In a nutshell
This study aimed to investigate if sorafenib (Nexavar) would be a suitable maintenance agent for patients with acute myeloid leukemia who received a stem cell transplant.
This study concluded that sorafenib is a safe and effective maintenance agent in these patients.
Patients with acute myeloid leukemia (AML) with FMS-like tyrosine kinase 3 (FLT3) mutations have a very poor prognosis. These patients are normally treated with allogeneic hematopoietic stem cell transplant (allo-HSCT) and salvage treatments. Allo-HSCT is a procedure in which a patient receives blood-forming stem cells from a genetically similar, but not identical, donor. This is often a sister or brother but could be an unrelated donor. Salvage treatment is a form of therapy given after cancer does not respond to standard therapy.
Sorafenib is a type of targeted therapy called a kinase inhibitor. It is approved for use in FLT3 AML. Maintenance therapy is a chemotherapy given in lower doses to assist in prolonging remission. It was unknown if sorafenib could be used as a maintenance therapy after allo-HSCT in AML patients.
Methods & findings
This study involved 27 patients with FLT3-mutated AML who received allo-HSCT as a treatment. Patients were followed up for an average of 40 months.
A previous report indicated that sorafenib was safe and effective as a maintenance agent after allo-HSCT in patients with FLT3-mutated AML. Significant overall survival (OS) and progression-free survival (PFS) has been seen in patients who received sorafenib before and/or after allo-HSCT.
In this updated study, the persistence of previously reported long-term disease control was demonstrated. The 2-years PFS was on average 73% and the 2-years OS was 80%.
The bottom line
This study concluded that sorafenib is a safe and effective maintenance agent for patients with FLT3-mutated AML who received allo-HSCT.
The fine print
This study had a very small number of patients. Further studies on larger populations are needed.
Published By :
Clinical lymphoma, myeloma & leukemia
Aug 01, 2019