Added Centimetres and Their Repercussions: How effective and safe is growth hormone in the treatment of short stature in girls with Turner syndrome and in children born small for gestational age?

The most common clinical characteristic of Turner syndrome (TS) is short stature. Although girls with TS are not growth hormone (GH) deficient, studies show that long-term GH treatment in TS leads to normalisation of height during childhood. In this chapter the results and conclusions are summarised of the multi-centre randomised dose-response growth hormone (GH) trial evaluating the efficacy, safety and psychosocial effect of long-term GH treatment on girls with TS. The TS trial was an open trial consisting of 68 untreated girls, aged between 2 and 11 years, with TS. The girls were randomly assigned to a group using 4 IU GH/m2/day, to a group using 4 IU GH/m2/day in the first year, and 6 IU GH/m2/day in the years thereafter, or to a group using 4 IU GH/m2/day in the first, 6 IU GH/m2/day in the second, and 8 IU GH/m2/day in the years thereafter (~ 0.045, 0.067, or 0.090 mg/kg/day). After at least 4 years of GH treatment, at a minimum age of 12 years, a low dose of micronised oestradiol was given to induce puberty.

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