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.
© 2019 by The American Society of Hematology.
Publication types
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Comparative Study
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Historical Article
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Review
MeSH terms
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Adrenal Cortex Hormones / adverse effects
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Adrenal Cortex Hormones / pharmacology
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Benzoates / administration & dosage
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Benzoates / pharmacokinetics*
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Benzoates / pharmacology
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Benzoates / therapeutic use
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Child, Preschool
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Clinical Trials as Topic
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Cloning, Organism / history
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Drug Development / statistics & numerical data*
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Drug Development / trends
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Hemorrhage / prevention & control
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History, 20th Century
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Humans
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Hydrazines / administration & dosage
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Hydrazines / pharmacokinetics*
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Hydrazines / pharmacology
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Hydrazines / therapeutic use
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Immunoglobulins / adverse effects
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Immunoglobulins / pharmacology
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Infant
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Injections, Subcutaneous
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Platelet Count / methods
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Platelet Count / trends
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Practice Guidelines as Topic
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Purpura, Thrombocytopenic, Idiopathic / chemically induced
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Purpura, Thrombocytopenic, Idiopathic / drug therapy*
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Purpura, Thrombocytopenic, Idiopathic / etiology
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Pyrazoles / administration & dosage
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Pyrazoles / pharmacokinetics*
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Pyrazoles / pharmacology
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Pyrazoles / therapeutic use
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Receptors, Fc / administration & dosage
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Receptors, Fc / therapeutic use
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Receptors, Thrombopoietin / agonists*
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Recombinant Fusion Proteins / administration & dosage
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Recombinant Fusion Proteins / pharmacokinetics*
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Recombinant Fusion Proteins / pharmacology
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Recombinant Fusion Proteins / therapeutic use
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Safety
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Splenectomy / adverse effects
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Splenectomy / methods
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Thrombocytopenia, Neonatal Alloimmune / drug therapy
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Thrombopoietin / administration & dosage
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Thrombopoietin / genetics*
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Thrombopoietin / pharmacokinetics
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Thrombopoietin / pharmacology
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Thrombopoietin / therapeutic use
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United States / epidemiology
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United States Food and Drug Administration
Substances
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Adrenal Cortex Hormones
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Benzoates
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Hydrazines
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Immunoglobulins
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Pyrazoles
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Receptors, Fc
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Receptors, Thrombopoietin
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Recombinant Fusion Proteins
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Thrombopoietin
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romiplostim
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eltrombopag