Division of Gastroenterohepatology, Department of Pediatrics, School of Medicine, Universitas Syiah Kuala - Dr. Zainoel Abidin Hospital, Banda Aceh, 23111, Indonesia.
Division of Hematology-Oncology, Department of Pediatrics, School of Medicine, Universitas Syiah Kuala - Dr. Zainoel Abidin Hospital, Banda Aceh, 32111, Indonesia.
F1000Res. 2023 Dec 19;12:154. doi: 10.12688/f1000research.128810.2. eCollection 2023.
Excess iron deriving from a chronic transfusion and dietary intake increases the risk for cardiac complications in β-thalassemia major patients. Deferiprone and deferasirox are commonly prescribed to thalassemic patients who are at risk of iron overload. This study aimed to compare the performance and toxicity of deferiprone and deferasirox in β-thalassemia major patients.
A cross-sectional observation was performed on 102 patients with β-thalassemia major. Serum ferritin along with total, indirect, and direct bilirubin levels were measured. Levels of liver enzymes, transaminase (ALT), and aspartate transaminase (AST), were also determined. Ferritin correlations with serum ALT, AST, and total bilirubin were constructed based on Spearman's rank correlation. Statistical differences based on the serum parameters were analyzed between deferiprone and deferasirox groups. The differences of iron chelators' effects between those receiving short-term (≤7 years) and long-term (>7 years) blood transfusion were also analyzed.
The averaged levels of bilirubin, ALT, AST, and ferritin were found to be high. Ferritin was positively correlated with ALT (r=0.508 and <0.001) and AST ((r=0.569; p<0.001). There was no statistical difference in ferritin levels between the deferiprone and deferasirox groups ( =0.776). However, higher total bilirubin and ALT were observed in the deferasirox group than in the deferiprone group ( =0.001 and 0.022, respectively). Total ( <0.001), indirect ( <0.001), and direct bilirubin levels ( =0.015) were significantly higher in patients with long-term transfusions than those receiving short-term transfusions. Higher ferritin was found with a statistical significance of =0.008 in the long-term transfusions group.
Ferritin is high in people with transfusion-dependent β-thalassemia major and positively correlated with ALT and AST. Deferasirox might pose a higher risk of developing hepatic injury as compared with deferiprone. Yet, no significant change of deferasirox efficacy (based on ferritin level) was found between those receiving short-term and long-term transfusions.
慢性输血和饮食摄入过多的铁会增加β-地中海贫血患者发生心脏并发症的风险。地拉罗司和去铁酮常用于有铁过载风险的地中海贫血患者。本研究旨在比较地拉罗司和去铁酮在β-地中海贫血患者中的疗效和毒性。
对 102 例β-地中海贫血患者进行横断面观察。检测血清铁蛋白及总胆红素、间接胆红素和直接胆红素水平。还测定了肝酶、丙氨酸转氨酶（ALT）和天门冬氨酸转氨酶（AST）水平。根据 Spearman 等级相关构建铁蛋白与血清 ALT、AST 和总胆红素的相关性。根据血清参数分析地拉罗司和去铁酮组之间的差异。还分析了接受短期（≤7 年）和长期（>7 年）输血的铁螯合剂效果的差异。
发现胆红素、ALT、AST 和铁蛋白的平均水平较高。铁蛋白与 ALT（r=0.508，p<0.001）和 AST（r=0.569；p<0.001）呈正相关。地拉罗司组和去铁酮组的铁蛋白水平无统计学差异（ =0.776）。然而，去铁酮组的总胆红素和 ALT 高于地拉罗司组（ =0.001 和 0.022）。长期输血患者的总胆红素（ <0.001）、间接胆红素（ <0.001）和直接胆红素（ =0.015）水平显著高于短期输血患者。长期输血组铁蛋白水平显著升高（ =0.008）。
依赖输血的β-地中海贫血患者铁蛋白水平较高，与 ALT 和 AST 呈正相关。与地拉罗司相比，去铁酮可能会增加肝损伤的风险。然而，在接受短期和长期输血的患者中，去铁酮的疗效（基于铁蛋白水平）没有明显变化。
