Patient-reported Outcomes Data Research Articles

Medicine Access Programmes: what do patients think – a patient‐reported outcome study on ribociclib in metastatic breast cancer in Australia

AbstractThis study evaluated patient‐reported outcomes (PROs) of Medicine Access Programmes (MAPs) for Australian metastatic breast cancer patients on ribociclib. Limited patient awareness of MAP enrolment was identified, emphasising the need for improved education and consent processes. Most patients expressed gratitude for accessing non‐funded medications and perceived enhanced medication adherence as a key benefit. Integrating PRO data with real‐world registry data provides comprehensive insight for future MAP development.

Biomarkers of microbial translocation and generalized inflammation are associated with frailty among people with HIV.

Frailty occurs at higher rates and younger ages among people with HIV (PWH) compared to the general population and is often attributed to chronic inflammation and subsequent immune exhaustion. We assessed how inflammatory biomarkers are associated with frailty among PWH. The Centers for AIDS Research Network of Integrated Clinical Systems (CNICS) cohort is comprised of adult PWH in care at 10 sites, and harmonizes demographic, clinical, and patient-reported outcomes (PRO) data. A panel of 13 inflammatory biomarkers was collected from a subset of virally suppressed PWH once per person between 2010-2018. Frailty was measured with a validated PRO phenotype, scored 0-4, from biomarker collection date through July 2022. With adjusted linear mixed models, we estimated longitudinal associations between standard deviation-scaled log2-transformed biomarkers and frailty score. Among 273 PWH, most were male (91%), average age at baseline was 45, 42% were non-Hispanic White while 35% were non-Hispanic Black, and average follow-up time was 5.5 years. Several biomarkers were associated with higher frailty, including those linked to microbial translocation (sCD14, LBP, KT ratio) and systemic inflammation (CRP, IL-6, suPAR, sTNFR1, sTNFR2). Higher IL-6 was associated with a 0.25-point higher frailty score (95%CI:0.12-0.39). Higher sTNFR1 (0.35 [0.13-0.56]), sCD14 (0.21 [0.11-0.31]), and suPAR (0.24 [0.11-0.36]) levels were also associated with higher frailty scores over follow-up. Higher levels of biomarkers linked to microbial translocation and systemic inflammation are associated with higher average frailty scores over time in a cohort of virally suppressed PWH, highlighting these pathways as potential interventional targets for mitigating frailty in PWH.

Comparison of Patient-Reported Outcomes after Local Flap Coverage versus Amputation for Complex Lower Extremity Trauma

Abstract Background There is a paucity of patient-reported outcomes (PROs) data in lower extremity salvage. Limb salvage can often be achieved with the use of local muscle flaps or fasciocutaneous flaps. The purpose of this study was to compare PROs of patients who underwent lower extremity salvage using local fasciocutaneous flaps or muscle flaps to lower extremity amputation. Materials and Methods The outcomes of 61 patients that underwent lower extremity local flap reconstruction (n = 33) or amputation (n = 28) between 2014 and 2020 were recorded. Chart reviews were performed to collect perioperative data. Patients were contacted via telephone for participation in the survey portion of our study. PROs were recorded utilizing both the Lower Extremity Functional Scale (LEFS) and the 36-Item Short-Form Health Survey (SF-36). Results Surveys were completed by 61 patients (response rate 59.2%). The mean time of survey after flap reconstruction or amputation was 2.7 ± 1.4 years. Recent trauma (within 90 days) was the most common indication for local flap coverage (n = 23). LEFS score and SF-36 physical functioning scores were significantly lower in patients who underwent muscle flaps compared with fasciocutaneous flaps (p = 0.021 and p = 0.022). Muscle flap patients had similar LEFS and SF-36 scores to amputation patients, while fasciocutaneous flap patients had significantly higher LEFS (p = 0.01), SF-36 physical functioning (p = 0.031), physical role functioning (p = 0.031), and emotional role functioning (p = 0.047) scores than amputation patients. Conclusion Patients who underwent local fasciocutaneous flaps for limb salvage reported higher PRO scores than those undergoing amputation, while patients undergoing muscle flaps reported outcomes similar to those undergoing amputation. PROs for muscle flap patients were significantly lower than those of fasciocutaneous flap patients. These data suggest that while fasciocutaneous and muscle flaps are both useful limb salvage procedures, fasciocutaneous flaps may confer advantages that result in improved patient-perceived outcomes. Further study is needed to better characterize outcomes in limb salvage.

