Kellee Howard

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 EXACTs structure and item pool, ready for empirically based item reduction and reliability and validity testing.

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

BackgroundPremature 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.MethodsQualitative 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.ResultsParticipants 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.ConclusionThis 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.

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 Observers 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 Observers 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.

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 Alzheimers 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.

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.

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.

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

ObjectivesSleep 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.MethodsResearch 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.ResultsThe 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.ConclusionsThe 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.

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.