Journal of Patient-Reported Outcomes最新文献

Background: Traditionally, surgical success in non-cancer operations, such as elective hernia repair, has been defined by clinical outcomes, including recurrence and complication rates. However, these measures do not capture the primary reason patients seek surgery: relief from symptoms and an improved quality of life. Despite the evident patient-centered goal of non-cancer surgical procedures, research has long prioritized clinical parameters over patient-reported outcomes (PROs). A shift is essential to ensure that surgical success aligns with what truly matters to patients.

Main body: Current surgical research and practice heavily rely on clinical benchmarks that do not adequately reflect patients' lived experiences. For non-cancer conditions, where surgery is elective and aims to enhance quality of life, PROs should serve as the primary indicators of success. Studies across various surgical disciplines have revealed discrepancies between clinical outcome measures and patient satisfaction, highlighting the need for validated, standardized PRO instruments. The Danish AFTERHERNIA Project exemplifies efforts to integrate PROs into surgical evaluations, utilizing digital health infrastructure to systematically capture patient experiences. Additionally, condition-specific tools, such as the Abdominal Hernia-Q, demonstrate the growing recognition of patient-centered metrics. However, the widespread implementation of PRO measurement faces challenges, including resource constraints and the need for clinician training. Addressing these barriers is important for redefining success in non-cancer surgical care.

Conclusions: A paradigm shift in non-cancer surgical evaluation is important. Success should be measured not only by technical outcomes but also by enhancements in patient-reported quality of life and satisfaction. Incorporating PROs into surgical research and practice is both a scientific necessity and an ethical responsibility to ensure that patient needs are addressed. Moving forward, the patient experience must become the foundation for defining surgical success in non-cancer conditions. Ultimately, the primary objective of surgical intervention should be to improve the patient's health status and overall well-being compared to their preoperative condition.

Background: Patient-reported outcome measures (PROMs) are standardized questionnaires for the assessment of health outcomes directly from the patient. A systematic evaluation of the quality of PROMs for acute bronchitis (AB) and acute cough due to (lower) respiratory tract infection or common cold has not yet been performed. The present study aimed to systematically review the quality of available PROMs for AB and acute cough due to (lower) respiratory tract infection or common cold for use in adults and children.

Methodology: Embase, PubMed and Web of Science were searched for studies reporting on the development and/or validation of any PROMs for AB and acute cough due to (lower) respiratory tract infection or common cold. We assessed the methodological quality of each included study, evaluated the quality of measurement properties per PROM and study, and graded the evidence according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology. Based on the overall evidence, we derived recommendations for use of the instruments.

Results: We included three studies on three PROMs for adults measuring disease severity (Acute Bronchitis Severity Score (ABSS); Symptom Diary) and cough-related quality of life (Leicester Cough Questionnaire (LCQ-acute)). For children, we included two studies on two PROMs assessing quality of life (Parent-proxy Children's Acute Cough-specific QoL Questionnaire (PAC-QoL₁₆) and its Short Form (PAC-QoL₆)), and one study on a PROM assessing cold symptoms (Child Cold Symptom Questionnaire (CCSQ)). All instruments were classified as COSMIN category B except for the PAC-QoL₆, indicating that they have the potential to be recommended, but require further validation. The PAC-QoL₆ cannot be recommended for use (COSMIN category C). Content validity is a shortcoming of all identified PROMs.

Conclusions: None of the identified PROMs can be unrestrictedly recommended for use in future research. For adults, the LCQ-acute appears the most suitable tool warranting further validation. Given the intensive work on scale development and testing for PROM design, the CCSQ is promising for use in children. Content validity assessments involving patients and experts are highly recommended for all identified PROMs.

Systematic review registration: OSF ( https://doi.org/10.17605/OSF.IO/3G6CP ).

Background: Recommendations suggest that children need to be ≥ 8 years-old to participate in concept elicitation (CE) and cognitive interviewing (CI) when developing patient reported outcome measures (PROMs). However, these recommendations have not been subject to thorough scrutiny and recent evidence suggests that younger children may be enabled to participate. This study audited current opinions of PROM developers regarding the feasibility of conducting CE and CI research with children.

Methodology: An online survey was developed to capture PROM developers' perspectives, recruited from existing networks (UK PROMs, International Society for Quality of Life Research) and outcomes research groups from English-speaking countries between August-November 2024. Survey questions explored the ages from which developers considered it feasible to include children in CE and CI research, their previous experiences conducting CE/CI research with children, and respondents' background experiences with children. Results were analysed descriptively, and exploratory comparisons were made based on developers' characteristics.

