Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients

[Background/Objectives]: Immune effector cell-associated hematotoxicity (ICAHT) is a frequent adverse event after chimeric antigen receptor (CAR)-T cell therapy. Grade ≥ 3 thrombocytopenia occurs in around one-third of patients, and many of them become platelet transfusion-dependent. Eltrombopag is...

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Autores: Mingot-Castellano, María Eva, Reguera-Ortega, Juan Luis, Zafra Torres, Denis, Hernani, Rafael, López-Godino, Oriana, Guerreiro, Manuel, Herrero, Blanca, López-Corral, Lucía, Luna, Alejandro, Gonzalez-Pinedo, Lesli, Chinea, Anabelle, Africa-Martín, Ana, Bailén, Rebeca, Martínez-Cibrián, Nuria, Balsalobre, Pascual, Filaferro, Silvia, Alonso-Saladrigues, Anna, Barba, Pere, Pérez-Martínez, Antonio, Pérez-Simón, José A., Sánchez-Pina, José María
Tipo de documento: artigo
Estado:Versão publicada
Data de publicação:2024
País:España
Recursos:Consejo Superior de Investigaciones Científicas (CSIC)
Repositório:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/382217
Acesso em linha:http://hdl.handle.net/10261/382217
Access Level:Acceso aberto
Palavra-chave:CAR-T
Thrombocytopenia
Platelet transfusion
Neutropenia
Red blood cell transfusion
Pan-cytopenia
Thrombopoietin receptor agonist
Eltrombopag
Adverse events
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spelling Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated PatientsMingot-Castellano, María EvaReguera-Ortega, Juan LuisZafra Torres, DenisHernani, RafaelLópez-Godino, OrianaGuerreiro, ManuelHerrero, BlancaLópez-Corral, LucíaLuna, AlejandroGonzalez-Pinedo, LesliChinea, AnabelleAfrica-Martín, AnaBailén, RebecaMartínez-Cibrián, NuriaBalsalobre, PascualFilaferro, SilviaAlonso-Saladrigues, AnnaBarba, PerePérez-Martínez, AntonioPérez-Simón, José A.Sánchez-Pina, José MaríaCAR-TThrombocytopeniaPlatelet transfusionNeutropeniaRed blood cell transfusionPan-cytopeniaThrombopoietin receptor agonistEltrombopagAdverse events[Background/Objectives]: Immune effector cell-associated hematotoxicity (ICAHT) is a frequent adverse event after chimeric antigen receptor (CAR)-T cell therapy. Grade ≥ 3 thrombocytopenia occurs in around one-third of patients, and many of them become platelet transfusion-dependent. Eltrombopag is a thrombopoietin receptor agonist (TPO-RA) able to accelerate megakaryopoiesis, which has been used successfully in patients with bone marrow failure and immune thrombocytopenia (ITP). Its role in managing thrombocytopenia and other cytopenias in CAR-T cell-treated patients has been scarcely addressed. Our aim was to report the safety and efficacy of this approach in patients included in the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC) registry.[Methods]: This is a retrospective, multicenter, observational study. Patients who developed platelet transfusion dependence subsequently to CAR-T cells and received eltrombopag to improve platelet counts were recruited in 10 Spanish hospitals.[Results]: Thirty-eight patients were enrolled and followed up for a median (interquartile range [IQR]) of 175 (99, 489) days since CAR-T cell infusion. At the moment eltrombopag was indicated, 18 patients had thrombocytopenia and another severe cytopenia, while 8 patients had severe pancytopenia. After 32 (14, 38) days on eltrombopag, 29 (76.3%) patients recovered platelet transfusion independence. The number of platelet units transfused correlated with the time needed to restore platelet counts higher than 20 × 109/L (Rho = 0.639, p < 0.001). Non-responders to eltrombopag required more platelet units (58 [29, 69] vs. 12 [6, 26] in responders, p = 0.002). Nineteen out of twenty-three (82.6%) patients recovered from severe neutropenia after 22 (11, 31) days on eltrombopag. Twenty-nine out of thirty-five (82.9%) patients recovered red blood cell (RBC) transfusion independence after 29 (17, 44) days. Seven patients recovered all cell lineages while on treatment. No thromboembolic events were reported. Only two transient toxicities (cholestasis, hyperbilirubinemia) were reported during eltrombopag treatment, none of which compelled permanent drug withdrawal.[Conclusions]: Eltrombopag could be safely used to manage thrombocytopenia and accelerate transfusion independence in CAR-T cell-treated patients.Peer reviewedMultidisciplinary Digital Publishing InstituteConsejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]202520252024info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versioninfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/10261/382217reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Ingléshttps://doi.org/10.3390/jcm13175117Síinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/3822172026-05-22T06:33:51Z
