Journal of Patient-Reported Outcomes最新文献

Objectives: To translate, cross-culturally adapt and validate the Serbian Ankylosing Spondylitis Quality of Life (ASQoL) questionnaire, e.g. according to the new nomenclature Radiographic-Axial Spondyloarthritis (r-axSpA), and to relate it to disease activity and functional status domains.

Methods: Four stages were accomplished: (1) Bilingual and lay panel for translation and cross-cultural adaptation (2) Cognitive debriefing interviews (assessing the language and cultural equivalence of the concepts used in the Serbian ASQoL translation) for face and content validity (3) Psychometric evaluation: (a) convergent validity by NHP as a comparator scale and (b) known group validity by correlations with disease activity and overall health status and reliability (internal consistency and test-retest reliability) (4) Independent regression analyses for relations between ASQoL and ASDAS, BASDAI, BASFI, Schober's test, respiratory index and SPARCC were used. The statistical program SPSS (version 21; IBM, Armonk, NY, USA) was used.

Results: The bilingual panel made a unified version of the translated documents, a lay panel confirmed the clarity of the questionnaire. Cognitive debriefing interviews with 10 patients evaluated the Serbian ASQoL as clear, precise, easy to complete. The psychometric properties with 60 randomly selected patients showed good convergent validity between ASQoL and NHP domains of pain (r = 0.79), emotional reactions (r = 0.78), physical activity (r = 0.77) and energy (r = 0.75). The internal reliability was 0.95 and 0.91 (1st and 2nd administration), respectively, and the test-retest reliability was 0.84. Regression analyses showed highly significant relationships (p < 0.001) between ASQoL and ASDAS (R²=0.403), BASDAI (R²=0.564) and BASFI (R²=0.444).

Conclusion: The Serbian ASQoL demonstrated good psychometric properties and significant relationships with disease activity and functional status and is recommended for quality of life assessment in Serbian-speaking ankylosing spondylitis (radiographic axial spondyloarthritis) patients, both in clinical practice and clinical research.

Purpose: Zirconia dental implants show excellent biocompatibility and tissue integration, low affinity for plaque, and favorable biomechanical properties. However, these objective measures do not adequately replicate the patient's perception. This systematic review evaluated the evidence on patient-reported outcome (PROs) in zirconia dental implant treatment.

Methods: A systematic literature review was conducted following the PRISMA guidelines, utilizing six electronic databases, and supplemented by a manual search of relevant journals and websites to ensure a thorough and comprehensive screening process. The identified studies were subjected to preidentified inclusion criteria. Only controlled clinical trials published in English were considered without limitations on the year of publication. Data on the study characteristics (follow-up, survival rate (%), implant system, number of implants, and type of treatment), PROMs, level of evidence, and Methodological Index for Nonrandomized Studies (MINORS) Bias Score were extracted.

Results: The initial database and hand search yielded 596 articles; 189 were included in the title and abstract screening after excluding the duplicates. Eighteen articles were selected based on the inclusion criteria, among which six were excluded because they did not match the research question. Thus, the final selection comprised 12 articles. Most PROMs (aesthetics, speaking, comfort, chewing ability, and general satisfaction) at prosthetic delivery revealed significantly improved average scores than those at pretreatment.

Conclusions: Despite the respective limitations of the articles included in this systematic review, patients revealed high satisfaction levels with regard to zirconia dental implants. A high level of heterogeneity was observed among the instruments used for measuring the patient-reported outcomes in patients with zirconia implants, thus highlighting the need to develop specific PROMs in the future.

Purpose: Informal caregivers of people with high grade glioma (HGG) often have high levels of unmet support needs. Routine screening for unmet needs can facilitate appropriate and timely access to supportive care. We aimed to develop a brief screening tool for HGG caregiver unmet needs, based on the Supportive Care Needs Survey-Partners & Caregivers (SCNS-P&C).

