Patient-reported Outcomes Research Research Articles

Extracting big data from the internet to support the development of a new patient-reported outcome measure for breast implant illness: a proof of concept study.

Individuals with health conditions often use online patient forums to share their experiences. These patient data are freely available and have rarely been used in patient-reported outcomes (PRO) research. Web scraping, the automated identification and coding of webpage data, can be employed to collect patient experiences for PRO research. The objective of this study was to assess the feasibility of using web scraping to support the development of a new PRO measure for breast implant illness (BII). Nine publicly available BII-specific web forums were chosen post-consultation with two prominent BII advocacy leaders. The Python Selenium and Pandas packages were used to automate extraction of de-identified text from the individual posts/comments into a spreadsheet. Data were coded using a line-by-line approach and constant comparison was used to create top-level domains and sub-domains. 6362 unique codes were identified and organized into four top-level domains of information needs, symptom experiences, life impact of BII, and care experiences. Information needs of women included seeking/sharing information pre-breast implant surgery, post-breast implant surgery, while contemplating explant surgery, and post-explant surgery. Symptoms commonlydescribed by women included fatigue, brain fog, and musculoskeletal symptoms. Many comments described BII's impact on daily activities and psychosocial wellbeing. Lastly, some comments described negative care experiences and experiences related toadvocating for themselves to providers. This proof-of-concept study demonstrated the feasibility of employing web scraping as a cost-effective, efficient method to understand the experiences of women with BII. These data will be used to inform the development of a BII-specific PROM.

Patient-reported outcomes: The unmet needs of the gastrointestinal cancer community.

731 Background: According to the World Health Organization International Agency for Research on Cancer, gastrointestinal (GI) cancers account for 1 in 4 cancer cases and 1 in 3 cancer-related deaths worldwide. An estimated 4.8 million new cases of GI cancers and 3.4 million related deaths occur annually. Understanding the need for increased GI cancer awareness, patient education and support, the GI Cancers Alliance developed a series of engagement activities for the GI cancer community as a needs assessment to identify critical gaps in support services, fragmented education, and disease awareness. Methods: Our 12-month patient-reported outcomes (PRO) research included an online anonymous survey, individual and small group interviews, focus groups, advisory boards, and an interactive workshop at the 2023 American Society of Clinical Oncology annual meeting. 1,122 patients participated during this 12-month PRO research period. Participants reported being diagnosed with one of the following GI cancer primary tumor types: Anus (3%), Appendix (2%), Bile Duct (6%), Colon (19%), Esophagus (7%), Gallbladder (5%), Gastric (14%), GI NET / GI Carcinoid Tumor (3%), Liver (7%), Pancreas (6%), Small Intestine (4%), Rectum (23%), Unknown primary location (1%). Results: Overarching areas of unmet need include: health disparities throughout the care continuum for underserved populations (21%); lack of adequate precision oncology patient education, including biomarker education and testing (19%); the rise of early-age onset (EAO) in GI cancers and the unique needs of the young adult (YA) population (18%); nutritional wellness education (17%); insufficient support for family caregivers and caregiver respite services (16%) scarcity of patient-centered care and adequate patient-clinician communication (11%). Conclusions: Our PRO research underscores the unmet needs and gaps in support services of the GI cancer community. Our global call to action includes partnering with our 100+ member organizations to amplify the patient voice and patient-lived experience, and cross-collaboration with our community of advocates, patients, caregivers, and clinicians. Our shared call to action and continued collaboration provides a greater impact for our GI cancer community to help meet patient needs and eliminate critical gaps in services.

Hydrometrocolpos: a Contemporary Review of the Last 5 Years.

The goal of this review is to provide a comprehensive overview of hydrometrocolpos, covering disease etiology, pathophysiology, clinical presentation, and diagnostic and management techniques, and known outcomes. This narrative review presents the literature on hydrometrocolpos in the pediatric population from the past 5years. We highlight the 69 reported cases of hydrometrocolpos and classify them based on type of obstruction or associated anomaly, discuss new diagnostic algorithms based on imaging, and present novel and underutilized surgical techniques for definitive management. Hydrometrocolpos, a condition characterized by retained fluid causing a distended vagina and uterus in the setting of a distal vaginal outflow obstruction, has a wide range of presentation severity based on the type of obstruction. Whether hydrometrocolpos is due to an isolated condition like imperforate hymen, a complex abnormality like cloacal malformation, or a part of a large congenital syndrome, the mainstay of treatment is decompression of the dilated vagina and surgical correction of the outflow obstruction. Imaging-based diagnostic algorithms and new treatment techniques reported in the literature, as well as longitudinal and patient-reported outcome research, can improve the lives of children affected by this condition.

