A 9-year-old boy presented with tiredness on exertion; cardiac auscultation reveals a murmur in aortic area; echo study showed aortic valve disease. On examination he had mild coarse facial features, corneal clouding, joint stiffness, and mild dysostosis multiplex but normal intelligence and stature. Of the following, the MOST likely diagnosis is

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Endocrine System Pediatrics Questions

Question 1 of 5

A 9-year-old boy presented with tiredness on exertion; cardiac auscultation reveals a murmur in aortic area; echo study showed aortic valve disease. On examination he had mild coarse facial features, corneal clouding, joint stiffness, and mild dysostosis multiplex but normal intelligence and stature. Of the following, the MOST likely diagnosis is

Correct Answer: B

Rationale: Scheie disease (mild MPS I) fits with normal intelligence, stature, corneal clouding, and cardiac issues, unlike severe Hurler (A), X-linked Hunter (C), skeletal Morquio (D), or CNS Sanfilippo (E).

Question 2 of 5

All the following are clinical features of acute intermittent porphyria EXCEPT

Correct Answer: D

Rationale: AIP features neuropathy (A), tachycardia (B), abdominal pain (C), and bladder issues (D). Photosensitivity (E) is absent, typical of cutaneous porphyrias.

Question 3 of 5

The least well-defined long-term sequelae of severe prolonged hypoglycemia is

Correct Answer: B

Rationale: Personality affection (B) is less well-defined as a hypoglycemia sequela compared to cerebral palsy (A), cognitive impairment (C), seizures (D), or autonomic issues (E), which are better documented.

Question 4 of 5

In X-linked adrenoleukodystrophy (ALD), MRI shows characteristic cerebral white matter lesions mainly in

Correct Answer: D

Rationale: ALD’s white matter lesions are characteristically parietooccipital on MRI, reflecting disease progression, distinct from other regions (A, B, C, E).

Question 5 of 5

The MOST common presentation of primary carnitine deficiency is

Correct Answer: B

Rationale: Primary carnitine deficiency most commonly presents with cardiomyopathy from impaired fatty acid transport, more frequent than liver (A), skeletal (C), fibroelastosis (D), or hypoglycemia (E).

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