Journal of Patient-Reported Outcomes最新文献

Purpose: Warm autoimmune hemolytic anemia (wAIHA) is a rare disorder characterized by premature destruction of red blood cells (hemolysis) and fatigue that affects patients' health-related quality of life. This study aimed to identify fatigue concepts important to patients and evaluate the content validity of the Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) in the wAIHA patient population.

Methodology: Two observational studies collected data via 60-minute, individual interviews. Eligible participants were English-speaking, US-resident adults diagnosed with wAIHA by a physician. Both studies gathered data regarding fatigue experiences that mattered to patients and comprehension and relevance of the FACIT-Fatigue; one study also gathered data about patient perspectives on meaningful change in item-level response selection.

Results: Each study enrolled 10 individuals with wAIHA (n = 20). All participants described fatigue as the most prominent or most bothersome symptom, with substantial impacts on daily functioning, family and social life, and emotional well-being. The FACIT-Fatigue was well understood, comprehensive, and relevant to participants' experiences with wAIHA. Data analysis indicated that at least a 3-point change in total score likely reflects a meaningful change in fatigue.

Conclusions: Fatigue is the primary patient-reported wAIHA symptom and important to measure from the patient perspective. Findings demonstrated that wAIHA patients found the FACIT-Fatigue understandable, comprehensive, and relevant, and the data indicated that the instrument can detect a meaningful change in fatigue symptoms and impacts. Study findings support the content validity of the FACIT-Fatigue and contribute to the evidence that the FACIT-Fatigue is fit-for-purpose to evaluate fatigue in adults with wAIHA.

Background: Genetic haemochromatosis (GH) is a long-term genetic condition which results in increased iron absorption into the blood and accumulation of iron into certain organs overtime. Increased absorption and accumulation can be fatal. GH can cause many symptoms including arthritis/joint pain, chronic fatigue, and cognitive difficulties. The aim of this study was to measure quality of life (QoL) in people diagnosed with GH (GH-diagnosed) compared to a healthy sample and identify possible explanations for this.

Methodology: QoL was measured in 535 healthy people and 1039 GH-diagnosed, through completion of the World Health Organisation Quality of Life-100 survey (WHOQOL-100). 985 GH-diagnosed respondents completed a GH-focussed survey, which was developed to get further details of the impact of GH.

Results: Comparison of the WHOQOL-100 overall QoL score between GH-diagnosed and the healthy sample found a significantly lower score in the GH-diagnosed. Physical, psychological, level of independence, and spiritual domains were significantly lower in the GH-diagnosed group. The GH-focussed survey found a high incidence of physical and mental symptoms, and some impact on social and work life. Areas in which participants suggest would improve their QoL included: improved healthcare especially with increased understanding of GH in medical professionals, increased access to appointments, in-person appointments, regular checks for organ damage, more nutrition or dietary advice, and local support groups.

Conclusions: Based on the WHOQOL-100 scores and GH-focussed survey, overall QoL is worse in people diagnosed with GH due to worse physical and psychological symptoms. Improved healthcare may aid in reducing the difference in QoL.

Background: The rise in artificial intelligence tools, especially those competent at language interpretation and translation, enables opportunities to enhance patient-centered care. One might be the ability to rapidly and inexpensively create accurate translations of English language patient-reported outcome measures (PROMs) to facilitate global uptake. Currently, it is unclear if machine translation (MT) tools can produce sufficient translation quality for this purpose.

Methodology: We used Generative Pretrained Transformer (GPT)-4, GPT-3.5, and Google Translate to translate the English versions of selected scales from the Breast-Q and Face-Q, two widely used PROMs assessing outcomes following breast and face reconstructive surgery, respectively. We used MT to forward and back translate the scales from English into Arabic, Vietnamese, Italian, Hungarian, Malay, and Dutch. We compared translation quality using the Metrics for Evaluation of Translation with Explicit Ordering (METEOR). We compared the scores between different translation versions using the Kruskal-Wallis test or analysis of variance as appropriate.

Results: In forward translations, the METEOR scores significantly varied depending on target languages for all MT tools (p < 0.001), with GPT-4 having the highest scores in most languages. We detected significantly different scores among translators for all languages (p < .05), except for Italian (p = 0.59). In backward translations, MTs (GPT-4: 0.81 ± 0.10; GPT-3.5: 0.78 ± 0.12; Google Translate: 0.80 ± 0.06) received higher or compatible scores to human translations (0.76 ± 0.11) for all languages. The differences in backward translation scores by different forward translators were significant for all languages (p < 0.01; except for Italian, p = 0.2). The scores between different languages were also significantly different for all translators (p < 0.001).

Conclusions: Our findings suggest that large language models provide high-quality PROM translations to support human translations to reduce costs. However, substituting human translation with MT is not advisable at the current stage.

Short stature in children aged 0-4 years presents unique physical, social, and emotional challenges that significantly impact health-related quality of life (HRQoL). The QoLISSY 0-4 questionnaire was developed as a Patient Reported Outcome measure (PROMS) to address the absence of an age-specific, condition-focused HRQoL assessment tool for this population. A mixed-method approach was employed to adapt the original QoLISSY questionnaire for children aged 5-18. Qualitative interviews were conducted with 24 parents of children diagnosed with short-stature conditions, including achondroplasia, small for gestational age, growth hormone deficiency, and Silver-Russell Syndrome. Cognitive debriefing sessions and iterative feedback guided the development of new items tailored to the needs of children aged 0-4. Pilot testing involved 20 parents, who evaluated the questionnaire's clarity, relevance, and comprehensiveness. The development process yielded an 8-domain, 55-item questionnaire addressing physical health, social interactions, emotions, coping mechanisms, medical care, future concerns, and parental impact. Cognitive debriefing results indicated high item clarity (100%), relevance (93%), and importance (94%), with parents confirming that the questionnaire effectively captured their child's HRQoL experiences. The QoLISSY 0-4 questionnaire provides a tailored, parent-reported tool for assessing HRQoL in children aged 0-4 with short stature. Its development reflects a rigorous, participant-informed process ensuring its relevance and usability. Future validation studies will explore its cross-cultural applicability and psychometric properties to establish its utility in research and clinical settings.

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.