去铁斯若薄膜包衣片（ Jadenu ）和去铁斯若分散片（ Exjade ）对重型β地中海贫血患者疗效的比较：关于对满意度、便利性、心脏 / 肝脏 MRI T2*、血清铁蛋白水平及生化指标影响的初步报告
Comparison of the effects of deferasirox film-coated tablets (Jadenu) and deferasirox dispersible tablets (Exjade) in patients with beta thalassemia major: a preliminary report of the effects on the satisfaction, convenience, cardiac/liver MRI T2*, serum ferritin level, and biochemical profiles.
作者信息
Mobinikhaledi Mahya, Falahati Vahid, Tajerian Amin, Hashiani Amir Almasi, Ghaffari Kazem, Ghasemi Ali
机构信息
Faculty of Medicine, Arak University of Medical Sciences, Arak, Iran.
Department of Pediatrics, Faculty of Medicine, Arak University of Medical Sciences, Arak, Iran.
出版信息
Front Pharmacol. 2024 Nov 19;15:1438611. doi: 10.3389/fphar.2024.1438611. eCollection 2024.
BACKGROUND
Deferasirox (DFX) is a once-daily oral iron chelator with proven dose-dependent efficacy in patients with thalassemia major (TM). The reason for switching from DFX dispersible tablets (Exjade) to DFX film-coated tablets (Jadenu) was intolerance. Many patients also reported that deferasirox did not taste good. In this study, we compared the effect of Jadenu and Exjade on satisfaction, convenience, cardiac/liver MRI T2*, serum ferritin levels, and biochemical profiles in patients with thalassemia major.
METHOD
Sixty-two patients with thalassemia over 2 years of age, who had iron overload indicated by chelation therapy, were randomly divided into two groups. The first group (n = 32) is treated with Exjade, and the second group (n = 30) is treated with Jadenu. Laboratory investigations included alkaline phosphatase (ALK), alanine transferase (ALT), aspartate transferase (AST), and serum ferritin levels. Cardiac/liver MRI T2* levels and patient satisfaction and convenience, were assessed before and 1 year after starting therapy.
RESULTS
The study found that 53.3% of Jadenu patients were satisfied with the taste of the medication compared to only 12.5% of Exjade patients, which was statistically significant ( = 0.001). Additionally, 40% of Jadenu patients were satisfied with the ease of taking the medication compared to 28.1% of Exjade patients, and again, the difference was statistically significant ( = 0.047). A comparison of the cardiac MRI T2* levels between the two studied groups showed no significant difference ( = 0.851).
CONCLUSION
Jadenu offers patients an improved formulation that can be taken on an empty stomach, has a better taste, and presents fewer gastrointestinal tolerability concerns. Overall, patient satisfaction is higher with Jadenu, which may improve adherence and reduce the frequency and severity of complications associated with iron overload. This, in turn, may help mitigate cardiovascular and hepatic complications from iron overload in the long term.
CLINICAL TRIAL REGISTRATION
https://irct.behdasht.gov.ir/search/result?query=IRCT20210830052346N1.
背景
地拉罗司（DFX）是一种每日服用一次的口服铁螯合剂，在重型地中海贫血（TM）患者中具有已证实的剂量依赖性疗效。从DFX分散片（Exjade）转换为DFX薄膜包衣片（Jadenu）的原因是不耐受。许多患者还报告说地拉罗司味道不好。在本研究中，我们比较了Jadenu和Exjade对重型地中海贫血患者满意度、便利性、心脏/肝脏MRI T2*、血清铁蛋白水平和生化指标的影响。
方法
62名2岁以上接受螯合治疗提示铁过载的地中海贫血患者被随机分为两组。第一组（n = 32）接受Exjade治疗，第二组（n = 30）接受Jadenu治疗。实验室检查包括碱性磷酸酶（ALK）、丙氨酸转氨酶（ALT）、天冬氨酸转氨酶（AST）和血清铁蛋白水平。在开始治疗前和治疗1年后评估心脏/肝脏MRI T2*水平以及患者满意度和便利性。
结果
研究发现，53.3%的Jadenu患者对药物味道满意，而Exjade患者中只有12.5%满意，差异具有统计学意义（P = 0.001）。此外，40%的Jadenu患者对服药便利性满意，而Exjade患者为28.1%，差异同样具有统计学意义（P = 0.047）。两个研究组之间心脏MRI T2*水平的比较显示无显著差异（P = 0.851）。
结论
Jadenu为患者提供了一种改进的制剂，可空腹服用，味道更好，胃肠道耐受性问题更少。总体而言，Jadenu患者满意度更高，这可能会提高依从性并减少与铁过载相关并发症的频率和严重程度。反过来，这可能有助于长期减轻铁过载引起的心血管和肝脏并发症。
临床试验注册
https://irct.behdasht.gov.ir/search/result?query=IRCT20210830052346N1 。
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