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多发性骨髓瘤的干细胞移植。

Stem Cell Transplantation in Multiple Myeloma.

机构信息

Clinica di Ematologia AOU Ospedali Riuniti di Ancona, Ancona. Italy.

Myeloma Unit, Division of Hematology, University of Torino, Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino, Torino. Italy.

出版信息

Curr Cancer Drug Targets. 2017;17(9):769-781. doi: 10.2174/1568009616666160920090236.

Abstract

High-dose therapy (HDT) followed by autologous stem cell transplantation (ASCT) remains the standard of care for patients younger than 65 years of age with multiple myeloma (MM). However, this therapeutic approach has undergone substantial advances in this last decade, mainly due to the introduction of new drugs such as thalidomide, lenalidomide and bortezomib. These new drugs, in different combinations, have shown to significantly increase response rates after induction therapy and ASCT. Moreover, the positive results obtained with these agents in consolidation and maintenance strategies after ASCT strongly support the concept of continuous therapy, whose ultimate goal is the long-term control of the disease and the improvement of outcome. Preliminary data from studies investigating next generation proteasome inhibitors, such as carfilzomib and ixazomib, used upfront as well as at subsequent therapeutic lines, demonstrate the possibility of achieving molecular remission in most of the patients. The deeper responses obtained with new drugcombinations questioned the role of ASCT, and large, ongoing, phase 3 trials will shed light on the role and the timing of ASCT.

摘要

高剂量化疗(HDT)联合自体造血干细胞移植(ASCT)仍然是 65 岁以下多发性骨髓瘤(MM)患者的标准治疗方法。然而,在过去的十年中,这种治疗方法取得了重大进展,主要是由于引入了沙利度胺、来那度胺和硼替佐米等新药。这些新药以不同的组合方式,在诱导治疗和 ASCT 后显著提高了缓解率。此外,这些药物在 ASCT 后的巩固和维持策略中取得的积极结果强烈支持了持续治疗的概念,其最终目标是长期控制疾病并改善预后。研究下一代蛋白酶体抑制剂(如卡非佐米和伊沙佐米)的初步数据表明,这些药物在治疗初期和后续治疗线中都有可能使大多数患者达到分子缓解。新药联合治疗获得的更深层次的缓解质疑了 ASCT 的作用,正在进行的大型 3 期试验将阐明 ASCT 的作用和时机。

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