Romiplostim prevents chemotherapy-induced thrombocytopenia in phase 3 trial

Results from a global phase 3 clinical trial led by investigators at Mass General Brigham show that the medication romiplostim can effectively prevent chemotherapy from destroying platelet-producing bone marrow cells, a common complication known as chemotherapy-induced thrombocytopenia. Romiplostim boosts the bone marrow's ability to withstand the assault of chemotherapy so that recipients of the medication can continue to make platelets that are needed to prevent bleeding.

The findings, which are published in the New England Journal of Medicine, offer hope for patients whose cancer care is compromised by this challenging and common chemotherapy complication.

"This work has been nearly a decade in the making, and it is so important because there are no available approved medications for chemotherapy-induced thrombocytopenia, which drastically increases a patient's risk of major or life-threatening bleeding," said lead author Hanny Al-Samkari, MD, a classical hematologist at Mass General Brigham Cancer Institute and the Peggy S. Blitz Endowed Chair in Hematology/Oncology. "In an effort to prevent life-threatening bleeding in these patients, oncologists are forced to dose reduce and delay chemotherapy, often repeatedly. We know from other studies that this chemotherapy intensity reduction results in worsened outcomes of cancer treatment, including reduced overall survival and lower chance of cancer cure. Therefore, we hope that romiplostim's ability to allow administration of full-dose chemotherapy delivered on time will translate into longer survival for patients."

Al-Samkari and his colleagues' phase 3 RECITE trial included 165 patients with advanced colorectal cancer, gastroesophageal cancer, or pancreatic cancer. There were 109 in the romiplostim group and 56 in the placebo group.

Patients taking romiplostim had more than 10-fold lower odds of having to reduce their chemotherapy dose due to chemotherapy-induced thrombocytopenia. No chemotherapy dose modifications were made in 84% of patients in the romiplostim group versus 36% in the placebo group. Adverse events of grade 3 or higher occurred in 37% of patients who received romiplostim and in 22% of those who received placebo, primarily reflecting the adverse events of the multiagent chemotherapy patients were receiving, and the fact that patients on romiplostim were able to receive higher doses of chemotherapy.

Adverse events related to romiplostim or placebo occurred in 12% of patients taking romiplostim and in 7% of those taking placebo, with the most frequent being nausea (2% in each group) and headache (2% in the romiplostim group). None of these were serious or led to death or discontinuation of romiplostim, placebo, or chemotherapy. Clotting-related adverse events occurred in 2% of patients taking romiplostim and in no patients taking placebo.