Exploring the patient experience of locally advanced or metastatic pancreatic cancer to inform patient-reported outcomes assessment.

PURPOSE: Pancreatic cancer and its treatments impact patients' symptoms, functioning, and quality of life. Content-valid patient-reported outcome (PRO) instruments are required to assess outcomes in clinical trials. This study aimed to: (a) conceptualise the patient experience of pancreatic cancer; (b) identify relevant PRO instruments; (c) review the content validity of mapped instruments to guide PRO measurement in clinical trials. METHODS: Qualitative literature and interviews with clinicians and patients were analysed thematically to develop a conceptual model of patient experience. PRO instruments were reviewed against the conceptual model to identify gaps in measurement. Cognitive debriefing explored PRO conceptual relevance and patients' understanding. RESULTS: Patients in the USA (N = 24, aged 35-84) and six clinicians (from US and Europe) were interviewed. Pre-diagnosis, pain was the most frequently reported symptom (N = 21). Treatments included surgery, radiation, chemotherapy, and immunotherapy. Surgery was associated with acute pain and gastrointestinal symptoms. Chemotherapy/chemoradiation side effects were cyclical and included fatigue/tiredness (N = 21), appetite loss (N = 15), bowel problems (N = 15), and nausea/vomiting (N = 15). Patients' functioning and well-being were impaired. The literature review identified 49 PRO measures; the EORTC QLQ-C30/PAN26 were used most frequently and mapped with interview concepts. Patients found the EORTC QLQ-C30/PAN26 to be understandable and relevant; neuropathic side effects were suggested additions. CONCLUSIONS: This is the first study to develop a conceptual model of patients' experience of metastatic/recurrent pancreatic cancer and explore the content validity of the EORTC QLQ-C30/PAN26 following therapeutic advances. The EORTC QLQ-C30/PAN26 appears conceptually relevant; additional items to assess neuropathic side effects are recommended. A recall period should be stated throughout to standardise responses.

Validity and performance of the Functional Assessment of Cancer Therapy-Bladder (FACT-Bl) among advanced urothelial cancer patients.

PURPOSE: The recent increase in emerging novel therapies in the bladder cancer therapeutic area has increased the need for fit-for-purpose patient-reported outcome (PRO) measures for these patients. This study evaluates the psychometric properties of the Functional Assessment of Cancer Therapy-Bladder (FACT-Bl) in 182 patients with advanced urothelial cancer (UC) and fills an important gap by demonstrating its validity for use in clinical trials. METHODS: Data were collected as part of a multicentre, open-label study of durvalumab in patients with inoperable or metastatic solid tumours. Psychometric properties evaluated include item and subscale characteristics (including correlation analysis), reliability (estimated using Cronbach's α), validity (by independent sample t test), responsiveness (using mixed models with repeated measures), and clinically meaningful changes using both anchor-based and distribution-based methods. RESULTS: One hundred and seventy-two patients completed the FACT-Bl questionnaire at baseline. Many individual items had floor or ceiling effects indicative of minimal symptoms and high functioning. The psychometric properties of the existing established scales were assessed and found to range from acceptable to very good. Internal consistency (most Cronbach's α coefficients range 0.66-0.85) and stability (test-retest reliability) generally exceeded standards for good reliability (most estimated intraclass correlations [ICCs] exceeded 0.70, although ICCs for some items [e.g. emotional well-being, ICC 0.58; social well-being, ICC 0.66] were lower than 0.70). Evidence for known-group validity of relevant FACT-Bl subscales and total score was demonstrated by significant differences between groups defined by baseline tumour burden and quality of life scores (difference of FACT-Bl total between low/high tumour burden groups 11.72 (p = 0.001); difference between low/high QoL scores groups 30.51 (p < 0.0001)). The FACT-Bl subscale and total scores were responsive to changes in bladder cancer symptom severity. Clinically meaningful changes in FACT-Bl scores were estimated. CONCLUSIONS: Results support the use of the FACT-Bl within this patient population in future clinical research.

Assessment of treatment-seeking behavior and healthcare utilization in an international cohort of subjects with overactive bladder.

