Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity

Purpose: Use of adoptive immunotherapy with virus-specific T cells (VST) in patients with inborn errors of immunity prior to hematopoietic stem cell transplantation (HSCT) has been reported in few patients. We report our experience, reviewing all the cases previously reported. Methods: We report fou...

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Autores: Alonso Recio, Laura, Méndez Echevarría, Ana, Rudilla, Francesc, Mozo, Yasmina, Soler Palacín, Pere, Sisinni, Luisa, Bueno, David, Riviere, Jacques, Paz, Raquel de, Sánchez Zapardiel, Elena, Querol, Sergi, Rodriguez-Pena, Rebeca, López Granados, Eduardo, Gimeno, Ramón, Díaz de Heredia, Cristina, Pérez Martínez, Antonio
Tipo de recurso: artículo
Fecha de publicación:2021
País:España
Institución:Universidad Autónoma de Madrid
Repositorio:Biblos-e Archivo. Repositorio Institucional de la UAM
Idioma:inglés
OAI Identifier:oai:repositorio.uam.es:10486/715872
Acceso en línea:http://hdl.handle.net/10486/715872
https://dx.doi.org/10.1007/s10875-020-00961-w
Access Level:acceso abierto
Palabra clave:immunotherapy
inborn errors of immunity
primary immunodeficiency diseases
viruses
Medicina
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spelling Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of ImmunityAlonso Recio, LauraMéndez Echevarría, AnaRudilla, FrancescMozo, YasminaSoler Palacín, PereSisinni, LuisaBueno, DavidRiviere, JacquesPaz, Raquel deSánchez Zapardiel, ElenaQuerol, SergiRodriguez-Pena, RebecaLópez Granados, EduardoGimeno, RamónDíaz de Heredia, CristinaPérez Martínez, Antonioimmunotherapyinborn errors of immunityprimary immunodeficiency diseasesvirusesMedicinaPurpose: Use of adoptive immunotherapy with virus-specific T cells (VST) in patients with inborn errors of immunity prior to hematopoietic stem cell transplantation (HSCT) has been reported in few patients. We report our experience, reviewing all the cases previously reported. Methods: We report four children with inborn errors of immunity who received VST infusion in a pre-HSCT setting in two reference centers in Spain and review all inborn errors of immunity cases previously reported. Results: Taking into account our four cases, nine children have been reported to receive VST prior to HSCT to date: 3 severe combined immunodeficiency, 2 CTPS1 deficiency, 1 dyskeratosis congenital, 1 ORAI1 deficiency, 1 Rothmund-Thomson syndrome, and 1 combined immunodeficiency without confirmed genetic defect. In four patients, immunotherapy resulted in clinical improvement, allowing to proceed to HSCT. In these cases, the infusion was started closely to viral diagnosis [mean time 28 days (IQR; 17–52 days)], and the VST was followed shortly thereafter by HSCT [mean time 28 days (IQR; 10–99 days)]. Viremia was controlled after HSCT in two cases (performed 7 and 36 days after the infusion). Multiple infusions were required in many cases. Five out of nine patients died before receiving HSCT. These patients presented with a prolonged and uncontrolled infection before VST administration [mean time from viral diagnosis to VST infusion was 176 days (IQR; 54–1687)]. Conclusions: In patients with inborn errors of immunity, the efficacy of VST for treating disseminated viral infections in pre-transplant settings seems to have a limited efficacy. However, this therapy could be used in a pre-emptive setting before severe viral disease occurs or closely to HSCTSpringerDepartamento de PediatríaFacultad de Medicina20212021-05-01research articlehttp://purl.org/coar/resource_type/c_2df8fbb1AMhttp://purl.org/coar/version/c_ab4af688f83e57aainfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10486/715872https://dx.doi.org/10.1007/s10875-020-00961-wreponame:Biblos-e Archivo. Repositorio Institucional de la UAMinstname:Universidad Autónoma de MadridInglésengopen accesshttp://purl.org/coar/access_right/c_abf2info:eu-repo/semantics/openAccessoai:repositorio.uam.es:10486/7158722026-06-23T12:46:27Z
dc.title.none.fl_str_mv Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
title Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
spellingShingle Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
Alonso Recio, Laura
immunotherapy
inborn errors of immunity
primary immunodeficiency diseases
viruses
Medicina
