Developing patient-reported outcome measures for pain clinical trials: IMMPACT recommendations.
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Clinical Trials as Topic↗
Biomedical subjects
Kathleen W Wyrwich
Publications and source records attributed to Kathleen W Wyrwich.
A comparison of traditional and Rasch cut points for assessing clinically important change in health-related quality of life among patients with asthma.
BACKGROUND: Patient-perceived change in health-related quality of life (HRQoL) domains has often been classified using a 15-point patient transition rating scale. However, traditional change levels of trivial ( - 1, 0, or 1), minimal (2, 3 or - 2, - 3), moderate (4, 5 or - 4, - 5) and large (6, 7 or - 6, - 7) on this scale have been arbitrarily defined and originally assumed that change related to an improvement was the same as that for a decline. OBJECTIVE: To compare traditional and Rasch partial credit model-derived cut points and the mean changes for each change categorization when assessing clinically important change in asthma-specific HRQoL. METHODS: Our sample included 396 asthmatic outpatients who completed bimonthly telephone interviews on the Asthma Quality of Life Questionnaire and transition rating items over 1 year of participation. We employed item response theory in a novel approach to identify cut points on domain-specific HRQoL change data and transition ratings. After determining natural cut points for minimal, moderate, and large differences on the transition rating anchor, we calculated mean changes under change categorizations for both improvements and declines for the two transition rating classification approaches. RESULTS: Although traditional and Rasch categorizations for small, moderate, and large changes slightly differed and displayed a lack of symmetry between improvements and declines, nearly all mean changes between classification approaches were comparable. CONCLUSIONS: In this study, traditional transition rating cut points remain suitable to assess HRQoL clinical significance in outpatients with asthma.
Adult↗
Physician-estimated disease severity in patients with chronic heart or lung disease: a cross-sectional analysis.
BACKGROUND: We evaluated how well physicians' global estimates of disease severity correspond to more specific physician-rated disease variables as well as patients' self-rated health and other patient variables. METHODS: We analyzed baseline data from 1662 primary care patients with chronic cardiac or pulmonary disease who were enrolled in a longitudinal study of health-related quality of life (HRQoL). Each patient's primary physician rated overall disease severity, estimated the two-year risk of hospitalization and mortality, and reported the use of disease-specific medications, tests, and subspecialty referrals. Patient variables included sociodemographic characteristics, psychosocial factors, self-rated health, and both generic and disease-specific HRQoL. RESULTS: Physicians rated 40% of their patients "about average", 30% "worse", and 30% "better" than the typical patient seen with the specific target disorder. The physician's global estimate of disease severity was strongly associated (P < 0.001) with each of the five more specific elements of physician-rated disease severity, but only marginally associated with patient self-rated health. Multivariable regression identified a set of patient variables that explained 16.4% of the variance in physician-rated disease severity. CONCLUSION: Physicians' global ratings may provide disease severity and prognostic information unique from and complementary to patient self-rated health and HRQoL measures. The elements influencing physician-rated disease severity and its predictive validity for clinical outcomes warrant prospective investigation.
Adult↗
Understanding global transition assessments.
OBJECTIVE: Several methods currently used for determining meaningful or important change in health-related quality of life (HRQoL) measures are anchored by global transition items. It can be empirically demonstrated that patients' responses to these gold standards for change do not adequately incorporate the prior condition, yet these transition items play an influential role in patient-clinician communications and cannot be disregarded. This follow-up study queried patients to better understand how they ascertained their transition assessments. DATA SOURCE: Prior to their qualitative interviews, the 41 participants in this study had completed 1 year of enrollment in a clinical study with bi-monthly HRQoL telephone interviews that included the SF-36 and a disease-specific HRQoL instrument (asthma, COPD, or heart disease), as well as global transition assessments for each instruments domains. STUDY DESIGN: We used face-to-face cognitive interview and the think aloud approaches to obtain qualitative clues for understanding patient reports of HRQoL changes over time. Interview transcripts were coded using the four components of the Rapkin-Schwarz Appraisal Model: frame of reference; sampling strategy; standards of comparison; and combinatory algorithm. PRINCIPAL FINDINGS: Patients' explanations for determining global transition item responses often focused on current mobility and fatigue patterns, physicians' statements about their conditions, comparisons of their current states with the prior health conditions, and strong personal convictions that reflected a resistance to voicing any emotional changes. Patients also expressed primary health concerns, like back pain, that were not related to the chronic conditions (asthma, COPD or heart disease) under investigation. CONCLUSIONS: HRQoL studies and patient-clinician communications that incorporate global transition assessments to anchor the interpretations of HRQoL changes should also consider additional relevant questions to understand the process patients uses to appraise and report changes in HRQoL.
