Efficacy and safety of Sorafenib as monotherapy to FLT3-ITD positive acute myeloid leukemia
10.3760/cma.j.issn.0253-2727.2016.12.002
- VernacularTitle:索拉非尼单药治疗FLT3-ITD突变阳性急性髓系白血病14例疗效及安全性分析
- Author:
Jinsong JIA
1
;
Honghu ZHU
;
Haixia FU
;
Lizhong GONG
;
Jun KONG
;
Xiaojun HUANG
;
Hao JIANG
Author Information
1. 100044,北京大学人民医院、北京大学血液病研究所
- Keywords:
Sorafinbe;
Gene,FLT3;
DNA mutational analysis;
Leukemia,myeloid,acute
- From:
Chinese Journal of Hematology
2016;37(12):1022-1026
- CountryChina
- Language:Chinese
-
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 samples t test before and after Sorafenib treatment.Results The peripheral blood WBC count [4.2 (0.9-11.8) × 109/L vs 39.6 (2.3-209.5) × 109/L,P＜0.001],the percentage of peripheral blast cell [0.07 (0-0.54) vs 0.53 (0-0.94),P＜0.001] and the percentage of bone marrow blast cell [0.266 (0.020-0.880) vs 0.604 (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.
