Thalassemia Major: Transplantation or Transfusion and Chelation
| Autor: | Said Y Mohamed |
|---|---|
| Rok vydání: | 2017 |
| Předmět: |
congenital
hereditary and neonatal diseases and abnormalities medicine.medical_specialty Thalassemia Graft vs Host Disease Disease Iron Chelating Agents lcsh:RC254-282 03 medical and health sciences 0302 clinical medicine Unrelated Donor hemic and lymphatic diseases medicine Humans Blood Transfusion Chelation therapy Sibling Intensive care medicine lcsh:RC633-647.5 Donor selection business.industry beta-Thalassemia lcsh:Diseases of the blood and blood-forming organs Hematology General Medicine lcsh:Neoplasms. Tumors. Oncology. Including cancer and carcinogens Allografts medicine.disease Surgery Transplantation Oncology Hematologic disease 030220 oncology & carcinogenesis Cord Blood Stem Cell Transplantation business 030215 immunology |
| Zdroj: | Hematology/Oncology and Stem Cell Therapy, Vol 10, Iss 4, Pp 290-298 (2017) |
| ISSN: | 1658-3876 |
| DOI: | 10.1016/j.hemonc.2017.05.022 |
| Popis: | Thalassemia is the most common monogenic hematologic disease that affects millions in the world and kills thousands of patients every year.Without transfusion or transplantation, patients with thalassemia major are expected to die within months of diagnosis. However, long-term transfusion and chelation therapy is highly challenging for many developing countries where the disease is prevalent, representing a major and unsustainable health burden.Stem cell transplantation is the only cure for thalassemia. It has witnessed major developments that have made it less toxic, more successful, and feasible for a larger number of patients with diverse comorbidities and from a wider range of donors. Advances in human leukocyte antigen typing have greatly refined alternate donor selection with results of matched unrelated donors similar to matched sibling donors.Novel strategies such as haploidentical and cord blood transplantation have increased the possibility of patients with no healthy donor to get a better opportunity to survive and avoid chronic transfusion complications.Cost-effectively, transplantation should be considered the primary treatment of choice in the presence of a suitable related or unrelated donor and at centers with a satisfactory experience in the field of transplantation and particularly, in managing those with thalassemia. Despite some complications such as graft-versus-host disease and late conditioning effects, the overall improvement in the quality of life of thalassemia is difficult to deny.Unfortunately, the number of transplants for thalassemia represents only a minority of all transplants conducted globally and the essential requirement for transplants for thalassemia in limited-resources countries should mandate the transplant societies, including Worldwide Network for Blood and Marrow Transplantation, to collaborate to help initiate and support specialized transfusion and transplant programs for managing thalassemia. Keywords: Chelation, Hemoglobinopathies, Thalassemia, Transfusion, Transplantation |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|