Successful discontinuation of eltrombopag in one child with refractory primary immune thrombocytopenia and literature review

The treatment of refractory primary immune thrombocytopenia (RITP) remains challenging because of the lack of well tolerated and effective drugs. Eltrombopag is approved for pediatric patients, who were aged at least 1 year, having chronic primary immune thrombocytopenia in 2015. Eltrombopag can quickly promote platelets and be conveniently used, thereby providing a new treatment option for patients with immune thrombocytopenia. Some patients may sustain their platelet response when treatment is withdrawn, but the mechanism of this phenomenon is unknown. We described a pediatric case of RITP successfully treated with eltrombopag. The platelet count remained normal after 4 years of drug withdrawal. No adverse effect was observed during the treatment. Eltrombopag should be considered well tolerated and effective and has minimal side effects in the treatment of RITP in children, and response can be maintained after eltrombopag is discontinued.