Results: Fifty-eight responses were analysed. The mean youngest ages considered feasible to include children in CE and CI research were 6.66 years and 7.36 years, respectively. The mean youngest ages respondents reported involving children in CE and CI research in practice were 7.67 years and 8.13 years, respectively. Concern that children would have insufficient cognitive and/or linguistic skills was the most often endorsed reason for considering the involvement of younger children to be infeasible. Respondents who had recent parental experience with younger children tended to consider it feasible to include children from younger ages. Those who had conducted CI with children considered it feasible to include children in CI from younger ages. Opposingly, those who had conducted CE with children considered it less feasible to include younger children in CE research.

Conclusions: In-line with established precedent, PROM developers included children from ∼ 8 years-old in CE and CI research, while in principle considering it feasible to include younger ages. Reasons for including (or not including) certain age groups in CE and CI research need critical evaluation and PROM developers may wish to consider ways in which more inclusive opportunities for younger children can be provided.

Aim: The rapid review aimed to analyse current practices and recommendations regarding case-mix adjustment for benchmarking Patient Reported Experience Measures (PREMs) across inpatient health centres. Findings will inform the applicability of case-mix adjustment to PREMs in the Swiss context.

Methods: We searched PubMed, Embase, and Web of Science for studies which met the following criteria: PREMs is a main outcome, study from a European country with a national inpatient PREMs survey, study with adult patients in acute care setting, and evaluates the effect of case-mix adjustment on PREMs. Screening and appraisal were performed by an experienced epidemiologist. A narrative evidence synthesis was undertaken to address the review question, with support of tables to summarize evidence on case-mix variables and statistical methods.

Results: Seven studies (n = 301,833) were included. All supported case-mix adjustment to some extent, though variables used for case mix varied, complicating standardization. Concerns included the risk of masking quality differences. To address this, several authors advocated reporting both adjusted and unadjusted scores. Only one study included language spoken as a case mix variable-a key factor in Switzerland.

Discussion: Case-mix adjustment can enhance fairness in PREM-based benchmarking but must be applied cautiously. For multilingual contexts like Switzerland, local relevance of adjustment variables should be evaluated. A stepwise, transparent approach is recommended to avoid obscuring true performance differences.

Background: Translation teams conducting translation and cultural adaptation find it paramount to properly describe concepts of items within clinical outcome assessments (COAs). To minimize potential threats to linguistic/conceptual equivalence, these teams must understand the concepts a COA intends to measure. This research provides recommendations for the process of developing concept definitions in general, as well as specific recommendations on who should be involved in the process and what a concept definition document should contain.

Methods: The Concept Definition Working Group of the International Society for Quality of Life Research (ISOQOL) Translation and Cultural Adaptation Special Interest Group (TCA-SIG) carried out a literature review and a survey of 20 professionals working in the area of translation and linguistic validation of COAs. The Working Group based recommendations on a combination of survey results and consensus building via online meetings.

Results: Translation teams should develop concept definitions during the preparation phase of the translation process, assuming they do not already exist, and include COA developers and project managers with experience in linguistic validation and conceptual analysis of COAs. The Working Group recommends that concept definitions consist of information related to the therapeutic area being studied, information related to the COA development process, definitions of concepts and domains, as well as elaboration of colloquialisms and acceptable/unacceptable translation alternatives. We recommend centralized distribution of concept definitions.

Conclusions: Concept definitions guide stakeholders and ensure all parties align on the intended meaning of items being translated. While experts have made recommendations for best practices around translation and linguistic validation methodology, they have not clearly delineated the process of defining concepts. The Concept Definition Working Group of the ISOQOL TCA-SIG has therefore developed a set of recommendations for the process of defining concepts. With these recommendations the Working Group intends to standardize the development of concept definitions with the goal of enhancing conceptual equivalence across translations to support data pooling and provide confidence that clinical trial data are comparable, interpretable, and can be relied upon in evaluating clinical benefit of treatments.

Background: Chronic obstructive pulmonary disease (COPD) is characterized by progressive airflow limitation, often associated with declining health status. It is widely believed that the burden of the disease increases over time, leading to continuous suffering in the patient. Understanding the long-term course of patient-reported outcomes (PROs) and the variability in disease progression is crucial for effective management. The purpose of this research was to investigate the long-term trajectories of health status, respiratory symptoms, and dyspnea in COPD patients over a seven-year period and to identify factors associated with different progression patterns.

