Deborah Miller

Implementing patient-reported outcomes assessment in clinical practice: a review of the options and considerations

PurposeWhile clinical care is frequently directed at making patients “feel better,” patients’ reports on their functioning and well-being (patient-reported outcomes [PROs]) are rarely collected in routine clinical practice. The International Society for Quality of Life Research (ISOQOL) has developed a User’s Guide for Implementing Patient-Reported Outcomes Assessment in Clinical Practice. This paper summarizes the key issues from the User’s Guide.MethodsUsing the literature, an ISOQOL team outlined considerations for using PROs in clinical practice; options for designing the intervention; and strengths, weaknesses, and resource requirements associated with each option.ResultsImplementing routine PRO assessment involves a number of methodological and practical decisions, including (1) identifying the goals for collecting PROs in clinical practice, (2) selecting the patients, setting, and timing of assessments, (3) determining which questionnaire(s) to use, (4) choosing a mode for administering and scoring the questionnaire, (5) designing processes for reporting results, (6) identifying aids to facilitate score interpretation, (7) developing strategies for responding to issues identified by the questionnaires, and (8) evaluating the impact of the PRO intervention on the practice.ConclusionsIntegrating PROs in clinical practice has the potential to enhance patient-centered care. The online version of the User’s Guide will be updated periodically.

Recent developments in the assessment of quality of life in Multiple Sclerosis (MS)

Multiple sclerosis (MS) and its treatment have broad-ranging effects on quality of life. This article reviews recent efforts to assess the impact of MS on activities of daily living (ADLs) and health-related quality of life (HRQL), and describes the development of the Multiple Sclerosis Quality of Life Inventory (MSQLI). The MSQLI is a modular MS-specific HRQL instrument consisting of a widely-used generic measure, the Health Status Questionnaire (SF-36), supplemented by nine symptom-specific measures (covering fatigue, pain, bladder function, bowel function, emotional status, perceived cognitive function, visual function, sexual satisfaction, and social relationships). Content validation consisted of evaluating its adherence to a conceptual model of the impact of MS, and review by MS specialists (neurologists and allied health professionals), HRQL experts, patients, and caregivers. The reliability and construct validity of the MSQLI were rigorously evaluated in a field test with 300 North American patients (198 female, 102 male) with definite MS (Poser criteria) and a broad range of physical impairment (EDSS=0.0-8.5). This article concludes by comparing the MSQLI with two other MS-specific HRQL measures (MS Quality of Life-54 (QOL-54) and Functional Assessment of Multiple Sclerosis (FAMS)) and discussing key issues to consider in selecting an HRQL instrument for a collaborative database.

The Goldman Consensus statement on depression in multiple sclerosis

Background. In January 2002 the New York City Chapter of the National Multiple Sclerosis Society convened a panel of experts to review the issue of depressive affective disorders associated with multiple sclerosis (MS). This Consensus Conference was supported by a grant from the Goldman family of New York City. Results. The panel reviewed summaries of current epidemiologic, neurobiologic, and therapeutic studies having to do with depressive disorders among MS patient populations. Depressive disorders occur at high rates among patients with MS, and there is reason to believe that the immunopathology of the disease is involved in the clinical expression of affective disorders. The depressive syndromes of MS have a major, negative impact on quality of life for MS sufferers, but are treatable. At the present time, most MS patients with depression do not receive adequate recognition and treatment. Conclusions. The Goldman Consensus Conference Study Group provides recommendations for improved screening, diagnosis, and clinical management for depressive affective disorders among patients suffering from MS.

Health-related quality of life in multiple sclerosis: Effects of natalizumab

To report the relationship between disease activity and health‐related quality of life (HRQoL) in relapsing multiple sclerosis, and the impact of natalizumab.

