Patient-reported Outcomes Data Research Articles

Integrating Patient-Reported Outcomes Into the Care of People With Advanced Cancer-A Practical Guide.

Patient-reported outcomes (PROs) are being increasingly integrated into routine clinical practice to enhance individual patient care. This has been driven by recognition of the benefits of PROs in enhancing symptom management, patient satisfaction, quality of life, and overall survival, and reductions in acute health care utilization. These benefits are reflected in the emergence of value-based health care initiatives incorporating PRO symptom monitoring such as the Enhancing Oncology Model in the United States. However, implementing PROs can be challenging and it can be difficult to know where to begin to select appropriate PROs, and effectively display and appropriately interpret PRO data. This manuscript summarizes an educational session at the 2024 ASCO Annual Meeting, which provided practical guidance to clinicians seeking to incorporate PROs into the care of people with advanced cancer. We focus on why it is important to collect PROs in routine care from a patient's perspective, how to select PROs for symptom monitoring (including using static patient-reported outcome measures and newer item libraries), and highlight key pearls and pitfalls in the display and interpretation of PROs. We highlight the breadth of existing resources available to guide clinicians in PRO implementation.

Short- and long-term patient-reported outcomes of subcutaneous implantable cardioverter-defibrillator therapy

Abstract Background The safety and efficacy of subcutaneous implantable cardioverter-defibrillator (S-ICD) therapy are well-established. However, there is a paucity of patient-reported outcome data, despite the increasing emphasis on incorporating patients' perspectives into quality care assessments. Current guidelines also recommend evaluating psychological status and addressing distress in defibrillator patients. Purpose This study aims to assess perioperative and postoperative pain during S-ICD implantation, along with patient acceptance during follow-up. We also explore associations between these outcomes, clinical characteristics, and implantation variables. Methods A total of 149 consecutive patients (87% male, mean age 53±13 years, body mass index 27±4 Kg/m², ejection fraction 39±14%) who underwent S-ICD implantation across 11 centers were analyzed. Short-term endpoints included pain during and 6 hours after the procedure. Pain intensity was rated on a 10-point visual analogue scale. Long-term endpoint was S-ICD patient acceptance measured (on a 0-to-100 point scale) by the Florida Patients Acceptance Survey (FPAS) score and its components: Return to Function (RTF), Device-Related Distress (DRD), Positive Appraisal (PA), and Body Image Concerns (BIC). The survey was administered 12 months post-implantation. Results The S-ICD generator was positioned in an intermuscular pocket in 140 (94%) patients. Serratus anterior plane block (SAPB) was performed in 103 (69%) patients. 72 (48%) patients underwent the defibrillation testing. Implantation success was reported in all patients without operative complications. Overall, pain intensity during implantation was 2 [25th-75th percentile: 1-4], and 1 [1-2] for static pain and 2 [1-3] for dynamic pain, 6 hours post-procedure. SAPB was associated with significantly lower pain intensity during implantation (2 [1-3] vs. 4 [2-5], p<0.001) and non-significantly lower values of dynamic pain intensity 6 hours post-procedure (1 [1-2] versus 2 [1-3], p=0.076). The other tested variables, i.e. age, sex, body habitus, ejection fraction, omission of defibrillation testing, were not associated with different pain levels. S-ICD acceptance scores were favorable overall (total FPAS 77 [63-87], RTF 69 [56-88], DRD 25 [10-45], PA 88 [75-100], BIC 13 [0-38]). Patients who underwent SAPB exhibited better RTF (75 [56-88] versus 63 [50-69], p=0.002) and PA (88 [75-100] versus 75 [69-94], p=0.004). Conclusions S-ICD implantation resulted in minimal discomfort, especially with novel anesthetic techniques. Overall, patients reported good acceptance of the S-ICD. Improved acceptance was observed in patients who underwent SAPB, highlighting the significance of enhanced patient implantation experience for long-term outcomes.

Abstract PO1-11-06: Evaluating Symptom Clusters in Patients with Breast Cancer Experiencing Aromatase Inhibitor-Associated Musculoskeletal Symptoms

