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 i...

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Autores: Mingot-Castellano ME, Reguera-Ortega JL, Zafra Torres D, Hernani R, Lopez-Godino O, Guerreiro M, Herrero B, López-Corral L, Luna A, Gonzalez-Pinedo L, Chinea-Rodriguez A, Africa-Martín A, Bailen R, Martinez-Cibrian N, Balsalobre P, Filaferro S, Alonso-Saladrigues A, Barba P, Perez-Martinez A, Calbacho M, Perez-Simón JA, Sánchez-Pina JM, On Behalf Of The Spanish Group Of Hematopoietic Transplant And Cell Therapy Get
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2024
País:España
Institución:Fundació Sant Joan de Déu
Repositorio:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
OAI Identifier:oai:fsjd.fundanetsuite.com:p26758
Acceso en línea:https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=26758
Access Level:acceso abierto
Palabra clave: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 MEReguera-Ortega JLZafra Torres DHernani RLopez-Godino OGuerreiro MHerrero BLópez-Corral LLuna AGonzalez-Pinedo LChinea-Rodriguez AAfrica-Martín ABailen RMartinez-Cibrian NBalsalobre PFilaferro SAlonso-Saladrigues ABarba PPerez-Martinez ACalbacho MPerez-Simón JASánchez-Pina JMOn Behalf Of The Spanish Group Of Hematopoietic Transplant And Cell Therapy GetCAR-Tthrombocytopeniaplatelet transfusionneutropeniared blood cell transfusionpan-cytopeniathrombopoietin receptor agonisteltrombopagadverse eventsBackground/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 x 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.MDPI2024info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionhttps://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=26758Journal of Clinical MedicineISSN: 20770383reponame:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déuinstname:Fundació Sant Joan de DéuInglésinfo:eu-repo/semantics/openAccessoai:fsjd.fundanetsuite.com:p267582026-05-27T12:37:41Z
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 ME
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 ME
Reguera-Ortega JL
Zafra Torres D
Hernani R
Lopez-Godino O
Guerreiro M
Herrero B
López-Corral L
Luna A
Gonzalez-Pinedo L
Chinea-Rodriguez A
Africa-Martín A
Bailen R
Martinez-Cibrian N
Balsalobre P
Filaferro S
Alonso-Saladrigues A
Barba P
Perez-Martinez A
Calbacho M
Perez-Simón JA
Sánchez-Pina JM
On Behalf Of The Spanish Group Of Hematopoietic Transplant And Cell Therapy Get
author Mingot-Castellano ME
author_facet Mingot-Castellano ME
Reguera-Ortega JL
Zafra Torres D
Hernani R
Lopez-Godino O
Guerreiro M
Herrero B
López-Corral L
Luna A
Gonzalez-Pinedo L
Chinea-Rodriguez A
Africa-Martín A
Bailen R
Martinez-Cibrian N
Balsalobre P
Filaferro S
Alonso-Saladrigues A
Barba P
Perez-Martinez A
Calbacho M
Perez-Simón JA
Sánchez-Pina JM
On Behalf Of The Spanish Group Of Hematopoietic Transplant And Cell Therapy Get
author_role author
author2 Reguera-Ortega JL
Zafra Torres D
Hernani R
Lopez-Godino O
Guerreiro M
Herrero B
López-Corral L
Luna A
Gonzalez-Pinedo L
Chinea-Rodriguez A
Africa-Martín A
Bailen R
Martinez-Cibrian N
Balsalobre P
Filaferro S
Alonso-Saladrigues A
Barba P
Perez-Martinez A
Calbacho M
Perez-Simón JA
Sánchez-Pina JM
On Behalf Of The Spanish Group Of Hematopoietic Transplant And Cell Therapy Get
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
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. 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 x 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.
publishDate 2024
dc.date.none.fl_str_mv 2024
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=26758
url https://fsjd.fundanetsuite.com/Publicaciones/ProdCientif/PublicacionFrw.aspx?id=26758
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv MDPI
publisher.none.fl_str_mv MDPI
dc.source.none.fl_str_mv Journal of Clinical Medicine
ISSN: 20770383
reponame:r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
instname:Fundació Sant Joan de Déu
instname_str Fundació Sant Joan de Déu
reponame_str r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
collection r-FSJD. Repositorio Institucional de Producción Científica de la Fundació Sant Joan de Déu
repository.name.fl_str_mv
repository.mail.fl_str_mv
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