Case Report: Eltrombopag in mosaic and gene therapy-treated patients with Fanconi anemia

Case Report: Eltrombopag in mosaic and gene therapy-treated patients with Fanconi anemia

Fanconi anemia (FA) constitutes the most common of the inherited bone marrow failure syndromes, a group of rare heterogeneous disorders characterized by cytopenia, predisposition to hematologic and solid malignancies and diverse clinical features. Currently, the only available hematopoietic curative...

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Bibliographic Details
Main Authors: Josune Zubicaray, June Iriondo, Elena Sebastián, Alejandro Sanz, Paula Rio, Jean Soulier, Sonsoles San Román, José J. Uriz, Susana Navarro, Eileen Nicoletti, Juan A. Bueren, Jonathan D. Schwartz, Julián Sevilla
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-08-01
Series:Frontiers in Pediatrics
Subjects:
Fanconi anemia
bone marrow failure
eltrombopag
gene therapy
mosaicism
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2025.1625751/full
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