TY - JOUR
T1 - 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
AU - Giordano, Paola
AU - Lassandro, Giuseppe
AU - Barone, Angelica
AU - Cesaro, Simone
AU - Fotzi, Ilaria
AU - Giona, Fiorina
AU - Gorio, Chiara
AU - Maggio, Angela
AU - Miano, Maurizio
AU - Marzollo, Antonio
AU - Nardi, Margherita
AU - Pession, Andrea
AU - Ruggiero, Antonio
AU - Russo, Giovanna
AU - Saracco, Paola
AU - Spinelli, Marco
AU - Tolva, Alessandra
AU - Tornesello, Assunta
AU - Palladino, Valentina
AU - Del Vecchio, Giovanni Carlo
PY - 2023
Y1 - 2023
N2 - 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 < 0.05). Patients of group 2 mostly did not achieve a stable platelet count in the first 6 months of treatment and underwent concomitant therapies during follow-up respect of group 1 and group 3 (p < 0.01). ConclusionOur study found that the benefits of eltrombopag treatment, in terms of platelet count improvement and use of additional therapies, are identifiable from the first 6 months of treatment.
AB - 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 < 0.05). Patients of group 2 mostly did not achieve a stable platelet count in the first 6 months of treatment and underwent concomitant therapies during follow-up respect of group 1 and group 3 (p < 0.01). ConclusionOur study found that the benefits of eltrombopag treatment, in terms of platelet count improvement and use of additional therapies, are identifiable from the first 6 months of treatment.
KW - bleeding disorders
KW - children
KW - eltrombopag
KW - immune thrombocytopenia
KW - thrombopoietin receptor agonists
KW - bleeding disorders
KW - children
KW - eltrombopag
KW - immune thrombocytopenia
KW - thrombopoietin receptor agonists
UR - http://hdl.handle.net/10807/270071
U2 - 10.3389/fmed.2023.1214308
DO - 10.3389/fmed.2023.1214308
M3 - Article
SN - 2296-858X
VL - 10
SP - N/A-N/A
JO - Frontiers in Medicine
JF - Frontiers in Medicine
ER -