[Efficacy of Cancer Treatment with Electronic Patient-Reported Outcome Monitoring and Future Expectations].

The Common Terminology Criteria for Adverse Events has been used to evaluate adverse events in oncology. However, it has been shown that medical practitioners who assess adverse events tend to underestimate symptoms more than the patients themselves who experience them, and monitoring of patient-reported outcomes(PROs)is considered essential to achieve patient-centered care. PRO is defined as"any report of a patient's health status obtained directly from the patient, without interpretation by a clinician or anyone else". PROs have been routinely collected in clinical practice using paper and pencil, but they can also be collected more easily and efficiently in the form of electronic PROs(ePROs)using tablets and smartphones. The ePRO system enables remote symptom monitoring and intervention, when necessary, as PROs entered by the patient are shared with the health care provider even when not in person, which not only improves the patient's quality of life but also has been reported to increase overall survival. In Japan, ePROs that can be used in routine medical care are already available, but they have not yet been widely adopted. This article outlines the necessity and usefulness of ePRO monitoring in the treatment of cancer patients, problems and solutions for its introduction and dissemination, and prospects in daily clinical practice.

Clinical variables influencing the perception of fatigue in people with multiple sclerosis: a cross-sectional study using FSIQ-RMS.

BACKGROUND: Physical fatigue is one of the most disabling symptoms in people with Multiple Sclerosis (PwMS). Several factors might influence the development of fatigue, such as gender, education, body mass index (BMI), Expanded Disability Status Scale (EDSS), disease duration, working status (Ws), physiotherapy (Ph), and disease-modifying therapies (DMTs). Fatigue Symptoms and Impacts Questionnaire-Relapsing Multiple Sclerosis (FSIQ-RMS) is a patient-reported outcome (PRO) that allows one to define the impact of fatigue in PwMS clearly. This study aimed to assess fatigue impact on PwMS by using FSIQ-RMS. METHODS: The participants were enrolled from May to July 2021 in MS Centers of Sant'Andrea Hospital and Policlinico Umberto I Hospital in Rome. Fatigue was evaluated using the FSIQ-RMS, validated, and culturally adapted in Italian. Clinical and demographic data were collected at the same time. RESULTS: We enrolled 178 PwMS [Female 74.16%; RMS 82.58%, SPMS 17.52%]. FSIQ-RMS scores were significantly correlated with EDSS (p-value < 0.01). Analysis of variance between means showed a statistically significant difference between the BMI groups at the 24hours_FSIQ-RMS score and the 7days_FSIQ-RMS score (p < 0.01), with the lower BMI group having the highest scores. Furthermore, perceived fatigue significantly improved both in subjects performing Ph (p < 0.05) and in those who actively work (p < 0.01). CONCLUSIONS: The use of FSIQ-RMS in a real-world setting confirmed that underweight and high levels of disability are closely related to fatigue. In addition, Ph and active Ws are strongly correlated with fatigue in PwMS.

Development and validation of a patient reported experience measure for experimental cancer medicines (PREM-ECM) and their carers (PREM-ECM-Carer).

BACKGROUND: Our aim was to develop a validated Patient Reported Experience Measure (PREM) to capture patient and carer experience during participation in experimental cancer medicine trials (ECM): called PREM-ECM. METHODS: Mixed method design, consisting of four stages. Questionnaire items were produced for both patients and carers using interviews, focus groups, and cognitive interviews with patients and carers separately. For both patient and carer PREMs, a cross-sectional questionnaire study was conducted to identify final items for inclusion using hierarchical item reduction and Rasch analysis. Questionnaire validity and reliability were assessed, including administration feasibility. RESULTS: Initial interview participants suggested the need for three PREMs, two specific to patients: (i) a 'prior' questionnaire that captured experiences of trial introduction, screening, consenting, and early trial experience (< 6 weeks post consent); and (ii) 'on-trial' that captured experiences of ongoing consent and trial participation; and (iii) a PREM specific for carers. The draft 25-item 'prior' questionnaire was completed by 162 patients and 162 patients completed the draft 35-item 'on-trial' questionnaire. Hierarchical and Rasch analysis produced a 14-item 'prior' list and a 15-item list for 'on-trial'. Both patient PREM's demonstrated a good fit to the Rasch model following Bonferroni correction (X2p = 0.008). The carer 34-draft item questionnaire was completed by 102 participants. Hierarchical and Rasch analysis produced a 13-item list for PREM-ECM-Carer, with good fit to the Rasch model ( X2p = 0.62). The pilot testing demonstrated the feasibility of all the PREMs in capturing patient and caregiver experiences in routine clinical settings. CONCLUSIONS: The three PREM-ECM questionnaires will be the first validated experience measures for ECM trial patients and their carers. These questionnaires may be used to assess patients' and their carers' experiences of ECM and enable robust comparisons across cancer trial units highlighting areas for service improvement.

