Set Of Patient-reported Outcome Measures Research Articles

Core Set of Patient-Reported Outcome Measures for Measuring Quality of Life in Clinical Obesity Care.

The focus of measuring success in obesity treatment is shifting from weight loss to patients' health and quality of life. The objective of this study was to select a core set of patient-reported outcomes and patient-reported outcome measures to be used in clinical obesity care. The Standardizing Quality of Life in Obesity Treatment III, face-to-face hybrid consensus meeting, including people living with obesity as well as healthcare providers, was held in Maastricht, the Netherlands, in 2022. It was preceded by two prior multinational consensus meetings and a systematic review. The meeting was attended by 27 participants, representing twelve countries from five continents. The participants included healthcare providers, such as surgeons, endocrinologists, dietitians, psychologists, researchers, and people living with obesity, most of whom were involved in patient representative networks. Three patient-reported outcome measures (patient-reported outcomes) were selected: the Impact of Weight on Quality of Life-Lite (self-esteem) measure, the BODY-Q (physical function, physical symptoms, psychological function, social function, eating behavior, and body image), and the Quality of Life for Obesity Surgery questionnaire (excess skin). No patient-reported outcome measure was selected for stigma. A core set of patient-reported outcomes and patient-reported outcome measures for measuring quality of life in clinical obesity care is established incorporating patients' and experts' opinions. This set should be used as a minimum for measuring quality of life in routine clinical practice. It is essential that individual patient-reported outcome measure scores are shared with people living with obesity in order to enhance patient engagement and shared decision-making.

Responsiveness of multiple patient-reported outcome measures for acute postsurgical pain: primary results from the international multi-centre PROMPT NIT-1 study

BackgroundPostsurgical outcome measures are crucial to define the efficacy of perioperative pain management; however, it is unclear which are most appropriate. We conducted a prospective study aiming to assess sensitivity-to-change of patient-reported outcome measures assessing the core outcome set of domains pain intensity (at rest/during activity), physical function, adverse events, and self-efficacy. MethodsPatient-reported outcome measures were assessed preoperatively, on day 1 (d1), d3, and d7 after four surgical procedures (total knee replacement, breast surgery, endometriosis-related surgery, and sternotomy). Primary outcomes were sensitivity-to-change of patient-reported outcome measures analysed by correlating their changes (d1–d3) with patients' global impression of change and patients' specific impression of change items as anchor criteria. Secondary outcomes included identification of baseline and patient characteristic variables explaining variance in change for each of the scales and descriptive analysis of various patient-reported outcome measures from different domains and after different surgeries. ResultsOf 3322 patients included (18 hospitals, 10 countries), data from 2661 patients were analysed. All patient-reported outcome measures improved on average over time; the median calculated sensitivity-to-change for all patient-reported outcome measures (overall surgeries) was 0.22 (range: 0.07–0.31, scale: 0–10); all changes were independent of baseline data or patient characteristics and similar between different procedures. ConclusionsPain-related patient-reported outcome measures have low to moderate sensitivity-to-change; those showing higher sensitivity-to-change from the same domain should be considered for inclusion in a core outcome set of patient-reported outcome measures to assess the effectiveness and efficacy of perioperative pain management.

Conceptual Framework for Patient-Reported Outcome Measures in Clinical Trials of Skeletal Muscle Cramping Experienced in Dialysis: A Kidney Health Initiative Workgroup Report.

