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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
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 |
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creator | 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 |
description | 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) ( |
doi_str_mv | 10.3389/fmed.2023.1214308 |
format | article |
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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) (
< 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 (
< 0.01).
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.</description><identifier>ISSN: 2296-858X</identifier><identifier>EISSN: 2296-858X</identifier><identifier>DOI: 10.3389/fmed.2023.1214308</identifier><identifier>PMID: 37521342</identifier><language>eng</language><publisher>Switzerland: Frontiers Media S.A</publisher><subject>bleeding disorders ; children ; eltrombopag ; immune thrombocytopenia ; Medicine ; thrombopoietin receptor agonists</subject><ispartof>Frontiers in medicine, 2023-07, Vol.10, p.1214308-1214308</ispartof><rights>Copyright © 2023 Giordano, Lassandro, Barone, Cesaro, Fotzi, Giona, Gorio, Maggio, Miano, Marzollo, Nardi, Pession, Ruggiero, Russo, Saracco, Spinelli, Tolva, Tornesello, Palladino and Del Vecchio.</rights><rights>Copyright © 2023 Giordano, Lassandro, Barone, Cesaro, Fotzi, Giona, Gorio, Maggio, Miano, Marzollo, Nardi, Pession, Ruggiero, Russo, Saracco, Spinelli, Tolva, Tornesello, Palladino and Del Vecchio. 2023 Giordano, Lassandro, Barone, Cesaro, Fotzi, Giona, Gorio, Maggio, Miano, Marzollo, Nardi, Pession, Ruggiero, Russo, Saracco, Spinelli, Tolva, Tornesello, Palladino and Del Vecchio</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c418t-cf96867ff060b588f04f4417df1fdb66feb6efbf263209a4ca780b009aa0bdfe3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC10375288/pdf/$$EPDF$$P50$$Gpubmedcentral$$Hfree_for_read</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC10375288/$$EHTML$$P50$$Gpubmedcentral$$Hfree_for_read</linktohtml><link.rule.ids>230,314,727,780,784,885,27924,27925,53791,53793</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/37521342$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Giordano, Paola</creatorcontrib><creatorcontrib>Lassandro, Giuseppe</creatorcontrib><creatorcontrib>Barone, Angelica</creatorcontrib><creatorcontrib>Cesaro, Simone</creatorcontrib><creatorcontrib>Fotzi, Ilaria</creatorcontrib><creatorcontrib>Giona, Fiorina</creatorcontrib><creatorcontrib>Gorio, Chiara</creatorcontrib><creatorcontrib>Maggio, Angela</creatorcontrib><creatorcontrib>Miano, Maurizio</creatorcontrib><creatorcontrib>Marzollo, Antonio</creatorcontrib><creatorcontrib>Nardi, Margherita</creatorcontrib><creatorcontrib>Pession, Andrea</creatorcontrib><creatorcontrib>Ruggiero, Antonio</creatorcontrib><creatorcontrib>Russo, Giovanna</creatorcontrib><creatorcontrib>Saracco, Paola</creatorcontrib><creatorcontrib>Spinelli, Marco</creatorcontrib><creatorcontrib>Tolva, Alessandra</creatorcontrib><creatorcontrib>Tornesello, Assunta</creatorcontrib><creatorcontrib>Palladino, Valentina</creatorcontrib><creatorcontrib>Del Vecchio, Giovanni Carlo</creatorcontrib><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><title>Frontiers in medicine</title><addtitle>Front Med (Lausanne)</addtitle><description>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) (
< 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 (
< 0.01).