Aged↗
Assessing fatigue in persons with cancer: further validation of the Wu Cancer Fatigue Scale.
Cancer-related fatigue (CRF) is a significant clinical symptom. Effective assessment of CRF attributes from the patients' perspective is essential. This study tested the psychometric properties of the Wu Cancer Fatigue Scale (WCFS). A total of 172 outpatients with breast cancer, who were at various stages and on various chemotherapy regimens, and were undergoing treatment at one of three cancer clinics in a Midwest metropolitan area, participated in this study. The participants were instructed to complete four instruments in the following order: the 16-item WCFS, Schwartz Cancer Fatigue Scale (SCFS), Geriatric Depression Scale (GDS), and Cancer-Related Fatigue Distress Scale (CRFDS). Structural equation modeling (LISREL 8.54) supported the one-factor measurement model with nine items remaining. Nonsignificant Satorra-Bentler Scaled Chi-square (27)=32.52, P=0.21, standardized root mean square residual=0.032, nonnormal fit index=0.97, comparative fit index=0.98, and incremental fit index=0.98 indicated a good model fit. Convergent validity with the SCFS was 0.78, concurrent validity with the GDS was 0.60, and predictive validity with the CRFDS was 0.73. Internal consistency reliability was alpha=0.91 for the nine-item scale. The revised WCFS is a reliable and valid instrument that aims to measure the subjective characteristics of CRF from the patients' perspective. It may prove useful in both clinical and research settings.
Adult↗
Interpreting quality-of-life data: methods for community consensus in asthma.
BACKGROUND: Change in health-related quality of life (HRQoL) is an important outcome in asthma treatment. Patient and provider consensus on how to determine thresholds for identifying important improvements and declines, however, has not been achieved. OBJECTIVE: To search for consensus in clinically important difference (CID) thresholds for HRQoL change from 3 points of view: (1) an expert panel of physicians who treat patients with asthma and measure the HRQoL of their patients, (2) asthmatic patients, and (3) the primary care physicians (PCPs) of these asthmatic patients. METHODS: The expert panel used Delphi and consensus methods to agree on CID levels for the Asthma Quality of Life Questionnaire and the Medical Outcomes Study 36-Item Short-Form Health Survey, version 2.0. A total of 396 patients attending midwestern primary care clinic sites completed these interviews bimonthly for 1 year. The 46 PCPs treating these patients completed baseline assessments on each patient's asthma and then evaluated the change in each patient's condition at subsequent office visits during the next year. RESULTS: The patient-perceived estimates were consistent with results of previous studies but were notably lower than those derived from the expert panel and the PCPs. The Short-Form Health Survey generally did not provide sufficient sensitivity to detect important changes. CONCLUSIONS: Although consensus on the interpretation of change in HRQoL measures was not achieved, this evidence-based approach demonstrates the value of patient and physician perspectives and the need for improved dialogue and understanding to optimize the HRQoL of patients with asthma.
Adult↗
Individual, social environmental, and physical environmental influences on physical activity among black and white adults: a structural equation analysis.