Comparison of statistical methods for the analysis of patient-reported outcomes in randomised controlled trials: A simulation study.

Patient-reported outcomes(PROs) that aim to measure patients' subjective attitudes towards their health or health-related conditions in various fields have been increasingly used in randomised controlled trials(RCTs). PRO data is likely to be bounded, discrete, and skewed. Although various statistical methods are available for the analysis of PROs in RCT settings, there is no consensus on what statistical methods are the most appropriate for use. This study aims to use simulation methods to compare the performance (in terms of bias, empirical standard error, coverage of the confidence interval, Type I error, and power) of three different statistical methods, multiple linear regression(MLR), Tobit regression (Tobit), and median regression (Median), to estimate a range of predefined treatment effects for a PRO in a two-arm balanced RCT. We assumed there was an underlying latent continuous outcome that the PRO was measuring, but the actual scores observed were equally spaced and discrete. This study found that MLR was associated with little bias of the estimated treatment effect, small standard errors, and appropriate coverage of the confidence interval under most scenarios. Tobit performed worse than MLR for analysing PROs with a small number of levels, but it had better performance when analysing PROs with more discrete values. Median showed extremely large bias and errors, associated with low power and coverage for most scenarios especially when the number of possible discrete values was small. We recommend MLR as a simple and universal statistical method for the analysis of PROs inRCT settings.

Patient-reported outcomes in cancer survivorship: insights from two decades of population-based PROFILES registry research.

When the field of cancer survivorship research was in its infancy, the PROFILES registry was set up in 2004 to monitor patient-reported outcomes (PROs) in survivors and a normative population. This scoping review aims to summarize lessons learned from developing a population-based PRO registry, focusing on study methodologies, data collection shifts, data utilization, multidisciplinary collaboration, societal impact, and data sharing. A systematic computerized literature search through PubMed was performed to collect all publications using data from the PROFILES registry between January 1, 2004, and December 31, 2023. The PROFILES registry's research today encompassed 249 papers from 35 studies. Key insights include the importance of multi-hospital collaboration, which enhances participant inclusion and result generalizability. Optimizing response rates and patient inclusion is achieved through proactive data collection methods such as inclusion by health care professionals, and using both web-based and paper questionnaires. Longitudinal studies, despite their intensive data collection efforts, provide critical insights into the consequences of cancer and its treatment on patient-reported outcomes (PROs) from diagnosis through survivorship. Combining PRO data with comprehensive clinical registry data ensures reliable datasets, crucial for drawing meaningful conclusions. The shift towards multidisciplinary collaboration, open-access publishing, and data sharing all contribute to accessible and impactful research. This review highlights key insights from the PROFILES registry, emphasizing multi-hospital collaboration, proactive data collection, and the integration of PROs with clinical data. These lessons can guide future research on cancer survivorship, improving methodologies to enhance survivorship care and quality of life through multidisciplinary collaboration and data sharing.

7377 CAM2029, Octreotide Subcutaneous Depot, Improves Patient-Reported Outcomes From Standard-of-Care Baseline in Patients With Acromegaly: Interim Results From a Phase 3 Study (ACROINNOVA 2)

