Successful Treatment of Severe Thalassemia

Figure 1. A six-year-old girl underwent successful allogeneic bone marrow transplantation for severe β-thalassemia in 1985. Before transplantation, the serum ferritin concentration was 4044 μg per liter and the serum aspartate aminotransferase and alanine aminotransferase concentrations were respectively 6 and 13 times the upper level of the normal range. Liver biopsy showed severe iron overload (hepatic iron concentration, 24.74 mg per gram of liver, dry weight), chronic active hepatitis, and bridging fibrosis (Ishak histologic score, 3) (Panels A and B). Six years later, the patient was treated with 54 sequential phlebotomies to reduce iron overload. Ten years after transplantation, the . . .