Patient-centered Outcome Measures Research Articles

Friedreich Ataxia Caregiver-Reported Health Index: Development of a Novel, Disease-Specific Caregiver-Reported Outcome Measure.

The Friedreich ataxia (FRDA) scientific community needs access to patient-centered outcome measures that satisfy regulatory guidelines and are capable of tracking clinically meaningful changes in FRDA disease burden. The objective of this research was to develop a novel, disease-specific caregiver-reported outcome measure for use in FRDA research and clinical care. In prior work, we conducted qualitative interviews and a cross-sectional study of FRDA caregivers and patients to determine the symptoms of greatest importance to individuals with FRDA. We designed the Friedreich Ataxia Caregiver-Reported Health Index (FACR-HI) to serially measure the symptoms of greatest importance to patients and utilized factor analysis, beta testing, reliability testing, and cross-sectional subgroup analysis to further evaluate and optimize this disease-specific outcome measure. The FACR-HI was designed to measure total disease burden and disease burden in 18 symptomatic domains. The FACR-HI total score demonstrated high internal consistency (Cronbach's α = 0.98) and test-retest reliability (intraclass correlation coefficient = 0.96). Beta interview participants found the FACR-HI to be highly relevant, comprehensive, and easy to use. FACR-HI total and subscale scores were associated with functional staging for ataxia scores and speech impairment. Initial evaluation of the FACR-HI supports its content validity, test-retest reliability, and construct validity as a caregiver-reported outcome measure for assessing how pediatric individuals with FRDA feel and function. The FACR-HI provides a potential mechanism to quantify changes in multifactorial FRDA disease burden during future clinical trials.

P.116 Days at home after traumatic brain injury: moving beyond mortality to evaluate patient-centered outcomes using population health data

Background: Despite the utility of administrative health data, there remains a lack of patient-centered outcome measures to meaningfully capture morbidity after traumatic brain injury (TBI). We sought to characterize and validate days at home (DAH) as a feasible measure to assess population-level moderate to severe TBI (msTBI) outcomes and health resource utilization. Methods: We utilized linked health administrative data sources to identify adults with msTBI patients presenting to trauma centers in Ontario injured between 2009-2021. DAH at 180 days reflects the total number of days spent alive and at home excluding the days spent institutionalized in acute care, rehabilitation, inpatient mental health settings or post-acute readmissions. Construct and predictive validity were determined; we additionally estimated minimally important difference (MID) in DAH180days. Results: There were 6340 patients that met inclusion criteria. Median DAH180days were 70 days (interquartile range 0-144). Increased health resource utilization at baseline, older age, increasing cranial injury severity and major extracranial injuries were significantly associated with fewer DAH180days. DAH180days was correlated to DAH counts at 1-3 years. The average MID estimate from anchor-based and distribution-based methods was 18 days. Conclusions: We introduce DAH180days as a feasible and sufficiently responsive patient-centered outcome measure with construct, predictive and face validity in an msTBI population.

