Multiple Myeloma Section, Metabolism Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
Eur J Haematol. 2014 Jan;92(1):1-12. doi: 10.1111/ejh.12205. Epub 2013 Nov 26.
Since smoldering multiple myeloma (SMM) was first described over three decades ago based on a case series of six patients, its definition and our understanding of the entity have evolved considerably. The risk of progression to symptomatic myeloma (MM) varies greatly among individuals diagnosed with myeloma precursor disease. Epidemiologic, molecular, flow cytometric and radiological techniques have demonstrated that this transformation to MM from precursor states is not sudden but rather a continuous overlapping series of events with evidence of end-organ damage that could manifest in the earliest stages of disease. Contemporary antimyeloma therapies can yield rapid, deep, and durable responses with manageable toxicities, and molecular-cell-based measures are now available to rule out minimal residual disease. With this information, clinical studies with correlative measures can now be developed to test the fundamental hypothesis that intervention in early myeloma may provide a measurable clinical benefit to patients by either delaying progression or eradicating plasma cell clones.
自三十多年前基于六例患者的病例系列首次描述冒烟型多发性骨髓瘤(SMM)以来,其定义和我们对该实体的认识已经发生了很大变化。诊断为骨髓瘤前体疾病的个体进展为有症状骨髓瘤(MM)的风险差异很大。流行病学、分子、流式细胞术和影像学技术已经表明,从前体状态向 MM 的这种转变并非突然发生,而是一系列连续重叠的事件,有证据表明终末器官受损,这可能在疾病的最早阶段表现出来。当代抗骨髓瘤疗法可以产生快速、深度和持久的反应,且毒性可控制,现在还可以采用基于分子和细胞的措施来排除微小残留疾病。有了这些信息,现在可以制定具有相关性措施的临床研究,以检验以下基本假设:早期骨髓瘤的干预可能通过延迟进展或消除浆细胞克隆,为患者提供可衡量的临床获益。