Adrenal Hyperplasia, Congenital Clinical Trial
Official title:
Evaluation of the Adult Height Gain With Growth Hormone Treatment in Children With Congenital Adrenal Hyperplasia (CDAH), Using the OPALE Prediction Model
Congenital adrenal hyperplasia (CAH) is a genetic rare disease, which alters the adrenal
production of gluco and mineralo corticoids. The treatment consists in supplementing
children using hydrocortisone. Despite care for these children has improve substantially
across decades, short adult height still remains an important consequence of the disease.
About 20 % of patients have an AH below 2 standard deviations compared to their expected
height.
In the OPALE model study, the investigators have collected data from a cohort of 496 French
patients, born between 1970 and 1991 and with a known genotype. Using their age, sex,
growth, disease, bone maturation and pubertal data, they have built a model which allows to
predict their AH using data available at 8 years of age. This model has shown that the
currently used formula to calculate the predicted AH (Bayley Pineau's method) is not
applicable to children with CAH.
In this project, the investigators plan to use the prediction model to compare the AH in
patients who have received GH treatment to their predicted AH using the model.
The hypothesis is that GH improves the AH in such patients. Existing cohorts have shown
improved growth celerity, and growth expectation using the Bayley-Pineau formula), but this
has not been shown on the actual AH.
This study will allow to reinforce the investigators' hypothesis.
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