The MOST common presentation of primary carnitine deficiency is

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Endocrine System Pediatrics Questions

Question 1 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).

Question 2 of 5

A 12-year-old female has a hypoglycemic seizure, weakness, and increased cutaneous pigmentation. She is also noted to have a chronic history of mucocutaneous candidiasis, which is especially severe on her nails. In addition, she has been on thyroid replacement medication since the age of 9 years. The most likely diagnosis is

Correct Answer: D

Rationale: Autoimmune polyendocrinopathy (e.g., APS-1) explains adrenal insufficiency (pigmentation, hypoglycemia), candidiasis, and hypothyroidism, a classic triad.

Question 3 of 5

McCune-Albright syndrome is associated with all of the following EXCEPT

Correct Answer: C

Rationale: Diabetes mellitus is not a feature of McCune-Albright syndrome, which involves endocrine overactivity and fibrous dysplasia.

Question 4 of 5

Holoprosencephaly is associated with all of the following EXCEPT

Correct Answer: D

Rationale: Holoprosencephaly is a developmental defect, not caused by congenital toxoplasmosis; others are midline anomalies.

Question 5 of 5

A 10-year-old presents with 1 year of polyuria and polydipsia. On voiding cystourethrogram, she is noted to have megacystis and hydroureter. Her most likely diagnosis is

Correct Answer: B

Rationale: Megacystis and hydroureter suggest obstructive uropathy causing polyuria, not central DI or psychogenic causes.

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