Generic Patient-reported Outcome Measures Research Articles

Towards Standardized Assessment of Outcomes in Back Pain—Validation of Linking Studies Between Disease-Specific and Generic Patient-Reported Outcome Measures

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.

PROs for RARE: protocol for development of a core patient reported outcome set for individuals with genetic intellectual disability

IntroductionRare genetic neurodevelopmental disorders and intellectual disability (ID), collectively called genetic ID (GID), can profoundly impact daily functioning and overall well-being of affected individuals. To improve our understanding of the impact of GID and advancing both care and research, measuring relevant patient reported outcomes (PROs) is crucial. Currently, various PROs are measured for GID. Given the shared comorbidities across disorders, we aim to develop a generic core PRO set for children and adults with GID.Methods and resultsDeveloping the generic core PRO set entails the following steps: 1) providing an overview of potentially relevant PROs by scoping reviews and qualitative research; 2) integrating and conceptualizing these PROs (i.e., describing the content of the PROs in detail) into a pilot generic core PRO set; and 3) prioritizing relevant PROs by a European Delphi survey and consensus meetings.ConclusionsThis protocol presents the steps for developing a generic core PRO set for children and adults with GID. The next step involves selecting suitable patient reported outcome measures (PROMs) to adequately measure these PROs: the generic core PROM set. This generic core PROM set needs validation in the GID population, and eventually implementation in care and research, facilitating the aggregation and analysis of PRO data and guaranteeing continuous integration of the patient perspective in both care and research.

Physical and Mental Components of Quality of Life after a Cardiac Rehabilitation Intervention: A Systematic Review and Meta-Analysis.

Background: This study aimed to analyze the effect of cardiac rehabilitation programs on the health-related quality of life of patients after a coronary cardiac event using patient-reported outcome measures (PROMs) for up to 6 months of evaluation. Methods: A comprehensive search was carried out in the MEDLINE, CINAHL, CENTRAL, and Web of Science databases for randomized controlled trials comparing the cardiac rehabilitation program with usual care. Two independent reviewers assessed the studies for inclusion, risk of bias using the Cochrane tool, and quality of evidence through the GRADE system. A meta-analysis was performed on studies assessing health-related quality of life with the SF-12 (Physical Component Summary and Mental Component Summary) up to 6 months after the program. Results: Twelve studies encompassed 2260 patients who participated in a cardiac rehabilitation program after a coronary event, with a mean age of 60.06 years. The generic PROMs used to assess quality of life were the SF-12, SF-36, EQ-5D-3L, EQ-5D-5L, and GHQ, and the specific coronary heart disease PROMs were MacNew and HeartQoL. There was a positive effect of participation in cardiac rehabilitation on the physical component of health-related quality of life at 6 months (MD [7.02]; p = 0.04] and on the mental component (MD [1.06]; p = 0.82) after applying the SF-12. Conclusions: This study highlights the significant benefits of cardiac rehabilitation programs on health-related quality of life, particularly in the physical domain at 6 months. Assessing outcomes over time through PROMs after coronary heart events is essential, thus making it possible to personalize patients' care and improve their health status.

A systematic review of crosswalks for converting patient-reported outcome measure scores in hip, knee, and shoulder replacement surgery.

We aimed to systematically review studies of crosswalks for converting patient-reported outcome measure (PROM) scores used in joint replacement, and develop a database of published crosswalks. 4 electronic databases were searched from January 2000 to May 2023 to identify studies reporting the development and/or validation of crosswalks to convert PROM scores in patients undergoing elective hip, knee, or shoulder replacement surgery. Data on study and sample characteristics, source and target PROMs, and crosswalk development and validation methods were extracted from eligible studies. Study reporting was evaluated using the Mapping onto Preference-based measures reporting Standards (MAPS) checklist. 17 studies describing 35 crosswalks were eligible for inclusion. Unidirectional crosswalks were available to convert hip-specific (Oxford Hip Score [OHS]) and knee-specific (Oxford Knee Score [OKS]) scores to the EQ-5D-3L/EQ-5D-5L. Similar crosswalks to convert disease-specific scores (WOMAC) to the EQ-5D-3L, EQ-5D-5L, and ICECAP-O Capability Index were identified. Bidirectional crosswalks for converting OHS and OKS to the HOOS-JR/HOOS-12 and KOOS-JR/KOOS-12, for converting WOMAC to the HOOS-JR/KOOS-JR, and for converting HOOS-Function/KOOS-Function to the PROMIS-Physical Function were also available. Additionally, crosswalks to convert generic PROM scores from the UCLA Activity Scale to the Lower Extremity Activity Scale in both directions were available. No crosswalks were identified for converting scores in shoulder replacement. Development methods varied with the type of target score; most studies used regression, item response theory, or equipercentile equating approaches. Reporting quality was variable, particularly for methods and results items, impacting crosswalk application. This is the first synthesis of published crosswalks for converting joint-specific (OHS, OKS, HOOS, KOOS), disease-specific (WOMAC), and generic PROMs scores (PROMIS-Physical Function, UCLA Activity Scale, Lower Extremity Activity Scale) used to assess joint replacement outcomes, providing a resource for data harmonization and pooled analysis. Crosswalks were developed using regression methods (9 studies), equipercentile equating methods (5 studies), a combination of equipercentile equating and item response theory methods (2 studies), and a combination of regression and equipercentile equating methods (1 study). A range of crosswalk validation approaches were adopted, including the use of external datasets, separate samples or subsets, follow-up data from additional time points, or bootstrapped samples. Efforts are needed to standardize crosswalk methodology and achieve consistent reporting.

