Since successful cloning of thrombopoietin (TPO) in 1994, significant advances have been made in the development of recombinant TPO receptor agonists. The US Food and Drug Administration (FDA) has approved 2 agents for use in patients with immune thrombocytopenia (ITP): eltrombopag and romiplostim. Romiplostim is a once-weekly subcutaneous injection that has been shown to increase the platelet count, lessen bleeding, and reduce concurrent medication use in adults with ITP. In December 2018, the US FDA approved romiplostim for use in pediatric patients ≥1 year of age with ITP of >6 months' duration and insufficient response to corticosteroids, immunoglobulins, or splenectomy, based on similarly favorable clinical trial data. In addition, romiplostim is well tolerated, making it an attractive option for the treatment of children. Expansion of off-label romiplostim use is being reported in children for ITP <6 months, neonatal thrombocytopenia, hereditary thrombocytopenias, and chemotherapy- and bone marrow transplant-associated thrombocytopenia. We review here the development of romiplostim with a focus on pediatric use.
© 2019 by The American Society of Hematology.
Conflict of interest statement
Conflict-of-interest disclosure: C.E.N. is a consultant for Sanofi-Genzyme. M.J.R. declares no competing financial interests.
Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP).Pediatr Blood Cancer. 2015 Feb;62(2):208-213. doi: 10.1002/pbc.25136. Epub 2014 Oct 24. Pediatr Blood Cancer. 2015. PMID: 25345874 Free PMC article. Clinical Trial.
Thrombopoietin receptor agonists in refractory immune thrombocytopenia: differential responses to eltrombopag and romiplostim: a case report and possible explanations.J Clin Pharm Ther. 2012 Dec;37(6):729-32. doi: 10.1111/j.1365-2710.2012.01353.x. Epub 2012 May 14. J Clin Pharm Ther. 2012. PMID: 22583038
The burden of immune thrombocytopenia in adults: evaluation of the thrombopoietin receptor agonist romiplostim.J Med Econ. 2012;15(5):956-76. doi: 10.3111/13696998.2012.688902. Epub 2012 May 23. J Med Econ. 2012. PMID: 22533524 Review.
Romiplostim in chronic immune thrombocytopenic purpura.Clin Ther. 2009 Sep;31(9):1887-907. doi: 10.1016/j.clinthera.2009.09.013. Clin Ther. 2009. PMID: 19843480 Review.
Romiplostim: a review of its use in immune thrombocytopenia.Drugs. 2012 Feb 12;72(3):415-35. doi: 10.2165/11208260-000000000-00000. Drugs. 2012. PMID: 22316355 Review.
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