title: Sorafenib and azacitidine as salvage therapy for relapse of FLT3-ITD mutated AML after allo-SCT.
abstract: OBJECTIVE
  Patients with acute myeloid leukemia (AML) carrying FLT3-ITD mutations (FLT3-ITD+) who relapse after allogeneic transplantation (allo-SCT) have a very dismal prognosis with the currently available treatment options.
  
  
  METHODS
  We treated eight patients with FLT3-ITD+ AML who had relapsed in median 91 d (range, 28-249) following allo-SCT with a combination of the multikinase inhibitor sorafenib and the DNA methyltransferase inhibitor azacitidine (Aza).
  
  
  RESULTS
  Patients received a median of five cycles of Aza (range, 2-9) and sorafenib with a median daily dosage of 750 mg (range 400-800) for 129 d (range, 61-221). Six of eight patients received donor lymphocyte infusions (DLI) with a median number of two DLI per patient (range, 1-4). Following this treatment, four patients (50%) achieved a complete remission and three of them a complete molecular remission. Median duration of CR was 182 d (range, 158-406), and two patients remain in ongoing remission for 406 and 168 d. Median overall survival was 322 d (range, 108-574 d) with three patients being currently alive.
  
  
  CONCLUSIONS
  Taken together, the combination of sorafenib, Aza, and DLI shows promising efficacy and deserves further evaluation in larger patient groups.
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Christina
authors.initials: C
authors.lastname: Rautenberg
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Kathrin
authors.initials: K
authors.lastname: Nachtkamp
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Ariane
authors.initials: A
authors.lastname: Dienst
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Pia Verena
authors.initials: PV
authors.lastname: Schmidt
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Claudia
authors.initials: C
authors.lastname: Heyn
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Mustafa
authors.initials: M
authors.lastname: Kondakci
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Ulrich
authors.initials: U
authors.lastname: Germing
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Rainer
authors.initials: R
authors.lastname: Haas
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Guido
authors.initials: G
authors.lastname: Kobbe
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Medical Faculty, University of Duesseldorf, Duesseldorf, Germany.
authors.forename: Thomas
authors.initials: T
authors.lastname: Schroeder
mesh_terms: D000328:Adult; D064591:Allografts; D001374:Azacitidine / Q000008:administration & dosage*; D018572:Disease-Free Survival; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000378:metabolism / Q000401:mortality* / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D009536:Niacinamide / Q000008:administration & dosage / Q000031:analogs & derivatives*; D010671:Phenylurea Compounds / Q000008:administration & dosage*; D012008:Recurrence; D016879:Salvage Therapy / Q000379:methods*; D000077157:Sorafenib; D015996:Survival Rate; D051941:fms-Like Tyrosine Kinase 3 / Q000235:genetics*
chemical_list: D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib; C497970:FLT3 protein, human; D051941:fms-Like Tyrosine Kinase 3; D001374:Azacitidine
keywords: FLT3; acute myeloid leukemia; allogeneic transplantation; azacitidine; relapse; sorafenib