Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review
Ataxia-Telangiectasia (A-T) is a rare autosomal recessive disorder, first reported in 1926, caused by a deficiency of ATM (Ataxia-Telangiectasia Mutated) protein. The disease is characterized by progressive cerebellar neurodegeneration, immunodeficiency, leukemia, and lymphoma cancer predisposition....
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doaj-5d6e1fb0ba2145879a82344a3f50f4842020-11-25T03:35:59ZengMDPI AGCancers2072-66942020-10-01123207320710.3390/cancers12113207Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic ReviewBruna Sabino Pinho de Oliveira0Sabrina Putti1Fabio Naro2Manuela Pellegrini3Department of Anatomical, Histological, Forensic Medicine and Orthopedic Sciences, Faculty of Pharmacy and Medicine, Sapienza University, Via Scarpa 16, 00161 Rome, ItalyInstitute of Biochemistry and Cell Biology, IBBC-CNR, Campus Adriano Buzzati Traverso, Via Ramarini 32, 00015 Monterotondo, Rome, ItalyDepartment of Anatomical, Histological, Forensic Medicine and Orthopedic Sciences, Faculty of Pharmacy and Medicine, Sapienza University, Via Scarpa 16, 00161 Rome, ItalyInstitute of Biochemistry and Cell Biology, IBBC-CNR, Campus Adriano Buzzati Traverso, Via Ramarini 32, 00015 Monterotondo, Rome, ItalyAtaxia-Telangiectasia (A-T) is a rare autosomal recessive disorder, first reported in 1926, caused by a deficiency of ATM (Ataxia-Telangiectasia Mutated) protein. The disease is characterized by progressive cerebellar neurodegeneration, immunodeficiency, leukemia, and lymphoma cancer predisposition. Immunoglobulin replacement, antioxidants, neuroprotective factors, growth, and anti-inflammatory hormones are commonly used for A-T treatment, but, to date, there is no known cure. Bone marrow transplantation (BMT) is a successful therapy for several forms of diseases and it is a valid approach for tumors, hemoglobinopathies, autoimmune diseases, inherited disorders of metabolism, and other pathologies. Some case reports of A-T patients have shown that BMT is becoming a good option, as a correct engraftment of healthy cells can restore some aspects of immunologic capacity. However, due to a high risk of mortality as a result of a clinical and cellular hypersensitivity to ionizing radiation and radiomimetic drugs, a specific non-myeloablative conditioning is required before BMT. Although BMT might be considered as one promising therapy for the treatment of immunological defects and cancer prevention in selected A-T patients, the therapy is currently not recommended or recognized and the eligibility of A-T patients for BMT is a point to deepen and deliberate.https://www.mdpi.com/2072-6694/12/11/3207ATMA-T patientsA-T treatmentsbone marrow transplantation |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Bruna Sabino Pinho de Oliveira Sabrina Putti Fabio Naro Manuela Pellegrini |
spellingShingle |
Bruna Sabino Pinho de Oliveira Sabrina Putti Fabio Naro Manuela Pellegrini Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review Cancers ATM A-T patients A-T treatments bone marrow transplantation |
author_facet |
Bruna Sabino Pinho de Oliveira Sabrina Putti Fabio Naro Manuela Pellegrini |
author_sort |
Bruna Sabino Pinho de Oliveira |
title |
Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review |
title_short |
Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review |
title_full |
Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review |
title_fullStr |
Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review |
title_full_unstemmed |
Bone Marrow Transplantation as Therapy for Ataxia-Telangiectasia: A Systematic Review |
title_sort |
bone marrow transplantation as therapy for ataxia-telangiectasia: a systematic review |
publisher |
MDPI AG |
series |
Cancers |
issn |
2072-6694 |
publishDate |
2020-10-01 |
description |
Ataxia-Telangiectasia (A-T) is a rare autosomal recessive disorder, first reported in 1926, caused by a deficiency of ATM (Ataxia-Telangiectasia Mutated) protein. The disease is characterized by progressive cerebellar neurodegeneration, immunodeficiency, leukemia, and lymphoma cancer predisposition. Immunoglobulin replacement, antioxidants, neuroprotective factors, growth, and anti-inflammatory hormones are commonly used for A-T treatment, but, to date, there is no known cure. Bone marrow transplantation (BMT) is a successful therapy for several forms of diseases and it is a valid approach for tumors, hemoglobinopathies, autoimmune diseases, inherited disorders of metabolism, and other pathologies. Some case reports of A-T patients have shown that BMT is becoming a good option, as a correct engraftment of healthy cells can restore some aspects of immunologic capacity. However, due to a high risk of mortality as a result of a clinical and cellular hypersensitivity to ionizing radiation and radiomimetic drugs, a specific non-myeloablative conditioning is required before BMT. Although BMT might be considered as one promising therapy for the treatment of immunological defects and cancer prevention in selected A-T patients, the therapy is currently not recommended or recognized and the eligibility of A-T patients for BMT is a point to deepen and deliberate. |
topic |
ATM A-T patients A-T treatments bone marrow transplantation |
url |
https://www.mdpi.com/2072-6694/12/11/3207 |
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