Neuro-QOL Brief measures of health-related quality of life for clinical research in neurology

Objective: To address the need for brief, reliable, valid, and standardized quality of life (QOL) assessment applicable across neurologic conditions. Methods: Drawing from larger calibrated item banks, we developed short measures (8–9 items each) of 13 different QOL domains across physical, mental, and social health and evaluated their validity and reliability. Three samples were utilized during short form development: general population (Internet-based, n = 2,113); clinical panel (Internet-based, n = 553); and clinical outpatient (clinic-based, n = 581). All short forms are expressed as T scores with a mean of 50 and SD of 10. Results: Internal consistency (Cronbach α) of the 13 short forms ranged from 0.85 to 0.97. Correlations between short form and full-length item bank scores ranged from 0.88 to 0.99 (0.82–0.96 after removing common items from banks). Online respondents were asked whether they had any of 19 different chronic health conditions, and whether or not those reported conditions interfered with ability to function normally. All short forms, across physical, mental, and social health, were able to separate people who reported no health condition from those who reported 1–2 or 3 or more. In addition, scores on all 13 domains were worse for people who acknowledged being limited by the health conditions they reported, compared to those who reported conditions but were not limited by them. Conclusion: These 13 brief measures of self-reported QOL are reliable and show preliminary evidence of concurrent validity inasmuch as they differentiate people based upon number of reported health conditions and whether those reported conditions impede normal function.

A meta-analysis of methylprednisolone in recovery from multiple sclerosis exacerbations.

Despite recent advances in multiple sclerosis treatment, patients experience relapses for which standard treatment remains glucocorticosteroids (GCS). However, there is limited information comparing doses or routes of administration for different GCS types or the benefit of GCS compared to natural recovery. Currently, high dose (HD) methylprednisolone (MP) is the preferred therapy. We conducted meta-analyses of published studies assessing MP at different doses and in comparison to other steroid products or no treatment. Relevant studies were identified through predetermined processes and five articles met the inclusion criteria. Three studies compared HD MP to placebo; two studies compared the effect of HD MP and low dose (LD) MP; only one accepted report compared HD MP to another GCS. This report could not be included in a meta-analysis. The meta-analysis of HD MP vs placebo studies indicated a mean treatment difference of 0.76 in Expanded Disability Status Score (EDSS) changes from baseline. The meta-analysis of HD and LD MP demonstrated no difference in EDSS change. Despite these rather obvious findings, these meta-analyses have been valuable in identifying further research questions. We recommend studies to determine optimum benefit related to dosage, timing for starting therapy and the most appropriate GCS type. Given the advances in MS therapeutics, these studies will have to include patients on additional disease modifying therapy.

The neurology quality-of-life measurement initiative.

OBJECTIVE To describe the development and calibration of the banks and scales of the Quality of Life in Neurological Disorders (Neuro-QOL) project, commissioned by the National Institute of Neurological Disorders and Stroke to develop a bilingual (English/Spanish), clinically relevant, and psychometrically robust health-related quality-of-life (HRQOL) assessment tool. DESIGN Classic and modern test construction methods were used, including input from essential stakeholder groups. SETTING An online patient panel testing service and 11 academic medical centers and clinics from across the United States and Puerto Rico that treat major neurologic disorders. PARTICIPANTS Adult and pediatric patients representing different neurologic disorders specified in this study, proxy respondents for select conditions (stroke, pediatric conditions), and English- and Spanish-speaking participants from the general population. INTERVENTIONS Not applicable. MAIN OUTCOME MEASURES Multiple generic and condition-specific measures used to provide construct validity evidence for the new Neuro-QOL tool. RESULTS Neuro-QOL has developed 14 generic item banks and 8 targeted scales to assess HRQOL in 5 adult (stroke, multiple sclerosis, Parkinsons disease, epilepsy, amyotrophic lateral sclerosis) and 2 pediatric conditions (epilepsy, muscular dystrophies). CONCLUSIONS The Neuro-QOL system will continue to evolve, with validation efforts in clinical populations and new bank development in health domains not presently included. The potential for Neuro-QOL measures in rehabilitation research and clinical settings is discussed.

