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Patient-Reported Outcomes Gain Attention at ECTRIMS 2019


Sessions and posters from the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis discussed various ways to measure patient-reported outcomes in multiple sclerosis.

Patient-reported outcomes (PROs), said Paul Matthews, MD, DPhil, FRCP, of Imperial College, London, are “ultimately the filter through which all this biology and pharmacology acts.” His comment about the importance of hearing from those living with multiple sclerosis (MS) occurred at ECTRIMS 2019, the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis, which took place September 11-13, 2019, in Stockholm, Sweden.

Matthews’ presentation on PROs, which was part of a satellite symposium on novel measures monitoring MS that are becoming more important in clinical practice, was just one of many presentations, posters, and educational sessions during ECTRIMS 2019 that examined both the necessity, challenges, and results of incorporating this data into measurement of progress in treating MS.

PROs, Matthews said, are “better at capturing the voice of the person with MS,” especially as cognition assessments become more important as measures of disability progression. Oncology, he said, has offered a path for measuring “how patients feel and function,” as well as examples of how PROs directly relate to “hard” clinical outcomes.

He discussed the benefits and downsides of the Multiple Sclerosis Impact Scale (MSIS-29), which has 20 items related to physical status and 9 concerning psychological status. The score can be converted into a score of 0 to 100.

Digital tools provide opportunities for capturing PROs easily, with an iPhone or a Fitbit. Matthews pointed to examples such as the PatientsLikeMe website, which offers patients with dozens of conditions to share data online and connect with others like themselves.

But PROs are still a work in progress he said. Measures are still being constructed and validated. More work is being done to (1) address new populations or aspects of health, (2) improve clinical meaningfulness, (3) enhance convenience and patient compliance, and (4) improvement measurement properties.

Allessandra Solari, MD, of Fondazione IRCCS Istituto Neurologico C. Besta, Milan, Italy, said during an educational session that implementing PROs can bring certain challenges, including the administration burden and the concern on the part of patients that there is no obvious purpose to being asked questions. For physicians and researchers, the subjective nature of questions can be a barrier, along with the ability to have data at the point of care, and to interpret it. Finally, data security can be an issue.

But Solari was among the researchers at ECTRIMS 2019 working to validate PROs for use in clinical, epidemiological, and quality of care studies, and several were presented during the meeting.

Researchers from the United Kingdom’s Swansea University in Wales and Imperial College London presented a poster highlighting the power of a multivariate linear regression (MLR) model using the Expanded Disability Status Scale (EDSS), along with measures already collected by the UK MS Register. The poster was based on data gathered from 83 patients were given the MSIS-29 v2 and the Multiple Sclerosis Walking Scale.1

“The model results had good predictive power; the selected variables in the MLR account for 94% of EDSS variance,” the authors wrote.

Deborah M. Miller, PhD, LISW, is member of the professional staff at the Mellen Center for Multiple Sclerosis Treatment and Research of Cleveland Clinic and an associate professor Cleveland Clinic Lerner College of Medicine of Case Western Reserve University, offered an update about the Neuro-QoL (Quality of Life in Neurological Conditions), a group of PROs measures developed by the National Institutes of Health and available since 2010. It is now supported by HealthMeasures, Miller said. Neuro-QoL was developed for 5 adult and 2 pediatric neurological disorders, through an extensive process that gathered input from patients, caregivers, and clinicians.

The measure includes 12 unidimensional scales that have been calibrated using item response theory. Scores are reported on a T-score metric (mean = 50; SD = 10), which allows for an interpretable reference for scores. Miller said administration can be done in many forms, but the most efficient method is on a computer. This method can be translated into other languages, the computer method limits the administrative burden.

It has 3 main domains: physical, mental, and social health. Miller said 3 functions that have not been adequately calibrated for use in clinical practice in MS include bowel function, urinary/bladder function, and sexual function.

One of the mental health areas measures is stigma. “It’s remarkable to me how significant stigma is for people living with MS,” Miller said. “We had included this primarily for people living with epilepsy. But there is a high level that people report even with low-level disability with people with multiple sclerosis.”

In this realm, the end-of-life concerns measure has not been calibrated, she said. At present, the metric is being used in 2 actively-enrolling global MS comparative effectiveness studies that will report data in 2-3 years. Many smaller studies assessing Neuro-QoL are ongoing. It’s too soon to say if NIH’s Neuro-QoL will become the “gold standard.”

With choices available, Matthews said in his talk, “it’s hard to give a fixed recommendation,” for a single measure. “But it’s important for all neurologists to think about incorporating some PROs into their practice.

The best recommendation is to use the simplest measure “that you can count on the patient to use.”


  1. Rodgers W, Middleton R, Downing-Wood H, et al. Towards a clinically validated PRO measurement of disability in people with MS. Presented at 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis; Stockholm, Sweden; September 11-13, 2019. Abstract P451.
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