title: [Improved clinical outcome of acute myeloid leukemia with FLT3-ITD mutation treated with sorafenib].
abstract: OBJECTIVE
  To analyze the efficacy of sorafenib on the treatment of patients diagnosed as acute myeloid leukemia(AML) with FLT3-ITD mutation.
  
  
  METHODS
  From January 2012 to February 2015, 42 cases of AML with FLT3-ITD mutation according to MICM (morphology, immunology, cytogenetics and molecular) diagnosis system in our hospital were retrospectively analyzed. Thirty-two cases were refractory to chemotherapy or relapsed, who were treated with sorafenib or combined with chemotherapy. Ten patients relapsed after allogeneic hematopoietic stem cell transplantation (allo-HSCT), who were retreated with sorafenib or combined with donor lymphocyte infusion (DLI) or chemotherapy. In the first group, 13 of 32 patients accepted allo-HSCT.
  
  
  RESULTS
  The overall response rate of all 42 patients was 73.8%, including 4 (9.5%) complete molecular remission (CMR), 9 (21.4%) complete remission (CR), 8 (19%) complete remission with incomplete hematologic recovery (CRi), 10 (23.8%) partial remission (PR), and 11 (26.2%) none remission (NR). The response rate of sorafenib alone for 17 patients was 70.6%, and that of sorafenib plus chemotherapy was 66.7% (P=0.555). Thirteen patients who received allo-HSCT included 6 CMR/CR/CRi, 4 PR, and 3 NR before transplant. The 2-year overall survival (OS) rate and progress free survival (PFS) rate in all patients were 36.9% and 28.7%, and the corresponding median time were 18 months and 9 months respectively. The 2-year OS rate in 23 patients who received sorafenib combined with allo-HSCT was superior to that in 19 patients not receiving allo-HSCT (45.5% vs 23.9%, P=0.041), so was PFS rate (44.0% vs 9.7%, P=0.014). Twelve cases died of disease progression, four of infection, and one of chronic graft versus host disease after transplant.
  
  
  CONCLUSIONS
  Sorafenib combined with chemotherapy improves response rate of AML patients with FLT3-ITD mutation. Those who are treated with sorafenib plus allo-HSCT obtain better long-term survival.
authors.affiliation: The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, Collaborative Innovation Center of Hematology, Suzhou Institute of Blood and Marrow Transplantation, Suzhou 215006, China.
authors.forename: B
authors.initials: B
authors.lastname: Gu
authors.forename: G H
authors.initials: GH
authors.lastname: Chen
authors.forename: H J
authors.initials: HJ
authors.lastname: Shen
authors.forename: X
authors.initials: X
authors.lastname: Ma
authors.forename: C C
authors.initials: CC
authors.lastname: Fu
authors.forename: Y
authors.initials: Y
authors.lastname: Han
authors.forename: X W
authors.initials: XW
authors.lastname: Tang
authors.forename: M
authors.initials: M
authors.lastname: Miao
authors.forename: H Y
authors.initials: HY
authors.lastname: Qiu
authors.forename: A N
authors.initials: AN
authors.lastname: Sun
authors.forename: D P
authors.initials: DP
authors.lastname: Wu
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D018572:Disease-Free Survival; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000235:genetics / Q000401:mortality / Q000628:therapy*; D009154:Mutation; D009536:Niacinamide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000627:therapeutic use*; D012074:Remission Induction; D012189:Retrospective Studies; D000077157:Sorafenib; D015996:Survival Rate; D016896:Treatment Outcome; D051941:fms-Like Tyrosine Kinase 3 / Q000235:genetics*
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib; D051941:fms-Like Tyrosine Kinase 3