Sophi Tatlock

What Outcomes are Important for Gout Patients? In-Depth Qualitative Research into the Gout Patient Experience to Determine Optimal Endpoints for Evaluating Therapeutic Interventions.

Background and ObjectivesCharacterized by sudden onset of severe joint pain, swelling, redness, and tenderness to touch, gout ‘flare ups’ have a substantial impact on quality of life (QoL). This research employed a patient-centered approach to explore the symptoms and impacts of gout, and assess the content validity of existing patient-reported outcomes (PROs).MethodsQualitative interviews were conducted with 30 US gout patients (non-tophaceous: n = 20, tophaceous: n = 10) and five expert rheumatologists. Each interview included both concept elicitation (CE) questioning to learn about the patient experience and cognitive debriefing to assess the content validity of three PRO instruments (HAQ-DI, GAQ, and TIQ-20). Nine of the patients provided further real-time qualitative data through a smart phone application. All qualitative data were subject to thematic analysis using Atlas.ti. Two patient advisors and three expert clinicians were engaged as advisors at key stages throughout the research.ResultsInterview and real-time data identified the same core symptoms and proximal impact concepts. Severe pain (typically in joints of extremities) was described as the cardinal symptom, often accompanied by swelling, redness, heat, sensitivity to touch, and stiffness. Domains of QoL impacted included physical functioning, sleep, daily activities, and work. The PRO instruments were generally well-understood by patients, but each included items with questionable relevance to at least some of the sample, dependent on the specific joints affected.ConclusionsGout patients experience severe pain in affected joints, resulting in substantial limitations in physical functioning. Both the HAQ-DI and the TIQ-20 are useful for specific research purposes in the gout population, although modifications are recommended.

Qualitative cross-cultural exploration of vaginal bleeding/spotting symptoms and impacts associated with hormone therapy in post-menopausal women to inform the development of new patient-reported measurement tools

OBJECTIVES To understand the vaginal bleeding/spotting experiences of postmenopausal (PM) women taking estrogen plus progestin therapies (EPT) and develop measures to assess these symptoms and their impact on womens daily lives in four countries. DESIGN (1) Concept elicitation interviews were conducted with PM women in the US (n=14), Italy (n=15), Mexico (n=15) and China (n=15) to explore vaginal bleeding/spotting symptoms associated with EPT. The Post-Menopausal Bleeding Questionnaire (PMBQ) was also debriefed to evaluate understanding and comprehensiveness. (2) Based on concept elicitation, a single item electronic daily diary was developed and the PMBQ modified to form a 12-item impact measure. (3) The measures were pilot-tested and then cognitively debriefed with US women receiving EPT. All qualitative data was subject to thematic analysis. MAIN OUTCOME MEASURES The Vaginal Bleeding/Spotting Daily Diary, (VBS-DD) and Post-Menopausal Bleeding Impact Questionnaire (PMBIQ) were developed in this study. RESULTS Concept elicitation identified vaginal bleeding and spotting as important symptoms for women taking EPT, impacting their emotional wellbeing, social life, ability to move freely, clothing and sexual activity. Based on pilot testing and cognitive debriefing, women demonstrated good understanding of the VBS-DD and the PMBQ was reduced to 10 items due to conceptual redundancy. CONCLUSIONS Women taking EPT in the US, China, Mexico and Italy reported vaginal bleeding/spotting symptoms that have a detrimental impact on their quality of life. Two new measures were developed to assess the severity and impact of vaginal bleeding/spotting specific to EPT. This work highlights the need for EPT-related symptoms to be a part of treatment decision-making.

Development and preliminary psychometric evaluation of an owner-completed measure of feline quality of life

