A wide variety of new therapeutic options for Multiple Myeloma (MM) have recently become available, extending progression-free and overall survival for patients in meaningful ways. However, these treatments are not curative, and patients eventually relapse, necessitating decisions on the appropriate choice of treatment(s) for the next phase of the disease. Additionally, an important subset of MM patients will prove to be refractory to the majority of the available treatments, requiring selection of effective therapies from the remaining options. Immunomodulatory agents (IMiDs), proteasome inhibitors, monoclonal antibodies, and alkylating agents are the major classes of MM therapies, with several options in each class. Patients who are refractory to one agent in a class may be responsive to a related compound or to a drug from a different class. However, rules for selection of alternative treatments in these situations are somewhat empirical and later phase clinical trials to inform those choices are ongoing. To address these issues the NCI Multiple Myeloma Steering Committee formed a relapsed/refractory working group to review optimal treatment choices, timing, and sequencing and provide recommendations. Additional issues considered include the role of salvage autologous stem cell transplantation, risk stratification, targeted approaches for genetic subsets of MM, appropriate clinical trial endpoints, and promising investigational agents. This report summarizes the deliberations of the working group and suggests potential avenues of research to improve the precision, timing, and durability of treatments for Myeloma.
近年来，多种针对多发性骨髓瘤（MM）的新型治疗方案相继问世，显著延长了患者的无进展生存期和总生存期。然而，这些治疗手段尚无法根治疾病，患者最终仍会复发，因此需要为疾病下一阶段的治疗选择合适的方案。此外，相当一部分多发性骨髓瘤患者会对大多数现有治疗方案产生耐药性，这就需要从剩余选项中筛选有效疗法。免疫调节剂、蛋白酶体抑制剂、单克隆抗体和烷化剂是多发性骨髓瘤的主要治疗类别，每类中均有多种选择。对某类药物中的一种制剂耐药的患者，可能对同类其他相关化合物或不同类别的药物产生反应。然而，在这些情况下选择替代治疗方案的规则尚缺乏充分依据，目前正在进行后期临床试验以指导相关选择。为解决这些问题，美国国家癌症研究院多发性骨髓瘤指导委员会成立了一个复发/难治性工作组，旨在评估最佳治疗方案、治疗时机与顺序，并提出建议。工作组还探讨了其他相关问题，包括挽救性自体干细胞移植的作用、风险分层、针对多发性骨髓瘤特定基因亚群的靶向疗法、恰当的临床试验终点以及具有前景的研究性药物。本报告总结了工作组的讨论内容，并为提升多发性骨髓瘤治疗的精准性、时效性和持久性提出了潜在研究方向。
肿瘤(癌症)患者之家