Patient-reported outcome measures for medication treatment satisfaction: a systematic review of measure development and measurement properties

BackgroundMedication Treatment Satisfaction (M-TS) from the patients’ perspective is important for comprehensively evaluating the effect of medicines. The extent to which current patient-reported outcome measures (PROMs) for M-TS are valid, reliable, responsive, and interpretable remains unclear. To assess the measurement properties of existing PROMs for M-TS and to highlight research gaps.MethodsUsing PubMed, Embase (Ovid), Cochrane library (Ovid), IPA (Ovid), PsycINFO, Patient-Reported Outcome and Quality of Life Questionnaires biomedical databases, and four Chinese databases, we performed a systematic search for studies addressing the development and validation of PROMs for M-TS. Based on the Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) guideline, pairs of reviewers independently assessed the measurement properties of the PROMs and rated the quality of evidence on the measurement properties of each PROM. (The Open Science Framework registration: https://doi.org/10.17605/OSF.IO/8S5ZM).ResultsThis review identified 69 PROMs for M-TS in 114 studies (four generic, 32 disease-specific, and 33 drug-specific) of which 60 were intended for adults. All provided limited or no information regarding interpretability. Most demonstrated appropriate construct validity including convergent validity (39/69) and discriminative or known-groups validity (40/69) (high to moderate quality of evidence). Only a few provided evidence of sufficient content validity (8/69), structural validity (13/69), and internal consistency (11/69). Of 38 PROMs reporting test–retest reliability, results in 24 provided evidence of satisfactory test–retest reliability (18 with high to moderate, 6 with low to very low quality of evidence). Few PROMs reported responsiveness (16/69). Two generic PROMs (Treatment Satisfaction Questionnaire for Medication initial Version 1.4, TSQM-1.4; Treatment Satisfaction with Medicines Questionnaire, SATMED-Q) and one drug-specific PROM (Insulin Treatment Satisfaction Questionnaire, ITSQ) demonstrated both satisfactory validity and reliability.ConclusionsMost existing PROMs for M-TS require further exploration of measurement properties. Reporting guidelines are needed to enhance the reporting quality of the development and validation of PROMs for M-TS.

Cross-cultural adaptation and validation of Carolinas Comfort Scale to Brazilian Portuguese for inguinal hernia.

Inguinal hernias are highly prevalent worldwide and its surgical repair is one of the most common procedures in general surgery. The broad use of mesh has decreased the recurrence rates of inguinal hernia to acceptable levels, thus centering the attention on Quality of Life as a pivotal postoperative outcome. Carolinas Comfort Scale is a well-studied questionnaire designed to identify Quality of Life changes following hernia repair with mesh techniques. The aim of this study is to validate the CCS in Brazilian Portuguese for inguinal hernias. The original CCS was translated into Brazilian Portuguese according to cross-cultural adaptation guidelines. We conducted a cross-sectional study in individuals aged 18 and above who had undergone inguinal laparo-endoscopic hernia repair for at least 6months prior, between January 2019 and August 2022, at a Brazilian tertiary hospital. Participants answered an online survey containing the Brazilian CCS and the generic Patient-Reported Outcome Measure (PROM) Short-Form Health 36 (SF-36). Participants answered the same questionnaires in the follow-up after at least three weeks, with an additional question about satisfaction with surgery results. The survey was completed by 115 patients, of whom 78 (67%) responded to the follow-up questionnaire after 3 to 10weeks. CCS showed excellent internal consistency, with Cronbach's α of 0.94. Intraclass correlation coefficient ranged from 0.60 to 0.82 in the test-retest analysis. Compared to SF-36, a strong correlation was observed in the physical functioning dimension, and a moderate correlation was found in role-physical and bodily pain (Pearson's Coefficient Correlation = 0.502, 0.338 and 0.332 respectively), for construct analysis. The mean CCS score was significantly lower (p < 0.001) among satisfied patients compared to the unsatisfied ones. The Brazilian version of CCS is a valid and reliable method to assess long-term quality of life after inguinal laparo-endoscopic hernia repair.

