西班牙青少年特发性关节炎患者的实际医疗效果和成本

Jordi Antón, Estefania Moreno Ruzafa, Mireia Lopez Corbeto, R. Bou, J. Sánchez Manubens, Sonia Carriquí Arenas, Joan Calzada Hernández, Violetta Bittermann, Carolina Estepa Guillén, Juan Mosquera Angarita, Lucía Rodríguez Díez, E. Iglesias, Miguel Marti Masanet, B. López Montesinos, M. I. González Fernandez, Alfonso de Lossada, Carmen Peral, Mónica Valderrama, N. Llevat, M. Montoro Álvarez, Immaculada Calvo Penadés
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The greatest contribution to direct costs is medication, but other expenses contribute to the consumption of resources, negatively impacting healthcare cost and the economic conditions of affected families. Objective: To assess the direct healthcare, indirect resource utilization, and associated cost of moderate-to-severe JIA in children in routine clinical practice in Spain. Methods: Children were enrolled in this 24-month observational, multicentric, cross-sectional, retrospective study (N = 107) if they had been treated with biologic disease-modifying anti-rheumatic drugs (bDMARDs), had participated in a previous study (ITACA), and continued to be followed up at pediatric rheumatology units at 3 tertiary Spanish hospitals. Direct costs included medication, specialist and primary care visits, hospitalizations, emergency visits or consultations, surgeries, physiotherapy, and tests. Indirect costs included hospital travel expenses and loss of caregiver working hours. Unitary costs were obtained from official sources (€, 2020). Results: Overall, children had inactive disease/low disease activity according to JADAS-71 score and very low functional disability as measured by Childhood Health Assessment Questionnaire score. Up to 94.4% of children received treatment, mainly with bDMARDs as monotherapy (84.5%). Among anti-TNFα treatments, adalimumab (47.4%) and etanercept (40.2%) were used in similar proportions. Annual mean (SD) total JIA cost was €7516.40 (€5627.30). Average cost of pharmacological treatment was €3021.80 (€3956.20), mainly due to biologic therapy €2789.00 (€3399.80). Direct annual cost (excluding treatments) was €3654.60 (€3899.00). Indirect JIA cost per family was €747.20 (€1452.80). Conclusion: JIA causes significant costs to the Spanish healthcare system and affected families. 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引用次数: 0

摘要

背景:幼年特发性关节炎(JIA)是儿童中最常见的慢性风湿病。如果炎症得不到适当治疗,可能会导致关节损伤、长期残疾和成年后疾病活跃。及早发现并实施适当的治疗对于改善临床疗效至关重要。在西班牙,JIA 给患儿、其家庭和医疗系统造成的负担尚未得到充分评估。对直接成本影响最大的是药物治疗,但其他费用也会造成资源消耗,对医疗成本和患者家庭的经济状况产生负面影响。目标:评估西班牙常规临床实践中儿童中重度 JIA 的直接医疗保健、间接资源利用和相关成本。方法:对西班牙儿童中度至重度 JIA 进行 24 个月的观察:在这项为期 24 个月的观察性、多中心、横断面、回顾性研究(N=107)中,如果儿童接受过生物改良抗风湿药(bDMARDs)治疗,参加过之前的研究(ITACA),并继续在西班牙 3 家三级医院的儿科风湿病科接受随访,则将其纳入研究。直接费用包括药物、专科和初级保健就诊、住院、急诊或会诊、手术、理疗和检查。间接成本包括医院差旅费和护理人员的工时损失。单位成本来自官方资料(欧元,2020 年)。研究结果总体而言,根据 JADAS-71 评分,患儿的疾病不活跃/疾病活动度低,根据儿童健康评估问卷评分,患儿的功能障碍程度非常低。多达94.4%的儿童接受了治疗,主要是bDMARDs单药治疗(84.5%)。在抗肿瘤坏死因子α治疗中,阿达木单抗(47.4%)和依那西普(40.2%)的使用比例相似。JIA的年平均(标度)总费用为7516.40欧元(5627.30欧元)。药物治疗的平均费用为 3021.80 欧元(3956.20 欧元),主要是生物疗法的费用 2789.00 欧元(3399.80 欧元)。每年的直接费用(不包括治疗)为 3654.60 欧元(3899.00 欧元)。每个家庭的间接JIA费用为747.20欧元(1452.80欧元)。结论:JIA给西班牙医疗系统和受影响家庭带来了巨大的成本。公共开支的部分原因是生物治疗费用高昂,但生物治疗仍是一种有效的长期治疗方法,可维持非活动性疾病/低疾病活动状态、极低的功能性残疾评分和良好的生活质量。
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Real-World Health Care Outcomes and Costs Among Patients With Juvenile Idiopathic Arthritis in Spain
Background: Juvenile idiopathic arthritis (JIA) is the most frequent chronic rheumatic disease in children. If inflammation is not adequately treated, joint damage, long-term disability, and active disease during adulthood can occur. Identifying and implementing early and adequate therapy are critical for improving clinical outcomes. The burden of JIA on affected children, their families, and the healthcare system in Spain has not been adequately assessed. The greatest contribution to direct costs is medication, but other expenses contribute to the consumption of resources, negatively impacting healthcare cost and the economic conditions of affected families. Objective: To assess the direct healthcare, indirect resource utilization, and associated cost of moderate-to-severe JIA in children in routine clinical practice in Spain. Methods: Children were enrolled in this 24-month observational, multicentric, cross-sectional, retrospective study (N = 107) if they had been treated with biologic disease-modifying anti-rheumatic drugs (bDMARDs), had participated in a previous study (ITACA), and continued to be followed up at pediatric rheumatology units at 3 tertiary Spanish hospitals. Direct costs included medication, specialist and primary care visits, hospitalizations, emergency visits or consultations, surgeries, physiotherapy, and tests. Indirect costs included hospital travel expenses and loss of caregiver working hours. Unitary costs were obtained from official sources (€, 2020). Results: Overall, children had inactive disease/low disease activity according to JADAS-71 score and very low functional disability as measured by Childhood Health Assessment Questionnaire score. Up to 94.4% of children received treatment, mainly with bDMARDs as monotherapy (84.5%). Among anti-TNFα treatments, adalimumab (47.4%) and etanercept (40.2%) were used in similar proportions. Annual mean (SD) total JIA cost was €7516.40 (€5627.30). Average cost of pharmacological treatment was €3021.80 (€3956.20), mainly due to biologic therapy €2789.00 (€3399.80). Direct annual cost (excluding treatments) was €3654.60 (€3899.00). Indirect JIA cost per family was €747.20 (€1452.80). Conclusion: JIA causes significant costs to the Spanish healthcare system and affected families. Public costs are partly due to the high cost of biologic treatments, which nevertheless remain an effective long-term treatment, maintaining inactive disease/low disease activity state; a very low functional disability score; and a good quality of life.
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