Long term results of growth hormone treatment in France in children of short stature: population, register based study

Objectives: To describe the growth of children treated with growth hormone and to evaluate the prognostic factors for height at the end of treatment. Design: Register based cohort study. Setting: French national register of all children treated with growth hormone. Subjects: 3233 short stature children (3165 of whom were deficient in growth hormone) who were treated with growth hormone (excluding children with Turner's syndrome) and whose treatment started between 1973 and 1989, last data being recorded in December 1993. Main outcome measures: Annual changes in height, and height at the end of treatment. Results: Mean height SD score at the end of treatment, after a mean of 4.3 years, was −2, corresponding to gain in mean height SD score of 1 and to a height SD score of 1.1 below target height. In all, 923 children prematurely stopped taking growth hormone treatment, mainly because of insufficient response (insufficient growth) or tiredness. Variables that predicted height at the end of treatment were age, target height, aetiology of short stature, use of puberty inhibitors, and type of growth hormone. Conclusions: The outcome of children of short stature with growth hormone deficiency who were treated with growth hormone has been less favourable than initially assumed. Growth hormone treatment has not restored normal growth to these children. The highly demanding nature and high costs of this treatment require an optimised prescription, and this remains to be determined. Key messages Few data are available on the long term results of growth hormone treatment in children of short stature Population based registers are ideal to provide information about the true use of treatments, characteristics of treated subjects and outcome as they are not distorted by selection criteria as in clinical trials or sponsored phase IV studies The analysis of the data from the French register up to 1993 leads to a less optimistic view of the efficacy of growth hormone treatment than that generally held Growth hormone treatment did not restore the genetic growth potential and thus should not be considered as a replacement treatment