PP80 The Value of Intraoperative Neural Monitoring During Thyroid Surgery In China: A Literature Review

IntroductionRecurrent laryngeal nerve (RLN) injury has been a frequent source of malpractice litigation following thyroid surgery. Intraoperative neural monitoring (IONM) has been widely applied to avoid RLN injury in thyroid surgery in developed countries, but China cannot achieve the same application rate currently. To improve the recognition and application of IONM in China, this literature review aims to synthesize the evidence on the value of IONM in China.MethodsA comprehensive literature review was conducted by searching through PubMed, CNKI and Wan Fang to identify studies about the IONM technology for protecting RLN during thyroid surgery in Chinese clinical data.ResultsNineteen Chinese clinical trials of IONM during thyroid surgery published from 2012 to 2022 were included for review. Almost all studies recruited adults who need thyroid surgery and only one study enrolled geriatric patients. There were three (3/19) prospective studies and two were randomized controlled trials that both showed that IONM helped surgeons to detect the RLN with less time and reduce the injury of RLN in reoperative thyroid surgery. Major studies (16/19) conducted retrospective analysis and six of them (6/16) only had an IONM group that showed that IONM technology can avoid damage to the RLN. The remaining ten studies (10/16) compared patients with and without IONM during the surgery. Compared with the non-IONM group, nine studies (9/10) stated IONM technology has an advantage in the reduction of RLN injury rate and seven studies (7/10) claimed IONM was helpful in reducing the time of surgery. One study believed IONM was beneficial to reducing bleeding during the surgery and another study reported a lower incidence of hypoparathyroidism with the IONM group.ConclusionsThe value of IONM for protecting RLN during thyroid surgery and improving operation efficiency has been thoroughly proven by the above Chinese clinical trials. Further economic evaluations and patient-reported outcomes research of IONM with Chinese hospital data will help better assess the value of IONM.

The value of co-creating a clinical outcome assessment strategy for clinical trial research: process and lessons learnt

BackgroundIn support of UCB pharmaceutical research programs, the aim of this research was to implement a novel process for patient involvement in a multidisciplinary research group to co-create a clinical outcome assessment strategy to accurately reflect the experience of people living with early-stage Parkinson’s. Patient experts were an integral part of the decision-making process for patient-reported outcome (PRO) research and instrument development.MethodsIn partnership with two patient organizations (Parkinson’s UK and the Parkinson’s Foundation), 6 patient experts were recruited into a multidisciplinary research group alongside clinical, patient engagement and involvement, regulatory science, and outcome measurement experts. The group was involved across two phases of research; the first phase identified what symptoms are cardinal to the experience of living with early-stage Parkinson’s and the second phase involved the development of PRO instruments to better assess the symptoms that are important to people living with early-stage Parkinson’s. Patient experts were important in performing a variety of roles, in particular, qualitative study protocol design, conceptual model development, and subsequent co-creation of two PRO instruments.ResultsInvolving people with Parkinson’s in PRO research ensured that the expertise of these representatives from the Parkinson’s community shaped and drove the research; as such, PRO instruments were being developed with the patient at the forefront. Working with patient experts required considerable resource and time allocation for planning, communication, document development, and organizing meetings; however, their input enriched the development of PRO instruments and was vital in developing PRO instruments that are more meaningful for people with Parkinson’s and clinicians.ConclusionsConducting PRO research, in the context of clinical development involving pharmaceutical companies, requires balancing regulatory and scientific rigor with tight time constraints. Incorporating a multi-stakeholder perspective, which included patient experts as joint investigators, had a strong positive impact on our research, despite the logistical complexities of their involvement. Due to the input of patient experts, the innovative clinical outcome assessment strategy and the co-created novel PRO instruments were more relevant and holistic to the patient experience of early-stage Parkinson’s.Graphical abstract

Missing Data in Patient-Reported Outcomes Research: Utilizing Multiple Imputation to Address an Unavoidable Problem.

Patient-reported outcomes (PROs) have become a focus in postoperative surgical care. Unfortunately, studies using PROs can be subject to missing data, which may lead to biases or inaccurate conclusions. Multiple imputation (MI) is a statistical method for addressing missing data in clinical research. The aim of this study was to explore MI as a way to address missing data in PRO research. A working example of MI using real-world data was performed using the BREAST-Q PRO measurein postmastectomy reconstruction. A retrospective review of immediate tissue expander breast reconstruction patients in 2019 was conducted to compare BREAST-Q physical well-being of the chest scores betweenprepectoral and subpectoral cohorts at 2 weeks postoperatively. The observed dataset and three hypothetical missingness situations were created to assess how increasing missingness affects MI results. Overall, 916 patients were included in the analysis. When excluding patients with missing information and solely performing analysis on the completed cases, prepectoral patients had significantly higher physical well-being of the chest scores at 2weeks postoperatively; however, this trend was reversedwith increasing missingness scenarios, where subpectoral patients had higher scores. In comparison, all MI results showed that prepectoral patients had higher scores on average compared with subpectoral patients regardless of missingness scenario. MI demonstrated consistent results with increasing missingness scenarios, whereas performing analysis in higher missingness scenarios without MI led to varying results. This working example emphasizes the need for missing data methodology to be considered in PRO research.

