Journal of Patient-Reported Outcomes最新文献

Background: Mindfulness has been studied as a treatment option for a large range of psychological disorders and is associated with a multitude of positive psychological outcomes. There are now several scales for measuring mindfulness as both a trait and a state. As mindfulness potentially plays a critical role in maintaining physical activity habits, the State Mindfulness Scale for Physical Activity (SMS-PA) was developed to specifically measure mindfulness in a physical activity context. This study aimed to provide validity evidence for a German version of the SMS-PA (henceforth called SMS-PA-G) in a clinical sample.

Methods: We used baseline data from 129 participants in the randomised controlled trial 'New Ways to Cope with Depression'. The sample, which was screened for clinical depression symptoms, completed the SMS-PA-G and several other psychometric scales for capturing state mindfulness, self-efficacy, sense of coherence, anxiety, depression, and physical activity. We conducted reliability and item analyses and ran a confirmatory factor analysis. Also, we assessed construct validity through correlations with the abovementioned scales and through differences in SMS-PA scores between physically active and nonactive participants.

Results: The mean SMS-PA-G score in our sample was 25.3 with a standard deviation of 8.5. The item and reliability analyses provided satisfactory Cronbach's alpha and discriminatory power values. The confirmatory factor analysis showed that physical activity mindfulness can best be described via a bifactor model, with specific mind and body factors and a general mindfulness factor. We found the expected relationships with the attention subscale of state mindfulness, self-efficacy, and sense of coherence but did not find them with the awareness subscale of state mindfulness, depression, and anxiety. As hypothesised, physically active participants exhibited higher SMS-PA-G values than nonactive participants.

Conclusions: The SMS-PA-G is an internally consistent test instrument that captures respondents' general physical activity mindfulness and their attention to mental and bodily events. Whereas validity evidence was generally supportive of the SMS-PA-G, its relationships with other constructs require further investigation.

Trial registration: ISRCTN, ISRCTN12347878. Registered 28 March 2022, https://www.isrctn.com/ISRCTN12347878 .

Professional advisory groups, with patient and public involvement (PPI) representatives, can be used for co-production within research projects. This paper aims to document the benefits and challenges of undertaking stakeholder and participant engagement for an implementation research project within NHS (National Health Service) Wales. A patient focused research project, initiated by clinicians, on the use of patient empowerment tools within standard patient care, used a professional advisory group to identify appropriate tools to use within the research proposal. The professional advisory group was made up of therapists, NHS stakeholders, academics and PPI representatives. A hybrid-meeting style was employed to optimise participation for all members of the group. Benefits of the professional advisory group included increased engagement and ownership of the study due to co-creation, and obtaining important contextual information and lived experience. However, challenges included keeping the discussion on topic due to pre-conceived agendas, pleasing everyone in the room due to varied backgrounds, and technological issues. Future professional advisory groups should consider how to facilitate the full involvement of PPI representatives within the discussion and having a variety of resources to present the topic of discussion. Furthermore, clearly communicating what the purpose and direction of the research project is and how it fits into the wider system, should be carefully considered. Overall, it was recognised that the professional advisory group was of significant value to shape the research proposal. Due to the situational challenges faced by healthcare professional within the NHS and preconceived ideas for solutions, it is hoped that by involving stakeholders early in the process there will be greater acceptance and usability of the research findings.

Background and objective: Individuals with Spinal Cord Injury (SCI) rank regaining sexual function as their top priority. Sexual dissatisfaction often leads to low self-esteem, reduced intimacy, and relationship difficulties and hence forms an essential part of the rehabilitation process. In Nepal, where people generally hold fairly traditional views about sex and sexual health, a sexual outcome measure that is culturally sensitive is a must for addressing sexual problems. No study has yet been carried out to investigate the impact of SCI on sexual attitudes and satisfaction in Nepal. Therefore, this study aims to translate and cross-culturally adapt the Sexual Interest and Satisfaction Scale (SIS) in the Nepali language.

Methods: (1) Forward translation: Three translators translated the English version of SIS into Nepali. (2) Synthesis: The three translated versions were synthesized into a draft version. (3) Expert committee review: The expert committee meetings were held to reach a consensus for a sensible and applicable pre-final version of SIS. (4) Pre-test: The pre-final version of SIS was tested on 25 individuals with SCI. Their opinions and comprehension were documented for each question, and subsequent modifications were made to form the final version of Nepali SIS (SIS-NP). The internal consistency of SIS-NP was calculated using Cronbach's alpha.

