Division of Hematology, Mayo Clinic, 200 First St SW, Rochester, Minnesota 55905, USA.
Department of Haematology, Erasmus Medical Center, Rotterdam, The Netherlands.
Nat Rev Dis Primers. 2017 Jul 20;3:17046. doi: 10.1038/nrdp.2017.46.
Multiple myeloma is a malignancy of terminally differentiated plasma cells, and patients typically present with bone marrow infiltration of clonal plasma cells and monoclonal protein in the serum and/or urine. The diagnosis of multiple myeloma is made when clear end-organ damage attributable to the plasma cell proliferative disorder or when findings that suggest a high likelihood of their development are present. Distinguishing symptomatic multiple myeloma that requires treatment from the precursor stages of monoclonal gammopathy of undetermined significance and smouldering multiple myeloma is important, as observation is the standard for those conditions. Much progress has been made over the past decade in the understanding of disease biology and individualized treatment approaches. Several new classes of drugs, such as proteasome inhibitors and immunomodulatory drugs, have joined the traditional armamentarium (corticosteroids, alkylating agents and anthracyclines) and, along with high-dose therapy and autologous haemopoietic stem cell transplantation, have led to deeper and durable clinical responses. Indeed, an increasing proportion of patients are achieving lasting remissions, raising the possibility of cure for this disease. Success will probably depend on using combinations of effective agents and treating patients in the early stages of disease, such as patients with smouldering multiple myeloma.
多发性骨髓瘤是一种终末分化的浆细胞恶性肿瘤,患者通常表现为骨髓中克隆性浆细胞浸润,血清和/或尿中有单克隆蛋白。当浆细胞增殖性疾病引起明确的终末器官损害,或存在高度提示其发展的发现时,即可诊断多发性骨髓瘤。区分需要治疗的有症状多发性骨髓瘤与意义未明的单克隆丙种球蛋白血症和冒烟型多发性骨髓瘤的前期阶段很重要,因为这些情况下的标准是观察。在过去十年中,人们对疾病生物学和个体化治疗方法的理解取得了很大进展。几种新的药物类别,如蛋白酶体抑制剂和免疫调节剂,已经加入了传统的治疗方法(皮质类固醇、烷化剂和蒽环类药物),以及大剂量化疗和自体造血干细胞移植,导致了更深和更持久的临床反应。事实上,越来越多的患者实现了持久缓解,这增加了治愈这种疾病的可能性。成功可能取决于使用有效的联合药物治疗和在疾病早期治疗患者,如冒烟型多发性骨髓瘤患者。