The role of socioeconomic factors as barriers to patient reported outcome measure completion following lumbar spine fusion

BACKGROUND CONTEXTAlthough incorporating patient reported outcomes (PROMs) into practice allows healthcare systems to evaluate the value of care provided, research and policy reflecting PROMs can only be valid if they represent all patients. Few studies have evaluated socioeconomic barriers to PROM completion, and none have done so in a spine patient population. PURPOSETo identify patient barriers to PROM completion one year following lumbar spine fusion. STUDY DESIGN/SETTINGRetrospective single-institution cohort study. PATIENT SAMPLEA total of 2,984 patients undergoing lumbar fusion between 2014 and 2020 OUTCOME MEASURESCompletion of Mental Component Score (MCS-12) and Physical Component Score (PCS-12) of Short Form-12 questionnaire 1 year postoperatively. METHODSA retrospective review was conducted of all patients undergoing 1-3-level lumbar fusion at a single urban tertiary center. PROMs were queried from our prospectively managed electronic outcomes database. Patients were considered to have complete PROMs if 1-year outcomes were available. Community-level characteristics were collected from patients’ zip codes using the Economic Innovation Group Distressed Communities Index. Bivariate analyses were performed to assess factors associated with PROM incompletion along with multivariate logistic regression to control for confounders. RESULTSA total of 1,968 (66.0%) had incomplete 1-year PROMs. Patients with incomplete PROMs were more likely to be Black (14.5% vs 9.3%, p<.001), Hispanic (2.9% vs 1.6%, p=.027), reside in a distressed community (14.7% vs 8.5%, p<.001), and be active smokers (22.4% vs 15.5%, p<.001). On multivariate regression, Black race (OR: 1.46, p=.014, Hispanic ethnicity (OR: 2.19, p=.027), distressed community status (OR: 1.47, p=.024), workers’ compensation status (OR: 2.82, p=.001), and active smoking (OR:1.31, p=.034) all were independently associated with PROM incompletion. Surgical characteristics, including primary surgeon, revision status, approach, and levels fused were not associated with PROM incompletion. CONCLUSIONSSocial determinants of health impact completion of PROMs. Patients completing PROMs are overwhelmingly White, non-Hispanic, and reside in wealthier communities. Efforts should be taken to provide better education regarding PROMs and ensure closer follow-up of certain subgroups of patients to avoid furthering disparities in PROM research.

Association of Traumatic Brain Injury, Mild Cognitive Impairment and Dementia in the Framingham Heart Study’s Original Cohort (S39.009)

<h3>Objective:</h3> To estimate the associations between traumatic brain injury (TBI), mild cognitive impairment (MCI), all-cause dementia, and Alzheimer’s disease (AD) dementia. <h3>Background:</h3> Research on the relationship between TBI, AD and AD related dementias is inconsistent. <h3>Design/Methods:</h3> The Framingham Heart Study (FHS) is a population-based cohort study which recruited its original cohort, ages 30–62, between 1948–1950. Information on TBIs was collected by self-report and comprehensive review of medical records obtained through FHS health history updates until death. TBI occurrence and severity were operationalized using modified American Congress of Rehabilitation Medicine and Veterans Affairs/Department of Defense criteria respectively. MCI, dementia, and AD dementia consensus diagnoses were operationalized using modified Peterson criteria, Diagnostic and Statistical Manual of Mental Disorders, 4^th edition, and National Institute of Neurological and Communicative Diseases and Stroke/AD and Related Disorders Association criteria respectively. <h3>Results:</h3> The study included 715 participants with TBI and 2145 age- and sex-matched participants without TBI who were cognitively normal at time of TBI and followed for an average of 16±17 years. 567 (26.43%), 461 (21.49%) and 373 (17.39%) participants without TBI developed MCI, all cause dementia and AD dementia respectively compared with 271 (37.9%), 218 (30.49%) and 131 (21.12%) of those with TBI. For mild TBI, hazard ratios (HRs) for MCI, all-cause dementia and AD dementia were 1.55 (95%CI 1.14–2.12), 1.47 (95%CI 1.02–2.14), and 1.06 (95%CI 0.68–1.66) respectively. For moderate-to-severe TBI, HRs for MCI, all cause dementia and AD dementia were 3.50 (95%CI 1.10–11.17), 4.61 (95%CI 0.96–22.14), and 6.36 (95%CI 0.72–56.50) respectively. <h3>Conclusions:</h3> In this cohort followed for more than a decade and comprehensively assessed for TBI and dementia-related outcomes, there were small to moderate sized associations of mild TBI and large associations of moderate-severe TBI with MCI and all-cause dementia. Association between mild TBI and dementia may be driven by non-AD etiologies. <b>Disclosure:</b> Dr. Durape has nothing to disclose. Dr. Joshi has nothing to disclose. Ms. Burton has nothing to disclose. Mr. Chien-Young has received research support from the National Heart, Lung, And Blood Institute of the National Institutes of Health under Award Number T35HL139444. Miss Price has nothing to disclose. Miss Shanabrook has nothing to disclose. Miss Yhang has nothing to disclose. Mrs. Liu has nothing to disclose. The institution of Dr. Ang has received research support from NIH. Dr. Devine has nothing to disclose. Dr. Gurnani has nothing to disclose. Dr. Katz has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Law firms. The institution of Dr. Katz has received research support from NINDS/NIH. Dr. Katz has received publishing royalties from a publication relating to health care. Yorghos Tripodis, 5406 has nothing to disclose. The institution of Michael Alosco, PHD has received research support from NIH. Dr. Dams-O’Connor has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Various law firms. The institution of Dr. Dams-O’Connor has received research support from NIH. The institution of Dr. Dams-O’Connor has received research support from Department of Defense. The institution of Dr. Dams-O’Connor has received research support from National Institute on Disability Independent Living and Rehabilitation Research. The institution of Dr. Dams-O’Connor has received research support from Patient Reported Outcomes Research Institute. Dr. Au has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Au has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Dr. Au has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Au has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NovoNordisk. Dr. Au has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for GSK. Dr. Au has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Eisai. Dr. Au has received publishing royalties from a publication relating to health care. Dr. Au has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant with Davos Alzheimer’s Collaborative. Dr. Au has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant with High Lantern. The institution of Dr. Mez has received research support from NIH, DOD.