Abstract Background: Although treatment with an aromatase inhibitor (AI) for 5-10 years decreases 10-year breast cancer mortality by about 40%, up to 25% of patients will stop treatment early because of treatment-related, intolerable joint and muscular pain and stiffness, called aromatase inhibitor-associated musculoskeletal symptoms (AIMSS). We previously identified that nociplastic pain, or chronic pain in the absence of tissue damage or inflammation, is associated with increased likelihood of developing AIMSS. Other studies have found that symptom clusters, such as the SPPADE symptoms (sleep disturbance, pain, physical function impairment, anxiety, depression, and low energy/fatigue) are prevalent and co-occur in patients with cancer. Here we examine the prevalence of SPPADE symptoms for patients experiencing AIMSS and willing to enroll on a clinical trial examining use of cannabidiol (CBD) to treat their symptoms. Methods: Patients with stage 0-3 hormone receptor-positive breast cancer experiencing AIMSS (defined as worst joint pain ≥4/10 during prior 7 days that developed or worsened since starting AI therapy) were eligible for enrollment in this single arm, single center phase 2 open label clinical trial (NCT04754399). Enrolled patients completed validated questionnaires at baseline and then received CBD (Epidiolex) for 15 weeks. Patient-reported outcomes (PRO) data was collected serially, including the following questionnaires which addressed SPPADE symptoms: Brief Pain Inventory (BPI) to assess pain, PROMIS Profile 29+2 v2.1 to assess multiple symptoms, and Fibromyalgia Survey (FMS) to assess nociplastic pain. Results: 39 patients enrolled on this clinical trial. The median age at enrollment was 57 (range 37-59) and 21% of patients were over age 70. The majority of patients were white (92%) and average BMI was 31 (SD 6.6). Anastrozole was the most used aromatase inhibitor (74%), followed by exemestane (18%), and letrozole (8%). 5 patients (13%) required concomitant GnRHa therapy. Baseline PRO data was obtained from 37 patients. BPI average pain score over the week prior to enrollment was 5.1 (SD 1.5) and worst pain score was 7.2 (SD 1.3). Pain interference reported on the BPI was 4.1 (SD 2.1). Compared to the general population, enrolled patients reported better physical function (T-score 60.2, SE 3.7), less depression (T-score 45.5, SE 4.7) and fatigue (T-score 46.7, SE 5.1), and similar levels of anxiety (T-score 51, SE 7.1) and sleep disturbance (T-score 49.5, SE 5.9) on the PROMIS Profile. On the FMS, 62% of patients had evidence of nociplastic pain, with average score 13.3 (SD 5.7). The median number of sites of pain in the body reported by patients was 8 (range 1-16). Conclusions: While patients enrolling on an AIMSS clinical trial reported a high degree of daily pain that regularly interferes with their activities, for most patients these symptoms did not appear to have a negative impact on mood, affect, or other symptoms that typically cluster with pain. Though it is difficult to extrapolate results from a small population, the unexpected absence of co-occurring symptoms in this population raises additional questions about the interplay of AIMSS and other symptoms commonly reported in interventional AIMSS studies. Follow-up PRO data will be obtained in this clinical trial, allowing us to examine the impact of CBD use on pain, pain interference, and other patient-reported symptoms, as well as to explore which patients are more likely to obtain benefit from CBD treatment. Funding support provided by Conquer Cancer, the ASCO Foundation and the Rising Tide Foundation for Clinical Cancer Research. Citation Format: Fleege Nicole, N. Lynn Henry. Evaluating Symptom Clusters in Patients with Breast Cancer Experiencing Aromatase Inhibitor-Associated Musculoskeletal Symptoms [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO1-11-06.

Abstract PO3-10-10: Are we adequately integrating patient reported outcomes in breast cancer clinical trials?

Abstract Background: Integrating patient reported outcomes (PROs) in cancer care enhances clinical outcomes and creates a paradigm shift in providing patient-centered care. Several studies performed in the past 15 years have demonstrated that systematic monitoring of patients using PROs through surveys improve patient-clinician communication, clinician awareness of symptoms, symptom management, patient satisfaction, quality of life, and overall survival. There has been uncertainty over the type of PRO system to implement in cancer care, how to evaluate PRO in clinical trials, and how to compare this to the clinician common terminology criteria for adverse events (CTCAE). The PRO concept was first introduced in 1975 with the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life (QoL) survey. In May 2018, the National Cancer Institute established the PRO-CTCAE to be utilized for cancer patients to report and grade their treatment related adverse events, like that of clinician reported CTCAE. The goal of our study was to evaluate frequency of PRO incorporation in phase III breast cancer clinical trials. Methods: New England Journal of Medicine, Journal of Clinical Oncology, and Lancet were systematically searched to identify phase III breast cancer clinical trials published between January 2018 and June 2023. A total of 113 phase III studies were identified, 87 of which had reported protocols. Studies that did not demonstrate protocols on these search engines were not included in the review. Of these 87 clinical trials, 36 studies documented evaluation of PRO. These studies were segregated by year of publication. For each year, the number of total phase III breast cancer clinical trials that were published were compared to those that were published and evaluated PRO. A percentage was generated and allowed for a longitudinal trend to be analyzed over a 5-year period to assess if there was improved frequency of PRO integration into breast cancer clinical trials. Summary statistics were calculated using SAS 9.4. Results: 36 of 113 trials demonstrated PRO use and 26 utilized EORTC-QoL, 1 used PRO-CTCAE, and 9 used another measure of PRO assessment within their study protocol (such as FACT-B and PHQ-9. Additionally, in 2018, 33% of published breast cancer studies included PRO data. Since then, there have been fluctuations in breast cancer studies collecting PRO; 2019: 33%, 2020: 47%, 2021: 43%, 2022: 73%, and since the start of 2023: 9%. Conclusions: Our data suggests a trend towards improvement in frequency of PRO data collection in breast cancer clinical trials over the past 5 years, exemplified by the marked increase in 2022. There remains a need to incorporate PROs in clinical trials and routine care to improve patient experience and provide patient-centered care. Citation Format: Yadav Rina, Zhengyan Huang, Jessica Moss, Garima Gupta. Are we adequately integrating patient reported outcomes in breast cancer clinical trials? [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO3-10-10.

