Evaluation of an Online Platform for Multiple Sclerosis Research: Patient Description, Validation of Severity Scale, and Exploration of BMI Effects on Disease Course
Evaluation of an Online Platform for Multiple Sclerosis Research: Patient Description, Validation of Severity Scale, and Exploration of BMI Effects on Disease Course
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DOI：
10.1371/journal.pone.0059707
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发表时间：
2013-03-20
期刊：
影响因子：
3.7
通讯作者：
De Jager, Philip L.
中科院分区：
文献类型：
--
作者：
Bove, Riley;Secor, Elizabeth;De Jager, Philip L.
目的：评估在线平台PatientsLikeMe的潜力。com（PLM），用于多发性硬化症（MS）的研究。身体质量指数（BMI）对MS疾病过程中的作用进行了调查，以说明该platform.Methods的效用：首先，我们比较了受试者从PLM和从区域MS中心的人口统计学特征。其次，我们验证了PLM的患者报告的结果测量（MS评定量表，MSRS）对标准的医生评定工具。结果：与4,039名MS中心患者相比，10,255名PLM成员更年轻，受教育程度更高，男性和白色较少。病程多为复发缓解型，症状发作较年轻，病程较短。由于样本量大，差异显著，但绝对值很小。121例MS中心患者的MSRS评分显示患者来源和医生来源的综合评分之间的一致性可接受（加权kappa = 0.46）。步行域显示患者和医生评分之间的最高加权Kappa（0.73）和相关性（rs = 0.86）。此外，患者报告的MSRS复合评分和步行评分与医生衍生的测量值之间存在良好的相关性：扩展残疾状态量表（复合评分= 0.61，步行评分= 0.74）、计时25英尺步行（复合评分= 0.70，步行评分= 0.69）和Ambassador指数（复合评分= 0.81，步行评分= 0.84）。最后，使用PLM数据，我们发现BMI和横截面MSRS（rho = 0.17）之间的适度相关性和BMI和疾病course.Conclusions之间没有关联：PLM人口是一个诊所人口，其患者报告的MSRS与现有的临床仪器。因此，该在线平台可以为MS研究提供独特优势（频繁的数据收集，大样本量）。为了说明其适用性，我们评估了BMI在MS病程中的作用，但没有发现BMI在这种情况下具有临床意义的作用。
Objectives: To assess the potential of an online platform, PatientsLikeMe. com (PLM), for research in multiple sclerosis (MS). An investigation of the role of body mass index (BMI) on MS disease course was conducted to illustrate the utility of the platform.Methods: First, we compared the demographic characteristics of subjects from PLM and from a regional MS center. Second, we validated PLM's patient-reported outcome measure (MS Rating Scale, MSRS) against standard physician-rated tools. Finally, we analyzed the relation of BMI to the MSRS measure.Results: Compared with 4,039 MS Center patients, the 10,255 PLM members were younger, more educated, and less often male and white. Disease course was more often relapsing remitting, with younger symptom onset and shorter disease duration. Differences were significant because of large sample sizes but small in absolute terms. MSRS scores for 121 MS Center patients revealed acceptable agreement between patient-derived and physician-derived composite scores (weighted kappa = 0.46). The Walking domain showed the highest weighted kappa (0.73) and correlation (rs = 0.86) between patient and physician scores. Additionally, there were good correlations between the patient-reported MSRS composite and walking scores and physician-derived measures: Expanded Disability Status Scale (composite rs = 0.61, walking rs = 0.74), Timed 25 Foot Walk (composite rs = 0.70, walking rs = 0.69), and Ambulation Index (composite rs = 0.81, walking rs = 0.84). Finally, using PLM data, we found a modest correlation between BMI and cross-sectional MSRS (rho = 0.17) and no association between BMI and disease course.Conclusions: The PLM population is comparable to a clinic population, and its patient-reported MSRS is correlated with existing clinical instruments. Thus, this online platform may provide a venue for MS investigations with unique strengths (frequent data collection, large sample sizes). To illustrate its applicability, we assessed the role of BMI in MS disease course but did not find a clinically meaningful role for BMI in this setting.