title_short Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
title_full Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
title_fullStr Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
title_full_unstemmed Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
title_sort Failure of Viral-Specific T Cells Administered in Pre-transplant Settings in Children with Inborn Errors of Immunity
dc.creator.none.fl_str_mv Alonso Recio, Laura
Méndez Echevarría, Ana
Rudilla, Francesc
Mozo, Yasmina
Soler Palacín, Pere
Sisinni, Luisa
Bueno, David
Riviere, Jacques
Paz, Raquel de
Sánchez Zapardiel, Elena
Querol, Sergi
Rodriguez-Pena, Rebeca
López Granados, Eduardo
Gimeno, Ramón
Díaz de Heredia, Cristina
Pérez Martínez, Antonio
author Alonso Recio, Laura
author_facet Alonso Recio, Laura
Méndez Echevarría, Ana
Rudilla, Francesc
Mozo, Yasmina
Soler Palacín, Pere
Sisinni, Luisa
Bueno, David
Riviere, Jacques
Paz, Raquel de
Sánchez Zapardiel, Elena
Querol, Sergi
Rodriguez-Pena, Rebeca
López Granados, Eduardo
Gimeno, Ramón
Díaz de Heredia, Cristina
Pérez Martínez, Antonio
author_role author
author2 Méndez Echevarría, Ana
Rudilla, Francesc
Mozo, Yasmina
Soler Palacín, Pere
Sisinni, Luisa
Bueno, David
Riviere, Jacques
Paz, Raquel de
Sánchez Zapardiel, Elena
Querol, Sergi
Rodriguez-Pena, Rebeca
López Granados, Eduardo
Gimeno, Ramón
Díaz de Heredia, Cristina
Pérez Martínez, Antonio
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Departamento de Pediatría
Facultad de Medicina
dc.subject.none.fl_str_mv immunotherapy
inborn errors of immunity
primary immunodeficiency diseases
viruses
Medicina
topic immunotherapy
inborn errors of immunity
primary immunodeficiency diseases
viruses
Medicina
description Purpose: Use of adoptive immunotherapy with virus-specific T cells (VST) in patients with inborn errors of immunity prior to hematopoietic stem cell transplantation (HSCT) has been reported in few patients. We report our experience, reviewing all the cases previously reported. Methods: We report four children with inborn errors of immunity who received VST infusion in a pre-HSCT setting in two reference centers in Spain and review all inborn errors of immunity cases previously reported. Results: Taking into account our four cases, nine children have been reported to receive VST prior to HSCT to date: 3 severe combined immunodeficiency, 2 CTPS1 deficiency, 1 dyskeratosis congenital, 1 ORAI1 deficiency, 1 Rothmund-Thomson syndrome, and 1 combined immunodeficiency without confirmed genetic defect. In four patients, immunotherapy resulted in clinical improvement, allowing to proceed to HSCT. In these cases, the infusion was started closely to viral diagnosis [mean time 28 days (IQR; 17–52 days)], and the VST was followed shortly thereafter by HSCT [mean time 28 days (IQR; 10–99 days)]. Viremia was controlled after HSCT in two cases (performed 7 and 36 days after the infusion). Multiple infusions were required in many cases. Five out of nine patients died before receiving HSCT. These patients presented with a prolonged and uncontrolled infection before VST administration [mean time from viral diagnosis to VST infusion was 176 days (IQR; 54–1687)]. Conclusions: In patients with inborn errors of immunity, the efficacy of VST for treating disseminated viral infections in pre-transplant settings seems to have a limited efficacy. However, this therapy could be used in a pre-emptive setting before severe viral disease occurs or closely to HSCT
publishDate 2021
dc.date.none.fl_str_mv 2021
2021-05-01
dc.type.none.fl_str_mv research article
http://purl.org/coar/resource_type/c_2df8fbb1
AM
http://purl.org/coar/version/c_ab4af688f83e57aa
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv http://hdl.handle.net/10486/715872
https://dx.doi.org/10.1007/s10875-020-00961-w
url http://hdl.handle.net/10486/715872
https://dx.doi.org/10.1007/s10875-020-00961-w
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Springer
publisher.none.fl_str_mv Springer
dc.source.none.fl_str_mv reponame:Biblos-e Archivo. Repositorio Institucional de la UAM
instname:Universidad Autónoma de Madrid
instname_str Universidad Autónoma de Madrid
reponame_str Biblos-e Archivo. Repositorio Institucional de la UAM
collection Biblos-e Archivo. Repositorio Institucional de la UAM
repository.name.fl_str_mv
repository.mail.fl_str_mv
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