A 7-month-old white female presents with severe developmental delay and episodes of vomiting. Physical examination reveals blue eyes, light skin with an eczematoid rash, and hyperactive deep tendon reflexes. There is failure to thrive and microcephaly. The most likely diagnosis is

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Pediatric Endocrine Exam Questions Questions

Question 1 of 5

A 7-month-old white female presents with severe developmental delay and episodes of vomiting. Physical examination reveals blue eyes, light skin with an eczematoid rash, and hyperactive deep tendon reflexes. There is failure to thrive and microcephaly. The most likely diagnosis is

Correct Answer: D

Rationale: Phenylketonuria (PKU) causes developmental delay, microcephaly, rash, and light pigmentation if untreated.

Question 2 of 5

Matching: Giant granules in granulocytes

Correct Answer: A

Rationale: Chédiak-Higashi syndrome features giant granules in neutrophils.

Question 3 of 5

The plasma amino acids in the patient in the previous two questions reveal a marked elevation of leucine, isoleucine, and valine. The most likely diagnosis is

Correct Answer: A

Rationale: Maple syrup urine disease (MSUD) causes elevated branched-chain amino acids (leucine, isoleucine, valine).

Question 4 of 5

A 10-day-old manifests profound coma one day after an illness characterized by emesis, poor oral intake, and hypotonia. Laboratory findings reveal 4+ ketonuria, an arterial blood gas of 6.9, PCO2 19, PO2 95, an anion gap of 37, and an absolute neutrophil count of 400. The most likely diagnosis is

Correct Answer: C

Rationale: Methylmalonic acidemia causes severe acidosis, ketonuria, and neutropenia during metabolic crises.

Question 5 of 5

During acute metabolic decompensation, all of the following are appropriate therapy EXCEPT

Correct Answer: B

Rationale: Albumin infusion is not standard for urea cycle defect crises; others help remove ammonia or provide energy.

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