Clinical and Experimental Pediatrics

Original Article

Hematology

Outcomes of hematopoietic stem cell transplantation for pediatric patients with transfusion-dependent thalassemia in Thailand

Kleebsabai Sanpakit, Kamala Laohverapanich, Bunchoo Pongtanakul, Nattee Narkbunnam, Chayamon Takpradit, Usanarat Anurathapan, Samart Pakakasama, Supanun Lauhasurayotin, Kanhatai Chiengthong, Thirachit Chotsampancharoen, Pornpun Sripornsawan, Piya Rujkijyanont, Duantida Songdej, Nongnuch Sirachainan, Suradej Hongeng

Clin Exp Pediatr. 2026;69(4):340-352. Published online March 13, 2026

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.

DOI: https://doi.org/10.3345/cep.2025.02173

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