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Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature

Since their license in 2008, studies on thrombopoietin receptor agonists (TPO-RAs) are proceeding at a fast pace. Their favorable efficacy and safety profile makes them good candidates for the management of thrombocytopenia in different settings, even beyond their current indications. In the last 10...

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Autores principales: Capecchi, Marco, Serpenti, Fabio, Giannotta, Juri, Pettine, Loredana, Reda, Gianluigi, Martinelli, Ida, Artoni, Andrea, Barcellini, Wilma, Fattizzo, Bruno
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8505995/
https://www.ncbi.nlm.nih.gov/pubmed/34650908
http://dx.doi.org/10.3389/fonc.2021.680411
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author Capecchi, Marco
Serpenti, Fabio
Giannotta, Juri
Pettine, Loredana
Reda, Gianluigi
Martinelli, Ida
Artoni, Andrea
Barcellini, Wilma
Fattizzo, Bruno
author_facet Capecchi, Marco
Serpenti, Fabio
Giannotta, Juri
Pettine, Loredana
Reda, Gianluigi
Martinelli, Ida
Artoni, Andrea
Barcellini, Wilma
Fattizzo, Bruno
author_sort Capecchi, Marco
collection PubMed
description Since their license in 2008, studies on thrombopoietin receptor agonists (TPO-RAs) are proceeding at a fast pace. Their favorable efficacy and safety profile makes them good candidates for the management of thrombocytopenia in different settings, even beyond their current indications. In the last 10 years, we faced patients with refractory thrombocytopenia that required treatment with off-label TPO-RA, despite the paucity of data in the literature and the possible risks, particularly that of thrombosis. We hereby report our 10-year real-life single-center experience of TPO-RA used off-label. Fourteen patients were divided into three groups according to the etiology of thrombocytopenia: myelodysplastic syndromes, post-transplantation, and lymphoproliferative diseases. Clinical features and results are reported within each group. Overall, TPO-RA proved effective in all these conditions achieving responses also in heavily pretreated patients. The overall response rate (ORR) was 100% in patients with thrombocytopenia after transplantation and in those with lymphoproliferative diseases and 75% in patients with myelodysplastic syndromes. The median duration of therapy was 285 days (range 93–1,513 days). Four patients (29%) discontinued treatment because of lack of response (n=2) or a sustained response (n=2). No grade 3–4 adverse events occurred, particularly no thrombosis. In our real-life experience, TPO-RAs were effective and safe and proved of value in the challenging management of patients with refractory thrombocytopenia associated with different conditions.
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spelling pubmed-85059952021-10-13 Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature Capecchi, Marco Serpenti, Fabio Giannotta, Juri Pettine, Loredana Reda, Gianluigi Martinelli, Ida Artoni, Andrea Barcellini, Wilma Fattizzo, Bruno Front Oncol Oncology Since their license in 2008, studies on thrombopoietin receptor agonists (TPO-RAs) are proceeding at a fast pace. Their favorable efficacy and safety profile makes them good candidates for the management of thrombocytopenia in different settings, even beyond their current indications. In the last 10 years, we faced patients with refractory thrombocytopenia that required treatment with off-label TPO-RA, despite the paucity of data in the literature and the possible risks, particularly that of thrombosis. We hereby report our 10-year real-life single-center experience of TPO-RA used off-label. Fourteen patients were divided into three groups according to the etiology of thrombocytopenia: myelodysplastic syndromes, post-transplantation, and lymphoproliferative diseases. Clinical features and results are reported within each group. Overall, TPO-RA proved effective in all these conditions achieving responses also in heavily pretreated patients. The overall response rate (ORR) was 100% in patients with thrombocytopenia after transplantation and in those with lymphoproliferative diseases and 75% in patients with myelodysplastic syndromes. The median duration of therapy was 285 days (range 93–1,513 days). Four patients (29%) discontinued treatment because of lack of response (n=2) or a sustained response (n=2). No grade 3–4 adverse events occurred, particularly no thrombosis. In our real-life experience, TPO-RAs were effective and safe and proved of value in the challenging management of patients with refractory thrombocytopenia associated with different conditions. Frontiers Media S.A. 2021-09-28 /pmc/articles/PMC8505995/ /pubmed/34650908 http://dx.doi.org/10.3389/fonc.2021.680411 Text en Copyright © 2021 Capecchi, Serpenti, Giannotta, Pettine, Reda, Martinelli, Artoni, Barcellini and Fattizzo 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 Oncology
Capecchi, Marco
Serpenti, Fabio
Giannotta, Juri
Pettine, Loredana
Reda, Gianluigi
Martinelli, Ida
Artoni, Andrea
Barcellini, Wilma
Fattizzo, Bruno
Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature
title Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature
title_full Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature
title_fullStr Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature
title_full_unstemmed Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature
title_short Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature
title_sort off-label use of thrombopoietin receptor agonists: case series and review of the literature
topic Oncology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8505995/
https://www.ncbi.nlm.nih.gov/pubmed/34650908
http://dx.doi.org/10.3389/fonc.2021.680411
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