Abstract Disclosure: D. Ferone: Consulting Fee; Self; Camurus AB, Ipsen, Novartis-AAA, Recordati. Grant Recipient; Self; Camurus AB, Ipsen, Novartis-AAA, Recordati. Research Investigator; Self; Camurus AB. P. Maffei: Research Investigator; Self; Camurus AB. J. Silverstein: Consulting Fee; Self; Xeris Pharmaceuticals. Research Investigator; Self; Camurus AB. J.L. Spencer-Segal: Advisory Board Member; Self; Camurus AB. Research Investigator; Self; Camurus AB. A. Gilis-Januszewska: None. M. Doknic: Speaker; Self; Pfizer, Inc., Novo Nordisk, Merck, Sandoz. P. Kadioglu: None. P. Freda: Research Investigator; Self; Camurus AB. L. Katznelson: Advisory Board Member; Self; Camurus AB, Recordati. G. Fidan Yaylali: None. M. Harrie: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. A. Svedberg: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. A.M. Pedroncelli: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. F. Tiberg: Employee; Self; Camurus AB. Stock Owner; Self; Camurus AB. Introduction: Standard-of-care (SoC) therapies for acromegaly typically require healthcare provider administration and can pose a significant treatment burden. CAM2029 is a novel subcutaneous octreotide depot designed for convenient once-monthly self-administration as a ready-to-use syringe or injection pen. In a 24-week Phase 3 trial (ACROINNOVA 1), CAM2029 achieved superior insulin-like growth factor 1 (IGF-1) response vs placebo in acromegaly patients (pts) previously controlled with SoC (octreotide LAR/lanreotide Autogel). We report patient-reported outcome (PRO) data from an interim analysis of a Phase 3, open-label trial of CAM2029 (ACROINNOVA 2; NCT04125836). Methods: ACROINNOVA 2 enrolled pts with acromegaly who completed ACROINNOVA 1 (prior CAM2029 or placebo groups) and new pts with IGF-1 ≤2x upper limit of normal during stable SoC. Pts received once-monthly CAM2029 20 mg for up to 52 weeks (28 weeks for prior placebo pts [week 24−52]). Primary endpoint was adverse events. PROs were assessed using Acromegaly Index of Severity (AIS, 0−18; sum of 6 scores [0−3; none−severe] for headache, sweating, fatigue, joint pain, paresthesia, soft tissue swelling) for symptoms; Acromegaly Quality of Life Questionnaire (AcroQoL, 0−100) for QoL; Treatment Satisfaction Questionnaire for Medication (TSQM, 0−100) and patient satisfaction scale (0−5) for treatment satisfaction; and Self-Injection Assessment Questionnaire (SIAQ v2.0, 0−10) for satisfaction with self-administration. Higher scores signify improvements, except for AIS. Data are shown for the overall population. Results: At data cutoff (May 23, 2023), 54 roll-over pts (prior CAM2029 n=36; prior placebo n=18) and 81 new pts were enrolled; mean CAM2029 exposure was 56.3 weeks. CAM2029 was well tolerated with no unexpected side effects. Mean AIS score improved from baseline SoC to week 52 (−1.2; 95% CI: −1.8, −0.7); estimated proportion of pts with any acromegaly symptoms reduced by 15.1% (95% CI: −23.9, −6.3). At week 52 vs baseline: mean total AcroQoL increased by 3.5 (95% CI: 1.3, 5.7); mean TSQM increased for ‘convenience’ by 15.6 (95% CI: 11.8, 19.3), ‘effectiveness’ by 6.3 (95% CI: 2.8, 9.8), ‘satisfaction’ by 4.7 (95% CI: 1.1, 8.4), and for ‘side effects’ by 2.4 (95% CI: −2.0, 6.8). Mean patient satisfaction score at week 52 was 4.1 (95% CI: 3.9, 4.3); 32.6% and 19.3% of pts reported a ‘much better’ or ‘slightly better’ experience, respectively, with CAM2029 vs prior SoC. Mean SIAQ increased at week 48 vs pre-treatment, ‘satisfaction with way of taking medication’ by 1.6 (95% CI: 0.9, 2.2) and ‘feelings about injections’ by 0.8 (95% CI: 0.3, 1.3). Conclusion: CAM2029 improved symptom burden, QoL, treatment convenience, and patient satisfaction vs baseline SoC, including in pts with stable or controlled disease at baseline, supporting the clinical benefit of CAM2029 and its potential to address unmet needs for pts with acromegaly. Presentation: 6/2/2024

Registry-based study comparing health-related quality of life between patients with primary rectal cancer and locally recurrent rectal cancer

AimNational clinical registries offer the benefits of a comprehensive dataset, particularly when linked with patient-reported outcome (PRO) data. This aim of this study was to utilise UK registry data to assess cross-sectional differences in health-related quality of life (HrQoL) in patients with primary rectal (PRC) and locally recurrent rectal cancer (LRRC). Materials and methodsData were extracted from the COloRECTal cancer Repository (CORECT-R) and the Locally Recurrent Rectal Cancer – Quality of Life (LRRC-QoL) datasets. Propensity score matching was undertaken in a 1:1 ratio using two covariates: age and sex. The primary outcome was the FACT-C Colorectal Cancer Subscale (CCS). Statistical significance was determined using p < 0.05 and clinical significance using effect size (ES) and minimally important clinical difference (MCID). ResultsA matched cohort with 72 patients in each group was identified. Overall FACT-C CCS scores were worse in patients with LRRC from a statistical (11.80 vs 18.03, p < 0.001) and clinically meaningful perspective (ES 1.63, MCID 6.23). Patients with PRC reported better digestion (p < 0.001, ES 0.85), better control over their bowels (p < 0.001, ES 1.03) and increased appetite (p < 0.001, ES 1.74, MCID 2.08). Patients with LRRC reported worse stomach swelling (p < 0,001, ES 0.97) and more diarrhoea (p < 0.001, ES 0.92), however they reported better body image (p < 0.001, ES 0.80). ConclusionPatients with LRRC reported significantly worse overall scores in the FACT-C CCS from both a statistical and clinical perspective, demonstrating the ability of the FACT-C to distinguish between these patient groups and the benefits of the inclusion of PROs within colorectal cancer registries, specifically including patients with advanced/recurrent disease.

