Mona L. Martin

Quality of Life of Persons With Urinary Incontinence: Development of a New Measure

OBJECTIVES Our objective was to develop a self-report quality of life measure specific to urinary incontinence (I-QOL) that could be used as an outcome measure in clinical trials and in patient care centers. METHODS The I-QOL was developed from interviews of 20 individuals with urinary incontinence. Refining the questionnaire was accomplished by structured interviews of 17 individuals with urinary incontinence. Testing the I-QOLs psychometric properties involved two administrations (n = 62) along with measures of psychologic well-being and functional status. RESULTS The rigorous development process ensured that the measure was complete and understandable. The I-QOL proved to be internally consistent (alpha 0.95) and highly reproducible (r = 0.93; 18 days; SD 4). For discriminant validity, severity of incontinence (P < 0.0001) and number of medical appointments in the past year to treat incontinence (P < 0.0001) significantly predicted I-QOL scores. Convergent validity analyses confirmed our predictions that the I-QOL scores were more closely related to overall well-being than bodily pain. CONCLUSIONS The I-QOL proved to be valid and reproducible as a self-administered measure for assessing quality of life of patients with urinary incontinence.

Quality of life of women with urinary incontinence: further development of the incontinence quality of life instrument (I-QOL)

OBJECTIVES To report on the further development of the Incontinence Quality of Life Instrument (I-QOL), a self-report quality of life measure specific to urinary incontinence (UI), including its measurement model, responsiveness, and effect size. METHODS Incontinent female patients (141 with stress, 147 with mixed UI) completed the I-QOL and comparative measures at screening, pretreatment, and four subsequent follow-up visits during participation in a multicenter, double-blind, placebo-controlled, randomized trial assessing the efficacy of duloxetine. Psychometric testing followed standardized procedures. RESULTS Factor analysis confirmed an overall score and three subscale scores (avoidance and limiting behaviors, psychosocial impacts, and social embarrassment). All scores were internally consistent (alpha = 0.87 to 0.93) and reproducible (ICC = 0.87 to 0.91). The pattern of previously reported correlations with the Short-Form 36-item Health Survey and Psychological Well-Being Schedule were confirmed. Responsiveness statistics using changes in the independent measures of stress test pad weight, number of incontinent episodes, and patient global impression of improvement ranged from 0.4 to 0.8. Minimally important changes ranged from 2% to 5% in association with these measures and effect sizes. CONCLUSIONS In a clinical trial, the I-QOL proved to be valid, reproducible, and responsive to treatment for UI in women.

Content Validity—Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part 2—Assessing Respondent Understanding

The importance of content validity in developing patient reported outcomes (PRO) instruments is stressed by both the US Food and Drug Administration and the European Medicines Agency. Content validity is the extent to which an instrument measures the important aspects of concepts developers or users purport it to assess. A PRO instrument measures the concepts most relevant and important to a patients condition and its treatment. For PRO instruments, items and domains as reflected in the scores of an instrument should be important to the target population and comprehensive with respect to patient concerns. Documentation of target population input in item generation, as well as evaluation of patient understanding through cognitive interviewing, can provide the evidence for content validity. Part 1 of this task force report covers elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument. Building on qualitative interviews and focus groups used to elicit concepts, cognitive interviews help developers craft items that can be understood by respondents in the target population and can ultimately confirm that the final instrument is appropriate, comprehensive, and understandable in the target population. Part 2 details: 1) the methods for conducting cognitive interviews that address patient understanding of items, instructions, and response options; and 2) the methods for tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies. The task force reports two parts are meant to be read together. They are intended to offer suggestions for good practice in planning, executing, and documenting qualitative studies that are used to support the content validity of PRO instruments to be used in medical product evaluation.

