Disease-specific Patient-reported Outcome Measures Research Articles

Determining the minimally clinically important difference for the pediatric liver transplant quality of life questionnaire.

The Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire is a disease-specific patient reported outcome measure for pediatric liver transplant (LT) recipients. To-date, threshold values above which a change in PeLTQL score is considered meaningful to patients are unavailable. This study proposes the first values for the minimally clinically important difference (MCID) for the PeLTQL. In this retrospective cohort study, anchor and distribution-based methods were used to estimate the MCID for the PeLTQL. Questionnaires completed between March 2013, and July 2022 were included if data from two sequential visits were available. An internal anchor question was used for anchor-based determination of the MCID. A final MCID estimate was ascertained from triangulation of all methods. PeLTQL data from 65 LT recipients (26 [40%] male, 17 [42%] biliary atresia, median age at LT 3.08 years [IQR 0.99-7.30]), and their caregivers were included for analysis. Median patient age at time of baseline PeLTQL completion was 13.84 (10.90-15.86) years. The MCID for self-PeLTQL total scores ranged from 4.53 to 8.46, and from 4.47 to 8.85 for proxy responses. By triangulation, the MCID of the PeLTQL total score was 6.45 and 6.78 for self and proxy responses respectively. A change in PeLTQL score of 6.5 or more points suggests a change in health status that is meaningful to the patient, providing the clinical team an opportunity to engage the patient's voice in reassessing current health status and management strategies.

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

BACKGROUND 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. AIM 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. METHODS 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. RESULTS 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. CONCLUSION 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.

Surgically-Relevant Quality of Life Thresholds for the Short Inflammatory Bowel Disease Questionnaire in Crohn’s disease

Background or PurposeDespite interest in patient-reported outcome measures to track progression in Crohn’s disease, frameworks to apply these questionnaires in the preoperative setting are lacking. Using the short inflammatory bowel disease questionnaire (sIBDQ), this study aimed to describe interpretable quality of life thresholds and examine potential associations with future bowel resection in Crohn’s disease. MethodsAdult Crohn’s disease patients completing an sIBDQ at a clinic visit between 2020-2022 were eligible. A stoplight framework was adopted for sIBDQ scores, including a ‘Resection Red zone’ suggesting poor quality of life that may benefit from discussions about surgery as well as a ‘Nonoperative Green’ zone. Thresholds were identified with both anchor- and distribution-based methods using receiver operating characteristic curve analysis and subgroup percentile scores, respectively. To quantify associations between sIBDQ scores and subsequent bowel resection, multivariable logistic regression models were fit with covariates of age, sex, body mass index, medications, disease pattern and location, resection history, and the Harvey Bradshaw Index. Incremental discriminatory value of the sIBDQ beyond clinical factors was assessed through area under the receiver operating characteristics curve (AUC) with an internal validation through bootstrap resampling. ResultsOf 2003 included patients, 102 underwent Crohn’s-related bowel resection. The sIBDQ Nonoperative Green zone threshold ranged from 61 to 64 and the Resection Red zone from 36 to 38. When adjusting for clinical covariates, a worse sIBDQ score was associated with greater odds of subsequent 90-day bowel resection when considered as a one-point (OR [95% CI], 1.05 [1.03-1.07]) or five-point change (OR [95% CI], 1.25 [1.13-1.40]). Inclusion of the sIBDQ modestly improved discriminative performance (AUC [95% CI] 0.85 [0.85 – 0.86]) relative to models that included only demographics (0.57 [0.57 – 0.58]) or demographics with clinical covariates (0.83 [0.83 – 0.84]). Discussion or ConclusionIn the decision-making process for bowel resection, disease-specific patient-reported outcome measures may be useful to identify Crohn’s disease patients with poor quality of life and promote shared understanding of personalized burden.

Developing a disease-specific patient reported outcome measure to enhance understanding of the lived experiences of ANCA associated vasculitis: A protocol paper.

Anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis (AAV) is a chronic, relapsing-remitting condition associated with increased morbidity. Previous research has shown patients with AAV report high levels of fatigue, pain, depression and anxiety. Over recent years successful work has been carried out to improve clinical outcomes, resulting in reduced mortality and end stage kidney disease (ESKD). Despite this, little work has been done to better understand the role of the patient within this condition. The prevalence of AAV is increasing and to date, there is a shortage of specific tools that assess and measure key features relating to patient reported outcomes (PROs). This protocol details how we can better understand the lived experiences of those with AAV through the development of a disease specific, patient reported outcome measure (PROM), to be used in clinic practice. This will allow us to recognise and validate PROs and the impact the disease and its treatment has on patients' health related quality of life (HRQoL). In addition, we aim to identify potential differences in PRO's between demographics, organ involvement and treatment subgroups in AAV as well as outcomes relating to the patient experience. Patients from a single centre in the UK will be recruited to take part in the exploratory qualitative study which will include focus groups and semi-structured interviews. The inclusion criteria comprise anyone with a diagnosis of AAV and willing to participate, including those who have active or relapsing disease, those are economically active, unemployed, retired and patients receiving renal replacement therapy. The aim of the project is to identify key issues patients experience in relation to their disease and its management and how these can be better assessed in a new PROM developed for use in the clinic setting. This will enable better delivery of individualised care and inform shared decision making, while also serving as a platform for future research looking at PROs in other glomerulonephritides.

The development of a cryptoglandular anal fistula quality of life scale (AF-QoL).

Quality of life (QoL) is a crucial and core outcome in assessing the effectiveness of treatments for cryptoglandular anal fistula. Despite its extensive impact, there is a lack of patient-centred, disease-specific QoL measurement instruments of adequate quality. The aim of this study is to develop a disease-specific measurement instrument that can accurately measure QoL for patients with cryptoglandular anal fistula. Semi-structured qualitative patient interviews and a systematic review of current instruments were used to generate items for the draft instrument. This underwent successive rounds of cognitive interviews to refine its wording and structure. Individual item and overall scale content validity were determined by asking experts to rate the relevance of each item and those deemed irrelevant were removed. The final instrument then underwent psychometric testing and test-retest analysis to determine its sensitivity and stability. A total of 148 patients were involved in item generation, scale development and psychometric testing. A 22-item measurement instrument has been developed; it is scored on a scale of 0-100, where 0 indicates the worst QoL and 100 demonstrates perfect QoL. The scale demonstrates excellent internal consistency (Cronbach-α = 0.927), strong content and construct validity [correlation with Perianal Disease Activity Index = -0.713, Hospital Anxiety and Depression Anxiety (-0.659) and Depression (-0.673) subscales and Short Form-12 physical (0.609) and mental (0.589) component scales] and strong reliability and responsiveness. We have developed a cryptoglandular Anal Fistula Quality of Life scale (AF-QoL), a comprehensive, disease-specific patient reported outcome measure assessing QoL in patients with cryptoglandular anal fistula.

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.

Chronic cough: more than just a persistent cough: a systematic literature review to understand the impact of chronic cough on quality of life.

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

Capturing Patient Value in an Economic Evaluation.