Skeletal muscle cramping is a common and bothersome symptom for patients on maintenance dialysis therapy, regardless of modality, and it has not been prioritized for innovative assessments or treatments. Research to prevent or treat skeletal muscle cramping in patients receiving dialysis is hindered by poorly understood pathophysiology, lack of an accepted definition, and the absence of a standardized measurement method. The Kidney Health Initiative, a public-private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a multidisciplinary workgroup to define a set of patient-reported outcome measures for use in clinical trials to test the effect of new dialysis devices, new KRTs, lifestyle/behavioral modifications, and medications on skeletal muscle cramping. Upon determining that foundational work was necessary, the workgroup undertook a multistep process to elicit concepts central to developing the basis for demonstrating content validity of candidate patient-reported outcome measures for skeletal muscle cramping in patients on dialysis. The workgroup sought to (1) create an accepted, patient-endorsed definition for skeletal muscle cramping that applies to all dialysis modalities, (2) construct a conceptual model for developing and evaluating a skeletal muscle cramping-specific patient-reported outcome measure, and (3) identify potential questions from existing patient-reported outcome measures that could be modified or adapted and subsequently tested in the dialysis population. We report the results of the workgroup's efforts, provide our recommendations, and issue a call to action to address the gaps in knowledge and research needs we identified. These action steps are urgently needed to quantify skeletal muscle cramping burden, assess the effect, and measure meaningful changes of new interventions to improve the experience of patients receiving dialysis and suffering from skeletal muscle cramping.

Patient and Treatment Characteristics Associated With Receipt of Core Patient-Reported Outcome Measures in VA Mental Health.

Measurement-based care (MBC) has been implemented in Veterans Affairs since 2016 and is increasingly used in other mental health care organizations. Little quantitative research exists that assesses patient-level barriers and facilitators of MBC. This study examines correlates of receiving patient-reported outcome measures (PROMs) among veterans receiving both psychotherapy and pharmacotherapy (Both), psychotherapy only (Psychotherapy), and pharmacotherapy only (Pharmacotherapy). Data on PROM administration were obtained for a 12-month period for 1,726,578 veterans who initiated outpatient mental health care during fiscal year 2019. Clinical, treatment, and PROM data were extracted from the electronic health record. Logistic regression was used to model the association between veteran and treatment characteristics and PROM administration. Thirty-two percent of veterans in Both, 26.0% in Psychotherapy, and 8.8% in Pharmacotherapy received at least one PROM. The probability of PROM administration was positively associated with the number of treatment encounters during the fiscal year 2019. Major depressive, generalized anxiety, and other depressive disorders were associated with an increased probability of PROM administration. Psychotic disorders, personality disorders, older age, dementia, and electronic health record suicide risk flag were associated with decreased odds of PROM administration across treatment types. Rates of PROM administration differ depending on the type of treatment received. The probability of PROM administration is influenced by the frequency of encounters and, to a lesser extent, having a diagnosis congruent with symptoms assessed in the set of PROMs considered. Consistent with hypotheses from the MBC implementation literature, potential indicators of clinical severity and cognitive impairment decrease the likelihood of PROM utilization.

A retrospective study of eating and psychosocial problems in patients with hepatic glycogen storage diseases and idiopathic ketotic hypoglycemia: Towards a standard set of patient-reported outcome measures.

There is a paucity in literature on eating and psychosocial problems in patients with hepatic glycogen storage disease (GSD) and idiopathic ketotic hypoglycemia (IKH), problems that can greatly affect quality of life. This is a monocentre, retrospective, observational mixed method study of patients with hepatic GSD or IKH treated at the Beatrix Children's Hospital Groningen, who had been referred to SeysCentra, a specialist centre for the treatment of eating problems. Additionally, a systematic literature review has been performed to identify instruments to quantify patient‐reported outcome measures of psychosocial problems in hepatic GSD patients. Sixteen patients from 12 families were included with ages ranging between 3 and 24 years. Five out of sixteen patients were diagnosed with Avoidant/Restrictive Food Intake Disorder and six patients showed characteristics of this disorder. Fourteen patients experienced sleeping problems, and 11 out of 12 parent couples experienced stress about the illness of their child. We subsequently identified 26 instruments to quantify patient‐reported outcome measures for GSD patients. This study demonstrates that GSD patients can develop Avoidant/Restrictive Food Intake Disorder influencing quality of life at multiple domains. The identification of instruments to assess psychosocial wellbeing is an important step towards a standard set of patient‐reported outcome measures.

