1. 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
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Paola Giordano, Giuseppe Lassandro, Angelica Barone, Simone Cesaro, Ilaria Fotzi, Fiorina Giona, Chiara Gorio, Angela Maggio, Maurizio Miano, Antonio Marzollo, Margherita Nardi, Andrea Pession, Antonio Ruggiero, Giovanna Russo, Paola Saracco, Marco Spinelli, Alessandra Tolva, Assunta Tornesello, Valentina Palladino, and Giovanni Carlo Del Vecchio
- Subjects
immune thrombocytopenia ,eltrombopag ,children ,thrombopoietin receptor agonists ,bleeding disorders ,Medicine (General) ,R5-920 - Abstract
BackgroundThe present multicenter retrospective study on eltrombopag administration in Italian children with chronic ITP aims to extend follow-up of our previous study.Materials and methodsThis 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.Results56 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 (n = 1), inefficacy (n = 10), stable platelet count (n = 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) (p
- Published
- 2023
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