Moral distress measurement in animal care workers: a systematic review.

OBJECTIVES: The mental health of veterinary and other animal health professionals is significantly impacted by the psychological stressors they encounter, such as euthanasia, witnessing animal suffering and moral distress. Moral distress, initially identified in nursing, arises when individuals are aware of the right action but are hindered by institutional constraints. We aimed to review existing research on moral distress scales among animal care workers by focusing on the identification and psychometric validity of its measurement. DESIGN: Two-step systematic review. First, we identified all moral distress scales used in animal care research in the eligible original studies. Second, we evaluated their psychometric validity, emphasising content validity, which is a critical aspect of patient-reported outcome measures (PROMs). This evaluation adhered to the Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN). The results were reported according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses. DATA SOURCES: PubMed, EMBASE and PsycINFO to search for eligible studies published between January 1984 and April 2023. ELIGIBILITY CRITERIA FOR SELECTING STUDIES: We included original (primary) studies that (1) were conducted in animal care workers; (2) describing either the development of a moral distress scale, or validation of a moral distress scale in its original or modified version, to assess at least one of the psychometric properties mentioned in COSMIN guidelines. DATA EXTRACTION AND SYNTHESIS: Two independent reviewers used standardised methods to search, screen and code included studies. We considered the following information relevant for extraction: study reference, name and reference of the moral distress scale used, psychometric properties assessed and methods and results of their assessments. The collected information was then summarised in a narrative synthesis. RESULTS: The review identified only one PROM specifically adapted for veterinary contexts: the Measure of Moral Distress for Animal Professionals (MMD-AP), derived from the Measure of Moral Distress for Healthcare Professionals (MMD-HP). Both MMD-HP and MMD-AP were evaluated for the quality of development and content validity. The development quality of both measures was deemed doubtful. According to COSMIN, MMD-HP's content validity was rated as sufficient, whereas MMD-AP's was inconsistent. However, the evidence quality for both PROMs was rated low. CONCLUSION: This is the first systematic review focused on moral distress measurement in animal care workers. It shows that moral distress is rarely measured using standardised and evidence-based methods and that such methods should be developed and validated in the context of animal care. PROSPERO REGISTRATION NUMBER: CRD42023422259.

Impact on diabetes control and patient-reported outcomes of a newer implantable continuous glucose monitoring system (Eversense® CGM System): a single-centre retro- and prospective observational study.

