UNASSIGNED: We conduct a decision-tree model, where we compare the clinical and economic outcomes of two novel Food and drug administration (FDA)-approved medications which are available in Qatar; l-glutamine and crizanlizumab over a time horizon of 1 year in a hypothetical cohort of adult sickle cell disease patients from a Qatar healthcare perspective. The main outcome is incremental cost per sickle cell disease-related acute pain crises averted. Model clinical parameters were derived from individual drug randomized trials, published literature, whereas cost parameters from Qatar healthcare payer system (2020-2021). A sensitivity analysis was carried out, and the study results were robust around model inputs. Costs were converted to 2020 US dollars.
UNASSIGNED: Study results showed that both treatment modalities\' costs were the main driver of this analysis, with an average annual cost of the treatments per patient being $189,014 for crizanlizumab (5 mg/kg), $143,798 for crizanlizumab (2.5 mg/kg), and $74,323 for l-glutamine. The probability of no first-time sickle cell disease-related vaso-occlusive crises averted was 0.001/year for glutamine, 0.26/year for crizanlizumab (5 mg/kg), and 0.34/year for crizanlizumab (2.5 mg/kg). Lower dose crizanlizumab (2.5 mg/kg) dominated the higher one (5 mg/kg). The incremental cost-effectiveness ratio of crizanlizumab (2.5 mg/kg), when compared to l-glutamine was $81,265 per sickle cell disease-related vaso-occlusive crises averted. When comparing crizanlizumab (5 mg/kg) and l-glutamine, crizanlizumab (5 mg/kg) showed higher efficacy, yet the crizanlizumab incremental cost-effectiveness ratio was at $459,620 than l-glutamine.
UNASSIGNED: Crizanlizumab (2.5 mg/kg) may be a cost-effective intervention, yet it is not the approved dose for preventing vaso-occlusive crises in adolescents and adults with sickle cell disease. Crizanlizumab (5 mg/kg) was more cost-effective than the approved l-glutamine per sickle cell disease vaso-occlusive crisis prevented. Of note, we primarily focused on modeling acute vaso-occlusive pain, which limited our ability to consider other key outcomes in sickle cell disease.
■我们进行决策树模型,我们比较了两种新的食品和药物管理局(FDA)批准的药物的临床和经济结果,这些药物在卡塔尔的医疗保健角度,在一个假设的成年镰状细胞病患者队列中,在1年的时间范围内使用l-谷氨酰胺和crizanlizumab。主要结果是避免了与镰状细胞疾病相关的急性疼痛危机的成本增加。模型临床参数来自个体药物随机试验,出版文献,而卡塔尔医疗付款人系统的成本参数(2020-2021年)。进行了敏感性分析,研究结果在模型输入周围是稳健的。成本转换为2020美元。
■研究结果表明,两种治疗方式的成本都是这项分析的主要驱动因素,每位患者的平均年治疗费用为$189,014(5mg/kg),$143,798的crizanlizumab(2.5mg/kg),还有74,323美元的L-谷氨酰胺.谷氨酰胺没有避免首次镰状细胞疾病相关的血管闭塞危机的概率为0.001/年,cizanlizumab为0.26/年(5mg/kg),和0.34/年的crizanlizumab(2.5mg/kg)。较低剂量的crizanlizumab(2.5mg/kg)占主导地位,较高的剂量(5mg/kg)。crizanlizumab(2.5mg/kg)的增量成本-效果比,与l-谷氨酰胺相比,每避免镰状细胞疾病相关的血管闭塞危机为81,265美元。当比较crizanlizumab(5mg/kg)和l-谷氨酰胺时,crizanlizumab(5mg/kg)显示出更高的疗效,然而,与l-谷氨酰胺相比,cizanlizumab增量成本-效果比为459,620美元.
■Crizanlizumab(2.5mg/kg)可能是一种具有成本效益的干预措施,然而,对于患有镰状细胞病的青少年和成人,它还不是预防血管闭塞危象的批准剂量.Crizanlizumab(5mg/kg)比批准的每镰状细胞疾病血管闭塞性危象预防的l-谷氨酰胺更具成本效益。值得注意的是,我们主要专注于模拟急性血管闭塞性疼痛,这限制了我们考虑镰状细胞疾病其他关键结果的能力。