PDF(Pubmed)
Abstract：
UNASSIGNED: Treatment options for preventing vaso-occlusive crises among sickle cell disease patients are on the rise, especially if hydroxyurea treatment has failed. This economic analysis is conducted to assess the comparative clinical effectiveness, safety, and acquisition cost of l-glutamine and crizanlizumab for older adolescents and adults (⩾16 years old) with sickle cell disease in Qatar, with an emphasis on treatment costs and acute pain crises.
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.
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.
摘要：
■预防镰状细胞病患者血管闭塞性危象的治疗方案正在增加，特别是如果羟基脲治疗失败。进行此经济分析是为了评估比较临床有效性，安全,1-谷氨酰胺和crizanlizumab的收购成本为年龄较大的青少年和成年人（16岁）在卡塔尔镰状细胞病，强调治疗成本和急性疼痛危机。
■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。
■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。
