Elicitation of disease concepts in patients with diabetic foot ulcers: a qualitative study.

OBJECTIVE: To identify in-depth information directly from patients with diabetic foot ulcers (DFU) on DFU symptoms, impacts on functioning and effects on health-related quality of life (HRQoL). METHOD: Semi-structured, qualitative concept elicitation interviews were conducted with patients with DFUs (Wagner grade 1 or 2) until saturation was reached. Qualitative analysis (using MAXQDA, VERBI GmbH, Germany) of interview transcripts was conducted to identify concepts relevant to patients with DFUs, based on the frequency of mentions, and elucidate themes regarding impacts on HRQoL. RESULTS: Of the 18 participants, most were male (n=14; 78%) and 10 (56%) presented with a Wagner grade of 1. Frequently reported symptoms were pain/discomfort (n=15; 83%), weeping/discharge (n=10; 56%), bleeding (n=10; 56%) and swelling (n=8; 44%). Overall, patients reported more impacts than symptoms-wound care/treatment burden (n=14; 78%), limitations on exercise/physical activity (n=13; 72%), mobility limitations (n=12; 67%), and offloading (n=12; 67%) were the most frequently mentioned. Based on findings from the patient interviews, a draft conceptual model was developed outlining interrelationships between DFU symptoms, impacts, and HRQoL from the patient perspective. CONCLUSION: Qualitative interviews captured the breadth of disease-related concepts of direct importance to patients. The draft conceptual model developed from the analysis can help identify measures or instruments for use in assessing patient-reported symptoms or HRQoL in clinical practice and may have wider research applicability, including evaluation of treatment benefits in patients with DFUs.

Patient-Reported Outcome Assessments as Endpoints in Studies in Infectious Diseases.

The goal of administering medical interventions is to help patients live longer or live better. In keeping with this goal, there has been increasing interest in taking the "voice" of the patient into account during the development process, specifically in the evaluation of treatment benefits of medical interventions, and use of patient-centered outcome data to justify reimbursement. Patient-reported outcomes (PROs) are outcome assessments (OAs) used to define endpoints that can provide direct evidence of treatment benefit on how patients feel or function. When PROs are appropriately developed, they can increase the efficiency and clinical relevance of clinical trials. Several PROs have been developed for OA in specific infectious diseases indications, and more are under development. PROs also hold promise for use in evaluating adherence, adverse effects, satisfaction with care, and routine clinical practice.

Evidence to Support Montgomery-Asberg Depression Rating Scale Administration Every 24 Hours to Assess Rapid Onset of Treatment Response.

OBJECTIVE: This study investigated the suitability of the Montgomery-Asberg Depression Rating Scale (MADRS), with a 24-hour recall period (MADRS-24hr), to assess the rapid onset of the antidepressant effect of a treatment in patients with treatment-resistant depression (TRD). Psychometric properties of the MADRS-24hr were assessed together with qualitative assessment of content validity. METHODS: Content validity was assessed using semistructured interviews conducted from November 2013 to December 2013 in patients (18-64 years old) with TRD who met DSM-IV diagnostic criteria and health care professionals (HCPs) experienced in treating major depressive disorder and familiar with using the MADRS. The psychometric properties of MADRS-24hr were evaluated using data from 2 randomized clinical studies involving patients with TRD. RESULTS: A total of 23 patients (15 [65%] women) with TRD (mean age = 45 years) and 11 HCPs were interviewed. With the exception of reduced sleep, the majority of patients and HCPs reported that the items captured in the MADRS can fluctuate in a 24-hour period. The majority of participants also reported that a meaningful change in depression symptoms could be assessed in a 24-hour recall period, except for reduced sleep and appetite. Assessment of the psychometric properties of the MADRS-24hr showed that this instrument had high internal consistency reliability (Cronbach α of 0.84 and 0.91) and test-retest reliability (intraclass correlation coefficients of 0.96 and 0.91), had construct validity, and was responsive to change following an intervention. CONCLUSIONS: Overall, results suggest that MADRS-24hr can be used to assess the rapid onset of antidepressant efficacy of a treatment in patients with TRD. TRIAL REGISTRATION: ClinicalTrials.gov identifiers: NCT01627782 and NCT01640080.