BACKGROUND: Social ecological models suggest that conditions in the social and physical environment, in addition to individual factors, play important roles in health behavior change. Using structural equation modeling, this study tested a theoretically and empirically based explanatory model of physical activity to examine theorized direct and indirect effects of individual (e.g., motivation and self-efficacy), social environmental (e.g., social support), and physical environmental factors (e.g., neighborhood quality and availability of facilities). METHOD: A community-based sample of adults (N = 910) was recruited from 2 public health centers (67% female, 43% African American, 43% < $20,000/year, M age = 33 years) and completed a self-administered survey assessing their current physical activity level, intrinsic and extrinsic motivation for physical activity, perceived social support, self-efficacy, and perceptions of the physical environment. RESULTS: Results indicated that (a) perceptions of the physical environment had direct effects on physical activity, (b) both the social and physical environments had indirect effects on physical activity through motivation and self-efficacy, and (c) social support influenced physical activity indirectly through intrinsic and extrinsic motivation. For all forms of activity, self-efficacy was the strongest direct correlate of physical activity, and evidence of a positive dose-response relation emerged between self-efficacy and intensity of physical activity. CONCLUSIONS: Findings from this research highlight the interactive role of individual and environmental influences on physical activity.
Adolescent↗
Improving pilot response to in-flight strokes: a randomized controlled trial.
BACKGROUND: When a passenger suffers an in-flight stroke (IFS), the pilot decides when to expedite access to ground care. Pilot pro-activeness towards IFS could improve outcome and should be promoted. Unfortunately, little is known about a pilot's stroke pro-activeness or limiting factors. METHODS: Randomized controlled trial of an educational intervention (American Stroke Association, 30-slide stroke awareness lecture) through an internet-based computer system using pilot students and instructors as subjects. Pilots completed pre-intervention and post-intervention tests of 25 simulated in-flight scenarios that describe strokes and other neurological and medical symptoms. Outcomes chosen were the percentage of pilots that would use a medical radio service, declare an emergency on board, or divert to the nearest airport for each scenario. RESULTS: Participant pilots (n = 104) were less likely to respond to IFS than to myocardial infarction (p < 0.001). Fear of retaliation by an employer was the most important modifiable limitation. The educational program increased the simulated rate of emergency declarations for in-flight vertebrobasilar strokes (p < 0.001) and subarachnoid hemorrhage (p < 0.001). CONCLUSIONS: Pilot-simulated response to certain IFS improves immediately after this educational intervention, which should be disseminated in schools and airlines. Further studies are needed to determine the long-term benefits of this intervention and the impact on actual diversion rates. Companies should also review their policies to shield pilots from retaliation when altering the flight plan for patients.
Aviation↗
Estimating clinically significant differences in quality of life outcomes.
OBJECTIVE: This report extracts important considerations for determining and applying clinically significant differences in quality of life (QOL) measures from six published articles written by 30 international experts, in the field of QOL assessment and evaluation. The original six articles were presented at the Symposium on Clinical Significance of Quality of Life Measures in Cancer Patients at the Mayo Clinic in April 2002 and subsequently were published in Mayo Clinic Proceedings. PRINCIPAL FINDINGS: Specific examples and formulas are given for anchor-based methods, as well as distribution-based methods that correspond to known or relevant anchors to determine important differences in QOL measures. Important prerequisites for clinical significance associated with instrument selection, responsiveness, and the reporting of QOL trial results are provided. We also discuss estimating the number needed to treat (NNT) relative to clinically significant thresholds. Finally, we provide a rationale for applying group-derived standards to individual assessments. CONCLUSIONS: While no single method for determining clinical significance is unilaterally endorsed, the investigation and full reporting of multiple methods for establishing clinically significant change levels for a QOL measure, and greater direct involvement of clinicians in clinical significance studies are strongly encouraged.
Cross-Sectional Studies↗
Bridging the gap: using triangulation methodology to estimate minimal clinically important differences (MCIDs).
