多发性骨髓瘤的1号染色体改变:精确治疗的考虑。

IF 2.3 3区 医学 Q2 HEMATOLOGY
Niamh McAuley, Izabela Cymer, Roisin McAvera, Ann M. Hopkins, Siobhan V. Glavey
{"title":"多发性骨髓瘤的1号染色体改变:精确治疗的考虑。","authors":"Niamh McAuley,&nbsp;Izabela Cymer,&nbsp;Roisin McAvera,&nbsp;Ann M. Hopkins,&nbsp;Siobhan V. Glavey","doi":"10.1111/ejh.14352","DOIUrl":null,"url":null,"abstract":"<p>Multiple myeloma (MM) is an incurable blood malignancy characterized by the clonal expansion of plasma cells and the secretion of monoclonal immunoglobulins. High-risk MM, defined by specific cytogenetic abnormalities, poses significant therapeutic challenges and is associated with inferior survival outcomes compared to standard-risk disease. Although molecularly targeted therapies have shown efficacy in other hematologic malignancies, currently venetoclax is the only targeted therapy approved for MM (t(11;14)). However, chromosome 1q gains, amplifications, and 1p deletions are frequently observed in MM, and have been linked to drug resistance and poor patient prognosis. Accordingly, this review focuses on emerging MM precision therapies capable of targeting dysregulated genes within these regions. It addresses gene therapies, small molecule inhibitors and monoclonal antibodies currently under investigation to antagonize oncogenic drivers including <i>MCL-1</i>, <i>BCL9</i>, <i>F11R</i>, and <i>CKS1B</i>, all of which are implicated in cell survival, proliferation or drug resistance. In conclusion, the link between chromosome 1 abnormalities and high-risk disease in MM patients offers a compelling rationale to identify and explore therapeutic targeting of chromosome 1 gene products as a novel precision medicine approach for a poorly served patient population.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"400-410"},"PeriodicalIF":2.3000,"publicationDate":"2024-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14352","citationCount":"0","resultStr":"{\"title\":\"Chromosome 1 Alterations in Multiple Myeloma: Considerations for Precision Therapy\",\"authors\":\"Niamh McAuley,&nbsp;Izabela Cymer,&nbsp;Roisin McAvera,&nbsp;Ann M. Hopkins,&nbsp;Siobhan V. Glavey\",\"doi\":\"10.1111/ejh.14352\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>Multiple myeloma (MM) is an incurable blood malignancy characterized by the clonal expansion of plasma cells and the secretion of monoclonal immunoglobulins. High-risk MM, defined by specific cytogenetic abnormalities, poses significant therapeutic challenges and is associated with inferior survival outcomes compared to standard-risk disease. Although molecularly targeted therapies have shown efficacy in other hematologic malignancies, currently venetoclax is the only targeted therapy approved for MM (t(11;14)). However, chromosome 1q gains, amplifications, and 1p deletions are frequently observed in MM, and have been linked to drug resistance and poor patient prognosis. Accordingly, this review focuses on emerging MM precision therapies capable of targeting dysregulated genes within these regions. It addresses gene therapies, small molecule inhibitors and monoclonal antibodies currently under investigation to antagonize oncogenic drivers including <i>MCL-1</i>, <i>BCL9</i>, <i>F11R</i>, and <i>CKS1B</i>, all of which are implicated in cell survival, proliferation or drug resistance. In conclusion, the link between chromosome 1 abnormalities and high-risk disease in MM patients offers a compelling rationale to identify and explore therapeutic targeting of chromosome 1 gene products as a novel precision medicine approach for a poorly served patient population.</p>\",\"PeriodicalId\":11955,\"journal\":{\"name\":\"European Journal of Haematology\",\"volume\":\"114 3\",\"pages\":\"400-410\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2024-12-04\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14352\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European Journal of Haematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1111/ejh.14352\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Journal of Haematology","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1111/ejh.14352","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

摘要

多发性骨髓瘤(MM)是一种无法治愈的血液恶性肿瘤,其特征是浆细胞克隆扩增和单克隆免疫球蛋白分泌。高风险MM是由特定的细胞遗传学异常定义的,与标准风险疾病相比,它带来了重大的治疗挑战,并且与较差的生存结果相关。尽管分子靶向治疗已经在其他血液系统恶性肿瘤中显示出疗效,但目前venetoclax是唯一被批准用于MM的靶向治疗(t(11;14))。然而,染色体1q增加、扩增和1p缺失在MM中经常观察到,并且与耐药和患者预后不良有关。因此,本综述的重点是能够靶向这些区域内失调基因的新兴MM精确疗法。它涉及目前正在研究的基因疗法、小分子抑制剂和单克隆抗体,以拮抗包括MCL-1、BCL9、F11R和CKS1B在内的致癌驱动因子,所有这些驱动因子都与细胞存活、增殖或耐药性有关。总之,1号染色体异常与MM患者高危疾病之间的联系为鉴定和探索1号染色体基因产物的靶向治疗提供了一个令人信服的理论依据,作为一种新的精准医学方法,用于服务差的患者群体。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Chromosome 1 Alterations in Multiple Myeloma: Considerations for Precision Therapy

Chromosome 1 Alterations in Multiple Myeloma: Considerations for Precision Therapy

Multiple myeloma (MM) is an incurable blood malignancy characterized by the clonal expansion of plasma cells and the secretion of monoclonal immunoglobulins. High-risk MM, defined by specific cytogenetic abnormalities, poses significant therapeutic challenges and is associated with inferior survival outcomes compared to standard-risk disease. Although molecularly targeted therapies have shown efficacy in other hematologic malignancies, currently venetoclax is the only targeted therapy approved for MM (t(11;14)). However, chromosome 1q gains, amplifications, and 1p deletions are frequently observed in MM, and have been linked to drug resistance and poor patient prognosis. Accordingly, this review focuses on emerging MM precision therapies capable of targeting dysregulated genes within these regions. It addresses gene therapies, small molecule inhibitors and monoclonal antibodies currently under investigation to antagonize oncogenic drivers including MCL-1, BCL9, F11R, and CKS1B, all of which are implicated in cell survival, proliferation or drug resistance. In conclusion, the link between chromosome 1 abnormalities and high-risk disease in MM patients offers a compelling rationale to identify and explore therapeutic targeting of chromosome 1 gene products as a novel precision medicine approach for a poorly served patient population.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
5.50
自引率
0.00%
发文量
168
审稿时长
4-8 weeks
期刊介绍: European Journal of Haematology is an international journal for communication of basic and clinical research in haematology. The journal welcomes manuscripts on molecular, cellular and clinical research on diseases of the blood, vascular and lymphatic tissue, and on basic molecular and cellular research related to normal development and function of the blood, vascular and lymphatic tissue. The journal also welcomes reviews on clinical haematology and basic research, case reports, and clinical pictures.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信