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Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents an effective treatment for a variety of inborn errors of immunity (IEI). We report the experience of children affected by IEI who received allo-HSCT over a period of 32 years at IRCCS Istituto Giannina Gaslini, Genoa, Italy. H...

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Autores principales: Dell’Orso, Gianluca, Bagnasco, Francesca, Giardino, Stefano, Pierri, Filomena, Ferrando, Giulia, Di Martino, Daniela, Micalizzi, Concetta, Guardo, Daniela, Volpi, Stefano, Sabatini, Federica, Miano, Maurizio, Gattorno, Marco, Dufour, Carlo, Faraci, Maura
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9941625/
https://www.ncbi.nlm.nih.gov/pubmed/36825011
http://dx.doi.org/10.3389/fimmu.2023.1103080
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author Dell’Orso, Gianluca
Bagnasco, Francesca
Giardino, Stefano
Pierri, Filomena
Ferrando, Giulia
Di Martino, Daniela
Micalizzi, Concetta
Guardo, Daniela
Volpi, Stefano
Sabatini, Federica
Miano, Maurizio
Gattorno, Marco
Dufour, Carlo
Faraci, Maura
author_facet Dell’Orso, Gianluca
Bagnasco, Francesca
Giardino, Stefano
Pierri, Filomena
Ferrando, Giulia
Di Martino, Daniela
Micalizzi, Concetta
Guardo, Daniela
Volpi, Stefano
Sabatini, Federica
Miano, Maurizio
Gattorno, Marco
Dufour, Carlo
Faraci, Maura
author_sort Dell’Orso, Gianluca
collection PubMed
description Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents an effective treatment for a variety of inborn errors of immunity (IEI). We report the experience of children affected by IEI who received allo-HSCT over a period of 32 years at IRCCS Istituto Giannina Gaslini, Genoa, Italy. HSCTs were performed in 67 children with IEI. Kaplan–Meier estimates of overall survival (OS) rate at 5 years in the whole group of patients was 83.4% after a median follow-up of 4 years. Median age at transplant was 2.5 years. Eight allo-HSCTs were complicated by either primary or secondary graft failure (GF), the overall incidence of this complication being 10.9%. Incidence of grade 3–4 acute GvHD (aGvHD) was 18.7%, significantly lower in the haploidentical transplant cohort (p = 0.005). Year of transplant (≤2006 vs. >2006) was the main factor influencing the outcome. In fact, a significant improvement in 5-year OS was demonstrated (92.5% >2006 vs. 65% ≤2006, p = 0.049). Frequency of severe aGvHD was significantly reduced in recent years (≤2006 61.5%, vs. >2006 20%, p = 0.027). A significant progress has been the introduction of the TCR αβ/CD19-depleted haploidentical platform, which was associated with the absence of severe aGvHD. However, it was associated with 23.5% incidence of GF. All but one patient experiencing GF in the this specific cohort were successfully retransplanted. In summary, allo-HSCT is confirmed to be an effective treatment for children with IEI, even in the absence of an HLA-matched donor.
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spelling pubmed-99416252023-02-22 Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience Dell’Orso, Gianluca Bagnasco, Francesca Giardino, Stefano Pierri, Filomena Ferrando, Giulia Di Martino, Daniela Micalizzi, Concetta Guardo, Daniela Volpi, Stefano Sabatini, Federica Miano, Maurizio Gattorno, Marco Dufour, Carlo Faraci, Maura Front Immunol Immunology Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents an effective treatment for a variety of inborn errors of immunity (IEI). We report the experience of children affected by IEI who received allo-HSCT over a period of 32 years at IRCCS Istituto Giannina Gaslini, Genoa, Italy. HSCTs were performed in 67 children with IEI. Kaplan–Meier estimates of overall survival (OS) rate at 5 years in the whole group of patients was 83.4% after a median follow-up of 4 years. Median age at transplant was 2.5 years. Eight allo-HSCTs were complicated by either primary or secondary graft failure (GF), the overall incidence of this complication being 10.9%. Incidence of grade 3–4 acute GvHD (aGvHD) was 18.7%, significantly lower in the haploidentical transplant cohort (p = 0.005). Year of transplant (≤2006 vs. >2006) was the main factor influencing the outcome. In fact, a significant improvement in 5-year OS was demonstrated (92.5% >2006 vs. 65% ≤2006, p = 0.049). Frequency of severe aGvHD was significantly reduced in recent years (≤2006 61.5%, vs. >2006 20%, p = 0.027). A significant progress has been the introduction of the TCR αβ/CD19-depleted haploidentical platform, which was associated with the absence of severe aGvHD. However, it was associated with 23.5% incidence of GF. All but one patient experiencing GF in the this specific cohort were successfully retransplanted. In summary, allo-HSCT is confirmed to be an effective treatment for children with IEI, even in the absence of an HLA-matched donor. Frontiers Media S.A. 2023-02-07 /pmc/articles/PMC9941625/ /pubmed/36825011 http://dx.doi.org/10.3389/fimmu.2023.1103080 Text en Copyright © 2023 Dell’Orso, Bagnasco, Giardino, Pierri, Ferrando, Di Martino, Micalizzi, Guardo, Volpi, Sabatini, Miano, Gattorno, Dufour and Faraci 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 Immunology
Dell’Orso, Gianluca
Bagnasco, Francesca
Giardino, Stefano
Pierri, Filomena
Ferrando, Giulia
Di Martino, Daniela
Micalizzi, Concetta
Guardo, Daniela
Volpi, Stefano
Sabatini, Federica
Miano, Maurizio
Gattorno, Marco
Dufour, Carlo
Faraci, Maura
Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
title Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
title_full Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
title_fullStr Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
title_full_unstemmed Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
title_short Hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
title_sort hematopoietic stem cell transplantation for inborn errors of immunity: 30-year single-center experience
topic Immunology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9941625/
https://www.ncbi.nlm.nih.gov/pubmed/36825011
http://dx.doi.org/10.3389/fimmu.2023.1103080
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