TY - JOUR
T1 - Targeting IDH1 and IDH2 Mutations in Acute Myeloid Leukemia
AU - Ragon, Brittany Knick
AU - DiNardo, Courtney D.
N1 - Publisher Copyright:
© 2017, Springer Science+Business Media, LLC.
PY - 2017/12/1
Y1 - 2017/12/1
N2 - Purpose of Review: Over the past decade, the pathogenic role of mutations in isocitrate dehydrogenases (IDH) 1 and 2, affecting approximately 20% of patients with AML, has been defined, allowing for the development of specific therapeutic strategies for IDH-mutant AML. In this review, the landscape and progress of targeted therapeutics aimed at IDH mutations in AML and related myeloid malignancies will be described. Recent Findings: Since 2013, several mutant IDH-targeted inhibitors have been developed, and nearly a dozen clinical trials have opened specifically for IDH-mutant hematologic malignancies. Preliminary results for several of these investigations have shown evidence of safety, tolerability, and encouraging evidence of efficacy. Summary: Targeting IDH mutations in AML is a biologically informed and rational strategy to promote clinical responses, primarily through differentiation and maturation of the malignant clone. The use of IDH targeted therapy is expected to soon become part of a genomically defined and individualized AML treatment strategy.
AB - Purpose of Review: Over the past decade, the pathogenic role of mutations in isocitrate dehydrogenases (IDH) 1 and 2, affecting approximately 20% of patients with AML, has been defined, allowing for the development of specific therapeutic strategies for IDH-mutant AML. In this review, the landscape and progress of targeted therapeutics aimed at IDH mutations in AML and related myeloid malignancies will be described. Recent Findings: Since 2013, several mutant IDH-targeted inhibitors have been developed, and nearly a dozen clinical trials have opened specifically for IDH-mutant hematologic malignancies. Preliminary results for several of these investigations have shown evidence of safety, tolerability, and encouraging evidence of efficacy. Summary: Targeting IDH mutations in AML is a biologically informed and rational strategy to promote clinical responses, primarily through differentiation and maturation of the malignant clone. The use of IDH targeted therapy is expected to soon become part of a genomically defined and individualized AML treatment strategy.
KW - AML
KW - Differentiation
KW - IDH1
KW - IDH2
KW - Novel therapeutics
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U2 - 10.1007/s11899-017-0418-6
DO - 10.1007/s11899-017-0418-6
M3 - Review article
C2 - 29064021
AN - SCOPUS:85032026480
SN - 1558-8211
VL - 12
SP - 537
EP - 546
JO - Current hematologic malignancy reports
JF - Current hematologic malignancy reports
IS - 6
ER -