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Patients with beta-thalassemia major die prematurely of heart and liver disease secondary to iron overload. They are exposed to the risks of transfusion and iron-chelation therapy, have stunted growth and other endocrine problems, and generally have poor quality of life.
To determine whether hematopoietic stem cell transplantation (HSCT) provides long-term improvement in health-related quality of life, investigators surveyed 109 thalassemic patients who underwent HSCT from human leukocyte antigen (HLA)-identical siblings during the 1980s and 1990s in Sardinia and Italy (median age at HSCT, 12 years; median age at follow-up, 34). All patients received a myeloablative conditioning regimen with busulfan and cyclophosphamide; graft-versus-host d…