■洛马他被批准用于降低纯合子家族性高胆固醇血症的低密度脂蛋白胆固醇(LDL-C),这是一种罕见的遗传疾病。来自小型临床试验的关于其安全性和有效性的证据需要在现实世界中进一步验证有效性和安全性。本研究旨在使用有关lomitapide有效性和安全性的机构数据,以协助制定将其添加到处方集中的观点。
■这是对在阿卜杜勒阿齐兹国王医疗城积极服用洛米他必特的患者的回顾性研究,利雅得,沙特阿拉伯,从2019年到2022年。数据收集包括人口统计,确认的基因突变结果,lomitapide治疗的持续时间,基线,在治疗上,最后的LDL-C水平,治疗1-3个月后LDL-C降低百分比(以先可用者为准),使用其他降低LDL-C的疗法,肝功能检查,不利影响,和合规。
■8名成人患者被纳入审查,平均年龄25.5岁.大约75%是女性,lomitapide的治疗时间为9个月至3年。没有患者进行连续LDL单采。提交给我们的设施时的平均基线LDL-C为17.2mmol/L(范围,11.78-21.97mmol/L),lomitapide的LDL-C平均下降百分比为34.1%(范围,0%-87%),50%的患者出现胃肠道紊乱,未见严重肝毒性或肝酶升高的病例。
■在我们的成年患者队列中,lomitapide显示LDL-C总体适度降低,没有肝酶升高和不耐受的病例,表明大多数患者可能不合规。这篇评论揭示了为罕见疾病报销昂贵药物时的重要考虑因素。实时的现实证据可以支持医疗保健系统进行价格谈判并达成共同协议,最终可以改善患者获得护理的机会。
UNASSIGNED: Lomitapide is approved for lowering low-density lipoprotein cholesterol (LDL-C) in homozygous familial hypercholesterolemia, which is a rare genetic disorder. The evidence regarding its safety and efficacy from a small clinical trial requires further validation for effectiveness and safety in the real world. This study aimed to use institutional data on the effectiveness and safety of lomitapide to assist in formulating a perspective on adding it to the formulary.
UNASSIGNED: This was a retrospective review of patients who were actively prescribed lomitapide at King Abdulaziz Medical City, Riyadh, Saudi Arabia, from 2019 to 2022. Data collection included demographics, confirmed gene mutation results, duration of lomitapide therapy, baseline, on-treatment, last LDL-C levels, percent reduction in LDL-C after 1-3 months of therapy (whichever was first available), other LDL-C lowering therapies used, liver function tests, adverse effects, and compliance.
UNASSIGNED: Eight adult patients were included in the review, with a mean age of 25.5 years. Approximately 75% were female, and the duration of treatment with lomitapide ranged from 9 months to 3 years. None of the patients were on continuous LDL apheresis. The mean baseline LDL-C at presentation to our facility was 17.2 mmol/L (range, 11.78-21.97 mmol/L), the mean percent drop in LDL-C with lomitapide was 34.1% (range, 0%-87%), gastrointestinal disturbances were documented in 50% of the patients, and no cases of severe liver toxicities or increase in liver enzymes were seen.
UNASSIGNED: In our cohort of adult patients, lomitapide showed an overall modest reduction in LDL-C, with no cases of increase in liver enzymes and documented intolerance, indicating that most patients were likely noncompliant. This review revealed important considerations when reimbursing expensive medications for rare diseases. Real-world evidence in real-time can support healthcare systems in price negotiations and reaching mutual agreements that can eventually improve patient access to care.