Inborn errors of metabolism (IEM) are hereditary biochemical disorders caused by single-gene mutations that encode specific proteins. The following are the common characteristics of IEM EXCEPT

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Pediatric Endocrine Nursing Questions Questions

Question 1 of 5

Inborn errors of metabolism (IEM) are hereditary biochemical disorders caused by single-gene mutations that encode specific proteins. The following are the common characteristics of IEM EXCEPT

Correct Answer: B

Rationale: Most IEMs are autosomal recessive (A), require early diagnosis (C), present after birth (D), and vary in severity (E). However, later symptom onset often indicates milder disease, not greater severity, making B the exception.

Question 2 of 5

The MOST appropriate method to confirm the diagnosis of tyrosinemia type 1 is by elevated level of

Correct Answer: D

Rationale: Serum succinylacetone is the specific diagnostic marker for tyrosinemia type 1, reflecting fumarylacetoacetate hydrolase deficiency, more definitive than α-fetoprotein (A), tyrosine (B), methionine (C), or ALA (E).

Question 3 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 4 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 5 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.

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