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Successful Use of Eltrombopag in a Pediatric Patient With Human Immunodeficiency Virus (HIV)-Associated Thrombocytopenia.

Thrombocytopenia and other hematologic manifestations related to HIV are not uncommon. Treatment of HIV-related thrombocytopenia is challenging: treatment options are not effective in all patients, or less well studied, particularly in the pediatric population. We aim to present and discuss the case of a 13-year-old with HIV and persistent thrombocytopenia who, after failing monthly IVIG infusions, showed normalization of platelet count on the novel thrombopoietin receptor agonist, eltrombopag. A retrospective chart review of the case patient's medical record was conducted. Additionally, a thorough literature review was performed on this topic, including the pathophysiology of underlying HIV-related thrombocytopenia and its treatment modalities. The patient was treated initially with monthly IVIG infusions for about 1 year but did not show a sustained response, particularly in between infusions. After initiation with eltrombopag 50 mg daily, the patient showed a sustained increase in his platelet count. During a brief lapse in eltrombopag treatment, his platelet count dropped, which then increased upon his reinitiation of therapy. He has continued to show a sustained platelet response and has not been symptomatic or required IVIG for more than 1 year. To our knowledge, this is the first report of a pediatric patient with HIV-related thrombocytopenia who has benefited from the use of eltrombopag.