{"title":"BCL2 inhibition for multiple myeloma and AL amyloidosis.","authors":"Lorenzo Cani, Vikas A Gupta, Jonathan L Kaufman","doi":"10.1111/bjh.20046","DOIUrl":null,"url":null,"abstract":"<p><p>Despite the development of novel treatments, multiple myeloma (MM) and light-chain (AL) amyloidosis remain incurable diseases. BCL2 inhibitors are a class of drugs under development for plasma cell disorders, with strong data supporting their use, particularly in patients with MM and AL amyloidosis harbouring the t(11;14). Venetoclax, the most extensively studied BCL2-specific inhibitor, was initially designed and evaluated for other malignant blood disorders. However, it has since shown promising efficacy in both randomized and real-world studies for MM and AL amyloidosis, either as a monotherapy or in combination with other agents. Nonetheless, toxicity concerns have been raised, underscoring the need for careful patient selection and precise dose optimization. Additionally, other BCL2-targeting drugs are under investigation in preclinical and clinical studies. This review focuses on the current role of BCL2 inhibitors in the treatment landscape of MM and AL amyloidosis.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1000,"publicationDate":"2025-03-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"British Journal of Haematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/bjh.20046","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Despite the development of novel treatments, multiple myeloma (MM) and light-chain (AL) amyloidosis remain incurable diseases. BCL2 inhibitors are a class of drugs under development for plasma cell disorders, with strong data supporting their use, particularly in patients with MM and AL amyloidosis harbouring the t(11;14). Venetoclax, the most extensively studied BCL2-specific inhibitor, was initially designed and evaluated for other malignant blood disorders. However, it has since shown promising efficacy in both randomized and real-world studies for MM and AL amyloidosis, either as a monotherapy or in combination with other agents. Nonetheless, toxicity concerns have been raised, underscoring the need for careful patient selection and precise dose optimization. Additionally, other BCL2-targeting drugs are under investigation in preclinical and clinical studies. This review focuses on the current role of BCL2 inhibitors in the treatment landscape of MM and AL amyloidosis.
期刊介绍：
The British Journal of Haematology publishes original research papers in clinical, laboratory and experimental haematology. The Journal also features annotations, reviews, short reports, images in haematology and Letters to the Editor.
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