Evaluating the Population-Based Usage and Benefit of Digitally Collected Patient-Reported Outcomes and Experiences in Patients With Chronic Diseases: The PROMchronic Study Protocol.

Chronic diseases are associated with a high disease burden. Under- and overprovision of care as well as quality variation between health care providers persists, while current quality indicators rarely capture the patients' perspective. Capturing patient-reported outcome measures (PROMs) as well as patient-reported experience measures (PREMs) is becoming more and more important to identify gaps in care provision, prioritize services most valuable to patients, and aid patients' self-management. This study aims to measure the potential benefits and effectiveness of using electronic patient-reported outcome measures (ePROMs) and electronic patient-reported experience measures in a structured and population-based manner to enhance health care for chronic disease patients in Germany. This prospective cohort study aims to evaluate the potential benefits of PROM usage in patients with chronic diseases. We evaluate whether (1) digitally collected PROMs and PREMs can be used for health system performance assessment by generating a representative response of chronically diseased individuals with asthma, chronic obstructive pulmonary disease, diabetes, and coronary artery disease across Germany, and (2) based on the PROMs and PREMs, low-value care can be identified. As patient-reported outcomes (PROs) are rarely presented back to patients, (3) this study also examines patients' reactions to their PROM scores in the form of digital PRO feedback. For these purposes, randomly selected patients from a nationwide German insurer are digitally surveyed with generic and disease-specific PROMs and PREMs, as well as additional questions on their health-related behavior, 4 times over 1 year. Individual PRO feedback is presented back to patients longitudinally and compared to a peer group after each survey period. Patient-reported data is linked with health insurance data. Response rates, changes in health and experience outcomes over time, self-reported changes in health behavior, and health care system usage will be analyzed. The PROMchronic study explores the usage of PROMs in patients with chronic diseases. Data collection began in October 2023, after the initial invitation letter. All the 200,000 potential patients have been invited to participate in the study. Data have not yet been analyzed. Publication of the interim results is planned for the autumn of 2024, and the results are planned to be published in 2025. We aim to fill the research gap on the population-based usage of PROMs and PREMs in patients with chronic diseases and add to the current understanding of PROM data-sharing with patients. The study's results can thereby inform whether a health care system-wide approach to collecting PROMs and PREMs can be used to identify low-value care, assess quality variation within and across chronic conditions, and determine whether PRO feedback is helpful and associated with any changes in patients' health behaviors. German Clinical Trials Register DRKS00031656; https://drks.de/search/en/trial/DRKS00031656. DERR1-10.2196/56487.

Responsiveness and minimal important change of specific and generic patient-reported outcome measures for back patients: the Norwegian Neck and Back Register

PurposeTo evaluate responsiveness and minimal important change (MIC) of Oswestry Disability Index (ODI), pain during activity on a numeric rating scale (NRSa) and health related quality of life (EQ-5D) based on data from the Norwegian neck and back registry (NNRR).MethodsA total of 1617 patients who responded to NNRR follow-up after both 6 and 12 months were included in this study. Responsiveness was calculated using standardized response mean and area under the receiver operating characteristic (ROC) curve. We calculated MIC with both an anchor-based and distribution-based method.ResultsThe condition specific ODI had best responsiveness, the more generic NRSa and EQ-5D had lower responsiveness. We found that the MIC for ODI varied from 3.0 to 9.5, from 0.4 to 2.5 for NRSa while the EQ5D varied from 0.05 to 0.12 depending on the method for calculation.ConclusionIn a register based back pain population, the condition specific ODI was more responsive to change than the more generic tools NRSa and EQ5D. The variations in responsiveness and MIC estimates also indicate that they should be regarded as indicative, rather than fixed estimates.

