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迈向多发性骨髓瘤治愈的步骤?

Steps towards a Multiple Myeloma Cure?

作者信息

Gozzetti Alessandro, Bocchia Monica

机构信息

Division of Hematology, University of Siena, Azienda Ospedaliera Universitaria Senese, 53100 Siena, Italy.

出版信息

J Pers Med. 2022 Sep 3;12(9):1451. doi: 10.3390/jpm12091451.

Abstract

Multiple myeloma survival has increased in last 20 years because of new treatments, better clinical management due to novel diagnostic tools such as imaging, and better understanding of the disease, biologically and genetically. Novel drugs have been introduced that act with different therapeutic mechanisms, but so have novel therapeutic strategies such as consolidation and maintenance after autologous stem cell transplant. Imaging (such as PET-CT and MRI) has been applied at diagnosis and after therapy for minimal residual disease monitoring. Multiparametric flow and molecular NGS may detect, with high-sensitivity, residual monoclonal plasma cells in the bone marrow. With this novel therapeutic and biological approach, a considerable fraction of multiple myeloma patients can achieve durable remission or even MGUS-like regression, which can ultimately lead to disease disappearance. The big dogma, "Myeloma is an incurable disease", is hopefully fading.

摘要

在过去20年中,由于新的治疗方法、借助影像学等新型诊断工具实现的更好的临床管理以及对该疾病在生物学和遗传学方面的更深入了解,多发性骨髓瘤患者的生存期有所延长。已引入了具有不同治疗机制的新型药物,同时也出现了如自体干细胞移植后的巩固和维持等新型治疗策略。影像学检查(如PET-CT和MRI)已应用于诊断以及治疗后微小残留病的监测。多参数流式细胞术和分子二代测序(NGS)能够高灵敏度地检测骨髓中残留的单克隆浆细胞。通过这种新型的治疗和生物学方法,相当一部分多发性骨髓瘤患者能够实现持久缓解,甚至达到类似意义未明的单克隆丙种球蛋白血症(MGUS)的病情逆转,并最终实现疾病消失。“骨髓瘤是一种无法治愈的疾病”这一传统观念有望逐渐淡化。

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