#2535 Development of a patient-centered outcome strategy for the CONVINCE trial

Abstract Background and Aims Patient-centered outcomes play an important role when assessing treatment effects. Patient-reported outcome (PRO) measures assessing symptoms and impacts are important tools to capture the patient perspective in clinical trials. However, careful consideration is needed when choosing PROs and PRO measures. Aspects such as the conceptual model, measurement model, validity, reliability, and responsiveness, as well as translation, interpretability of scores and burden for participants and investigators have to be considered when developing a patient-centered outcome strategy. The CONVINCE randomized controlled trial was funded by the European Union (HORIZON2020) to assess effects of high-dose hemodiafiltration (HDF) versus high-flux hemodialysis (HD). The primary outcome is all-cause mortality and one of the secondary outcomes is the experienced health status of the patients as measured by patient-reported outcomes. The aim of this study is to describe the approach for the development of a patient-centered outcome strategy using a domain-based approach that was implemented in the CONVINCE trial. Method The development of the patient-centered outcome strategy for the CONVINCE trial followed three consecutive steps: (1) targeted literature review to identify core outcome sets, as well as concepts and PRO measures in the field of dialysis; (2) semi-structured interviews and focus groups with dialysis patients (n = 13) and clinicians (n = 4) to determine health aspects, i.e. symptoms and impacts most relevant to patient population; (3) triangulation of results to determine the most relevant health domains to assess within the context of the CONVINCE trial and determination of PRO measures in due consideration of psychometric properties of respective instruments. Results We identified 97 PRO measures reported in previous dialysis studies, assessing more than 11 health domains and 41 disease-specific symptoms/problems relevant for dialysis patients. Core outcome sets, such as the SONG-HD and the ICHOM standard set for chronic kidney disease, indicated in total 19 outcomes that we classified as potential PROs. Interviews with patients and clinicians confirmed health domains and outcomes that were identified in the literature and provided further insights in terms of the level of importance of health domains. To balance the comprehensiveness of assessments and respondent burden, we decided to concentrate on 9 domains and 20 symptoms/problems according to the objectives of the trial. For the more proximal outcomes, i.e. recovery time and recovery time pattern, new items were developed by the CONVINCE consortium applying established state-of-the-art methods. For generic outcomes, e.g. fatigue, physical function, we decided to apply psychometrically advanced tools from the PROMIS® initiative, validated within a general population as well as various chronic health conditions. Conclusion Building upon core outcome sets by confirming health concepts most relevant for dialysis patients within the specific context of the CONVINCE trial, enabled us to develop a robust patient-centered outcome measurement strategy for the CONVINCE trial that assesses relevant health domains of dialyses patients in granular fashion using a domain-based approach. Further validation of the PRO measures should be considered, especially establishing meaningful change estimates to support interpretation of change in PRO measurement scores. Conference Abstract ERA EDTA; May 23-26, 2024; Stockholm, Sweden Kathrin I. Fischer, Peter J. Blankestijn, Krister Cromm, Michiel L. Bots, Claudia Barth, Bernard Canaud, Jörgen Hegbrant, Giovanni Strippoli, Andrew Davenport, Mark Woodward, Matthias Rose for the CONVINCE Scientific Committee and CONVINCE Investigators

An International, Multidisciplinary Consensus Set of Patient-Centered Outcome Measures for Substance-Related and Addictive Disorders.

Background: In 1990, the United States' Institute of Medicine promoted the principles of outcomes monitoring in the alcohol and other drugs treatment field to improve the evidence synthesis and quality of research. While various national outcome measures have been developed and employed, no global consensus on standard measurement has been agreed for addiction. It is thus timely to build an international consensus. Convened by the International Consortium for Health Outcomes Measurement (ICHOM), an international, multi-disciplinary working group reviewed the existing literature and reached consensus for a globally applicable minimum set of outcome measures for people who seek treatment for addiction. Methods: To this end, 26 addiction experts from 11 countries and 5 continents, including people with lived experience (n = 5; 19%), convened over 16 months (December 2018-March 2020) to develop recommendations for a minimum set of outcome measures. A structured, consensus-building, modified Delphi process was employed. Evidence-based proposals for the minimum set of measures were generated and discussed across eight videoconferences and in a subsequent structured online consultation. The resulting set was reviewed by 123 professionals and 34 people with lived experience internationally. Results: The final consensus-based recommendation includes alcohol, substance, and tobacco use disorders, as well as gambling and gaming disorders in people aged 12 years and older. Recommended outcome domains are frequency and quantity of addictive disorders, symptom burden, health-related quality of life, global functioning, psychosocial functioning, and overall physical and mental health and wellbeing. Standard case-mix (moderator) variables and measurement time points are also recommended. Conclusions: Use of consistent and meaningful outcome measurement facilitates carer-patient relations, shared decision-making, service improvement, benchmarking, and evidence synthesis for the evaluation of addiction treatment services and the dissemination of best practices. The consensus set of recommended outcomes is freely available for adoption in healthcare settings globally.

Early Quality of Recovery after Elective Cardiothoracic and Aortic Surgeries in Adult Patients: A Pilot Report.

