Imetelstat leads to durable red blood cell transfusion independence

Imetelstat results in sturdy pink blood cell transfusion independence

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Imetelstat leads to durable red blood cell transfusion independence

For closely transfused sufferers with lower-risk myelodysplastic syndromes (LR-MDS) who usually are not responding to or are ineligible for erythropoiesis-stimulating brokers (ESAs), the aggressive telomerase inhibitor imetelstat results in sturdy pink blood cell (RBC) transfusion independence, in line with a research revealed on-line Dec. 1 in The Lancet.

Uwe Platzbecker, M.D., from College Hospital Leipzig in Germany, and colleagues in contrast the RBC transfusion independence charge with imetelstat versus placebo in sufferers with RBC transfusion-dependent LR-MDS in a double-blind trial performed at 118 websites. Total, 178 sufferers (aged 18 years and older) with ESA-relapsed, ESA-refractory, or ESA-ineligible LR-MDS have been enrolled and randomly assigned to imetelstat or placebo (118 and 60, respectively), administered each 4 weeks as a two-hour intravenous infusion.

Sufferers have been adopted for a median of 19.5 and 17.5 months within the imetelstat and placebo teams, respectively. The researchers discovered that 40 and 15 p.c of sufferers had an RBC transfusion independence of a minimum of eight weeks within the imetelstat and placebo teams, respectively. Of the sufferers receiving imetelstat and placebo, 91 and 47 p.c, respectively, had grade 3 to 4 treatment-emergent adversarial occasions, with the most typical occasions being neutropenia and thrombocytopenia for these taking imetelstat. There have been no experiences of treatment-related deaths.

“Taken collectively, IMerge part 3 outcomes validate the observations from the part 2 half and present that imetelstat gives clinically vital profit to a closely transfusion-dependent LR-MDS affected person inhabitants,” the authors write.

Janssen Analysis & Improvement and Geron Company funded the trial earlier than and after April 18, 2019, respectively. Geron manufactures imetelstat.

Extra info:
Uwe Platzbecker et al, Imetelstat in sufferers with lower-risk myelodysplastic syndromes who’ve relapsed or are refractory to erythropoiesis-stimulating brokers (IMerge): a multinational, randomised, double-blind, placebo-controlled, part 3 trial, The Lancet (2023). DOI: 10.1016/S0140-6736(23)01724-5

Mrinal M Patnaik, Telomerase inhibition in haematological neoplasms—are we prepared for primetime?, The Lancet (2023). DOI: 10.1016/S0140-6736(23)02187-6

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