Validation and correlations of the Angioedema Activity Score (AAS), Angioedema Quality of Life (AE-QoL) questionnaire, and Angioedema Control Test (AECT) in Chinese patients with angioedema

BackgroundThe impact of recurrent angioedema can be severely debilitating and remains difficult to quantify. Several standardized patient-reported outcome measures (PROMs), including the Angioedema Activity Score (AAS), Angioedema Quality of Life Questionnaire (AE-QoL) and Angioedema Control Test (AECT), have been developed and translated into different languages. However, these PROMs have yet to be validated in Chinese and their correlations in the Chinese population remain unknown. ObjectiveTo validate the Chinese AAS, AE-QoL, and AECT and to assess their inter-correlations. MethodsA prospective cohort of 118 Chinese patients with recurrent angioedema at the Angioedema and Urticaria Centre of Reference and Excellence in Hong Kong completed the traditional Chinese versions of the AAS, AE-QoL, and AECT. We analysed the reliability and validity of these PROMs and their correlations with each other and with generic PROMs. ResultsThe Chinese AAS, AE-QoL and AECT demonstrated excellent internal consistency (Cronbach’s α: 0.920, 0.976, and 0.832; McDonald’s ω: 0.972, 0.977, and 0.901; respectively). Confirmatory factor analysis for the AE-QoL showed an acceptable fit to the 4-dimensional model (comparative fit index=0.869, Tucker–Lewis’s index=0.842). The AECT showed significant correlations with both the AAS and AE-QoL (ρ=-0.750 and -0.456 respectively, both p<0.05). The AE-QoL moderately correlated with certain domains of generic PROMs such as WPAI-GH V2.0 and SF-12v2 (all ρ<0.60). ConclusionThe Chinese AE-QoL, AAS, and AECT are valid and reliable tools for Chinese patients. More validated tools should be made available to improve patient care and research for all angioedema patients globally.

Can prediction models for hospital readmission be improved by incorporating patient-reported outcome measures? A systematic review and narrative synthesis.

To investigate the roles, challenges, and implications of using patient-reported outcome measures (PROMs) in predicting the risk of hospital readmissions. We systematically searched four bibliometric databases for peer-reviewed studies published in English between 1 January 2000 and 15 June 2023 and used validated PROMs to predict readmission risks for adult populations. Reported studies were analysed and narratively synthesised in accordance with the CHARMS and PRISMA guidelines. Of the 2858 abstracts reviewed, 23 studies met predefined eligibility criteria, representing diverse geographic regions and medical specialties. Among those, 19 identified the positive contributions of PROMs in predicting readmission risks. Seven studies utilised generic PROMs exclusively, eleven used generic and condition-specific PROMs, while 5 focussed solely on condition-specific PROMs. Logistic regression was the most used modelling approach, with 13 studies aiming at predicting 30-day all-cause readmission risks. The c-statistic, ranging from 0.54 to 0.84, was reported in 22/23 studies as a measure of model discrimination. Nine studies reported model calibration in addition to c-statistic. Thirteen studies detailed their approaches to dealing with missing data. Our study highlights the potential of PROMs to enhance predictive accuracy in readmission models, while acknowledging the diversity in data collection methods, readmission definitions, and model evaluation approaches. Recognizing that PROMs serve various purposes beyond readmission reduction, our study supports routine data collection and strategic integration of PROMs in healthcare practices to improve patient outcomes. To facilitate comparative analysis and broaden the use of PROMs in the prediction framework, it is imperative to consider the methodological aspects involved.

Facilitators and Barriers in the Implementation and Adoption of Patient-Reported Outcomes Measurements in Daily Practice

ObjectivesAt the Erasmus Medical Center, patient-reported outcomes measures (PROMs) are implemented on a hospital-wide scale. However, less than half of the patients and healthcare professionals (HCPs) use these PROMs. Therefore, this study aimed to investigate facilitators and barriers for adoption of PROMs to develop guidance around implementation. MethodsA mixed-methods study with a combination of interviews and focus groups and questionnaires was conducted, involving patients, both PROM nonresponders and PROM responders, HCPs, and medicine students and nurse specialists in training (hereafter “students”). Interview transcripts were subjected to thematic content analysis. Subsequently, questionnaires were developed and presented to all stakeholders to validate the findings. Finally, identified themes and implementation recommendations were presented in a final questionnaire to the Value-Based Healthcare Erasmus Medical Center expert group to prioritize findings. ResultsInterviews were conducted with 15 patients, 14 HCPs and 4 students, and 2 focus groups with 5 students. The questionnaire was completed by 370 of 999 responders (37.0%), 173 of 1395 nonresponders (12.5%), and 44 of 194 HCPs (22.7%), and 40 students were reached via an open link. The identified facilitators and barriers were grouped into 4 overarching themes: training on PROMs at different levels in the education of (future) HCPs, motivate and reduce the burden for the HCP, implement generic and disease-specific PROMs simultaneously, and motivate, activate, and reduce the patient burden. ConclusionsProviding end users with digital tools, implementation support, and a clear hospital-wide vision is important, yet this does not guarantee successful adoption of PROMs. Successful adoption necessitates ongoing efforts to engage, motivate, and train end users.