Special issue PRO] A demonstration of estimands and sensitivity analyses for time-to-deterioration of patient reported outcomes

ABSTRACT In oncology trials, health-related quality of life (HRQoL), specifically patient-reported symptom burden and functional status, can support the interpretation of survival endpoints, such as progression-free survival. However, applying time-to-event endpoints to patient-reported outcomes (PRO) data is challenging. For example, in time-to-deterioration analyses clinical events such as disease progression are common in many settings and are often handled through censoring the patient at the time of occurrence; however, disease progression and HRQoL are often related leading to informative censoring. Special consideration to the definition of events and intercurrent events (ICEs) is necessary. In this work, we demonstrate time-to-deterioration of PRO estimands and sensitivity analyses to answer research questions using composite, hypothetical, and treatment policy strategies applied to a single endpoint of disease-related symptoms. Multiple imputation methods under both the missing-at-random and missing-not-at-random assumptions are used as sensitivity analyses of primary estimands. Hazard ratios ranged from 0.52 to 0.66 over all the estimands and sensitivity analyses modeling a robust treatment effect favoring the treatment in time to disease symptom deterioration or death. Differences in the estimands include how people who experience disease progression or discontinue the randomized treatment due to AEs are accounted for in the analysis. We use the estimand framework to define interpretable and principled approaches for different time-to-deterioration research questions and provide practical recommendations. Reporting the proportions of patient events and patient censoring by reason helps understand the mechanisms that drive the results, allowing for optimal interpretation.

Gas and Bloat in Female Patients after Anti-Reflux Procedures: Analysis of 934 Cases.

Anti-reflux procedures (ARPs) are effective treatments for gastroesophageal reflux disease (GERD). However, variation in objective and patient reported outcomes persists. Limited evidence and anecdotal experience suggest that patient sex may play a role. The objective of this study was to compare outcomes after ARPs between male and female patients. We performed a retrospective review of a prospectively maintained database at a single institution. All patients who underwent an ARP for GERD were included. Demographic, clinical, and patient reported outcomes data (GERD-Health Related Quality of Life, Reflux Symptom Index), as well as radiographic hernia recurrence were collected and stratified by sex. Uni- and multivariable logistic and mixed effects linear regression were used to control for confounding effects. Between 2009 and 2022, 934 patients (291 males, 643 females) underwent an ARP. Reflux Symptom Index, GERD-HRQL, and gas/bloat scores improved uniformly for both sexes, though female patients were more likely to have higher gas/bloat scores one year post-procedure (mean ± SD 1.7 ± 1.4 vs 1.4 ± 1.3, p=0.03) and higher GERD-HRQL scores two years post-procedure (6.3 ± 8.1 vs 4.7 ± 6.8, p=0.04). Higher gas/bloat scores in females persisted on regression controlling for confounders. Hernia recurrence rates were low (85 patients, 9%) and were similar for both sexes. A final intraprocedural DI ≥3mm 2/mmHg was significantly associated with a 7 times higher rate of recurrence (95% CI 1.62-31.22, p=0.01). While patients of either sex experience symptom improvement and low rates of recurrence after ARPs, females are more likely to endorse gas/bloat compared to males. Final distensibility ≥3mm 2/mmHg carries a high risk of recurrence. These results may augment how physicians prognosticate during consultations and tailor their treatments in patients with GERD.