Influence of Social Deprivation on Patient-Reported Outcomes in Foot and Ankle Patients.

The impact of social health on patient-reported outcomes (PROs) is gaining increasing attention within the orthopaedic community. Few studies have explored any relationship between social deprivation levels and PROs in orthopaedic foot and ankle patients. We retrospectively identified patients who presented to an orthopaedic foot and ankle clinic for new evaluation. Patients completed PROs including PROMIS physical function (PF), PROMIS pain interference (PI), and the Foot and Ankle Ability Measure (FAAM). Social deprivation was measured using the Area Deprivation Index (ADI), a metric that incorporates various domains of poverty, education, housing, and employment. The ADI score quantifies the degree of social deprivation based on the 9-digit home zip code but is not a specific measure to an individual patient. Briefly, a lower ADI indicates less deprivation whereas a higher score denotes greater deprivation. Patient characteristics and outcomes were summarized and stratified by the nationally defined median ADI. Multivariable linear regression models assessed the relationships between PROs and continuous ADI controlling for demographics (age, sex, race/ethnicity, marital status, and employment status). Our cohort consisted of 1565 patients with PRO and appropriate zip code data. Patients in the most-deprived median ADI split had more pain (median PROMIS-PI 62.7 vs 61.2, P = .001) and less function (median PROMIS-PF 37.1 vs 38.6, P = .021) compared with the least-deprived median ADI split. The clinical significance of these findings is unclear, though, given the minimal differences between groups for PROMIS measures. There was no relationship between ADI and FAAM scores. More socially deprived patients presented to the clinic with marginally less function and greater pain. Although statistically significant, the clinical significance of these relationships is unclear and merits further exploration. We plan to continue to study the connection between social deprivation and patient outcomes in specific clinical conditions as well as before/after surgical interventions. Level IV, retrospective cases series.

Enhancing pediatric oncology clinical trials through patient reported outcomes (PROs)

Survival rates of children with cancer have significantly increased over the last decades in high income countries. However, cancer survivors often face significant long-term adverse effects on physical, psychosocial, and neurocognitive health. The use of Patient-Reported Outcomes (PROs) in clinical trials may help to further optimize treatment regimens to minimize acute and late adverse events. Despite clear recommendations of regulatory agencies, the use of PROs in pediatric clinical trials remains limited. In this article, we discuss the rationale to assess PROs in pediatric clinical trials with a specific highlight on electronic PRO (ePRO) assessment. In addition, we underscore the importance of recent international guideline developments such as the Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL-IMI) and the CONsolidated Standards of Reporting Trials – Patient-Reported Outcomes (CONSORT-PRO) to improve the robustness of PRO data assessment in pediatric oncology, thus facilitating better patient care and more accurate assessments of treatment effects. Best practice examples for the use of modern technologies in ePRO assessments are presented and potential barriers and challenges are discussed. We conclude that – despite some challenges and barriers – the routine assessment of (e)PROs in pediatric clinical trials has the potential to substantially improve the quality of the trials and facilitate the usability of outcome data. We call for a joint effort to take proactive steps to incorporate PROs into clinical trials in pediatric oncology, thus giving children a voice.

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.

Systematic literature review of the humanistic and economic burden of focal epilepsy and primary generalized tonic-clonic seizures in adults.

