René Gauducheau Cancer Center, Boulevard Jacques Monod, Nantes/St Herblain, France.
Clin Lymphoma Myeloma Leuk. 2010 Dec;10(6):424-42. doi: 10.3816/CLML.2010.n.076.
In 2000, the most important therapeutic improvement in multiple myeloma (MM) had been the introduction of high-dose therapy (HDT) supported by autologous stem cell transplantation, but this strategy was proposed only to younger patients without comorbidities. In the past decade, progress in the management of MM has been dramatic and more rapid than in the four previous decades. The main reason for this has been the introduction of three novel agents in less than five years. These three agents, two immunomodulatory agents (IMIDs; thalidomide and lenalidomide) and one proteasome inhibitor (bortezomib) have been initially used in relapsed or refractory patients and rapidly added to frontline therapy both in younger and older patients. They have already increased overall survival but are also changing the objectives of treatment and the therapeutic strategy; other important advances have been made during these ten years with new diagnostic and staging procedures and new prognostic factors. These clinical improvements have been possible mostly by a better understanding of the disease pathophysiology, including genetic changes, and by the development of preclinical in vitro and vivo tests to better evaluate the potential clinical activity of new agents.
2000 年，多发性骨髓瘤（MM）最重要的治疗进展是采用大剂量化疗（HDT）联合自体造血干细胞移植，但该策略仅适用于无合并症的年轻患者。过去十年中，MM 的治疗进展迅速，比前四十年更为迅速。主要原因是不到五年内引入了三种新的药物。这三种药物，两种免疫调节剂（IMIDs；沙利度胺和来那度胺）和一种蛋白酶体抑制剂（硼替佐米）最初用于复发或难治性患者，并迅速在年轻和老年患者的一线治疗中得到应用。它们已经提高了总体生存率，但也改变了治疗目标和治疗策略；在这十年中，还取得了其他重要进展，包括新的诊断和分期程序以及新的预后因素。这些临床进展主要得益于对疾病病理生理学的更好理解，包括遗传变化，以及开发了临床前体外和体内试验，以更好地评估新药物的潜在临床活性。
