Long term use of eltrombopag in children with chronic immune thrombocytopenia: extended real life retrospective multicenter experience of the Italian Association of Pediatric Hematology and Oncology

The present multicenter retrospective study on eltrombopag administration in Italian children with chronic ITP aims to extend follow-up of our previous study. This retrospective multicenter study was conducted in 17 centers affiliated to the Italian Association of Pediatric Hematology and Oncology (...

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Published in:Frontiers in medicine 2023-07, Vol.10, p.1214308-1214308
Main Authors: Giordano, Paola, Lassandro, Giuseppe, Barone, Angelica, Cesaro, Simone, Fotzi, Ilaria, Giona, Fiorina, Gorio, Chiara, Maggio, Angela, Miano, Maurizio, Marzollo, Antonio, Nardi, Margherita, Pession, Andrea, Ruggiero, Antonio, Russo, Giovanna, Saracco, Paola, Spinelli, Marco, Tolva, Alessandra, Tornesello, Assunta, Palladino, Valentina, Del Vecchio, Giovanni Carlo
Format: Article
Language:English
Subjects:
bleeding disorders
children
eltrombopag
immune thrombocytopenia
Medicine
thrombopoietin receptor agonists
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Summary:The present multicenter retrospective study on eltrombopag administration in Italian children with chronic ITP aims to extend follow-up of our previous study. This retrospective multicenter study was conducted in 17 centers affiliated to the Italian Association of Pediatric Hematology and Oncology (AIEOP). Patients were classified into three subgroups: group 1 included patients who discontinued treatment due to a stable platelet count; group 2 included patients who discontinued treatment due to ineffectiveness; group 3 included patients who did not permanently discontinue treatment. 56 patients were eligible for analysis. The median duration of eltrombopag treatment was 40 months (7-71 months). Twenty patients (36%) discontinued permanently eltrombopag. The reasons of permanent discontinuation were adverse effects ( = 1), inefficacy ( = 10), stable platelet count ( = 9). All patients of group 1 maintained a durable response without additional treatments after eltrombopag discontinuation. We found that patients of group 2 were on treatment for less time (median treatment time: 13.5 months, min: 6.0 - max: 56.0) than patients of group 1 (median treatment time: 34 months, min: 16.0 - max: 62.0) (
ISSN:2296-858X
2296-858X
DOI:10.3389/fmed.2023.1214308