AIMS OF THE STUDY: The Eversense® CGM System is the first and only continuous glucose monitoring system (CGMS) that uses a fully subcutaneous implanted sensor. This study aimed to evaluate effectiveness, safety and patient-reported outcomes in patients using the Eversense® CGM System in a realistic clinical setting, assessed at a single Swiss diabetes centre (Luzerner Kantonsspital) with prolonged follow-up. METHODS: This was a prospective and retrospective observational study that included patients with type 1 diabetes mellitus in whom at least one Eversense® glucose sensor was implanted between 2017 and 2022. The primary endpoint was the change in HbA1c levels from the baseline (before implantation of the sensor) to 6 ± 2 and 12 ± 2 months and the last follow-up (newest available value) after implantation. The secondary outcome measures were the number of premature sensor breakdowns, adverse events related to the implantation procedure (infection, bleeding, difficulties with implantation or explantation) and patient-related outcomes (assessed with a questionnaire). RESULTS: A total of 33 patients participated in this study. The median follow-up time was 50 (IQR 22.3-58.5) months. In total, 178 sensor implantations were performed. Valid HbA1c results were available for 26 participants. Compared to the baseline values, HbA1c levels at 6 and 12 months and the last follow-up changed by -0.25%, -0.45 and -0.2 (p = 0.278, 0.308 and 0.296, respectively). We recorded 16 (9%) premature sensor breakdowns, all occurring between 2019 and 2020. Apart from one late-onset infection and four complicated sensor removals, no major complications were assessed. The results of the questionnaire showed a subjective improvement in hypoglycaemia rates, a better perception of hypoglycaemia and the impression of better diabetes management. Common issues with the device reported by the patients were technical errors (connection problems) and problems with the removal procedure. CONCLUSIONS: The use of the Eversense® CGM System resulted in changes in HbA1c of between -0.2% and -0.45%. The rate of premature sensor breakdown was low. Major complications following sensor implantation or removal were absent, apart from one case of infection and four cases of complicated removal. Patient-reported outcomes with the Eversense® CGM System showed a subjective positive impact on hypoglycaemia rates, greater confidence in managing hypoglycaemia and diabetes in general, and easy handling of the transmitter and mobile app. Technical issues must be considered but are nowadays, with the use of the newest sensor generation, very rare.

Linking genetic counseling communication skills to patient outcomes and experiences using a community-engagement and provider-engagement approach: research protocol for the GC-PRO mixed methods sequential explanatory study.

INTRODUCTION: In over 50 years since the genetic counseling (GC) profession began, a systematic study of GC communication skills and patient-reported outcomes in actual sessions across multiple clinical specialties has never been conducted. To optimize GC quality and improve efficiency of care, the field must first be able to comprehensively measure GC skills and determine which skills are most critical to achieving positive patient experiences and outcomes. This study aims to characterise GC communication skills using a novel and pragmatic measure and link variations in communication skills to patient-reported outcomes, across clinical specialties and with patients from diverse backgrounds in the USA. Our community-engagement and provider-engagement approach is crucial to develop recommendations for quality, culturally informed GC care, which are greatly needed to improve GC practice. METHODS AND ANALYSIS: A mixed methods, sequential explanatory design will be used to collect and analyze: audio-recorded GC sessions in cancer, cardiac, and prenatal/reproductive genetic indications; pre-visit and post-visit quantitative surveys capturing patient experiences and outcomes and post-visit qualitative interview data. A novel, practical checklist will measure GC communication skills. Coincidence analysis will identify patterns of GC skills that are consistent with high scores on patient-reported measures. Two-level, multilevel models will be used to evaluate how GC communication skills and other session/patient characteristics predict patient-reported outcomes. Four community advisory boards (CABs) and a genetic counselor advisory board will inform the study design and analysis. ETHICS AND DISSEMINATION: This study has been approved by the single Institutional Review Board of the University of Minnesota. This research poses no greater than minimal risk to participants. Results from this study will be shared through national and international conferences and through community-based dissemination as guided by the study's CABs. A lay summary will also be disseminated to all participants.

Influence of Role Expectancy on Patient-Reported Outcomes Among Patients With Migraine: A Randomized Clinical Trial.

