Abstract

Background: Thalassemia is highly prevalent in Vietnam with 2.000 new severe cases yearly. Bone marrow transplantation remains the only established long-term cure for severe thalassemia. This report describes the baseline characteristics of severe thalassemia and evaluates the feasibility and effectiveness of bone marrow transplantation therapy at Hue Central Hospital, Vietnam.

Methods: This is a prospective, interventional cohort study conducted at Hue Central Hospital, Vietnam for severe thalassemia who received allogeneic stem cell transplantation between September 2024 (start of program) and June 2025. Data were analyzed in SPSS v.18.0

Results: A total of 8 matched-related BMTs have been performed during this time. Now, all of whom are disease- and GVHD-free. The median age at BMT was 5.6 years old (2.5-9.0). The male and female accounted for 62.5% and 37.5% respectively. There were 5 cases with HbE/Beta-Thalassemia and 3 cases with Alpha-Thalassemia. The median ferritin level pre-transplant was 1078.5 ng/ml (236-2917). The median dose of nucleated cells was 9.55x10⁸ cells/kg (8.4-10x10⁸). The median platelet and neutrophile engraftment time was 19 days (16-24) and 21 days (13-21), respectively. There were two cases (25%) with skin aGVHD-grade I and two cases (25%) with hemorrhagic cystitis. The median chimerism after day 30 was 97.9%. The median Hb after transplant was 12.7 (10,6-14.7) g/dl. All patients are very healthy now without red blood cells transfusion.

Conclusions: Allogeneic stem cell transplantation is a good therapy to cure severe thalassemia with HLA-matched siblings. This therapy demonstrates effective and safe therapy.