Dr. Perl on Genetic Testing and Targeted Therapies in AML

Alexander E. Perl, MD, MS, associate professor, medicine, the Hospital of the University of Pennsylvania, discusses why genetic testing is important for determining targeted therapies for patients with acute myeloid leukemia (AML).

Newer targeted therapies in the AML treatment landscape are providing impressive benefits for patients. For instance, although single-agent gemtuzumab ozogamicin (Mylotarg) did not display a prominent overall survival (OS) improvement in the relapsed/refractory setting, when moved to the frontline setting and combined with induction and consolidation chemotherapy, the agent demonstrated an impressive OS benefit in patients with core binding factor AML, Perl says.

Similar advances have been made in patients with FLT3-mutated AML. The phase 3 RATIFY trial (NCT00651261) led to the FDA approval of midostaurin (Rydapt) in FLT3-mutant AML based on the survival improvements it showed with the agent in the ITD and TKD subtypes of FLT3-positive disease, Perl explains. Additionally, the phase 3 QuANTUM-First study (NCT02668653) showed OS improvements with quizartinib, a FLT3 TKI, in older patients with the ITD subtype of FLT3-mutant AML, Perl notes. Determining patients’ genetic risk profiles is important for recognizing potential therapeutic targets, so patients can receive optimal therapies early in their treatment courses, Perl concludes.