Importance: It is usually assumed that an individual's classification as a patient or a healthy person is determined by the presence or absence of disease, but little is known about whether the mere awareness of being a patient or a healthy control can play an important role for reporting outcomes. Objective: To investigate whether assignment to the role of a patient or a healthy control has an effect on patient-reported outcomes. Design, Setting, and Participants: This single center, double-blind, 3-group randomized clinical trial included consecutive patients from a tertiary headache clinic based at a single center in Germany who were invited to participate between October 2019 and June 2023. Statistical analysis was performed from January to March 2024. Intervention: Patients with migraine were randomized into 2 groups. The first group was told that this study was centered on migraine symptoms, whereas the second group was told that healthy controls were being sought for a study about patients with vertigo. A third group of age- and sex-matched headache-free participants served as controls. All participants viewed 2 standardized roller coaster videos and provided ratings of their perceived levels of motion sickness and dizziness. Main Outcomes and Measures: The primary outcome was self-reported vestibular symptoms. Secondary outcomes included differences in motion sickness, headache burden, and migraine disability. Outcomes were assessed using standardized questionnaires. Results: The final sample included 366 participants: 122 patients with migraine assigned the role of patient (MP) (migraine as patient): mean [SD] age, 37.56 [12.93] years; 105 [86.1%] female), 122 patients with migraine assigned the role of healthy participant (MH) (migraine as healthy): mean [SD] age, 37.03 [13.10] years; 107 [87.7%] female), and 122 headache-free controls (HC): mean [SD] age, 37.55 [11.56] years; 100 [82.0%] female). The assigned role of the individuals with migraine (MP vs MH) had a significant effect on self-disclosure of (1) estimation that symptoms (dizziness) will occur under specific conditions (self-reported vestibular symptoms: 79 MP [64.8%]; 29 MH [23.8%]; 9 HC [7.4%]; P < .001), (2) the increase of such symptoms (dizziness) after viewing the roller coaster video, and (3) the reported frequency (median [IQR] self-reported monthly headache days for MP: 7 [4-15] days; for MH: 5 [2-10] days; P = .008) and severity (median [IQR] migraine disability assessment score for MP: 35 [20-64] points; for MH: 25 [11-47] points; P = .005) of migraine symptoms. Statistically significant changes were also found for self-reported headache frequency and disability caused by migraine. Conclusions and Relevance: This randomized clinical trial found an effect of expectations regarding the role of a patient with respect to clinical and study outcomes. These findings suggest that role expectations should be taken into account when, for example, invasive treatments are discussed. Trial Registration: ClinicalTrials.gov Identifier: NCT06322550.

Quality-of-Life Assessment in Pediatric Advanced Cancer: Development of the Patient-Reported Outcome Measure Advance QoL.

A recent measure was developed to assess the Quality of Life (QoL) of young people with advanced cancer and is available for parents and professionals (Advance QoL). The present study aimed to elaborate self-reported versions for children and adolescents with advanced cancer. We adopted a four-phase research plan: (1) to elaborate the Advance QoL questionnaire for youth (8-12 and 13-18 years old) with a team of young research partners; (2) to evaluate the understandability of these versions in a sample of 12 young patients from the target population using cognitive interviews; (3) to assess social validity in the same group using a questionnaire and the content validity index (CVI); and (4) to refine the questionnaires according to these results. Four major themes were identified: (1) issues affecting the understanding of the tool; (2) issues that did not affect the understanding of the tool; (3) modifications to improve the tool; and (4) positive features of the tool. Advance QoL was well received, and feedback was positive. Adjustments were made according to young people's comments and two self-reported versions are now available. It is essential to measure the key domains of QoL in advanced cancer. Advance QoL self-report versions will help target the specific needs of young people with this condition and their families.

Exploring patients' experience using PROMs within routine post-discharge follow-up assessment after stroke: a mixed methods approach.

BACKGROUND: Patient Reported Outcomes Measures (PROMs) are being used increasingly to measure health problems in stroke clinical practice. However, the implementation of these PROMs in routine stroke care is still in its infancy. To understand the value of PROMs used in ischemic stroke care, we explored the patients' experience with PROMs and with the consultation at routine post-discharge follow-up after stroke. METHODS: In this prospective mixed methods study, patients with ischemic stroke completed an evaluation questionnaire about the use of PROMs and about their consultation in two Dutch hospitals. Additionally, telephone interviews were held to gain in-depth information about their experience with PROMs. RESULTS: In total, 63 patients completed the evaluation questionnaire of which 10 patients were also interviewed. Most patients (82.2-96.6%) found completing the PROMs to be feasible and relevant. Half the patients (49.2-51.6%) considered the PROMs useful for the consultation and most patients (87.3-96.8%) reported the consultation as a positive experience. Completing the PROMs provided 51.6% of the patients with insight into their stroke-related problems. Almost 75% of the patients found the PROMs useful in giving the healthcare provider greater insight, and 60% reported discussing the PROM results during the consultation. Interviewed patients reported the added value of PROMs, particularly when arranging further care, in gaining a broader insight into the problems, and in ensuring all important topics were discussed during the consultation. CONCLUSIONS: Completing PROMs appears to be feasible for patients with stroke attending post-discharge consultation; the vast majority of patients experienced added value for themselves or the healthcare provider. We recommend that healthcare providers discuss the PROM results with their patients to improve the value of PROMs for the patient. This could also improve the willingness to complete PROMs in the future.

