A 13-month-old infant is found comatose in bed after sleeping later than usual. On physical examination, the infant is afebrile and of normal size, and the liver is palpated 4 cm below the costal margin. The plasma glucose level is 15 mg/dL, the bicarbonate level is 20 mEq/L, BUN is 35 mg/dL, ammonia is 295 μmol/L, AST is 320 U/L, ALT is 425 U/L, and bilirubin is normal. Urinalysis is negative for glucose, ketones, protein, and reducing substances. Which of the following is the most likely diagnosis?

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Endocrine System Pediatrics Questions

Question 1 of 5

A 13-month-old infant is found comatose in bed after sleeping later than usual. On physical examination, the infant is afebrile and of normal size, and the liver is palpated 4 cm below the costal margin. The plasma glucose level is 15 mg/dL, the bicarbonate level is 20 mEq/L, BUN is 35 mg/dL, ammonia is 295 μmol/L, AST is 320 U/L, ALT is 425 U/L, and bilirubin is normal. Urinalysis is negative for glucose, ketones, protein, and reducing substances. Which of the following is the most likely diagnosis?

Correct Answer: B

Rationale: Type I glycogen storage disease (von Gierke) causes severe hypoglycemia, hepatomegaly, and elevated ammonia/liver enzymes due to glucose-6-phosphatase deficiency.

Question 2 of 5

Appropriate treatment of the child in the previous question before the onset of serious symptoms is helpful in preventing severe retardation. Overtreatment may result in

Correct Answer: D

Rationale: Over-restricting phenylalanine in PKU can lead to protein deficiency, causing anorexia, lethargy, and rash.

Question 3 of 5

In general, albinism is characterized by all of the following EXCEPT

Correct Answer: D

Rationale: Nail hypoplasia is not a feature of albinism; others relate to melanin deficiency.

Question 4 of 5

A 4-year-old blue-eyed white female manifests a malar flush, mild mental retardation, subluxation of the ocular lens, iridodonesis, and Marfanoid-like features (tall, thin, arachnodactyly). The most likely diagnosis is

Correct Answer: D

Rationale: Homocystinuria mimics Marfan syndrome with lens dislocation and retardation due to homocysteine accumulation.

Question 5 of 5

Matching: Isovaleric acidemia

Correct Answer: D

Rationale: Isovaleric acidemia can present with pancytopenia during crises due to bone marrow suppression.

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