Pediatrics · Pediatric Endocrinology (Thyroid, CAH, Diabetes, Puberty Disorders)

A female neonate presents with ambiguous genitalia, hyperpigmentation, hyponatraemia (Na 126 mEq/L), hyperkalaemia (K 6.8 mEq/L), and low blood pressure on day 5 of life. Serum 17-hydroxyprogesterone is markedly elevated. Which enzyme deficiency is responsible for the MOST common form of congenital adrenal hyperplasia?

  • A 21-hydroxylase deficiency
  • B 11-beta-hydroxylase deficiency
  • C 3-beta-hydroxysteroid dehydrogenase deficiency
  • D 17-alpha-hydroxylase deficiency
Correct answer: A. 21-hydroxylase deficiency

Explanation

21-hydroxylase deficiency (CYP21A2 gene mutation) accounts for >95% of all congenital adrenal hyperplasia cases. It leads to impaired cortisol and aldosterone synthesis with accumulation of 17-hydroxyprogesterone, which is shunted into androgen pathways causing virilisation of female fetuses. The salt-wasting form (most severe) presents with hyponatraemia and hyperkalaemia due to aldosterone deficiency. 11-beta-hydroxylase deficiency causes the second most common form (5%) and is associated with hypertension (due to 11-deoxycorticosterone accumulation). 17-alpha-hydroxylase deficiency causes hypertension and absence of sex hormones.

Reference: Ghai Essential Pediatrics, 10th ed.

High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP

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