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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

Many youths given growth hormone because of growth delay may actually have pubertal delay, and a spontaneous potential for growth.

A long term follow-up by French investigators has found that half of the patients treated for idiopathic isolated growth hormone deficiency stop treatment and still achieve adult heights similar to those of patients who completed their treatment.

A multi-center study led by Dr Jean-Claude Carel, professor of paediatrics, Groupe hospitalier Cochin-Saint Vincent de Paul and Faculté Cochin-Université Paris, investigated all 2,852 French children diagnosed as having isolated idiopathic growth hormone deficiency whose treatment started between 1987 and 1992 and ended before 1996.

The investigators point out: "Growth hormone has been used for four decades, initially as an extract and now in recombinant form, but we still know little about its long term effects on adult height. No long term controlled trial has been performed, and evaluation of the effect of growth hormone is based on comparisons with historical controls or on changes in height."

In the study, 2,165 of the patients (76 percent) reached their adult height. Patients who completed treatment gained 1.0 SD score of height in 3.6 years. Patients who stopped treatment early gained 0.6 SD score in 2.7 years while receiving treatment and a further 0.4 SD score after the end of treatment.

A small proportion of patients with severe growth hormone deficiency respond better to treatment than patients with less severe growth hormone deficiency.

The researchers suggest that long-term treatment with growth hormone has no clear cut benefits for a majority of patients treated for idiopathic isolated growth hormone deficiency. Most of them, in fact, have pubertal delay and a potential for spontaneous catch up.

A diagnosis of idiopathic isolated growth hormone deficiency should be restricted to patients with severely and permanently altered growth hormone secretion, the investigators add.

Because so many children are being treated with growth hormone by clinicians around the world, there is a need for long term controlled trials to evaluate the effects in patients who do not have growth hormone deficiency, the investigators conclude. " We should try to identify predictive markers for short stature in adults and focus intervention on patients at higher risk."
BMJ 2002;325:70.

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