Multiple endocrine abnormalities in a child with Blackfan-Diamond anemia and hemochromatosis. Significant improvement of growth velocity and predicted adult height following growth hormone treatment despite liver damage

We evaluated a short, prepubertal 13.9 year-old boy with Blackfan-Diamond a nemia and significant liver iron stores due to multiple blood transfusions and found him to have several endocrine abnormalities, including hypothyroi dism, hypoparathyroidism, primary and secondary hypogonadism and IGF-I insu fficiency. Growth velocity was poor despite treatment with levothyroxine, c alcitriol, calcium and aggressive therapy with chelating agents. After 25 m onths of treatment with rhGH his growth velocity, height for age and PAH in creased significantly, suggesting a degree of sensitivity to GH despite his liver damage.