Romiplostim was beneficial in treating chemotherapy-induced thrombocytopenia, according to findings from the global phase III RECITE trial published in The New England Journal of Medicine.
“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 the 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.”
Study Methods
The international, double-blind, randomized, placebo-controlled phase III RECITE trial enrolled patients with gastrointestinal cancers who had persistent chemotherapy-induced thrombocytopenia from their oxaliplatin-based cytotoxic chemotherapy regimens. Patients (n = 165) were randomly assigned 2:1 to receive either romiplostim or placebo for three cycles of chemotherapy.
The primary endpoint was the absence of modification of their chemotherapy dose in both the second and third cycles due to chemotherapy-induced thrombocytopenia.
Key Findings
Eighty-four percent of patients in the romiplostim arm had no chemotherapy dose modifications due to chemotherapy-induced thrombocytopenia compared with 36% of patients who received placebo (odds ratio = 10.16; 95% confidence interval [CI] = 4.44–23.72; P < .001; risk ratio = 2.77; 95% CI = 1.78–4.30; P < .001).
Adverse events of grade 3 or higher were reported in 37% of patients in the romiplostim arm and in 22% of patients in the placebo arm. Treatment-related adverse events were observed in 12% and 7% of the romiplostim and placebo arms, respectively. The most common adverse events were nausea and headache, though no events led to death or treatment discontinuation. Thromboembolic events were reported in 2% of patients in the romiplostim arm.
DISCLOSURES: The study was supported by Amgen and the Biomedical Advanced Research and Development Authority. Dr. Al-Samkari is a consultant for Amgen and receives research funding to his institution from Amgen. For full disclosures of the study authors, visit nejm.org.
