Abstract Details

Clinical measures of outcome eg the EDSS are the gold standard in multiple sclerosis (MS) assessement. However, the EDSS requires a clinical examination and varies within patient, and between clinicians, centres and countries. PROs are self-reported measures that can be performed online, frequently and at low cost. It is not known how PROs compare to clinical measures and whether they have the potential to replace them. 

To assess self-reported patient reported outcome measures (PROs) as an equivalent and complementary alternative metric to the clinical Expanded Disability Status Scale (EDSS) for disability in neurology clinic, registries and clinical trials.

Participants had to have had at least 3 visits in the longitudinal cohort spaced no more than 270 days apart; the MS-SMART clinical trial [NCT01910259] collected scores every 48 weeks. A progression or improvement event was measures by a +/- 6 point change in the PRO normalised MS impact scale (MSIS)-29 Physical score; a +/- 1 point change in the web EDSS; and a worsening in the clinical EDSS score (if base score < 6.0 then +/- 1 else +/- 0.5).

234 patients from NHS clinics and1315 pwMS with self-reported PRO data; and 161 participants from the MS-SMART trial were included.  There was no significant difference in confirmed worsening (p=0.83) or confirmed improvement (p=0.21) between clinical EDSS scores and self-reported webEDSS. The MS-SMART participants were propensity score matched with the MSIS-29 PRO data and there was no difference (p=1) between time to a confirmed MSIS-29 worsening when self-reported or trial reported. 

There is no significant difference in the time to confirmed to time to worsening and improvement using PROS and clinical EDSS data. PROs have the potential to complement clinical data collection.