Themes that Determine Quality of Life in Patients with Peripheral Arterial Disease: A Systematic Review.

Generic and disease-specific patient-reported outcome measures (PROMs) may lack relevance and sensitivity on a patient-level in chronic diseases with differential disease expression and high individual variability, such as Cystic Fibrosis (CF). This study aimed to develop and validate a novel personalized electronic PROM (ePROM) that captures relevant aspects of quality of life in individuals with CF. The Q-Life app was developed as a short personalized ePROM to assess individual quality of life. Psychometric properties were assessed in a single-center cross-sectional study between September 2019 and September 2021 and in a prospective cohort study between September 2021 and September2022. Combined studies included 223 participants (median age: 24 years, IQR: 19.0-32.5 years, range: 12.0-58.0 years). Internal consistency (Cronbach's alpha: 0.83-0.90) and test-retest reliability (intraclass correlation coefficient: 0.90; 95% CI: 0.65-0.92; p<0.001) of quality of life (Q-Life) scores were strong. Q-Life scores were associated with overall Cystic Fibrosis Questionnaire-Revised (CFQ-R) scores (ρ=0.71; p<0.001), CFQ-R respiratory domain scores (ρ=0.57; p<0.001) and forced expiratory volume in 1s (ρ=0.41; p<0.001). Furthermore, Q-Life scores improved from 65.0 (IQR: 45.0-63.3) at baseline to 84.2 (IQR: 75.0-95.0) and 87.5 (IQR: 75.0-100.0) after 3 and 6 months of elexacaftor/tezacaftor/ivacaftor treatment (change: 20.8; 95% CI: 17.5-25.0; p<0.001), comparable to CFQ-R respiratory domain scores (change: 22.2, 95% CI: 19.4-25.0, p<0.001). The Q-Life app is a reliable, valid and sensitive personalized ePROM to measure all aspects of quality of life that really matter to individuals with Cystic Fibrosis. This patient-centered approach could provide important advantages over generic and disease-specific PROMs in the era of personalized medicine and value-based healthcare. Dutch Cystic Fibrosis Foundation, Health-Holland.

Chronic cough (CC), defined as a cough persisting ≥ 8weeks, can have a substantial negative impact on health-related quality of life (HRQoL). This is exacerbated by challenges with timely diagnosis and a lack of approved therapies. A systematic literature review (SLR) was conducted to identify evidence on HRQoL and health state utility values associated with refractory CC or unexplained CC. Electronic database searches were supplemented with searches of conference proceedings and health technology assessment body websites. Two independent reviewers assessed all citations for inclusion based on predefined inclusion/exclusion criteria. Key inclusion criteria were patient populations with CC and reporting of patient-reported outcomes or utilities using generic or disease-specific measures. Following screening, 65 studies were identified for inclusion in the SLR. Of these, 23 studies assessed HRQoL among patients with CC who were not treated or treated with unspecified interventions, and 42 studies in patients who were treated with specified interventions. The studies indicated a substantial decrement to HRQoL as a result of CC, characterized by generic and disease-specific patient-reported outcome measures. HRQoL was impacted across multiple domains, including physical, psychological, and social functioning. The studies also demonstrated the potential for treatments to have a significant positive impact on HRQoL. CC can substantially affect HRQoL in patients, across physical, psychological, and social domains. Although treatments can improve HRQoL in these patients, the available evidence is limited. There remains an unmet need for approved pharmacological treatments to alleviate CC and improve HRQoL for these patients.

