New Treatment Reduces Need for Blood Transfusions for Patients with Certain Blood Cancers, Study Shows

By Corrie Pellegrino - December 09, 2023

Blood transfusions are a regular part of life for many patients undergoing treatment for myelodysplastic syndromes, a group of blood cancers that are commonly diagnosed in older people. Patients may need transfusions as often as every week to combat low red blood cell counts and the related symptoms such as fatigue, cognitive decline and diminished cardiopulmonary function. However, a new treatment option is giving patients with lower risk myelodysplastic syndromes hope for achieving red blood cell transfusion independence and better outcomes.   

Imetelstat is a first-in-class telomerase inhibitor that prevents cancer cells from continuing to replicate. In a phase 3 global study, the treatment had a significant impact on transfusion independence for patients with lower risk myelodysplastic syndromes (MDS) who were relapsed or refractory to or ineligible for erythropoiesis-stimulating agents, a common treatment used to stimulate the production of red blood cells.

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"Currently there are only a few treatment options for people with lower risk MDS, and a lot of these patients end up needing regular red blood cell blood transfusions. So there is always an unmet need for new treatments."

- Dr. Rami Komrokji, Malignant Hematology Department

“Currently there are only a few treatment options for people with lower risk MDS, and a lot of these patients end up needing regular red blood cell blood transfusions,” explained Dr. Rami Komrokji, vice chair of the Malignant Hematology Department at Moffitt Cancer Center, who is presenting the IMerge phase 3 study findings at the American Society of Hematology Annual Meeting in San Diego. “So there is always an unmet need for new treatments.”

In the placebo-controlled study, 178 patients were enrolled across 17 countries. For 118 patients, imetelstat was given intravenously every four weeks. The other 60 patients received a placebo. Overall, significantly higher rates of red blood cell transfusion independence were achieved in the patients receiving imetelstat, compared with those receiving the placebo:   

  • 39.8% of patients receiving imetelstat achieved eight-week transfusion independence, compared with 15% of patients receiving the placebo
  • 28% of patients receiving imetelstat achieved 24-week transfusion independence, compared with 3.3% of patients receiving the placebo
  • 13.6% of patients receiving imetelstat achieved one-year transfusion independence, compared with 1.7% of patients receiving the placebo

The study also analyzed response rates for various subgroups of MDS classified by the International Prognostic Scoring System as low or intermediate-1 risk. These subgroup classifications have evolved and grown more accurate over the length of the IMerge trial, with some patients who were originally considered low risk being eventually reclassified as higher risk.

“But when we look at the responses on the trial, the benefit of imetelstat compared to the placebo was seen across all those risk groups, even in patients who had a higher stage of the disease,” Komrokji said.  

Details of the phase 3 IMerge trial have been published in The Lancet, with Komrokji as a co-senior author. The next step is presenting the data to the Food and Drug Administration for approval in early 2024. 

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Corrie Pellegrino Senior Managing Editor 813-745-0833 More Articles

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