This systematic literature review (SLR) assessed the humanistic and economic burden of focal epilepsy and primary generalized tonic-clonic seizures (PGTCS) in adults to evaluate these domains in both populations and identify evidence gaps to inform future research. A search was conducted on December 7, 2022, using MEDLINE and Embase to identify studies published from 2012 onwards reporting humanistic burden (patient-reported or caregiver-reported outcomes or utilities, qualitative evaluations), economic burden (productivity loss, caregiver and societal costs of epilepsy), and sleep-related outcomes. Of the 2830 citations identified, 136 were included. Most studies were in the focal epilepsy population; very few studies reported outcomes in the PGTCS population. The presence of epilepsy-specific instruments varied based on the domain evaluated. Epilepsy exerted considerable humanistic and economic burden. Indicators of poor disease control (e.g., high seizure frequency, resistance to anti-seizure medications, polypharmacy) increased epilepsy burden. Seizure frequency and type, disease severity, and polypharmacy also affected work productivity. Adults with epilepsy, particularly focal epilepsy, reported higher indirect costs, more sick days accrued, and early entry into retirement. Caregivers similarly reported high productivity loss and absenteeism related to caregiving duties. The results of this SLR highlight the high humanistic and economic burden of focal epilepsy and PGTCS, although limited data were available for the PGTCS population. The results include patient-reported outcome data specific to focal epilepsy and PGTCS, expanding the limited humanistic burden evidence identified in previous reviews, and show the effect of poor disease control on individuals' lives and as a driver of indirect costs. PLAIN LANGUAGE SUMMARY: Our systematic literature review identified studies that evaluated the impact of focal epilepsy and primary generalized tonic-clonic seizures on patients and their caregivers. We found that focal epilepsy negatively impacted patients' mental health and sleep and was associated with higher indirect costs and lower work productivity in people with more severe disease. The impact of primary generalized tonic-clonic seizures on patients was rarely reported, and future research is needed.

Real-world data on tolerability of COVID-19 vaccination in patients with rheumatoid arthritis based on patient-reported outcomes.

To assess tolerability of COVID-19 vaccination in patients with RA and controls based on patient-reported outcomes (PROs). In total, 266 study participants were included at 6 ± 1 weeks after their second vaccination (BioNTech/Pfizer (72.2%), AstraZeneca (18.8%) and Moderna (9.0%)). In a cross-sectional, observational study design, PRO data were recorded regarding both total and symptom-level tolerability. Overall tolerability was very high according to the patients' self-assessment scores (1.71 for the first and 1.72 for the second vaccination, 6-point Likert scale [1 (very good) to 6 (very poor)]) and did not differ significantly between patients with RA (n = 204) and controls (n = 62). Self-rated overall tolerability regarding first vaccination was significantly better (P = 0.002) in patients receiving mRNA vaccines (n = 193, mean tolerability 1.59) as compared with vector-vaccinated patients (n = 73, mean tolerability 2.04). Homologous or heterologous vaccination regimens had no statistically significant effect on vaccine tolerability (P = 0.131). Reservations about the vaccination were rare (6.4% for the first and 6.0% for the second vaccination) but significantly associated with poorer overall tolerability (P < 0.001) and significantly reduced willingness to recommend vaccination to others (P < 0.001 for the first and P = 0.004 for the second vaccination). Based on these real-world data, tolerability of COVID-19 vaccination was very good in both RA patients and controls. Reservations against COVID-19 vaccination were rare overall, but if present, associated with a significantly worse tolerability and a significantly lower degree of recommendation.

Patient-reported outcomes and treatment adherence in type 2 diabetes using natural language processing: Wave 8 of the Observational International Diabetes Management Practices Study.