Methods: Secondary analysis was performed on responses to the SCNS-P&C from 188 HGG caregivers, who participated in the Care-IS trial. SCNS-P&C items were assessed against four criteria: factor loadings; prevalence; variation in domain score; diagnostic accuracy. Supplementary analysis was conducted at two timepoints (T1 & T2) on the final selected items to identify caregivers indicating no needs on the screening items but reported a need on the original SCNS-P&C, suggesting they would be "missed" by the screening items.

Results: Six items performed best against psychometric criteria, capturing two domains: Cancer impact needs and Information and communication needs. Supplementary analysis showed screening items failed to identify only 7.4% (14/188) of caregivers with other unmet needs at T1 and 11.4% (18/158) at T2. Of those missed at T1, only four were missed again at T2.

Conclusions: We identified six-items for inclusion in a brief screening tool, the SCNS-P&C-6, demonstrating good sensitivity in detecting unmet needs of caregivers of people with HGG. Use of this tool in clinical practice has the potential to improve access to care and the cancer experience for both the caregiver and person with brain tumor.

Background: Patients with Rheumatic and Musculoskeletal Diseases, including axial spondyloarthritis (axSpA), may suffer from stressors like pain and functional impairments leading to limitations in their self-perceived health status. The COping with Rheumatic Stressors (CORS) questionnaire was developed to analyze how patients cope with these stressors. The CORS is currently not available in German.

Objective: First, to translate, cross-culturally adapt and to linguistically validate the original Dutch CORS into German. Second, to test the pre-final German translation through cognitive debriefing in patients with axSpA.

Methodology: The original Dutch CORS underwent a multistep cross-cultural adaptation process, as described by Beaton. It was first independently translated into German by bilingual Dutch-German lay and expert translators. Subsequently, it was translated back from the German version into Dutch. Remaining discrepancies were resolved by a scientific committee, resulting in a pre-final German version. This version was then tested through cognitive debriefing by 10 patients with axSpA across a broad spectrum of sociodemographic backgrounds.

Results: Forward and backward translations of the CORS revealed minor discrepancies, mainly based on the degree of formal versus informal language usage, minor semantic errors or unusual syntax, which led to minor modifications in the wording. Reviewed by the scientific committee, the pre-final consensus German version was linguistically validated by cognitive debriefing by 10 patients with axSpA. Cognitive debriefing confirmed and ensured closest linguistic validity for German in Germany and highest equivalence to the Dutch original version.

Conclusion: The German CORS was shown to have high cross-cultural and face validity for the assessment of coping with rheumatic stressors.

Background: Patient-reported outcomes measures (PROMs) are standardized self-administered tools that assess the patient's opinion on the level of health, quality of life, and disability among other aspects. The objective of this study was to gather information on physical and mental health in patients with major mental illness using PROMs.

Methods: This was an observational, naturalistic, prospective study carried out in adult stabilized outpatients attended at nine Adult Mental Health Centers in Barcelona, Spain. All participants had a confirmed diagnosis of major depression disorder or schizophrenia (DSM-5) and were currently on drug treatment. Participants (n = 508) self-completed a baseline questionnaire for clinical data and PROMs scales (PHQ-19, SF-12, and WHODAS 2.0) at baseline and 9 months thereafter (n = 482).

Results: Mean (SD) age was 50.9 (13.2) years, and 83% of patients lived with their families. Although 93.9% of patients recognized having a mental illness, 15.7% did not know their diagnosis. When asked if they considered that during the last year their treatment had offered some type of improvement, 83.9% answered affirmatively. Patients reported that their degree of adherence to treatment was high (77%) and most of them (80%) believed the medication had a beneficial effect. Depressed patients showed both at baseline and at follow up significantly more depressive symptoms than the group with schizophrenia. In the schizophrenia group, a statistically significant improvement in depressive symptoms was noted at 9 months follow-up. We did not find significant differences within or between groups in quality of life (SF-12 scores) obtained at baseline and after 9 months of follow-up Both at baseline and at follow-up, patients with depression reported a significantly higher degree of disability (WHODAS scores) than those with schizophrenia.