Patient-centered outcome measures should be evaluated postoperatively as the recovery after surgery varies between patients. We aimed to evaluate the feasibility, reliability, and trajectory of the quality of recovery-15 (QoR-15) in patients undergoing cardiothoracic and aortic surgeries. This retrospective study included adult patients who underwent elective cardiothoracic and aortic surgeries. The primary outcome was the QoR-15, with a minimal clinically important difference of 6.8, assessed on postoperative days (POD) 2, 4, and 7. The final analysis included patients with at least one valid outcome. Feasibility and reliability were assessed by the successful completion rate on each POD and using Cronbach's alpha of the QoR-15 on POD 4. A linear mixed model was used to evaluate the trajectory of the postoperative QoR-15 scores. Of the 36 eligible patients, 30 with a mean age of 70 years were included in the final analysis. The successful completion rates on POD 2, 4, and 7 were 72.7%, 87.8%, and 87.8%, respectively. The mean QoR-15 scores on POD 2, 4, and 7 were 89.9, 98.0, and 108.3, respectively. The QoR-15 scores on POD 2 and 4 were not statistically different (P = 0.06) but were clinically significant. The QoR-15 score on POD 7 was statically (P < 0.001) and clinically higher than the QoR-15 score on POD 2. Cronbach's alpha for the QoR-15 score measured on POD 4 was 0.85. The QoR-15 is a feasible and valid measurement after elective cardiothoracic surgery, which increases significantly over time after surgery.

A patient centered assessment of the 2016 ACR-EULAR Myositis Response Criteria: evaluating the meaningfulness of response.

The ACR-EULAR Myositis Response Criteria (Total Improvement Score [TIS]) is a composite measure calculated using changes in myositis core set measures. It is unclear if achieving improvement per TIS reflects improvement in any symptoms of myositis patients. In this study, we examined the association between achieving TIS improvement and patient-centered outcome measures (PCOMs). Adults with myositis were enrolled in a prospective study with baseline and 6-month visits. Six core set measures were collected at each visit along with the following PCOMs: Fatigue (visual analogue scale [VAS] and short form 36 [SF36]), pain (VAS, SF36), health-related quality of life (SF-36), physical function (PROMIS-physical function, SF36, sit-to-stand, timed up-and-go, and six-min walk) and physical activity (actigraphy). Mann-Whitney U was used to compare PCOMs between improvement groups. Spearman correlation and regression models were used for correlation and association between TIS and PCOMs, respectively. Of 50 patients (six polymyositis, 24 dermatomyositis, 9 necrotizing myopathy, 11 anti-synthetase syndrome) enrolled (mean age: 52, 60% female), 21 patients satisfied the TIS improvement criteria at 6-months. PCOMs including fatigue, pain, quality of life, physical activity and physical function demonstrated significantly greater improvement in patients who had minimal TIS improvement compared with those with no improvement. Greater PCOM improvements were seen with moderate-major TIS improvement. TIS correlated moderately-strongly with most PCOMs. Achieving improvement criteria was accompanied by significant clinical improvements in fatigue, pain, health-related quality of life, physical function, and physical activity. These results support the use of TIS as a clinically meaningful metric of improvement.

The impacts of migraine on functioning: Results from two qualitative studies of people living with migraine.

To describe the impact of migraine on functioning based on comprehensive data collection, analysis, and reporting of patients' experiences. Qualitative research conducted to understand patients' perspectives on living with migraine has often focused on narrow topics or specific groups of patients or has been selectively reported. Qualitative interviews with 71 participants were conducted during two concept elicitation studies as part of the Migraine Clinical Outcome Assessment System (MiCOAS) project, an FDA grant-funded program designed to develop a core set of patient-centered outcome measures for migraine clinical trials. Participants self-reported being diagnosed with migraine by a healthcare professional and participated in semi-structured qualitative interviews about their experiences with the symptoms and impacts of migraine. Interview transcripts were coded to identify and define concepts, which were then grouped into broad domains based on conceptual similarities. A total of 66 concepts were identified: 12 for physical functioning, 16 for cognitive functioning, 10 for social role functioning, 19 for emotional and psychological functioning, and 9 related to migraine management. Participants described a complex and varied relationship between migraine attack symptoms and impacts on functioning. Impacts from migraine were further influenced by numerous contextual factors, such as people's individual social environments and the level of day-to-day demand for functioning they face. Findings showed that migraine impacted individual functioning in multiple ways and the nature of these impacts was dependent on social-contextual factors. The results are being used in the development of core measures designed to improve our understanding of the burden of migraine and the efficacy of migraine therapies. The results also offer new insights and raise new questions about migraine experience that can be used to guide future research.

