Imetelstat Reduce Red Blood Cell Transfusions in Lower-Risk Myelodysplastic Syndromes

The key findings of this study were:

1.From September 11, 2019, to October 13, 2021, a total of 178 patients were enrolled and randomly assigned, with 111 (62%) being male and 67 (38%) female.

2.By the data cutoff, 91 (77%) out of 118 patients had discontinued treatment in the imetelstat group, and 45 (75%) in the placebo group; additionally, one patient in the placebo group did not receive treatment.

3.The median follow-up was 19.5 months in the imetelstat group and 17.5 months in the placebo group. In the imetelstat group, 47 patients achieved an RBC-TI of at least 8 weeks, compared to nine in the placebo group.

4.Overall, 107 (91%) out of 118 patients receiving imetelstat and 28 (47%) out of 59 patients receiving placebo experienced grade 3–4 treatment-emergent adverse events.

5.The most common grade 3–4 adverse events in patients taking imetelstat were neutropenia and thrombocytopenia.

Imetelstat, a competitive telomerase inhibitor, provides lasting independence from red blood cell (RBC) transfusions for extensively transfused individuals with LR-MDS who do not respond to or are ineligible for ESAs.

Source: 

Platzbecker, U., Santini, V., Fenaux, P., Sekeres, M. A., Savona, M. R., Madanat, Y. F., Díez-Campelo, M., Valcárcel, D., Illmer, T., Shah, S., Xia, Q., Sun, L., Wan, Y., Huang, F., … Zeidan, A. M. (2023). Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. In The Lancet. Elsevier BV. https://doi.org/10.1016/s0140-6736(23)01724-5