This paper proposes the use of triangulation methodology to derive guidelines for interpreting change scores on health outcome measures. Triangulation integrates results from global ratings with clinical benchmarks of change, statistical estimates of magnitude, and qualitative data from patients and/or clinicians to derive guidelines that are not field-specific or method bound. A case study is presented to illustrate how this methodology can be applied. Secondary analyses were performed on blinded data from 2,971 patients enrolled in three phase IlIa clinical trials to develop guidelines for interpreting change scores on the Breathlessness Diary (BD), a relatively new approach for evaluating dyspnea outcomes in patients with chronic obstructive pulmonary disease. BD scores were examined by disease severity and rescue medication use. In addition, mean BD change scores by physician global ratings of efficacy were juxtaposed with changes in forced expiratory volume (FEV1) and St. George's Respiratory Questionnaire scores. Percent change, effect size, one-half standard deviation, and the standard error of measurement were used as statistical indicators of magnitude. Data from qualitative interviews provided insight into patient perspectives of change in dyspnea. Taking into consideration results across estimation methods, guidelines were developed for defining large, moderate, and small group-level mean changes on the BD. Areas of divergence and convergence across statistical indicators and clinical benchmarks in this case study highlight the importance of using triangulation methodology to derive guidelines that are both empirically sound and clinically relevant.
Adult↗
A comparison of clinically important differences in health-related quality of life for patients with chronic lung disease, asthma, or heart disease.
OBJECTIVE: On the eight scales of the Medical Outcomes Study Short-Form 36-Item Health Survey (SF-36), Version 2, we compared the clinically important difference (CID) thresholds for change over time developed by three separate expert panels of physicians with experience in quality of life assessment among patients with chronic obstructive pulmonary disease (COPD), asthma, and heart disease. STUDY DESIGN: We used a modified Delphi technique combined with a face-to-face panel meeting within each disease to organize and conduct the consensus process among the expert panelists, who were familiar with the assessment and evaluations of health-related quality of life (HRQL) measures among patients with the panel-specific disease. PRINCIPAL FINDINGS: Each of the expert panels first determined the magnitude of the smallest numerically possible change on each SF-36 scale, referred to as a state change, and then built their CIDs from this metric. All three panels attained consensus on the scale changes that constituted small, moderate, and large clinically important SF-36 change scores. The CIDs established by the heart disease panel were generally greater than the CIDs agreed on by the asthma and COPD panels. CONCLUSIONS: These panel-derived thresholds reflect possible differences in disease management among the represented panel-specific diseases, and are all greater than the minimal CID thresholds previously developed for the SF-36 scales among patients with arthritis. If confirmed among patients with the relevant diseases and those patients' physicians, these disease-specific CIDs could assist both researchers and practicing clinicians in the use and interpretation of HRQL changes over time.
Asthma↗
Clinically important differences in health status for patients with heart disease: an expert consensus panel report.
BACKGROUND: The purpose of the study was to develop clinically important difference (CID) standards for patients with coronary artery disease and congestive heart failure that identify small, moderate, and large intraindividual changes with time in a modified version of the Chronic Heart Failure Questionnaire (CHQ) and the Medical Outcomes Study Short-Form 36-Item Health Survey (SF-36, version 2). Prior work in ascertaining important difference standards for the CHQ have centered on patient-perceived changes. No important difference standards for the SF-36 have been published for patients with heart disease. This development of CIDs would facilitate the use of health status measures in daily clinical decision-making. METHODS: We used a modification of the RAND Appropriateness Method to assemble and guide a 9-member consensus panel of physicians with substantial experience in using the CHQ or the SF-36 among patients with heart disease. RESULTS: On the basis of their own experience using these measures and an extensive review of articles describing the development and use of these instruments, the expert panel achieved consensus on small, medium, and large clinically relevant changes in scores for the CHQ and SF-36. The CID standards established by this panel were slightly higher than the minimal important difference standards previously established for the CHQ using patient-perceived changes. CONCLUSIONS: The CID standards established by this expert panel provide an important and useful tool for determining whether routine clinical health status assessments will benefit patients and enhance physicians' decision-making capacity in clinical settings.
Chronic Disease↗
Minimal important difference thresholds and the standard error of measurement: is there a connection?
