In a phase 2 trial in patients with gastrointestinal cancers, 70% of patients receiving chemotherapy were able to stick to their treatment schedule with Avatrombopag, compared with 17% of patients receiving a placebo.
Researchers at Massachusetts General Brigham Cancer Institute have found that the oral drug avatrombopag is safe and effective in managing persistent chemotherapy-induced thrombocytopenia (CIT) in more than half of patients with gastrointestinal cancers in a phase 2 clinical trial, providing a potential new option for managing a common and difficult complication associated with cancer treatment. Their works are Journal of Clinical Oncology.
CIT occurs when chemotherapy lowers platelet levels, reducing blood cells that help control bleeding. Decreased platelet counts are common in patients receiving chemotherapy, often leading to treatment delays and chemotherapy dose reductions. This can compromise important cancer treatments and shorten the chances of cure and survival of these patients. However, there are currently no approved treatments widely available to address this complication.
Chemotherapy remains the cornerstone of gastrointestinal cancer treatment, so we hope that patients can continue with their planned treatment. The results of our trial may help improve care for patients who experience persistent CIT, a complication that has historically been very difficult to manage. ”
Hany Al Samkari, MD, first and corresponding author, classical hematologist at the Massachusetts General Brigham Cancer Institute, Peggy S. Blitz Endowed Chair in Hematology/Oncology at the Massachusetts General Brigham, and associate professor at Harvard Medical School.
Researchers enrolled 47 adults with gastrointestinal cancer and persistent CIT from four cancer centers in the United States, including Massachusetts General Hospital, and randomly assigned them to receive avatrombopag or a placebo. Participants’ ages ranged from 25 to 84 years. Thirty percent were female, 17% were nonwhite, and 11% were Hispanic or Latino. The study was stopped early after an independent monitoring board determined that the trial met predefined stopping criteria for efficacy.
The researchers found that 70% of patients receiving avatrombopag were successfully treated for CIT, defined as platelet recovery and prevention of chemotherapy delay or dose reduction, compared with 17% of patients receiving placebo. Platelet counts recovered in 83% of patients who received the drug, compared with 46% of those who did not receive it. There were no serious adverse events attributable to avatrombopag, and no patients required platelet transfusions.
The authors note that further research is needed to determine whether improved CIT management leads to improved long-term outcomes.
sauce:
Reference magazines:
Al-Samkari, H. Others. (2026) Avatrombopag versus placebo for persistent chemotherapy-induced thrombocytopenia in gastrointestinal cancer: Phase II ACT-GI trial. Journal of Clinical Oncology DOI: 10.1200/JCO-26-00568. https://ascopubs.org/doi/10.1200/JCO-26-00568
