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Ref Type Journal Article
PMID (35549749)
Authors He S, Zhang M, Li J, Zhao W, Yu L, Han Y, Pang Y
Title The FLT3 Y842D mutation may be highly sensitive to midostaurin: a case report.
URL
Abstract Text A Y842D mutation within the activation loop of fms-like tyrosine kinase 3 (FLT3) has been shown to confer strong resistance to sorafenib in vitro. Whether this type of mutation exerts clinically significant effects in patients with acute myeloid leukaemia (AML) remains unclear. Here, a novel Y842D activating mutation within the kinase domain of FLT3, in a pregnant patient with de novo hyperleucocyte acute myeloid leukaemia, is described. Following induction failure with standard dose idarubicin and cytarabine (IA), the patient received re-induction combined with midostaurin, a promising agent targeting mutant-FLT3, and IA regimen. Fortunately, morphological remission was achieved. During the period of midostaurin treatment, the patient exhibited a symptom that was characteristic of differentiation syndrome, which disappeared following treatment with methylprednisolone. The present case revealed that Y842D, an uncommon activating mutation in the activation loop of FLT3, may be a midostaurin-sensitive mutation type in patients with acute myeloid leukaemia.

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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
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FLT3 Y842D acute myeloid leukemia predicted - sensitive Cytarabine + Idarubicin + Midostaurin Case Reports/Case Series Actionable In a clinical case study, Rydapt (midostaurin), Cytosar-U (cytarabine), and Idarubicin combination therapy resulted in complete morphological remission, allowing for subsequent allogeneic stem cell transplantation, in a patient with acute myeloid leukemia harboring FLT3 Y842D (PMID: 35549749). 35549749