Economic evaluations predominantly use generic outcomes, such as the Euro Quality of Life-5 Dimension (EQ-5D), to assess health status. However, because of the generic nature, they are less suitable to capture the quality of life of patients with specific conditions. Given the transition to patient-centered (remote) care delivery, this study aims to evaluate the possibility of using disease-specific measures in a cost-effectiveness analysis. A real-life cohort from Maasstad Hospital (2020-2021) in the Netherlands, with 772 patients with rheumatoid arthritis (RA), was used to assess the cost-effectiveness of electronic consultations (e-consultations) compared with face-to-face consultations. The Incremental Cost-Effectiveness Ratio (ICER), based on the generic EQ-5D, was compared with ICER's based on RA-specific measures: the Rheumatoid Arthritis Impact of Disease (RAID) and Health Assessment Questionnaire-Disability Index (HAQ-DI). To compare the cost-effectiveness of these different measures, HAQ-DI and RAID were expressed in quality-adjusted life-years (QALYs) via estimated conversion equations. Disease-specific patient-reported outcome measures (PROMs) offer a promising alternative for traditional measures in economic evaluations, capturing patient-relevant domains more comprehensively. Because PROMs are increasingly applied in clinical practice, the next step entails modeling of an RA patient-wide conversion equation to implement PROMs in economic evaluations. The conventional ICER (eg, EQ-5D) indicates that e-consultations are cost-effective with cost savings of -€161,000 per QALY gained for a prevalent RA cohort treated in a secondary trainee hospital. RA-specific measures show similar results, with ICERs of -€163,000 per HAQ-DI (QALY) and -€223,000 per RAID (QALY) gained. RA-specific measures capture patient-relevant domains and offer the opportunity to improve the assessment and treatment of the disease impact.

12-MONTH CLINICAL OUTCOMES OF ARTHROSCOPIC LATERAL ANKLE LIGAMENT RECONSTRUCTION FOR CHRONIC ANKLE INSTABILITY: A PROSPECTIVE CASE SERIES

IntroductionChronic ankle instability is a common condition that can be effectively treated with arthroscopic lateral ankle ligament reconstruction to restore ankle stability and function. The aim of this study was to assess the functional outcomes of arthroscopic lateral ligament reconstruction using the MOXFQ, VAS, and EQ5D patient-reported outcome measures (PROMs).MethodsThis prospective series included 38 patients who underwent arthroscopic lateral ligament reconstruction for chronic ankle instability between December 2019 and April 2022. All patients completed the MOXFQ, VAS, and EQ5D PROMs preoperatively, as well as at6, and 12 months postoperatively. The MOXFQ is a disease-specific PROM that assesses foot and ankle function, while the VAS measures pain and the EQ5D evaluates health-related quality of life.ResultsAt the 12-month follow-up, the mean MOXFQ Index score had improved significantly from 53.3 ± 23.1 preoperatively to 16.0 ±21.1 (p < 0.001). Similarly, the mean VAS score had improved from 36.2 ± 22.4 preoperatively to 14.7 ± 15.0 (p < 0.001), and the meanEQ5D score had improved from 0.55 ± 0.26 preoperatively to 0.87 ± 0.12 (p < 0.001). No major complications were observed.ConclusionArthroscopic lateral ligament reconstruction is an effective treatment for chronic ankle instability, with significant improvements in clinical and health-related quality of life outcomes.

Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment-naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial

BackgroundThe Phase 3 COMET trial (NCT02782741) comparing avalglucosidase alfa and alglucosidase alfa included health-related quality of life (HRQoL) assessments in treatment-naïve patients with late-onset Pompe disease (LOPD). Here, we further characterize results from disease-specific and general patient-reported outcome (PRO) measures. MethodsAdults who participated in the COMET trial receiving avalglucosidase alfa or alglucosidase alfa (both 20 mg/kg biweekly) during the 49-week double-blind treatment period were included in the analysis. Proportions of patients exceeding meaningful change thresholds at Week 49 were compared post hoc between treatment groups. PROs and their meaningful change thresholds included: Pompe Disease Severity Scale (PDSS; decrease 1.0–1.5 points), Pompe Disease Impact Scale (PDIS; decrease 1.0–1.5 points), Rasch-built Pompe-specific Activity Scale (R-PAct; change from unable to able to complete activity), 12-item Short Form Health Survey (SF-12; physical component summary [PCS] score: increase ≥6 points, mental component summary [MCS] score: increase ≥7 points), EuroQol 5 Dimension 5 Level (EQ-5D-5L; improvement of ≥1 category), and Patient Global Impression of Change (PGIC; any improvement). ResultsThe analysis included 99 adult patients (avalglucosidase alfa n = 50; alglucosidase alfa n = 49). Patients who received avalglucosidase alfa had significantly greater odds of achieving a meaningful change versus alglucosidase alfa for the PDSS Shortness of Breath (OR [95% CI] 11.79 [2.24; 62.18]), Fatigue/Pain (6.24 [1.20; 32.54]), Morning Headache (13.98 [1.71; 114.18]), and Overall Fatigue (5.88 [1.37; 25.11]) domains, and were significantly more likely to meet meaningful change thresholds across multiple PDSS domains (all nominal p < 0.05). A numerically greater proportion of patients in the avalglucosidase alfa group were able to complete selected activities of the R-PAct compared with the alglucosidase alfa group. Significantly greater proportions of patients who received avalglucosidase alfa achieved meaningful improvements for EQ-5D-5L usual activities dimension, EQ visual analog scale, and all four PGIC domains. The proportion of patients with improvements in SF-12 PCS and MCS was greater in the avalglucosidase alfa group versus alglucosidase alfa group, but was not significant (p > 0.05). ConclusionsThese analyses show that avalglucosidase alfa improves multiple symptoms and aspects of daily functioning, including breathing and mobility. This supports the clinical relevance of the effects of avalglucosidase alfa on HRQoL for patients with LOPD.