Results: The translation and cross-cultural adaptation segregated the questions into two divisions: first, which could be answered by everyone irrespective of their sexual partner status, and second, which could only be answered by those with a sexual partner. No changes were made to the original questions; however, essential clarifications and definitions were added. A total of 66 individuals with SCI participated to evaluate internal consistency with a median duration of injury of 9.5 years (IQR = 9.25). Unmarried participants accounted for 40% (n = 27). Internal consistency was found to be 0.74.

Conclusion: SIS was translated to Nepali, adapting standard recommended guidelines. SIS-NP demonstrated adequate internal consistency to be used in SCI.

Background: As one of the main goals of hip and knee replacements is to improve patients' health-related quality of life, a meaningful evaluation can be achieved by calculating minimal important changes (MICs) for improvements in patient-reported outcome measures (PROMs). This study aims at providing MICs adjusted for patient characteristics for EQ-5D-3L index score improvements after hip and knee replacements. It adds to existing literature by relying on a large national sample and precise clustering algorithms, and by employing a state-of-the-art methodology for the calculation of improved adjusted MICs.

Methodology: A retrospective observational study was conducted using the publicly available National Health Service (NHS) PROMs dataset for primary hip and knee replacements. We used information on 252,331 hip replacements and 279,668 knee replacements from all NHS-funded providers in England between 2013 and 2020. Clusters of patients were created based on pre-operative EQ-VAS, depression status, and sex. Unstratified and stratified estimates for meaningful EQ-5D-3L improvements were obtained through anchor-based predictive MICs corrected for the proportion of improved patients and the reliability of transition ratings.

Results: Stratifying patients showed that MICs varied across subgroups based on pre-operative EQ-VAS, depression status, and sex. MICs were larger for patients with worse pre-operative EQ-VAS scores, while patients with better pre-operative scores required smaller MICs to achieve a meaningful change. We show how after stratification the percentage of patients achieving their stratified MIC was better in line with the actual share of improved patients. Larger MICs were found for patients with depression and for female patients. MICs calculated for knee replacements were consistently lower than those for hip replacements.

Conclusions: Our findings show the importance of adjusting MICs for patients' characteristics and should be considered for quality-related choices and policy initiatives.

Background: This study examines the impact of SARS-CoV-2 (i.e., coronavirus, COVID, COVID-19) using data from a measurement-based care (MBC) system utilized in an outpatient psychiatric clinic providing telemedicine care. A novel Patient Rated Outcome Measure (PROM), the COVID-19 Events Checklist (CEC) was administered in a hospital system based ambulatory clinic beginning April 2020 to track COVID-19-19's impact on patients' mental, emotional, and health-related behaviors during the pandemic. The study (1) provides descriptive CEC data, and (2) compares CEC results with PROMs evaluating anxiety (Generalized Anxiety Disorder-7; GAD-7), depression (Patient Health Questionnaire; PHQ-9), and psychological distress (Brief Adjustment Scale-6; BASE-6).

Methods: This retrospective observational study included patient intake data collected from April 2020 to March 2021. Patient (N = 842) reports on the CEC's five domain questions were aggregated to calculate average reports of COVID-19 related impacts at intake over the initial 12 months of the pandemic. Trends in COVID-19 related impacts were examined, and non-aggregated scores on the PHQ-9, GAD-7, and BASE-6 were compared to primary dichotomous (yes/no) CEC survey questions via Wilcoxon rand sum testing.

Results: Results capture the relationship between COVID-19 exposure, COVID-19- related sequelae and behaviors, and psychological symptom severity. Specifically, Wilcoxon rank-sum tests indicate that social determinants of health (SDOH), negative mental health impacts, and positive coping skill use were significantly associated with psychological symptomatology including overall psychological functioning via the BASE-6, anxiety via the GAD-7, and depressive symptoms via the PHQ-9. Results regarding SDOH were as follows: BASE-6 (w = 44,005, p < 0.001), GAD-7 (w = 44,116, p < 0.001), and PHQ-9 (w = 43,299, p < 0.001). Regarding negative mental health outcomes, the results were: BASE-6 (w = 38,374, p < 0.001), GAD-7 (w = 39,511, p < 0.001), and PHQ-9 (w = 40,154, p < 0.001). As the initial year of the pandemic elapsed, incoming patients demonstrated increased rates of suspected or confirmed exposure to COVID-19, (+2.29%, t = 3.19, p = 0.01), reported fewer negative impacts of COVID-19 on SDOH (-3.53%, t= -2.45, p = 0.034), and less engagement in positive coping strategies (-1.47%, t = -3.14, p = 0.010).

