A 5-year-old mentally retarded girl presented with repeated convulsions and abnormal EEG finding. On examination, she had a light skin, small head, and exaggerated reflexes. Of the following, the MOST appropriate method to confirm the diagnosis is

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Pediatric Endocrine Nursing Questions Questions

Question 1 of 5

A 5-year-old mentally retarded girl presented with repeated convulsions and abnormal EEG finding. On examination, she had a light skin, small head, and exaggerated reflexes. Of the following, the MOST appropriate method to confirm the diagnosis is

Correct Answer: D

Rationale: Plasma phenylalanine concentration confirms PKU in this classic presentation (mental retardation, seizures, light skin, microcephaly), offering definitive diagnosis over screening (A, C) or secondary tests (D). Flow cytometry (B) is unrelated.

Question 2 of 5

The MOST appropriate treatment of homocystinuria is

Correct Answer: D

Rationale: Vitamin B6 (pyridoxine) is the primary treatment for homocystinuria, reducing homocysteine levels in responsive patients, more foundational than betaine (A), folic acid (B), vitamin C (C), or methionine restriction (E).

Question 3 of 5

A 2-year-old boy presented with failure to thrive, regression of developmental milestone, and difficult to contact with surroundings. On examination, there are hepatosplenomegaly and moderate lymphadenopathy. Of the following, the MOST likely diagnosis is

Correct Answer: D

Rationale: Niemann-Pick disease (type A/B) fits with hepatosplenomegaly, lymphadenopathy, and developmental regression, unlike Gaucher (A), Sandhoff (B), Tay-Sachs (C), or von Gierke (D).

Question 4 of 5

A 2-year-old boy presented with growth retardation, hepatomegaly, and recurrent attacks of hypoglycemia; biochemical study reveals elevated blood lactate, triglyceride, and uric acid levels. All the following are complications of this disease EXCEPT

Correct Answer: C

Rationale: Von Gierke disease (GSD I) leads to pancreatitis (A), renal failure (B), hepatic adenoma (D), and pulmonary hypertension (E) from metabolic derangements. Cardiomyopathy (C) is more typical of GSD II.

Question 5 of 5

A 28-day-old boy presented with prolonged neonatal jaundice, lethargy, poor feeding, and failure to regain birth weight. On examination there are hepatomegaly and cataract. Of the following, the MOST likely injured organs in this disease are

Correct Answer: A

Rationale: Galactosemia injures eyes (cataracts), liver (jaundice, hepatomegaly), and brain (lethargy) from galactose-1-phosphate toxicity, not kidney (B, C, D) or pancreas (E) prominently.

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