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

Questions 64

ATI RN

ATI RN Test Bank

Endocrine System Pediatric Questions

Question 1 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 2 of 5

A 7-month-old girl presented with recurrent chest infection, chocking during feeding, large protruded tongue, persistent head lag, and hepatomegaly. Chest X-ray showed cardiomegaly. All the following are beneficial in the treatment of this patient EXCEPT

Correct Answer: A

Rationale: Pompe disease benefits from ERT (E), ventilatory support (D), and transplantation (C). Exercise therapy (A) is ineffective due to muscle glycogen accumulation; high-protein diet (B) may help marginally.

Question 3 of 5

All the following should be restricted in hereditary fructose intolerance EXCEPT

Correct Answer: A

Rationale: HFI requires restricting fructose sources (sorbitol, juice, sugar, cereal, B-E). Milk (A), primarily lactose, is safe and not restricted.

Question 4 of 5

Which of the following mucopolysaccharidoses have normal intelligence?

Correct Answer: D

Rationale: Morquio disease (MPS IV) typically preserves intelligence, unlike Sly (A), Hurler (B), Hunter (C), and Sanfilippo (E), which involve cognitive decline.

Question 5 of 5

A definitive diagnosis of mucopolysaccharidoses disorder is established by

Correct Answer: B

Rationale: Enzyme assay confirms MPS by detecting specific enzyme deficiencies, more definitive than biopsy (A), imaging (C), marrow (D), or GAG screening (E).

Access More Questions!

ATI RN Basic


$89/ 30 days

ATI RN Premium


$150/ 90 days

Similar Questions