Disease progression among multiple sclerosis patients before and during a disease-modifying drug program: a longitudinal population-based evaluation
Disease progression among multiple sclerosis patients before and during a disease-modifying drug program: a longitudinal population-based evaluation
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DOI：
10.1177/1352458509350307
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发表时间：
2009-11-01
影响因子：
5.8
通讯作者：
Bhan, V.
中科院分区：
文献类型：
--
作者：
Veugelers, P. J.;Fisk, J. D.;Bhan, V.
随机对照试验已经证明了疾病调节药物（DMD）在复发缓解型多发性硬化症（MS）和继发性进展型MS叠加复发患者中的疗效。然而，这些对选定患者的简短研究主要集中在减少发作上，必须辅之以“现实世界”临床环境中的评估，以确定DMD项目在减缓疾病进展方面的有效性，并为卫生政策和项目决策提供信息。我们评估了在一个全面的公共资助的药物保险计划中管理DMD的有效性，该计划为符合特定资格标准的地理定义的MS患者人群提供DMD。分析了1980年至2004年期间在加拿大新斯科舍省一个区域MS诊所为整个人口服务的1752例MS患者（10，312次评估）的数据。使用生存方法，我们观察到在引入该计划后，疾病进展到特定扩展残疾状态量表终点的统计学显著减少。使用分层线性回归方法对符合治疗条件的患者进行的亚组分析也表明，接受首个DMD处方治疗的患者的疾病进展减缓。这些发现提供了支持DMD计划有效性的证据，可用于为此类计划的更广泛实施提供信息。
Randomized controlled trials have demonstrated the efficacy of disease-modifying drugs (DMDs) in persons with relapsing-remitting multiple sclerosis (MS) and secondary progressive MS with superimposed relapses. However, these brief studies of selected patients have focused mainly on reducing attacks and must be complemented by evaluations in 'real-world' clinical settings to establish the effectiveness of DMD programs in slowing disease progression and to inform health policy and program decision-making. We assessed the effectiveness of DMDs as administered in a comprehensive publicly funded drug insurance program that provides DMDs to a geographically defined population of MS patients who meet specific eligibility criteria. Data from 1752 MS patients (10,312 assessments) seen between 1980 and 2004 at a regional MS Clinic serving the entire population of Nova Scotia, Canada were analysed. Using survival methods we observed a statistically significant reduction in disease progression to specific Expanded Disability Status Scale endpoints following the introduction of this program. Subgroup analyses of patients eligible for treatment using hierarchical linear regression methods also suggested that disease progression was slowed in patients treated with the first DMD prescribed. These findings provide evidence supporting DMD program effectiveness that can be used to inform the broader implementation of such programs.