#1030 Addressing the unmet need of patient reported outcomes in ANCA associated vasculitis, in routine clinical practice

Abstract Background and Aims ANCA associated vasculitis (AAV) is associated with increased morbidity. Recent years has seen improvement in clinical outcomes yet little work has been done to better understand the patient's role within this condition. Patient reported outcome measures (PROMs) are increasingly standard in clinical research outcomes, as well as an overall indicator for good quality patient care. With the exception of Robson et al AAV-PRO [1], little research has been done to develop validated disease specific tools for patients living with AAV. We aim to develop a disease specific tool that can be used in clinical practice to aid patients and clinicians in understanding and track changes in health-related quality of life (HRQoL) over time. Method AAV patients from a single centre were invited to take part in focus groups conducted using a semi-structured topic guide. Participants were asked to discuss topics relating to four broad areas; 1) the effects of AAV on everyday life and living, 2) the effects of treatment on everyday life and living, 3) the role of patients in health management and the patient experience 4) a review of existing generic and disease specific PROM tools (SF-36 and AAV-PRO). Audio transcript of each focus group was transcribed verbatim and data was analysed using a combination of inductive and deductive framework analysis [2]. Results Twenty-eight patients with AAV were invited to participate, 19 attended one of three, 2.5 hour focus groups, 2 patients failed to attend, 4 declined and 3 were unavailable. The mean age of participants was 66.6 +9.4 years and there was a near equal male to female split (10:9). The median time from diagnosis as 7.4 years. Three participants had active or newly diagnosed AAV with BVAS scores of &amp;gt;10. Five participants had previously required dialysis with 1 receiving a renal transplant. All participants received glucocorticoid and immunosuppressive therapy (cyclophosphamide and or Rituximab), 15 (78.9%) were still receiving immunotherapy. Full analysis of the qualitative data is being undertaken. Preliminary findings outlined what was most important to participants when considering HRQoL. Areas acknowledged included; self-identity, social roles e.g. fulfilling caring responsibilities, fears of relapse and disease and treatment burden. Fifty-six deductive and inductive codes were identified which have been further categorised. Participants felt existing tools lacked consideration of some key issues. Scoring systems were considered to be negativity worded e.g. mild, moderate, severe, with the term ‘none’ being the only positive option. Participants reported that existing tools overlooked positive factors which impact HRQoL, such as symptom validation, close clinician monitoring and improvement with treatment. Limitations were noted when trying to address transient or inconsistent symptoms and difficulty separating vasculitis from other health conditions when answering questions. Participants expressed a preference for using a PROM as part of their clinical care and felt that completing it prior to clinic would be most helpful and facilitate discussions with healthcare professionals. The use of an electronic-PROM was widely supported and would support clinicians and patients to monitor changes over time. Conclusion The AAV-PRO represents a significant advancement over generic tools such as the SF-36, however, existing PROMs are not without limitations and are primarily used in research practices. Our study revealed that patients desire active participation in HRQoL discussions and further work to incorporate this into the clinic setting is needed. We are continuing to develop a PROM tool that can be used to assess patients HRQoL in relation to their AAV in the outpatient setting. We believe this will allow a deeper understanding of the disease burden and could support a comprehensive, patient-centred approach to decision-making, offering new insights and identifying the need for supportive care. On a broader scale, these insights can be utilized to compare treatments, shed light on inequalities and facilitate enhancements in services.

Unicompartmental knee arthroplasty vs high tibial osteotomy for knee osteoarthritis: A comparison of clinical and radiological outcomes.

