Inborn errors of metabolism (IEM) are hereditary biochemical disorders caused by single-gene mutations that result in alteration of

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Endocrine System Pediatric Questions

Question 1 of 5

Inborn errors of metabolism (IEM) are hereditary biochemical disorders caused by single-gene mutations that result in alteration of

Correct Answer: D

Rationale: IEMs result from single-gene mutations altering enzyme function, typically affecting protein structure or production (e.g., enzymes), not chromosomes (A), mitochondria broadly (B), fats (C), or carbohydrates (E) as primary defects.

Question 2 of 5

A 15-month-old boy had recurrent hospital admissions because of fever, jaundice, subcutaneous bleeding, and hypoglycemic fits; on examination, there is hepatomegaly. In the last attack, the child developed severe legs pain associated with retraction of the neck and trunk. Of the following, the MOST likely diagnosis is

Correct Answer: D

Rationale: Tyrosinemia type 1 fits with recurrent fever, jaundice, bleeding, hypoglycemia, hepatomegaly, and neurological symptoms (pain, retraction) from tyrosine accumulation, unlike other conditions (A, B, C, E).

Question 3 of 5

A 6-year-old girl that looks tall and thin with light skin. On examination she has a peculiar malar flush, subluxation of the ocular lens, developmental delay, and severe hypertension. Of the following, the MOST likely cause of hypertension is

Correct Answer: C

Rationale: Homocystinuria’s hypertension often stems from thromboembolism due to homocysteine’s vascular effects, fitting this presentation, unlike atherosclerosis (A), hyperthyroidism (B), coarctation (D), or renal stenosis (E).

Question 4 of 5

A 9-year-old boy admitted to the hospital because of fracture of left femur due to a minor trauma. Past history revealed recurrent attacks of epistaxis and intermittent generalized bone pain. On examination, there is massive splenomegaly. Of the following, the MOST appropriate practical treatment of this disease is

Correct Answer: C

Rationale: Enzyme replacement therapy (ERT) is the practical, standard treatment for Gaucher disease, reducing splenomegaly and bone issues, more widely used than gene therapy (A), transplantation (B, E), or substrate reduction (D).

Question 5 of 5

A 2-year-old girl presented with rapid deep breathing, hypoglycemia, recurrent epistaxis, easy bruising, failure to thrive, fat cheek, and hepatomegaly. Of the following, the MOST likely cause of bleeding tendency is

Correct Answer: C

Rationale: In von Gierke disease, impaired hepatic function from glycogen accumulation reduces clotting factor synthesis, causing bleeding, more than vascular issues (A), platelet count (D), or function (E).

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