dc.title.none.fl_str_mv Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
title Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
spellingShingle Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
Mingot-Castellano, María Eva
CAR-T
Thrombocytopenia
Platelet transfusion
Neutropenia
Red blood cell transfusion
Pan-cytopenia
Thrombopoietin receptor agonist
Eltrombopag
Adverse events
title_short Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
title_full Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
title_fullStr Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
title_full_unstemmed Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
title_sort Use of Eltrombopag to Improve Thrombocytopenia and Tranfusion Requirement in Anti-CD19 CAR-T Cell-Treated Patients
dc.creator.none.fl_str_mv Mingot-Castellano, María Eva
Reguera-Ortega, Juan Luis
Zafra Torres, Denis
Hernani, Rafael
López-Godino, Oriana
Guerreiro, Manuel
Herrero, Blanca
López-Corral, Lucía
Luna, Alejandro
Gonzalez-Pinedo, Lesli
Chinea, Anabelle
Africa-Martín, Ana
Bailén, Rebeca
Martínez-Cibrián, Nuria
Balsalobre, Pascual
Filaferro, Silvia
Alonso-Saladrigues, Anna
Barba, Pere
Pérez-Martínez, Antonio
Pérez-Simón, José A.
Sánchez-Pina, José María
author Mingot-Castellano, María Eva
author_facet Mingot-Castellano, María Eva
Reguera-Ortega, Juan Luis
Zafra Torres, Denis
Hernani, Rafael
López-Godino, Oriana
Guerreiro, Manuel
Herrero, Blanca
López-Corral, Lucía
Luna, Alejandro
Gonzalez-Pinedo, Lesli
Chinea, Anabelle
Africa-Martín, Ana
Bailén, Rebeca
Martínez-Cibrián, Nuria
Balsalobre, Pascual
Filaferro, Silvia
Alonso-Saladrigues, Anna
Barba, Pere
Pérez-Martínez, Antonio
Pérez-Simón, José A.
Sánchez-Pina, José María
author_role author
author2 Reguera-Ortega, Juan Luis
Zafra Torres, Denis
Hernani, Rafael
López-Godino, Oriana
Guerreiro, Manuel
Herrero, Blanca
López-Corral, Lucía
Luna, Alejandro
Gonzalez-Pinedo, Lesli
Chinea, Anabelle
Africa-Martín, Ana
Bailén, Rebeca
Martínez-Cibrián, Nuria
Balsalobre, Pascual
Filaferro, Silvia
Alonso-Saladrigues, Anna
Barba, Pere
Pérez-Martínez, Antonio
Pérez-Simón, José A.
Sánchez-Pina, José María
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]
dc.subject.none.fl_str_mv CAR-T
Thrombocytopenia
Platelet transfusion
Neutropenia
Red blood cell transfusion
Pan-cytopenia
Thrombopoietin receptor agonist
Eltrombopag
Adverse events
topic CAR-T
Thrombocytopenia
Platelet transfusion
Neutropenia
Red blood cell transfusion
Pan-cytopenia
Thrombopoietin receptor agonist
Eltrombopag
Adverse events
description [Background/Objectives]: Immune effector cell-associated hematotoxicity (ICAHT) is a frequent adverse event after chimeric antigen receptor (CAR)-T cell therapy. Grade ≥ 3 thrombocytopenia occurs in around one-third of patients, and many of them become platelet transfusion-dependent. Eltrombopag is a thrombopoietin receptor agonist (TPO-RA) able to accelerate megakaryopoiesis, which has been used successfully in patients with bone marrow failure and immune thrombocytopenia (ITP). Its role in managing thrombocytopenia and other cytopenias in CAR-T cell-treated patients has been scarcely addressed. Our aim was to report the safety and efficacy of this approach in patients included in the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC) registry.
publishDate 2024
dc.date.none.fl_str_mv 2024
2025
2025
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Publisher's version
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/382217
url http://hdl.handle.net/10261/382217
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv https://doi.org/10.3390/jcm13175117

dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
collection DIGITAL.CSIC. Repositorio Institucional del CSIC
repository.name.fl_str_mv
repository.mail.fl_str_mv
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