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
R. Lanes et al., 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, J PED END M, 13(3), 2000, pp. 325-328
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.