OA04 Association of clinical and demographic factors with quality of life, functional status, depression, and fatigue in patients presenting with, and at risk of, rheumatoid arthritis

Abstract Background/Aims Rheumatoid arthritis (RA) is often preceded by symptomatic phases during which classification criteria are not fulfilled. The health burden of these “at-risk” stages is not well described. This study assessed health-related quality of life (HRQoL), functioning, fatigue and depression in newly presenting patients with clinically suspect arthralgia (CSA), unclassified arthritis (UA) and RA. Methods Cross-sectional analysis of baseline Patient-Reported Outcome Measures (PROMs) was conducted in 838 patients with CSA, UA or RA recruited to the Birmingham Early Arthritis Cohort. HRQoL, function, depression and fatigue at presentation were assessed using EQ-5D, HAQ-DI, PHQ-9 and FACIT-F, respectively. Descriptive statistics and multivariate linear regression were performed to assess associations between clinical/demographic variables and PROMs. Results Patients with RA at initial presentation had worse PROMs than those with CSA or UA. However, HRQoL was decreased in all groups. In patients with CSA/UA, HRQoL was comparable to data relating to chronic conditions such as heart failure, severe COPD or mild angina from the Health Survey for England (HSE). Associations between predictor variables and PROMs are summarised in Table 1. Increased functional disability was associated with female sex, older age, obesity, lower quintiles of social deprivation, increased disease activity and polypharmacy. HRQoL was associated with increased disease activity and polypharmacy. The severity of depression (PHQ-9) increased with older age, increasing BMI, living in areas with the lowest quintile of social deprivation, increased disease activity and polypharmacy. Fatigue (FACIT-F) was associated with female sex, increasing BMI, disease activity and polypharmacy. After adjusting for the demographic and clinical factors, the diagnosis assigned at baseline did not affect any of the studied PROMs, but disease activity and polypharmacy were strongly linked to poorer scores across all PROMs. Conclusion Patients with musculoskeletal symptoms who are at risk of RA present with a high health burden. Early intervention may be needed to improve quality of life from initial presentation. Disclosure B. Torlinska: Corporate appointments; BT is employed by Visible Analytics Ltd.. Grants/research support; BT receives funding from the NIHR. K. Raza: Consultancies; KR declares personal fees from Abbvie and Sanofi outside of the submitted work. Grants/research support; KR declares funding from Bristol Myers Squibb outside of the submitted work, KR is supported by the NIHR Birmingham Biomedical Research Centre, the MRC Versus Arthritis Centre for Musculoskeletal Ageing Research and the Research into Inflammatory Arthritis Centre Versus Arthritis, University of Birmingham, UK. A. Filer: Grants/research support; AF is supported by the NIHR Birmingham Biomedical Research Centre, the MRC Versus Arthritis Centre for Musculoskeletal Ageing Research and the Research into Inflammatory Arthritis Centre Versus Arthritis, University of Birmingham, UK, AF declares funding outside of the submitted work from Abbvie, Roche, Janssen, UCB, Nascient, Mestag, GSK. G. Jutley: None. I. Sahbudin: None. R. Singh: None. P. de Pablo: None. E. Rankin: None. B. Rhodes: None. N. Amft: None. E. Justice: None. C. McGrath: None. S. Baskar: None. J. Trickey: None. M. Calvert: Consultancies; MC has received personal fees outside of the submitted work from Astellas, Aparito Ltd., CIS Oncology, Takeda, Merck, Daiichi Sanko, Glaukos, GSK, and the Patient-Centered Outcomes Research Institute. Grants/research support; MC is Director of the Birmingham Health Partners Centre for Regulatory Science and Innovation, Director of the Centre for Patient Reported Outcomes Research and is a NIHR Senior Investigator, MC receives funding from NIHR, UKRI, NIHR Birmingham Biomedical Research Centre, NIHR Surgical Reconstruction and Microbiology Research Centre, NIHR ARC West Midlands, UK Spine and Health Data Research at the University of Birmingham and University Hospitals Birmingham, UK, Innovate UK (part of UK Research and InnovatioRin), Macmillan Cancer Support, UCB Pharma, Janssen, GSK, and Gilead. M. Falahee: None.

