{"title":"Advancing AML Treatment: Evidence-Based Regimens and Guideline Updates for Targeted Treatments in R/R AML [Podcast].","authors":"Amer M Zeidan, Eunice Wang","doi":"10.2147/BLCTT.S548242","DOIUrl":null,"url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) is a heterogeneous malignancy characterized by diverse genetic mutations, including <i>IDH1</i> and <i>IDH2</i>, which are present in approximately 15-20% of cases. Recent clinical practice guidelines, including the 2025 NCCN guidelines, emphasize the importance of comprehensive mutational profiling at diagnosis and at relapse to guide targeted treatment strategies for patients with refractory or relapsed (R/R) AML. IDH1-mutations, which occur in 5-7% of AML cases, result in the production of the oncometabolite 2-hydroxyglutarate (2-HG), disrupting cellular differentiation. IDH1-inhibitors, such as ivosidenib and olutasidenib, block this aberrant metabolic pathway, allowing for differentiation and apoptosis of leukemia cells. Given the rarity of these mutations, comprehensive molecular testing remains essential to optimize therapeutic decision-making.</p>","PeriodicalId":42368,"journal":{"name":"Blood and Lymphatic Cancer-Targets and Therapy","volume":"15 ","pages":"69-75"},"PeriodicalIF":3.9000,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12235439/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood and Lymphatic Cancer-Targets and Therapy","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.2147/BLCTT.S548242","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Acute myeloid leukemia (AML) is a heterogeneous malignancy characterized by diverse genetic mutations, including IDH1 and IDH2, which are present in approximately 15-20% of cases. Recent clinical practice guidelines, including the 2025 NCCN guidelines, emphasize the importance of comprehensive mutational profiling at diagnosis and at relapse to guide targeted treatment strategies for patients with refractory or relapsed (R/R) AML. IDH1-mutations, which occur in 5-7% of AML cases, result in the production of the oncometabolite 2-hydroxyglutarate (2-HG), disrupting cellular differentiation. IDH1-inhibitors, such as ivosidenib and olutasidenib, block this aberrant metabolic pathway, allowing for differentiation and apoptosis of leukemia cells. Given the rarity of these mutations, comprehensive molecular testing remains essential to optimize therapeutic decision-making.
期刊介绍：
Blood and Lymphatic Cancer: Targets and Therapy is an international, peer reviewed, open access journal focusing on blood and lymphatic cancer research, identification of therapeutic targets, and the optimal use of preventative and integrated treatment interventions to achieve improved outcomes, enhanced survival, and quality of life for the cancer patient. Specific topics covered in the journal include: Epidemiology, detection and screening Cellular research and biomarkers Identification of biotargets and agents with novel mechanisms of action Optimal clinical use of existing anticancer agents, including combination therapies Radiation, surgery, bone marrow transplantation Palliative care Patient adherence, quality of life, satisfaction Health economic evaluations.
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