Rapid Growth of Targeted Therapies in AML Spurs Need for More Learning

Christina T. Loguidice
Published: Thursday, Nov 14, 2019
Harry P. Erba, MD, PhD

Harry P. Erba, MD, PhD

Prior to 2017, the treatment of acute myeloid leukemia (AML) had been stagnant for several decades, with many patients experiencing poor outcomes if they were unfit to undergo intensive therapy.1 The advent of next-generation sequencing, however, led to the identification of several cytogenetic and molecular markers that both aided prognostication and prompted the development and approval of 8 novel agents over the past 3 years. These include targeted therapies for specific AML subpopulations (Timeline2).1-3 Collectively, these agents have improved outcomes for patients with AML.

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Gilteritinib was approved in the R/R setting by the FDA based on an interim analysis of data from the phase III ADMIRAL trial, which randomly assigned 371 previously treated patients 2:1 to single-agent gilteritinib (n = 247) or 1 of 4 prerandomization- selected salvage chemotherapy regimens (n = 124), 2 of which were intensive and 2, low intensity.2,9 “[All patients had] an FLT3 mutation because the prior studies in the phase I/II setting showed very limited activity in patients who didn’t have the target mutation,” Perl explained.

“The [ADMIRAL] study showed a higher response rate in the gilteritinib arm [and] a better survival, with a median overall survival of 9.3 months [compared with] a median overall survival of 5.6 months in the chemotherapy arm,” Perl said.9 Furthermore, he explained, there was a higher rate of transplantation in the gilteritinib arm, and the patients who underwent transplantation and then resumed gilteritinib after engraftment had some of the study’s best OS results. Based on these data, gilteritinib became the first FLT3 inhibitor approved as single-agent therapy in adults with relapsed or refractory AML with an FLT3 mutation, and it has an NCCN category 1 recommendation for this indication.2,7

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