Department of Pharmacy Practice, JSS College of Pharmacy, JSS Academy of Higher Education and Research, Mysuru, Karnataka, India.
Department of Paediatrics, JSS Medical College and Hospital, JSS Academy of Higher Education and Research, Mysuru, Karnataka, India.
Indian J Pharmacol. 2020 Nov-Dec;52(6):514-519. doi: 10.4103/ijp.IJP_333_19.
Chronic iron overload in beta-thalassemia patients after continuous blood transfusions has caused notable morbidity and mortality in these patients. The once-a-day oral iron chelator, deferasirox has established efficacy and bearable safety in adults and pediatric thalassemia patients. It is now extensively used for the management of transfusional hemosiderosis. However, a number of studies have revealed a few patients continued to be none respondent or intolerant toward the once-a-day regimen of deferasirox even after the administration of maximum dose recommended by the World Health Organization. In the literature, there were three studies showing the boon of twice in a day dosing of deferasirox among transfusional-dependent beta thalassemia patients. Therefore, a nonsystematic review was conducted on above three studies to ascertain the enhanced effectiveness and tolerability of twice per day regimen of deferasirox with the same total dose as that of once daily regimen of deferasirox in unresponsive or intolerant transfusion-dependent beta-thalassemia (TDT) patients. All the above studies concluded that the twice per day regimen of deferasirox was more efficacious and tolerable among TDT patients when compared to the once-a-day regimen with the same total daily dose. Although there was a significant good results from these studies, there is a need to conduct either muticenter study or randomized control study in a larger number of patients for the better confirmation of the results as all the above studies were conducted in the small number of TDT patients.
慢性铁过载在接受持续输血的β-地中海贫血患者中导致了这些患者显著的发病率和死亡率。每天一次的口服铁螯合剂地拉罗司在成人和儿科地中海贫血患者中已被证实具有疗效和可耐受的安全性。它现在被广泛用于输血性血色素沉着症的治疗。然而，一些研究表明，即使给予世界卫生组织推荐的最大剂量，仍有少数患者对地拉罗司每天一次的方案无反应或不耐受。在文献中，有三项研究表明，对于依赖输血的β地中海贫血患者，每天两次给予地拉罗司剂量会带来益处。因此，对上述三项研究进行了非系统性综述，以确定在无反应或不耐受的依赖输血的β-地中海贫血（TDT）患者中，每天两次给予地拉罗司与每天一次给予地拉罗司相同的总剂量的方案的增强疗效和耐受性。所有上述研究都得出结论，与每天一次的方案相比，每天两次给予地拉罗司在 TDT 患者中更有效且更耐受。尽管这些研究有显著的良好结果，但仍需要在更多的患者中进行多中心研究或随机对照研究，以更好地确认结果，因为所有这些研究都是在少数 TDT 患者中进行的。
