Nhận xét kết quả điều trị thải sắt phối hợp deferiprone và deferoxamine ở trẻ em thalassemia tại Bệnh viện Đa khoa Xanh Pôn

Abstract

Thalassemia is a common hereditary hemolytic anemia in the world as well as in Vietnam, requiring regular blood transfusions with consequences of severe iron overload and iron accumulation in vital organs such as heart and liver. The study was conducted at the Saint Paul General Hospital on 46 thalassemia pediatric patients with indications for iron chelation treatment participating in the study from March 1, 2023 to October 30, 2023. All patients received oral daily dose of 75 mg/kg Deferiprone at home in combination with monthly intravenous dose of 25 - 35 mg/kg for 5 days of Deferoxamine in the hospital. Pre and post therapeutic gradients of serum ferritin and LIC levels were used as indicators of chelation therapy. The objective of the study was to evaluate the treatment efficacy of iron chelation therapy in children with Thalassemia at Saint Paul General Hospital by combining daily oral Deferiprone and monthly IV Deferoxamine in 5 days, using liver iron concentration (LIC) and serum ferritin (SF) levels as indicators. The results of the study showed that: Post-treatment serum ferritin (SF) decreased by 10 - 20% compared to baseline ferritin levels (p < 0.05). In average, LIC significantly reduced of 1.21 mg/g dry liver weight (p < 0.05). In this preliminary study, the chelation therapy in children with severe iron overload using a combination of Deferiprone and Deferoxamine significantly reduces serum ferritin and liver iron concentration after 6 months of treatment, worthy to further investigate in an ongoing study with large size in order to confirm the efficacy of this therapeutic modality.