OBJECTIVE: To study the status of growth and development and the relationship between growth disorders and iron overload in children with betathalassemia major. METHODS: Fifty children with beta-thalassemia major and who received blood transfusion therapy regularly (age: 9 months-17 years) were enrolled. They were subjected to a thorough history taking, clinical examinations, and laboratory examinations, including complete blood count, alanine transferasa (ALT) and serum ferritin. The physical growth parameters, such as height and weight, were compared with the reference values of Chinese children. RESULTS: Twenty-four patients (48%) were of short stature with height under the 3th percentile. Among them, 15 cases presented with their height and weight both under the 3th percentile. Spontaneous sex development was seen in 7 cases out of 21 over 10-year-old patients. No sex development was found in 4 out of 8 patients who were over 14 years old. The patients with a height under the 10th percentile (n=31) had higher serum ferritin levels (8 239.2±5 865.5 mg/L vs 5 028.1±3 885.7 mg/L; P<0.05) and lower hemoglobin levels (68.2±12.3 g/L vs 79.7±14.5 g/L; P<0.05) as well as hepatomegaly when compared with those patients with a height over the 10th percentile (n=19). Serum ferritin levels in 20 patients with a weight under the 10th percentile were significantly higher than those in 30 patients with a height over the 10th percentile (9 165.5±6 042.5 mg/L vs 5 567.3±4 447.3 mg/L; P<0.05). CONCLUSIONS: Short stature, low weight and sex development delay are common in children with beta-thalassemia major. This may be related to iron overload.