Conclusions: Psychosocial factors related to COVID-19 are discussed, as well as opportunities for further research on the relationship between psychological symptomatology and the impact of COVID-19 on health-related behaviors.

Background: To meaningfully understand outcomes of gender-affirming care, patient-reported outcome measures (PROMs) that are grounded in what matters to individuals seeking care are urgently needed. The objective of this study was to develop a comprehensive PROM to assess outcomes of gender-affirming care in clinical practice, research, and quality initiatives (the GENDER-Q).

Methods: Internationally established guidelines for PROM development were used to create a field test version of the GENDER-Q. In-depth interviews were conducted from December 2018 to March 2020 with youth and adults aged 16 years and older who were seeking or had received gender-affirming care at outpatient clinics providing gender-affirming care located within tertiary care centers or communities in Canada, Denmark, the Netherlands or the US. Data were analyzed and used to develop a conceptual framework and an item pool, which was used to develop preliminary scales. Between February 2021 to November 2021, iterative feedback was sought from clinicians and patient participants on the scales and used to refine or develop new scales. The revised scales were pilot-tested using a crowd-sourcing platform between February 2022 and April 2022.

Results: Data from interviews with 84 participants (aged 34 ± 14 years) resulted in a conceptual framework of the GENDER-Q with 13 domains measuring health-related quality of life, sexual, urination, gender practices, voice, hair, face and neck, body, breasts, genital feminization, chest, genital masculinization, and experience of care. Preliminary versions of 44 scales were developed covering most concepts in the conceptual framework. Iterative feedback was obtained from clinician experts (4 to 37 experts per scale; response rate, 67%) and 7-14 patient participants (depending on scale). All scales were refined, and 15 new scales were developed, resulting in 55 scales in the field test version of the GENDER-Q. In total, 601 transgender and gender diverse (TGD) people (aged 25 ± 6 years) participated in the pilot field test and the data were used to make changes to the field test survey.

Conclusion: The GENDER-Q was developed using extensive input from TGD individuals and clinician experts and represents the most comprehensive set of independently functioning scales that are available to date. An international field test of the GENDER-Q was completed in 2024 and the GENDER-Q is available for use in patient care, clinical research and quality improvement efforts.

Background: Patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) are becoming essential parts of a learning health system, and using these measures is a promising approach for value-based healthcare. However, evidence regarding healthcare professional and patient organizations' knowledge, use and perception of PROMs and PREMs is lacking.

Objectives: The objectives of the study were to: 1- Describe the current knowledge and use of PROMs and PREMs by healthcare professional and patient organizations, 2- Describe the determinants of PROMs and PREMs implementation according to healthcare professional and patient organizations.

Methods: We conducted an environmental scan using semi-structured interviews with representatives from healthcare professional and patient organizations. Interviews were recorded and live coded based on the Franklin framework. We used inductive and deductive thematic analysis to extract information about the main themes addressed during the interview (awareness of PROMs and PREMs, examples of implementation and use of PROMs and PREMs, tools used, vision for future implementation, barriers and facilitators to implementation and the best way to collect PROMs and PREMs data).

Results: 63% of healthcare professional organizations (n = 19) and 41% of patient organizations (n = 9) that were contacted agreed to have a representative interviewed. The representatives from both the healthcare professional and patient organizations acknowledged the importance of assessing patients' experience and outcomes. However, they considered the implementation of PROMs and PREMs tools to be scarce within their organizations, in clinical practice and in the education system. Patient organizations were worried that overuse of PROMs and PREMs could lead to depersonalization of practice. Barriers to implementing PROMs and PREMs included lack of awareness of tools, resistance to change and lack of motivation to complete or explain the questionnaire. Barriers also included factors such as lack of financial, technological and human resources and issues with integration of data and inconsistency of digital platforms.

Conclusions: This environmental scan revealed a lack of awareness of tools by healthcare professional and patient organizations' representatives and limited implementation. Adequate training, technological integration, and demonstration of PROMs and PREMs benefits to foster broader adoption in clinical and organizational settings is dearly needed. Addressing these challenges is essential for enhancing value-based care.

