Congenital Adrenal Hyperplasia Clinical Trial
Official title:
Final Height in Patients With Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is the most common inherited disorder in the adrenal gland in children. Growth is usually affected in CAH patients either due to the disease itself or treatment consequences.
CAH comprises a group of autosomal recessive disorders caused by a deficiency of one of five
enzymes needed for the synthesis of cortisol leading to defect in cortisol synthesis with or
without aldosterone deficiency and an increase in the production of adrenocorticotropic
hormone through negative feedback.
The most common form is 21-hydroxylase deficiency (21OHD), which forms more than 90 % of the
cases.
In classic CAH, 75% of the patients have the salt wasting (SW) and 25% have the non
salt-wasting phenotype (NSW).There are no clinical signs at birth in male infants and in
female patients, CAH is suspected shortly after birth if there is genital ambiguity, ranging
from slight clitromegaly to complete masculinization with acceleration of growth and pubertal
development.
The non-classic (late onset) form of CAH is a less severe form of 21OHD, and is diagnosed
later in life.
Final height in early and late onset patients has been reported as diminished (Hauffa et al,
1997).This could be attributed to androgen excess or treatment with steroids. Androgen excess
can occur at any age leading to accelerated growth, early epiphyseal closure and compromised
final adult height.
Despite that all forms of CAH differ in their degree of enzymatic deficiency, they all
represent a therapeutic challenge to pediatric endocrinologists attempting to optimize
growth.
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