Pediatrics · Pediatric Hematology and Oncology

A 5-year-old boy with known beta-thalassemia major on regular blood transfusions develops increasing serum ferritin (>2500 ng/mL). The first-line iron chelation agent for this child is:

  • A Desferrioxamine (deferoxamine) IV infusion
  • B Deferiprone oral tablets
  • C Phlebotomy every 2 weeks
  • D Deferasirox oral tablets
Correct answer: D. Deferasirox oral tablets

Explanation

Deferasirox (oral, once daily) is currently the first-line iron chelation agent for transfusion-dependent thalassemia in children due to its oral administration, convenient once-daily dosing, and proven efficacy in reducing liver iron concentration. Deferoxamine (desferrioxamine) was the original gold standard but requires subcutaneous or IV infusion over 8–12 hours nightly, limiting compliance. Deferiprone is used when deferasirox is inadequate or contraindicated; it has specific efficacy for cardiac iron. Phlebotomy is used in hemochromatosis, not thalassemia.

Reference: Ghai Essential Pediatrics, 10th ed.

High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP

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