Profiles of patients\u2019 self-reported health after acute stroke

BackgroundWe aimed to identify groups of patients with similar health status after stroke, assessed by patient reported outcome measures (PROMs), to improve initial risk stratification.MethodsIn a prospective study, inpatients were recruited during acute stroke treatment. Demographics, history, and cardio-vascular risk factors were assessed at baseline. Self-reported functional status, physical and mental health as well as anxiety and depressive symptoms were assessed 3 and 12 months after stroke and used to identify latent classes. The association of patient characteristics with latent class membership was investigated with multinomial logistic regression.ResultsOf the 650 patients included with a mean age of 75 years and 48% female, 70% had ischemic, 6% hemorrhagic strokes, and 24% transient ischemic attacks. Median NIHSS on admission was 2 (IQR:0,5). Values of PROMs remained comparable at 3 and 12 months. A three-class model was developed, differentiating between patients with mildly (75%), moderately (17%), and severely (8%) impaired self-reported health status. Adjusted for univariately significant baseline characteristics, initial NIHSS distinguished mild- from moderate-, and moderate- from severe-class-membership (p < 0.001). Length of inpatient stay (p < 0.001;OR = 1.1), diabetes (p = 0.021;OR = 1.91), and atrial fibrillation (p = 0.004;OR = 2.20) predicted allocation to the moderately vs. mildly affected class.ConclusionsGrading stroke patients by a standard set of PROMs up to 1 year after stroke allows to distinguish the diverse impact of baseline characteristics on differently affected groups. In addition to initial stroke severity, longer inpatient stay, presence of diabetes and atrial fibrillation correlate with greater impairment of self-reported health in the less affected groups.Trial registrationhttp://www.ClinicalTrials.gov; Unique identifier: NCT03795948.

Developing an International Standard Set of Patient-Reported Outcome Measures for Psychotic Disorders.

The objective of this project was to develop a set of patient-reported outcome measures for adolescents and adults who meet criteria for a psychotic disorder. A research team and an international consensus working group, including service users, clinicians, and researchers, worked together in an iterative process by using a modified Delphi consensus technique that included videoconferencing calls, online surveys, and focus groups. The research team conducted systematic literature searches to identify outcomes, outcome measures, and risk adjustment factors. After identifying outcomes important to service users, the consensus working group selected outcome measures, risk adjustment factors, and the final set of outcome measures. International stakeholder groups consisting of >100 professionals and service users reviewed and commented on the final set. The consensus working group identified four outcome domains: symptoms, recovery, functioning, and treatment. The domains encompassed 14 outcomes of importance to service users. The research team identified 131 measures from the literature. The consensus working group selected nine measures in an outcome set that takes approximately 35 minutes to complete. A set of patient-reported outcome measures for use in routine clinical practice was identified. The set is free to service users, is available in at least two languages, and reflects outcomes important to users. Clinicians can use the set to improve clinical decision making, and administrators and researchers can use it to learn from comparing program outcomes.

A Multi-Site Mood Outcomes Program: Preliminary Results and Lessons Learned From the National Network of Depression Centers

The National Network of Depression Centers (NNDC) is a consortium of academic medical centers specializing in the treatment of mood disorders. The NNDC's longitudinal Mood Outcomes Program where 17 participating programs administer a standard set of patient-reported outcome measures at the point of care.

Pediatric PROMIS Computer Adaptive Tests Are Highly Correlated With Adult PROMIS Computer Adaptive Tests in Pediatric Sports Medicine Patients

