Caregiver Burden of Autosomal Dominant Polycystic Kidney Disease: A Qualitative Study.

Rationale & Objective: There is limited published research on how autosomal dominant polycystic kidney disease (ADPKD) impacts caregivers. This study explored how caregivers of individuals with ADPKD perceive the burdens placed on them by the disease. Study Design: Qualitative study consisting of focus groups and interviews. Discussions were conducted by trained interviewers using semi-structured interview guides. Setting & Participants: The research was conducted in 14 countries in North America, South America, Asia, Australia, and Europe. Eligible participants were greater than or equal to 18 years old and caring for a child or adult diagnosed with ADPKD. Analytical Approach: The concepts reported were coded using qualitative research software. Data saturation was reached when subsequent discussions introduced no new key concepts. Results: Focus groups and interviews were held with 139 participants (mean age, 44.9 years; 66.9% female), including 25 participants who had a diagnosis of ADPKD themselves. Caregivers reported significant impact on their emotional (74.1%) and social life (38.1%), lost work productivity (26.6%), and reduced sleep (25.2%). Caregivers also reported worry about their financial situation (23.7%). In general, similar frequencies of impact were reported among caregivers with ADPKD versus caregivers without ADPKD, with the exception of sleep (8.0% vs 28.9%, respectively), leisure activities (28.0% vs 40.4% respectively), and work/employment (12.0% vs 29.8%, respectively). Limitations: The study was observational and designed to elicit concepts, and only descriptive analyses were conducted. Conclusions: These findings highlight the unique burden on caregivers in ADPKD, which results in substantial emotional, social, and professional/financial impact.

Polycystic Kidney Disease-Related Disease Burden in Adolescents With Autosomal Dominant Polycystic Kidney Disease: An International Qualitative Study.

Rationale & Objective: Little is known about symptoms and disease impacts in adolescents with autosomal dominant polycystic kidney disease (ADPKD). The objective of the study was to explore these issues from the adolescent patient's perspective. Study Design: Observational, qualitative study. Setting & Participants: Eligible participants were 12-17 years old and had a diagnosis of ADPKD. Semi-structured interviews were conducted in 18 cities in 13 countries to elicit participant experiences of ADPKD-related symptoms and physical, social, and emotional impacts. Analytical Approach: Interviews were recorded, transcribed, and coded. Symptom and impact frequencies from the interviews were calculated, and representative quotes concerning elicited concepts were collated. Results: Thirty-three participants (mean age, 14.6 years; 42.4% female) completed interviews. Frequently reported symptoms included urinary urgency (n = 10; 30.3%) and back pain (n = 9; 27.3%). Consistent with previous findings in adults, participants experienced 3 primary types of pain: dull kidney pain, severe or sharp kidney pain, and a feeling of fullness and/or discomfort. Reported disease impacts included avoiding sports and physical activity (n = 10; 30.3%), missing school (n = 6; 18.2%) and social activities (n = 6; 18.2%), and feeling worried (n = 6; 18.2%), sad (n = 4; 12.1%), or frustrated (n = 3; 9.1%) about the disease and their future. Approximately one-fifth of participants (n = 7; 21.2%) reported that they were bothered or impacted by dietary limitations (primarily the need for reduced sodium intake and increased water intake). Limitations: The study had a small sample size. The researchers were unable to conduct focus groups with participants because of parental preferences. Conclusions: The findings from this exploratory study indicate that a substantial proportion of adolescents with ADPKD experience physical, social, and emotional impacts from their disease.

The development of a patient-reported outcome measure for assessing nighttime symptoms of chronic obstructive pulmonary disease.

BACKGROUND: The assessment of symptoms of chronic obstructive pulmonary disease (COPD) is important for monitoring and managing the disease and for evaluating outcomes of interventions. COPD patients experience symptoms during the day and night, and symptoms experienced at night often disturb sleep. The aim of this paper is to describe methods used to develop a patient-reported outcome (PRO) instrument for evaluating nighttime symptoms of COPD, and to document evidence for the content validity of the instrument. METHODS: Literature review and clinician interviews were conducted to inform discussion guides to explore patients' nighttime COPD symptom experience. Data from focus groups with COPD patients was used to develop a conceptual framework and the content of a new PRO instrument. Patient understanding of the new instrument was assessed via cognitive interviews with COPD patients. RESULTS: The literature review confirmed that there is no instrument with evidence of content validity currently available to assess nighttime symptoms of COPD. Additionally, the literature review and clinician interviews suggested the need to understand patients' experience of specific symptoms in order to evaluate nighttime symptoms of COPD. Analyses of patient focus group data (N = 27) supported saturation of concepts and aided in development of a conceptual framework. Items were generated using patients' terminology to collect data on concepts in the framework including the occurrence and severity of COPD symptoms, use of rescue medication at night, and nocturnal awakening. Response options were chosen to reflect concepts that were salient to patients. Subsequent cognitive interviewing with ten COPD patients demonstrated that the items, response options, recall period, and instructions were understandable, relevant, and interpreted as intended. CONCLUSIONS: A new PRO instrument, the Nighttime Symptoms of COPD Instrument (NiSCI), was developed with documented evidence of content validity. The NiSCI is ready for empirical testing, including item reduction and evaluation of psychometric properties.