Conclusions: PROMs can be used in real-world conditions to assess severity of disease, quality of life, and disability in major depression and schizophrenia. The present results are relevant for both patients and clinicians.

Purpose: Eosinophilic esophagitis (EoE), a chronic immune-mediated progressive disease, causes dysphagia, food impaction, abdominal pain, vomiting, and heartburn. EoE requires long-term monitoring and can affect quality of life owing to its symptoms and associated emotional and social burden. This study aimed to understand patients' experiences with EoE.

Methods: Semi-structured longitudinal interviews were conducted with patients from MESSINA, a phase 3 placebo-controlled trial evaluating benralizumab for EoE. Interviews were held at two different times to assess the impact of EoE on patients' lives before and during trial participation. Data were analyzed qualitatively to develop detailed patient profiles.

Results: The MESSINA trial was terminated prematurely. Of the 34 patients recruited for the first interview, 15 (44%) completed the second interview and 11 patient profiles were developed. Patients were a demographically diverse group with varying experiences. The primary reported symptom was difficulty swallowing (n = 11), leading to serious consequences like choking and hospitalization (n = 2). Other symptoms included pain when swallowing (n = 7), reflux (n = 6), and stomachache (n = 6). In the second interview, most (n = 9) patients reported moderate improvements in symptoms, while others experienced symptom recurrence or worsening. EoE had a significant negative impact on social and emotional well-being, and professional lives. Trial participation improved emotional well-being for some; however, concerns about the need for ongoing treatment were noted.

Conclusion: This study highlighted emotional and social burdens of EoE. The encouraging feedback on study participation underscores the importance of patient insights in developing holistic management strategies for EoE.

Aims: This study aims to improve the interpretability and clinical utility of the COmprehensive Score for financial Toxicity-Functional Assessment of Chronic Illness Therapy (COST-FACIT) by identifying distinct financial toxicity classes in adults with diabetes.

Methods: Data included a sample of 600 adults with Type 1 or Type 2 diabetes and high A1c. Latent Class Analysis was used to identify subgroups of patients based on COST-FACIT score patterns.

Results: We identified 3 financial toxicity classes (high, medium and low) with strong indicators of membership classification. Multiple indicators of financial stress, maladaptive cost-coping behaviors, more comorbidities, more prescribed medications, more diabetes distress, more depressive symptoms, closer to the federal poverty level, female, having lower educational attainment and being single were all significant predictors of high financial toxicity class membership. A score of 26 on the COST-FACIT was the strongest threshold for sorting high vs. medium/low financial toxicity, with a positive predictive value (PPV) of 76% and negative predictive value (NPV) of 93%.

Conclusion: The COST-FACIT can be used to reliably identify people with diabetes that have high financial toxicity. Integrating this new cut-score into clinical practice may help clinical teams identify people in need of additional support due to financial toxicity.

The Dysphagia Handicap Index (DHI) is commonly utilized for evaluating how dysphagia impacts the quality of life (QoL) of patients across physical, functional, and emotional dimensions. The primary aim of the research was to linguistically validate and culturally adapt the DHI to the Bangla version. A cross-sectional study design was chosen, with Beaton's protocol as the guiding framework for validating and adapting the DHI. It has followed a systematic process of forward translation, participation in expert discussions, and subsequent back translation to obtain a reviewed version. The Bangla version, DHI-Ban, was administered purposefully to 50 dysphagia patients in the Clinical Speech and Language Therapy (SLT) Department of the Centre for the Rehabilitation of the Paralyzed (CRP) and was also administered to 50 healthy individuals for comparison. Of the fifty, eighteen dysphagia subjects were assigned again after two weeks for the retest. The DHI-Ban demonstrated strong internal consistency (Cronbach's α = 0.89) and good test-retest reproducibility (ICC = 0.86). The Spearman test confirmed significant construct validity (p < 0.01), and the Wilcoxon test identified significant differences (p < 0.001) between patients and healthy individuals. Feedback from participants was also taken into account for acceptance and clarity. In conclusion, the adapted DHI-Ban has emerged to be a reliable patient-reported tool for assessing dysphagia in Bangla-speaking individuals. Incorporating the Bangla language framework facilitates its comprehension and effectiveness, further solidifying its reliability.