We analyzed patient-reported outcomes of people with type 2 diabetes to better understand perceptions and experiences contributing to treatment adherence. In the ongoing International Diabetes Management Practices Study, we collected patient-reported outcomes data from structured questionnaires (chronic treatment acceptance questionnaire and Diabetes Self-Management Questionnaire) and free-text answers to open-ended questions to assess perceptions of treatment value and side-effects, as well as barriers to, and enablers for, adherence and self-management. Free-text answers were analyzed by natural language processing. In 2018-2020, we recruited 2,475 patients with type 2 diabetes (43.3% insulin-treated, glycated hemoglobin (HbA1c) 8.0 ± 1.8%; 30.9% with HbA1c <7%) from 13 countries across Africa, the Middle East, Europe, Latin America and Asia. Mean ± standard deviation scores of chronic treatment acceptance questionnaire (acceptance of medication, rated out of 100) and Diabetes Self-Management Questionnaire (self-management, rated out of 10) were 87.8 ± 24.5 and 3.3 ± 0.9, respectively. Based on free-text analysis and coded responses, one in three patients reported treatment non-adherence. Overall, although most patients accepted treatment values and side-effects, self-management was suboptimal. Treatment duration, regimen complexity and disruption of daily routines were major barriers to adherence, whereas habit formation was a key enabler. Treatment-adherent patients were older (60 ± 11.6 vs 55 ± 11.7 years, P < 0.001), and more likely to have longer disease duration (12 ± 8.6 vs 10 ± 7.7 years, P < 0.001), exposure to diabetes education (73.1% vs 67.8%, P < 0.05), lower HbA1c (7.9 ± 1.8% vs 8.3 ± 1.9%, P < 0.001) and attainment of HbA1c <7% (29.7% vs 23.3%, P < 0.01). Patient perceptions/experiences influence treatment adherence and self-management. Patient-centered education and support programs that consider patient-reported outcomes aimed at promoting empowerment and developing new routines might improve glycemic control.

Mental health provider perspectives on a mobile health application to support remote measurement-based care: Challenges and impacts.

Measurement-based care (MBC) comprises collecting patient-reported outcomes data using validated assessments and using that information to support treatment. The Veterans Health Administration (VHA) has developed technology platforms to support MBC, including the Mental Health Checkup (MHC) mobile health application (app). Our objective was to examine VHA mental health provider perspectives on the MHC app. We completed a mixed-methods, sequential explanatory evaluation of MHC. We surveyed 284 VHA mental health providers who used MHC, then conducted semistructured telephone interviews with a purposefully selected subset of survey respondents (n = 20). Approximately half of survey respondents agreed that MHC allowed them to collect assessment data from veterans more frequently than before (51%) and that they more frequently discussed assessment results with veterans because of MHC (50%) and used those results to inform goal-setting discussions (50%) and treatment decision making (51%). Bivariate analyses indicated a positive relationship between frequency of MHC use and the aforementioned impacts on care. Interview data conveyed both advantages (e.g., increased treatment efficiency, improved treatment decision making) and challenges (e.g., limited assessment availability, difficulties engaging veterans in completing assessments through the app) to using MHC. This evaluation demonstrated how MHC supported providers working to implement MBC. The app enhanced their ability to reach and engage veterans and incorporate assessment data into clinical encounters. Still, many did not perceive that MHC was impactful on mental health care delivery; given that providers who used MHC more frequently reported more positive impressions of MHC, this may be related to how frequently they used the app. (PsycInfo Database Record (c) 2024 APA, all rights reserved).

Comparable Real-World Patient-Reported Outcomes Data Across Health Conditions, Settings, and Countries: The PROMIS International Collaboration

The patient voice is critical to achieving value-based care, improving health outcomes, and advancing medical research. However, a key challenge is how to translate this “voice” into scientifically valid data that can inform evidence-based clinical decisions. One of the biggest barriers is the sheer variety of available patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs) and the associated challenges of translation, validation, implementation, and interpretation, making it difficult to obtain valid and comparable health outcomes. The authors present a harmonized global approach to international standardization of PROs and PROMs. This approach has the potential to accelerate patient-centered care by facilitating the collection of accurate and comparable real-world evidence on health outcomes that matter most to patients. This proposed approach consists of two elements: a data collection process based on a common set of PROs and a state-of-the-art measurement approach based on item response theory. First, there is growing evidence that outcomes such as pain, fatigue, anxiety, depression, sleep disturbance, physical function, and the ability to participate in social roles and activities are relevant for most people, irrespective of their health condition. Measuring these outcomes routinely in all patients could increase outcome comparability and utility for a range of stakeholders. Second, a measurement strategy based on a state-of-the-art psychometric approach — using item response theory (IRT)-based item banks — offers short, flexible, sustainable, and universally applicable PROMs with robust measurement properties and a common measurement scale. The unique integration of these two elements offers the potential to collect comparable PROM data across patients and providers to support shared decision-making, which may lead to better outcomes. The Patient-Reported Outcomes Measurement Information System (PROMIS) is a globally used example of such an approach. The PROMIS Profile measures serve as a resource for measuring a harmonized core set of PROs across medical conditions, languages, and countries. To meet the United Nations Sustainable Development Goal of ensuring healthy lives and promoting well-being for all, at all ages, a collaborative effort is needed to achieve consensus on international standardization of PROs and PROMs to accelerate patient-centered care across health conditions, settings, and countries. The authors propose to routinely measure a core set of broadly relevant PROs in all patients, regardless of their health condition, with universally applicable IRT-based PROMs.