Validation of the Malaysian Chinese-version of the EORTC QLQ-CR29 among Colorectal cancer patients in Malaysia

Patient reported outcome research are becoming a very important way of understanding patient’s satisfaction and tolerance with the treatment they receive. This study examined the validity and reliability of the Malaysian Chinese version of the European Organization for Research and Treatment of Cancer (EORTC) colorectal cancer-specific quality of life questionnaire (QLQ-CR29). Patients were recruited at the outpatient clinics of the University of Malaya and Universiti Kebangsaan Medical Centre, Malaysia. The QLQ-CR29 and Karnofsky Performance Scales (KPS) were used. Multi-traits scaling analysis such as Cronbach’s alpha, inter-class correlation (ICC) and known-groups comparisons (Wilcoxon-rank sum test) were performed. Significant level was pre-set at ≤ 0.05. Patients mean age (standard deviation, SD) was 64 (10) years, and 60% were male. Colon cancer was 50%. While 60% had advanced stage (Dukes’ C/D) cancers and about 30% of patients had a stoma. The mean KPS was 80% (SD 10). The internal consistency coefficient (ICC) was 0.89, 0.84, 0.46, and 0.73 for body image, urinary frequency, blood &amp; mucus in stool and stool frequency scales respectively. The correlation coefficients for convergent validity were moderate to large (r ranged from 0.72 to 0.93) across all scales. For the discriminant validity measurements, small to moderate correlations were observed across the scales. The psychometric properties of the Malaysian Chinese version of the QLQ-CR29 were comparable to the original English version. This work provides a translated, culturally adapted, reliable and valid measure of HRQOL for use among Malaysian Chinese patients with colorectal cancer.

MSR22 Strategies for Effective Patient Advocacy Group Engagement in the Development and Fielding of Online Patient Burden Surveys

Patient burden surveys capture patient reported outcome (PRO) data, but can be limited by poor understanding of the disease area and a lack of patient access. Patient advocacy groups’ (PAGs) unique understanding of, and access to, their patient community can facilitate PRO research on the patient experience. However, there is a lack of research exploring effective methods for collaboration with PAGs. Here we establish a best practice framework for successful PAG engagement in future PRO research.

Implementation of thyroid-related patient-reported outcomes in routine clinical practice.

Patient-reported outcomes (PROs) are increasingly used in clinical practice to improve clinical care. Multiple studies show that systematic use of PROs can enhance communication with patients and improve patient satisfaction, symptom management and quality of life. Further, such data can be aggregated to examine health levels for patient groups, improve quality of care, and compare patient outcomes at the institutional, regional or national level. However, there are barriers and challenges that should be handled appropriately to achieve successful implementation of PROs in routine clinical practice. This paper briefly overviews thyroid-related PROs, describes unsolved quality of life issues in benign thyroid diseases, provides examples of routine collection of PROs, and summarizes key points facilitating successful implementation of thyroid-related PROs in routine clinical practice. Finally, the paper touches upon future directions of PRO research.

The application of patient reported outcomes (PROs) to build personalized patient education aids

Background After diagnosis, cancer patients receive a large volume of information in a short period of time when they are usually feeling anxious and stressed. In particular, prostate cancer patients can be overwhelmed by multiple treatment options and the expectation of making a life-altering personal choice during shared decision-making. Consent forms attempt to summarize potential side effects but are rarely personalized to each patient. Clinicians can underestimate symptoms/side effects experienced by patients, compared to patient-reported outcomes (PROs). PROs are the best estimate of how our patients will feel during/after treatments. Methods We used real world data including PROs and clinical/radiation data from the Enterprise Data Warehouse and our departmental radiation database to explore patient characteristics associated with PRO changes over time. We created several different visualizations showing how PROs changed over time, and tried to pick visualizations that were most accessible to patients with different backgrounds, languages, data/health literacy. In the future, we hope to conduct patient interviews to improve visualizations, and to automate real-time generation of personalized patient education aids for use at consultations. Results Our cohort included ∼3,900 patients who completed >35,000 PROMIS-10, >5,000 EPIC-26, and >11,000 weekly radiation treatment visit questionnaires about urinary, bowel, erectile, and overall quality of life. Among significant predictors of toxicity were initial symptom severity. Discussion Patient reported outcomes (PROs) improve outcomes and have become standard in clinical trials and are gradually being incorporated into more cancer clinics. The next phase of PRO research is continuing to expand applications into general clinical practices and routine daily workflows using real world data (collected from each clinic because clinical practices/outcomes can vary). It is time to use our PROs work back to directly benefit patients. We are working on real-time generation of personalized patient education resources in the routine clinical workflows. Resources include data visualizations that aim to be accessible to patients with diverse backgrounds, languages, data/health literacy. Prostate cancer patients could especially benefit from personalized resources for standard-of-care shared decision-making after initial diagnosis. This work in progress describes how to combine PROs and clinical data to create personalized PRO visualizations for patient education, and potential mechanisms for real-time generation of these resources. After diagnosis, cancer patients receive a large volume of information in a short period of time when they are usually feeling anxious and stressed. In particular, prostate cancer patients can be overwhelmed by multiple treatment options and the expectation of making a life-altering personal choice during shared decision-making. Consent forms attempt to summarize potential side effects but are rarely personalized to each patient. Clinicians can underestimate symptoms/side effects experienced by patients, compared to patient-reported outcomes (PROs). PROs are the best estimate of how our patients will feel during/after treatments. We used real world data including PROs and clinical/radiation data from the Enterprise Data Warehouse and our departmental radiation database to explore patient characteristics associated with PRO changes over time. We created several different visualizations showing how PROs changed over time, and tried to pick visualizations that were most accessible to patients with different backgrounds, languages, data/health literacy. In the future, we hope to conduct patient interviews to improve visualizations, and to automate real-time generation of personalized patient education aids for use at consultations. Our cohort included ∼3,900 patients who completed >35,000 PROMIS-10, >5,000 EPIC-26, and >11,000 weekly radiation treatment visit questionnaires about urinary, bowel, erectile, and overall quality of life. Among significant predictors of toxicity were initial symptom severity. Patient reported outcomes (PROs) improve outcomes and have become standard in clinical trials and are gradually being incorporated into more cancer clinics. The next phase of PRO research is continuing to expand applications into general clinical practices and routine daily workflows using real world data (collected from each clinic because clinical practices/outcomes can vary). It is time to use our PROs work back to directly benefit patients. We are working on real-time generation of personalized patient education resources in the routine clinical workflows. Resources include data visualizations that aim to be accessible to patients with diverse backgrounds, languages, data/health literacy. Prostate cancer patients could especially benefit from personalized resources for standard-of-care shared decision-making after initial diagnosis. This work in progress describes how to combine PROs and clinical data to create personalized PRO visualizations for patient education, and potential mechanisms for real-time generation of these resources.