Validation of a patient interview for assessing reasons for antipsychotic discontinuation and continuation.

INTRODUCTION: The Reasons for Antipsychotic Discontinuation Interview (RAD-I) was developed to assess patients' perceptions of reasons for discontinuing or continuing an antipsychotic. The current study examined reliability and validity of domain scores representing three factors contributing to these treatment decisions: treatment benefits, adverse events, and distal reasons other than direct effects of the medication. METHODS: Data were collected from patients with schizophrenia or schizoaffective disorder and their treating clinicians. For approximately 25% of patients, a second rater completed the RAD-I for assessment of inter-rater reliability. RESULTS: All patients (n = 121; 81 discontinuation, 40 continuation) reported at least one reason for discontinuation or continuation (mean = 2.8 reasons for discontinuation; 3.4 for continuation). Inter-rater reliability was supported (kappas = 0.63-1.0). Validity of the discontinuation domain scores was supported by associations with symptom measures (the Positive and Negative Syndrome Scale for Schizophrenia, the Clinical Global Impression - Schizophrenia Scale; r = 0.30 to 0.51; all P < 0.01), patients' primary reasons for discontinuation, and adverse events. However, the continuation domain scores were not significantly associated with these other indicators. DISCUSSION: Results support the reliability, convergent validity, and known-groups validity of the RAD-I for assessing patients' reasons for antipsychotic discontinuation. Further research is needed to examine validity of the RAD-I continuation section.

Validation of a clinician questionnaire to assess reasons for antipsychotic discontinuation and continuation among patients with schizophrenia.

The Reasons for Antipsychotic Discontinuation Questionnaire (RAD-Q) was designed to assess clinicians' perceptions of reasons for antipsychotic discontinuation or continuation. The current study examined psychometric properties of this instrument and patterns of antipsychotic discontinuation. The sample of 121 patients (81 discontinuation, 40 continuation) with schizophrenia or schizoaffective disorder was 66.9% male, with a mean age of 41.6 years. Treating clinicians reported a mean of 4.1 reasons for discontinuation and 7.5 reasons for continuation. RAD-Q domain scores were derived to quantify the impact of three factors on the decision to discontinue or continue: treatment benefits, adverse events, and distal reasons other than direct effects of the medication. Analysis of inter-rater reliability indicated an acceptable degree of agreement between clinicians (weighted Kappa for discontinuation scores=0.70-0.78). Correlations with symptom measures (Clinical Global Impression-Schizophrenia Scale (CGI-SCH), Positive and Negative Syndrome Scale (PANSS)) supported convergent validity of the benefits domain score (r=0.28-0.47; all p<0.05). Domain scores discriminated among groups of patients differing in clinician and patient-reported clinical variables. Results suggest that the RAD-Q is a useful detailed measure of reasons for antipsychotic discontinuation and continuation. Findings indicate that clinicians usually report multiple reasons for discontinuation, rather than a single reason for each patient.

Utilities and disutilities for attributes of injectable treatments for type 2 diabetes.

INTRODUCTION: Although cost-utility models are frequently used to estimate treatment outcomes for type 2 diabetes, utilities are not available for key attributes of injectable treatments. The purpose of this study was to identify the utility or disutility of three injection-related attributes (dose frequency, dose flexibility, injection site reaction) that may influence patient preference. METHODS: Patients with type 2 diabetes in Scotland completed standard gamble (SG) interviews to assess the utility of hypothetical health states and their own current health state. The EQ-5D, PGWB, IWQOL-Lite, and QIDS were also administered. Construct validity and differences among health states were examined. RESULTS: A total of 151 patients completed interviews. Of the three injection-related attributes, dose frequency was the only attribute with a statistically significant impact on utility (in a multiple regression model, p = 0.01). Weekly injections were associated with an average added utility of 0.023 in comparison to everyday injections. Flexible dosing and injection site reactions resulted in somewhat smaller utility shifts that were in the expected directions (+0.006 and -0.011, respectively). SG utility of current health (mean = 0.897) demonstrated construct validity through statistically significant correlations with patient-reported outcome measures. DISCUSSION: The three injection attributes were associated with small utility shifts in the expected directions. Dose frequency appears to be the most important of the three attributes from the patients' perspective. The vignette-based SG approach was feasible and useful for assessing added utility or disutility of injection-related attributes associated with treatments for type 2 diabetes.