Using normalization process theory to evaluate the use of patient-centred outcome measures in specialist palliative home care—a qualitative interview study

BackgroundStandardised use of patient-centred outcome measures (PCOMs) improves aspects of quality of care. Normalization Process Theory (NPT) considers the social (inter-)actions of implementation processes operationalised through four constructs: coherence-building, cognitive participation, collective action and reflexive monitoring. The aim of the study was to identify barriers and enablers for the successful use of PCOMs in specialist palliative home care (SPHC) using NPT, to collect clinically meaningful and reliable data to improve patient outcomes.MethodsQualitative study using semi-structured interviews with palliative care professionals from German SPHC teams who participated in a study using PCOMs. Data were analysed using Framework analysis, and contextualised within NPT.ResultsSeventeen interviews across five teams were conducted. Some teams already had an understanding of what PCOMs are and how to use them, based on previous experience. In other teams, this understanding developed through the perception of the benefits (coherence). Participation and engagement depended on individuals and was decisive for coherence-building. The attitude of the management level also played a major role (cognitive participation). Integration of PCOMs into everyday clinical practice varied and depended on the manifestation of the first two constructs and other already established routines (collective action). In the context of appraisal, both positive (e.g. focus on patient) and negative aspects (e.g. additional work) of using PCOMs were mentioned (reflexive monitoring).ConclusionsAlthough benefits of using PCOMs were partly recognised, not all teams continued standardised use. Here, not only the social (inter-)actions, but also the influence of the context (working environment) were decisive. Future implementation strategies should consider integrating PCOMs in existing electronic patient records, education sessions supporting coherence-building, internal facilitators/local champions, and ensuring frequent data analyses as it is beneficial and increases the readiness of using PCOMs.

Development and validation of TreatHSP-QoL: a patient-reported outcome measure for health-related quality of life in hereditary spastic paraplegia.

Hereditary spastic paraplegia (HSP) is a rare neurodegenerative disease that lacks specific and validated patient-centered outcome measures (PCOMs). We aimed to develop and validate a health-related quality of life (HRQoL) questionnaire specific to HSP ("TreatHSP-QoL") that could be used as a PCOM. The pilot-items of the TreatHSP-QoL (45 five-level Likert scale items, with values per item between 0 and 4) were developed based on a qualitative data analysis of 54 semi-structured interviews, conducted in person with 36 HSP patients and 18 caregivers. It was then reduced and modified through the validation process to 25 items. The main validation was performed using the online questionnaire in 242 HSP patients and 56 caregivers. The exploratory factor analysis defined five subdomains. Cronbach's alpha ranged from 0.57 to 0.85 for the subdomains and reached 0.85 for the total score. The test-retest Pearson correlation reached 0.86 (95% Confidence Interval (CI) [0.79, 0.91]). Pearson correlations with the EuroQol-5 Dimension (5 levels) (EQ-5D-5L) and Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) questionnaires varied strongly among the subdomains, with the total scores reaching 0.53 (95% CI [0.42, 0.61]) and -0.45 (95% CI [- 0.55, - 0.35]), respectively. The caregiver-patient response Pearson correlation ranged between 0.64 and 0.82 for subdomains and reached 0.65 (95% CI [0.38, 0.81]) for the total score. TreatHSP-QoL can be used in high-quality clinical trials and clinical practice as a disease-specific PCOM (i.e., HRQoL measure) and is also applicable as a proxy questionnaire. Score values between 0 and 100 can be reached, where higher value represents better HRQoL. The Pearson correlations to the EQ-5D-5L and FARS-ADL support the additional value and need of HSP-specific PCOM, while non-specific QoL-assessment and specific clinical self-assessment tools already exist. All in all, the results demonstrate good validity and reliability for this new patient-centered questionnaire for HSP.