Recruiting Adolescent and Young Adult Cancer Survivors for Patient-Reported Outcome Research: Experiences and Sample Characteristics of the SURVAYA Study.

Background: Participation of Adolescents and Young Adults with cancer (AYAs: 18–39 years at time of diagnosis) in patient-reported outcome studies is warranted given the limited knowledge of (long-term) physical and psychosocial health outcomes. We examined the representativeness of AYAs participating in the study, to observe the impact of various invitation methods on response rates and reasons for non-participation. Methods: A population-based, cross-sectional cohort study was performed among long-term (5–20 years) AYA cancer survivors. All participants were invited using various methods to fill in a questionnaire on their health outcomes, including enclosing a paper version of the questionnaire, and sending a reminder. Those who did not respond received a postcard in which they were asked to provide a reason for non-participation. Results: In total, 4.010 AYAs (response 36%) participated. Females, AYAs with a higher socio-economic status (SES), diagnosed more than 10 years ago, diagnosed with a central nervous system tumor, sarcoma, a lymphoid malignancy, stage III, or treated with systemic chemotherapy were more likely to participate. Including a paper questionnaire increased the response rate by 5% and sending a reminder by 13%. AYAs who did not participate were either not interested (47%) or did want to be reminded of their cancer (31%). Conclusions: Study participation was significantly lower among specific subgroups of AYA cancer survivors. Higher response rates were achieved when a paper questionnaire was included, and reminders were sent. To increase representativeness of future AYA study samples, recruitment strategies could focus on integrating patient-reported outcomes in clinical practice and involving AYA patients to promote participation in research.

Evaluation of Patient-Reported Outcomes (PROs) Protocol Content and Reporting for Clinical Trials that Lead to the approval of frontline Immune Checkpoint Blockade Combination for Patients with Advanced Renal Cell Carcinoma - The Patients' Voice or a Missed Opportunity.

Immune checkpoint blockade (ICB) is a rapidly emerging field of oncology that has revolutionized the metastatic renal cell carcinoma (mRCC) treatment. Four recent treatment regimens Nivolumab-Ipilimumab, Pembrolizumab-Axitinib, Nivolumab-Cabozantinib, and Pembrolizumab-Lenvatinib-have demonstrated improved clinical endpoints compared to standard of care and are endorsed by NCCN (2021). However, data on patient-reported outcomes (PROs) for patients receiving these regimens are limited. We conducted a comparative assessment of the quality and standardization of PROs endpoints and data reported for these randomized controlled trials (RCTs). We systematically identified all RCTs evaluating combination ICB for ccRCC. PROs-specific data were abstracted from the final version of 4 RCT protocols, as well as clinical and PROs specific manuscripts published between April 2018 and April 2021. We used 3 previously published guides standardizing PROs research to objectively score the data: (i) 24-point PROEAS; (ii) 12-point SPIRIT-PRO; and (iii) 14-point CONSORT-PRO. The CheckMate 214, KEYNOTE 426, CheckMate 9ER, and CLEAR studies had PROEAS scores of 88% (21/24), 37% (9/24), 83% (20/24), and 16% (4/24), respectively, and SPIRIT-PRO scores of 50% (6/12), 75% (9/12), 66% (8/12), and 41% (5/12) respectively. The CONSORT-PRO scores were 86% (12/14) for CheckMate 214 and 43% (6/14) for CheckMate 9ER, but scores were not available for the CLEAR and KEYNOTE 426 studies because of a lack of sufficient data. The average SPIRIT-PRO score across the 4 RCTs was 58%, indicating a reasonable adoption of PROs research in data management and analysis. The CheckMate 214 trial had the longest follow-up and most comprehensive published PROs data. Our analysis identified the limitations of current PROs data in combination ICB approved for mRCC. This analysis will enable clinicians to better interpret the current PROs results and emphasize the importance of better incorporation of PROs endpoints in future mRCC trial design.

