Imetelstat Versus Best Available Therapy in Relapsed/Refractory Myelofibrosis

By Ariel Jones - Last Updated: October 25, 2021

An analysis published in Annals of Hematology sought to add real-world context to findings from the phase II MYF2001 trial, which demonstrated that treatment with imetelstat improved overall survival (OS) in patients with relapsed/refractory myelofibrosis (MF).

The MYF2001 trial enrolled patients with intermediate-2 or high-risk MF) that had relapsed after or was refractory to JAK inhibitor treatment. Treatment with imetelstat 9.4 mg/kg every three weeks demonstrated an “encouraging” median overall survival (OS) of 29.9 months, the authors reported.

For comparison, the researchers collected external real-world data from a study of 96 patients who had discontinued ruxolitinib and were subsequently treated with best available therapy (BAT) at Moffitt Cancer Center. Matched cohorts were developed using the MYF2001 eligibility criteria, including patients with MF who had discontinued ruxolitinib due to lack or loss of response.

The present analysis included 57 patients treated with imetelstat 9.4 mg/kg from MYF2001 and 38 patients treated with BAT from the real-world study. OS was longer among patients treated with imetelstat versus BAT (30 vs. 12 months), and analysis showed a significantly lower risk of death with imetelstat compared with BAT (hazard ratio = 0.35; p = 0.0019).

Results were similar after additional adjustment for baseline covariates and in sensitivity analyses. These findings “warrant further evaluation of imetelstat in this poor-prognosis patient population,” the authors concluded.