Congenital Adrenal Hyperplasia Clinical Trial
Official title:
An Adult Height Prediction Model for Congenital Adrenal Hyperplasia From a National Cohort (OPALE Model Study)
Congenital Adrenal Hyperplasia (CAH) is a genetic rare disease, which alters the adrenal
production of gluco and mineralo corticoïds. The treatment consists in supplementing children
with hydrocortisone. Despite care for these children has improved substantially across
decades, short adult height (AH) still remains an important consequence of the disease. About
20% of patients have an AH below 2 standard deviations compared to their expected AH.
In the OPALE-Model study, the investigators want to collect data from a cohort of 496 CAH
French patients, born between 1970 and 1991 with a known genotype. Using their age, sex,
growth, disease, bone maturation and pubertal data, the investigators will build a model
which allows to predict their AH using data available at 8 years of age. The growth charts
built from this cohort have shown that currently used formula to calculate the predicted AH
(Bayley-Pineau's formula) is not applicable to children with CAH.
In this project, the investigators plan to compute an AH prediction model using data from
children born between 1970 and 1993, and to validate the model using data from a different
cohort (i.e. children born between 1994 and 1998). this choice was due to availability of
data for computing the model first, and in a second stage, data from more recently born
patients.
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