HMN 2026: What is the major treatment advance for tough-to-treat high-risk blood cancer

Results from a new clinical trial led by Wake Forest University School of Medicine and Atrium Health Levine Cancer show that a new therapy could provide significantly better outcomes for patients with high-risk essential thrombocythaemia (ET), a type of blood cancer. The findings were recently published in The Lancet Haematology.

ET is a rare blood cancer resulting in excessive platelet production due to abnormal stem cells in bone marrow. This can cause a stroke or heart attack due to clotting, progression to more severe cancers and symptoms affecting a patient’s quality of life.

Ruben Mesa, M.D., president of Advocate Health’s cancer national service line, which includes Atrium Health Levine Cancer, was the study’s principal investigator. The trial involved nearly 175 patients across more than 50 clinical sites in Asia and North America.

The study, known as SURPASS-ET, looked at outcomes for patients with ET who are unable to tolerate or don’t respond to hydroxyurea, the current standard treatment. It compared ropeginterferon alfa-2b, currently used to treat another type of blood cancer, to anagrelide, an oral medication often used when hydroxyurea doesn’t work.

Nearly a third of patients who use hydroxyurea eventually develop intolerance or resistance. “Treatment options for patients with essential thrombocythemia who can’t use hydroxyurea have been limited,” Mesa said. “This study shows ropeginterferon alfa-2b not only controls the disease more effectively than current alternatives, but also improves symptoms, reduces serious complications and improves outcomes for patients and clinicians.”

The study found ropeginterferon alfa-2b worked better than anagrelide at controlling ET in patients who couldn’t use hydroxyurea. Patients who took the new drug had better blood counts, fewer symptoms and fewer serious problems.

The U.S. Food and Drug Administration has recently accepted an application seeking approval to expand the use of ropeginterferon alfa-2b for the treatment of ET. The FDA is expected to complete its review by Aug. 30.

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