Background: The Patient-Reported Outcomes Measurement Information System (PROMIS) is a powerful set of patient-reported outcome measures (PROMs) that are gaining popularity throughout orthopaedic surgery. The use of both adult and pediatric PROMIS questionnaires in orthopaedic sports medicine limits the value of the PROMIS in routine sports medicine clinical care, research, and quality improvement. Because orthopaedic sports medicine surgeons see patients across a wide age range, simplifying the collection of PROMIS computer adaptive tests (CATs) to a single set of questionnaires, regardless of age, is of notable value. Purpose/Hypothesis: The purpose was to determine the strength of the correlation between the pediatric and adult PROMIS questionnaires. We hypothesized that there would be a high correlation between the adult and pediatric versions for each PROMIS domain, thereby justifying the use of only the adult version for most sports medicine providers, regardless of patient age. Study Design: Cohort study (Diagnosis); Level of evidence, 2. Methods: Between December 2018 and December 2019, all pediatric sports medicine patients presenting to a single, academic, orthopaedic sports medicine clinic were asked to participate in the present study with their parents’ consent. Patients were asked to complete a set of adult PROMIS domains (Physical Function and/or Upper Extremity, Pain Interference, and Depression) as well as a set of pediatric PROMIS domains (Mobility and/or Upper Extremity, Pain Interference, and Depressive Symptoms). Concurrent validity was assessed using Pearson correlation coefficients (r). Ceiling and floor effects were determined. Results: A total of 188 patients met our inclusion criteria. The correlation between the adult and pediatric PROMIS Upper Extremity, Physical Function and Mobility, Pain Interference, and Depression and Depressive Symptoms forms were high-moderate (r = 0.68; P < .01), high-moderate (r = 0.69; P < .01), high (r = 0.78; P < .01), and high (r = 0.85; P < .01), respectively. Both adult and pediatric depression-related PROMIS domains demonstrated notable floor effects (adult: 38%; pediatric: 24%). The pediatric PROMIS Upper Extremity domain demonstrated a ceiling effect (20%). Conclusion: Adult PROMIS CATs may be used in an orthopaedic sports medicine clinic for both adult and pediatric patients. Our findings will help decrease the amount of resources needed for the implementation and use of PROMs for patient care, research, and quality improvement in orthopaedic sports medicine clinics.

A value-based healthcare approach: Health-related quality of life and psychosocial functioning in women with Turner syndrome.

ObjectiveAs part of the value‐based healthcare programme in our hospital, a set of patient‐reported outcome measures was developed together with patients and implemented in the dedicated Turner Syndrome (TS) outpatient clinic. This study aims to investigate different aspects of health‐related quality of life (HR‐QoL) and psychosocial functioning in women with TS in order to establish new possible targets for therapy.Design/ParticipantsA comprehensive set of questionnaires (EQ‐5D, PSS‐10, CIS‐20, Ferti‐QoL, FSFI) was developed and used to capture different aspects of HR‐QoL and psychosocial functioning in a large cohort of adult women with Turner syndrome. All consecutive women, ≥18 years, who visited the outpatient clinic of our tertiary centre were eligible for inclusion.ResultsOf the eligible 201 women who were invited to participate, 177 women (age 34 ± 12 years, mean ± SD) completed at least one of the validated questionnaires (88%). Women with TS reported a lower health‐related quality of life (EQ‐5D: 0.857 vs 0.892, P = .003), perceived more stress (PSS‐10:14.7 vs 13.3; P = .012) and experienced increased fatigue (CIS‐20: P < .001) compared to the general Dutch population. A relationship between noncardiac comorbidities (eg diabetes, orthopaedic complaints) and HR‐QoL was found (R = .508).ConclusionsWe showed that TS women suffer from impaired HR‐QoL, more perceived stress and increased fatigue compared to healthy controls. A relationship between noncardiac comorbidities and HR‐QoL was found. Especially perceived stress and increased fatigue can be considered targets for improvement of HR‐QoL in TS women.

Feasibility of Collecting Multiple Patient-Reported Outcome Measures Alongside the Dutch Arthroplasty Register.

