In a phase III trial (RECITE) reported in The New England Journal of Medicine, Al-Samkari et al found that the thrombopoietin receptor agonist romiplostim was effective in treating chemotherapy-induced thrombocytopenia (CIT) among patients receiving oxaliplatin-based multiagent cytotoxic chemotherapy for gastrointestinal cancers.
Study Details
In the international double-blind trial, 165 patients with CIT (platelet count ≤ 85 × 109/L on trial day 1) were randomly assigned 2:1 between September 2019 and October 2023 to receive weekly subcutaneous doses of romiplostim (n = 109) or placebo (n = 56), adjusted to maintain platelet counts of 100 to 250 × 109/L, for three chemotherapy cycles. Stage IV disease was present in 72% of the romiplostim group and 61% of the placebo group. The primary outcome measure was absence of CIT-induced modifications of the chemotherapy dose (reduction, delay, omission, or discontinuation) in both the second and third chemotherapy cycles.
Key Findings
The proportion of patients with no CIT-induced modification of the chemotherapy dose was 84% in the romiplostim group vs 36% in the placebo group, corresponding to an odds ratio of 10.16 (95% confidence interval [CI] = 4.44–23.72, P < .001) and a risk ratio of 2.77 (95% CI = 1.78–4.30, P < .001).
Adverse events of grade 3 or higher occurred in 37% of the romiplostim group and 22% of the placebo group, with most being attributable to chemotherapy toxicity. Adverse events attributed to romiplostim vs placebo occurred in 12% vs 7% of patients; the most common adverse events attributed to romiplostim were nausea (2% vs 2% in the placebo group) and headache (2% vs 0%). Adverse events led to discontinuation of romiplostim vs placebo in 3% vs 2% of patients. Thromboembolic events occurred in 2% of patients in the romiplostim group and none in the placebo group.
The investigators concluded: “In this phase 3, placebo-controlled trial, romiplostim was efficacious in treating CIT.”
Hanny Al-Samkari, MD, of Division of Classical Hematology, Mass General Brigham Cancer Institute, Boston, is the corresponding author for the New England Journal of Medicine article.
DISCLOSURE: The study was funded by Amgen and the Biomedical Advanced Research and Development Authority. For full disclosures of the study authors, visit nejm.com.