Assessment of sleep in patients with fibromyalgia: qualitative development of the fibromyalgia sleep diary.

OBJECTIVES: Sleep disturbance is a common experience in fibromyalgia (FM). The field lacks a sleep specific patient reported outcome (PRO) measure developed and validated in a FM population. The study objective is to gain an in-depth understanding of sleep in FM and to develop a PRO measure of it. METHODS: Research involved the following stages: 1) A literature review conducted to identify key concepts associated with FM patient experience of sleep and PRO measures that have been used to assess this; 2) Qualitative interviews with therapeutic area experts; 3) Focus groups with FM patients who experienced sleep disturbance; 4) Development of a conceptual framework and the Fibromyalgia Sleep Diary (FMSD); and 5) Cognitive interviews with patients to explore content validity of the FMSD. RESULTS: The literature review and expert interviews supported sleep disturbance being an important aspect of the FM patient experience, and underscored the need for a new FM specific sleep PRO measure. Results from the focus groups demonstrated that FM patients experience sleep disturbances that they attribute to their FM symptoms, such as pain and stiffness, confirming the importance of understanding more about sleep changes. Aspects of sleep raised by FM patients included poor sleep quality and insufficient quantity including difficulty with falling asleep, getting comfortable, and staying asleep; restlessness; light sleep; not feeling rested upon awakening; and difficulty starting the day. Cognitive interview results showed that the 8-item FMSD, developed to reflect the concepts identified above, was relevant to FM patients with content that was interpreted as intended. CONCLUSIONS: The FMSD was developed in line with the recommendations of the FDA PRO guidance and ISPOR PRO Task Force. The qualitative evidence generated thus far strongly supports the content validity of the FMSD as a PRO measure of sleep disturbance in FM populations. Psychometric evaluation of the FMSD to demonstrate reliability, validity and sensitivity to change is recommended as a next step.

Reliability, validity, and interpretation of the dependence scale in mild to moderately severe Alzheimer's disease.

INTRODUCTION: The Dependence Scale (DS) was designed to measure dependence on others among patients with Alzheimer's disease (AD). The objectives of this research were primarily to strengthen the psychometric evidence for the use of the DS in AD studies. METHODS: Patients with mild to moderately severe AD were examined in 3 study databases. Within each data set, internal consistency, validity, and responsiveness were examined, and structural equation models were fit. RESULTS: The DS has strong psychometric properties. The DS scores differed significantly across known groups and demonstrated moderate to strong correlations with measures hypothesized to be related to dependence (|r| ≥ .31). Structural equation modeling supported the validity of the DS concept. An anchor-based DS responder definition to interpret a treatment benefit over time was identified. DISCUSSION: The DS is a reliable, valid, and interpretable measure of dependence associated with AD and is shown to be related to--but provides information distinct from--cognition, functioning, and behavior.

Item selection, reliability and validity of the Shortness of Breath with Daily Activities (SOBDA) questionnaire: a new outcome measure for evaluating dyspnea in chronic obstructive pulmonary disease.

