A 7-year-old child with beta-thalassemia major on chronic transfusion therapy (every 3–4 weeks for 5 years) develops increasing skin pigmentation, growth failure, and elevated serum ferritin of 5500 ng/mL. Liver biopsy shows severe hepatic iron deposition. Which chelation strategy is MOST appropriate at this point?

• A Combined deferoxamine plus deferiprone intensification regimen for severe iron overload (ferritin >5000 ng/mL) ✓
• B Deferoxamine (IV/SC) monotherapy at the maximum tolerated dose
• C Deferasirox (oral) monotherapy, which is now preferred over deferoxamine for children above 2 years with severe iron overload
• D Therapeutic phlebotomy to rapidly reduce iron stores

Explanation

With severe iron overload (ferritin >5000 ng/mL) and evidence of end-organ damage (hepatic iron deposition, growth failure), combination chelation therapy using deferoxamine (DFO) plus deferiprone (DFP) has been shown to be superior to monotherapy in reducing body iron load and cardiac iron. The synergistic mechanism is that DFP is a small molecule that enters cells and shuttles iron to DFO, which then excretes it. For severe overload, combination therapy is recommended in current guidelines. Therapeutic phlebotomy is contraindicated in thalassemia major due to the underlying hemolytic anemia.

Reference: Ghai Essential Pediatrics, 10th ed.

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Written and medically reviewed by the StethoPrep medical team.