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Enasidenib Well-Tolerated, Active in Patients With IDH2-Mutated MDS
Enasidenib was well-tolerated and clinically active in patients with relapsed or refractory IDH2-mutated myelodysplastic syndromes (MDS), according to results from a subgroup analysis of the phase 1 dose-escalation AG221-C-001 clinical trial (Lancet Haematol. 2020 Mar 5. Epub ahead of print).
“We aimed to establish the clinical outcomes of enasidenib monotherapy in a subgroup of patients with [MDS] harbouring mutations in IDH2 from the AG221-C-001 trial,” explained Eytan M. Stein, MD, Memorial Sloan Kettering Cancer Center, New York, and colleagues, who noted that enasidenib has already been shown to induce responses in patients with relapsed or refractory acute myeloid leukemia with IDH2-mutation.
A total of 17 patients enrolled in the study between February 18, 2014, and September 1, 2015, were included in the subgroup analysis if they had MDS that relapsed or was refractory to standard treatments.
Therapy consisted of enasidenib 60 mg to 300 mg daily administered in 28-day cycles, with a median of 3 cycles.The primary end points were the safety and activity of enasidenib.
No dose-limiting toxicities were reported and the most common adverse events were diarrhea and nausea (53% each). Indirect hyperbilirubinemia (35%), pneumonia (29%), and thrombocytopenia (24%) were the most common grade 3-4 adverse events.
No treatment-related deaths occurred.
A total of 9 (53%; 95% CI, 28-77) patients had overall responses with a median duration of 9.2 months (95% CI, 1.0 to not reached).
The median overall survival was 16.9 months, and median event-free survival was 11.0 months.
Ultimately, Dr Stein et al found that enasidenib was generally well-tolerated and can induce responses in patients with mutant IDH2 myelodysplastic syndromes.
“Testing for IDH2 mutations in [MDS] is essential for identifying patients who might benefit from enasidenib therapy, including those patients in whom conventional treatments have been unsuccessful,” they concluded.—Kaitlyn Manasterski
