Enasidenib was approved by the Food and Drug Administration in 2017 for the treatment of patients with relapsed or refractory (RR)
acute myeloid leukemia (AML) with an
isocitrate dehydrogenase-2 (IDH2) mutation. Given limited data in clinical practice, this study assessed real-world clinical outcomes and healthcare resource use in patients with RR AML. Physicians performed chart abstraction of patients with RR IDH2-mutated AML treated with
enasidenib (between 1/2018 and 6/2019) or other first-line (1 L) RR
therapy (between 1/2016 and 7/2017). Progression-free survival (PFS) and overall survival (OS) were estimated by the Kaplan-Meier method, and adjusted risk of progression and death were estimated by multivariable Cox proportional hazard models. Among 124 patients treated with
enasidenib and 76 patients treated with other 1 L RR
therapy, overall response rate was higher among patients treated with
enasidenib vs. other 1 L RR
therapies (77% vs. 52%, p < 0.01). After a median follow-up of 9 and 6 months, median PFS was 8 months in
enasidenib-treated patients and 5 months in patients receiving other 1 L RR
therapy, respectively (adjusted HR=0.36, 95% CI: 0.23-0.57, p < 0.01). Median OS was 11 and 6 months in
enasidenib-treated patients and patients receiving other 1 L RR
therapy, respectively (adjusted HR=0.37, 95% CI: 0.22-0.60, p < 0.01). Fewer
enasidenib-treated patients were hospitalized during 1 L RR
therapy vs. those receiving other
therapies (14% vs. 46%, p < 0.01). Results from this real-world study confirm the effectiveness of
enasidenib among patients with IDH2-mutated RR AML and demonstrate that hospitalizations were significantly lower vs. other 1 L RR treatment in clinical practice.