According to a new study published in the journal Pediatric Blood and Cancer, researchers at Fred Hutchinson Cancer Research Center and Seattle Children's Hospital in Seattle, Washington, have found that sorafenib treatment is feasible and well-tolerated in pediatric patients with FLT3/ITD+ acute myeloid leukemia (AML) following allogenic stem cell transplantation (HSCT).
For the retrospective study, researchers identified 15 pediatric patients with FLT3/ITD+ AML treated with sorafenib within 18 months of receiving HSCT. These patients either received sorafenib as prophylaxis because they were at very high risk for relapse or at the time of recurrence. Results showed that 73% of patients experienced significant toxicities.
Of those, researchers found that 55% had received sorafenib at doses above the maximum tolerated dose of sorafenib for pediatric leukemia.
Of note, sorafenib was not found to exacerbate graft versus host disease. In addition, all patients who received sorafenib for minimal residual disease immediately before receiving a transplant or with emergence post-HSCT are alive and continue to be in remission at a median of 48 months after transplant.
The findings suggest that sorafenib is feasible and well-tolerated in pediatric patients with FLT3/ITD+ AML, particularly those with minimal residual disease, but prospective controlled studies are warranted to determine the most appropriate sorafenib dose in these patients.
FLT3/ITD is associated with poor outcomes in adult and pediatric acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation (HSCT) can improve cure rates, however relapse is still common. The case series suggests that sorafenib administration is feasible and tolerable in pediatric FLT3/ITD+ AML patients early post HSCT.