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BACKGROUND: Hypoparathyroidism is a rare endocrine disease frequently associated with serious physical and cognitive symptoms. This study's purpose was to understand the impacts of the phase 3 PaTHway clinical trial treatment, TransCon PTH, on patients' overall, physical, and cognitive hypoparathyroidism signs/symptoms and what patients consider meaningful improvement. METHODS: Individual telephone exit interviews were conducted with patients who recently completed the PaTHway trial blinded period. Using a semi-structured interview guide, interviews focused on trial treatment impact on hypoparathyroidism symptoms following the symptom list in the Hypoparathyroidism Patient Experience Scale-Symptom (HPES-Symptom). Meaningful changes in hypoparathyroidism symptoms were assessed with the Patient Global Impression of Severity (PGIS) and Patient Global Impression of Change (PGIC) measures. Interviewees were probed on the meaningfulness of reported changes in symptoms from prior to starting trial treatment to the past 2 weeks/current time. Interviews were audiotaped and transcribed. Transcripts were coded for emerging concepts and themes/subthemes covered in the interview guide based on an adapted grounded theory approach. RESULTS: Nineteen adults with hypoparathyroidism participated in interviews in the United States (n = 13, 68.4%) and Canada (n = 6, 31.6%). Marked improvements in physical and cognitive symptoms were described among trial treatment group respondents. The majority of participants who reported experiencing hypoparathyroidism physical symptoms pre-trial indicated symptom improvement with treatment, including muscle twitching (100%, n = 15), low energy (92.9%, n = 13), feeling tired (92.3%, n = 12), muscle weakness (92.9%, n = 13), tingling without numbness (84.6%, n = 11), trouble sleeping (92.3%, n = 12), muscle cramping (92.3%, n = 12), tingling with numbness (92.3%, n = 12), muscle spasms (100%, n = 12), and pain (90.9%, n = 10). Most participants who reported experiencing cognitive symptoms pre-trial reported symptom improvement with treatment, including difficulty finding the right words (86.7%, n = 13), difficulty concentrating (93.3%, n = 14), trouble remembering (92.9%, n = 13), trouble thinking clearly (85.7%, n = 12), and difficulty understanding information (83.3%, n = 10). Those in the placebo group reported limited or no improvement. The vast majority of participants affirmed that the improvements they experienced in symptom frequency on the PGIS/PGIC and HPES-Symptom were meaningful. CONCLUSIONS: Findings indicate that TransCon PTH treatment improved participants' physical and cognitive hypoparathyroidism symptoms in meaningful ways, while reducing the daily burden associated with conventional therapy. TRIAL REGISTRATION: NCT04701203 Registered: 06 January 2021. https://clinicaltrials.gov/study/NCT04701203?term=NCT04701203&rank=1 .

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BACKGROUND: Chronic obstructive pulmonary disease (COPD) and muscle weakness can cause impaired physical function, significantly impacting patients' health-related quality of life (HRQoL). Loss of muscle strength is usually assessed through clinical and performance outcome (PerfO) assessments, which consists of tasks performed in a standardized manner, providing evidence of a patient's functional ability. However, evidence documenting the patient experience of COPD and muscle weakness is limited. METHODS: This two-stage qualitative study used semi-structured interviews in patients aged 45-80 years with COPD (post-bronchodilator forced expiratory volume in 1s [FEV1]/forced vital capacity ratio < 0.70, and FEV1% predicted of 30-80%) and muscle weakness. In Stage 1, 30-minute concept elicitation interviews were conducted with participants recruited across three US sites to explore impacts on physical functioning and activities of daily living. In Stage 2, interviews were performed with participants exiting a Phase IIa trial investigating the efficacy of a selective androgen receptor modulator (GSK2881078) on leg strength, whereby PerfOs were used to evaluate strength and physical functioning endpoints. These participants completed either 60-minute in-depth (n = 32) or 15-minute confirmatory (n = 35) interviews exploring trial experience, completion of outcome measures, disease experience and treatment satisfaction. RESULTS: In Stage 1 (n = 20), most participants described their muscles as weak (83.3%). Difficulties with walking (100%) and lifting heavy objects (90%) were reported. In Stage 2, 60-minute interviews, all participants (n = 32) reported a positive trial experience. Most participants reported that the home exercise program was easy to fit into daily life (77.8%), the PROactive daily diary was easy to complete (100%) and wearable sensors were easy to use (65.6%). However, technical issues were reported (71%), and few participants (19.4%) found physical assessments easy to complete. Improvements in muscle strength and functional limitations were reported by most participants. The shorter 15-minute confirmatory interviews (n = 35) supported the in-depth interview results. CONCLUSION: The qualitative interviews generated in-depth evidence of key concepts relevant to patients with COPD and muscle weakness and support the assessments of patient strength and physical function as outcome measures in this population in future studies. TRIAL NUMBER: GSK Stage 1: 206869; Stage 2: 200182, NCT03359473; Registered December 2, 2017, https://clinicaltrials.gov/ct2/show/NCT03359473 .

