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

**Authors:** Antonella Sau, Gianfranco Di Prinzio, Daniela Onofrillo, Anna Caterina Russo, Nicole Santoro, Mauro Di Ianni

PMC · DOI: 10.3389/fimmu.2025.1576053 · Frontiers in Immunology · 2025-04-15

## TL;DR

A 6-year-old girl with persistent ITP showed improvement after treatment with avatrombopag when standard therapies failed.

## Contribution

Demonstrates avatrombopag's effectiveness in treating refractory pediatric ITP.

## Key findings

- Standard therapies failed to improve the patient's ITP.
- Avatrombopag led to significant clinical improvement in the patient.

## Abstract

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.

## Linked entities

- **Chemicals:** avatrombopag (PubChem CID 9852519)
- **Diseases:** immune thrombocytopenia (MONDO:0002048), ITP (MONDO:0008558)

## Full-text entities

- **Genes:** MPL (MPL proto-oncogene, thrombopoietin receptor) [NCBI Gene 4352] {aka C-MPL, CD110, MPLV, THCYT2, THPOR, TPOR}
- **Diseases:** hematologic disorder (MESH:D006402), ITP (MESH:D016553)
- **Chemicals:** avatrombopag (MESH:C533238)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

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## Figures

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## References

11 references — full list in the complete paper: https://tomesphere.com/paper/PMC12037552/full.md

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Source: https://tomesphere.com/paper/PMC12037552