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Review
. 2019 Jun 25;3(12):1907-1915.
doi: 10.1182/bloodadvances.2019000279.

Romiplostim for the Management of Pediatric Immune Thrombocytopenia: Drug Development and Current Practice

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Free PMC article
Review

Romiplostim for the Management of Pediatric Immune Thrombocytopenia: Drug Development and Current Practice

Cindy E Neunert et al. Blood Adv. .
Free PMC article

Abstract

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.

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.

Figures

Figure 1.
Figure 1.
Romiplostim peptide body.
Figure 2.
Figure 2.
TPO receptor pathway.

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