ORIGINAL ARTICLES  TRENDS IN MOLECULAR DIAGNOSIS AND THERAPY OF β-THALASSEMIA AND SICKLE CELL ANEMIA 

Minerva Biotecnologica 2003 June;15(2):99-105

Copyright © 2003 EDIZIONI MINERVA MEDICA

language: English

Bone marrow transplantation for β-thalassemia

Giardini C., Lucarelli G.

Hematology/Oncology Department and Bone Marrow Transplant Center, “San Salvatore” General Hospital, Pesaro, Italy

β-thalassemia is a common genetic disease worldwide. Supportive therapies such as chronic lifelong transfusions and iron chelation have significantly ameliorated clinical manifestations of this disease, but cannot eliminate disease and treatment related complications that result in end-organ damage. Allogeneic bone marrow transplantation (BMT) is the only cure for patients with hemoglobinopathies. Results of transplants have steadily improved over the last decades due to effective control of transplant related complications and development of new preparative regimens. Our understandings of mixed chimerism (MC) in patients with hemoglobinopathies provide a rational for the use of less intensive conditioning regimens and gene therapy in these disorders. This review summarizes the current status of BMT for thalassemia major.