Personalized hematopoietic stem cell transplantation for inborn errors of immunity
Patients with inborn errors of immunity (IEI) have been transplanted for more than 50 years. Many long-term survivors have ongoing medical issues showing the need for further improvements in how hematopoietic stem cell transplantation (HSCT) is performed if patients in the future are to have a norma...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-04-01
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| Series: | Frontiers in Immunology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fimmu.2023.1162605/full |
| _version_ | 1797852218059128832 |
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| author | Mary Slatter Mary Slatter Su Han Lum Su Han Lum |
| author_facet | Mary Slatter Mary Slatter Su Han Lum Su Han Lum |
| author_sort | Mary Slatter |
| collection | DOAJ |
| description | Patients with inborn errors of immunity (IEI) have been transplanted for more than 50 years. Many long-term survivors have ongoing medical issues showing the need for further improvements in how hematopoietic stem cell transplantation (HSCT) is performed if patients in the future are to have a normal quality of life. Precise genetic diagnosis enables early treatment before recurrent infection, autoimmunity and organ impairment occur. Newborn screening for severe combined immunodeficiency (SCID) is established in many countries. For newly described disorders the decision to transplant is not straight-forward. Specific biologic therapies are effective for some diseases and can be used as a bridge to HSCT to improve outcome. Developments in reduced toxicity conditioning and methods of T-cell depletion for mismatched donors have made transplant an option for all eligible patients. Further refinements in conditioning plus precise graft composition and additional cellular therapy are emerging as techniques to personalize the approach to HSCT for each patient |
| first_indexed | 2024-04-09T19:30:28Z |
| format | Article |
| id | doaj.art-e39968aec84c4521bd12c32ed4566466 |
| institution | Directory Open Access Journal |
| issn | 1664-3224 |
| language | English |
| last_indexed | 2024-04-09T19:30:28Z |
| publishDate | 2023-04-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Immunology |
| spelling | doaj.art-e39968aec84c4521bd12c32ed45664662023-04-05T04:39:51ZengFrontiers Media S.A.Frontiers in Immunology1664-32242023-04-011410.3389/fimmu.2023.11626051162605Personalized hematopoietic stem cell transplantation for inborn errors of immunityMary Slatter0Mary Slatter1Su Han Lum2Su Han Lum3Paediatric Immunology and HSCT, Newcastle University, Newcastle upon Tyne, United KingdomTranslational and Clinical Research Institute, Great North Children’s Hospital, Newcastle upon Tyne, United KingdomPaediatric Immunology and HSCT, Newcastle University, Newcastle upon Tyne, United KingdomTranslational and Clinical Research Institute, Great North Children’s Hospital, Newcastle upon Tyne, United KingdomPatients with inborn errors of immunity (IEI) have been transplanted for more than 50 years. Many long-term survivors have ongoing medical issues showing the need for further improvements in how hematopoietic stem cell transplantation (HSCT) is performed if patients in the future are to have a normal quality of life. Precise genetic diagnosis enables early treatment before recurrent infection, autoimmunity and organ impairment occur. Newborn screening for severe combined immunodeficiency (SCID) is established in many countries. For newly described disorders the decision to transplant is not straight-forward. Specific biologic therapies are effective for some diseases and can be used as a bridge to HSCT to improve outcome. Developments in reduced toxicity conditioning and methods of T-cell depletion for mismatched donors have made transplant an option for all eligible patients. Further refinements in conditioning plus precise graft composition and additional cellular therapy are emerging as techniques to personalize the approach to HSCT for each patienthttps://www.frontiersin.org/articles/10.3389/fimmu.2023.1162605/fullhematopoietic stem cell transplantationinborn errors of immunitySCID-severe combined immunodeficiencyconditioningT cell depletion |
| spellingShingle | Mary Slatter Mary Slatter Su Han Lum Su Han Lum Personalized hematopoietic stem cell transplantation for inborn errors of immunity Frontiers in Immunology hematopoietic stem cell transplantation inborn errors of immunity SCID-severe combined immunodeficiency conditioning T cell depletion |
| title | Personalized hematopoietic stem cell transplantation for inborn errors of immunity |
| title_full | Personalized hematopoietic stem cell transplantation for inborn errors of immunity |
| title_fullStr | Personalized hematopoietic stem cell transplantation for inborn errors of immunity |
| title_full_unstemmed | Personalized hematopoietic stem cell transplantation for inborn errors of immunity |
| title_short | Personalized hematopoietic stem cell transplantation for inborn errors of immunity |
| title_sort | personalized hematopoietic stem cell transplantation for inborn errors of immunity |
| topic | hematopoietic stem cell transplantation inborn errors of immunity SCID-severe combined immunodeficiency conditioning T cell depletion |
| url | https://www.frontiersin.org/articles/10.3389/fimmu.2023.1162605/full |
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