Abstract
The thrombopoietin receptor agonists (TPO-RAs) are a class of platelet growth factors used to treat immune thrombocytopenia (ITP) in children and adults. Romiplostim is a peptide TPO-RA approved for over a decade to treat adults with ITP but was just recently US Food and Drug Administration approved to manage ITP in children 1 year of age and older who have had an inadequate response to corticosteroids, intravenous immunoglobulin, or splenectomy. Like the small molecule TPO-RA eltrombopag, romiplostim offers a high clinical response rate in pediatric patients with ITP, but requires use over an extended, and possibly indefinite, duration. This review is a critical appraisal of the role of romiplostim in pediatric ITP, discussing the safety and efficacy of this agent in clinical trials of children and adults and defining the patients most likely to benefit from romiplostim treatment. The treating hematologist is additionally provided guidance with treatment goals, dosing strategies, toxicity management, and indications for discontinuation.