Academic-industry partnership advancing cannabis science: A Complementary Care Practice-Based Research Network

ObjectivesData collected during routine care holds the potential to support hypothesis generation, study feasibility, and provide insight regarding how to address problems under real world conditions. Currently there are no practice-based research networks in Florida that focus on complementary care in general or medical marijuana specifically. Through an academic-industry partnership, we sought to develop a practice-based research network focused on cannabis science and create a de-identified database for analyses that support hypothesis generation, study feasibility estimation, and a network that also facilitates recruitment into future research studies. DesignThe Complementary Care Practice-Based Research Network (CC-PBRN) is a centralized repository, which contains electronic health records (EHR) from a private medical cannabis health system in the state of Florida. ResultsThis paper provides cross-sectional descriptive analyses of the CC-PBRN’s 43,802 currently active patients. There are 24 clinics in the network with nine in North Florida, 11 in Central Florida, and 4 in South Florida. ConclusionThis network serves as a statewide resource for patient-reported outcomes (PRO) research in medical marijuana. The network currently has numerous outpatient practices with thousands of patients that are a potential source of research participants for both observational studies as well as traditional clinical trials. The outpatient clinical practices can also serve as units of analysis for pragmatic trials comparing different care protocols and organizational structure.

Incorporating Patient-Reported Outcomes as a Vital Sign for Dermatologic Clinical Care and Clinical Investigations

Patient-reported outcomes (PROs) provide quantitative assessments of patients' experiences with their skin diseases. PROs are usually much more comprehensive than what can be gleaned from a brief clinical history and more informative than what dermatologists can gather on clinical examination. Correlations between PROs and clinician assessments (e.g., investigator global assessment, PASI) are poor to moderate at best, and therefore data from each source are not redundant and can complement one another. PROs should serve as skin vital signs in dermatology. PROs can offer snapshots of the intensity of a symptom as well as the effects of symptoms, emotions, and functioning on a patient's skin-related QOL. Just as clinicians obtain a baseline blood pressure before starting antihypertensives, dermatology-specific PROs serve as a baseline from which clinicians can monitor (even remotely) for improvement or side effects with treatment and for flares. Both PROs and conventional vital signs are usually normal. It is when they are abnormal or different than expected that they become informative. We conclude by offering a roadmap for investigators to conduct the next steps in PRO research necessary to establish guidelines for transitioning PROs from clinical research and trials to routine clinical use.

Patient-reported outcomes: The anal cancer patient lived experience.

2 Background: Anal Cancer incidence and deaths from the disease have been rising in the United States for the past two decades. Data published in the Journal of the National Cancer Institute (11/19/2019) indicate from 2001 to 2015, anal cancer overall incidence increased by 2.7% per year and deaths rose by 3.1 % each year. This data indicates that anal cancer may be one of the fastest growing causes of cancer incidence and mortality. Now, especially with data indicating the rise in incidence rate, it is imperative that the anal cancer patient lived experience be better understood, including gaps in awareness and prevention education that may lead to earlier diagnosis. The Raymond Foundation in partnership with the anal cancer patient community is amplifying the patient voice by facilitating a series of impactful roundtable conversations to underscore the patient lived experience. Methods: From March–August 2021, the Raymond Foundation convened virtual roundtable conversations and individual interviews with 171 anal cancer patients and survivors. Overarching themes from our conversations included: Barriers to earlier diagnosis and awareness, Frustration and anger by the lack of new treatment protocols, Quality of life after diagnosis, including daily distress levels leading to allostatic load. Results: 94% of patients/survivors reported lack of anal cancer awareness and prevention education that may have led to a later stage diagnosis; 93% reported feeling stigmatized with a marked decrease in quality of life post diagnosis; 90% reported feeling embarrassed when discussing their diagnosis with family and friends; 86% reported frustration and anger by the lack of new treatment options; Anxiety (81%), Fear (78%) Depression (73%) were common daily distress concerns; Reported physical effects of the disease and treatment, include Radiation Proctitis (71%) Fecal Incontinence (68%) Urinary Incontinence (65%). Less than half (44%) of patients felt empowered and comfortable advocating for themselves regarding their healthcare and treatment decisions. Conclusions: Anal cancer patients have many unmet needs–both physical and psycho-social. Their voices and lived experiences provide a roadmap to better understand these unmet needs. Based on our patient-reported outcomes research, we will continue our outreach and partnership with the anal cancer community to provide patient support, education and awareness programs and campaigns that help meet the needs of the anal cancer patient community.

