title: [Efficacy and safety of Sorafenib as monotherapy to FLT3-ITD positive acute myeloid leukemia].
abstract: Objective:To explore the efficacy and safety of Sorafenib as monotherapy to FLT3 positive acute myeloid leukemia (AML).Methods:From April 2014 to December 2015, fourteen AML patients with FLT3 positive, 7 males and 7 females with a median age of 42 (range: 14-81) years old, were enrolled in this study. Of the 14 cases, 4 were de novo cases, 9 refractory cases and 1 relapsed case, including 78.6% patients with severe complications and 57.1% patients with KPS score less than 60 [the median KPS score was 45 (20-70) ]. The administration of Sorafenib was 400 mg twice daily and Sorafenib was continued if tolerated. The treatment response was evaluated by MICM and the data were analyzed by paired samplesttest before and after Sorafenib treatment.Results:The peripheral blood WBC count [4.2 (0.9-11.8) ×109/Lvs39.6 (2.3-209.5) ×109/L,P<0.001 ], the percentage of peripheral blast cell [0.07 (0-0.54)vs0.53 (0-0.94),P<0.001] and the percentage of bone marrow blast cell [0.266 (0.020-0.880)vs0.604 (0.180-0.900),P=0.003] were significantly decreased after Sorafenib monotherapy compared with before. The overall response rate was 57.1% (8/14), including 5 cases (35.7%) with complete remission (CR). Of 4 de novo cases, 2 achieved CR, 1 with PR, 1 with NR; 3 of 10 refractory and relapsed patients achieved CR and 2 cases achieved PR, 5 cases NR. The median duration of achieving molecular remission (FLT3-ITD negative) after Sorafenib was 46(33-72) days, and the median progression free survival (PFS) was 53 (28-175) days.Conclusion:Sorafenib shows activity in FLT3-ITD mutation positive AML patients. Sorafenib monotherapy could be used as a treatment option for elderly patients or patients with severe complications, and refractory and relapsed patients with not suitable for intensive chemotherapy.
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing 100044, China.
authors.forename: J S
authors.initials: JS
authors.lastname: Jia
authors.forename: H H
authors.initials: HH
authors.lastname: Zhu
authors.forename: H X
authors.initials: HX
authors.lastname: Fu
authors.forename: L Z
authors.initials: LZ
authors.lastname: Gong
authors.forename: J
authors.initials: J
authors.lastname: Kong
authors.forename: X J
authors.initials: XJ
authors.lastname: Huang
authors.forename: H
authors.initials: H
authors.lastname: Jiang
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy / Q000235:genetics; D008297:Male; D008875:Middle Aged; D009154:Mutation; D012074:Remission Induction; D012189:Retrospective Studies; D000077157:Sorafenib / Q000627:therapeutic use*; D055815:Young Adult; D051941:fms-Like Tyrosine Kinase 3
chemical_list: D000970:Antineoplastic Agents; D000077157:Sorafenib; D051941:fms-Like Tyrosine Kinase 3