Due to improved healthcare and pet longevity, measurement of health-related quality of life (HRQoL) is increasingly important in companion animal medicine. The aim of this study was to develop and evaluate the content and psychometric properties of an owner-completed assessment of health and quality of life (QoL) in cats for use in general veterinary clinical practice. A 23-item feline QoL measure, drafted based on findings from an online survey completed by 45 pet owners, was revised following qualitative interviews with 10 pet owners of healthy cats to assess content validity. The resulting 22-item measure was completed twice by 199 owners of healthy cats to assess the reliability and validity of the measure via psychometric evaluation, including assessment of missing data, item response distributions, item correlations, factor analysis, internal consistency, test-retest reliability, multi-trait analysis, known groups analyses and estimation of minimally important differences. There were no missing data. Responses for all items were heavily skewed due to the sample being healthy. Analysis of items and factor analysis supported deletion of six items and calculation of two domain scores and a total score. Internal consistency and test-retest reliability were strong for all domains (0.70-0.80), indicating good reliability. All but three items demonstrated strong item convergent validity (item-scale correlation>0.40) and correlated highest with their respective domain (item discriminant validity). Significant between-group differences in scores differing according to a global impression of feline health item provided evidence of discriminative validity. Findings provide evidence that the final 16-item feline QoL measure has strong cross-sectional psychometric properties.

Qualitative cross-cultural exploration of breast symptoms and impacts associated with hormonal treatments for menopausal symptoms to inform the development of new patient-reported measurement tools

To explore cross-cultural experiences of women taking estrogen plus progestin therapies (EPT) and develop a symptom-based electronic diary and impact questionnaire for EPT-related breast symptoms. (1) Concept elicitation interviews were conducted with women in the US (n=14), Italy (n=15), Mexico (n=15) and China (n=15) to explore breast symptoms associated with EPT. Patients completed the Breast Sensitivity Questionnaire (BSQ) to evaluate understanding and comprehensiveness. (2) Based on concept elicitation, a 6-item eDiary (Breast Pain/Tenderness Daily Diary - BPT-DD) was generated and the BSQ modified forming the 18-item Breast Sensations Impact Questionnaire (BSIQ). (3) The measures were pilot-tested and then cognitively debriefed with US women receiving EPT. All qualitative data was subject to thematic analysis. Concept elicitation identified breast pain/tenderness, swollen breasts and sensitivity to contact as important symptoms, impacting womens emotional well-being, relationships with family/friends, social life, sleep, ability to move freely, contact, clothing and sexual activity. Experiences were relatively consistent across the country samples. Based on pilot testing and cognitive debriefing, the BPT-DD was reduced to 4 items (and renamed the Breast Pain Daily Diary - BP-DD) and the BSIQ was reduced to 13 items due to conceptual redundancy. Women taking EPT in the US, China, Mexico and Italy reported breast sensations that have a detrimental impact on quality of life. Two new measures were developed to assess the severity and impact of breast pain specific to EPT. This work highlights that EPT-related symptoms should be part of treatment decision-making, and treatments with less burdensome side effects are needed.

Evaluation of a crosswalk between the European Quality of Life Five Dimension Five Level and the Menopause-Specific Quality of Life questionnaire

Abstract Objectives: Postmenopausal (PM) women taking therapies using estrogens plus progestogens (EPTs) can experience side effects (breast pain, vaginal spotting/bleeding). Sensitivity of the European Quality of Life Five Dimension Five Level (EQ-5D-5L) in measuring quality of life of PM women experiencing side effects of EPTs is unknown. A crosswalk between the Menopause-Specific Quality of Life (MENQOL) questionnaire and the EQ-5D-5L was assessed. Methods: The measures were administered to 352 PM women (side effects = 202; control = 75; untreated = 75) in a non-interventional study. MENQOL total scores, treated as continuous and categorical predictors, were mapped onto EQ-5D-5L utilities using regression. Ordinary least-squares regression using averaged scores over time, goodness of fit, and estimated coefficients was also assessed. Results: Mean age was 53.7 years. The first model (MENQOL as a continuous variable) showed a moderate correlation (−0.589) and statistically significant relationship with the EQ-5D-5L (p < 0.001), with an equation of EQ-5D-5L = 0.992 − 0.042 × MENQOL. The EQ-5D-5L mean scores were comparable (side effects = 0.854; control = 0.927; untreated = 0.836) to MENQOL mean scores estimated in the first model (side effects = 0.865 [standard deviation 0.07]; control = 0.909; untreated = 0.833). Linearity assumptions were supported with MENQOL scores as a categorical predictor. Goodness of fit was moderate (R2 = 0.347; root mean squared error = 0.093). Conclusion: The crosswalk supports conversion of MENQOL scores to EQ-5D-5L-derived health utilities for group-level analyses in PM women.