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Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders

The basis of allogeneic hemopoietic stem cell (HSC) transplantation in thalassemia consists in substituting the ineffective thalassemic erythropoiesis with and allogeneic effective one. This cellular replacement therapy is an efficient way to obtain a long lasting, probably permanent, clinical effec...

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Autores principales: Angelucci, Emanuele, Pilo, Federica, Targhetta, Clara, Pettinau, Martina, Depau, Cristina, Cogoni, Claudia, Usai, Sara, Pani, Mario, Dessì, Laura, Baronciani, Donatella
Formato: Texto
Lenguaje:English
Publicado: Università Cattolica del Sacro Cuore 2009
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3033161/
https://www.ncbi.nlm.nih.gov/pubmed/21415993
http://dx.doi.org/10.4084/MJHID.2009.015
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author Angelucci, Emanuele
Pilo, Federica
Targhetta, Clara
Pettinau, Martina
Depau, Cristina
Cogoni, Claudia
Usai, Sara
Pani, Mario
Dessì, Laura
Baronciani, Donatella
author_facet Angelucci, Emanuele
Pilo, Federica
Targhetta, Clara
Pettinau, Martina
Depau, Cristina
Cogoni, Claudia
Usai, Sara
Pani, Mario
Dessì, Laura
Baronciani, Donatella
author_sort Angelucci, Emanuele
collection PubMed
description The basis of allogeneic hemopoietic stem cell (HSC) transplantation in thalassemia consists in substituting the ineffective thalassemic erythropoiesis with and allogeneic effective one. This cellular replacement therapy is an efficient way to obtain a long lasting, probably permanent, clinical effective correction of the anaemia avoiding transfusion requirement and subsequent complications like iron overload. The first HSC transplant for thalassemia was performed in Seattle on Dec 2, 1981. In the early eighties transplantation procedure was limited to very few centres worldwide. Between 17 December 1981 and 31 January 2003, over 1000 consecutive patients, aged from 1 to 35 years, underwent transplantation in Pesaro. After the pioneering work by the Seattle and Pesaro groups, this therapeutic approach is now widely applied worldwide. Medical therapy of thalassemia is one of the most spectacular successes of the medical practice in the last decades. In recent years advances in knowledge of iron overload patho-physiopathology, improvement and diffusion of diagnostic capability together with the development of new effective and safe oral chelators promise to further increase success of medical therapy. Nevertheless situation is dramatically different in non-industrialized countries were the very large majority of patients live today. Transplantation technologies have improved substantially during the last years and transplantation outcome is likely to be much better today than in the ‘80s. Recent data indicated a probability of overall survival and thalassemia free survival of 97% and 89% for patients with no advanced disease and of 87% and 80% for patients with advanced disease. Thus the central role of HSC in thalassemia has now been fully established. HSC remains the only definitive curative therapy for thalassemia and other hemoblobinopathies. The development of oral chelators has not changed this position. However this has not settled the controversy on how this curative but potentially lethal treatment stands in front of medical therapy for adults and advanced disease patients. In sickle cell disease HSC transplantation currently is reserved almost exclusively for patients with clinical features that indicate a poor outcome or significant sickle-related morbidity.
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spelling pubmed-30331612011-03-17 Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders Angelucci, Emanuele Pilo, Federica Targhetta, Clara Pettinau, Martina Depau, Cristina Cogoni, Claudia Usai, Sara Pani, Mario Dessì, Laura Baronciani, Donatella Mediterr J Hematol Infect Dis Review Article The basis of allogeneic hemopoietic stem cell (HSC) transplantation in thalassemia consists in substituting the ineffective thalassemic erythropoiesis with and allogeneic effective one. This cellular replacement therapy is an efficient way to obtain a long lasting, probably permanent, clinical effective correction of the anaemia avoiding transfusion requirement and subsequent complications like iron overload. The first HSC transplant for thalassemia was performed in Seattle on Dec 2, 1981. In the early eighties transplantation procedure was limited to very few centres worldwide. Between 17 December 1981 and 31 January 2003, over 1000 consecutive patients, aged from 1 to 35 years, underwent transplantation in Pesaro. After the pioneering work by the Seattle and Pesaro groups, this therapeutic approach is now widely applied worldwide. Medical therapy of thalassemia is one of the most spectacular successes of the medical practice in the last decades. In recent years advances in knowledge of iron overload patho-physiopathology, improvement and diffusion of diagnostic capability together with the development of new effective and safe oral chelators promise to further increase success of medical therapy. Nevertheless situation is dramatically different in non-industrialized countries were the very large majority of patients live today. Transplantation technologies have improved substantially during the last years and transplantation outcome is likely to be much better today than in the ‘80s. Recent data indicated a probability of overall survival and thalassemia free survival of 97% and 89% for patients with no advanced disease and of 87% and 80% for patients with advanced disease. Thus the central role of HSC in thalassemia has now been fully established. HSC remains the only definitive curative therapy for thalassemia and other hemoblobinopathies. The development of oral chelators has not changed this position. However this has not settled the controversy on how this curative but potentially lethal treatment stands in front of medical therapy for adults and advanced disease patients. In sickle cell disease HSC transplantation currently is reserved almost exclusively for patients with clinical features that indicate a poor outcome or significant sickle-related morbidity. Università Cattolica del Sacro Cuore 2009-12-03 /pmc/articles/PMC3033161/ /pubmed/21415993 http://dx.doi.org/10.4084/MJHID.2009.015 Text en This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review Article
Angelucci, Emanuele
Pilo, Federica
Targhetta, Clara
Pettinau, Martina
Depau, Cristina
Cogoni, Claudia
Usai, Sara
Pani, Mario
Dessì, Laura
Baronciani, Donatella
Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders
title Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders
title_full Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders
title_fullStr Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders
title_full_unstemmed Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders
title_short Hematopietic Stem Cell Transplantation in Thalassemia and Related Disorders
title_sort hematopietic stem cell transplantation in thalassemia and related disorders
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3033161/
https://www.ncbi.nlm.nih.gov/pubmed/21415993
http://dx.doi.org/10.4084/MJHID.2009.015
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