Validation of the Italian version of a patient-reported outcome measure for Hereditary Spastic Paraplegia.

BACKGROUND AND AIM: Patient-reported outcome measures (PROMs) are recognized as valuable measures in the clinical setting. In 2018 we developed the Italian version of the "Hereditary Spastic Paraplegia-Self Notion and Perception Questionnaire" (HSP-SNAP), a disease-specific questionnaire that collects personal perception on motor symptoms related to HSP such as stiffness, weakness, imbalance, reduced endurance, fatigue and pain. In this study our primary aim was to assess the questionnaire validity and reliability. Our secondary aim was to characterize the symptoms "perceived" by patients with HSP and compare them with those "perceived" by age-matched healthy subjects. METHODS: The 12-item HSP-SNAP questionnaire was submitted to 20 external judges for comprehensibility and to 15 external judges for content validity assessment. We recruited 40 subjects with HSP and asked them to fill the questionnaire twice for test-retest procedure. They also completed the Medical Outcome Survey Short Form (SF-36) and were evaluated by the Spastic Paraplegia Rating Scale and the Six-Minute Walk Test. We also recruited 44 healthy subjects who completed the HSP-SNAP once to test score variability. RESULTS: The HSP-SNAP content validity index was high (0.8±0.1) and the test-retest analysis showed high reliability (ICC = 0.94). The mean HSP-SNAP score (score range 0-48) of the HSP group was 22.2±7.8, which was significantly lower than healthy subjects (43.1±6.3). The most commonly perceived symptom was stiffness, followed by weakness and imbalance. CONCLUSION: Although HSP-SNAP does not investigate non-motor symptoms and we validated only its Italian version, it showed good validity and reliability and it could be used in combination with other objective outcome measures for clinical purposes or as endpoints for future clinical rehabilitation studies. TRIAL REGISTRATION: Trial Registration: ClinicalTrial.gov, NCT04256681. Registered 3 February 2020.

Development and validation of the patient-reported outcome for older people living with HIV/AIDS in China (PROHIV-OLD).

BACKGROUND: The involvement of quality of life as the UNAIDS fourth 90 target to monitor the global HIV response highlighted the development of patient-reported outcome (PRO) measures to help address the holistic needs of people living with HIV/AIDS (PLWHA) beyond viral suppression. This study developed and tested preliminary measurement properties of a new patient-reported outcome (PROHIV-OLD) measure designed specifically to capture influences of HIV on patients aged 50 and older in China. METHODS: Ninety-three older people living with HIV/AIDS (PLWHA) were interviewed to solicit items and two rounds of patient cognitive interviews were conducted to modify the content and wording of the initial items. A validation study was then conducted to refine the initial instrument and evaluate measurement properties. Patients were recruited between February 2021 and November 2021, and followed six months later after the first investigation. Classical test theory (CTT) and item response theory (IRT) were used to select items using the baseline data. The follow-up data were used to evaluate the measurement properties of the final instrument. RESULTS: A total of 600 patients were recruited at the baseline. Of the 485 patients who completed the follow-up investigation, 483 were included in the validation sample. The final scale of PROHIV-OLD contained 25 items describing five dimensions (physical symptoms, mental status, illness perception, family relationship, and treatment). All the PROHIV-OLD dimensions had satisfactory reliability with Cronbach's alpha coefficient, McDonald's ω, and composite reliability of each dimension being all higher than 0.85. Most dimensions met the test-retest reliability standard except for the physical symptoms dimension (ICC = 0.64). Confirmatory factor analysis supported the structural validity of the final scale, and the model fit index satisfied the criterion. The correlations between dimensions of PROHIV-OLD and MOS-HIV met hypotheses in general. Significant differences on scores of the PROHIV-OLD were found between demographic and clinical subgroups, supporting known-groups validity. CONCLUSIONS: The PROHIV-OLD was found to have good feasibility, reliability and validity for evaluating health outcome of Chinese older PLWHA. Other measurement properties such as responsiveness and interpretability will be further examined.