BACKGROUND: Chronic obstructive pulmonary disease (COPD) is characterized by irreversible, progressive obstruction of lung airflow. Dyspnea (shortness of breath [SOB]) is the COPD symptom which most negatively impacts patients' daily activities. To assess how SOB affects daily activities, 37 items were drafted through focus group discussions and cognitive interviews with COPD patients to develop a patient-reported outcome instrument: the Shortness of Breath with Daily Activities questionnaire (SOBDA). Psychometric analysis was conducted to reduce the number of items and evaluate the measurement properties of the final SOBDA. METHODS: Prospective, observational study of 334 COPD patients, recruited from 24 pulmonology and internal medicine clinics in the United States. The 37-item SOBDA was administered to patients each evening for 28 days using an electronic diary. Patients answered every item and rated their level of SOB experienced that day during specific activities. Item selection was conducted by examining item characteristics, dimensionality, and Rasch model analysis results. The decision to delete an item was based on psychometric evidence, content validity, and expert clinical input. The final SOBDA instrument was evaluated for internal consistency, reproducibility, convergent validity, known-groups validity, and responsiveness. RESULTS: Twenty-four items from the 37-item pool were removed following the item selection process: nine items were removed due to high item-to-item correlations; five due to floor effects; three due to infrequent activity; one due to gender bias; two due to low factor loadings; three due to unordered response options; and one due to expert's discretion. Internal consistency and reproducibility of the final SOBDA were demonstrated by Cronbach Alpha = 0.87, and intra-class correlation coefficient = 0.91. Convergent validity was demonstrated by high correlation with the CRQ-SAS (0.60) and SGRQ-C (0.61). Known groups validity was demonstrated by significant difference between ratings of the mMRC and clinical global rating of severity. Evaluation of the ability to detect change was not performed owing to too few responders at the end of the study. CONCLUSIONS: Through the empirical item reduction process, 13 items were selected from the 37-item pool generated during qualitative development. The final 13-item SOBDA is a reliable and valid instrument for use in clinical trials.

Development of the Shortness of Breath with Daily Activities questionnaire (SOBDA).

OBJECTIVES: Based on qualitative research of patients with chronic obstructive pulmonary disease (COPD), the Shortness of Breath (SOB) with Daily Activities (SOBDA) questionnaire was developed as a patient-reported outcome instrument to evaluate the impact of therapy on SOB and assess how SOB affects daily activities. METHODS: Development of the SOBDA questionnaire consisted of three components. First, focus groups of patients with COPD were asked to describe their experiences of SOB with daily activities. A pool of items was drafted on the basis of information from the focus groups and literature reviews, and then discussed among instrument development and clinical experts. Cognitive debriefing interviews of patients were conducted to assess the draft item pool, and their feedback was used to develop newer versions of the questionnaire. Input was also sought from the Food and Drug Administration, patients, and clinicians. RESULTS: Forty patients participated in seven focus groups. The terms most often used to describe SOB were "short of breath" or "difficulty breathing." Patients were clearly able to distinguish SOB from chest congestion and wheezing, other common symptoms associated with COPD. The resulting item pool contained 37 items to assess SOB associated with everyday activities, and concept saturation was reached. Thirty-seven patients participated in the subsequent cognitive debriefing interviews. Patients found the items clear and easy to understand with relevance to their everyday experiences, and easy to use in an electronic format. CONCLUSIONS: Instructions and response options to the SOBDA questionnaire were well understood by patients with COPD, and item relevance was confirmed. Prospective validation and item reduction studies are highly anticipated.

Patient self-report for evaluating mild cognitive impairment and prodromal Alzheimer's disease.

Patient-reported outcome (PRO) measures are used to evaluate disease and treatments in many therapeutic areas, capturing relevant aspects of the disorder not obtainable through clinician or informant report, including those for which patients may have a greater level of awareness than those around them. Using PRO measures in mild cognitive impairment (MCI) and prodromal Alzheimer's disease (AD) presents challenges given the presence of cognitive impairment and loss of insight. This overview presents issues relevant to the value of patient report with emphasis on the role of insight. Complex activities of daily living functioning and executive functioning emerge as areas of particular promise for obtaining patient self-report. The full promise of patient self-report has yet to be realized in MCI and prodromal AD, however, in part because of lack of PRO measures developed specifically for mild disease, limited use of best practices in new measure development, and limited attention to psychometric evaluation. Resolving different diagnostic definitions and improving clinical understanding of MCI and prodromal AD will also be critical to the development and use of PRO measures.

Development of the EXAcerbations of Chronic Obstructive Pulmonary Disease Tool (EXACT): a patient-reported outcome (PRO) measure.

