Oral drug avatrombopag reduces chemotherapy treatment delays, trial finds

In a phase 2 trial of patients with gastrointestinal cancers, avatrombopag helped 70% of those receiving chemotherapy to stay on treatment schedule, compared with 17% receiving placebo.

In a phase 2 clinical trial, Mass General Brigham Cancer Institute researchers found that the oral drug avatrombopag was safe and effective in managing persistent chemotherapy-induced thrombocytopenia (CIT) in more than half of patients with gastrointestinal cancers, offering a potential new option for managing a common and challenging complication from cancer treatment. Their work is published in the Journal of Clinical Oncology.

CIT occurs when chemotherapy lowers platelet levels, reducing the blood cells that help control bleeding. For patients receiving chemotherapy, low platelet counts are common and frequently lead to treatment delays or dose reductions of chemotherapy. This compromises vital cancer care, potentially reducing the chance of cure or length of survival in these patients. However, there are currently no widely available approved therapies to address this complication.

Chemotherapy remains a cornerstone of therapy for gastrointestinal cancers, so we want patients to be able to stay on their planned treatment. The findings from our trial may help improve care for patients who experience persistent CIT, which has historically been a very challenging complication to manage."

Hanny Al-Samkari, MD, lead and corresponding author, classical hematologist at the Mass General Brigham Cancer Institute, the Peggy S. Blitz Endowed Chair in Hematology/Oncology at Mass General Brigham, and Associate Professor of Medicine at Harvard Medical School

Researchers enrolled 47 adults with gastrointestinal cancers and persistent CIT from across four U.S. cancer centers, including Massachusetts General Hospital, and randomly assigned them to receive avatrombopag or a placebo. Participants ranged from 25 to 84 years old; 30% were women, 17% were non-White, and 11% were Hispanic or Latino. The study was stopped early after an independent monitoring board determined that the trial had met its prespecified stopping criteria for efficacy.

Investigators found that 70% of patients receiving avatrombopag achieved successful treatment of CIT, defined as platelet recovery and prevention of chemotherapy delays or dose reductions, compared with 17% of those receiving placebo. Platelet counts recovered in 83% of patients who received the drug, compared with 46% of those who did not. No serious adverse events were attributed to avatrombopag, and no patients required platelet transfusions.

The authors note that further research is needed to determine whether improving CIT management leads to improved long-term outcomes.