Background: Late-onset Pompe disease (LOPD), a rare autosomal recessive multisystemic disorder, substantially impacts patients' day-to-day activities, outcomes, and health-related quality of life (HRQoL). The PROPEL trial compared cipaglucosidase alfa plus miglustat (cipa+mig) with alglucosidase alfa plus placebo (alg+pbo) in adult patients with LOPD over 52 weeks and showed improved motor and respiratory function in patients switching treatment from standard-of-care enzyme replacement therapy (ERT) to cipa+mig at baseline. This study evaluated the impact of cipa+mig on patient-reported outcomes (PROs), including HRQoL in ERT-experienced patients, using data from PROPEL.

Methods: PROs evaluated included the Subject's Global Impression of Change (SGIC), Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 20a, PROMIS Fatigue Short Form 8a, Rasch-built Pompe-specific Activity (R-PAct), and European Quality of Life-5 Dimensions 5 Response Levels (EQ-5D-5L). The proportions of responders in the cipa+mig arm and the alg+pbo arm were compared via chi-squared or Fisher's exact test (patient-level responder analysis), and least squares (LS) mean differences were calculated for change from baseline at Week 52 of the PRO measures (group-level analysis).

Results: At Week 52, patient-level SGIC responder and group-level SGIC analyses favored cipa+mig compared with alg+pbo across all SGIC domains (e.g. 90 vs. 59% responders in the cipa+mig vs. the alg+pbo group for SGIC ability to move around; P = 0.0005; and LS mean difference 0.385; P = 0.02). Similarly, PROMIS Physical Function and Fatigue domains numerically favored cipa+mig in both analyses (e.g. 50 vs. 40% responders in the cipa+mig vs. alg+pbo arm for PROMIS Physical Function; P = 0.37; and LS mean difference 3.1; P = 0.11). R-PAct for both treatment groups was similar in the patient-level responder analysis, but numerically favored alg+pbo in the group-level analysis (35% responders in both arms; P = 0.95; and LS mean difference -0.8; P = 0.48). Self-care, usual activities, and depression/anxiety domains of EQ-5D-5L numerically favored cipa+mig in both analyses (e.g. 20 vs. 12% responders in the cipa+mig vs. alg+pbo arm for EQ-5D-5L self-care; P = 0.54; and LS mean difference -0.108; P = 0.52).

Conclusions: Overall, switching treatment from alglucosidase alfa to cipa+mig positively impacted PRO measurements during the double-blind period of PROPEL.

Trial registration: NCT03729362; Registration date: November 1, 2018; https://clinicaltrials.gov/study/NCT03729362.

Background: Bowel urgency, the sudden and immediate need to have a bowel movement, is common in patients with ulcerative colitis (UC) and Crohn's disease (CD). While its impact in UC is well established, less is known about its importance in CD. Further, what level of bowel urgency control patients with UC or CD would consider to be acceptable or in remission has not been established. This qualitative study aimed to understand perceptions of bowel urgency and remission in these patients.

Methods: Semi-structured combined concept elicitation and cognitive interviews were conducted to explore how adults with moderate-to-severe UC or CD and current or recent bowel urgency think about the concept of bowel urgency and its remission. The Urgency Numeric Rating Scale (UNRS) was used to examine different levels of bowel urgency severity and to investigate what level of bowel urgency patients would consider as representing remission.

Results: Forty adults (n = 19 UC, n = 21 CD) recruited from six US sites completed the study. Sociodemographic and clinical characteristics were similar in both groups. Both groups reported impacts of bowel urgency on physical, social, professional, and emotional aspects of their lives. Most participants (n = 15 UC, n = 18 CD) reported having experienced one or more incidents of urgency-related fecal incontinence. Most participants considered remission to be a state with a normal or reduced number of bowel movements and no or less frequent bowel urgency, and they noted that remission would increase their ability to engage in daily activities without fear of fecal incontinence. Participants were able to map different levels of severity of bowel urgency to UNRS score ranges (scale: 0-10), and they indicated that a mean UNRS score of 5 (UC) or 4 (CD) would be the highest point on the NRS at which they would still consider their bowel urgency to be in remission.

Conclusions: Bowel urgency is an important issue for patients with either moderate-to-severe UC or CD, and its remission would improve their lives. Further, these patients may still consider bowel urgency to be in remission even at UNRS scores as high as 4 or 5.