Remote Symptom Monitoring Using Patient-Reported Outcomes in Patients With Chronic Kidney Disease: Process Evaluation of a Randomized Controlled Trial.

In Denmark, outpatient follow-up for patients with chronic kidney disease (CKD) is changing from in-hospital visits toward more remote health care delivery. The nonuse of remote patient-reported outcomes (PROs) is a well-known challenge, and it can be difficult to explain which mechanisms of interventions influence the outcome. Process evaluation may, therefore, be used to answer important questions on how and why interventions work, aiming to enhance the implications for clinical practice. This study aimed to provide insight into the intervention process by evaluating (1) the representativity of the study population, (2) patient and physician use patterns, (3) patient adherence to the intervention, and (4) clinical engagement. A process evaluation determining the reach, dose, fidelity, and clinical engagement was carried out, alongside a multicenter randomized controlled trial (RCT). We developed and implemented an intervention using PRO measures to monitor outpatients remotely. Data were collected for the PRO intervention arms in the RCT from 4 sources: (1) PRO data from the participants to determine personal factors, (2) the web-based PRO system to identify key usage intervention patterns, (3) medical records to identify clinical factors relating to the use of the intervention, and (4) semistructured interviews conducted with involved physicians. Of the 320 patients invited, 152 (47.5%) accepted to participate. The study population reflected the target population. The mean adherence rate to the PRO intervention arms was 82% (95% CI 76-87). The questionnaire response rate was 539/544 (99.1%). A minority of 13 (12.9%) of 101 patients needed assistance to complete study procedures. Physicians assessed 477/539 (88.5%) of the questionnaires. Contact was established in 417/539 (77.4%) of the cases, and 122/539 (22.6%) of the patients did not have contact. Physicians initiated 288/417 (69.1%) and patients requested 129/417 (30.9%) of all the contacts. The primary causes of contact were clinical data (242/417, 58%), PRO data (92/417, 22.1%), and medication concerns and precautionary reasons (83/417, 19.9%). Physicians found the use of PRO measures in remote follow-up beneficial for assessing the patient's health. The inclusion of self-reported clinical data in the questionnaire motivated physicians to assess patient responses. However, some barriers were emphasized, such as loss of a personal relationship with the patient and the risk of missing important symptoms in the absence of a face-to-face assessment. This study demonstrates the importance and practical use of remote monitoring among patients with CKD. Overall, the intervention was implemented as intended. We observed high patient adherence rates, and the physicians managed most questionnaires. Some physicians worried that distance from the patients made it unfeasible to use their "clinical glance," posing a potential risk of overlooking crucial patients' symptoms. These findings underscore key considerations for the implementation of remote follow-up. Introducing a hybrid approach combining remote and face-to-face consultations may address these concerns. ClinicalTrials.gov NCT03847766; https://clinicaltrials.gov/study/NCT03847766.

Comparison of early patient-reported outcomes between uniportal thoracoscopic segmentectomy and wedge resection for peripheral small-sized non-small-cell lung cancer

BackgroundAnalysis of patient-reported outcomes (PROs) offers valuable insights into distinguishing the effects of closely related medical procedures from the patient’s perspective. In this study we compared symptom burden in patients undergoing uniportal thoracoscopic segmentectomy and wedge resection for peripheral small-sized non-small cell lung cancer (NSCLC).MethodsThis study included patients with peripheral NSCLC from an ongoing longitudinal prospective cohort study (CN-PRO-Lung 3) who underwent segmentectomy or wedge resection with tumor diameter ≤ 2 cm and consolidation tumor ratio (CTR) ≤ 0.5. PROs data were collected using the Perioperative Symptom Assessment for Lung Surgery questionnaire pre-operatively, daily post-surgery up to the fourth hospitalization day, and weekly post-discharge up to the fourth week. Propensity score matching and a generalized estimation equation model were employed to compare symptom severity. In addition, short-term clinical outcomes were compared.ResultsIn total, data of 286 patients (82.4%) undergoing segmentectomy and 61 patients (17.6%) undergoing wedge resection were extracted from the cohort. No statistically significant differences were found in the proportion of moderate-to-severe symptoms and mean scores for pain, cough, shortness of breath, disturbed sleep, fatigue, drowsiness, and distress during the 4-day postoperative hospitalization or the 4-week post-discharge period before or after matching (all p > 0.05). Compared with segmentectomy, wedge resection showed better short-term clinical outcomes, including shorter operative time (p = 0.001), less intraoperative bleeding (p = 0.046), and lower total hospital costs (p = 0.002).ConclusionsThe study findings indicate that uniportal thoracoscopic segmentectomy and wedge resection exert similar early postoperative symptom burden in patients with peripheral NSCLC (tumor diameter ≤ 2 cm and CTR ≤ 0.5).Clinical trial registrationNot applicable.

