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Ref Type
PMID (26012842)
Authors Chatain N, Perera RC, Rossetti G, Rossa J, Carloni P, Schemionek M, Haferlach T, Brümmendorf TH, Schnittger S, Koschmieder S
Title Rare FLT3 deletion mutants may provide additional treatment options to patients with AML: an approach to individualized medicine.
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Abstract Text

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
FLT3 E598_Y599del deletion gain of function FLT3 E598_Y599del results in the deletion of two amino acids in the juxtamembrane domain of the Flt3 protein from amino acid 598 to 599 (PMID: 11756186). E598_Y599del confers a gain of function to Flt3 as demonstrated by increased Flt3 downstream signaling, decreased apoptosis, clonogenic growth, and cytokine-independent growth in cultured cells (PMID: 26012842).
FLT3 F590_D593delinsLY indel gain of function FLT3 F590_D593delinsLY results in a deletion of four amino acids in the juxtamembrane domain of the Flt3 protein from amino acids 590 to 593, combined with the insertion of a leucine (L) and a tyrosine (Y) at the same site (PMID: 11756186). F590_D593delinsLY confers a gain of function to Flt3 as demonstrated by increased Flt3 downstream signaling, decreased apoptosis, weak clonogenic growth, and cytokine-independent growth in cultured cells (PMID: 26012842).
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FLT3 E598_Y599del hematologic cancer sensitive Midostaurin Preclinical - Cell culture Actionable In a preclinical study, Rydapt (midostaurin) inhibited Flt3 and Stat5 phosphorylation and viability in cells expressing FLT3 E598_Y599del in culture (PMID: 26012842). 26012842
FLT3 F590_D593delinsLY hematologic cancer sensitive Midostaurin Preclinical - Cell culture Actionable In a preclinical study, Rydapt (midostaurin) inhibited Flt3 and Stat5 phosphorylation and viability in cells expressing FLT3 F590_D593delinsLY in culture (PMID: 26012842). 26012842
FLT3 E598_Y599del hematologic cancer sensitive Quizartinib Preclinical - Cell culture Actionable In a preclinical study, Vanflyta (quizartinib) inhibited Flt3 and Stat5 phosphorylation and viability in cells expressing FLT3 E598_Y599del in culture (PMID: 26012842). 26012842
FLT3 F590_D593delinsLY hematologic cancer sensitive Quizartinib Preclinical - Cell culture Actionable In a preclinical study, Vanflyta (quizartinib) inhibited Flt3 and Stat5 phosphorylation and viability in cells expressing FLT3 F590_D593delinsLY in culture (PMID: 26012842). 26012842