BACKGROUND: This article describes the qualitative methods used to develop the EXAcerbation of Chronic Pulmonary Disease Tool (EXACT), a new patient-reported outcome (PRO) instrument for evaluating frequency, severity, and duration of exacerbations of chronic obstructive pulmonary disease (COPD). METHODS: Focus groups and interviews were conducted in the United States with COPD patients treated for exacerbations during the past 6 months. Participants were asked to describe exacerbation attributes, care-seeking cues, and indications of progression and recovery. An iterative process was used to identify themes in the data to inform instrument content and structure. Cognitive debriefing interviews were performed to evaluate and revise the draft item pool. Experts in COPD, instrument development, and clinical research participated in the process. RESULTS: Eighty-three subjects participated in elicitation focus groups or interviews (n=48); elicitation interviews with cognitive debriefing (n=23), or cognitive interviews alone (n=12). Mean age of the sample was 65 years (SD=10); 45% were male; mean FEV-1% predicted was 44% (SD=16). Participants characterized exacerbations as a persistent increase in the severity of respiratory symptoms and other systemic manifestations accompanied by a dramatic reduction in activity. Specific attributes included shortness of breath, chest congestion, cough, sputum, chest discomfort, feeling weak or tired, sleep disturbances, and concern or worry. The diary card of 23 candidate items was debriefed in booklet and electronic format. CONCLUSIONS: Qualitative data from patients and input from experts formed the basis of the EXACT's structure and item pool, ready for empirically based item reduction and reliability and validity testing.

A qualitative assessment of the concept of dependence in Alzheimer's disease.

BACKGROUND: The Dependence Scale (DS) was designed to assess levels of patient need for care due to deficits typical of Alzheimer's disease (AD). This study examined content validity of the DS based on input from patients, caregivers, and clinicians. METHODS: Qualitative interviews with experts, patients, and caregivers were used to collect information on the concept of dependence and to assess content validity. RESULTS: Nine clinicians rated item relevance ''high'' with consensus on the primacy of functional abilities and dependence in the measurement of AD progression. Twenty-two US, 11 UK, and 14 informal caregivers from Spain participated in focus groups; 18 patients participated in 3 separate focus groups. Discussion supported DS hierarchy of dependence, capture of mild-to-severe dependence, suitability of response options, and short recall time frame. CONCLUSIONS: Clinicians, caregivers, and patients support content validity of the DS in mild-to-moderate AD. The DS may be valuable to capture dependence within future clinical dementia trials.

Development and validation of the patient and clinician sedation satisfaction index for colonoscopy and upper endoscopy.

BACKGROUND & AIMS: Recent increases in gastrointestinal endoscopic procedures and sedation options show a need for better sedation evaluation. This article describes the development and validation of 2 instruments: the Patient Satisfaction with Sedation Instrument (PSSI) and the Clinician Satisfaction with Sedation Instrument (CSSI) for assessing satisfaction in patients undergoing outpatient upper endoscopy and colonoscopy. METHODS: A total of 118 patients who underwent outpatient colonoscopy or esophagogastroduodenoscopy and 22 physicians were recruited across 5 US gastroenterology practices. Physicians completed the CSSI and patients completed the PSSI after each procedure. Patients completed the SF-12 Health Survey, Patient Satisfaction Questionnaire, and Socially Desirable Response Scale; clinicians completed the Observer's Assessment of Alertness Scale after procedures. RESULTS: Study patients were mostly women (59%), white (88%), had a mean age of 57.5 +/- 15.7 years, and had a colonoscopy (70%). Internal consistency reliabilities assessed result consistencies across test items. Coefficients greater than 0.70 indicate good reliability; greater than 0.85 indicate excellent reliability. Internal consistency reliabilities of the PSSI and CSSI total and subscale scores were greater than 0.76. Correlations were assessed using Pearson product moment correlation. PSSI and CSSI scores were correlated (P < .05). PSSI totals scores were correlated significantly with the SF-12 Health Survey (P < .05), the Patient Satisfaction Questionnaire (P < .05), and CSSI total scores were correlated with the Observer's Assessment of Alertness Scale (P < .01) and a visual analogue scale (P < .01). PSSI scores were not correlated with the Socially Desirable Response Scale (P > .05). CONCLUSIONS: The PSSI and CSSI provide for feasible, reliable, and valid assessment of procedural sedation satisfaction for outpatient colonoscopy and esophagogastroduodenoscopies and can be used for future sedation studies.

