Abstract
Management of patients with corticosteroid-refractory immune thrombocytopaenia (ITP) possesses a significant challenge to practitioners. Until recently, options included splenectomy and immunosuppression. With improved knowledge of both thrombopoiesis and the pathophysiology of ITP, novel drug development with thrombopoietin-receptor agonists (TPO-RAs) was undertaken. Two agents, romiplostim and eltrombopag, are currently approved for use in patients with chronic ITP. Both agents have been shown to increase the platelet count, improve health-related quality of life and reduce bleeding symptoms and concomitant medication use. This review will highlight the discovery of TPO-RA agents, appraise key clinical trials and explore future directions.
Georg Thieme Verlag KG Stuttgart · New York.
MeSH terms
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Administration, Oral
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Adult
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Benzoates / administration & dosage
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Benzoates / adverse effects
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Benzoates / therapeutic use*
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Child
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Child, Preschool
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Clinical Trials as Topic
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Hemorrhage / drug therapy
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Hemorrhage / prevention & control
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Humans
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Hydrazines / administration & dosage
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Hydrazines / adverse effects
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Hydrazines / therapeutic use*
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Immunosuppression Therapy / methods
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Infant
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Infant, Newborn
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Platelet Count
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Purpura, Thrombocytopenic, Idiopathic / drug therapy*
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Purpura, Thrombocytopenic, Idiopathic / prevention & control
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Purpura, Thrombocytopenic, Idiopathic / psychology
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Purpura, Thrombocytopenic, Idiopathic / surgery
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Pyrazoles / administration & dosage
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Pyrazoles / adverse effects
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Pyrazoles / therapeutic use*
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Quality of Life
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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 / adverse effects
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Recombinant Fusion Proteins / therapeutic use*
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Safety
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Splenectomy / methods
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Thrombopoiesis / drug effects*
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Thrombopoiesis / physiology
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Thrombopoietin / administration & dosage
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Thrombopoietin / adverse effects
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Thrombopoietin / therapeutic use*
Substances
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Benzoates
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Hydrazines
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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