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.

2516. Development of a de novo Patient-Reported Outcome (PRO) Measure to Assess the Impacts of Disseminated Coccidioidomycosis [Valley Fever] on Patients Living with the Condition

Abstract Background Extrapulmonary or disseminated coccidioidomycosis (cocci) is a rare, severe form of fungal disease acquired by inhaling spores of Coccidioides spp., a dimorphic fungus endemic in the southwest United States (US) and parts of Central/South America. Currently, there are no measures that assess the patient experience of health-related quality of life (HRQL) impacts associated with disseminated cocci. Preliminary work to develop a disease-specific PRO has been completed (i.e., literature review, conceptual model development [fig 1], clinician interviews). The focus of this research was to gather qualitative evidence directly from patients living with disseminated cocci. Initial draft of a Patient-Centered Conceptual Model of Coccidioidomycosis A conceptual model of the symptoms and impacts of coccidioidomycosis was developed by analyzing 29 online patient stories after a literature review revealed qualitative, patient experience research was nonexistent in fungal infections. Methods Concept elicitation (CE) interviews were conducted with 25 patients with chronic, symptomatic disseminated cocci. Audio recordings of 60–110-minute telephone interviews were transcribed and qualitatively analyzed using NVivo 1.6. Saturation analysis was performed to determine if all concepts had emerged such that additional interviews would not add to the findings. Item generation meetings were then held between F2G, COS and a patient advocate to develop draft items reflecting the HRQL impacts of the condition. Results Thirteen (52.0%) CNS/multi-site and 12 (48.0%) non-CNS/multi-site patients participated; 18 (72.0%) were English-speakers and 7 (28.0%) were native Spanish-speakers; 3 (12.0%) were Black and 12 (48.0%) were Hispanic. Key impacts were identified across 5 domains, all reaching saturation (fig 2,3): physical function (physical activity, walking, climbing stairs, standing), activities of daily living (household chores, self-care), cognitive function (memory, focus), social-role impacts (work/school, socializing) and emotional (depression, anxiety). No major differences were found between CNS/non-CNS and English/Spanish patient experiences. Physical function and ADL Impacts Reported by Patients with Disseminated Cocci: Subgroup Analyses 1 CE interview results showed disseminated patients were significantly impacted in their physical functioning and activities of daily living. A surprising result was that in general, CNS and non-CNS patients had similar impact experiences as a result of disseminated cocci. (Total number of patients who experienced the impact is shown at the top of the column and the number of patients within each subgroup who experienced the impact is shown within the column). Social/Role, Emotional and Cognitive Impacts Reported by Patients with Disseminated Cocci: Subgroup Analyses 2 Disseminated patients were also affected socially, cognitively and emotionally, as well as in their roles professionally, and as caregivers and providers. Impact on communication consisted of difficulty retrieving words and following conversation. Cognitive impacts experienced by non-CNS patients were more prevalent than anticipated. Conclusion The CE interviews provided rich qualitative data on symptom and impact experiences of patients with disseminated cocci. The findings were used to draft a disease-specific PRO measure assessing HRQL impacts of disseminated cocci, consisting of 19 items across 5 domains (fig 4). Cognitive debrief interviews are now planned with disseminated cocci patients and clinicians to further evaluate the validity of the measure. Hypothesized Conceptual Framework of PRO measure 'Patient-Reported Impacts of Disseminated Coccidioidomycosis (Valley Fever)' Conceptual frameworks represent how a PRO measure may generate a score, broadly illustrating which patient experiences will be measured and how they will be measured. Disclosures Emma L. Harvey, BSc, MBBS, MRCP, FFPM, F2G Ltd: Employee|F2G Ltd: Stocks/Bonds Mark Bresnik, MD, F2G, Ltd.: Stocks/Bonds Elliott Blatt, MPH, F2G Ltd: Advisor/Consultant Rob Purdie, n/a, Clinical Outcomes Solutions: Consulting

