According to research presented at AACR 2019, the FLT3 inhibitor gilteritinib successfully increased overall survival compared to chemotherapy, in patients with relapsed or refractory acute myeloid leukemia.

The targeted therapy specifically inhibits the Fms-like tyrosine kinase 3 (FLT3) gene, found in patients who typically respond worse to standard care.

Leukemia cells. Source: Getty

“Patients with FLT3 mutations who have relapsed or refractory AML have very low response rates to chemotherapy at the time of relapse, and their survival is poor as a result,” Alexander Perl, MD, MS, associate professor of Hematology-Oncology at the Perelman School of Medicine at the University of Pennsylvania, said in a press release. “This drug is specifically designed to help this group of patients, and now we've shown it can make a huge difference for those who, until recently, had no specific therapies available beyond chemotherapy.”

The ADMIRAL trials randomized 247 patients to receive gilteritinib and 124 patients to receive chemotherapy.

According to the clinical trial results, the gilteritinib study arm had increased median overall survival, at 9.3 months vs 5.6 months in the chemotherapy group. Furthermore, survival at 1 year was reported in 37% of patients in the gilteritinib group vs 16.7% in the chemotherapy group.

"Across the board, this trial shows gilteritinib carries a clear survival benefit, meaning we now have a targeted, highly-effective, and well-tolerated treatment option for a group of truly high-risk patients," Perl said in the press release.

The researchers also found that complete remissions (CR) or CR with partial hematologic recovery rates were 34% in the gilteritinib group vs 15% in the chemotherapy group.

Perl and colleagues also found that the safety profile of gilteritinib was similar to that of chemotherapy; however, 11% of patients had to discontinue the treatment due to adverse events.

"Although the incidence of various side effects was similar across the study arms, patients took gilteritinib for considerably longer than they underwent chemotherapy," Perl said. "This actually means the likelihood of side effects on a daily basis is lower on this drug, and this favorable side effect profile allowed us to give gilteritinib as an outpatient treatment, a huge shift for these patients."