Background:Compliance rates with patient-reported outcome measures (PROMs) collected alongside arthroplasty registries vary in the literature. We described the feasibility of a routinely collected set PROMs alongside the Dutch Arthroplasty Register.Methods:The longitudinal Leiden Orthopaedics Outcomes of OsteoArthritis Study is a multicenter (7 hospitals), observational study including patients undergoing total hip or total knee arthroplasty (THA or TKA). A set of PROMs: Short Form-12, EuroQol 5 Dimensions, Hip/Knee injury and Osteoarthritis Outcome Score, Oxford Hip/Knee Score was collected preoperatively and at 6, 12, 24 months, and every 2 years thereafter. Participation rates and response rates were recorded.Results:Between June 2012 and December 2014, 1796 THA and 1636 TKA patients were invited, of whom 1043 THA (58%; mean age 68 years [standard deviation, SD: 10]) and 970 TKA patients (59%; mean age 71 years [SD 9.5]) participated in the study. At 6 months, 35 THA/38 TKA patients were lost to follow-up. Response rates were 90% for THA (898/1000) and 89% for TKA (827/932) participants. At 1 and 2 years, 8 and 18 THA and 17 and 11 TKA patients were lost to follow-up, respectively. The response rates among those eligible were 87% (866/992) and 84% (812/972) for THA and 84% (771/917) and 83% (756/906) for TKA patients, respectively. The 2-year questionnaire was completed by 78.5% of the included THA patients and by 77.9% of the included TKA patients.Conclusions:About 60% of patients undergoing THA or TKA complete PROMs preoperatively, with more than 80% returning follow-up PROMs. To increase the participation rates, more efforts concerning the initial recruitment of patients are needed.

Outcome of knee arthroplasty in patients aged 65 years or less: a prospective study of 232 patients with 2-year follow-up

Previous studies have reported lower implant survival rates, residual pain, and higher patient dissatisfaction rates following knee arthroplasty in younger knee arthroplasty patients. We aimed to assess the real-world effectiveness of knee arthroplasty in a prospective non-selected cohort of patients aged 65 years or less with 2-year follow-up. In total, 250 patients (272 knees) aged 65 years or less were enrolled into this prospective cohort study. Patient-reported outcome measures were used to assess the outcome. The mean Oxford Knee Score and all Knee Injury and Osteoarthritis Outcome Score subscales increased significantly (p < 0.001) from preoperative situation to the 2-year follow-up. Significant increase (p < 0.001) in physical activity was detected in High-Activity Arthroplasty Score and RAND-36 Physical Component Score (PCS). Pain was also significantly (p < 0.001) relieved during the follow-up. Total disappearance of pain was rare at 2 years. Patients with milder (Kellgren-Lawrence grade 2) osteoarthritis were less satisfied and reported poorer patient-reported outcome measure than those with advanced osteoarthritis (Kellgren-Lawrence grade 3-4). There was no difference in the outcome (any patient-reported outcome measure) between patients who underwent total knee arthroplasty and those who received unicondylar knee arthroplasty. We found that measured with a wide set of patient-reported outcome measures, both total knee arthroplasty and unicondylar knee arthroplasty resulted in significant pain relief, as well as improvement in physical performance and quality of life in patients aged 65 years or less. Real-world effectiveness of these procedures seems to be excellent. 15% of patients still had residual symptoms and were dissatisfied with the outcome at 2 years after the operation.

Conceptualizing patient-reported outcome measures for use within two Danish psychiatric clinical registries: description of an iterative co-creation process between patients and healthcare professionals

Background: Denmark has national clinical indicator programs for adult patients diagnosed with depression and schizophrenia, respectively. Within each program, the responsible steering group (SG) decided to add some indicators based upon patient-reported outcome measures (PROMs).Aims: The primary aim was to describe the process of selecting PROMs and defining a national measurement concept for use in clinical practice and for indicator monitoring and the secondary aim s to collect patient recommendations for implementation.Methods: An interdisciplinary SG of healthcare professionals and a Patient Peer Board (PPB) representing both patient groups co-created the output in an iterative process.The work included literature search, PPB workshops, SG meetings, ratings of PROM topics and items, and a pilot. The PPB discussed the following: item relevance, mode of data collection, graphical format of the online PROMs, and display of results. Finally, requirements for PROM patient information were identified. Based upon input from the PPB, the SG selected the items and specified the measurement concept.Results: The PPB prioritized 20 of 53 suitable items and suggested alternative wording and answer categories. A pilot was performed and 19 items covering well-being, lack of well-being, impairment of functioning, and overall health were selected for clinical testing.The patients recommended concrete, unambiguous, easily understandable information and procedures for data collection and display of results.Conclusions: The iterative co-creation process based upon a high degree of patient involvement resulted in a set of PROMs, a national measurement concept, and patient recommendations for implementation. The cooperation between patients and professionals was successful.

