Eltrombopag for Newly Diagnosed Pediatric Immune Thrombocytopenia Requiring Treatment

What is the efficacy of eltrombopag compared with standard therapy in children with newly diagnosed immune thrombocytopenia requiring treatment?In this randomized clinical trial including 118 pediatric patients with newly diagnosed immune thrombocytopenia (63% of whom experienced initial treatment failure after observation or medical therapy), the percentage achieving a sustained platelet response was significantly higher in those treated with eltrombopag (65%) vs those treated with standard therapy (35%).In pediatric patients with newly diagnosed immune thrombocytopenia and nonsevere bleeding who require pharmacological treatment, eltrombopag is more likely to achieve a sustained platelet response vs standard therapy.

Corresponding Author: Kristin A. Shimano, MD, UCSF Benioff Children’s Hospital, 550 16th St, San Francisco, CA 94158 (kristin.shimano@ucsf.edu).

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