Item Response Theory Quantifies the Relationship Between Improvements in Serum Phosphate and Patient-Reported Outcomes in Adults With X-Linked Hypophosphatemia.

Burosumab is indicated for treatment of a rare bone disease, X-linked hypophosphatemia (XLH). The aim of this analysis was to evaluate the relationship between a treatment response biomarker and patient-reported outcomes (PROs). Longitudinal data for PROs were obtained from adults with XLH from a phase III study. Individual rich time profiles of the biomarker, serum phosphate were simulated using a prior population pharmacokinetic-pharmacodynamic model to calculate serum phosphate exposure metrics for each 28-day treatment cycle, which were then merged with PROs data. Item response theory parameters were first estimated to map a latent variable, ψ, that is, disability score, relative to baseline. Next, the relationships between serum phosphate exposures and ψ were modeled using a nonlinear mixed-effect (NLME) modeling approach. A combined item response theory-NLME model with average serum phosphate as a predictor of ψ described PROs data well. The model estimates suggested 28%, 31%, and 25% reduction in Western Ontario and McMaster Universities Osteoarthritis Index, brief pain inventory, and brief fatigue inventory scores, respectively, with every unit increase in average serum phosphate from the lower limit of normal (2.5 mg/dL). Additionally, a time effect of ~ 0.08% improvements each week was estimated. The analysis suggested that burosumab treatment-induced improvements in serum phosphate levels are associated with improvements in PROs in adults with X-linked hypophosphatemia. The analyses confirmed the importance of prolonged serum phosphate level correction in adult patients with XLH. These results can be useful to guide the design of further studies and to design treatment optimization strategies.

A Hybrid Markov-SPC Approach to Assess Cost Differences in Urgent Care Utilization Using Patient-Reported Data in Inflammatory Bowel Disease.

Cost is a key outcome in quality and value, but it is often difficult to estimate reliably and efficiently for use in real-time improvement efforts. We describe a method using patient-reported outcomes (PROs), Markov modeling, and statistical process control (SPC) analytics in a real-time cost-estimation prototype designed to assess cost differences between usual care and improvement conditions in a national multicenter improvement collaborative-the IBD Qorus Learning Health System (LHS). The IBD Qorus Learning Health System (LHS) collects PRO data, including emergency department utilization and hospitalizations from patients prior to their clinical visits. This data is aggregated monthly at center and collaborative levels, visualized using Statistical Process Control (SPC) analytics, and used to inform improvement efforts. A Markov model was developed by Almario et al to estimate annualized per patient cost differences between usual care (baseline) and improvement (intervention) time periods and then replicated at monthly intervals. We then applied moving average SPC analyses to visualize monthly iterative cost estimations and assess the variation and statistical reliability of these estimates over time. We have developed a real-time Markov-informed SPC visualization prototype which uses PRO data to analyze and monitor monthly annualized per patient cost savings estimations over time for the IBD Qorus LHS. Validation of this prototype using claims data is currently underway. This new approach using PRO data and hybrid Markov-SPC analysis can analyze and visualize near real-time estimates of cost differences over time. Pending successful validation against a claims data standard, this approach could more comprehensively inform improvement, advocacy, and strategic planning efforts.

Patient-reported outcomes and daily activity assessed with a digital wearable device in patients with paroxysmal nocturnal hemoglobinuria treated with ravulizumab: REVEAL, a prospective, observational study

BackgroundParoxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder. Symptoms such as fatigue can have a substantial impact on patients’ physical activity levels, sleep, quality of life, and work productivity. Ravulizumab treatment can reduce thrombosis risk, improve survival and quality of life, and reduce fatigue in PNH, but information is limited on how it impacts sleep and physical activity. Here, data on resting heart rate, daily physical activity, and sleep in ravulizumab-treated patients with PNH were passively collected via a digital wearable activity-tracking device and patient-reported outcome (PRO) data were collected via weekly surveys in the same cohort.MethodsREVEAL was a 32-week prospective observational cohort study in individuals with PNH receiving ravulizumab in the USA. A wrist-worn Fitbit™ collected data on resting heart rate, daily step count, and sleep duration from eligible patients. Patients also completed the following electronic weekly surveys: Functional Assessment of Chronic Illness Therapy (FACIT) – Fatigue, Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health, PROMIS Global Mental Health, PROMIS Sleep-Related Impairment and Sleep Disturbance, and Work Productivity and Activity Impairment Questionnaire – Specific Health Problem (WPAI-SHP). Data collected from the activity trackers and surveys were compared against US general population values reported in the literature.ResultsTwenty-eight ravulizumab-treated patients were included (median age: 34 years; 54% female). PRO scores were within US general population normative values, including FACIT-Fatigue (40.0), PROMIS Global Physical Health (51.0), Global Mental Health (51.0), Sleep-Related Impairment (50.0), and Sleep Disturbance (49.0). Similarly, mean resting heart rate (67 bpm), daily step count (7476), and sleep duration (7.7 h) were within the range of US general population values. Daily step count was positively correlated with PROMIS Global Physical and Mental Health scores.ConclusionsThis was the first study to use digital monitoring technology to collect data on physical activity and sleep in patients with PNH. The findings indicate that ravulizumab treatment enables patients with PNH to achieve activity levels (heart rate, sleep duration, step count) and quality of life that are comparable to those of the US general population. A weak positive correlation was identified between patient-reported physical and mental health and daily physical activity levels.

Inflammatory Bowel Disease-Associated Arthritis Is Associated with Concomitant Autoimmune and Inflammatory Disorders.

Extraintestinal Manifestations (EIMs) are a common and potentially debilitating complication of Inflammatory Bowel Diseases (IBD), sometimes requiring additional treatment beyond those used to control intestinal disease. IBD-associated arthritis (IAA), a form of spondyloarthritis, is associated with several factors including disease location, sex, and IBD type. However, much remains unknown about other clinical factors predicting development of EIMs. Our goal was to identify additional factors associated with IAA. Participants in the LOCATION-IBD cohort were included in this analysis. We performed univariate and multivariate analysis of demographics, clinical data, and patient-reported outcomes data. The LOCATION-IBD cohort included 182 participants with (n = 53) and without (n = 110) joint EIMs and with joint pain of unclear etiology (n = 19). In a multivariate analysis comparing those with and without joint EIMs, female sex (OR = 2.5, p = 0.014), the presence of concomitant autoimmune and inflammatory disorders (OR = 2.5, p = 0.038), and Crohn's disease (OR = 2.9, p = 0.026) were associated with the presence of joint EIMs. This analysis reveals patients with IAA are more likely to have concomitant autoimmune disorders. Further studies are needed to confirm this association, understand the mechanisms underlying the common pathogenesis of these concurrent disorders, and evaluate their impact on the treatment of IAA.

Patient-Reported Outcomes 10 Years After Breast-Conserving Surgery for Early-Stage Breast Cancer.

Patient-reported outcomes (PROs) are a critical component of value-based care. Limited data exist describing long-term PROs in patients undergoing breast-conserving surgery (BCS). Patients undergoing surgery for stage 0-III breast cancer at our institution from 2002 to 2012 who agreed to be contacted were invited to participate in a cross-sectional PRO study. Health-related quality of life outcomes using BREAST-Q, EORTC QLQ-C30, and EORTC QLQ-BR45 were collected. Patients reporting chemotherapy within 6 months of receiving the survey were excluded. For this work, we focused on patients who underwent BCS. Multivariable linear regression was performed to identify factors associated with PRO scores, adjusting for age, time since surgery, anatomic stage, molecular subtype, receipt of systemic and/or radiation therapy (RT), locoregional recurrence, or contralateral breast cancer. Among 562 interested and eligible patients, 437 (78%) responded; median time from surgery to survey completion was 10.4 years (interquartile range: 8.0-13.5). Median age at surgery was 53 years (standard deviation 9.8 years), ≥ 90% were white, had upfront surgery for early-stage disease, and completed adjuvant RT. Physical and psychological well-being scores were generally high, with more variation seen for sexual well-being and satisfaction with breasts. This study provides long-term PRO data for patients treated with BCS, demonstrating the ongoing association of breast cancer surgery with quality of life in the survivorship period and highlighting the importance of examining PROs beyond the perioperative period. These data also provide important reference values for the interpretation of PROs among women treated with BCS as we move towards value-based care.