Romiplostim was found to effectively prevent chemotherapy-induced thrombocytopenia, a complication where chemotherapy destroys platelet-producing bone marrow cells, according to results from a global phase 3 clinical trial led by investigators at Mass General Brigham and published on Mar. 12 in the New England Journal of Medicine.
This finding is significant because there are currently no approved medications for this condition, which can increase a patient's risk of major or life-threatening bleeding. The lack of treatment options often forces oncologists to reduce or delay chemotherapy doses, potentially leading to worse cancer outcomes.
"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."
The phase 3 RECITE trial included 165 patients with advanced colorectal cancer, gastroesophageal cancer, or pancreatic cancer. Of these participants, 109 received romiplostim while 56 received placebo. Patients taking romiplostim had more than ten times lower odds of needing to reduce their chemotherapy dose due to thrombocytopenia compared with those receiving placebo. No modifications were made in 84% of patients in the romiplostim group versus 36% in the placebo group.
Adverse events rated grade 3 or higher occurred in 37% of those who received romiplostim and in 22% who received placebo; these rates primarily reflected side effects from multiagent chemotherapy regimens and the fact that patients on romiplostim could receive higher doses. Adverse events related specifically to romiplostim or placebo occurred in 12% and 7% respectively; nausea and headache were most common but none were serious nor led to discontinuation or death.