Current status of patient-reported outcome measures in vascular surgery

Development of National Research and Clinical Agendas for Patient-Reported Outcomes in IR: Proceedings from a Multidisciplinary Consensus Panel

Health-related quality of life and patient-reported outcome measures in NASH-related cirrhosis

BackgroundFollow-up care provided via telemedicine (TM) is intended to be a more integrated care pathway to manage diabetes-related foot ulcers (DFU) than traditionally-delivered healthcare. However, knowledge of the effect of TM follow-up on PROMs including self-reported health, well-being and QOL in patients with DFUs is lacking and often neglected in RCT reports in general. Therefore, in this study of secondary outcomes from the DiaFOTo trial, the aim was to compare changes in self-reported health, well-being and QOL between patients with DFUs receiving telemedicine follow-up care in primary healthcare in collaboration with specialist healthcare, and patients receiving standard outpatient care.MethodsThe current study reports secondary endpoints from a cluster randomized controlled trial whose primary endpoint was ulcer healing time. The trial included 182 adults with diabetes-related foot ulcers (94/88 in the telemedicine/standard care groups) in 42 municipalities/districts, recruited from three clinical sites in Western Norway. Mean (SD) diabetes duration for the study population was 20.8 (15.0). The intervention group received care in the community in collaboration with specialist healthcare using an asynchronous telemedicine intervention. The intervention included an interactive web-based ulcer record and a mobile phone enabling counseling and communication between the community nurses and specialist healthcare; the control group received standard outpatient care. In total 156 participants (78/78) reported on secondary endpoints: self-reported health, well-being and quality of life evaluated by generic and disease-specific patient-reported outcome measures (e.g. Euro-QOL, the Hospital Anxiety and Depression Scale (HADS), Problem Areas in Diabetes (PAID), Neuropathy and Foot Ulcer–Specific Quality of Life Instrument (NeuroQOL)). Linear mixed-effects regression was used to investigate possible differences in changes in the scores between the intervention and control group at the end of follow-up.ResultsIn intention to treat analyses, differences between treatment groups were small and non-significant for the health and well-being scale scores, as well as for diabetes-related distress and foot ulcer-specific quality of life.ConclusionsThere were no significant differences in changes in scores for the patient reported outcomes between the intervention and control group, indicating that the intervention did not affect the participants’ health, well-being and quality of life.Trial registrationClinicaltrials.gov, NCT01710774. Registered October 19th, 2012.

To discuss the importance of validated tools that measure patient-reported outcomes and their use in ambulatory surgery. Sustained increases in ambulatory surgical care reflect advances in surgical techniques and perioperative anaesthetic care. Use of patient-reported outcomes allows identification of minor adverse events that are more common in this population compared with traditional endpoints such as mortality. Variability in reported outcomes restricts research potential and limits the ability to benchmark providers. The standardized endpoints in perioperative medicine initiative's recommendations on patient-reported outcomes and patient comfort measures are relevant to evaluating ambulatory care. Combining validated generic and disease-specific patient-reported outcome measures (PROMs) examines the widest spectrum of outcomes. Technological advances can be used to facilitate outcome measurement in ambulatory surgery with digital integration optimizing accurate real-time data collection. Telephone or web-based applications for reviewing ambulatory patients were found to be acceptable in multiple international settings and should be harnessed to allow remote follow-up. Use of validated tools to measure patient-reported outcomes allows internal and external quality comparison. Tools can be combined to measure objective outcomes and patient satisfaction. These are both key factors in driving forward improvements in perioperative ambulatory surgical care.

Varicose veins and chronic venous disease are common, and some funding bodies ration treatment based on a minimum diameter of the incompetent truncal vein. This study assessed the effect of maximum vein diameter on clinical status and patient symptoms. A prospective observational cohort study of patients presenting with symptomatic varicose veins to a tertiary referral public hospital vascular clinic between January 2011 and July 2012. Patients underwent standardized assessment with venous duplex ultrasonography, and completed questionnaires assessing quality of life (QoL) and symptoms (Aberdeen Varicose Vein Questionnaire, EuroQol Five Domain QoL assessment and EuroQol visual analogue scale). Clinical scores (Venous Clinical Severity Score (VCSS) and Clinical Etiologic Anatomic Pathophysiologic (CEAP) class) were also calculated. Regression analysis was used to investigate the relationship between QoL, symptoms and vein diameter. Some 330 patients were assessed before surgery. The median maximum vein diameter was 7·0 (i.q.r. 5·3-9·2) mm overall, 7·9 (6·0-9·8) mm for great saphenous vein and 6·0 (5·2-8·9) mm for small saphenous vein. In linear regression analysis, vein diameter was shown to have a significant association with VCSS (P = 0·041). For every 1-mm increase in vein diameter, there was a 2·75-fold increase in risk of being in CEAP class C4 compared with C2. No other QoL or symptom measures were related to vein diameter. Incompetent truncal vein diameter was associated with increasing VCSS, but not a variety of other varicose vein disease-specific and generic patient-reported outcome measures.

