A practical guide to the use of eltrombopag in children with chronic immune thrombocytopenia

ABSTRACT

Introduction: Pediatric immune thrombocytopenia (ITP) may be associated with significant burden on children and their parents/caregivers. Thrombopoietin (TPO) receptor agonists (RAs) have been used to treat adult patients with chronic ITP (cITP) for nearly a decade and following pediatric studies Eltrombopag has been recently approved for pediatric cITP in the United States and Europe. TPO-RA s may help reduce the risk of bleeding and the need for conventional ITP therapies.

Review: In this review, the clinical data demonstrating the efficacy and safety of TPO-RAs in pediatric ITP are evaluated, key recommendations regarding safe administration of eltrombopag are provided, and potential future directions in management of pediatric ITP are discussed.

KEYWORDS:

• Eltrombopag
• pediatric
• romiplostim
• thrombocytopenia
• thrombopoietin receptor agonists

Acknowledgments

All listed authors meet the criteria for authorship set forth by the International Committee of Medical Journal Editors.

Funding and declaration of interest

JDG reports personal fees and nonfinancial support from Novartis, GlaxoSmithKline, Amgen, and grant support from ITP Support Association (UK). ST has nothing to disclose.

Funding

Editorial support for the preparation of this manuscript was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.