Sorafenib in treatment of acute myeloid leukemia patients with Fms-like tyrosine kinase 3-internal tandem duplication mutation

Objective To analyze the effect and safety of sorafenib in treatment of acute myeloid leukemia (AML) patients carrying Fms-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) mutation. Methods From January 2012 to April 2018,42 AML patients with FLT3-ITD who did not receive sorafenib treatment and did not achieve complete remission in the first induction chemotherapy in our center were included in this study.According to the presence or absence of sorafenib in the following treatment regime,the patients were divided into observation group (treated with sorafenib) and control group (treated without sorafenib).The complete remission (CR) rate,overall survival (OS) and relapse-free survival (RFS) were compared after chemotherapy between the two groups and the adverse events were assessed. Results There were 15 patients in the observation group and 27 patients in the control group.No significant difference was found in the general data and clinical features between the two groups (P>0.05).The complete remission rate of the observation group and the control group were 73.3% and 40.7%,respectively,and the difference was statistically significant (P=0.043).The 1-year OS and 1-year RFS of the observation group were higher than those of the control group (1-year OS:53.3% vs 25.9%;1-year RFS:54.5% vs 18.2%),without statistically significant difference (P>0.05).There was no significant difference in the types and incidence of adverse events between the two groups. Conclusion Application of sorafenib is safe and effective for patients with FLT3-ITD-positive acute myeloid leukemia.It can improve the complete remission rate,thus providing the opportunity for allogeneic hematopoietic stem cell transplantation.