title: Sorafenib plus all-trans retinoic acid for AML patients with FLT3-ITD and NPM1 mutations.
abstract: Knowledge of the molecular basis of acute myeloid leukaemia has increased considerably in the past few years, and therapies targeting specific molecular defects of this disease are intensively investigated. Patients with both NPM1 and FLT3-ITD mutations encompass 20% of cytogenetically normal AML. The multikinase and FLT3 inhibitor, sorafenib, has shown some efficacy in patients with relapsed FLT3-ITD(+) AML. In addition, it is suggested that all-trans retinoic acid (ATRA) used in combination with chemotherapy has shown to improve outcome of patients harbouring NPM1 mutations. We report here the clinical course of three patients with refractory or relapsed FLT3-ITD(+) /NPM1(+) AML who achieved significant response upon sorafenib and ATRA combination.
authors.affiliation: Service d'Hématologie, Centre Hospitalier Universitaire de Toulouse, Hôpital Purpan, Toulouse, France.
authors.forename: Sarah
authors.initials: S
authors.lastname: Guenounou
authors.forename: Eric
authors.initials: E
authors.lastname: Delabesse
authors.forename: Christian
authors.initials: C
authors.lastname: Récher
mesh_terms: D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D017809:Fatal Outcome; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis / Q000188:drug therapy* / Q000235:genetics*; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009536:Niacinamide / Q000008:administration & dosage / Q000031:analogs & derivatives; D009687:Nuclear Proteins / Q000235:genetics*; D000090243:Nucleophosmin; D010671:Phenylurea Compounds / Q000008:administration & dosage; D000077157:Sorafenib; D016896:Treatment Outcome; D014212:Tretinoin / Q000008:administration & dosage; D055815:Young Adult; D051941:fms-Like Tyrosine Kinase 3 / Q000235:genetics*
chemical_list: C000716967:NPM1 protein, human; D009687:Nuclear Proteins; D010671:Phenylurea Compounds; D000090243:Nucleophosmin; D009536:Niacinamide; D014212:Tretinoin; D000077157:Sorafenib; D051941:fms-Like Tyrosine Kinase 3
keywords: Acute myeloid leukemia; FLT3-ITD mutation; NPM1 mutation; all trans retinoid acid; sorafenib; targeted therapy