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BACKGROUND: Evidence indicates that Nigeria's high maternal mortality rate is attributable primarily to events that occur during the intrapartum period. This study determines the effectiveness of multifaceted interventions in improving the quality of intrapartum care in Nigeria's referral hospitals. METHODS: Data collected through an exit interview with 752 women who received intrapartum care in intervention and control hospitals were analyzed. The interventions were designed to improve the quality indicators in the WHO recommendations for positive childbirth and assessed using 12 quality indicators. Univariate, bivariate, Poisson, and logistic regression analyses were used to compare twelve quality indicators at intervention and control hospitals. RESULTS: The interventions showed a 6% increase in composite score of quality of care indicators at intervention compared with control hospitals. Five signal functions of intrapartum care assessed were significantly (< 0.001) better at intervention hospitals. Quality scores for segments of intervention periods compared to baseline were higher at intervention than in control hospitals. CONCLUSIONS: We conclude that multiple interventions that address various components of the quality of intrapartum care in Nigeria's referral hospitals have demonstrated effectiveness. The interventions improved five of ten quality indicators. We believe that this approach to developing interventions based on formative research is important, but a process of integrating the implementation activities with the normal maternal health delivery processes in the hospitals will enhance the effectiveness of this approach. TRIAL REGISTRATION: The study was registered at the Nigeria Clinical Trials Registry. Trial Registration Number NCTR No: 91,540,209 (14/04/2016) http://www.nctr.nhrec.net/ and retrospectively with the ISRCTN. Trial Registration Number 64 ISRCTN17985403 (14/08/2020) https://doi.org/10.1186/ISRCTN17985403 .

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INTRODUCTION: Lichen planus (LP) is an inflammatory skin disorder that can present in various forms across the body, including lesions on the skin (cutaneous LP [CLP]), scalp (lichen planopilaris [LPP]), and mucosal regions (mucosal LP [MLP]). Qualitative exploration of the patient experience of LP, notably symptoms and impacts on health-related quality of life (HRQoL), is limited. A scarcity of research was also identified relating to emotional wellbeing impacts of CLP patients. Two qualitative studies were conducted with LP patients to address these gaps. METHODS: Study 1 consisted of exit interviews conducted with a subset of adult patients with MLP (n = 5), CLP (n = 4), and LPP (n = 4) enrolled in an LP clinical study in the United States (US) to explore the patient experience. Study 2 consisted of independent qualitative interviews conducted with adult CLP patients (n = 13) from the US and Germany to further explore impacts on emotional wellbeing. RESULTS: Exit interviews found that itch , pain, and skin lesions were most frequently reported as signs/symptoms of LP. Itch and skin lesions were experienced across all LP subtypes, while pain was only reported by CLP and MLP patients. These signs/symptoms impacted HRQoL including emotional wellbeing (frustration, embarrassment), daily activities (oral hygiene, clothing options), social functioning (intimacy, social activities), and physical functioning (chewing/swallowing, opening/moving mouth). Impacts on activities of daily living (ADL) and physical functioning were mostly experienced by MLP patients. Independent qualitative interviews, which further explored impacts of CLP on patients' emotional wellbeing, identified frustration, worry, sadness, embarrassment, and depression as the most frequently experienced. CONCLUSION: The findings contribute to the literature by providing qualitative insights into signs/symptoms and HRQoL impacts of LP, from the adult patient perspective. The findings also highlight the importance of considering assessment of HRQoL impacts in future clinical LP research, particularly impacts on emotional wellbeing when selecting instruments for assessment of HRQoL in the CLP population. TRIAL REGISTRATION: NCT04300296.