UNLABELLED: Abstract Objective: To assess the association between incontinence severity, treatment-seeking behavior, and healthcare resource utilization (HRU) among participants with overactive bladder (OAB) in eight countries. RESEARCH DESIGN AND METHODS: A cross-sectional online survey of subjects ≥18 years old in Australia, Europe, and North America, who had a past OAB diagnosis and/or experienced ≥1 urinary incontinence (UI) episode in the preceding 12 months, were eligible to participate. Subjects contacted for the survey were primarily from a voluntary medication monitoring registry, MediGuard. Predominantly stress incontinence subjects were excluded. Incontinence severity was assessed by the number of UI episodes over 3 days and grouped as 0 ('dry'), 1-2, 3-4, and ≥5 UI episodes/day. Subject demographics, employment status, comorbidities, treatment-seeking behavior (past OAB diagnosis; spoken to healthcare provider [HCP]), and HRU (diagnostic tests; HCP visits in 6 months before screening) were analyzed by incontinence severity. RESULTS: Overall, 1341 subjects with OAB (mean age 54.5 years; 70.7% female) were surveyed; 20.2%, 47.7%, 18.8%, and 13.3% of subjects reported 0, 1-2, 3-4, and ≥5 UI episodes/day, respectively. Employment status and comorbidities were significantly (p < 0.05) associated with incontinence severity. The two measures of treatment-seeking behavior were significantly (p < 0.05) associated with incontinence severity groups; the proportion of subjects with a past diagnosis of OAB were 35.8%, 44.8%, 52.4%, and 64.0% in the 0, 1-2, 3-4, and ≥5 UI episodes/day groups, respectively; and 59.0%, 63.6%, 65.9%, and 78.1% of subjects in the respective UI severity groups talked to a HCP about their OAB symptoms. Multivariate linear regression analyses showed a positive and consistent association between incontinence severity and HRU; subjects reported a mean of 2.7, 4.1, 4.4, and 7.7 diagnostic tests overall (p < 0.001), and a mean of 1.4, 2.2, 2.7, and 4.0 HCP visits in the 0, 1-2, 3-4, and ≥5 UI episodes/day groups, respectively (p < 0.001). A potential limitation of the study is the cross-sectional survey methodology which limits the ability to draw causal inferences from the results. Additionally, since this is a web-based survey it is possible respondents who have access to/are familiar with technology were more likely to be enrolled. CONCLUSIONS: Incontinence severity was positively associated with both treatment-seeking behavior and HRU among subjects with OAB.

A cultural lens to understanding daily experiences with type 2 diabetes self-management among clinic patients in M'bour, Senegal.

Diabetes is a steadily increasing threat in Sub-Saharan Africa (SSA). Factors such as urbanization, obesity, physical inactivity, and inadequate access to healthcare are believed to contribute to the increasing burden of diabetes. Interventions that optimize diabetes self-management are critically important since obtaining diabetes medications is challenging due to cost constraints and availability. Culture is a significant factor in shaping health behaviors such as diabetes self-management, where individual health behaviors operate in confluence with family, community, and social structures. This study examined experiences with diabetes self-management among clinic patients residing in M'bour, Senegal, using the PEN3 model as a cultural framework. Results indicate that financial challenges related to accessing medical care and adhering to the prescribed diabetic diet were the main barriers to diabetes management. Family dynamics serve as both supportive and inhibiting forces that influence the aforementioned barriers.

Cost analysis and efficiency of sub-district health facilities in two districts in Ghana.

To establish the full costs borne by sub-district health facilities in providing services, we analysed the costs and revenues of 10 sub-district health facilities located in two districts in Ghana. The full costs were obtained by considering staff costs, cost of utilities, cost of using health facility equipment, cost of non-drug consumables, equipment maintenance expenses, amounts spent on training, community information sessions and other outreach activities as well as all other costs incurred in running the facilities. We found that (i) a large proportion of sub-district health facility costs is made up of staff salaries; (ii) at all facilities, internally generated funds (IGFs) are substantially lower than costs incurred in running the facilities; (iii) average IGF is several times higher in one district than the other; (iv) wide variations exist in efficiency indicators and (v) there is some evidence that sub-district health facilities may not necessarily be financially more efficient than hospitals in using financial resources. We suggest that the study should be replicated in other districts; but in the mean time, the health authorities should take note of the conclusions and recommendations of this study. Efforts should also be made to improve record keeping at these facilities.