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Review
. 2018 Feb 27;2(4):454-461.
doi: 10.1182/bloodadvances.2017010660.

Eltrombopag for use in children with immune thrombocytopenia

Taylor Olmsted Kim  1   2 , Jenny Despotovic  1   2 , Michele P Lambert  3   4
Affiliations
Review

Eltrombopag for use in children with immune thrombocytopenia

Taylor Olmsted Kim et al. Blood Adv. .

Abstract

Eltrombopag is currently the only US Food and Drug Administration-approved thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia (ITP) in children. This oral, once-per-day therapy has shown favorable efficacy and adverse effect profiles in children. Two multicenter, double-blind, placebo controlled clinical trials (PETIT [Efficacy and Safety Study of Eltrombopag in Pediatric Patients With Thrombocytopenia From Chronic Idiopathic Thrombocytopenic Purpura (ITP)] and PETIT2 [Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body]) demonstrated efficacy in raising platelet counts, reducing bleeding, and reducing the need for concomitant ITP therapies with relatively few adverse effects. The most commonly reported drug-related adverse effects include headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety data in children are limited, and studies in adults have not revealed a clinically significant increased incidence of thrombosis, marrow fibrosis, or cataract formation. Eltrombopag has also been approved for treating refractory severe aplastic anemia (AA) and has potential for expanded use in ITP and severe AA as well as in other conditions associated with thrombocytopenia.

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Conflict of interest statement

Conflict-of-interest disclosure: M.P.L. served on advisory boards for Novartis and Bayer and was a consultant for Novartis. J.D. was a consultant for Sanofi-Genzyme. T.O.K. declares no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Important molecules for thrombopoietin signaling, including the site of eltrombopag binding. GRB2, growth factor receptor-bound protein 2; RAF, rapidly accelerated fibrosarcoma; SOS: son of sevenless.
Figure 2.
Figure 2.
Molecular (2D) structure of eltrombopag olamine.
Figure 3.
Figure 3.
Eltrombopag dosing titration.
See this image and copyright information in PMC

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