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Case Report: A case of pediatric persistent refractory ITP responsive to avatrombopag

Antonella Sau1, Gianfranco Di Prinzio1, Daniela Onofrillo1

  • 1Department of Hematology and Oncology, Santo Spirito Hospital, Pescara, Italy.

Frontiers in Immunology
|April 30, 2025
PubMed

Immune thrombocytopenia (ITP) is a rare hematologic disorder characterized by low platelet counts due to an immune-mediated destruction of platelets. While corticosteroids, intravenous immunoglobulin (IVIG) are the mainstays of treatment, a subset of patients may remain refractory to these therapies. Here, we present a case of a 6-year-old girl diagnosed with refractory ITP, who failed to respond to standard therapies but showed a remarkable clinical improvement with avatrombopag, a thrombopoietin receptor agonist.

Keywords:
avatrombopagchildrenimmune thrombocytopaenia (ITP)persistent or chronic immune thrombocytopeniaplateletthrombopoietin receptor (TPOr) agonists

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