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本文引用的文献

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The Role of Diagnosis and Clinical Follow-up of Monoclonal Gammopathy of Undetermined Significance on Survival in Multiple Myeloma.意义未明的单克隆丙种球蛋白病在多发性骨髓瘤中的诊断和临床随访作用对生存的影响。
JAMA Oncol. 2015 May;1(2):168-74. doi: 10.1001/jamaoncol.2015.23.
2
Targeting the bone marrow microenvironment in multiple myeloma.靶向多发性骨髓瘤中的骨髓微环境。
Immunol Rev. 2015 Jan;263(1):160-72. doi: 10.1111/imr.12233.
3
Dilemmas in treating smoldering multiple myeloma.治疗冒烟型多发性骨髓瘤的困境
J Clin Oncol. 2015 Jan 1;33(1):115-23. doi: 10.1200/JCO.2014.56.4351. Epub 2014 Nov 24.
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How I treat smoldering multiple myeloma.我如何治疗冒烟型多发性骨髓瘤。
Blood. 2014 Nov 27;124(23):3380-8. doi: 10.1182/blood-2014-08-551549. Epub 2014 Oct 8.
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[Imaging in smoldering (asymptomatic) multiple myeloma. Past, present and future].[冒烟型(无症状)多发性骨髓瘤的影像学:过去、现在与未来]
Radiologe. 2014 Jun;54(6):572, 574-81. doi: 10.1007/s00117-014-2694-7.
6
Monoclonal gammopathy of undetermined significance and smoldering multiple myeloma: biological insights and early treatment strategies.意义未明的单克隆丙种球蛋白病和冒烟型多发性骨髓瘤:生物学见解与早期治疗策略
Hematology Am Soc Hematol Educ Program. 2013;2013:478-87. doi: 10.1182/asheducation-2013.1.478.
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Smoldering multiple myeloma requiring treatment: time for a new definition?冒烟型多发性骨髓瘤需要治疗:是否需要新的定义?
Blood. 2013 Dec 19;122(26):4172-81. doi: 10.1182/blood-2013-08-520890. Epub 2013 Oct 21.
9
Lenalidomide plus dexamethasone for high-risk smoldering multiple myeloma.来那度胺联合地塞米松治疗高危冒烟型多发性骨髓瘤。
N Engl J Med. 2013 Aug 1;369(5):438-47. doi: 10.1056/NEJMoa1300439.
10
Intraclonal heterogeneity is a critical early event in the development of myeloma and precedes the development of clinical symptoms.克隆内异质性是骨髓瘤发生过程中的一个关键早期事件,且发生在临床症状出现之前。
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多发性骨髓瘤的治愈之路始于冒烟型骨髓瘤。

The road to cure in multiple myeloma starts with smoldering disease.

作者信息

Salem Karma Z, Ghobrial Irene M

出版信息

Expert Opin Orphan Drugs. 2015 Apr 12;3(6). doi: 10.1517/21678707.2015.1036740.

DOI:10.1517/21678707.2015.1036740
PMID:25995973
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4435605/
Abstract

INTRODUCTION

Smoldering multiple myeloma (SMM) is a heterogeneous clinical entity that defines patients in the spectrum of disease progression from monoclonal gammopathy of undetermined significance to multiple myeloma (MM). Current standard of care is observation until end organ damage occurs. In spite of this, the scientific community has begun to question whether the strategy of watchful waiting should be replaced with earlier therapeutic intervention with the ultimate goal of preventing clonal heterogeneity and end organ damage.

AREAS COVERED

In this review, we challenge the concept of observation as the best option of therapy in SMM. We present current data on diagnosis, prognostic factors of disease progression and studies that have been conducted to date to determine whether earlier therapeutic interventions will lead to an improvement in overall survival of patients with MM.

EXPERT OPINION

If the recommendations of treatment of SMM were to change, the scientific body of evidence would have to overcome four major hurdles: to demonstrate that early intervention leads to prolonged survival and delay in development of end organ damage, that it does not have long-term toxicities, that it is implemented in patients with a high-likelihood of developing myeloma and that it does not lead to the outgrowth of more resistant clones. Only well-designed clinical trials will determine whether cure can be achieved with earlier interventions.

摘要

引言

冒烟型多发性骨髓瘤(SMM)是一种异质性临床实体,用于定义处于从意义未明的单克隆丙种球蛋白病到多发性骨髓瘤(MM)疾病进展范围内的患者。当前的治疗标准是观察,直至出现终末器官损害。尽管如此,科学界已开始质疑观察等待策略是否应被早期治疗干预所取代,其最终目标是预防克隆异质性和终末器官损害。

涵盖领域

在本综述中,我们对将观察作为SMM最佳治疗选择的概念提出质疑。我们展示了关于诊断、疾病进展预后因素的当前数据,以及迄今为止为确定早期治疗干预是否会改善MM患者总生存期而进行的研究。

专家观点

如果SMM的治疗建议要改变,科学证据体系将必须克服四个主要障碍:证明早期干预能延长生存期并延缓终末器官损害的发展,证明其没有长期毒性,证明其适用于有高骨髓瘤发生可能性的患者,以及证明其不会导致更具耐药性的克隆的出现。只有精心设计的临床试验才能确定早期干预是否能实现治愈。