EHA 2021 | Imetelstat in heavily-transfused lower-risk R/R MDS

This is an analysis of published study results. The study was actually investigating the imetelstat in heavily transfusion-dependent, lower-risk MDS patients. And these patients, of course, were EPO and ESA refractory, and the majority of them were- had a ring sideroblastic phenotype, or an SF3B1 mutation.

So, the very meaningful response rates were achieved actually regardless of baseline characteristics, also including mutations and equal level in transfusion burden. So, this study now investigated the effect of the imetelstat with regards to reducing of transfusion burden, transfusion independence in these specific molecular subsets. As I said before, SF3B1 mutation was most common, also cytogenetic subgroups were what you would have expected.

And to summarize, I think the data basically did not show a specific mutation or a cytogenic abnormality being associated with the lower response rate compared to other subgroup of patients. The study also showed a decline of SF3B1 allelic burden in some of the responders, suggesting a disease modifying activity of imetelstat. The agent is currently in a running Phase II program in the same patient population, and I think the study is therefore very important.