Patient-reported outcome measures for primary hyperparathyroidism: a systematic review of measurement properties.

BACKGROUND: The quality of patient-reported outcome measures (PROMs) used to assess the outcomes of primary hyperparathyroidism (PHPT), a common endocrine disorder that can negatively affect patients' health-related quality of life due to chronic symptoms, has not been rigorously examined. This systematic review aimed to summarize and evaluate evidence on the measurement properties of PROMs used in adult patients with PHPT, and to provide recommendations for appropriate measure selection. METHODS: After PROSPERO registration (CRD42023438287), Medline, EMBASE, CINAHL Complete, Web of Science, PsycINFO, and Cochrane Trials were searched for full-text articles in English investigating PROM development, pilot studies, or evaluation of at least one PROM measurement property in adult patients with any clinical form of PHPT. Two reviewers independently identified studies for inclusion and conducted the review following the Consensus-Based Standards for the Selection of Health Measurement Instruments (COSMIN) Methodology to assess risk of bias, evaluate the quality of measurement properties, and grade the certainty of evidence. RESULTS: From 4989 records, nine PROM development or validation studies were identified for three PROMs: the SF-36, PAS, and PHPQoL. Though the PAS demonstrated sufficient test-retest reliability and convergent validity, and the PHPQoL sufficient test-retest reliability, convergent validity, and responsiveness, the certainty of evidence was low-to-very low due to risk of bias. All three PROMs lacked sufficient evidence for content validity in patients with PHPT. CONCLUSIONS: Based upon the available evidence, the SF-36, PAS, and PHPQoL cannot currently be recommended for use in research or clinical care, raising important questions about the conclusions of studies using these PROMs. Further validation studies or the development of more relevant PROMs with strong measurement properties for this patient population are needed.

Service Evaluation of MyChristie-MyHealth, an Electronic Patient-Reported Outcome Measure Integrated Into Clinical Cancer Care.

PURPOSE: Electronic patient-reported outcome measures (ePROMs) are digitalized health questionnaires used to gauge patients' subjective experience of health and disease. They are becoming prevalent in cancer care and have been linked to a host of benefits including improved survival. MyChristie-MyHealth is the ePROM established at the Christie NHS Foundation Trust in 2019. We conducted an evaluation of this service to understand user experiences, as well as strategies to improve its functioning. METHODS: Data collection: Patients who had opted never to complete MyChristie-MyHealth (n = 87), and those who had completed at least one (n = 87) were identified. Demographic data included age, sex, ethnicity, postcode, diagnosis, treatment intent, and trial status. Semistructured interviews were held with noncompleters (n = 30) and completers (n = 31) of MyChristie-MyHealth, as well as clinician users (n = 6), covering themes such as accessibility, acceptability and usefulness, and open discourse on ways in which the service could be improved. RESULTS: Noncompleters of MyChristie-MyHealth were older (median age 72 v 66 years, P = .005), receiving treatment with curative rather than palliative intent (odds ratio [OR], 1.45; P = .045), and less likely to be enrolled on a clinical trial (OR, 0.531; P = .011). They were less likely to own a smartphone (33% v 97%) or have reliable Internet access (45% v 100%). Satisfaction with MyChristie-MyHealth was high in both groups: 93% (n = 29) of completers and 87% (n = 26) noncompleters felt generally happy to complete. Completers of MyChristie-MyHealth wanted their results to be acknowledged by their clinicians. Clinicians wanted results to be displayed in a more user-friendly way. CONCLUSION: We have broadly characterized noncompleters of the Christie ePROM to identify those in need of extra support or encouragement in the clinic. An action plan resulting from this review has been compiled and will inform the future development of MyChristie-MyHealth.

