The role of high-dose melphalan with autologous stem-cell transplant in multiple myeloma: is it time for a paradigm shift?
The role of high-dose melphalan with autologous stem-cell transplant in multiple myeloma: is it time for a paradigm shift?
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DOI：
10.1111/bjh.16764
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发表时间：
2020-12
影响因子：
6.5
通讯作者：
Richardson PG
中科院分区：
文献类型：
--
作者：
Kazandjian D;Mo CC;Landgren O;Richardson PG
多发性骨髓瘤的最新进展包括许多具有前所未有疗效的新疗法的批准，新药开发的快速和持续节奏，以及对预测的进一步改进，包括最小残留病(MRD)测试和改进的风险分层。预先使用免疫调节药物和蛋白酶体抑制剂组合，然后进行维护，已经产生了转折性的临床益处。在没有强化的情况下，结合了单抗的四种药物方案报告了前所未有的完全应答率和MRD阴性。在这些进展的背景下，大剂量马法兰联合自体干细胞移植(HDM-ASCT)的附加值是一个关键问题。从安全的角度来看，HDM-ASCT既与降低生活质量的急性毒性有关，也与可能限制某些患者预期寿命的长期毒性有关。本文讨论了诱导治疗的最新进展、这些进展对HDM-ASCT的影响、MRD检测的演变作用以及HDM-ASCT的短期和长期风险。认识到预期数据仍然有限，我们建议HDM-ASCT不被认为是符合条件的新诊断患者的强制性治疗，这些患者接受了高效的方案治疗并取得了深入的反应，而是保留了这种方法所固有的临床和基因组毒性的早期暴露。
Recent advances in multiple myeloma include numerous approvals of novel therapies with unprecedented efficacy, a rapid and sustained tempo of new drug development, and further refinements to prognostication to include minimal residual disease (MRD) testing and improved risk stratification. The upfront use of immunomodulatory drug and proteasome inhibitor combinations followed by maintenance has resulted in transformative clinical benefit. Four-drug regimens incorporating monoclonal antibodies are reporting unprecedented rates of complete response and MRD negativity in the absence of intensification. In the context of these advances, the added value of high-dose melphalan with autologous stem-cell transplant (HDM-ASCT) is a key question. From a safety standpoint, HDM-ASCT is associated with both acute toxicities that reduce quality of life and long-term toxicities that may limit life expectancy for some patients. The present review discusses the recent advances in induction therapy, the impact of these advances on HDM-ASCT, the evolving role of MRD testing and the short- and long-term risks of HDM-ASCT. Recognising that prospective data remains limited, we suggest that HDM-ASCT not be considered mandatory for eligible newly diagnosed patients who are treated with highly efficacious regimens and achieve deep responses, but rather be held in reserve without early exposure to the clinical and genomic toxicity inherent to this approach.
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