Psychometric validation of the Pyruvate Kinase Deficiency Diary and Pyruvate Kinase Deficiency Impact Assessment in adults in the phase 3 ACTIVATE trial

BackgroundPyruvate kinase (PK) deficiency is a rare hereditary disorder characterized by chronic hemolytic anemia and serious sequalae which negatively affect patient quality of life. This study aimed to psychometrically validate the first disease-specific patient-reported outcome (PRO) instruments: the 7-item PK Deficiency Diary (PKDD) and 12-item PK Deficiency Impact Assessment (PKDIA), designed to assess signs, symptoms, and impacts of PK deficiency in patients enrolled in the ACTIVATE global phase 3 study of mitapivat versus placebo (NCT03548220).MethodsAll validation analyses for the PKDD and PKDIA were performed on blinded data, with analyses on item integrity, scoring, reliability, and validity conducted on data from screening and baseline. Completion rates and baseline response distributions were characterized using descriptive statistics. Item response modelling was used to inform a weighted scoring system. Reliability was assessed by internal consistency and test–retest reliability; and validity by convergent and known-groups analyses.ResultsOf the 80 adults enrolled, baseline data were available for 77 (96.3%) and 78 (97.5%) patients for the PKDD and PKDIA, respectively. Item responses skewed right, indicating that mean values exceeded median values, especially for items utilizing a 0–10 numeric scale, which were subsequently recoded to a 0–4 scale; 4 items were removed from the PKDIA due to redundancy or low relevance to the trial population. Both the PKDD and PKDIA demonstrated high internal consistency (McDonald’s coefficient ω = 0.86 and 0.90, respectively), test–retest reliability (intra-class coefficients of 0.94 and 0.87, respectively), and convergent validity with other PROs (linear correlation coefficients [|r|] between 0.30–0.73 and 0.50–0.82, respectively).ConclusionsThe findings provide evidence of validity and reliability for the PKDD and PKDIA, the first disease-specific PRO measures for PK deficiency, and can therefore increase understanding of, and more accurately capture, the wider impact of PK deficiency on health-related quality of life.Trial registration ClinicalTrials.gov, NCT03548220. Registered June 07, 2018; https://www.clinicaltrials.gov/ct2/show/NCT03548220.

