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Recent advances in treatment of severe primary immunodeficiencies

Primary immunodeficiencies are rare, inborn errors that result in impaired, disordered or uncontrolled immune responses. Whilst symptomatic and prophylactic treatment is available, hematopoietic stem cell transplantation is an option for many diseases, leading to cure of the immunodeficiency and est...

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Detalles Bibliográficos
Autor principal: Gennery, Andrew
Formato: Online Artículo Texto
Lenguaje:English
Publicado: F1000Research 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754032/
https://www.ncbi.nlm.nih.gov/pubmed/26918153
http://dx.doi.org/10.12688/f1000research.7013.1
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author Gennery, Andrew
author_facet Gennery, Andrew
author_sort Gennery, Andrew
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description Primary immunodeficiencies are rare, inborn errors that result in impaired, disordered or uncontrolled immune responses. Whilst symptomatic and prophylactic treatment is available, hematopoietic stem cell transplantation is an option for many diseases, leading to cure of the immunodeficiency and establishing normal physical and psychological health. Newborn screening for some diseases, whilst improving outcomes, is focusing research on safer and less toxic treatment strategies, which result in durable and sustainable immune function without adverse effects. New conditioning regimens have reduced the risk of hematopoietic stem cell transplantation, and new methods of manipulating stem cell sources should guarantee a donor for almost all patients. Whilst incremental enhancements in transplantation technique have gradually improved survival outcomes over time, some of these new applications are likely to radically alter our approach to treating primary immunodeficiencies.
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spelling pubmed-47540322016-02-24 Recent advances in treatment of severe primary immunodeficiencies Gennery, Andrew F1000Res Review Primary immunodeficiencies are rare, inborn errors that result in impaired, disordered or uncontrolled immune responses. Whilst symptomatic and prophylactic treatment is available, hematopoietic stem cell transplantation is an option for many diseases, leading to cure of the immunodeficiency and establishing normal physical and psychological health. Newborn screening for some diseases, whilst improving outcomes, is focusing research on safer and less toxic treatment strategies, which result in durable and sustainable immune function without adverse effects. New conditioning regimens have reduced the risk of hematopoietic stem cell transplantation, and new methods of manipulating stem cell sources should guarantee a donor for almost all patients. Whilst incremental enhancements in transplantation technique have gradually improved survival outcomes over time, some of these new applications are likely to radically alter our approach to treating primary immunodeficiencies. F1000Research 2015-12-16 /pmc/articles/PMC4754032/ /pubmed/26918153 http://dx.doi.org/10.12688/f1000research.7013.1 Text en Copyright: © 2015 Gennery A http://creativecommons.org/licenses/by/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution Licence, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Gennery, Andrew
Recent advances in treatment of severe primary immunodeficiencies
title Recent advances in treatment of severe primary immunodeficiencies
title_full Recent advances in treatment of severe primary immunodeficiencies
title_fullStr Recent advances in treatment of severe primary immunodeficiencies
title_full_unstemmed Recent advances in treatment of severe primary immunodeficiencies
title_short Recent advances in treatment of severe primary immunodeficiencies
title_sort recent advances in treatment of severe primary immunodeficiencies
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4754032/
https://www.ncbi.nlm.nih.gov/pubmed/26918153
http://dx.doi.org/10.12688/f1000research.7013.1
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