Imetelstat for transfusion-dependent MDS – pro
The efficacy of imetelstat (Rytelo) was evaluated in the IMerge trial (NCT02598661), which is a multicenter study involving 178 patients with low- or intermediate-1-risk MDS according to the International Prognostic Scoring System. The transfusion independence rates were 39.8% for >=8 weeks, 28.0% for >=24 weeks, and 17.8% for >=1 year, compared to 15.0%, 3.3%, and 1.7% with placebo, respectively.
Imetelstat as the first FDA-approved telomerase inhibitor for transfusion-dependent MDS.
Javed A, Khattak AA, Khan FU, Khattak AMA, Shrestha B. Targeting telomerase in myelodysplastic syndrome: imetelstat as a disease-modifying therapy for transfusion-dependent patients. Ann Med Surg (Lond). 2025 Sep 4;87(10):6252-6255. doi: 10.1097/MS9.0000000000003805. PMID: 41181461; PMCID: PMC12578050.
Santini V, Zeidan AM, Fenaux P, et al. Impact of mutational status on clinical response to imetelstat in patients with lower-risk myelodysplastic syndromes in the Imerge phase 3 study. Blood 2023;142:4603
Rytelo, Prescribing Information 2025