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多发性骨髓瘤诊断与治疗中的挑战。

Challenges in multiple myeloma diagnosis and treatment.

作者信息

Girnius S, Munshi N C

机构信息

Boston VA Healthcare System, Boston, MA, USA; Boston University Medical Center, Boston, MA, USA.

Boston VA Healthcare System, Boston, MA, USA; Dana Farber Cancer Institute, Boston, MA, USA; Harvard Medical School, Boston, MA, USA.

出版信息

Leuk Suppl. 2013 May;2(Suppl 1):S3-9. doi: 10.1038/leusup.2013.2. Epub 2013 May 8.

Abstract

Although multiple myeloma (MM) remains an incurable disease, the advent of novel treatment paradigms has improved survival outcomes in the past two decades. This includes widespread use of high-dose chemotherapy with autologous stem cell transplantation (HDT-ASCT) and the development of the novel agents thalidomide, lenalidomide and bortezomib. The efficacy and tolerability of these novel agents have allowed for the exploration of continuous therapy approaches. Maintenance therapy after HDT-ASCT, for example, may help prolong progression-free survival by providing sustained control of residual disease. Trials are also under way to evaluate lenalidomide in patients with high-risk smoldering MM, with the aim of delaying progression to symptomatic MM. Other research is focusing on improving HDT-ASCT protocols and integrating novel agents, such as bortezomib, as an induction or consolidation therapy. Despite these advances, more effective strategies are needed, particularly for the management of older, less fit patients who are ineligible for HDT-ASCT. Preliminary results on the use of lenalidomide maintenance therapy in elderly patients are encouraging. Taken together, these observations indicate that in this era of novel agents, optimal treatment of MM requires a long-term perspective that focuses on providing sustained disease control while maintaining quality of life.

摘要

尽管多发性骨髓瘤(MM)仍然是一种无法治愈的疾病,但在过去二十年中,新型治疗模式的出现改善了生存结果。这包括大剂量化疗联合自体干细胞移植(HDT-ASCT)的广泛应用以及新型药物沙利度胺、来那度胺和硼替佐米的研发。这些新型药物的疗效和耐受性使得连续治疗方法的探索成为可能。例如,HDT-ASCT后的维持治疗可能通过持续控制残留疾病来帮助延长无进展生存期。目前也正在进行试验,以评估来那度胺在高危冒烟型MM患者中的疗效,目的是延缓进展为有症状的MM。其他研究则集中在改进HDT-ASCT方案以及整合新型药物,如硼替佐米,作为诱导或巩固治疗。尽管取得了这些进展,但仍需要更有效的策略,特别是对于不符合HDT-ASCT条件的老年、身体状况较差的患者的管理。来那度胺维持治疗在老年患者中的初步结果令人鼓舞。综上所述,这些观察结果表明,在这个新型药物的时代,MM的最佳治疗需要一个长期的视角,专注于在维持生活质量的同时提供持续的疾病控制。

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