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多发性骨髓瘤的治愈——炒作多于现实。

Cure of multiple myeloma -- more hype, less reality.

作者信息

Hari P, Pasquini M C, Vesole D H

机构信息

Medical College of Wisconsin, Milwaukee, WI 53226, USA.

出版信息

Bone Marrow Transplant. 2006 Jan;37(1):1-18. doi: 10.1038/sj.bmt.1705194.

Abstract

Randomized studies have firmly established the role of autologous transplant as initial therapy in multiple myeloma (MM). Indeed, MM has emerged as the commonest indication for autologous SCT in North America. The conceptual basis for high-dose therapy is the goal of complete remission (CR) through steep reduction in tumor burden affected by single and tandem transplants. Careful analysis of the data challenges the notion of CR as a surrogate to success. Intrinsically aggressive MM, defined by known unfavorable biologic risk factors, overrides the benefit of CR. In contrast, subgroups of patients with favorable biological risk factors may achieve prolonged survival, often without ever achieving CR. Unfortunately, even with tandem transplants, there is no plateau in survival curves. To this end, sequential autologous followed by nonmyeloablative allotransplants are a novel attempt at 'curing' myeloma, but the results thus far have failed to show a definite plateau in survival. Given the improvements in supportive care and concomitant reduction in transplant-related mortality, conventional myeloablative allogeneic transplants need to be re-examined as an option in high-risk aggressive myeloma. At the same time, novel antimyeloma therapies, newer risk stratification and staging tools are transforming the treatment algorithm. We examine the changing role of transplantation in myeloma in the context of novel drug therapy, biologic risk stratification and improving supportive care while arguing that the current 'one size fits all' transplant approaches are far from a cure.

摘要

随机研究已经明确确立了自体移植在多发性骨髓瘤(MM)初始治疗中的作用。事实上,在北美,MM已成为自体造血干细胞移植(SCT)最常见的适应证。大剂量治疗的理论基础是通过单次和串联移植大幅降低肿瘤负荷,从而实现完全缓解(CR)的目标。对数据的仔细分析对将CR作为成功替代指标的观念提出了挑战。由已知不良生物学风险因素定义的具有内在侵袭性的MM,会掩盖CR带来的益处。相比之下,具有良好生物学风险因素的患者亚组可能实现长期生存,且往往从未达到CR。不幸的是,即使进行串联移植,生存曲线也没有出现平台期。为此,序贯自体移植后进行非清髓性异基因移植是“治愈”骨髓瘤的一种新尝试,但迄今为止的结果未能显示出生存方面有明确的平台期。鉴于支持治疗的改善以及移植相关死亡率的相应降低,传统的清髓性异基因移植作为高危侵袭性骨髓瘤的一种选择需要重新审视。与此同时,新型抗骨髓瘤疗法、更新的风险分层和分期工具正在改变治疗方案。我们在新型药物治疗、生物学风险分层和改善支持治疗的背景下,审视移植在骨髓瘤治疗中不断变化的作用,同时认为当前“一刀切”的移植方法远非治愈之道。

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