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Question: Can hematopoietic stem cell transplantation (HSCT) cure pediatric transfusion-dependent thalassemia in Thailand? What influences its outcomes?
Finding: Among 266 HSCT procedures, 5-year overall survival and event-free survival rates were 91.3% and 81.0%, respectively. Outcomes were comparable between related and unrelated donors. Pre-HSCT ferritin >2,500 ng/mL, low CD34+ cell dose, and oral busulfan conditioning were associated with unfavorable survival. Longterm complications affected 22.7% of survivors.
Meaning: Optimizing pre-HSCT care, ensuring adequate grafting, and long-term surveillance are crucial. |