Impact of omitting annual reviews for COPD on patient reported care quality- outcomes from the Asthma+Lung COPD patient passport.

BACKGROUND: Regular clinical reviews of people with COPD provide an opportunity to optimise management and are recommended in national and international guidelines. However, there are limited data about the relationship between having an annual review and other aspects of care quality, which might influence decision-making by healthcare professionals and commissioners. METHOD: Using data from 74 827 people with COPD completing the Asthma+Lung UK COPD Patient Passport, between 2014 and 2022, we conducted adjusted logistic regression (adjusting for year) and compared receipt of key items of care between those reporting that they had had an annual review (65.3%) and those who did not (34.7%). To further capture patient experience, we also analysed 4228 free-text responses to the 2021 Asthma+Lung UK annual COPD survey to the question 'What is the one thing that could improve your COPD care?' RESULTS: We found that the absence of an annual review was associated with significantly worse COPD care across all domains studied; in particular, inhaler training (yes: 80.8% vs no: 38.4%, adjusted OR (AOR): 8.18, 95% CI (7.89 to 8.47), having a written care plan (89.6% vs 56.9%, AOR 6.68 (95% CI 6.35 to 7.05) and medication knowledge (72.6% vs 33.6%, AOR 5.73 (95% CI 5.51 to 5.96). Thematic analysis of the 2021 COPD survey responses identified three areas to improve care: (1) access and support from healthcare services, (2) improved treatment effectiveness and (3) interaction between COPD and the social environment. DISCUSSION: Failure to deliver annual COPD reviews is associated with worse patient-reported experience of care quality. In parallel, people with COPD express a desire for greater support and access to healthcare services.

Patient-reported experience with Fabry disease and its management in the real-world setting: results from a double-blind, cross-sectional survey of 280 respondents.

BACKGROUND: Fabry disease (FD) is a rare X-linked lysosomal storage disorder with a heterogeneous clinical presentation. Patients with FD may exhibit early signs/symptoms including neuropathic pain, gastrointestinal complaints, and dermatologic manifestations. FD may ultimately progress to renal, neurologic, and cardiac dysfunction. Current treatments for FD have significantly improved the management and outcomes for patients with FD, but important clinical and convenience limitations still exist. METHODS: To illuminate the impact of FD on daily life from the patient's perspective, we asked adult patients (≥ 18 years old) with FD in the United States and Canada to complete a 33-question online survey to assess patient-reported disease severity, management, and treatment outcomes. RESULTS: A total of 280 respondents with FD completed the survey; they had a mean age of 47 years, and 68% (191/280) were women. Most were currently receiving FD treatment (84%, 234/280) with enzyme replacement therapy (ERT) (89%, 208/234) or chaperone therapy (11%, 26/234). Common symptoms included low energy/fatigue (72%, 201/280), tingling (62%, 174/280) or pain in the hands/feet (60%, 168/280), ringing in ears/hearing loss (54%, 151/280), general body pains/pain crises (51%, 143/280), and abdominal/stomach pain (50%, 140/280). More than half (51%, 144/280) of respondents reported their symptoms as bothersome (38%, 106/280) or difficult to control (14%, 38/280). Temporary symptom worsening between infusions was reported by about half of respondents: 51% (108/211) currently receiving ERT and 48% (14/29) previously receiving ERT. Only 48% (59/122) of respondents reported their symptom worsening to their physician. Of those who reported it, 41% (24/59) said that their physician prescribed medication to manage their symptoms or changed their treatment regimen. CONCLUSIONS: Our analysis highlights the gap between current standard-of-care in disease monitoring and patient perception of disease progression among patients with FD. This information may be helpful for healthcare providers and drug developers seeking to improve the care of patients with FD by addressing unmet needs of high relevance.

Developing feasible and acceptable strategies for integrating the use of patient-reported outcome measures (PROMs) in gender-affirming care: An implementation study.

