Growth and development in thalassaemia major patients with severe bone lesions due to desferrioxamine

Abstract

Nine transfusion-dependent beta-thalassaemia major patients (seven males and two females), aged 4-15 years, with growth retardation and severe rickets-like radiological lesions due to continuous subcutaneous chelation therapy with desferrioxamine (45-75 mg/kg body weight, 6-7 time/week), were seen in our centre during the last 8 years. Serum ferritin levels ranged from 976 to 4115 micrograms/l. There was a progressive decline in growth velocity in these patients 2-3 years before the appearance of rickets-like radiological lesions. All patients underwent surgery to correct genu valgum and/or slipped capital epiphyses. The final height was below the 3rd percentile in six patients (SDS: from -2.9 to -5.2). The short stature was mainly due to a disproportion between upper and lower segments. Six of the patients had an associated sensorineural hearing loss.

Conclusion: Our data emphasize the importance of an accurate surveillance of the toxic effects of desferrioxamine treatment and warn of the risk of overtreating patients with low iron overload and also suggest a possible individual idiosyncrasy to the adverse effects of chelation therapy.