Gandolfi Sara, Vekstein Carolyn, Laubach Jacob P, O'Brien Alexandra, Masone Kelly, Munshi Nikhil C, Anderson Kenneth C, Richardson Paul G
Dana-Farber Cancer Institute, Boston, Massachusetts.
Clin Adv Hematol Oncol. 2018 Aug;16(8):564-574.
Autologous stem cell transplant (ASCT) is an established frontline standard of care for the younger, fitter patients with newly diagnosed multiple myeloma (NDMM) who are eligible for the procedure, and has contributed to improved overall survival. In the current era of novel therapies, the treatment landscape and prognosis have changed. The outstanding efficacy seen with regimens based on novel agents has led to a questioning of the frontline treatment paradigm with respect to ASCT. A key current question is whether to use transplant early or to collect stem cells early but save ASCT for salvage therapy. In this review, we evaluate the clinical data for each approach as well as the arguments in favor of early or delayed ASCT. We also consider the clinical/clonal heterogeneity of myeloma and review the evidence regarding which patient subgroups may benefit most from each approach. We summarize current treatment guidelines for transplant-eligible patients with NDMM and review the evolving role of minimal residual disease evaluation and its potential effect on the debate over early vs delayed ASCT. We conclude that frontline ASCT remains a standard of care for a substantial proportion of patients; however, delayed/salvage ASCT is increasingly being used in the context of highly active frontline regimens based on novel agents and the ongoing personalization of myeloma treatment.
自体干细胞移植（ASCT）是适合该手术的新诊断多发性骨髓瘤（NDMM）的年轻、健康患者既定的一线标准治疗方法，并有助于提高总生存率。在当前的新型疗法时代，治疗格局和预后已经发生了变化。基于新型药物的方案所展现出的卓越疗效引发了对ASCT一线治疗模式的质疑。当前一个关键问题是，是尽早进行移植，还是尽早采集干细胞，将ASCT留作挽救性治疗。在本综述中，我们评估了每种方法的临床数据以及支持早期或延迟ASCT的论据。我们还考虑了骨髓瘤的临床/克隆异质性，并回顾了关于哪些患者亚组可能从每种方法中获益最多的证据。我们总结了适合移植的NDMM患者的当前治疗指南，并回顾了微小残留病评估的不断演变的作用及其对早期与延迟ASCT争论的潜在影响。我们得出结论，一线ASCT仍然是相当一部分患者的标准治疗方法；然而，在基于新型药物的高度活跃的一线方案以及骨髓瘤治疗不断个性化的背景下，延迟/挽救性ASCT的使用越来越多。
