http://bmj.com/cgi/content/abstract/325/7355/70
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which this article appears: Other
Endocrinology Pharmacology
and toxicology Drugs:
endocrine system Other
Pediatrics |
Jean-Claude Carel
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@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.
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What is already known on this topic 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 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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