Patient-Reported Outcomes in Multiple Sclerosis: An Overview

At a recent visit to her neurologist, Marion reviews her health diary, in which she has been tracking her fatigue levels throughout the day and when she has to visit the bathroom. The PRO diary also helps her remember details that she might not otherwise be able to recall at the time of her clinic visit. They review the diary entrees together to develop a shared understanding of what Marion has been experiencing and identify trends in the PRO data. They discuss symptom management and use the PRO information from the diary to help guide adjustments to her physical therapy routine and medication regimen.

Part of Marion’s “PRO package” includes the Center for Epidemiologic Studies Depression Scale (CES-D), a validated depression screening and symptom severity questionnaire that she completes every 3 months. Although she denies being depressed, she has noticed that her CES-D scores in recent months have been consistently increasing. This prompts a discussion about mental health in MS and a referral to work on depression with the MS mental health specialist. Marion and the mental health specialist use CES-D measures at baseline and during treatment to set a remission target and to track progress during treatment. Marion finds this helpful because she says it is hard for her to “wrap my hands around depression… it’s not something that there is a blood test or a MRI for.” Marion is encouraged by being able to see her CES-D scores change as her depression severity decreases, and this helps motivate her to keep engaged in treatment.

PROs and PROMs: General Applications

PROs are measures obtained directly from an individual without a priori interpretation by a clinician.^9,17 PROs capture individual perspectives on symptoms, capability, disability, and health-related quality of life.⁹ With increasing emphasis on patient-centered care,¹⁸ individual perspectives and preferences elicited using PROMs may be able to inform better quality of care and patient-centered disease treatment and management.^19-21

PROMs are standardized, validated questionnaires used to assess PROs and can be generic or condition-specific. Generic PROMs can be used in any patient population. The SF-36²² is a set of quality of life measures that assess perceived ability to complete physical tasks and routine activities, general health status, fatigue, social functioning, pain, and emotional and mental health.²³ Condition-specific PROMs can be used for particular patient populations and are helpful in identifying changes in health status for a specific disease, disability, or surgery. For example, the PDQ-39 assesses 8 dimensions of daily living, functioning, and well-being for people with Parkinson’s disease.²⁴

PROMs have been used in some MS clinical trials and research studies to determine the effectiveness of experimental treatments from the viewpoint of study participants.^9,25,26 PROs can also be utilized in clinical care to facilitate communication of needs and track health outcomes,²⁷ and can inform improvement in outcomes for health systems and populations. They can also be used to assess experience of care,²⁸ encouraging a focus on high-quality outcomes through PRO-connected reimbursement mechanisms,²⁹ and provide aggregate data to evaluate clinical practice, population health outcomes, and the effectiveness of public policies.²⁷