未明的单克隆丙种球蛋白血症相比，进展为多发性骨髓瘤（MM）的风险增加。SMM 患者是否应该在出现症状性疾病之前进行治疗存在激烈争议，这是一个高度活跃的研究领域。
ECOG E3A06 研究表明，与观察相比，高危 SMM 患者用来那度胺早期治疗可显著降低进展为 MM 的风险。最近，国际骨髓瘤工作组（IMWG）验证了一种风险分层模型，包括细胞遗传学和针对个体患者的个性化风险计算器。除此之外，最近还对促进无症状疾病进展为 MM 的分子基因组异常和免疫现象进行了特征描述，这可能有助于更精确地识别最适合早期干预的患者。随着针对浆细胞疾病的高度有效和耐受治疗方法的发展，该领域正在发生范式转变，涉及对有进展为症状性骨髓瘤高风险的 SMM 患者进行干预，以预防发病率和死亡率。这篇综述强调了我们对 SMM 患者生物学的现有认识，阐明了早期干预的基本原理，并总结了高危冒烟型骨髓瘤患者各种治疗策略的早期结果。