Automating Patient-reported Data Collection: Does it Work?

There are several electronic patient-reported outcomes (ePROs) vendors that are being used at institutions to automate data collection. However, there is little known about their success in collecting patient-reported outcomes (PROs) and it is unknown which patients are more likely to complete these surveys. In this study, we assessed rates of PRO completion, as well as determined factors that contributed to the completion of baseline and follow-up surveys. We queried our ePRO platform to assess rates of completion for baseline and follow-up surveys for patients from October 2019 to June 2022. All baseline surveys were administered before pediatric orthopaedic procedures, and follow-up surveys were sent at 3 months, 6 months, 1 year, and 2 years after surgery to patients with baseline data. Descriptive statistics were used to summarize the data. Univariate and multivariate analyses were performed to assess differences in patients who did and did not complete surveys. This study included 1313 patients during the study period. Baseline surveys were completed by 66% of the cohort (n = 873 patients). There was a significant difference in race/ethnicity and language spoken in the patients who did and did not complete baseline surveys (P < 0.01) with lower rates of completion in African American, Hispanic, and Spanish-speaking patients. At least one follow-up was obtained for 68% of patients with baseline surveys (n = 597 patients). There were significant differences in completion rates based on race/ethnicity (P = 0.03) and language spoken (P = 0.01). There were lower rates of baseline completion for patients with government insurance in our multivariate analysis (odds ratio: 0.6, P < 0.01). Baseline and follow-up PRO data can be obtained from the majority of patients using automated ePRO platforms. However, additional focus is needed on collecting data from traditionally underrepresented patient groups to better understand outcomes in these patient populations. Level III-retrospective cohort study.

Psychometric evaluation of the near activity visual questionnaire presbyopia (NAVQ-P) and additional patient-reported outcome items.

The Near Visual Acuity Questionnaire Presbyopia (NAVQ-P) is a patient-reported outcome (PRO) measure that was developed in a phakic presbyopia population to assess near vision function impacts. The study refined and explored the psychometric properties and score interpretability of the NAVQ-P and additional PRO items assessing near vision correction independence (NVCI), near vision satisfaction (NVS), and near vision correction preference (NVCP). This was a psychometric validation study conducted using PRO data collected as part of a Phase IIb clinical trial (CUN8R44 A2202) consisting of 235 randomized adults with presbyopia from the US, Japan, Australia, and Canada. Data collected at baseline, week 2, and months 1, 2, and 3 during the 3-month trial treatment period were included in the analyses to assess item (question) properties, NAVQ-P dimensionality and scoring, reliability, validity, and score interpretation. Item responses were distributed across the full response scale for most NAVQ-P and additional PRO items. Confirmatory factor analysis supported the pre-defined unidimensional structure and calculation of a NAVQ-P total score as a measure of near vision function. Item deletion informed by item response distributions, dimensionality analyses, item response theory, and previous qualitative findings, including clinical input, supported retention of 14 NAVQ-P items. The 14-item NAVQ-P total score had excellent internal consistency (α = 0.979) and high test-retest reliability (Intraclass Correlation Coefficients > = 0.898). There was good evidence of construct-related validity for all PROs supported by strong correlations with concurrent measures. Excellent results for known-groups validity and ability to detect change analyses were also demonstrated. Anchor-based and distribution-based methods supported interpretation of scores through generation of group-level and within-individual estimates of meaningful change thresholds. A meaningful within-patient change in the range of 8-15-point improvement on the NAVQ-P total score (score range 0-42) was recommended, including a more specific responder definition of 10-point improvement. The NAVQ-P, NVCI, and NVS are valid and reliable instruments which have the ability to detect change over time. Findings strongly support the use of these measures as outcome assessments in clinical/research studies and in clinical practice in the presbyopia population.

