Back to Search Start Over

The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review

Authors :
Maria Maddalena Marrapodi
Annamaria Mascolo
Domenico Roberti
Martina Di Martino
Concetta Rafaniello
Consiglia Riccardi
Francesca Rossi
Source :
Frontiers in Pediatrics, Vol 11 (2023)
Publication Year :
2023
Publisher :
Frontiers Media S.A., 2023.

Abstract

BackgroundAcquired aplastic anemia (AAA) in pediatric patients is a rare disorder characterized by hypocellular bone marrow and pancytopenia. Eltrombopag, an oral thrombopoietin receptor agonist, provides a hematologic improvement in adults with severe aplastic anemia (SAA) refractory to immunosuppressive therapy (IST). The association of ELT and IST was approved by the US Food and Drug Administration (FDA) for adults and children ≥2 years of age as a first-line treatment for SAA. However, the effects of ELT on pediatric patients with SAA remain controversial and limited.Methods and findingsWe conducted a systematic review of the most recent literature from Pubmed, Web of Science, and Embase, published up to 20th December 2022, in order to evaluate the available evidence on the efficacy and safety of ELT added to IST for the treatment of SAA in the pediatric population.ConclusionEltrombopag added to the IST has shown a good safety profile, without manifestations of excessive toxic effects, although not all the results obtained from our studies support the addition of ELT to the IST in the first-line treatment of children with SAA.Systematic Review Registrationhttps://www.crd.york.ac.uk/prospero/, identifier: CRD42022325859.

Details

Language :
English
ISSN :
22962360
Volume :
11
Database :
Directory of Open Access Journals
Journal :
Frontiers in Pediatrics
Publication Type :
Academic Journal
Accession number :
edsdoj.2ece9ad6f59456899aae326d9a0c669
Document Type :
article
Full Text :
https://doi.org/10.3389/fped.2023.1149718