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BACKGROUND AND OBJECTIVE: The chest-related electronic patient reported outcome (ePRO) diary was recently developed to assess chest-related symptoms experienced by pediatric and adolescent populations during upper respiratory tract infections (URTI). The objective of this research was the psychometric evaluation of the chest-related ePRO diary in pediatric, adolescent and adult participants. METHODS: This non-interventional, psychometric validation study involved participants (N = 195; n = 42 6-8 years; n = 47 9-11 years; n = 55 12-17 years, n = 51 18+ years) completing the chest-related ePRO diary twice daily for 10 days while experiencing an acute URTI. Preliminary item-level performance and dimensionality results, along with consideration of previous qualitative findings, were used to inform item reduction decisions, the structure of the measure and scoring algorithm development. Subsequent analyses on the finalized measure included assessments of reliability (internal consistency and test-retest reliability), construct validity (convergent validity and known groups validity) and ability to detect change. Comparisons of findings were made between the different age groups as part of the analyses to assess the psychometric properties of the chest-related ePRO diary and to characterize potential differences in the symptom experience of children, adolescents, and adults. RESULTS: The measure demonstrated strong quality of completion and showed relatively similar trajectories of symptom scores over time within different age subgroups and good item response distribution properties. Exploratory factor analysis supported a one-factor solution in the total population and within age subgroups, and test-retest reliability of the measure was strong (Intra-class correlation: 0.843-0.894 between Visit 1 and Day 1). The measure also demonstrated strong construct validity through high correlations with relevant items on the Child Cold Symptom Questionnaire (CCSQ), strong known groups validity (with statistically significant differences between severity groups) and was responsive to change over time with change groups defined based on change on global items. CONCLUSION: The findings demonstrate that the chest-related ePRO diary provides a valid, reliable, responsive measure of chest congestion symptoms experienced with the common cold in pediatric and adolescent populations, and that only minor differences are present in the disease trajectory when comparing adults to younger participants, supporting the use of the measure in interventional studies.

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Increasing coverage of evidence-based maternal, neonatal, child, reproductive health and nutrition (MNCRHN) programs in low- and middle-income countries has coincided with dramatic improvements in health despite variable quality of implementation. Comprehensive evaluation to inform program improvement requires standardized but adaptable tools, which the Real Accountability, Data Analysis for Results (RADAR) project has developed. To inform selection of tools and methods packages ('packages') to measure program quality of care (QoC), we documented experiences testing the packages, which were developed and adapted based on global and local expertise, and pre- and pilot-testing. We conducted cross-sectional studies in 2018-2019 on the quality of 1) integrated community case management, 2) counseling on maternal, infant, and young child feeding, 3) intrapartum care, and 4) family planning counseling in Mali, Mozambique, Tanzania, and Malawi. Herein we describe package performance and highlight experiences that inform their selection and use. Direct observation packages provided high-quality, immediately applicable results but they required specialized expertise, in-person collection, adequate patient volume, reasonable wait times, and unambiguously 'correct' provision of care. General satisfaction questions from exit interview packages produced unvaryingly positive responses despite variable observed quality of care. Variation increased when questions were more targeted, but findings on caregiver and client's recall of recommendations were more actionable. When interactive, clinical vignettes can capture knowledge of clinical care. But for conditions that can be simulated, like provision of family planning counseling, we could capture provider practice from simulated clients. Clinicians could more easily demonstrate tactile aspects of intrapartum care using observed structured clinical examinations, but this method required storage and transport of the required mannequins. Based on our findings we recommend ten questions upon which evaluators can base package selection. Findings from these packages inform programs and, in the context of comprehensive program evaluation enable us to link programs with impact.

