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Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency
PURPOSE OF REVIEW: Hematopoietic cell transplantation (HCT) is an established curative treatment for children with primary immunodeficiencies. This article reviews the latest developments in conditioning regimens for primary immunodeficiency (PID). It focuses on data regarding transplant outcomes ac...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer US
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6861349/ https://www.ncbi.nlm.nih.gov/pubmed/31741098 http://dx.doi.org/10.1007/s11882-019-0883-1 |
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author | Lum, S. H. Hoenig, M. Gennery, A. R. Slatter, M. A. |
author_facet | Lum, S. H. Hoenig, M. Gennery, A. R. Slatter, M. A. |
author_sort | Lum, S. H. |
collection | PubMed |
description | PURPOSE OF REVIEW: Hematopoietic cell transplantation (HCT) is an established curative treatment for children with primary immunodeficiencies. This article reviews the latest developments in conditioning regimens for primary immunodeficiency (PID). It focuses on data regarding transplant outcomes according to newer reduced toxicity conditioning regimens used in HCT for PID. RECENT FINDINGS: Conventional myeloablative conditioning regimens are associated with significant acute toxicities, transplant-related mortality, and late effects such as infertility. Reduced toxicity conditioning regimens have had significant positive impacts on HCT outcome, and there are now well-established strategies in children with PID. Treosulfan has emerged as a promising preparative agent. Use of a peripheral stem cell source has been shown to be associated with better donor chimerism in patients receiving reduced toxicity conditioning. Minimal conditioning regimens using monoclonal antibodies are in clinical trials with promising results thus far. SUMMARY: Reduced toxicity conditioning has emerged as standard of care for PID and has resulted in improved transplant survival for patients with significant comorbidities. |
format | Online Article Text |
id | pubmed-6861349 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | Springer US |
record_format | MEDLINE/PubMed |
spelling | pubmed-68613492019-12-03 Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency Lum, S. H. Hoenig, M. Gennery, A. R. Slatter, M. A. Curr Allergy Asthma Rep Immune Deficiency and Dysregulation(C Kuo, Section Editor) PURPOSE OF REVIEW: Hematopoietic cell transplantation (HCT) is an established curative treatment for children with primary immunodeficiencies. This article reviews the latest developments in conditioning regimens for primary immunodeficiency (PID). It focuses on data regarding transplant outcomes according to newer reduced toxicity conditioning regimens used in HCT for PID. RECENT FINDINGS: Conventional myeloablative conditioning regimens are associated with significant acute toxicities, transplant-related mortality, and late effects such as infertility. Reduced toxicity conditioning regimens have had significant positive impacts on HCT outcome, and there are now well-established strategies in children with PID. Treosulfan has emerged as a promising preparative agent. Use of a peripheral stem cell source has been shown to be associated with better donor chimerism in patients receiving reduced toxicity conditioning. Minimal conditioning regimens using monoclonal antibodies are in clinical trials with promising results thus far. SUMMARY: Reduced toxicity conditioning has emerged as standard of care for PID and has resulted in improved transplant survival for patients with significant comorbidities. Springer US 2019-11-18 2019 /pmc/articles/PMC6861349/ /pubmed/31741098 http://dx.doi.org/10.1007/s11882-019-0883-1 Text en © The Author(s) 2019 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. |
spellingShingle | Immune Deficiency and Dysregulation(C Kuo, Section Editor) Lum, S. H. Hoenig, M. Gennery, A. R. Slatter, M. A. Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency |
title | Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency |
title_full | Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency |
title_fullStr | Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency |
title_full_unstemmed | Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency |
title_short | Conditioning Regimens for Hematopoietic Cell Transplantation in Primary Immunodeficiency |
title_sort | conditioning regimens for hematopoietic cell transplantation in primary immunodeficiency |
topic | Immune Deficiency and Dysregulation(C Kuo, Section Editor) |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6861349/ https://www.ncbi.nlm.nih.gov/pubmed/31741098 http://dx.doi.org/10.1007/s11882-019-0883-1 |
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