Assessment of fatigue and its impact in chronic liver disease

Patient reported outcomes (PROs) such as health related quality of life (HRQL) are important outcome measures for patients with chronic liver diseases (CLDs). Presence of cirrhosis and advanced liver disease have been associated with worsened HRQL and fatigue. On the other hand, some patients with earlier stages of CLD such as primary biliary cholangitis (PBC), chronic hepatitis C viral infection (HCV) and Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD), also experience fatigue causing PRO impairment. Treatment for some CLDs may improve HRQL and, sometimes, levels of fatigue. We aimed to provide an in-depth expert review of concepts related to fatigue and HRQL in patients with PBC, HCV and MASLD. As such, a panel of experts in fatigue and CLD reviewed and discussed the literature and collaborated to provide this expert review of fatigue in CLD. Herein, we review and report on the complexity of fatigue highlighting that fatigue is comprised of peripheral (neuromuscular failure, often in conjunction with sub-maximal cardiorespiratory function) and central (central nervous system dysfunction) causes. Fatigue and HRQL are measured using validated self-report instruments. Additionally, fatigue can be measured through objective tests (e.g. grip strength). Fatigue has deleterious effects on HRQL and one’s ability to be physically active and socially engaged but does not always correlate with CLD severity. Treatments for HCV and MASLD can improve levels of fatigue and HRQL, but current treatments for PBC do not seem to affect levels of fatigue. We conclude that obtaining PRO data to include HRQL and fatigue are essential for determining the comprehensive burden of CLD and its potential treatments. Impact and ImplicationsFatigue is a complex phenomenon associated with both a peripheral cause (neuromuscular failure and sub-maximal cardiorespiratory function) and a central cause (central nervous system dysfunction). Although fatigue is common among patients with CLD, it does not always correlate with the severity of the liver disease, but fatigue is related with significant decreases in patients’ health related quality of life (HRQL). Appreciating the impact of fatigue using validated self-report measurements is important to better understand the burden of CLD and the effectiveness of treatment.

Estimation of health utility values for alopecia areata

PurposeAlopecia areata (AA) is an autoimmune-mediated inflammatory dermatological disease characterised by non-scarring hair loss affecting the scalp and sometimes other hair-bearing sites. This study aimed to elicit health state utility values (HSUVs) from the UK general population for AA using time trade off (TTO) interviews.MethodsVignette descriptions of health states defined by the extent of hair loss were developed (as well as one describing caregiver burden). These were developed using data from standardised patient reported outcome (PRO) measures, a literature review and qualitative interviews. Health states were defined based on the severity of alopecia tool (SALT), which assesses extensiveness of scalp hair loss. HSUVs were then elicited for each health state in TTO interviews with the UK public.ResultsOne caregiver and five patient health states were developed based on the literature review findings, clinical trial PRO (Hospital Anxiety and Depression Scale and Alopecia Areata Patient Priority Outcomes Questionnaire) data and qualitative interviews with patients (N = 11), clinical experts (N = 4) and caregivers of adolescents with AA (N = 10). These data showed a more severe impact among patients with more extensive hair loss. One hundred and twenty participants evaluated the vignettes in TTO interviews. Patient HSUVs ranged from 0.502 for the most extensive hair loss health state (SALT 50–100 + eyebrow and eyelash loss) to 0.919 (SALT 0–10) for the mildest health state. The caregiver HSUV was 0.882.ConclusionQuantitative and qualitative data sources were used to develop and validate vignettes describing different AA health states. Patient and caregiver HSUVs demonstrate a large impact associated with AA, especially for states defined by more extensive hair loss.

End-of-Life Symptom Burden among Patients with Cancer Who Were Provided Medical Assistance in Dying (MAID): A Longitudinal Propensity-Score-Matched Cohort Study.

Cancer is the primary underlying condition for most Canadians who are provided Medical Assistance in Dying (MAID). However, it is unknown whether cancer patients who are provided MAID experience disproportionally higher symptom burden compared to those who are not provided MAID. Thus, we used a propensity-score-matched cohort design to evaluate longitudinal symptom trajectories over the last 12 months of patients' lives, comparing cancer patients in Alberta who were and were not provided MAID. We utilized routinely collected retrospective Patient-Reported Outcomes (PROs) data from the Edmonton Symptom Assessment System (ESAS-r) reported by Albertans with cancer who died between July 2017 and January 2019. The data were analyzed using mixed-effect models for repeated measures to compare differences in symptom trajectories between the cohorts over time. Both cohorts experienced increasing severity in all symptoms in the year prior to death (β from 0.086 to 0.231, p ≤ .001 to .002). Those in the MAID cohort reported significantly greater anxiety (β = -0.831, p = .044) and greater lack of appetite (β = -0.934, p = .039) compared to those in the non-MAID cohort. The majority (65.8%) of patients who received MAID submitted their request for MAID within one month of their death. Overall, the MAID patients did not experience disproportionally higher symptom burden. These results emphasize opportunities to address patient suffering for all patients with cancer through routine collection of PROs as well as targeted and early palliative approaches to care.

Conservative treatment of 3- and 4-part proximal humeral fractures: Can poor outcomes be predicted?

