A 10-day-old boy presented with lethargy, poor feeding, repeated vomiting, and recurrent hypoglycemic fits not responding to IV glucose. On examination, he was hypertonic with severe opisthotonos alternating with flaccidity. Of the following, the MOST likely diagnosis is

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Pediatric Endocrine Exam Questions Questions

Question 1 of 5

A 10-day-old boy presented with lethargy, poor feeding, repeated vomiting, and recurrent hypoglycemic fits not responding to IV glucose. On examination, he was hypertonic with severe opisthotonos alternating with flaccidity. Of the following, the MOST likely diagnosis is

Correct Answer: D

Rationale: Maple syrup urine disease (MSUD) presents neonatally with lethargy, vomiting, hypoglycemia, and neurological signs (opisthotonos, tone changes) from branched-chain amino acid buildup, unlike others (A-D).

Question 2 of 5

All the following are recognized feature of Niemann-pick disease type B EXCEPT

Correct Answer: C

Rationale: Niemann-Pick type B features normal IQ (A), lung disease (B), minimal neurology (D), and occasional cherry-red spots (E). Prolonged jaundice (C) is more typical of type A or other conditions.

Question 3 of 5

A 3-year-old girl presented with growth retardation, recurrent attacks of otitis media, diarrhea, and hypoglycemic seizures. On examination, there is hepatomegaly and her absolute neutrophil count (ANC) is 600/μL. Of the following, the MOST likely diagnosis is

Correct Answer: D

Rationale: GSD Ib causes hypoglycemia, hepatomegaly, and neutropenia (ANC 600) from impaired glucose-6-phosphate transport, unlike other neutropenia causes (A, B, C, E).

Question 4 of 5

Irreversible complication of untreated galactosemia is

Correct Answer: D

Rationale: Untreated galactosemia causes irreversible intellectual disability from brain damage, more permanent than cataracts (B), sepsis (A), or organ injuries (C, D), which may be treatable.

Question 5 of 5

Enzyme replacement is the main therapy of Hurler disease, it improve all the following EXCEPT

Correct Answer: D

Rationale: ERT in Hurler syndrome (MPS I) improves growth (A), mobility (B), organomegaly (C), and apnea (E), but cognitive impairment (D) persists due to CNS GAG accumulation.

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