First-line enasidenib is effective for relapsed/refractory IDH2-mutated acute myeloid leukemia (AML) in the real-world setting, demonstrating similar efficacy as pivotal phase 3 trials, according to a study presented at the virtual 62nd American Society of Hematology (ASH) Annual Meeting.
Since 2017 several new therapies were approved by the US Food and Drug administration for the treatment of AML. Enasidenib, a once daily oral tablet, was approved by the FDA in August 2017 for patients with relapsed refractory AML with an IDH2 mutation,” explained Andrew Klink, PhD, MPH, Cardinal Health Specialty Solutions, Dublin, Ohio, who presented the study.
The aim of the retrospective, observational cohort study was to assess real-world outcomes including response to therapy, survival, rates of emergency department (ED) visits, and hospitalizations, among patients with relapsed/refractory IDH2-mutated AML treated with enasidenib, or other systemic therapies.
Patient characteristics, treatment patterns, physician-reported response to first-line relapsed/refractory therapy, ED visits, hospitalizations, and date of death were collected from patient medical records.
A total of 200 patients were included in the study; 124 treated with enasidenib and 76 control patients treated with another first-line relapsed/refractory therapy. Approximately 23% of enasidenib patients were refractory to induction therapy versus 40% of control patients (P = .02).
Among patients treated with enasidenib, complete/partial response rates were higher (77%) in comparison to those treated with control regimens (52%; P <.01). After a median follow-up of 9 months in patients who received enasidenib and 6 months in patients who received control regimens, median PFS was 8.4 months and 4.8 months (P <.01) and median OS was 11 months and 6.4 months (P <.01)., respectively.
Rates of ED visits were similar for both enasidenib (11%) and control patients (18%; P = .59). However, there were fewer hospitalizations reported for patients treated with enasidenib (14%) in comparison to other therapies (46%; P <.01).
“Results from this large real-world cohort study confirm the effectiveness of enasidenib in relapsed/refractory AML patients with IDH2 mutations outside the trial setting,” Dr Klink concluded.—Janelle Bradley
Klink A, Copher R, Knoth R, et al. eal-World Use of Enasidenib in Relapsed or Refractory Acute Myeloid Leukemia Is Associated with Reduced Risk of Disease Progression and Death. Presented at: the virtual 62nd ASH Annual Meeting and Exposition; December 5-8, 2020. Abstract 1912.