3- and 4-part proximal humeral fractures are frequently treated conservatively. This study aims to combine radiographic, and patient reported outcome data to identify factors leading to poor outcomes following conservative treatment. A retrospective local database analysis identified 3- and 4-part fractures. Radiographic and functional outcomes including Oxford Shoulder Score (OSS), QuickDash (QD), Subjective Shoulder Score (SSV) and VAS pain scores were collected. 104 patients were included at mean follow-up of 55months. Analysis highlighted significant OSS differences in 3- versus 4-part (p = 0.027), dominant arm injury (p = 0.046), age > 65 (p = 0.006), varus coronal neck-shaft angle < 115 versus 115-155 degrees (p = 0.008), posterior head tilt > 155 degrees (p = 0.005), greater tuberosity (GT) displacement > 5mm (p = 0.001), GT comminution (p = 0.01), medial calcar hinge displacement > 2mm (p = 0.032). According to QD scores; age > 65 (p = 0.012), varus neck-shaft angle (p = 0.01), GT displacement > 5mm (p = 0.001), GT comminution (p = 0.01), medial calcar hinge displacement > 2mm (p = 0.006). SSV varied significantly with 3- versus 4-part fractures (p = 0.005), age > 65 (p = 0.04), varus neck-shaft angle (p = 0.001), posterior head tilt (p = 0.005), GT displacement > 5mm (p = 0.001), GT comminution (p = 0.003), and medial calcar hinge displacement > 2mm (p = 0.001). We highlight risk factors for unfavourable outcomes following conservative management, aiding surgeons in shared decision-making and patient expectation management.

Impact of Procedural Variation in Endoscopic Dilation for Idiopathic Subglottic Stenosis.

A small number of Idiopathic subglottic stenosis (iSGS) patients are treated at institutions across the country. Divergence in operative techniques for endoscopic dilation (ED) of iSGS has been anecdotally recognized but not formally characterized. Additionally, the relationship between procedural variation and clinical outcome has not been studied. Secondary analysis of the NoAAC iSGS1000 cohort investigated variation in procedural techniques and treatment outcomes in patients treated with ED across high-enrolling treatment centers (enrolled >10 patients in PR-02 trial). Thirteen NoAAC centers each enrolled >10 patients treated with ED for a total of 281 subjects. There was significant variation in procedural details and rate of recurrence among institutions. Hierarchal cluster analysis revealed significant heterogeneity among institutions and clusters in all procedural variables. However, analysis demonstrated a transient delay in disease recurrence in cluster 2 which disappeared with longer longitudinal follow-up. Patient-reported outcome and peak expiratory flow data supported the potential benefit of the technical variation in Cluster 2. Distinct to cluster 2, however, was routine use of adjuvant triple medical therapy (proton pump inhibitor (PPI), antibacterial agent, and steroid inhaler). Both outcome and procedural technique vary among centers employing ED to treat iSGS. A transient delay in recurrence was observed among centers that routinely prescribed adjuvant medical therapy (antibiotic, inhaled corticosteroid, and PPI) to iSGS patients after endoscopic dilation, which was further supported by patient-reported data and peak expiratory flow data. Prospective studies are needed to understand the effects of adjuvant medical therapy on recurrence after endoscopic dilation. 4 Laryngoscope, 2024.

Patient-reported outcomes in integrated health and social care: A scoping review.

Patient-reported outcomes (PROs) have potential to support integrated health and social care research and practice; however, evidence of their utilisation has not been synthesised. To identify PRO measures utilised in integrated care and adult social care research and practice and to chart the evidence of implementation factors influencing their uptake. Scoping review of peer-reviewed literature. Six databases (01 January 2010 to 19 May 2023). Articles reporting PRO use with adults (18+ years) in integrated care or social care settings. We screened articles against pre-specified eligibility criteria; 36 studies (23%) were extracted in duplicate for verification. We summarised the data using thematic analysis and descriptive statistics. We identified 159 articles reporting on 216 PRO measures deployed in a social care or integrated care setting. Most articles used PRO measures as research tools. Eight (5.0%) articles used PRO measures as an intervention. Articles focused on community-dwelling participants (35.8%) or long-term care home residents (23.9%), with three articles (1.9%) focussing on integrated care settings. Stakeholders viewed PROs as feasible and acceptable, with benefits for care planning, health and wellbeing monitoring as well as quality assurance. Patient-reported outcome measure selection, administration and PRO data management were perceived implementation barriers. This scoping review showed increasing utilisation of PROs in adult social care and integrated care. Further research is needed to optimise PROs for care planning, design effective training resources and develop policies and service delivery models that prioritise secure, ethical management of PRO data.