Unicompartmental knee arthroplasty (UKA) and high tibial osteotomy (HTO) are well-established operative interventions in the treatment of knee osteoarthritis. However, which intervention is more beneficial to patients with knee osteoarthritis remains unknown and a topic of much debate. Simultaneously, there is a paucity of research assessing the relationship between radiographic parameters of knee joint alignment and patient-reported clinical outcomes, preoperatively and following HTO or UKA. To compare UKAs and HTOs as interventions for medial-compartment knee osteoarthritis: Examining differences in clinical outcome and investigating the relationship of joint alignment with respect to this. This longitudinal observational study assessed a total of 42 patients that had undergone UKA (n = 23) and HTO (n = 19) to treat medial compartment knee osteoarthritis. Patient-reported outcome measures (PROMs) were collected to evaluate clinical outcome. These included two disease-specific (Knee Injury and Osteoarthritis Outcome Score, Oxford Knee Score) and two generic (EQ-5D-5L, Short Form-12) PROMs. The radiographic parameters of knee alignment assessed were the: Hip-knee-ankle angle, mechanical axis deviation and angle of Mikulicz line. Statistical analyses demonstrated significant (P < 0.001), preoperative to postoperative, improvements in the PROM scores of both groups. There were, however, no significant inter-group differences in the postoperative PROM scores of the UKA and HTO group. Several significant correlations associated a more distolaterally angled Mikulicz line with worse knee function and overall health preoperatively (P < 0.05). Postoperatively, two clusters of significant correlations were observed between the disease-specific PROM scores and knee joint alignment parameters (hip-knee-ankle angle, mechanical axis deviation) within the HTO group; yet no such associations were observed within the UKA group. UKAs and HTOs are both efficacious operations that provide a comparable degree of clinical benefit to patients with medial compartment knee osteoarthritis. Clinical outcome has a limited association with radiographic parameters of knee joint alignment postoperatively; however, a more distolaterally angled Mikulicz line appears associated with worse knee function/health-related quality of life preoperatively.

The state of art on the use of patient reported outcomes in migraine.

This review aims to explore the use of patient-reported outcome measures (PROMs) in migraine. Traditionally assessed through specific features, recent adoption of PROMs allows for a more objective and quantifiable evaluation. PROMs, which are standardized questionnaires collecting health information directly from a patients' perspective, cover various aspects, including migraine specific aspects. The review focuses on delineating the applications and interpretation of commonly used PROMs in migraine research, with an emphasis on their integration in clinical care. Generic and migraine-specific PROMs play a crucial role in clinical research, particularly in assessing health-related quality of life, disability, impact, and associated comorbidities. Some of these measures are strongly recommended to be used by the International Guidelines and are, in fact, mandated by the FDA for product labeling. Recently, there has been an expansion in the use of PROMs to assess migraine in diverse populations, in particular pediatric patients. However, the application of these measures in clinical care shows considerable heterogeneity, and some have not been validated specifically for migraine. The existing multitude of PROMs, coupled with ongoing development of new ones to better capture patient concerns, creates complexity in their research and clinical application. To address these challenges, it becomes imperative to streamline their use, focusing on those that are more validated and better aligned with the patients' perspective including different populations' needs. The utilization of PROMs in evaluating migraine enables a more holistic assessment, helps quantify the impact of the disease facilitating change measurement, improves communication between healthcare providers and patients and, guides treatment decisions for improved outcomes. However, the increasing number of PROMs questionnaires, underscores the importance of validating these tools for migraine and, the dynamic nature of the disease makes it relevant to decide with whom, why and when these should be used.

Hip-specific and generic patient-reported outcome measure scores after primary hip replacement are associated with early revision surgery: a national registry study

BackgroundThe ability to efficiently identify patients at higher risk of poor outcomes after joint replacement would enable limited resources for post-operative follow-up to be directed to those with the greatest clinical need. This is particularly important as joint replacement rates continue to grow internationally, stretching health system capabilities. Patient-reported outcome measures (PROMs) are routinely administered in many settings and offer an opportunity to detect suboptimal patient outcomes early. This study aimed to determine whether hip-specific and generic PROM scores are associated with early revision hip replacement within six to 24 months after the primary procedure.MethodsPre-operative and six-month post-operative PROM scores for patients undergoing primary total hip replacement (THR) were obtained from the Australian Orthopaedic Association National Joint Replacement Registry and Arthroplasty Clinical Outcomes Registry National and linked to revision surgery data. Clinically important improvement was defined using anchor-based thresholds. Associations between PROM scores (hip pain, Oxford Hip Score, HOOS-12, EQ-5D-5L, EQ VAS, patient-perceived change, satisfaction) and revision surgery were evaluated using t-tests, chi-square tests and regression models.ResultsData were analysed for 21,236 primary THR procedures between 2013 and 2022. Eighty-eight revision procedures were performed at six to 24 months. Patients who were revised had more back pain and worse HOOS-12 scores pre-operatively but between-group differences were small. Worse post-operative PROM scores (hip pain, Oxford, HOOS-12, EQ-5D-5L, EQ VAS) were associated with early revision, after adjusting for age and sex (p < 0.001 for all analyses). Patient dissatisfaction (relative risk (RR) 10.18, 95%CI 6.01–17.25) and patient-perceived worsening (RR 19.62, 95%CI 11.33–33.98) were also associated with a higher likelihood of revision. Patients who did not achieve clinically important improvement in hip pain, function, or quality of life had a higher revision risk (RRs 2.54–5.64), compared with those who did (reference).ConclusionSix-month hip-specific and generic PROM scores can identify patients at higher risk of early revision surgery. Our data highlight the utility of routine post-operative PROM assessment for signaling suboptimal surgical outcomes.

