Abstract
We reviewed the growth and endocrine status of 13 pts (8F, 5M) with TM on a hypertransfusion program (Hb>llg%), receiving Desferal (DF) > 6 days/wk since age 10.5 ± 0.9 yr (mean ± SEM) for a period of 5 ± 1.2 yr. With treatment, serum ferritin fell from a mean of 4900 to 2100ng/ml (p<.001) while SGOT resolved to normal in 11/13 pts. Despite these changes, growth failure and delayed sexual maturation was observed in 7/8 females and 4/5 males. Significant reduction in bone age (BA) was noted, in parallel with Tanner pubertal stage. At age 14 yr. growth velocity was 0.7 cm/yr. in F (BA 11.5 ± 1.5 yr.) and 1.8 cm/yr. in M (BA 11 ± 2.0 yr.), compared with 5.8 cm/yr and 6.5 cm/yr at age 6-8 yrs. In 6 pts studied (age 12-16 yrs.), T4 and TSH levels were normal, but sex steroids were low for chronological age. Failure of LH release after LH-RH in all pts. indicated hypothalamic dysfunction. Cortisol and GH responses to insulin-hypoglycemia, and the TSH response to TRH were normal. The growth pattern observed was similar to that in 10 pts in whom chelation was suboptimal judged by compliance and serum ferritin.
We conclude that DF, while achieving net negative iron balance, does not significantly influence growth/maturation in TM if begun after age 10 yrs. Such failure may be secondary to central hypogonadism, although abnormalities in somatomedin generation, perhaps secondary to liver damage, may also be important.
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Olivieri, N., Holland, F. & Freedman, M. THE EFFECT OF NIGHTLY SUBCUTANEOUS DESFERAL ON GROWTH AND SEXUAL MATURATION IN CHILDREN WITH THALASSEMIA MAJOR. Pediatr Res 18 (Suppl 4), 246 (1984). https://doi.org/10.1203/00006450-198404001-00918
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DOI: https://doi.org/10.1203/00006450-198404001-00918