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BACKGROUND: Patient-reported outcome (PRO) instruments provide robust and effective means of evaluating patients' treatment experience; however, none adequately cover experience using self-injection devices with enhanced features, such as an electromechanical autoinjector (e-Device). The aim of this study was to develop a PRO instrument that accurately assesses patient experience of using an e-Device and to evaluate its psychometric properties. METHODS: A mixed-methods approach was taken; two parallel, targeted literature reviews were conducted to identify relevant concepts and existing self-injection PRO instruments that could be adapted. Patient feedback obtained from two focus groups was used to inform initial instrument development. The pilot instrument was then administered in a multicenter, open-label, phase 3 clinical study in which patients self-injected certolizumab pegol using an e-Device, to gather evidence of its psychometric qualities. Exit interviews were conducted with a sub-sample of patients enrolled in the study to confirm the appropriateness and clarity of the items included and cognitively debrief the instrument. Confirmatory factor analysis (CFA) was conducted on all items, and each domain's internal consistency was measured using Cronbach's ɑ. RESULTS: The literature searches identified several e-Device-specific concepts related to device features, device function, side effects/reactions/pain, confidence, and interference/convenience in daily life. Seven existing PRO instruments were identified. The Self-Injection Assessment Questionnaire (SIAQ), containing pre- and post-injection questionnaire modules, was selected as most suitable and adapted using feedback from 19 patients in the two focus groups to form the pilot Assessment of Self-Injection (ASI) questionnaire. CFA resulted in some changes to the grouping of items in the post-injection module domains following psychometric evaluation of the ASI. Internal consistency was satisfactory for all pre- and post-injection domains (ɑ > 0.8). Cognitive debriefing results from 12 patient exit interviews confirmed the ASI's appropriateness and clarity. CONCLUSIONS: The ASI was developed iteratively with patient input and was evaluated in its intended clinical context of use. Psychometric analyses indicated promising cross-sectional results; the ASI was well understood and considered relevant by patients self-injecting using the e-Device, suggesting that it could be used in real-world settings to aid with clinical decision making. TRIAL REGISTRATION: NCT03357471.

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AIM: The objectives of the study were to determine the level of satisfaction with Mental Health and Psycho-social Support Services (MHPSS) provided to Syrians under temporary protection (SuTP) in Turkey and identify predictors that significantly determine the satisfaction with the MHPSS. This study is part of an effort by Ministry of Health (MoH) and World Health Organization (WHO) to evaluate the improvement in the MHPSS following capacity strengthening of social and health providers. METHODS: 357 Syrians under temporary protection were interviewed as they exited the Refugee Health Training Centers in 7 provinces in Turkey (Adana, Gaziantep, Hatay, Istanbul, Izmir and Sanliurfa), using patient exit interviews during mid-Oct - mid-Nov 2019. Uni and bivariate analysis for association was done using Chi square test for categorical variables, looking for significance at p < 0.05. Multivariate analysis (logistic regression) was used to determine the profile of service users and the predictors of satisfaction with MHPSS. RESULTS: Overall satisfaction with services was 93%. The profile of the MHPSS user suggests that the odds of using the service are twice as much for people aged 40+ years (OR 2.016, CI95% [1.129-3.601]), and five times less for married women (OR 0.180, CI95% [0.083-0.391]). The service characteristics that can predict service satisfaction are "having the needs met" (OR 138.73, CI95% [27.99-687.54]) and "satisfaction with the length of the appointments" (OR 54.50, CI95% [6.07-489.57]). There was no multicollinearity detected between the predictors. CONCLUSIONS: MHPSS services provided by professionals trained by MoH and WHO, have a high satisfaction rate and are serving the SuTP population in need. The high satisfaction rate is predicted by having MHPSS needs met, which is a key indication of the usefulness of these services.

