Patient-reported outcomes and survival in multiple sclerosis: A 10-year retrospective cohort study using the Multiple Sclerosis Impact Scale–29

Background There is increasing emphasis on using patient-reported outcomes (PROs) to complement traditional clinical outcomes in medical research, including in multiple sclerosis (MS). Research, particularly in oncology and heart failure, has shown that PROs can be prognostic of hard clinical endpoints such as survival time (time from study entry until death). However, unlike in oncology or cardiology, it is unknown whether PROs are associated with survival time in neurological diseases. The Multiple Sclerosis Impact Scale–29 (MSIS-29) is a PRO sensitive to short-term change in MS, with questions covering both physical and psychological quality of life. This study aimed to investigate whether MSIS-29 scores can be prognostic for survival time in MS, using a large observational cohort of people with MS. Methods and findings From 15 July 2004 onwards, MSIS-29 questionnaires were completed by people with MS registered with the MS Society Tissue Bank (n = 2,126, repeated 1 year later with n = 872 of the original respondents). By 2014, 264 participants (12.4%) had died. Higher baseline MSIS-29 physical (MSIS-29-PHYS) score was associated with reduced survival time (subgroup with highest scores versus subgroup with lowest scores: hazard ratio [HR] 5.7, 95% CI 3.1–10.5, p < 0.001). Higher baseline MSIS-29 psychological score was also associated with reduced survival time (subgroup with highest scores versus subgroup with lowest scores: HR 2.8, 95% CI 1.8–4.4, p < 0.001). In those with high baseline MSIS-29 scores, mortality risk was even greater if the MSIS-29 score worsened over 1 year (HR 2.3, 95% CI 1.2–4.4, p = 0.02). MSIS-29-PHYS scores were associated with survival time independent of age, sex, and patient-reported Expanded Disability Status Scale score in a Cox regression analysis (per 1-SD increase in MSIS-29-PHYS score: HR 1.8, 95% CI 1.1–2.9, p = 0.03). A limitation of the study is that this cohort had high baseline age and disability levels; the prognostic value of MSIS-29 for survival time at earlier disease stages requires further investigation. Conclusions This study reports that PROs can be prognostic for hard clinical outcomes in neurological disease, and supports PROs as a meaningful clinical outcome for use in research and clinical settings.

[1]  C. Gotay,et al.  Patient‐reported Outcomes in Cancer: A Review of Recent Research and Policy Initiatives , 2007, CA: a cancer journal for clinicians.

[2]  A Thompson,et al.  The Multiple Sclerosis Impact Scale (MSIS-29): a new patient-based outcome measure. , 2001, Brain : a journal of neurology.

[3]  J. Kurtzke.  Rating neurologic impairment in multiple sclerosis , 1983, Neurology.

[4]  A. John,et al.  The Physical and Psychological Impact of Multiple Sclerosis Using the MSIS-29 via the Web Portal of the UK MS Register , 2013, PloS one.

[5]  Galina Velikova,et al.  Measuring quality of life in routine oncology practice improves communication and patient well-being: a randomized controlled trial. , 2004, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[6]  Joanne Greenhalgh,et al.  Implementing patient-reported outcomes assessment in clinical practice: a review of the options and considerations , 2012, Quality of Life Research.

[7]  P. Dorman,et al.  Doctors and patients don't agree: cross sectional study of patients' and doctors' perceptions and assessments of disability in multiple sclerosis , 1997, BMJ.

[8]  M. Clanet,et al.  Observer disagreement in rating neurologic impairment in multiple sclerosis: facts and consequences. , 1991, European neurology.

[9]  C. Polman,et al.  Detecting clinically-relevant changes in progressive multiple sclerosis , 2015, Multiple sclerosis.

[10]  R. Reynolds,et al.  The neuropathological basis of clinical progression in multiple sclerosis , 2011, Acta Neuropathologica.

[11]  Amy P Abernethy,et al.  Review of electronic patient-reported outcomes systems used in cancer clinical care. , 2014, Journal of oncology practice.

[12]  C. Polman,et al.  Outcome measurement in multiple sclerosis: detection of clinically relevant improvement , 2010, Multiple sclerosis.

[13]  X. Montalban,et al.  Responsiveness and predictive value of EDSS and MSFC in primary progressive MS , 2008, Neurology.

[14]  M. Versavel,et al.  Can the Expanded Disability Status Scale be assessed by telephone? , 2003, Multiple sclerosis.

