| | |  | | | | Record from Web of Science® | | | | | | |
| Stern cell transplantation for hemoglobinopathies |
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| Author(s): Gaziev J, Lucarelli G |
| Source: CURRENT OPINION IN PEDIATRICS Volume: 15 Issue: 1 Pages: 24-31 Published: FEB 2003 |
| Times Cited: 20 References: 44 |
| Abstract: Hereditary anemias caused by beta-thalassemia and sickle cell disease are the most common genetic diseases worldwide. Supportive therapies such as chronic lifelong transfusions, iron chelation for thalassemia, and transfusions or hydroxyurea for sickle cell anemia have significantly ameliorated clinical manifestations of these diseases but cannot eliminate disease and treatment-related complications that result in end-organ damage. Allogeneic hematopoietic stem cell transplantation is the only cure for patients with hemoglobinopathies. Results of transplants have steadily improved over the last few decades due to effective control of transplant-related complications and development of new preparative regimens. Our understandings of mixed chimerism in patients with hemoglobinopathies provide a rationale for the use of less intensive conditioning regimens and gene therapy in these disorders. Although the role of stem cell transplantation for thalassemia major is well defined, few transplants have been carried out in sickle cell disease, and, in light of recent advances, the role of stem cell transplantation in this disease should be revised. This review summarizes the current status of stem cell transplantation for hemoglobinopathies. Curr Opin Pediatr 2003, 15:24-31 (C) 2003 Lippincott Williams Wilkins, Inc. |
| Document Type: Review |
| Language: English |
| Reprint Address: Gaziev, J (reprint author), Osseo Muraglia, Unita Operat Ematol, Via Lombrosso, I-61100 Pesaro, Italy |
Addresses:
1. Azienda Osped San Salvatore, Dept Hematol Oncol, Pesaro, Italy
2. Azienda Osped San Salvatore, Bone Marrow Transplant Ctr, Pesaro, Italy |
| Publisher: LIPPINCOTT WILLIAMS & WILKINS, 530 WALNUT ST, PHILADELPHIA, PA 19106-3621 USA |
| Subject Category: Pediatrics |
| IDS Number: 636BL |
| ISSN: 1040-8703 |
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| | | | | | | | | Record from Web of Science® | | | | | | | | | |