BMJ 2002;325:70 ( 13 July )

Papers

Adult height after long term treatment with recombinant growth hormone for idiopathic isolated growth hormone deficiency: observational follow up study of the French population based registry

Jean-Claude Carel, professor of paediatrics, ^a Emmanuel Ecosse, statistician, ^b Marc Nicolino, associate professor of paediatrics, ^e Maïté Tauber, professor of paediatrics, ^f Juliane Leger, associate professor of paediatrics, ^c Sylvie Cabrol, associate professor of paediatrics, ^d Irène Bastié-Sigeac, clinical research associate, ^a Jean-Louis Chaussain, professor of paediatrics, ^a Joël Coste, associate professor of biostatistics. ^b

^a Groupe hospitalier Cochin-Saint Vincent de Paul and Faculté Cochin-Université Paris V, 75014 Paris, France Department of Paediatric Endocrinology and INSERM U561, ^b Department of Biostatistics, ^c Department of Paediatric Endocrinology, Hôpital Robert Debré, 75019 Paris, ^d Paediatric Endocrinology Unit, Hôpital Trousseau, 75571 Paris, ^e Department of Paediatric Endocrinology, Hôpital Debrousse, Lyon, 69322 France, ^f Department of Paediatric Endocrinology, Hôpital des Enfants, 31026 Toulouse, France

Correspondence to: J-C Carel carel{at}cochin.inserm.fr

Objective: To evaluate the efficacy of recombinant growth hormone for increasing adult height in children treated for idiopathic isolated growth hormone deficiency.
Design: Observational follow up study.
Setting: Population based registry.
Participants: All 2852 French children diagnosed as having isolated idiopathic growth hormone deficiency whose treatment started between 1987 and 1992 and ended before 1996.
Main outcome measures: Change in height between the start of treatment and adulthood; classification of patients according to whether treatment was completed as scheduled or stopped early.
Results: Adult height was obtained for 2165 (76%) patients. The mean dose of growth hormone at start of treatment was 0.42 IU/kg/week. Height gain was 1.1 (SD 0.9) standard deviation (SD) scores, resulting in an adult height of -1.6 (0.9) SD score (girls, 154 (5) cm; boys, 167 (6) cm). Patients who completed the treatment gained 1.0 (0.7) SD score of height in 3.6 (1.4) years. Patients with treatments stopped early gained 0.6 (0.6) SD score in 2.7 (1.4) years while receiving treatment and a further 0.4 (0.9) SD score after the end of treatment. Most of the variation in height gain was explained by regression towards the mean, patients' characteristics, and delay in starting puberty. Severe growth hormone deficiency was associated with better outcome. Each year of treatment was associated with a gain of 0.2 SD score(1.3 cm).
Conclusion: The effect of growth hormone is unclear in many patients treated for so called idiopathic isolated growth hormone deficiency. Most of the patients have pubertal delay and a spontaneous growth potential, which must be taken into account when measuring the effect and cost effectiveness of treatments. Growth hormone deficiency should be clearly distinguished from pubertal delay, and criteria should restrict the definition to patients with severely and permanently altered growth hormone secretion as our results support the use of growth hormone in such patients. Long term trials are required for most patients currently treated.

What is already known on this topic Large numbers of children are treated with recombinant growth hormone for so called idiopathic isolated growth hormone deficiency The effect on adult height is unclear because of a lack of controlled trials and analysis, and that subgroups, rather than entire populations, are analysed. What this study adds Half the patients treated for idiopathic isolated growth hormone deficiency stop treatment before reaching adult height and achieve adult heights similar to those of patients who complete their treatment Many patients diagnosed as having growth hormone deficiency actually have pubertal delay A small proportion of patients with severe growth hormone deficiency respond better to treatment than patients with less severe growth hormone deficiency

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