[15]  James A Hanley,et al.  Random measurement error and regression dilution bias , 2010, BMJ : British Medical Journal.

[16]  A. Thompson,et al.  How responsive is the Multiple Sclerosis Impact Scale (MSIS-29)? A comparison with some other self report scales , 2005, Journal of Neurology, Neurosurgery & Psychiatry.

[17]  N. Aaronson,et al.  Use of health‐related quality‐of‐life assessments in daily clinical oncology nursing practice , 2008, Cancer.

[18]  J. Hobart,et al.  Kurtzke scales revisited: the application of psychometric methods to clinical intuition. , 2000, Brain : a journal of neurology.

[19]  R. Fitzpatrick,et al.  Impact of patient-reported outcome measures on routine practice: a structured review. , 2006, Journal of evaluation in clinical practice.

[20]  E. Wolfel,et al.  Quality of life and prognosis in heart failure: results of the Beta-Blocker Evaluation of Survival Trial (BEST). , 2007, Journal of cardiac failure.

[21]  C. Polman,et al.  Multiple Sclerosis Impact Scale (MSIS-29): relation to established measures of impairment and disability , 2004, Multiple sclerosis.

[22]  N. Devlin,et al.  Getting the Most out of PROMs: Putting Health Outcomes at the Heart of NHS Decision-Making , 2010 .

[23]  Yinshan Zhao,et al.  Relative mortality and survival in multiple sclerosis: findings from British Columbia, Canada , 2011, Journal of Neurology, Neurosurgery & Psychiatry.

[24]  J. Noseworthy,et al.  Interrater variability with the Expanded Disability Status Scale (EDSS) and Functional Systems (FS) in a multiple sclerosis clinical trial , 1990, Neurology.

[25]  R. Lyons,et al.  Physical Disability, Anxiety and Depression in People with MS: An Internet-Based Survey via the UK MS Register , 2014, PloS one.

[26]  A Smith,et al.  Feasibility and compliance of automated measurement of quality of life in oncology practice. , 2003, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[27]  C. Gotay,et al.  The prognostic significance of patient-reported outcomes in cancer clinical trials. , 2008, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[28]  J. Greenhalgh.  The applications of PROs in clinical practice: what are they, do they work, and why? , 2009, Quality of Life Research.

[29]  L. McLeod,et al.  Interpreting patient-reported outcome results: US FDA guidance and emerging methods , 2011, Expert review of pharmacoeconomics & outcomes research.

[30]  B. Weinshenker,et al.  Natural history of multiple sclerosis. , 2005, Neurologic clinics.

[31]  D. Ontaneda,et al.  Clinical trials in progressive multiple sclerosis: lessons learned and future perspectives , 2015, The Lancet Neurology.

[32]  D. Howell,et al.  Patient-reported outcomes in routine cancer clinical practice: a scoping review of use, impact on health outcomes, and implementation factors. , 2015, Annals of oncology : official journal of the European Society for Medical Oncology.

[33]  Jeffrey A Cohen,et al.  Disability outcome measures in multiple sclerosis clinical trials: current status and future prospects , 2012, The Lancet Neurology.

[34]  H. Krumholz,et al.  Health status identifies heart failure outpatients at risk for hospitalization or death. , 2006, Journal of the American College of Cardiology.

[35]  Gabriel E. Soto,et al.  Identifying Heart Failure Patients at High Risk for Near-Term Cardiovascular Events With Serial Health Status Assessments , 2007, Circulation.

[36]  G. Stimson.  Obeying doctor's orders: a view from the other side. , 1974, Social science & medicine.

[37]  R. L. Coleman,et al.  Patient-reported outcomes as end points and outcome indicators in solid tumours , 2015, Nature Reviews Clinical Oncology.

[38]  P. Fayers,et al.  Quality of Life: The assessment, analysis and interpretation of patient-reported outcomes , 2007 .

[39]  M. Kenward,et al.  Multiple imputation for missing data in epidemiological and clinical research: potential and pitfalls , 2009, BMJ : British Medical Journal.

[40]  T. Pekmezović,et al.  Prognostic factors for survival in multiple sclerosis , 1999, Multiple sclerosis.

[41]  D. Goodin,et al.  A comparative analysis of Patient-Reported Expanded Disability Status Scale tools , 2015, Multiple sclerosis.

[42]  M Hutchinson,et al.  The multiple sclerosis impact scale (MSIS-29) is a reliable and sensitive measure , 2004, Journal of Neurology, Neurosurgery & Psychiatry.