INTRODUCTION: Patient reported outcome measures (PROMs) play important role in addressing health related quality of life of patients and their outcome satisfaction. Neurosurgery is lagging behind in disease specific PROMs and there is an urgent and emerging need for disease specific PROMs for neurosurgery especially neuro-oncology to address specific quality of life issues, outcome satisfaction and patient safety. METHODS: A search in MEDLINE, PUBMED, EMBASE and WEB of Science databases was done for articles related to neurosurgery PROMs and 72 relevant articles were identified among 4473 articles. Twenty nine different PROMs were analysed from the relevant articles in terms of factions tested, advantages, disadvantages, limitations and scope in neurosurgery. RESULTS: The extensive search resulted in the data suggesting that generic PROMs are still in use in neurosurgery and multiple disadvantages has been identified giving rise to a strong scope and need of disease specific neurosurgical PROMs. CONCLUSION: Currents generic PROMs do not address the disease specific outcome issues of neurosurgery and thus there is a definite need of disease specific PROMs especially neuro-oncology so that the patient satisfaction and safety as well as quality of life issues can be addressed systematically. KEYWORDS: PROMs, Neurosurgery, brain tumours

Background Efficient use of health resources requires accurate outcome assessment. Disease-specific patient-reported outcome (PRO) measures are designed to be highly relevant to patients with a specific disease. They have advantages over generic PROs that lack relevance to patient groups and miss crucial impacts of illness. It is thought that disease-specific measurement cannot be used in comparative effectiveness research (CER). The present study provides further evidence of the value of disease-specific measures in making valid comparisons across diseases.Methods The Asthma Life Impact Scale (ALIS, 22 items), Living with Chronic Obstructive Pulmonary Disease (LCOPD, 22 items) scale, and Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR, 25 items) were completed by 140, 162, and 91 patients, respectively. The three samples were analyzed for fit to the Rasch model, then combined into a scale consisting of 58 unique items and re-analyzed. Raw scores on the three measures were co-calibrated and a transformation table produced.Results The scales fit the Rasch model individually (ALIS Chi2 probability value (p-Chi2) = 0.05; LCOPD p-Chi2 = 0.38; CAMPHOR p-Chi2 = 0.92). The combined data also fit the Rasch model (p-Chi2 = 0.22). There was no differential item functioning related to age, gender, or disease. The co-calibrated scales successfully distinguished between perceived severity groups (p < 0.001).Limitations The samples were drawn from different sources. For scales to be co-calibrated using a common item design, they must be based on the same theoretical construct, be unidimensional, and have overlapping items.Conclusions The results showed that it is possible to co-calibrate scores from disease-specific PRO measures. This will permit more accurate and sensitive outcome measurement to be incorporated into CER. The co-calibration of needs-based disease-specific measures allows the calculation of γ scores that can be used to compare directly the impact of any type of interventions on any diseases included in the co-calibration.