Methodology: This longitudinal study followed 70 COPD patients for seven years, with evaluations every six months. Participants underwent pulmonary function tests and completed four PRO measures: St. George's Respiratory Questionnaire (SGRQ), COPD Assessment Test (CAT), Evaluating Respiratory Symptoms in COPD (E-RS), and Dyspnoea-12 (D-12). Annual changes were estimated using linear mixed models and linear regression analysis. The patients were categorized into quartiles based on the rate of decline in forced expiratory volume in one second (FEV₁) and changes in PROs.

Results: The group showed a significant deterioration in the FEV₁ and PRO measures. FEV₁ declined by 25 milliliters annually, while SGRQ Total and CAT scores worsened by 1.4 and 0.6 units per year, respectively. However, substantial variability was observed between individuals. The SGRQ Total score worsened significantly after 1.0 year in the 4th quartile, while the 1st quartile showed improvements at 2.0, 2.5, 3.0, 4.0, 6.0 and 6.5 years. Similarly, while the CAT, E-RS Total and D-12 Total scores deteriorated in the fourth quartile, they remained stable or improved in the first quartile.

Conclusions: The progression of COPD varies widely among individuals. Although some patients experience significant declines, others remain stable or even improve for seven years. These findings challenge the belief that COPD inevitably leads to a constant increase in the burden of disease.

Purpose: To identify available patient-reported outcome measures (PROMs) used to evaluate central sensitization (CS) manifestations in chronic pain conditions and evaluate the quality of psychometric properties of those instruments.

Methods: A comprehensive search across multiple electronic databases was conducted for relevant studies following the specification of eligibility criteria and development of key search terms. After screening and full-text review, the methodological quality of studies and psychometric properties of PROMs were assessed and summarized using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist and scoring manual. The results were statistically pooled in a meta-analysis, specifically test-retest reliability, based on data availability and consistency of findings across studies.

Results: A total of fifty-eight studies evaluating eight instruments in adult patients with chronic pain were included. The methodological quality of the included studies was varied. Most identified PROMs have limited evidence regarding their measurement properties. The Central Sensitization Inventory (CSI) received the highest overall ratings for most measurement properties among all the instruments, followed by Pain Sensitivity Questionnaire (PSQ) and Fibromyalgia Survey Questionnaire (FSQ). Based on pooled data from available studies, the test-retest reliability of the CSI was found to be excellent, with an intra-class correlation coefficient (ICC) of 0.93 (95% CI: 0.91-0.95) for overall chronic pain, 0.90 (95% CI: 0.87-0.93) for chronic musculoskeletal pain and 0.93 (95% CI: 0.88-0.99) for chronic neck pain. PSQ also demonstrated excellent test-retest reliability, showing an ICC of 0.86 (95% CI: 0.72-0.99) for chronic pain.

Conclusion: Although not all properties have been studied, the CSI, which received the highest overall ratings, could serve as a reliable PROM assessing CS in chronic pain. More studies should be performed to comprehensively evaluate all measurement properties of all included instruments.

Background: Communicative participation is the most important outcome of speech and language therapy, but there are no measurement instruments for children, adolescents, and young adults. This paper describes the development of MyCommunication-Youth: an item bank to measure self-reported communicative participation in children, adolescents and young adults with various communication disorders.

Aims: 1) To develop a comprehensive, comprehensible item bank for measuring communicative participation in children, adolescents, and young adults with communication problems. 2) To assess the content validity of the item bank in a sample of these groups. 3) To define criteria for the minimal age at which children with communication difficulties can self-report their communicative participation.

Method: Based on a literature review and two concept elicitation studies three initial versions of item pools were developed: one for children, one for adolescents and one for young adults. These pools were pilot tested, using cognitive debriefing interviews, on comprehensibility and comprehensiveness in a diverse group of participants with communication difficulties, whereafter a second version of the item pools were created. Hereafter, the content validity was assessed in the target population and in a group of speech and language therapists.

