A 6-year-old boy with beta-thalassemia major has been on regular transfusions since infancy. He has an HLA-identical sibling donor. Which statement about curative treatment is correct?
- A Allogeneic stem cell transplant is contraindicated in patients under 5 years
- B Transplant outcome is best when performed before significant iron overload develops ✓
- C Myeloablative conditioning is not required due to the patient's young age
- D Gene therapy has replaced stem cell transplant as first-line curative therapy
Correct answer: B. Transplant outcome is best when performed before significant iron overload develops
Explanation
Allogeneic stem cell transplant offers the best chance of cure when performed early, before iron-related organ damage (hepatic, cardiac, endocrine) occurs. Younger age (<5 years) is a favorable prognostic factor, not a contraindication. Myeloablative conditioning is standard. Gene therapy is promising but not yet established as first-line curative therapy.
Reference: Williams Hematology, 10th ed.
High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP
Written and medically reviewed by the StethoPrep medical team.