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Establishing meaningful change thresholds for Clinical Outcome Assessments (COA) is critical for score interpretation. While anchor- and distribution-based statistical methods are well-established, qualitative approaches are less frequently used. This commentary summarizes and expands on a symposium presented at the International Society for Quality of Life Research (ISOQOL) 2017 annual conference, which provided an overview of qualitative methods that can be used to support understanding of meaningful change thresholds on COAs. Further published literature and additional examples from multiple disease areas which have also qualitatively explored the concept of meaningful change are presented.Semi-structured interviews conducted independently from a clinical trial, exit interviews conducted in the context of a clinical trial, focus groups, vignettes and the Delphi panel method can be used to obtain data regarding meaningful change thresholds, with advantages and disadvantages to each method. Semi-structured interviews using concept elicitation (CE) or cognitive debriefing (CD) methods conducted independently from a clinical trial can be an efficient way to gain in-depth patient/caregiver insights. However, there can be challenges with reconciling heterogeneous data across diverse samples and in interpreting the qualitative insights in the context of quantitative score changes. Semi-structured qualitative interviews using CE/CD methods embedded as exit interviews in a clinical trial context with patients/caregivers can provide insights which can augment quantitative findings based on analysis of clinical trial data. However, there are logistical challenges relating to embedding the interviews in a clinical trial.Focus groups and the Delphi panel method can be valuable for reaching consensus regarding meaningful change thresholds; however, for face-to-face interactions, social desirability bias can affect responses. Finally, using vignettes and taking a mixed methods approach can aid in achieving consensus on the minimum score change endorsed by respondents as a meaningful improvement/decrement. However, the approach can be cognitively challenging for participants and reaching a consensus is not guaranteed.Anchor- and distribution- based methods remain critical in establishing responder definitions. Nonetheless, qualitative data has the potential to provide complementary support that a certain level of change on the target COA, which has been statistically supported, is truly important and meaningful for the target population.

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INTRODUCTION: To improve understanding of the diabetic gastroparesis (DGP) patient experience and inform the patient-reported outcome measurement strategy for future trials in DGP, qualitative interviews were conducted with participants in a phase 2 clinical trial of a novel DGP treatment. METHODS: Trial participants were invited to participate in interviews at both the pretreatment visit (PTV) and the end-of-treatment visit (EOTV). The interviews were conducted by experienced qualitative researchers and followed a semistructured interview guide. The PTV interviews focused on patients' DGP symptoms and the impact of DGP on their lives, and the EOTV interviews focused on any symptom changes patients experienced during the trial. RESULTS: Of 90 enrolled trial participants, 78 (86.7%) opted to participate in the interview study. Bloating, stomach fullness, upper abdominal pain, vomiting, constipation, and heartburn or reflux were each reported spontaneously by a majority of the 73 PTV interview participants with evaluable data. These patients commonly reported bloating (n = 20), upper abdominal pain (n = 12), and nausea (n = 11) as their most bothersome DGP symptom. Of 51 EOTV interview participants, 44 (86.3%) reported improvement in at least one DGP symptom either spontaneously or when asked about specific symptoms reported during their PTV interview. CONCLUSION: Bloating, abdominal pain, nausea, constipation, stomach fullness, vomiting, and heartburn were frequently reported by patients as the most bothersome and important-to-treat symptoms. These results support the assessment of these symptoms in future DGP clinical trials, whether for symptom improvement or worsening. FUNDING: Ironwood Pharmaceuticals. TRIAL REGISTRATION: ClinicalTrials.gov identifier NCT02289846.