Abstract 14025: Health Status Profiles in Patients With New or Worsening Peripheral Artery Disease Symptoms and 12-month Hospitalization Risk From the Portrait Registry

Introduction: Risk stratification for healthcare utilization in PAD is critical given rising costs. The association between health status measures and hospitalization is unknown. We examined the association between a disease-specific patient-reported outcome measure and risk of hospitalization at 12 months. Methods: Patients with new or worsened lower extremity claudication enrolled at US sites in the PORTRAIT registry from 2011 to 2015 were included. The Peripheral Artery Questionnaire, a PAD-specific patient-reported outcome measure, was used to measure health status. PAQ summary scores range from 0 to 100 (better health status). Kaplan-Meier failure curves and adjusted Cox proportional hazards models assessed the association between baseline PAQ summary score and (1) the combined endpoint of all-cause hospital admission and ED visit (AD-ED) and (2) all-cause hospital admission (AD) at 12 months. Results: Of the 796 patients (mean age 69 ± 10 years, 42% female, 72% white, mean baseline PAQ summary score 46.8 ± 22.0) included, 349 (44%) had a hospital admission or ED visit at 12 months, with a total of 661 visits. Patients in the lowest PAQ quartile had higher rates of AD-ED at 30 days (16.1% vs. 4.3%), 90 days (29.8% vs. 12.8%), and 12 months (53.3% vs. 22.4%). In the fully adjusted model, lower PAQ score was associated with higher risk for AD-ED (HR per 10-point decrease, 1.12, 95% CI, 1.06-1.19, P&lt;0.001) and AD (HR per 10-point decrease, 1.14, 95% CI, 1.06-1.22, P&lt;0.001). Conclusion: Lower PAD-specific health status is associated with an increased risk in the combined endpoint of hospital admission or ED visits and all-cause hospital admission. These results support the use of baseline health status as a strong predictor for healthcare utilization in a high-risk population and may help to offset costs.

Clinical Utility of Patient-Reported Outcome Instruments in the Management of Pulmonary Hypertension: A Systematic Review

BackgroundDespite the greater sensitivity and specificity of disease-specific patient-reported outcome measures (PROM) to detect clinical change, only recently have such instruments been developed for pulmonary hypertension (PH), specifically pulmonary arterial hypertension (PAH) and chronic thromboembolic disease (CTEPH). Although these valuable tools are now being incorporated into clinical studies of PH, they have not yet reached widespread integration into routine clinical care. ObjectivesIn this systematic review, the authors assess the psychometric properties of PROM developed for PH, compare PROM with other clinical outcomes in PH, and address the utility of PROM in clinical care. MethodsThe authors performed a systematic search of papers published between January 1, 2006, and October 1, 2022, using the MEDLINE database to identify PROM developed and validated for PH. The identified PROM were found to have been developed only in groups with PAH and CTEPH. The authors evaluated the identified instruments according to established psychometric criteria. An additional search was performed to identify randomized controlled trials (RCTs) utilizing these PROM for comparison with clinical outcomes. ResultsFrom 527 papers retrieved, a total of 35 PROM were identified. Of these, 5 disease-specific instruments were included in the final analysis. While both CAMPHOR (Cambridge Pulmonary Hypertension Outcome Review) and emPHasis-10 performed well in patients with PAH and CTEPH with regard to their psychometric properties, emPHasis-10 demonstrated superior feasibility for use in clinical practice due to its concise format. The Pulmonary Arterial Hypertension–Symptoms and Impacts Questionnaire performed well in the authors’ analysis, though additional data is needed regarding interpretability and feasibility. ConclusionsEmPHasis-10 demonstrated strong psychometric properties and the greatest feasibility for clinical use. Further study assessing the integration of PROM into routine clinical care for PH is needed.

Development and validation of a novel personalized electronic patient-reported outcome measure to assess quality of life (Q-LIFE): a prospective observational study in people with Cystic Fibrosis