OBJECTIVE: Use CFIR guidance to create comprehensive, evidence-based, feasible, and acceptable gender-affirming care PROM implementation strategies. DESIGN, SETTING, PARTICIPANTS: A 3-Phase participatory process was followed to design feasible and acceptable strategies for integrating PROMs in gender-affirming care. In Phase 1, barriers and enablers to PROM implementation for gender-affirming care were identified from a previous systematic review and our prior qualitative study. We used the CFIR-ERIC tool to match previously identified barriers and enablers with expert-endorsed implementation strategies. In Phase 2, implementation strategy outputs from CFIR-ERIC were organised according to cumulative percentage value. In Phase 3, gender-affirming care PROM implementation strategies underwent iterative refinement based on rounds of stakeholder feedback with seven patient and public partners and a gender-affirming healthcare professional. RESULTS: The systematic review and qualitative study identified barriers and enablers to PROM implementation spanning all five CFIR domains, and 30 CFIR constructs. The top healthcare professional-relevant strategies to PROM implementation from the CFIR-ERIC output include: identifying and preparing implementation champions, collecting feedback on PROM implementation, and capturing and sharing local knowledge between clinics on implementation. Top patient-relevant strategies include: having educational material on PROMs, ensuring adaptability of PROMs, and collaborating with key local organisations who may be able to support patients. CONCLUSIONS: This study developed evidence-based, feasible, and acceptable strategies for integrating PROMs in gender-affirming care, representing evidence from a systematic review of 286 international articles, a qualitative study of 24 gender-affirming care patients and healthcare professionals, and iteration from 7 patient and public partners and a gender-affirming healthcare professional. The finalised strategies include patient- and healthcare professional-relevant strategies for implementing PROMs in gender-affirming care. Clinicians and researchers can select and tailor implementation strategies best applying to their gender-affirming care setting.

Patient-Reported Outcome Measures (PROMs) with Refractive and Diffractive Extended Depth of Focus (EDOF) Intraocular Lenses.

BACKGROUND: Extended depth of focus intraocular lenses (EDOF-IOLs) provide unaided far- and mid-range vision. Refractive IOLs, in contrast to diffractive designs, are associated with a lower depth of focus and absence of dysphotopsia. The aim of this study was to assess spectacle independence for far-range, mid-range, and near-vision activities in patients after implantation of refractive or diffractive EDOF-IOLs using patient-reported outcome measures (PROMs) in a real-world setting. PATIENTS AND METHODS: In 2021 and 2022, all patients in a single center referred for bilateral cataract surgery were assigned to 7 experienced cataract surgeons who either bilaterally implanted only a diffractive EDOF-IOL (Carl Zeiss Meditec AG, AT LARA Jena, Germany, three surgeons) or refractive EDOF-IOL (Johnson & Johnson Vision Inc., Tecnis Eyhance Irvine, California, USA, four surgeons) at the surgeon's discretion, with the aim of bilateral emmetropia. Six months after both cataract surgeries, all patients were contacted by telephone and asked to report their outcomes using a structured questionnaire investigating their spectacle usage for various daily activities and their experience with night glare or halos. Inclusion criteria were a normal postoperative visual potential based on the preoperative examination and completion of the questionnaire regarding postoperative visual experience. RESULTS: Of the patients, 514 underwent bilateral cataract surgery aiming for bilateral emmetropia with the implantation of EDOF-IOLs (422 with Tecnis Eyhance and 92 with AT LARA). A complete questionnaire was obtained from 472 (92%) patients, who were included in the study (393 vs. 79). Comparing Tecnis Eyhance with AT LARA IOL, 54 vs. 57% patients were able to perform most of their daily activities without spectacles, 9 vs. 19% reported not being dependent on spectacles at all, 25 vs. 29% reported using their smartphones without spectacles, 15 vs. 49% patients reported experiencing glares or halos at night, and 1 vs.15% with driving disturbance. Overall, 95 vs. 93% patients described themselves as "satisfied" or "highly satisfied" with their IOL. CONCLUSIONS: With both types of EDOF-IOLs, the majority of patients could perform most of their daily activities without spectacles, except reading, and were highly satisfied with their IOLs. Patients with diffractive Zeiss AT LARA EDOF-IOLs were more likely to accomplish unaided near-range visual tasks; however, they were also at a higher risk of experiencing glares or halos at night.