Critical Comments by Food and Drug Administration Reviewers on Patient-Reported Outcomes in Food and Drug Administration Regulatory Submissions (2018-2021)

ObjectivesThis article examined the inclusion of patient-reported outcome (PRO) data in new drug applications (NDAs) submitted to the Food and Drug Administration (FDA) and approved from 2018 to 2021. The importance of assessing PROs, which capture patients’ perspectives on the disease and treatment experience, has been underscored by many stakeholders, including regulatory authorities. Despite the increasing inclusion of PRO assessments in registration trials, inclusion of language related to PRO results in approved product labeling varies widely. MethodsThis study examined FDA submission packages for NDAs approved by the FDA from 2018 to 2021 to identify critical reviewer comments related to PROs. Comments were identified and categorized by the type of criticism. Reviewers considered both oncology and nononcology indications. ResultsAssessment of PROs was included in 66.2% of the 210 submissions reviewed. Critical comments were identified in 45.3% of these applications; comments most commonly related to statistical analysis considerations, fit for purpose, and study design. Other categories of critical comment included data quality, lack of treatment benefit, administrative considerations, and miscellaneous issues. Differences were observed between oncology and nononcology NDAs with regard to the number and type of comments included in each of these categories. The findings highlight the importance of planning statistical analyses, establishing content validity, carefully considering study design, maximizing data quality, and demonstrating treatment benefit, among other issues. ConclusionsOverall, this study offers insight into the landscape of PRO data included in recently approved NDAs, along with recommendations for improving the quality and reporting of PROs in clinical trials.

Exploring patient perspectives on EQ-5D-5L data visualization within an individualized decision aid for total knee arthroplasty (TKA) in Alberta, Canada

BackgroundDecision aids can help patients set realistic expectations. In this study, we explored alternative presentations to visualise patient-reported outcomes (EQ-5D-5L) data within an online, individualized patient decision aid for total knee arthroplasty (TKA) that, in part, generates individualized comparisons based on age, sex and body mass index, to enhance usability prior to implementation into routine clinical practice.MethodsWe used data visualization techniques to modify the presentation of EQ-5D-5L outcomes data within the decision aid. The EQ-5D-5L data was divided into two parts allowing patients to compare themselves to similar individuals (1) pre-surgery and (2) 1-year post-surgery. We created 2 versions for each part and sought patient feedback on comprehension, usefulness, and visual appeal. Patients from an urban orthopedic clinic were recruited and their ratings and comments were recorded using a researcher-administered checklist. Data were managed using Microsoft Excel, R version 3.6.1 and ATLAS.ti V8 and analyzed using descriptive statistics and directed content analysis.ResultsA total of 24 and 25 patients participated in Parts 1 and 2, respectively. Overall, there was a slight preference for Version 1 in Part 1 (58.3%) and Version 2 in Part 2 (64%). Most participants demonstrated adequate comprehension for all versions (range 50–72%) and commented that the instructions were clear. While 50–60% of participants rated the content as useful, including knowing the possible outcomes of surgery, some participants found the information interesting only, were unsure how to use the information, or did not find it useful because they had already decided on a treatment. Participants rated visual appeal for all versions favorably but suggested improvements for readability, mainly larger font and image sizes and enhanced contrast between elements.ConclusionsBased on the results, we will produce an enhanced presentation of EQ-5D-5L data within the decision aid. These improvements, along with further usability testing of the entire decision aid, will be made before implementation of the decision aid in routine clinical practice. Our results on patients’ perspectives on the presentation of EQ-5D-5L data to support decision making for TKA treatments contributes to the knowledge on EQ-5D-5L applications within healthcare systems for clinical care.

Impact of dry eye disease treatment on patient quality of life.

Dry eye disease (DED) is a common multifactorial disease affecting a substantial proportion of the population worldwide. Objective tests and subjective symptoms evaluation are necessary to assess DED. Although various treatments have been introduced, accurately evaluating the efficacy of those treatments is difficult because of the disparity between diagnostic tests and patient-reported symptoms. We reviewed the questionnaires used to evaluate DED and the improvements of quality of life with various treatments. In addition, we highlighted the importance of patient-reported outcomes (PRO) assessments for evaluating the effect of DED treatments. Given that the assessment of DED treatment effectiveness substantially relies on individual ocular experiences, acquiring qualitative PRO data is essential for comprehensive evaluation and optimal treatment management. Clinicians should not only focus on improving objective symptoms but also prioritize the well-being of patients in clinical management.

Clinical implications and insights from patient-reported outcome data in KarMMa-3

Clinical implications and insights from patient-reported outcome data in KarMMa-3