Treatment of growth hormone deficiency in very young children.

Growth hormone (GH) deficiency is a rare disease in very young children and a challenge to the physician in terms of clinical recognition, diagnosis and treatment. Here, we review the available information regarding substitution of GH and other pituitary hormones in this patient group. Our results confirm the severity of the clinical presentation and the rapid loss of height (measured in standard deviation scores) in hypopituitary patients that occurs early in life. GH therapy induces a rapid catch-up of growth and the frequency of injections appears to be a more important determinant than the size of the dose itself. Long-term results are available in only a small percentage of patients, yet they show a favourable auxological outcome, compared with target height or historical height data from patients with severe hypopituitarism. In conclusion, although tremendous progress has been made in providing for those children with early onset and severe hypopituitarism, there is still a long way to go before we can claim that we have completely normalized their condition. In particular, careful assessments of the psychological and neurodevelopmental outcomes will become necessary in order to evaluate the 'final results' of endocrine replacement therapies.