Multimodal and Interdisciplinary Interventions for the Treatment of Localized Provoked Vulvodynia: A Scoping Review of the Literature from 2010 to 2023.

Localized provoked vulvodynia (LPV) is a chronic condition characterized by pain in the vulvar vestibule, which can be provoked by pressure or touch and which is not tied to a clear underlying cause. Research into the etiology of and most appropriate treatment strategy for LPV is still limited. Using Arksey and O'Malley's model for scoping reviews, we evaluated the research question: what is the current evidence regarding the efficacy/effectiveness of multimodal or interdisciplinary interventions for the treatment of LPV? We collated and analyzed articles from 2010 to 2023 to capture the current research landscape. Our review identified 27 studies, which either compared treatments between classes (eg pharmacologic versus psychologic modalities) or described interdisciplinary treatment programs. We identify several trends in the literature. First, outcome measures are inconsistent between studies, often unvalidated, and may not adequately mirror patient concerns. Second, the absence of appropriate comparator groups in many studies restricts providers' ability to appraise which treatments may be most efficacious. Third, selection bias and demographic homogeneity limit generalizability. Finally, we highlight the need for head-to-head trials of vestibulectomy with other treatments considered first line for vulvodynia management. There is insufficient evidence to suggest the superiority of one treatment modality for LPV relative to others or to recommend a particular interdisciplinary management strategy. Future research should use a head-to-head design where sham control is impossible, incorporate patient-centered outcome measures, and investigate impacts of treatment among diverse samples of LPV patients.

Are the Newest Anti-Alzheimer’s Medicines Being Evaluated Under Outdated Pricing Paradigms?

The 2021 approval of aducanumab in the United States, was not followed by approvals in Europe, Canada, Australia or elsewhere; nonetheless, the initial pricing of $56,00.00/year created a firestorm of controversy. The 2023 Food and Drug Administration approval of lecanemab has been followed by approvals in Japan and China. The current (2024) costs are $26,500 in USA, $20,438 (National Health Insurance) in Japan and $28,180 (private market) in China. In Europe, a health technology assessment will drive coverage decisions and set single-payer prices for each health system for lecanemab and subsequently approved new treatments. For comparison, 2024 average pricing for the top five selling drugs for cancer [$198,273], arthritis [$83,666], hepatitis C [$75,118] and multiple sclerosis [$86,765] far exceed lecanemab’s annual cost. Could there be an anti-brain-health bias? The International Consortium for Health Outcomes Measurement notes that patient-centered outcome measures represent the ultimate measure of quality and they are always multi-dimensional. Quality-adjusted life-years (QALY) seem to be absent from the computation of pricing for anti-dementia treatments. Although global consensus on QALY calculations remains elusive, currently extant data are compelling. The QALY calculation is the change in utility value induced by the treatment, which is then multiplied by the duration of treatment effect to provide the number of QALYs gained. QALY ranges differ quite significantly, even amongst the most industrialized nations. Value-based pricing from double-blind, placebo-controlled trial results, demonstrating statistically significant beneficial effects on cognition and/or global function, would reward manufacturers for innovation whilst also enabling payer systems to remain solvent. Now that the first truly disease-modifying treatment for Alzheimer’s (lecanemab) has been approved, a second disease-modifying treatment (donanemab) appears to be heading for regulatory approval as well. The full value of these newest anti-dementia treatments may be understated when quantified under older pricing models and, hence, could unwittingly be a disincentive to further innovation. We now have disease-modifying and symptomatic relief treatments for Alzheimer’s disease to be included within the treating clinicians’ armamentarium and, when priced appropriately, access should be widely available whilst also encouraging further innovative anti-Alzheimer’s clinical research.

Embedding Patient Input in Outcome Measures for Long-Term Disease-Modifying Parkinson Disease Trials.