Content Validity—Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part 1—Eliciting Concepts for a New PRO Instrument

The importance of content validity in developing patient reported outcomes (PRO) instruments is stressed by both the US Food and Drug Administration and the European Medicines Agency. Content validity is the extent to which an instrument measures the important aspects of concepts that developers or users purport it to assess. A PRO instrument measures the concepts most significant and relevant to a patients condition and its treatment. For PRO instruments, items and domains as reflected in the scores of an instrument should be important to the target population and comprehensive with respect to patient concerns. Documentation of target population input in item generation, as well as evaluation of patient understanding through cognitive interviewing, can provide the evidence for content validity. Developing content for, and assessing respondent understanding of, newly developed PRO instruments for medical product evaluation will be discussed in this two-part ISPOR PRO Good Research Practices Task Force Report. Topics include the methods for generating items, documenting item development, coding of qualitative data from item generation, cognitive interviewing, and tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies. Part 1 covers elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument. Part 2 covers the instrument development process, the assessment of patient understanding of the draft instrument using cognitive interviews and steps for instrument revision. The two parts are meant to be read together. They are intended to offer suggestions for good practices in planning, executing, and documenting qualitative studies that are used to support the content validity of PRO instruments to be used in medical product evaluation.

Validation of the United States' version of the World Health Organization Quality of Life (WHOQOL) instrument

In 1991, the World Health Organization initiated a project to simultaneously develop a quality of life (QOL) instrument in 15 countries: The World Health Organization Quality of Life (WHOQOL) instrument. This was intended as a generic QOL tool for use with patients across varying disease types, severities of illness, and cultural subgroups. The objective of the current study was to evaluate the WHOQOL-100 in the U.S., one of the original 15 participating countries. The WHOQOL is a 100-item self-report instrument consisting of 24 subscales within six domains: Physical, Psychological, Independence, Social, Environment, and Spiritual. Four additional items pertain to overall QOL/health. We tested the WHOQOL-100 (U.S. version) in a sample of 443 adults (n = 251 chronically ill, n = 128 healthy, and n = 64 childbearing) in the U.S. to test its reliability (internal consistency, test-retest), construct validity (convergent, discriminant), responsiveness, and factor structure. The WHOQOL-100 (U.S. version) has acceptable internal consistency (alpha range: 0.82-0.95 across domains) and reproducibility (ICC range: 0.83-0.96 at 2-week retest interval). It is responsive to change in clinical conditions, as evidenced by predicted score change (effect size) in women after childbirth. Construct validity was demonstrated by (1) its correlation with the Short Form-36 and Subjective Quality of Life Profile, and (2) its ability to discriminate between the diverse samples in this study. The conceptual structure was confirmed exactly with the exception of four facets that did not correlate most highly with the domains to which they were originally assigned, but these differences were minor. The WHOQOL measurement system is suitable for evaluating the QOL of adults in the U.S. The psychometric properties will be continually evaluated as more data become available in the U.S.

Multinational Trials—Recommendations on the Translations Required, Approaches to Using the Same Language in Different Countries, and the Approaches to Support Pooling the Data: The ISPOR Patient‐Reported Outcomes Translation and Linguistic Validation Good Research Practices Task Force Report

OBJECTIVES With the internationalization of clinical trial programs, there is an increased need to translate and culturally adapt patient-reported outcome (PRO) measures. Although guidelines for good practices in translation and linguistic validation are available, the ISPOR Patient-Reported Outcomes Translation and Linguistic Validation Task Force identified a number of areas where they felt that further discussion around methods and best practices would be beneficial. The areas identified by the team were as follows: 1) the selection of the languages required for multinational trials; 2) the approaches suggested when the same language is required across two or more countries; and 3) the assessment of measurement equivalence to support the aggregation of data from different countries. METHODS The task force addressed these three areas, reviewed the available literature, and had multiple discussions to develop this report. RESULTS Decision aid tools have also been developed and presented for the selection of languages and the approaches suggested for the use of the same language in different countries. CONCLUSION It is hoped that this report and the decision tools proposed will assist those involved with multinational trials to 1) decide on the translations required for each country; 2) choose the approach to use when the same language is spoken in more than one country; and 3) choose methods to gather evidence to support the pooling of data collected using different language versions of the same tool.

