Patient-Reported Outcomes in Multiple Sclerosis: An Overview

Patient-reported experience measures (PREMs) assess patient satisfaction and experience of health care.^30,31 CollaboRATE³² is a PREM that assesses the degree of shared decision-making occurring between patients and clinicians during clinical care. PREMs are currently used for assessing self-efficacy and in shared decision-making and health care improvement applications. PREMs have yet to be developed specifically for persons with MS.

PROMs in MS Care

Generic PROMs have shown that persons with MS are disproportionately burdened by poor quality of life.^33-35 Other generic PROMs, like the SF-36,³⁶ the Sickness Impact Profile,³⁷ and versions of the Health Utilities Index,³⁸ can be used to gather information on dysfunction and to determine quality and duration of life modified by MS-related dysfunction and disability. MS-specific PROMs are used to assess MS impairments, including pain, fatigue, cognition, sexual dysfunction, and depression.^12,39-42 PROMs have also been used in MS clinical trials, including the Multiple Sclerosis Impact Scale-29 (MSIS-29),^43,44 the Leeds MS QoL (LMSQoL),^45,46 the Functional Assessment of MS (FAMS),⁴⁷ the Hamburg Quality of Life Questionnaire in MS (HAQUAMS),⁴⁸ the MS Quality of Life-54 (MSQoL-54),⁴⁹ the MS International QoL (MUSIQoL),⁵⁰ and the Patient-Reported Indices for MS Activity Limitations Scale (PRIMUS).⁵¹

Condition-specific PROMs are more sensitive to changes in health status and functioning for persons with MS compared to generic PROMs. They are also more reliable during MS remission and relapse periods.^44,52 For example, the SF-36 has floor and ceiling effects in MS populations—a high proportion of persons with MS are scored at the maximum or minimum levels of the scale, limiting discriminant capability.²² As a result, a “combined approach” using both generic and MS-specific measures is often recommended.⁵³ Some MS PROMs (eg, MSQoL-54) include generic questions found in the SF-36 as well as additional MS-specific questions or scales.

The variety of PROMs available (see Table for a selected listing) introduces a significant challenge to using them—limited generalizability and difficulty comparing PROs across MS studies. Efforts to establish common PROMs have been undertaken to address this problem.⁵⁴ The National Institute of Neurologicical Disorders and Stroke (NINDS) sponsored the development of a neurological quality of life battery, the Neuro-QOL.⁵⁵ Neuro-QOL measures the physical, mental, and social effects of neurological conditions in adults and children with neurological disorders and has the capability to facilitate comparisons across different neurological conditions. Additionally, the Patient-Reported Outcomes Measure Information System (PROMIS) has been developed to assess physical, mental, and social effects of chronic disease. PROMIS has a hybrid design that includes generic and MS-specific measures (such as PROMIS FatigueMS).⁵⁶ PROMIS can be used to assess persons with MS as well as to compare the MS population with other populations with chronic illness.

PROMs have varying levels of reliability and validity. The Evaluating the Measure of Patient-Reported Outcomes⁵⁷ study evaluated the development process of MS PROMs,⁴³ and found that the MSIS-29 and LMSQoL had the highest overall reliability among the most common MS PROMs. However, both scored poorly on validity due to lack of patient involvement during development. This questions the overall capability of existing MS PROMs to accurately and consistently assess PROs in persons with MS.