Objective: A key component of determining that a patient-reported outcome (PRO) measure is fit-for-purpose is to ensure that respondents understand its instructions and items. Any modification to a measure should be evaluated for relevance and understandability. The objective of the study was to assess comprehension of the adapted Urticaria Activity Score (UAS) questionnaire among adolescents aged populations with chronic spontaneous urticaria (CSU) and the modification to UAS question 2 to include patient-friendly terminology "wheals (hives)." A patient guidance document for completing the adapted UAS was also examined.

Methods: A non-interventional, cross-sectional, qualitative study involving hybrid concept elicitation and cognitive interviews was conducted among adults and adolescents with CSU. Eligibility included clinician confirmation of CSU and experience of itch and hives for ≥ 6 weeks. Study participants were recruited from US clinical sites and online CSU patient communities. Telephone interviews were conducted using a semi-structured interview guide. Participants reviewed the UAS and provided their input on the UAS guidance document.

Results: Twenty-two interviews were conducted (seven adolescents and 15 adults; mean age 34 ± 18 years, 64% female, 77% White, 77% non-Hispanic, and 59% moderate to severe CSU symptoms). All participants (n = 22/22, 100%) stated that the adapted UAS was clear and "easy" to understand. For the adapted UAS "wheals (hives)" item, several participants were unfamiliar with the term "wheals," but the term "hives" was well understood by US participants. Most participants reported that it was "easy" or "not difficult" to count and recall the number of hives they had over the past 24 h. Participants found the adapted UAS guidance document "helpful" and "easy to understand" for determining and counting hives in a 24-hour period. Suggestions for improving the guide included adding a picture to aid in counting hives.

Conclusions: Itch and hives are important symptoms of CSU. Results support the content validity of an adapted UAS as a daily measure of severity of itch and hives and provided valuable suggestions for improving its patient guidance, which can be used in future clinical trials involving adults and adolescents ≥ 12 years old with CSU to assess the severity of itch and hives.

Objectives: Systemic lupus erythematosus (SLE) is a chronic autoimmune disease with heterogeneous clinical manifestations which significantly impacts the daily lives of patients. Herein, we aimed to (i) investigate patients' perspectives on and experience with SLE; (ii) identify meaningful aspects of health (MAHs) and concepts of interest (COIs) in SLE that could be evaluated using digital clinical measures (DCMs); and (iii) identify target DCMs for their assessment.

Methods: A mixed-methods, multistep approach was deployed for (i) exploring patients' experience with SLE through a social media listening study and focused group discussions with patients; (ii) mapping patients' experiences to define MAHs and identify COIs measurable using DCMs; (iii) selecting DCMs for the target COIs; and (iv) identifying types of wearable sensors for measuring COIs in the patients.

Results: Six MAHs related to physical behavior and sleep were identified: difficulty in ambulating, lack of energy, inability to perform activities of daily living, difficulty engaging in sustained walking, inability to perform leisure activities and exercise, and lack of restful sleep. Measurable COIs represented walking (fatigue and pain) and sleep (sleep and pain) characteristics. Five and six DCMs related to stepping behavior and sleep quality, respectively, were identified. Several wearable sensors are available to derive DCMs for physical behavior and sleep; however, patients showed a strong preference for a wrist-worn actigraphy sensor.

Conclusion: We identified DCMs for physical behavior and sleep that are relevant and meaningful to patients with SLE, measurable in a real-world environment with wearable sensors, and have the potential to aid personalized patient care.