Clinical trials of disease-modifying therapies in PD require valid and responsive primary outcome measures that are relevant to patients. The objective is to select a patient-centered primary outcome measure for disease-modification trials over three or more years. Experts in Parkinson's disease (PD), statistics, and health economics and patient and public involvement and engagement (PPIE) representatives reviewed and discussed potential outcome measures. A larger PPIE group provided input on their key considerations for such an endpoint. Feasibility, clinimetric properties, and relevance to patients were assessed and synthesized. Although initial considerations favored the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III in Off, feasibility, PPIE input, and clinimetric properties supported the MDS-UPDRS Part II. However, PPIE input also highlighted the importance of nonmotor symptoms, especially in the longer term, leading to the selection of the MDS-UPDRS Parts I + II sum score. The MDS-UPDRS Parts I + II sum score was chosen as the primary outcome for large 3-year disease-modification trials. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

PCR85 Facilitating the Identification of Appropriate Patient-Centered Outcome Measures (PCOMS) in Rare Disease (RD) Clinical Research Using Functional Impacts Matching

PCR85 Facilitating the Identification of Appropriate Patient-Centered Outcome Measures (PCOMS) in Rare Disease (RD) Clinical Research Using Functional Impacts Matching

Effect of COVID‐19 and telehealth implementation on Patient Centered Outcome Measures completion by patients with Alzheimer’s and related disorders in an academic memory center

AbstractBackgroundPatient Centered Outcome Measures (PCOMs) are increasingly recognized as a means of improving clinical efficiency, particularly when completed electronically ahead of clinic visits. However, PCOM completion is often far from consistent. The shift to telehealth introduced by the Covid‐19 pandemic changed how patients and caregivers engaged with medical care, possibly encouraging increased participation in electronic assessments. We questioned how the pandemic influenced electronic PCOM completion by our patients with Alzheimer’s disease (AD) and their caregivers.MethodsPatients and caregivers were routinely invited to fill electronic PCOM questionnaires prior to visits both before and during the Covid‐19 pandemic. PCOMs assessed the predominant cognitive domain of concern, other diagnostically useful symptoms, disease severity, neuropsychiatric symptoms, and caregiver wellbeing. A date of 3/15/2020 was set as the onset of the pandemic for this study, after which time our clinic converted to telehealth visits only. We tallied rates of completion of electronic surveys by patients and/or caregivers three and a half months before and after that time, comparing completion rates by two proportion Z‐test.ResultsDuring this timeframe, across a total of 402 visits (total: 333 individuals, 69 returns; AD: 73 individuals, 6 returns), caregivers and/or patients were offered an opportunity to fill PCOMs ahead of the visit. Across all clinical diagnoses combined, the probability of a visit being associated with filled PCOMS was greater after switching to only telehealth (caregivers: P = 0.005, patients: P = 0.016). Additionally, after the shift to telehealth, AD‐related visits were twice as likely to have completed preclinical electronic PCOMs (caregivers: P = 0.002, patients: P = 0.008). Approximately 33.3% of PCOMS to be filled by AD caregivers were completed before the telehealth shift, and 67.5% were afterwards. Approximately 26.7% of patient PCOMs were filled before the change, and afterwards this percentage rose to 55.0%.ConclusionThe beginning of the Covid‐19 pandemic and subsequent shift towards telehealth‐only practice was associated with greater use of electronic PCOMs by all patients, including those with AD and their caregivers. Increased acceptance of electronic PCOMs seems to offer promising opportunities for remote assessment in the future of dementia diagnosis and care.

Developing an Integrated Longitudinal Dataset for Patient-Centered Outcome Measures in Cost-Related Medication Nonadherence.