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

The SOS-SAH questionnaire in clinical practice: a multi-method evaluation study

BackgroundIn patients with mild disabilities after aneurysmal subarachnoid hemorrhage (aSAH), invisible symptoms might be easily overlooked during consultations in the outpatient clinic. We hypothesize that the Questionnaire for the Screening of Symptoms in aneurysmal Subarachnoid Hemorrhage (SOS-SAH), a disease-specific patient-reported outcome measure, might aid in screening for symptoms after aSAH. The objective of this explorative study is to evaluate the perceived impact of using the SOS-SAH in daily clinical practice for patients after aSAH, as well as to explore potential barriers to further implementation.MethodsThis multi-method study consists of a quantitative and a qualitative component. To evaluate differences in quality of care, a patient experience survey was sent to patients receiving usual care and to patients who received the SOS-SAH. A multiple linear regression model was applied, with the intervention group and case mix adjusters as independent variables. We described differences in the number of symptoms discussed between patients receiving usual care and those receiving care post-implementation. Following implementation, 16 patients and 6 healthcare professionals were interviewed about their perceptions concerning the impact of and barriers to using the SOS-SAH. A thematic analysis was performed to identify the main themes.ResultsThe survey did not reveal any differences between the usual-care group and the post-implementation group on the scales of the patient experience survey. After implementation of the SOS-SAH, the number of symptoms discussed during consultation did not increase. The interviews suggest that the SOS-SAH may improve the preparation of patients by providing them with greater insight into their complaints and by raising issues for the consultation. It could also enhance the structure and efficiency of consultation, in addition to improving communication about issues that matter to patients. All patients and healthcare professionals recommended continuing the use of the SOS-SAH in daily practice.ConclusionsAlthough no quantitative improvements were found in patient experience and symptoms discussed during consultation, implementation of the SOS-SAH could aid in screening for symptoms in patients after aSAH, and it might have a positive influence on patient preparation, while helping to structure consultations between patients and healthcare professionals.

Utility of Patient-Reported Symptom and Functional Outcomes to Indicate Recovery after First 90 Days of Radical Cystectomy: A Longitudinal Study.

This is a longitudinal prospective study that tracked multiple symptom burden and functioning status for bladder cancer (BLC) patients for 3 months post-radical cystectomy at The University of Texas MD Anderson Cancer Center, using a validated disease-specific patient-reported outcome measure (PROM) tool, the MD Anderson Symptom Inventory (the MDASI-PeriOp-BLC). The feasibility of collecting an objective measure for physical functioning, using "Timed Up & Go test" (TUGT) and PRO scores at baseline, discharge and end of study, was tested. Patients (n = 52) received care under an ERAS pathway. The more severe scores of fatigue, sleep disturbance, distress, drowsiness, frequent urination and urinary urgency at baseline predicted poor functional recovery postoperatively (OR = 1.661, 1.039-2.655, p = 0.034); other more severe symptoms at discharge (pain, fatigue, sleep disturbance, lack of appetite, drowsiness, bloating/abdominal tightness) predicted poor functional recovery (OR = 1.697, 1.114-2.584, p = 0.014) postoperatively. Compliance rates at preoperative, discharge and end of study were 100%, 79% and 77%, while TUGT completion rates were 88%, 54% and 13%, respectively. This prospective study found that more severe symptom burden at baseline and discharge is associated with poor functional recovery post-radical cystectomy for BLC. The collection of PROs is more feasible than using performance measures (TUGT) of function following radical cystectomy.

PCR8 Understanding and Assessing the Patient Experience of Symptoms for Polycystic Liver Disease (PLD): Development of a New Disease-Specific Symptom Patient-Reported Outcome Measure (PROM)

PLD is a rare condition in which multiple cysts develop in the liver. Approximately 20% of patients experience a high symptom burden. Limited patient-centered research exists for this condition, and much of the patient-reported data results from generic measures. To address this gap, a disease-specific PROM, the PLD-Symptom was developed.

PCR107 A Systematic Review and Quality Assessment of Cardiovascular Disease-Specific Patient Reported Outcome Measures Part I: Instrument Development and Content Validity

Health-related Quality of Life (HRQoL) instruments for cardiovascular diseases (CVDs) have been commonly used to measure important patient-reported outcomes (PROs) in clinical trials and practices. This study aimed at systematically identifying and evaluating the content validity of CVD-specific HRQoL instruments.