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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
BACKGROUND: The present multicenter retrospective study on eltrombopag administration in Italian children with chronic ITP aims to extend follow-up of our previous study. MATERIALS AND METHODS: This retrospective multicenter study was conducted in 17 centers affiliated to the Italian Association of...
Autores principales: | , , , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10375288/ https://www.ncbi.nlm.nih.gov/pubmed/37521342 http://dx.doi.org/10.3389/fmed.2023.1214308 |
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author | 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 |
author_facet | 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 |
author_sort | Giordano, Paola |
collection | PubMed |
description | BACKGROUND: The present multicenter retrospective study on eltrombopag administration in Italian children with chronic ITP aims to extend follow-up of our previous study. MATERIALS AND METHODS: 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. RESULTS: 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 (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). CONCLUSION: Our 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. |
format | Online Article Text |
id | pubmed-10375288 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-103752882023-07-29 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 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 Front Med (Lausanne) Medicine BACKGROUND: The present multicenter retrospective study on eltrombopag administration in Italian children with chronic ITP aims to extend follow-up of our previous study. MATERIALS AND METHODS: 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. RESULTS: 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 (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). CONCLUSION: Our 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. Frontiers Media S.A. 2023-07-14 /pmc/articles/PMC10375288/ /pubmed/37521342 http://dx.doi.org/10.3389/fmed.2023.1214308 Text en Copyright © 2023 Giordano, Lassandro, Barone, Cesaro, Fotzi, Giona, Gorio, Maggio, Miano, Marzollo, Nardi, Pession, Ruggiero, Russo, Saracco, Spinelli, Tolva, Tornesello, Palladino and Del Vecchio. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Medicine 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 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 |
title | 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 |
title_full | 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 |
title_fullStr | 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 |
title_full_unstemmed | 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 |
title_short | 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 |
title_sort | 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 |
topic | Medicine |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10375288/ https://www.ncbi.nlm.nih.gov/pubmed/37521342 http://dx.doi.org/10.3389/fmed.2023.1214308 |
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