Characteristics of Patients With Early-Onset Arthritis in Latin America: Description of the REPANARC Cohort.

While many studies have tried to show that early intervention improves the clinical outcomes of early-onset arthritis, only a few were carried out in Latin America. The aim of this study was to describe the Pan-American Registry of Early-Onset Arthritis (REPANARC) project and report the preliminary outcomes of a cohort of patients. The REPANARC cohort consisted of a sample of patients from 6 Latin American countries. Patients with arthritis of 1 or more joints of 1-year duration or less were assessed by a rheumatologist during 6 consecutive clinical visits for a follow-up period of 2 years. The registry included clinical characteristics, medical history, physical examination, disease activity, analytical chemistries, imaging, current treatment, and a set of patient-reported outcome measures evaluating disability, psychological distress, and quality of life. A total of 173 patients were included with mean age of 41.9 ± 13.2 years; 83.8% were women. The predominant presentations at onset were insidious, polyarticular, additive, bilateral, and symmetrical. The initial diagnoses were rheumatoid arthritis (50.6%), undifferentiated arthritis (40.5%), and other arthritis (8.9%). With Disease Activity Score in 28 Joints, 76.9% had moderate to high disease activity, and 61.9% had moderate to severe disability (Health Assessment Questionnaire). Considering undifferentiated arthritis, 60.3% persisted undifferentiated, 29.4% evolved as rheumatoid arthritis, 4.4% remained self-limited, and 5.9% to other forms. The frequencies of depression and anxiety were high as measured with the Hospital Anxiety and Depression Scale, and approximately 20% had significant decrements in quality of life measured with the Medical Outcomes Study Short-Form 36 Health Survey Version 2. Mean time from the first symptoms to the first visit to a rheumatologist was 126 days. Shorter delays were confirmed to be associated with better outcomes. The REPANARC project is a useful tool to provide valuable information regarding patients with early-onset arthritis attending rheumatology centers in Latin-America.

A promising method for identifying cross-cultural differences in patient perspective: the use of Internet-based focus groups for content validation of new Patient Reported Outcome assessments

ObjectivesThis proof of concept (POC) study was designed to evaluate the use of an Internet-based bulletin board technology to aid parallel cross-cultural development of thematic content for a new set of patient-reported outcome measures (PROs).MethodsThe POC study, conducted in Germany and the United States, utilized Internet Focus Groups (IFGs) to assure the validity of new PRO items across the two cultures – all items were designed to assess the impact of excess facial oil on individuals' lives. The on-line IFG activities were modeled after traditional face-to-face focus groups and organized by a common 'Topic' Guide designed with input from thought leaders in dermatology and health outcomes research. The two sets of IFGs were professionally moderated in the native language of each country. IFG moderators coded the thematic content of transcripts, and a frequency analysis of code endorsement was used to identify areas of content similarity and difference between the two countries. Based on this information, draft PRO items were designed and a majority (80%) of the original participants returned to rate the relative importance of the newly designed questions.FindingsThe use of parallel cross-cultural content analysis of IFG transcripts permitted identification of the major content themes in each country as well as exploration of the possible reasons for any observed differences between the countries. Results from coded frequency counts and transcript reviews informed the design and wording of the test questions for the future PRO instrument(s). Subsequent ratings of item importance also deepened our understanding of potential areas of cross-cultural difference, differences that would be explored over the course of future validation studies involving these PROs.ConclusionThe use of IFGs for cross-cultural content development received positive reviews from participants and was found to be both cost and time effective. The novel thematic coding methodology provided an empirical platform on which to develop culturally sensitive questionnaire content using the natural language of participants. Overall, the IFG responses and thematic analyses provided a thorough evaluation of similarities and differences in cross-cultural themes, which in turn acted as a sound base for the development of new PRO questionnaires.