Acute Myeloid Leukemia Mutations: Therapeutic Implications

Papayannidis, Cristina; Sartor, Chiara; Marconi, Giovanni; Fontana, Maria Chiara; Nanni, Jacopo; Cristiano, Gianluca; Parisi, Sarah; Paolini, Stefania; Curti, Antonio

doi:10.3390/ijms20112721

Open AccessReview

Acute Myeloid Leukemia Mutations: Therapeutic Implications

by

Cristina Papayannidis

^*,

Chiara Sartor

,

Giovanni Marconi

,

Maria Chiara Fontana

,

Jacopo Nanni

,

Gianluca Cristiano

,

Sarah Parisi

,

Stefania Paolini

and

Antonio Curti

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

^*

Author to whom correspondence should be addressed.

Int. J. Mol. Sci. 2019, 20(11), 2721; https://doi.org/10.3390/ijms20112721

Submission received: 28 April 2019 / Revised: 31 May 2019 / Accepted: 31 May 2019 / Published: 3 June 2019

(This article belongs to the Special Issue Advances in Molecular Biology and Targeted Therapy of Leukemias)

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Abstract

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.

Keywords: acute myeloid leukemia; mutations; FLT3; IDH1-2; resistance

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MDPI and ACS Style

Papayannidis, C.; Sartor, C.; Marconi, G.; Fontana, M.C.; Nanni, J.; Cristiano, G.; Parisi, S.; Paolini, S.; Curti, A. Acute Myeloid Leukemia Mutations: Therapeutic Implications. Int. J. Mol. Sci. 2019, 20, 2721. https://doi.org/10.3390/ijms20112721

AMA Style

Papayannidis C, Sartor C, Marconi G, Fontana MC, Nanni J, Cristiano G, Parisi S, Paolini S, Curti A. Acute Myeloid Leukemia Mutations: Therapeutic Implications. International Journal of Molecular Sciences. 2019; 20(11):2721. https://doi.org/10.3390/ijms20112721

Chicago/Turabian Style

Papayannidis, Cristina, Chiara Sartor, Giovanni Marconi, Maria Chiara Fontana, Jacopo Nanni, Gianluca Cristiano, Sarah Parisi, Stefania Paolini, and Antonio Curti. 2019. "Acute Myeloid Leukemia Mutations: Therapeutic Implications" International Journal of Molecular Sciences 20, no. 11: 2721. https://doi.org/10.3390/ijms20112721

APA Style

Papayannidis, C., Sartor, C., Marconi, G., Fontana, M. C., Nanni, J., Cristiano, G., Parisi, S., Paolini, S., & Curti, A. (2019). Acute Myeloid Leukemia Mutations: Therapeutic Implications. International Journal of Molecular Sciences, 20(11), 2721. https://doi.org/10.3390/ijms20112721

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