Andrew Klink, PhD, MPH, Cardinal Health Specialty Solutions, Dublin, Ohio, discusses results from a real-world cohort study confirming the effectiveness of enasidenib for relapsed/refractory acute myeloid leukemia (AML) outside the trial setting.
These results were presented at the virtual 62nd American Society of Hematology (ASH) Annual Meeting.
Transcript
My name is Andy Klink. I'm a Director in the Real-World Evidence and Insights team at Cardinal Health. We conducted a retrospective cohort study of relapsed/refractory AML patients treated in US clinical practice outside the trial setting that aimed to assess real-world outcomes, including response to therapy, survival, and rates of ED and hospitalizations.
Since 2017, several new therapies were approved by the US Food & Drug Administration for the treatment of AML. Enasidenib is a once-daily oral tablet that was approved by the FDA in August 2017 for patients with relapsed/refractory AML with an IDH2 mutation.
Data were collected from patient medical records abstracted by 20 community oncologists from across the US for patients with relapsed/refractory AML with IDH2 mutations, treated with enasidenib in 2018 and '19, or with another approved relapsed/refractory AML therapy, including cytotoxic chemotherapy, hypomethylating agents, and targeted therapies, such as Venetoclax in 2016 and '17 as their first-line relapsed/refractory therapy.
Data were collected on 124 patients treated with enasidenib and 76 control patients with another non-enasidenib, first-line relapsed/refractory therapy.
Those treated with enasidenib were significantly older than those treated with other first-line relapsed/refractory therapies, with median ages of 68 and 63 years respectively.
Just over half the patients in each treatment group were ECOG performance status 2 or greater, and 29% of patients in each treatment group were of poorer cytogenetic risk.
77% of patients treated with enasidenib had a complete or partial response or morphologic leukemia-free state. This was significantly higher than among patients treated with other first-line relapsed/refractory therapies, among whom 53% had a complete or partial response or MLFS.
Progression-free survival was significantly longer among those treated with enasidenib compared to those treated with other relapsed/refractory therapies, with a media PFS of 8.4 and 4.8 months respectively.
Patients treated with enasidenib had a 74% lower risk of progression after adjusting for potential confounders. Those treated with enasidenib had a median overall survival of over 4.5 months longer compared to those treated with other first-line, relapsed/refractory therapies, with median OS of 11.0 and 6.4 months respectively.
Those treated with enasidenib had a 73% lower adjusted risk of death compared to those treated with other first-line relapsed/refractory therapies.
While rates of ED visits were similar across treatment groups, around 30% fewer first-line relapsed/refractory enasidenib patients were hospitalized during therapy versus those treated with other first-line relapsed/refractory therapies.
Results from this large, real-world cohort study confirmed the effectiveness of enasidenib in relapsed/refractory AML, with an IDH2 mutation outside of the trial setting.
These results are similar to results of a pivotal Phase 2 trial. Superior response rates, PFS, and OS were observed in enasidenib-treated patients compared with other commonly-used, first-line relapsed/refractory therapies.
Additionally, rates of hospitalizations were significantly lower in the enasidenib cohort. Further research is needed to understand how and when physicians test for mutations in applied targeted therapies and how practicing physicians assess response to contemporary relapsed/refractory AML therapies in real-world clinical practice.