Several recently published investigations have examined the relationship between the magnitude of the standard error of measurement (SEM) and established thresholds for a minimal clinically important difference (MCID) or a minimal important difference (MID) for change scores on health-related quality of life (HRQOL) or health status measures. These investigations, however, have resulted in differing SEM criteria for the MCID or MID. This study reviews and compares two sets of studies: (1) three investigations using a disease-specific HRQOL measure among patient samples with the chronic disease (heart disease, chronic obstructive pulmonary disease, or asthma) that have consistently demonstrated a 1 SEM correspondence with the established MCIDs or MIDs and (2) three investigations among patients referred to physical therapists with back, lower extremity, and neck pain showing that approximately 2.3 SEMs estimated the established MCID standards for three different measures of health status. Chronic disease patients were classified to have a MCID or MID if their global change ratings for the better or the worse were 1, 2, or 3 on a Likert scale ranging from 1 (almost the same, hardly any better, or worse at all) to 7 (a very great deal better or worse). Back pain patients, however, needed average global transition scores of 5, 6, or 7 (a good, a great, or a very great deal better) on the same 7-point Likert scale in order to experience an MCID in their condition. Charting these change levels against their respective SEM-MID criteria provides insight and promise for linking SEM-based criteria to MCID standards for other HRQOL and health status measures.
Data Interpretation, Statistical↗
Test-retest reliability of the Mirowsky-Ross 2 x 2 Index of the Sense of Control.
This study investigated the short-term stability of the 1991 Mirowsky-Ross 2 x 2 Index of the Sense of Control. From an ongoing longitudinal study, 304 subjects were randomly selected for test-retest interviews occurring 1 to 4 days after their regularly scheduled first follow-up interview. Test-retest reliability was assessed at the item level using percent agreement and weighted kappa. At the scale score level, reliability was assessed with the intraclass correlation coefficient (ICC). ICCs were also calculated within categories of demographic, socioeconomic, psychosocial, and functional status characteristics. There was moderate to substantial item-level agreement (mean weighted kappa = 51; weighted kappa range = .38 to .66). At the scale score level there was substantial agreement (ICC = .71). No appreciable differences in ICC values were found in the demographic, socioeconomic, psychosocial, and functional comparisons of status characteristics. Thus, this sense of control measure has acceptable test-retest reliability and is appropriate for use in longitudinal research.
Adult↗
Continuous or emergent EEG: can bedside caregivers recognize epileptiform discharges?
OBJECTIVE: When emergent or continuous EEG monitoring is carried out to detect subclinical seizures, expert interpretation of the results is usually not immediately available. We assessed the ability to recognize epileptiform discharges among various nonexpert bedside caregivers. A second objective was to determine whether such skill improves with a simple educational intervention. DESIGN AND SETTING: A prospective cohort study with an educational intervention in a university-based tertiary medical center. PARTICIPANTS: All 125 neurology and neurosurgery residents, intensive care fellows, critical care and neurological floor nurses, and EEG technicians were offered participation. Of these, 50 completed the study and provided 2,398 EEG responses. INTERVENTIONS: A computer-based PowerPoint lecture designed to teach recognition of epileptiform discharges. MEASUREMENTS AND RESULTS: (a) Average number of correct responses in identifying epileptiform discharges in a computer-based pre-test consisting of 24 EEG questions. (b) Impact of the educational intervention as measured by change in the mean average score in a similar post-test. The rate of correct responses by group ranged from 46% for medical critical care nurses to 94% for EEG technicians. Overall mean correct response rate was 14.72 (61%) for the pre-test, and increased to 16.06 (67%) in for post-test (p=0.002). CONCLUSIONS: Recognition of epileptiform discharges by bedside caregivers is disturbingly low. A simple educational intervention only modestly improved such ability. Therefore we should not rely on nontrained personnel to interpret emergent or continuous recordings. EEG technicians are an exception, and their skill could be utilized during emergent studies.
Cohort Studies↗
Clinically important differences in health-related quality of life for patients with asthma: an expert consensus panel report.
