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Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia
BACKGROUND AND OBJECTIVES: Fanconi anemia (FA) is a rare genetic disorder caused by an impaired DNA repair mechanism which leads to an increased tendency toward malignancies and progressive bone marrow failure. The only curative management available for hematologic abnormalities in FA patients is he...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Università Cattolica del Sacro Cuore
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5111526/ https://www.ncbi.nlm.nih.gov/pubmed/27872734 http://dx.doi.org/10.4084/MJHID.2016.054 |
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author | Fumani, Hosein Kamranzadeh Zokaasadi, Mohammad Kasaeian, Amir Alimoghaddam, Kamran Mousavi, Asadollah Bahar, Babak Vaezi, Mohammad Ghavamzadeh, Ardeshir |
author_facet | Fumani, Hosein Kamranzadeh Zokaasadi, Mohammad Kasaeian, Amir Alimoghaddam, Kamran Mousavi, Asadollah Bahar, Babak Vaezi, Mohammad Ghavamzadeh, Ardeshir |
author_sort | Fumani, Hosein Kamranzadeh |
collection | PubMed |
description | BACKGROUND AND OBJECTIVES: Fanconi anemia (FA) is a rare genetic disorder caused by an impaired DNA repair mechanism which leads to an increased tendency toward malignancies and progressive bone marrow failure. The only curative management available for hematologic abnormalities in FA patients is hematopoietic stem cell transplantation (HSCT). This study aimed to report the results of HSCT in adult or adolescent FA patients. PATIENTS AND METHODS: Twenty FA patients with ages of 16 or more who underwent HSCT between 2002 and 2015 enrolled in this study. The stem cell source was peripheral blood, and all patients had a full human leukocyte antigen (HLA) matched donor, 19 patients had a sibling donor, and one had full matched other related. Indications for HSCT were severe bone marrow failure or dependence on blood products transfusion and failure of medical treatment to sustain peripheral blood elements at an acceptable level. RESULTS: Eleven patients were female and 9 male (55% and 45%). Mean age was 24.05 years. Mortality rate was 50% (n=10), and the leading cause of death was graft versus host disease (GVHD) which occurred in 5 patients (4 cases from acute GVHD and one from chronic GVHD). Survival analysis showed an overall 5-year survival of 53.63% (95% confidence interval: 29.53%–72.74%) and 13 year survival of 45.96 % (95% confidence interval: 22.08%–67.03%) among patients. CONCLUSION: HSCT is the only curative management for bone marrow failure in FA patients. But the high rate of mortality and morbidity in adolescent and adult patients makes it a challenging issue. |
format | Online Article Text |
id | pubmed-5111526 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2016 |
publisher | Università Cattolica del Sacro Cuore |
record_format | MEDLINE/PubMed |
spelling | pubmed-51115262016-11-21 Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia Fumani, Hosein Kamranzadeh Zokaasadi, Mohammad Kasaeian, Amir Alimoghaddam, Kamran Mousavi, Asadollah Bahar, Babak Vaezi, Mohammad Ghavamzadeh, Ardeshir Mediterr J Hematol Infect Dis Original Article BACKGROUND AND OBJECTIVES: Fanconi anemia (FA) is a rare genetic disorder caused by an impaired DNA repair mechanism which leads to an increased tendency toward malignancies and progressive bone marrow failure. The only curative management available for hematologic abnormalities in FA patients is hematopoietic stem cell transplantation (HSCT). This study aimed to report the results of HSCT in adult or adolescent FA patients. PATIENTS AND METHODS: Twenty FA patients with ages of 16 or more who underwent HSCT between 2002 and 2015 enrolled in this study. The stem cell source was peripheral blood, and all patients had a full human leukocyte antigen (HLA) matched donor, 19 patients had a sibling donor, and one had full matched other related. Indications for HSCT were severe bone marrow failure or dependence on blood products transfusion and failure of medical treatment to sustain peripheral blood elements at an acceptable level. RESULTS: Eleven patients were female and 9 male (55% and 45%). Mean age was 24.05 years. Mortality rate was 50% (n=10), and the leading cause of death was graft versus host disease (GVHD) which occurred in 5 patients (4 cases from acute GVHD and one from chronic GVHD). Survival analysis showed an overall 5-year survival of 53.63% (95% confidence interval: 29.53%–72.74%) and 13 year survival of 45.96 % (95% confidence interval: 22.08%–67.03%) among patients. CONCLUSION: HSCT is the only curative management for bone marrow failure in FA patients. But the high rate of mortality and morbidity in adolescent and adult patients makes it a challenging issue. Università Cattolica del Sacro Cuore 2016-11-01 /pmc/articles/PMC5111526/ /pubmed/27872734 http://dx.doi.org/10.4084/MJHID.2016.054 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 | Original Article Fumani, Hosein Kamranzadeh Zokaasadi, Mohammad Kasaeian, Amir Alimoghaddam, Kamran Mousavi, Asadollah Bahar, Babak Vaezi, Mohammad Ghavamzadeh, Ardeshir Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia |
title | Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia |
title_full | Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia |
title_fullStr | Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia |
title_full_unstemmed | Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia |
title_short | Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients with Fanconi Anemia |
title_sort | allogeneic hematopoietic stem cell transplantation for adult patients with fanconi anemia |
topic | Original Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5111526/ https://www.ncbi.nlm.nih.gov/pubmed/27872734 http://dx.doi.org/10.4084/MJHID.2016.054 |
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