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PURPOSE: Telotristat ethyl, an oral tryptophan hydroxylase inhibitor, is intended to treat carcinoid syndrome by reducing serotonin production. Telotristat ethyl was evaluated in TELESTAR, a Phase III study for patients who had carcinoid syndrome with at least 4 bowel movements (BMs) per day and who were receiving somatostatin analogue therapy. This interview substudy was conducted to provide insight into the patient experience in TELESTAR and to help understand whether reductions in BM frequency (the primary end point) and other symptoms were clinically meaningful. METHODS: Participating sites were asked to invite (before randomization) all eligible patients to telephone interviews scheduled at the end of the double-blind treatment period. Patients and interviewers were blinded to treatment. FINDINGS: All 35 interviewed participants reported diarrhea and/or excessive BMs at baseline. Patients reported that these symptoms negatively affected emotional, social, physical, and occupational well-being. Prespecified criteria for treatment response (achieving ≥30% reduction in BM frequency for at least 50% of the days) were met by 8 of 26 patients taking telotristat ethyl and 1 of 9 patients taking placebo. All 8 patients taking telotristat ethyl described clinically meaningful reductions in BM frequency and were very satisfied with the ability of the study drug to control their carcinoid syndrome symptoms. Overall, reports of being very satisfied were observed in 12 patients taking telotristat ethyl and 0 taking placebo. IMPLICATIONS: Patient interviews revealed that TELESTAR patients, at baseline, were significantly affected by their high BM frequency. Patient reports of their clinical trial experience supported the significance of the primary end point and clinical responder analysis in TELESTAR, helping identify and understand clinically meaningful change produced by telotristat ethyl.

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OBJECTIVE: We examined the extent to which retrospective proxy reports of well-being mirror participant self-reports at 12-24 months before death and how proxy reports of well-being change over the last year of life. We also explored the role of sociodemographic, cognitive, and health factors of both participants and proxies in moderating such associations. METHOD: We used retrospective proxy ratings obtained in the German Socio-Economic Panel Study (N = 164; age at death = 19-99 years). RESULTS: Results revealed moderate agreement between self- and proxy reports (r = .42), but proxies, on average, overestimated participants' life satisfaction by two thirds of a scale point on a 0-10 scale (or 0.4 SD). Discrepancies were particularly pronounced when proxies themselves reported low life satisfaction. Over the last year of life, participants were viewed to have experienced declines in life satisfaction (-0.54 SD). Declines were stronger for ill participants and proxies who reported low life satisfaction. DISCUSSION: Results qualify theoretical expectations and empirical results based on self-report data that are typically available 1 or 2 years before death. We discuss that retrospective proxy reports in panel surveys can be used as a hypothesis-generating tool to gather insights into late life.

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AIM: In this study, we sought to assess patient awareness and perceptions of an opt-out biorepository. MATERIALS & METHODS: We conducted exit interviews with adult patients and parents of pediatric patients having their blood drawn as part of their clinical care at Vanderbilt University Medical Center (TN, USA). RESULTS: 32.9% of all patients and parents of pediatric patients report having heard of the opt-out biorepository, while 92.4% approve of this research effort based on a brief description. Awareness that leftover blood could be used for research increased among adult patients during the study period, from 34.3 to 50.0%. CONCLUSION: These findings will inform ongoing assessments of the suitability of opt-out and opt-in methods as alternatives to written informed consent for inclusion in a biorepository.

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INTRODUCTION: The Libyan National Health System (LNHS) is debated for the paradox of its performance versus impact. It has poor performance, but the national health statistics are good and competitive. There are concerted efforts to manage health care services and to regain the lost trust. A primary health care (PHC) system that focuses on preventive and promotive care is the core focus of LNHS efforts. OBJECTIVES: To assess patient satisfaction with quality of PHC assessed in terms of (a) customer profile, (b) patient satisfaction, and (c) health care-seeking behavior. METHODOLOGY: A sample of nine health centers and seven polyclinics from various locations in Benghazi, Libya were selected for gathering information by structured face-to-face interviews. A total of 310 beneficiaries were interviewed by using an Arabic translation of the Charleston Psychiatric Outpatient Satisfaction Scale. RESULTS: The beneficiaries appear to be quite satisfied with the quality of services. Geographical zone, marital status of beneficiary, and type of facility are satisfaction-related factors. There are preferences for facilities located within the City Centre over those located elsewhere. There is also an interaction effect of the geographical zone and the type of facility in creating differences in satisfaction. CONCLUSIONS: A customer-friendly facility concept that emphasizes reception, physician interaction, and cordiality shall add value. Polyclinics require more attention as does the Al Slawy area. A few utility services might also be considered.