Systematic review of the patient burden of generalised myasthenia gravis in Europe, the Middle East, and Africa

BackgroundMyasthenia gravis (MG) is a rare autoimmune disease characterised by muscle weakness, and progression from ocular (oMG) to generalised (gMG) symptoms results in a substantial negative impact on quality of life (QoL). This systematic review aimed to provide an overview of the patient burden experienced by people living with gMG.MethodsElectronic database searches (conducted March 2022), supplemented by interrogation of grey literature, were conducted to identify studies reporting patient burden outcomes in patients with gMG in Europe, the Middle East and Africa. Results were synthesised narratively due to the heterogeneity across trials.ResultsIn total, 39 patient burden publications (representing 38 unique studies) were identified as relevant for inclusion in the systematic review, consisting of 37 publications reporting formal patient-reported outcome measures (PROMs), and two publications describing alternative qualitative assessments of patient experience. The studies included a variety of measures including generic and disease-specific PROMs, as well as symptom-specific PROMs focusing on key comorbidities including depression, anxiety, fatigue and sleep disturbance. The findings showed some variation across studies and PROMs; however, in general there was evidence for worse QoL in patients with gMG than in healthy controls or in patients with oMG, and a trend for worsening QoL with increasing MG severity.ConclusionsThis review highlights the importance of considering patient QoL when developing and assessing treatment and management plans for patients with gMG. However, the heterogeneity identified across studies illustrates the need for further representative and well-powered studies in large cohorts administering consistent, validated questionnaires.Trial registrationThe protocol for this systematic review was registered in PROSPERO: CRD42022328444.

Can a generic patient reported outcome measure substitute a condition specific measure at assessing care effectiveness in low back pain?

Quality of care assessments beside the provider level should also include the patient perspective, however, there aren't widespread solutions for this approach. One possibility is to apply a generic, thus, widely applicable Patient Reported Outcomes Measure (PROM) to assess care outcomes. Taking this notion into consideration, this study aimed to investigate if a generic questionnaire can reliably substitute a disease-specific questionnaire when measuring care effectiveness with patient-reported outcome measures among patients with low back pain. Between January and December 2019, we conducted a before and after survey in three Hungarian hospitals. Adults with confirmed low back pain expecting spinal surgery were eligible to enter the study. SF-36 Health Survey (SF-36) and Roland‐Morris Disability Questionnaire (RMDQ) were used. Multivariate linear regression analyses were conducted to explore the relationship between the results of the two questionnaires and how service provider, sex, and education level could explain the differences in the calculated PROM-based performance measures. During the pre-intervention survey, 11 individuals decided to either not sign the consent form or complete the questionnaire. As a result, 116 individuals participated in the first – baseline – survey and 86 in the second – follow-up – survey. The drop-out rate varied by providers: the lowest rate was 15%, and the highest 39%. Of the SF-36 subscale-based performance measures, role limitations due to physical health and physical functioning significantly correlated with the RMDQ performance measure. Considering the necessary minimum clinically important difference, the explanatory analysis showed that the SF-36 physical functioning subscale-based and the RMDQ-based performance measures established the same performance rank-order among the participating hospitals. The physical functioning subscale of the SF-36 provided similar results to the RMDQ regarding care effectiveness. Thus, the SF-36 may be able to measure and compare care effectiveness among providers in low-back pain. If future studies investigating other health conditions come to the same conclusion, then the SF-36 could be used by itself to incorporate the patient perspective into health care quality assessments, thereby increasing comparability and lowering administrative costs.

Comparison of clinical outcomes between total hip replacement and total knee replacement.

