A 12-year-old boy presented with epistaxis, mild bruising, intermittent bone pain, and massive splenomegaly. Complete blood count shows thrombocytopenia and mild anemia. Radiological study revealed lytic lesions and Erlenmeyer flask deformity of the distal femur. Of the following, the MOST likely diagnosis is

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Endocrine System in Pediatrics Questions

Question 1 of 5

A 12-year-old boy presented with epistaxis, mild bruising, intermittent bone pain, and massive splenomegaly. Complete blood count shows thrombocytopenia and mild anemia. Radiological study revealed lytic lesions and Erlenmeyer flask deformity of the distal femur. Of the following, the MOST likely diagnosis is

Correct Answer: A

Rationale: Gaucher disease presents with splenomegaly, bone pain, cytopenias, and Erlenmeyer flask deformity from glucocerebroside accumulation, unlike others (B, C, D, E).

Question 2 of 5

A 9-month-old boy presented with persistent head lag, rapid breathing, difficult feeding, and hepatomegaly. Chest X- ray showed cardiomegaly. Of the following, the MOST appropriate confirmatory test is

Correct Answer: D

Rationale: Pompe disease (GSD II) with hypotonia, hepatomegaly, and cardiomegaly is confirmed by enzyme assay (acid α-glucosidase) in dried blood spots, more practical than biopsies (A, B) or nonspecific tests (C, D).

Question 3 of 5

All the following are common features between hereditary fructose intolerance and galactosemia EXCEPT

Correct Answer: D

Rationale: HFI and galactosemia share failure to thrive (A), renal (B) and hepatic (C) impairment, and reducing substances (D). Intellectual disability (E) is specific to untreated galactosemia, not HFI.

Question 4 of 5

All the following mucopolysaccharidoses are inherited as autosomal recessive EXCEPT

Correct Answer: C

Rationale: Hunter disease (MPS II) is X-linked, unlike autosomal recessive Hurler (A), Scheie (B), Morquio (D), and Sanfilippo (E).

Question 5 of 5

The characteristic radiographic finding of Hurler disease is

Correct Answer: D

Rationale: Hook-shaped L1 vertebra is a characteristic Hurler (MPS I) finding from GAG deposition, distinct from other features (A, B, C, E).

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