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.</description><subject>bleeding disorders</subject><subject>children</subject><subject>eltrombopag</subject><subject>immune thrombocytopenia</subject><subject>Medicine</subject><subject>thrombopoietin receptor agonists</subject><issn>2296-858X</issn><issn>2296-858X</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2023</creationdate><recordtype>article</recordtype><sourceid>DOA</sourceid><recordid>eNpVUstuEzEUHSEQrUo_gA3ykk2CX5nxsEFVBTRSpLIAiZ3lsa8TVx578HgK-cF-Vz1JqNqV7-Pcc2zfU1XvCV4yJtpPtgezpJiyJaGEMyxeVeeUtvVCrMTv18_is-pyHO8wxoTRFSfsbXXGmhUljNPz6mETwxZlSD2aRkDRIvA5xb6Lg9oiF5DeOW8SBPTX5V3JUgxOI9f3UwCUdweo3uc4QHDqM4J_GYIBgxIoj7yzUKJCOA6gs7sH1E8-Ow2hSBbwAMlB0AfhvAO0zso7FdDVOEbtVHYxzK0fYEqSivAN9CpHH7d7pIJBt0EfknfVG6v8CJen86L69e3rz-ubxeb2-_r6arPQnIi80LatRd1Yi2vcrYSwmFvOSWMssaarawtdDbaztGYUt4pr1Qjc4RIq3BkL7KJaH3lNVHdySK5XaS-jcvJQiGkrVSrv8yAJNaZpDeC2bTmmrWAt7hqKNVeNghoXri9HrmHqyi7nP0nKvyB92QluJ7fxXhI870-IwvDxxJDinwnGLHs3avBeBYjTKKngHLec86ZAyRGqyy7GBPZJh2A5-0nOfpKzn-TJT2Xmw_MLPk38dw97BOvyzgs</recordid><startdate>20230714</startdate><enddate>20230714</enddate><creator>Giordano, Paola</creator><creator>Lassandro, Giuseppe</creator><creator>Barone, Angelica</creator><creator>Cesaro, Simone</creator><creator>Fotzi, Ilaria</creator><creator>Giona, Fiorina</creator><creator>Gorio, Chiara</creator><creator>Maggio, Angela</creator><creator>Miano, Maurizio</creator><creator>Marzollo, Antonio</creator><creator>Nardi, Margherita</creator><creator>Pession, Andrea</creator><creator>Ruggiero, Antonio</creator><creator>Russo, Giovanna</creator><creator>Saracco, Paola</creator><creator>Spinelli, Marco</creator><creator>Tolva, Alessandra</creator><creator>Tornesello, Assunta</creator><creator>Palladino, Valentina</creator><creator>Del Vecchio, Giovanni Carlo</creator><general>Frontiers Media S.A</general><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>5PM</scope><scope>DOA</scope></search><sort><creationdate>20230714</creationdate><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><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></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c418t-cf96867ff060b588f04f4417df1fdb66feb6efbf263209a4ca780b009aa0bdfe3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2023</creationdate><topic>bleeding disorders</topic><topic>children</topic><topic>eltrombopag</topic><topic>immune thrombocytopenia</topic><topic>Medicine</topic><topic>thrombopoietin receptor agonists</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Giordano, Paola</creatorcontrib><creatorcontrib>Lassandro, Giuseppe</creatorcontrib><creatorcontrib>Barone, Angelica</creatorcontrib><creatorcontrib>Cesaro, Simone</creatorcontrib><creatorcontrib>Fotzi, Ilaria</creatorcontrib><creatorcontrib>Giona, Fiorina</creatorcontrib><creatorcontrib>Gorio, Chiara</creatorcontrib><creatorcontrib>Maggio, Angela</creatorcontrib><creatorcontrib>Miano, Maurizio</creatorcontrib><creatorcontrib>Marzollo, Antonio</creatorcontrib><creatorcontrib>Nardi, Margherita</creatorcontrib><creatorcontrib>Pession, Andrea</creatorcontrib><creatorcontrib>Ruggiero, Antonio</creatorcontrib><creatorcontrib>Russo, Giovanna</creatorcontrib><creatorcontrib>Saracco, Paola</creatorcontrib><creatorcontrib>Spinelli, Marco</creatorcontrib><creatorcontrib>Tolva, Alessandra</creatorcontrib><creatorcontrib>Tornesello, Assunta</creatorcontrib><creatorcontrib>Palladino, Valentina</creatorcontrib><creatorcontrib>Del Vecchio, Giovanni Carlo</creatorcontrib><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><collection>Directory of Open Access Journals</collection><jtitle>Frontiers in medicine</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Giordano, Paola</au><au>Lassandro, Giuseppe</au><au>Barone, Angelica</au><au>Cesaro, Simone</au><au>Fotzi, Ilaria</au><au>Giona, Fiorina</au><au>Gorio, Chiara</au><au>Maggio, Angela</au><au>Miano, Maurizio</au><au>Marzollo, Antonio</au><au>Nardi, Margherita</au><au>Pession, Andrea</au><au>Ruggiero, Antonio</au><au>Russo, Giovanna</au><au>Saracco, Paola</au><au>Spinelli, Marco</au><au>Tolva, Alessandra</au><au>Tornesello, Assunta</au><au>Palladino, Valentina</au><au>Del Vecchio, Giovanni Carlo</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>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</atitle><jtitle>Frontiers in medicine</jtitle><addtitle>Front Med (Lausanne)</addtitle><date>2023-07-14</date><risdate>2023</risdate><volume>10</volume><spage>1214308</spage><epage>1214308</epage><pages>1214308-1214308</pages><issn>2296-858X</issn><eissn>2296-858X</eissn><abstract>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) (
< 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 (
< 0.01).
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.</abstract><cop>Switzerland</cop><pub>Frontiers Media S.A</pub><pmid>37521342</pmid><doi>10.3389/fmed.2023.1214308</doi><tpages>1</tpages><oa>free_for_read</oa></addata></record> |
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subjects | bleeding disorders children eltrombopag immune thrombocytopenia Medicine thrombopoietin receptor agonists |
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 |
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