Validity and reliability of the MSQLI in cognitively impaired patients with multiple sclerosis.

Multiple sclerosis (MS) has important effects on quality of life but it is unknown how cognitive impairment affects the ability to assess or report this. O ur objective was to determine whether cognitive impairment negatively affects the construct validity and the reliability of the Multiple Sclerosis Q uality of Life Inventory (MSQLI). A neuropsychological test batter y and the Multiple Sclerosis Functional C omposite (MSFC) were administered to a sample of 136 patients referred for cognitive testing by their neurologists. A ge, sex, educatio n and ethnicity-adjusted T scores were calculated for each cognitive variable. C ognitive impairment was defined as any T score less than the fifth percentile. The MSQ LI was administered prior to neuropsychological testing and readministered one to four weeks later. C orrelations between the MSFC and the SF-36 were determined and compared between the cognitively impaired and unimpaired groups as the main test of construct validity. Test -retest and internal consistency reliability of each of the scales were compared for the impaired and unimpaired groups. Seventy-six (56%) patients were cognitively impaired. C onstruct validity and internal consistency reliability did not differ between the cognitively impaired and unimpaired groups. Test -retest reliability was lower for the bladder and vision scales in the impaired group, but remained acceptable for the bladder scale (r >0.7). C ognitive impairment, a common MS manifestation, does not appear to reduce the reliability or validity of the MSQ LI as a patient self-report measure of health status and quality of life.

Association of fatigue and brain atrophy in multiple sclerosis

Functional imaging studies have demonstrated a relationship between fatigue and altered cerebral activation patterns in multiple sclerosis patients, but no relationship between fatigue and brain atrophy has been demonstrated. We hypothesized that the subjective complaint of fatigue would predict the severity of destructive brain pathology. We assessed the relationship between fatigue and brain atrophy longitudinally in a cohort of 134 patients previously enrolled in a 2-year clinical trial of interferon beta-1a and re-evaluated 8 years after randomization into the trial. Brain atrophy was measured using the brain parenchymal fraction (BPF), and disability was measured using the Multiple Sclerosis Functional Composite at baseline, year 2 and year 8. Fatigue was measured using the Sickness Impact Profiles Sleep and Rest Scale (SIPSR) at baseline, year 2 and year 8. Linear regression analyses were used to assess the relationship between changes in fatigue and atrophy progression. Worsening fatigue on the SIPSR during the initial 2 years was significantly associated with progressive brain atrophy over the subsequent 6 years. The relationships between fatigue and brain atrophy were independent of changes in disability, mood, or other MRI characteristics. This suggests that the subjective complaint of fatigue is linked to destructive pathologic processes in RRMS patients.

Health-Related Quality of Life in Multiple Sclerosis Current Evidence, Measurement and Effects of Disease Severity and Treatment

Health-related quality of life (HR-QOL) is an important measure of health in patients with multiple sclerosis (MS), given that MS symptoms affect many aspects of everyday living. Physicians may tend to focus on physical or cognitive changes in patients with MS because these measures involve physician-or psychologist-administered tests rather than patient self-reporting. However, a number of validated instruments are available to evaluate HR-QOL in clinical studies. Several studies have used these instruments to evaluate the effects of traditional disease-modifying therapies (DMTs), i.e. interferon-β and glatiramer acetate on HR-QOL in patients with MS. The results of many of these studies showed that DMTs improved some aspects of patients’ HR-QOL, but study design issues such as small patient numbers or lack of placebo control for comparison have made it difficult to interpret these results. Two large, randomized, placebo-controlled studies of the newest DMT, natalizumab, showed that this therapy resulted in significant improvements in HR-QOL in patients with relapsing MS. Furthermore, the effects of natalizumab on HR-QOL were apparent, regardless of disease characteristics. The natalizumab studies definitively show that HR-QOL measures can be informative in a clinical trial setting and support the position that patient-reported outcomes, including HR-QOL measures, should be included in clinical trials to more fully assess therapeutic efficacy.