Survey of 275 Patients and Caregivers Affected By Pyruvate Kinase Deficiency: Impact of Communication with Hematologists on Mental Health and Quality of Life

Background. Pyruvate Kinase deficiency (PKD) is a rare congenital hemolytic anemia which affects approximately three people per million individuals worldwide. With no disease-modifying treatments currently available, management tends to focus on supportive symptom control such as blood transfusions. PKD has a profound, wide-ranging impact on quality of life (QoL). Patient advocacy and patient-reported outcomes research has been limited. The PKD Advocacy Advisory Council (AAC), a group of patients, caregivers, patient advocates and physicians, was formed in 2020, by Agios Pharmaceuticals, to improve both timely diagnosis and access to education, support and care for individuals with PKD.Objective. The AAC initiated a survey to explore communication between patients/ caregivers affected by PKD and their hematologists to inform future best practices and improve outcomes.Methods. A web-based, quantitative and qualitative survey was conducted amongst adult patients with PKD, and adult caregivers, with respondents from 11 countries including France, Germany, Italy, Spain, the UK and US. Question types included closed-ended, multiple choice, Likert scale and binary choice plus free text. The survey was carried out according to British Healthcare Business Intelligence Association Legal and Ethical Guidelines, as well as guidelines established by the UK's Market Research Society. Participants were recruited via online panels and via AAC member channels, including PKD Facebook groups.Results: The survey was completed by 200 adult patients with PKD and 75 adult caregivers (n=275). Twenty percent of patients were >50 years of age, 64% were ages 31-50 years and 17% were 18-30 years of age. Half of the patients had been diagnosed for >10 years. Although 82% of patients reported that their hematologist manages PK deficiency “well”, the survey revealed gaps in hematologists' understanding. Only 56% of respondents stated their hematologist is able to answer disease management questions and only 44% say their hematologist searches for and finds solutions to optimize disease management. Less than half of respondents, 44%, reported that their hematologists demonstrate a deep knowledge of PKD.The survey revealed an unmet need regarding emotional and psychosocial support with 25% of respondents reporting feeling neither positive nor negative, or somewhat negative, after hematologist interactions and 29% reporting at least one negative emotion following interactions. After meeting with their hematologist, 21% of respondents report feeling worried, 17% anxious, and 17% depressed.Communication with the hematologist was reported to be negative most often among patients who receive 0 transfusions per year. Within this group, only 62% report that their hematologist manages their condition well compared to 82% overall (p=0.003). Only 51% state that their hematologist understands the impact of PK deficiency on their QoL (compared to 83% amongst respondents receiving one or more transfusions per year, p<0.001). Of those who are not transfused, only 44% state their hematologist takes their perspective and experiences into consideration for their disease management plan (compared to 75% amongst respondents receiving one or more transfusions per year, p<0.001).ConclusionsPKD is a lifelong chronic disease that significantly impacts QoL of patients and families. The results of this largest ever survey of patients and caregivers with PKD point to a need to adapt clinical approaches to improve outcomes. Hematologists should seek to improve their understanding of the disease and its burden, particularly in non-transfused patients. Care should attend to emotional and psychosocial health aspects. Clinicians should consult with other medical specialists, including hematologists who specialize in PKD, to ensure complications are managed effectively. More research is needed to build on the survey insights, drive further awareness and understanding of the needs of those living with PKD among hematologists, and inform approaches to improve outcomes.The authors would like to acknowledge and thank members of the AAC that made this work possible. [Display omitted] DisclosuresGrace: Dova: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding; Agios: Research Funding; Principia: Membership on an entity's Board of Directors or advisory committees. Barcellini: Novartis: Honoraria; Bioverativ: Membership on an entity's Board of Directors or advisory committees; Agios: Honoraria, Research Funding; Alexion Pharmaceuticals: Honoraria; Incyte: Membership on an entity's Board of Directors or advisory committees.

Optimal Methods for Reducing Proxy-Introduced Bias on Patient-Reported Outcome Measurements for Group-Level Analyses.

Caregivers, or proxies, often complete patient-reported outcomes (PROs) on behalf of patients; yet, research has demonstrated proxies rate patient outcomes worse than patients rate their own outcomes. To improve interpretability of PROs in group-level analyses, our study aimed to identify optimal approaches for reducing proxy-introduced bias in the analysis of PROs. Data were simulated based on 200 patients with stroke and their proxies who both completed 9 PROMIS domains as part of a cross-sectional study. The sample size was varied as 50, 100, 200, and 500, and the proportion of patients with proxy-respondents was varied as 10%, 20%, and 50%. Six methods for handling proxy-completions were investigated: (1) complete case analysis; (2) proxy substitution; (3) Method 2 plus proxy adjustment; (4) Method 3 including inverse-probability of treatment weighting; (5) multiple imputation; (6) linear equating. These methods were evaluated by comparing average bias in PROMIS T-scores (estimated versus observed patient-only responses), as well as by comparing estimated regression coefficients to models using patient-only responses. Overall mean T-score differences ranged from 0 to 1.75. The range of mean differences varied by the 6 methods with methods 1 and 5 providing estimates closest to the observed mean. In regression models, all but inverse-probability of treatment weighting resulted in low bias when proxy-completions were 10% to 20%. With 50% proxy-completions, method 5 resulted in less accurate estimations while methods 1 to 3 provided less proxy-introduced bias. Bias remained low across domain and varying sample sizes but increased with larger percentages of proxy-respondents. Our study found modest proxy-introduced bias when estimating PRO scores or regression estimates across multiple domains of health. This bias remained low, even when sample size was 50 and there were large proportions of proxy-completions. While many of these methods can be chosen for including proxies in stroke PRO research with <20% proxy-respondents, proxy substitution with adjustment resulted in low bias with 50% proxy-respondents.