Results: Initially, three item pools were created for children (58 items), adolescents (78 items), and young adults (84 items). In the pilot test with 33 children adolescents and young adults with communication difficulties, items were revised for comprehensibility, some items were added for comprehensiveness, and some items were deleted because they appeared irrelevant, resulting in updated pools of 50, 69, and 72 items. In the content validity study, cognitive interviews with 27 participants and a focus group with 8 professionals identified additional revisions. Most items were comprehensible, but some were irrelevant for specific subpopulations of communication difficulties. Two new items were added after the input of professionals, whereafter the item bank was found comprehensive.

Conclusions: MyCommunication-Youth is an item bank for measuring communicative participation in children, adolescents and young adults with various communication difficulties. Three versions of the instrument were created: MyCommunication-Children of 49 items, MyCommunication-Adolescents of 70 items and MyCommunication-YoungAdults of 73 items. The item bank is comprehensible, relevant and comprehensive according to the target population and target professionals.

Purpose: The Australian and New Zealand Bariatric Surgery Registry is developing a bariatric-specific patient-reported outcome measure (PROM) to capture patient outcomes. This study aimed to establish an item bank and questionnaire to assess which outcomes are considered the most important by pre- & post-surgical patients and healthcare practitioners.

Methods: Initial qualitative studies were undertaken to provide an in-depth understanding of patients' lived experiences, and a targeted literature search was conducted to identify appropriate PROMs. Items from identified PROMs were pooled and categorised to form the basis of a questionnaire developed to interrogate bariatric patients' and healthcare practitioners' opinions on the importance of the various outcomes.

Results: 1,867 items from 76 instruments were extracted and pooled to form the item bank. Items were categorised and refined to generate an Outcome Importance Questionnaire containing 68 items across 10 domains. 313 participants completed the survey, including 48 pre-surgical patients, 180 post-surgical patients, and 85 Healthcare Practitioners. 52 outcomes (of 68; 76.5%) were prioritised by at least 1 group with 'Overall mental health', 'Co-morbidities', 'Satisfaction with surgery' and 'Satisfaction with quality of life' rated as the most important outcomes.

Conclusion: The item bank and outcome importance questionnaire demonstrated good coverage of patient-reported outcomes considered important to all stakeholders. Initial results identified distinct differences in preference votes by patient and healthcare practitioner groups, with sufficient variation to identify those outcomes considered the most important. Additional rounds of testing, including participant-suggested outcomes and forced-choice questions, will facilitate consensus on the most important outcomes for future inclusion in a Registry-based bariatric-specific PROM.

Background: Chronic hepatitis D (CHD) is the most severe form of viral hepatitis, which results in accelerated progression to cirrhosis and poor prognosis compared with other hepatitis infections, impacting patients' health-related quality of life (HRQoL). To adequately capture patient perspectives of new hepatitis D virus (HDV) treatments in clinical trials, patient-reported outcome (PRO) measures that are valid and assess key concepts relevant to the patient are needed. This study aimed to explore the patient experience of CHD and evaluate the content validity of the Hepatitis Quality of Life Questionnaire (HQLQv2) and the Fatigue Severity Scale (FSS) for use in an HDV population.

Methods: Combined qualitative concept elicitation (CE) and cognitive debriefing (CD) interviews were conducted with 39 patients in Germany, Italy, Spain, and the US with a clinician-confirmed diagnosis of CHD. Participants described their experience of CHD, informing the development of a conceptual model, and then completed the HQLQv2 and FSS using a think-aloud technique to assess understanding, relevance, and comprehensiveness of items, instructions, response scales, and recall periods. Interviews were conducted in the principal language of each country; official translations of the instruments were used, and all patient-facing study documents and the interview guide were translated by certified translators.

Results: The sample included participants with a range of liver fibrosis stages, including 11 with compensated (n = 9) and decompensated (n = 2) cirrhosis. Fatigue, loss of appetite, nausea, joint pain, and pain over the liver were the most frequently reported signs/symptoms. Fatigue was most commonly mentioned and was described as a severe and particularly burdensome symptom, that impacted several aspects of patients' daily lives. Participants reported that CHD impacted their emotional wellbeing (low mood, anxiety), physical functioning (difficulty walking), social functioning (attending social events), activities of daily living (household chores), and work. Participants demonstrated a good understanding of the HQLQv2 and FSS items, instructions, response scales and recall periods, and the concepts assessed were considered relevant to CHD by most participants.

Conclusion: Findings contribute to the understanding of the patient experience of CHD and support content validity of the HQLQv2 and FSS as outcome assessments for use in an HDV population.