Cost-related medication nonadherence (CRN) is an important patient-centered outcome measure. Longitudinal follow-up of CRN is rare. We propose to develop a novel integrated dataset to study CRN longitudinally. A dataset of 2000 Medicare beneficiaries at high risk of hospitalization surveyed quarterly on CRN and followed up individually for 8 quarters between 2013 and 2018 was linked to Medicare files. A metric of CRN categorizing persistent, intermittent, and transient CRN during the 8 quarters was developed. An ordered logit model and a logit model were developed to assess the factors influencing CRN overall and persistent CRN, respectively. A total of 1761 patients were included in the analysis, among whom 869 (49.3%) reported CRN at least once in the 8-quarter study period, 178 (10%) reported persistent CRN, 395 (22.4%) reported intermittent CRN, and 296 (16.8%) reported transient CRN. The conditional effect in the logit model for persistent CRN revealed that baseline dual eligibility was negatively associated (adjusted odds ratio = 0.45, P < 0.01) and depression positively associated (adjusted odds ratio = 1.55, P = 0.01) with persistent CRN. The marginal analysis in the ordered logit model revealed a clear pattern of higher probabilities of persistent and intermittent CRN at younger ages while transient CRN was flat. Among the 252 subjects who were deceased, 31 (12.3%) reported persistent CRN, compared with 147 (9.74%) who were alive (P = 0.21 by χ2 test). A significant number of patients reported persistent CRN, including those who were at the end of life. Research is critically needed to understand behavioral patterns among the younger Medicare population.

Implementing patient-centred outcome measures in palliative care clinical practice for adults (IMPCOM): Protocol for an update systematic review of facilitators and barriers.

Background: Despite the development of patient-centred or patient-reported outcome measures (PCOMs or PROMs) in palliative and end-of-life care over recent years, their routine use in practice faces continuing challenges. Objective: To update a highly cited literature review, identify and synthesise new evidence on facilitators, barriers, lessons learned, PCOMs used, models of implementation, implementation outcomes, costs, and consequences of implementing PCOMs in palliative care clinical practice. Methods: We will search MEDLINE, PsycINFO, CINAHL, Embase, Emcare, SCI-Expanded, SSCI, ESCI, and BNI. The database search will be supplemented by a list of studies from the expert advisory committee, hand-searching of reference lists for included articles, and citations of the original review. We will include primary studies using a PCOM during clinical care of adult patients with advanced disease in palliative care settings and extract data on reported models of implementation, PCOMs, facilitators, barriers, lessons learned, costs, and implementation outcomes. Gough's Weight of Evidence Framework will be used to assess the robustness and relevance of the studies. We will narratively synthesise and tabulate the findings. This review will follow PRISMA, PRISMA-Abstract, PRISMA-P, and PRISMA-Search as the reporting guidelines. Source of funding: Marie Curie. The funder is not involved in designing or conducting this study. Protocol registration: CRD42023398653 (13/02/2023).

Patient-reported outcome measures in children, adolescents, and young adults with palliative care needs—a scoping review

BackgroundMeasuring outcomes facilitates evaluation of palliative services for children, adolescents, and young adults (CAYAs) with life-limiting and/or life-threatening (LL/LT) conditions. Implementation of patient-reported, proxy-reported, or patient-centered outcome measures (hereafter PROMs) is recommended to ensure palliative services. The purpose of this scoping review was to provide an overview of PROMs relevant for CAYAs living with LL/LT conditions eligible for pediatric palliative care (PPC).MethodsArksey and O’Malley’s 6-stage scoping review framework was used to guide the review. The identified citations had to report on PROMs in any context including CAYAs with LL/LT conditions up to 25 years of age. A systematic search of Medline, EMBASE, CINAHL, APA PsycInfo, Health and Psychosocial Instruments, and AMED took place in January 2021 and was updated in June 2022. Citations were screened independently by pairs of researchers. The scoping review protocol was registered, and peer-review published.ResultsOf 3690 identified citations, 98 reports were included, of which the majority were from Western countries and about PROMs in CAYAs living with cancer or organ failure. A total of 80 PROMs were identified, assessing a range of phenomena, where quality of life and symptoms (especially pain) during the stage of ongoing care were the most frequent. There were only a few reports about outcome measures at time of diagnosis or in end-of-life care. CAYAs self-reported on the PROMs or collaborated with their parents in about half of the reports, while the remaining had proxies answering on behalf of the CAYAs. In the identified reports, PROMs were used to characterize a sample through cross-sectional or longitudinal research, and less often to assess effects of interventions.ConclusionThe identified PROMs in the CAYA population eligible for PPC is characterized by studies in high-income countries during ongoing care, primarily in patients with cancer or organ failure. More research is needed in patients living with other LL/LT conditions, and during different stages of the disease course, especially at time of diagnosis, during transition to adulthood, and in end-of-life care. This scoping review of PROMs relevant for young patients eligible for PPC may inform future research about patient-/proxy-reported or patient-centered outcome measures in PPC.Trial registrationReview registration: (https://osf.io/yfch2/) and published protocol (Holmen et al. Syst Rev. 10:237, 2021).

