Ruutiainen Juhani, Viita Anna-Mari, Hahl Jarmo, Sundell Jesse, Nissinen Helena
a a Finnish Neuro Society, Masku, Finland, and University of Turku , Turku , Finland.
b b Novartis Finland Oy , Espoo , Finland.
J Med Econ. 2016;19(1):21-33. doi: 10.3111/13696998.2015.1086362. Epub 2015 Sep 11.
Although multiple sclerosis (MS) is one of the most common causes of non-traumatic disability among young adults, no published data on its economic and health-related quality-of-life (HRQoL) burden is available from Finland. The DEFENSE study aimed to estimate the costs and HRQoL of patients with MS (PwMS) in Finland and explore how these variables are influenced by disease severity and relapses.
Overall, 553 PwMS registered with the Finnish Neuro Society, a national patient association in Finland, completed a self-administered questionnaire capturing information on demographics, disease characteristics and severity (Expanded Disease Severity Scale [EDSS]), relapses, resource consumption and HRQoL.
The PwMS had a mean EDSS score of 4.0. Overall, 44.1% had relapsing-remitting form of the disease (RRMS). The mean age was 53.8 years and 55.7% had retired prematurely due to MS. Disease-modifying therapies (DMTs) were used by 42.7% of the study population, and 21.5% across all disease types and severities had experienced relapses during the previous year. The mean total annual cost of MS was €46,994, which increased with advancing disease from €10,835 (EDSS score = 0) to €109,901 (EDSS score = 8-9). The mean utility was 0.644. HRQoL decreased with increasing disease severity. Relapses imposed an additional utility decrement among the PwMS with RRMS and EDSS ≤5 and had a trend-like effect on total costs.
The cross-sectional setting did not allow assessment of the significance of relapses in early MS or the use of DMTs on the prognosis of the disease.
The study confirms previous findings from other countries regarding a significant disease burden associated with MS and provides, for the first time, published numerical estimates from Finland. Treatments that slow disease progression and help PwMS retain employment for a longer duration have the highest potential to reduce the disease burden associated with MS.
尽管多发性硬化症（MS）是年轻成年人非创伤性残疾的最常见原因之一，但芬兰尚无关于其经济负担和健康相关生活质量（HRQoL）负担的公开数据。DEFENSE研究旨在评估芬兰MS患者（PwMS）的成本和HRQoL，并探讨这些变量如何受疾病严重程度和复发的影响。
总体而言，在芬兰全国患者协会芬兰神经学会注册的553名PwMS完成了一份自我管理问卷，该问卷收集了有关人口统计学、疾病特征和严重程度（扩展疾病严重程度量表[EDSS]）、复发、资源消耗和HRQoL的信息。
PwMS的平均EDSS评分为4.0。总体而言，44.1%患有复发缓解型疾病（RRMS）。平均年龄为53.8岁，55.7%因MS过早退休。42.7%的研究人群使用了疾病修正疗法（DMTs），所有疾病类型和严重程度的21.5%在前一年经历了复发。MS的年平均总成本为46,994欧元，随着疾病进展，从10,835欧元（EDSS评分=0）增加到109,901欧元（EDSS评分=8-9）。平均效用为0.644。HRQoL随着疾病严重程度的增加而下降。复发在RRMS且EDSS≤5的PwMS中导致额外的效用降低，并对总成本有类似趋势的影响。
横断面研究设计无法评估早期MS复发的意义或DMTs对疾病预后的影响。
该研究证实了其他国家先前关于MS相关重大疾病负担的研究结果，并首次提供了芬兰的公开数值估计。减缓疾病进展并帮助PwMS更长时间保持就业的治疗方法具有降低MS相关疾病负担的最大潜力。