BACKGROUND: Standards for change in health-related quality of life (HRQoL) measures used among asthmatic patients have been established by considering only patient preferences to determine important differences and may not reflect an informed clinical evaluation of change. OBJECTIVE: To establish clinically important difference (CID) standards through the consensus of an expert physician panel for the Juniper Asthma Quality of Life Questionnaire (AQLQ) and Medical Outcomes Study Short-Form 36-Item Health Survey (SF-36, version 2) when used to measure HRQoL among asthmatic patients. METHODS: We organized an 8-person panel of physicians familiar with measuring HRQoL among asthmatic patients with the AQLQ and the SF-36. This expert panel participated in 2 rounds of a modified Delphi process before attending an in-person consensus meeting to establish the CID standards for small, moderate, and large changes in the AQLQ and the SF-36. After the consensus meeting, the panel used an iterative improvement process to cooperatively complete their final report. RESULTS: The expert panel's thresholds for detecting CIDs in the domains of the AQLQ were much higher than previously established levels using patient-perceived changes. CIDs for asthma were also ascertained on the scales of the SF-36, version 2, which were markedly greater than previously cited cross-sectional differences between patient groups. CONCLUSIONS: The CID standards established by this expert panel elucidate a potential distinction between patient and physician perspectives of important HRQoL changes. The many stakeholders of HRQoL difference standards should consider this distinction when adopting standards to evaluate patient change.
Asthma↗
Clinically important changes in health-related quality of life for patients with chronic obstructive pulmonary disease: an expert consensus panel report.
OBJECTIVE: Without clinical input on what constitutes a significant change, health-related quality of life (HRQoL) measures are less likely to be adopted by clinicians for use in daily practice. Although standards can be determined empirically by within-person change studies based on patient self-reports, these anchor-based methods incorporate only the patients' perspectives of important HRQoL change, and do not reflect an informed clinical evaluation. The objective of this study was to establish clinically important difference standards from the physician's perspective for use of 2 HRQoL measures among patients with chronic obstructive pulmonary disease (COPD). DESIGN: We assembled a 9-person expert panel of North American physicians familiar with the use of the Chronic Respiratory Questionnaire (CRQ), a disease-specific HRQoL measure, or the generic Medical Outcomes Study Short-Form 36-Item Health Survey (SF-36, Version 2.0) among patients with COPD. RESULTS: Using 2 rounds of the Delphi process, 1 in-person meeting, and an iterative improvement process for circulating and correcting the final report, the expert panel established small, moderate, and large clinically important change levels for the CRQ and SF-36. CONCLUSIONS: For this expert physician panel, levels for detecting clinically important differences on the CRQ were equal to or slightly higher than previous studies based on patient-reported differences. Clinically important differences on the SF-36, Version 2.0, were noticeably larger than previous estimates based on cross-sectional differences between clinically defined patient groups.
Health Status Indicators↗
9-11, personal stress, mental health, and sense of control among older adults.
OBJECTIVES: We assessed whether the events of 9-11 affected the personal stress, mental health, or sense of control of older adults participating in an ongoing longitudinal study, and whether baseline characteristics were associated with the magnitude of any such changes. METHODS: Personal stress, mental health, and sense of control were measured at baseline and at six bimonthly follow-up interviews among 1662 patients. Of these, 437 had the opportunity to complete three interviews before and after 9-11, with 291 (67%) completing all six. We performed graphic comparisons, paired t tests, classification based on standard errors of measurement (SEMs), and multiple linear regressions for patients who completed all six interviews. RESULTS: No noticeable changes in aggregate trends for personal stress or mental health were associated with 9-11. However, 9-11 was associated with an aggregate decline in sense of control. This decline in sense of control was greater among those who were working for pay, had more comfortable incomes, and reported greater religiosity. DISCUSSION: Older adults more closely resembling those who died during 9-11 and those with greater levels of religiosity were most likely to have their sense of control affected by this catastrophic event.
Activities of Daily Living↗