Medical Research Council Clinical Trials Research Articles

Effect of human growth hormone treatment for 1 to 7 years on growth of 100 children, with growth hormone deficiency, low birthweight, inherited smallness, Turner's syndrome, and other complaints.

(1) Human growth hormone (HGH) has been given for one whole year or longer to 100 patients, aged 1·5 to 19 years, participating in the Medical Research Council Clinical Trial of HGH. Each patient was measured 3-monthly for a control year before treatment, and the majority for a control year after the first treatment year. All measurements were made by one anthropometrist. Radiographic measurements of widths of bone, muscle, and fat in calf and upper arm were made. Methods and standards for assessing the significance of a given height acceleration are presented. (2) The characteristics at diagnosis are given of 35 patients with isolated GH deficiency or hyposomatotrophism (HS), 18 with craniopharyngiomas and other CNS lesions, 3 with multiple trophic hormone deficiency, 18 with low birthweight short stature, 4 with hereditary smallness and/or delay in growth, 4 with psychosocial short stature, 1 with high resting HGH and low somatomedin, 6 with Turner9s syndrome, and 11 with other diagnoses. (3) 29 of the 35 HS patients were boys and 13 had an abnormally small penis and ill-developed scrotum. Only 2 were sibs. Parents averaged 40th centile for height. 4 children developed growth-suppressing antibodies, and had to cease treatment. The mean standard deviation score (SDS) for height at diagnosis was -4·7, range -2·6 to -7·3. Bone age SDS averaged -3·2, range -0·8 to -5·7. Skinfold SDS averaged +0·91. Limb muscle width SDS averaged about -3·0. GH peak in insulin hypoglycaemia averaged 4·7 ± 0·7 μU/ml, range 1 to 13. (4) A category of partial growth hormone deficiency is defined as patients with GH peaks of 7-20 μU/ml inclusive and height velocity SDS in the year before treatment between -1 and -2. Total HS patients have GH peaks of 1 to 6 μU/ml inclusive and height velocity SDS of < -2. Partial HS patients are accelerated by HGH and should be treated; but their average acceleration is below that of total HS patients. (5) There was a highly significant relation (r = -0·64) between blood GH peak level and pretreatment height velocity in the HS patients. (6) The LBW patients were 10 boys and 7 girls; all the boys had normal genitalia. The average height SDS at diagnosis was -3·7; parents9 height centile averaged 50th, bone age SDS -1·8, skinfold SDS -0·9. GH peaks were all above 30