Characterizing the burden of premature ejaculation from a patient and partner perspective: a multi-country qualitative analysis.

BACKGROUND: Premature ejaculation (PE) is a common sexual dysfunction among men which affects men and their partners. Little qualitative data are available to characterize the impact of PE on men and their partners about ejaculatory control, sexual satisfaction, emotional distress and relationships. The objective of this study was to assess the impact of PE from the perspective of men with PE and the female partners of men with PE on their sexual experience, distress and relationships. METHODS: Qualitative data were collected through 14 focus groups in the US and through one-on-one interviews in the US, UK, Italy, France, Germany, and Poland. Study participants included heterosexual men with PE and female partners of males with PE. All participants were asked about how PE affects their daily life, including emotional impacts. One-on-one interviews also included obtaining feedback on the male and female versions of 4-single item measures of PE focusing on ejaculatory control, satisfaction with intercourse, interpersonal distress, and relationship difficulty. RESULTS: Participants included 172 males with PE and 67 female partners of men with PE. Lack of control over ejaculation and dissatisfaction with intercourse emerged as central themes of PE. Lack of ejaculatory control resulted in greater dissatisfaction and greater emotional distress, including feelings of inadequacy, disappointment, and anxiety. Continued PE ultimately leads to greater problems with partners and often disrupts partner relationships. Participants indicated that PE was keeping them from attaining complete intimacy in their relationships even when their partners were generally satisfied with sexual intercourse. Impacts of PE on sexual satisfaction, emotional distress and partner relationships were consistent across countries. Feedback on the single-item PE measures confirmed relevance of the item content and further confirmed major themes identified from the qualitative data. CONCLUSION: This qualitative study provides valuable insights on the substantial psychosocial burden of PE in the US and the Europe. Lack of control over ejaculation resulted in dissatisfaction with intercourse and increased emotional distress, and wide-ranging impact for both men with PE and their partners of men with PE. Data collected in this study may help inform the content of new patient reported measures for use in PE research.

Undertreatment of osteoporosis in residents of nursing homes: population-based study with use of the Systematic Assessment of Geriatric Drug Use via Epidemiology (SAGE) database.

OBJECTIVE: To determine the proportion of nursing home residents >65 years of age with osteoporosis who were receiving antiosteoporosis pharmacotherapy and to identify the predictors of administration of such drugs. METHODS: We identified 29,357 patients with osteoporosis documented on the Minimum Data Set collected on residents of all nursing home facilities in 5 states during the period from 1992 through 1996. A multiple logistic regression model was used for analysis, with the dependent variable being use of any antiosteoporosis drug. RESULTS: Among the nursing home residents with osteoporosis, 25% received antiosteoporosis drugs. Women were more likely than men to receive antiosteoporosis drugs (adjusted odds ratio [OR], 1.41; 95% confidence interval [CI], 1.26 to 1.57). Both increasing age and level of cognitive impairment were inversely related to receipt of antiosteoporosis drugs. A history of fracture or falls was not predictive of use of such drugs. Treatment was less likely for nursing home residents with > or = 6 medical conditions (OR 0.55, 95% CI 0.51 to 0.59); those admitted to a nursing home from a hospital (OR 0.86, 95% Cl 0.80 to 0.92); and those with a terminal prognosis (OR 0.60,95% CI 0.42 to 0.87). CONCLUSION: The majority of nursing home residents with osteoporosis in this study did not receive drug therapy for this disabling and treatable disease. Although acceptable reasons may exist in some of these residents,others--especially the oldest old--may not be receiving adequate care.