PIH33 Generic Patient-Reported Outcome Measures (PROMS) for Use in Pediatric Age Group: Review of Available Measures

Patient-reported outcome measures (PROMs) play an important role in the evaluation of health outcomes, quality of life, and satisfaction, and have been successfully utilized in many areas of clinical medicine and surgical practice. The prevalence of PROMs in neurosurgery is not known. To review the PROMs that have been utilized in the published neurosurgery literature to date. Articles were searched in MEDLINE, EMBASE, HMIC Health Management Information Consortium, PsycARTICLES, and PsycINFO using search terms related to neurosurgery and PROMs, published from 1806 to August 2016. A total of 268 articles were identified that were stratified by the inclusion and exclusion criteria leading to a total of 137 articles. Twenty-six PROMs, involving both adult and pediatric populations, were identified. A large number of generic and disease-specific PROMs are used in the neurosurgical literature. Generic PROMs are usually nonspecific measures of health status. Disease-specific PROMs may not address issues relevant to neurosurgical procedures. There are very few neurosurgery-specific PROMs that take into account the impact of a neurosurgical procedure on a specific condition. PROMs that currently feature in the neurosurgical literature may not address the specific outcomes relevant to neurosurgical practice. There is an emergent need for generic and disease-specific PROMs to be validated in neurosurgical patients and neurosurgery-specific PROMs developed to address unmet needs of patients undergoing neurosurgical procedures.

Background: Comparing outcomes across different health measurement tools is essential where various patient-reported outcome measures (PROMs) are used. In spinal surgery, where recent studies show that over 30 different PROMs are applied, this need becomes even more pressing. Although several statistical transformations between the Oswestry Disability Index (ODI) and the PROMIS Profile 29 have been proposed, validation studies on conversion equations and cross-walk tables remain limited. In this study, we examined the agreement between observed ODI scores and those predicted from the PROMIS Profile 29 in a large sample of patients with low back pain, collected from routine clinical care. Methods: We compared the performance of regression and linking models at both the individual and group levels. Using Bland–Altman plots, we assessed the mean difference, 95% limits of agreement, root mean squared error (RMSE), and standardized mean differences (Cohen’s d) between predicted and observed ODI scores. Results: While group-level agreement was satisfactory, with negligible effect sizes, individual prediction accuracy was relatively poor. Additionally, regression models showed inconsistent performance across the ODI score range, though incorporating more domains marginally improved predictions. Conclusions: The equipercentile linking approach demonstrated stable agreement across all ODI scores, making it the preferred method. Future regression models should account for nonlinear relationships between PROMs to enhance prediction accuracy.

Disease-specific patient-reported outcome (PRO) measures are designed to be highly relevant to one disease. It is widely believed that comparisons of outcomes between patients with different diseases are only possible using generic measures. The present study employs a novel method of using Rasch analysis to co-calibrate scores from different disease-specific PRO measures, allowing scores to be compared across diseases. Psoriasis patients (n = 146, mean age = 44.4, males = 50 %) completed the Psoriasis Quality of Life scale (PSORIQoL) and atopic dermatitis patients (n = 146, mean age = 45.5, males = 50 %) the Quality of Life in Atopic Dermatitis scale (QoLIAD). Both measures employ the needs-based model of QoL, and they share five common items-providing a link between assessments. The groups were analysed separately, and then combined to test fit to the Rasch model. Both scales showed good fit to the Rasch model after minor adjustments (PSORIQoL: χ (2) p = 0.25; QoLIAD: χ (2) p = 0.51). For the combined dataset, one common item showing differential item functioning by disease was removed and fit to the Rasch model was achieved (χ (2) p = 0.08). The co-calibrated scale successfully distinguished between perceived severity groups (p < 0.001). It is possible to co-calibrate scores on the PSORIQoL and QoLIAD. This is one of the first studies in health research to demonstrate how Rasch analysis can be used to make comparisons across diseases using different disease-specific measures. Such an approach maintains the greater relevance and, consequently, accuracy associated with disease-specific measurement.

Long-term patient reported sexual and urological dysfunction in males after operatively treated pelvic ring injuries Do generic outcome measures identify genitourinary health problems?