Quality of life and parkinson's disease: Translation and validation of the US Parkinson's Disease Questionnaire (PDQ-39)

Parkinsons disease is a common progressive neurodegenerative disorder affecting an estimated 4 million people worldwide. A number of general health status measures exist but few fully capture the subjective evaluation of life quality associated with Parkinsons disease. We report here the results of: (1) translating the British PDQ-39 into a US version, (2) validity and reliability of the new US PDQ-39 questionnaire, and (3) parallel validation analyses following the method published in the development of the British version of the PDQ-39. Data were collected by postal survey on 150 patients recruited from neurology clinics in the Seattle area. A short, generic health status measure (SF-36) was used to test convergent validity, and a three-day test–retest assessed the reliability of the PDQ-39. The US version of the PDQ-39 demonstrated acceptable internal consistency (α = 0.51 to 0.96) and proved to be reproducible (0.86 to 0.96). Subscales of the PDQ-39 showed convergence with like scales of the SF-36 and was able to discriminate between levels of symptom severity.

Cultural Adaptation of a Quality-of-Life Measure for Urinary Incontinence

Objectives: To translate and validate a urinary incontinence-specific measure of quality of life (I-QOL) in French, Spanish, Swedish, and German and provide translations only into seven other languages and variants of these languages. Methods: Quality of life and linguistic experts prepared two forward translations from American English to their native languages and helped to harmonize these translations at a meeting. In the four European countries, the adapted versions of the I-QOL were administered to 259 women with stress, urge, and mixed incontinence. Principal component analyses were used to confirm the proposed measurement model suggested by patient interviews. Psychometric testing was conducted using standardized procedures. Results: Translation procedures resulted in a change in the original instrument’s Likert response scale from 4 to 5 points. Principal component analyses confirmed three patient-derived subscales and higher-order factor analysis confirmed a total summary score. In all countries, the internal consistency (alpha) and reproducibility (ICC) were high (alpha ranged between 0.87 and 0.93); (ICC ranged between 0.92 and 0.95). In all countries, I-QOL scores were significantly worse (p < 0.001) as perceived severity of incontinence, use of services, and number of incontinent episodes increased. Conclusions: The I-QOL has been adapted successfully into eleven languages and six variants of these languages. The cross-sectional psychometric properties of the US version were confirmed in four European countries. The I-QOL fills the need for a valid, international quality-of-life instrument for incorporation in clinical trials covering patients with varying types and severity of urinary incontinence.

Further evidence of the reliability and validity of the premature ejaculation diagnostic tool

This study details the further validation of the Premature Ejaculation Diagnostic Tool (PEDT), a five-item tool, developed to systematically apply the Diagnostic and Statistical Manual of Mental Disorders, revised version 4 (DSM-IV-TR), criteria in diagnosing presence or absence of premature ejaculation (PE). A total of 102 men completed the PEDT and were then interviewed by one of the seven clinical experts, who made a diagnosis of presence or absence of PE. The diagnoses from these two methods were compared to assess the convergent validity of PEDT. Retest reliability was also assessed, by men completing the PEDT a second time, approximately 2 weeks after the first administration. The level of agreement between clinical expert and PEDT diagnoses was very high (κ-statistic=0.80 (95% CI=0.68–0.92)), and retest reliability was very good – Intraclass correlation coefficient=0.88. In summary, the PEDT is extensively validated, self-report measure that can systematically assess DSM-IV-TR criteria to provide accurate diagnoses of PE/no-PE.

Synchrony of change in depressive symptoms, health status, and quality of life in persons with clinical depression

BackgroundLittle is known about longitudinal associations among measures of depression, mental and physical health, and quality of life (QOL). We followed 982 clinically depressed persons to determine which measures changed and whether the change was synchronous with change in depressive symptoms.MethodsData were from the Longitudinal Investigation of Depression Outcomes (LIDO). Depressive symptoms, physical and mental health, and quality of life were measured at baseline, 6 weeks, 3 months, and 9 months. Change in the measures was examined over time and for persons with different levels of change in depressive symptoms.ResultsOn average, all of the measures improved significantly over time, and most were synchronous with change in depressive symptoms. Measures of mental health changed the most, and physical health the least. The measures of change in QOL were intermediate. The 6-week change in QOL could be explained completely by change in depressive symptoms. The instruments varied in sensitivity to changes in depressive symptoms.ConclusionIn clinically depressed persons, measures of physical health, mental health, and quality of life showed consistent longitudinal associations with measures of depressive symptoms.