Eltrombopag for Treatment of Fanconi Anemia

Summary

An open-label, phase II study to assess the efficacy and safety of eltrombopag for the treatment of children and adolescents with Fanconi anemia.

Timeline

Start

2020-12-02

Primary completion

2023-06-19

Completion

2023-08-29

Publications

• Iriondo J, Zubicaray J, Rio P, Catala A, Solsona M, Sanz A, Gomez A, Sebastian E, de la Cruz A, Galan A, Navarro S, Larcher L, de Andoin NG, Uriz JJ, Vagace JM, Gonzalez de Pablo J, Pujol MR, Nicoletti E, Surralles J, Martin-Prado S, Schwartz JD, Soulier J, Bueren JA, Sevilla J. Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV. Eur J Haematol. 2025 Oct;115(4):403-412. doi: 10.1111/ejh.70007. Epub 2025 Jul 16.

Drugs

Indications