Long-term chelation therapy in thalassaemia major: effect on liver iron concentration, liver histology, and clinical progress

Abstract

A prospective trial of continuous chelation therapy in children with homozygous thalassaemia on a high transfusion regimen was started in April 1966. The effect of treatment on iron concentration in the liver and on hepatic histology was examined in 49 biopsy specimens obtained from nine chelator-treated patients and nine control patients between April 1966 and April 1973.Chelation therapy was associated with a significant reduction in liver iron concentration, and by the end of the trial the values for the two groups showed no overlap. A similar difference was seen in the amount of hepatic fibrosis, which showed little or no change in the chelator-treated patients but was progressive in the controls.No major clinical differences were seen, probably because both groups included several older children who were heavily loaded with iron at the start of the trial. Among the younger patients, however, puberty was delayed in four of five controls but in only one of four chelator-treated children. Prepubertal growth rate was significantly greater in the chelator-treated patients than in the controls.