Total hip replacements (THR) and total knee replacements (TKR) are effective treatments for severe osteoarthritis (OA). Some studies suggest clinical outcomes following THR are superior to TKR, the reason for which remains unknown. This study compares clinical outcomes between THR and TKR. To compare the clinic outcomes of THR anad TKR using a comprehensive range of patient reported outcome measures (PROMs). A prospective longitudinal observational study of patients with OA undergoing THR and TKR were evaluated using a comprehensive range of generic and joint specific PROMs pre- and post-operatively. A total of 131 patients were included in the study which comprised the THR group (68 patients) and the TKR group (63 patients). Both groups demonstrated significant post-operative improvements in all PROM scores (P < 0.001). There were no significant differences in post-operative PROM scores between the two groups: Hip and Knee Osteoarthritis Outcome scores (P = 0.140), Western Ontario and McMaster Universities Osteoarthritis Index pain (P = 0.297) stiffness (P = 0.309) and function (P = 0.945), Oxford Hip and Knee Score (P = 0.076), EuroQol-5D index (P = 0.386) and Short-Form 12-item survey physical component score (P = 0.106). Subgroup analyses showed no significant difference (P > 0.05) between cruciate retaining and posterior stabilised prostheses in the TKR group and no significant difference (P > 0.05) between cemented and uncemented fixation in the THR group. Obese patients had poorer outcomes following TKR but did not significantly influence the outcome following THR. Contrary to some literature, THR and TKR are equally efficacious in alleviating the pain and disability of OA when assessed using a comprehensive range of PROMs. The varying knee prosthesis types and hip fixation techniques did not significantly influence clinical outcome. Obesity had a greater influence on the outcome following TKR than that of THR.

Patient Acceptable Symptom State Thresholds for the Knee Society Score, Oxford Knee Score, and 36-Item Short Form Survey Ten Years Following Unicompartmental Knee Arthroplasty

BackgroundThe patient acceptable symptom state (PASS) refers to a cutoff value on any patient-reported outcome measures (PROMs) scale, beyond which patients consider themselves as having achieved an acceptable outcome. This study aimed to identify PASS thresholds for knee-specific and generic PROMs at 10 years post-unicompartmental knee arthroplasty (UKA). MethodsThere were 269 patients who underwent UKA for medial osteoarthritis from 2004 to 2007 at a single institution and were surveyed preoperatively and 10 years postoperatively using the Knee Society Function Score (KSFS), Knee Society Knee Score (KSKS), Oxford Knee Score (OKS), 36-Item Short Form Survey (SF-36) Mental Component Score (MCS), and SF-36 Physical Component Score (PCS). Treatment outcomes and expectations were assessed using an anchor question, and PASS attainment was determined using the Youden index on a receiver operating characteristic (ROC) curve. Also, a similar study that identified 2-year long-term PROM PASS thresholds for UKA was referenced and compared. ResultsOverall, 91.1% reported acceptable outcomes. The area under the curve for ROCs of KSKS, OKS, and PCS were 0.80, 0.75, and 0.71, respectively. The area under the curve for ROCs of KSFS and MCS were both 0.64. The PASS thresholds were 67.5 for KSFS, 70.5 for KSKS, 39.5 for OKS, 44.6 for PCS, and 43.8 for MCS. Patients who achieved a PASS were at least 3 times more likely to have satisfactory outcomes. ConclusionsTo our knowledge, this is the first study that identified 10-year long-term PROM PASS thresholds for UKA. Accounting for our finding that a decade-long follow-up yielded lower PASS thresholds, time-specific UKA PROM PASS thresholds should be considered. Level of evidenceIII.

Patient-Reported Outcomes in Venous Thromboembolism: A Systematic Review of the Literature, Current Challenges, and Ways Forward.

Venous thromboembolism (VTE) affects >1.2 million Americans annually. Although the clinical outcomes and economic burdens of VTE have been well described, the impact of VTE on patients' health status has yet to be summarized. This systematic review summarizes how patient-reported outcome measures (PROMs) have been used in VTE to date. PubMed/MEDLINE was queried for literature published through March 2023 using PROMs in a population of patients with VTE. Studies were excluded if the reference was an editorial, review, or case report, or if the study included patients with conditions other than VTE. Qualitative analyses were performed. After screening and exclusion, 136 references were identified; 5 described PROM development, 20 focused on PROM validation, and 111 used PROMs in outcomes research. The most used generic PROMs were the 36-item Short-Form Health Survey and EuroQol 5-dimensional questionnaire, and the most common disease-specific PROMs were the Venous Insufficiency Epidemiological and Economic Study-Quality of Life/Symptoms and the Pulmonary Embolism Quality of Life Questionnaire. PROMs were used to quantify the changes in health status after diagnosis, characterize the trajectory of subsequent improvement, and identify drivers of continued impairments in health status like postthrombotic syndrome and postpulmonary embolism syndrome. PROMs were also used to investigate the impact of novel treatment modalities on quality of life. This review demonstrates the many benefits of PROM use, including quantifying changes in health status with treatment, capturing patients' experiences with the treatment itself, and identifying complications of VTE. Incorporating PROMs into VTE care will be an essential component of evaluating the effectiveness of novel therapies and should lead to improved shared decision-making for patients with VTE.