Acute Myeloid Leukemia Mutations: Therapeutic Implications

International Journal of Molecular Sciences

Cristina Papayannidis¹  Gianluca Cristiano¹  Giovanni Marconi¹  MariaChiara Fontana¹  Sarah Parisi¹  Stefania Paolini¹  Jacopo Nanni¹  Antonio Curti¹  Chiara Sartor¹ 

[1] Istituto di Ematologia e Oncologia Medica “L. e A. Seràgnoli”, S.Orsola-Malpighi Hospital, 40138 Bologna, Italy;

关键词: acute myeloid leukemia;    mutations;    FLT3;    IDH1-2;    resistance;   

DOI  :  10.3390/ijms20112721

来源: DOAJ

【 摘 要 】

Acute Myeloid Leukemia (AML) is an extremely heterogeneous group of hematological neoplasms, for which allogeneic stem cell transplantation (HSCT) still represents the only potentially curative option in the majority of cases. However, elderly age and clinically severe comorbidities may often exclude a wide amount of patients from this therapeutic approach, underlying the urgent need for alternative strategies. Thanks to the introduction of advanced high-throughput techniques, light is being shed on the pathogenesis of AML, identifying molecular recurrent mutations as responsible for the onset, as well as progression, of disease. As a consequence, and in parallel, many new compounds, including targeted therapies (FMS-like tyrosine kinase 3 (FLT3) and Isocitrate dehydrogenase 1-2 (IDH1-2) inhibitors), have found a wide room of application in this setting, and are now available in daily practice, or in late phases of clinical development. Moreover, several further innovative molecules are currently under investigation, and promising results for many of them have already been reported. In this review, we will present an update on the most relevant molecular alterations of AML, focusing on the most frequent genomic mutations of the disease, for which compounds have been approved or are still currently under investigation.

【 授权许可】

Unknown