Insurance Not Socioeconomic Status is Associated With Access to Postacute Care After Injury: A Multicenter Cohort Study

IntroductionAccess to postacute care services in rehabilitation or skilled nursing facilities is essential to return trauma patients to their preinjury functional level but is often hindered by systemic barriers. We sought to study the association between the type of insurance, socioeconomic status (SES) measures, and postacute care utilization after injury. MethodsAdult trauma patients with an Injury Severity Score (ISS) ≥9 admitted to one of three Level I trauma centers were contacted 6-12 mo after injury to gather long-term functional and patient-centered outcome measures. In addition to SES inquiry specifically focused on education and income levels, patients were asked to subjectively categorize their perceived SES (p-SES) as high, mid-high, mid-low, or low. Insurance and income data were retrieved from trauma registries. Multivariable regression models were built to determine the association between type of insurance, SES, and discharge disposition after adjusting for patient and injury characteristics and hospitalization events. ResultsA total of 1373 patients were included, of which 44% were discharged to postacute care facilities. The median age (IQR) was 65 (46, 76) years, 56% of patients were male, 11% were on Medicaid, 68% had attained education higher than high school, 27% had low income, and 29% reported a low/mid-low p-SES. Medicaid patients were less likely to be discharged to postacute care compared to privately insured (OR [95% CI]: 0.41 [0.29-0.58]) and Medicare patients (OR [95% CI]: 0.29 [0.16-0.50]). The latter relationship was true across p-SES categories. P-SES, income and educational level were not associated with discharge destination. ConclusionsInsurance status, specifically having Medicaid, can pose a barrier to access to postacute care services in the trauma patient population across patients of all SES. Initiatives and policies that aim at reducing these access disparities are warranted.

1612P Demonstrating the complexity of need and the effectiveness of integrated palliative care: An analysis of routinely collected patient centred outcome measures

1612P Demonstrating the complexity of need and the effectiveness of integrated palliative care: An analysis of routinely collected patient centred outcome measures

Health Status of US Patients With One or More Health Conditions: Using a Novel Electronic Patient-reported Outcome Measure Producing Single Metric Measures.

Most existing research studying health status impacted by morbidity has focused on a specific health condition, and most instruments used for measuring health status are neither patient-centered nor preference-based. This study aims to report on the health status of patients impacted by one or more health conditions, measured by a patient-centered and preference-based electronic patient-reported outcome measure. A cross-sectional study was conducted among patients with one or more health conditions in the United States. A novel generic, patient-centered, and preference-based electronic patient-reported outcome measure: Château Santé-Base, was used to measure health status. Individual health state was expressed as a single metric number (value). We compared these health-state values between sociodemographic subgroups, between separate conditions, between groups with or without comorbidity, and between different combinations of multimorbidity. The total sample comprised 3913 patients. Multimorbidity was present in 62% of the patients. The most prevalent health conditions were pain (50%), fatigue/sleep problems (40%), mental health problems (28%), respiratory diseases (22%), and diabetes (18%). The highest (best) and lowest health-state values were observed in patients with diabetes and mental health problems. Among combinations of multimorbidity, the lowest values were observed when mental health problems were involved, the second lowest values were observed when fatigue/sleep problems and respiratory diseases coexisted. This study compared health status across various single, and multiple (multimorbidity and comorbidity) health conditions directly, based on single metric health-state values. The insights are valuable in clinical practice and policy-making.