Online Patient-reported Outcomes Research Articles

Patient-reported symptom monitoring linked to an alert algorithm: Effects on progression-free and overall survival in the SYMPRO-Lung trial.

12099 Background: Physician-initiated (active) monitoring of patient-reported outcomes (PROs) improves health-related quality of life (HRQOL) and overall survival (OS) in comparison to care as usual. We recently reported data from the SYMPRO-Lung trial showing for the first time that patient-initiated (reactive) PRO monitoring is equally effective as physician-initiated (active) monitoring in improving HRQOL. Here we investigate whether online PRO symptom monitoring via the active and reactive approach is also associated with improvements in 1-year progression-free survival (PFS) and OS compared to care as usual in lung cancer patients. Methods: SYMPRO-Lung is a Dutch multicenter randomized trial using a stepped wedge design. Inclusion criteria were (non-)small cell lung cancer stage I-IV, starting treatment. Patients in the intervention groups reported PRO symptoms weekly up to 1 year follow-up using the PRO-CTCAE lung cancer subset. If symptoms exceeded a pre-determined threshold, an alert was sent to a physician/nurse (active intervention group) or to the patient themselves (reactive intervention group). Survival was defined as time from start of treatment until progression (PFS), death (OS) or loss to follow-up. Follow-up time of all groups was calculated using the reverse Kaplan-Meier method. Cox proportional hazard analyses were performed to estimate the effect of PRO symptom monitoring on PFS and OS, adjusting for potential confounding (i.e. clinical characteristics and baseline EORTC QLQ-C30 domain scores). Results: 515 patients were included in the study (266 control, 249 intervention). The most pronounced uneven distribution in baseline characteristics was that the active intervention group had significantly more stage IV patients compared to the control group and the reactive intervention group (63% vs. 44% vs. 44%, p = 0.001). The median follow-up duration was 12 months for all groups. Age, sex, cancer staging, ECOG performance status and some baseline EORTC domain scores affected the association and were included as covariates in the analyses. Although not significant, we observed a tendency towards a PFS benefit for the active intervention group compared to care as usual (HR .78, 95%CI: .58 – 1.04) and an OS benefit for both of the intervention groups compared to care as usual (active: HR .80, 95% CI: .55 – 1.15; reactive: HR .69, 95% CI: .42 – 1.15). Conclusions: Although not significant, weekly PRO symptom monitoring linked to an alert algorithm showed a tendency towards a small 1-year survival benefit in lung cancer patients. Since we did not power the trial to demonstrate a survival benefit, the non-significant association might be due to a limited sample size. However, the positive results of symptom monitoring in improving HRQOL within our trial still provide sufficient evidence for implementation of PROMs in standard (lung) cancer care. Clinical trial information: NL7897 .

Online Patient-Reported Outcome Measure Engagement is Dependent on Demographics and Locality: Findings From an Observational Cohort.

Online patient-reported outcome measures (PROMs) enable remote collection of perceptions of health status, function, and well-being. We aimed to explore patterns of PROM completion in patients with early inflammatory arthritis (EIA) recruited to the National Early Inflammatory Arthritis Audit (NEIAA). NEIAA is an observational cohort study design; we included adults from this cohort with a new diagnosis of EIA from May 2018 to March 2020. The primary outcome was PROM completion at baseline, 3 months, and 12 months. Mixed effects logistic regression and spatial regression models were used to identify associations between demographics (age, gender, ethnicity, deprivation, smoking, and comorbidity), clinical commissioning groups, and PROM completion. Eleven thousand nine hundred eighty-six patients with EIA were included, of whom 5331 (44.5%) completed at least 1 PROM. Patients from ethnic minority backgrounds were less likely to return a PROM (adjusted odds ratio [aOR] 0.57, 95% CI 0.48-0.66). Greater deprivation (aOR 0.73, 95% CI 0.64-0.83), male gender (aOR 0.86, 95% CI 0.78-0.94), higher comorbidity burden (aOR 0.95, 95% CI 0.91-0.99), and current smoker status (aOR 0.73, 95% CI 0.64-0.82) also reduced odds of PROM completion. Spatial analysis identified 2 regions with high (North of England) and low (Southeast of England) PROM completion. We define key patient characteristics (including ethnicity) that influence PROM engagement using a national clinical audit. We observed an association between locality and PROM completion, with varying response rates across regions of England. Completion rates could benefit from targeted education for these groups.

Sexual well-being in patients with early-stage breast cancer at 1- and 2-year follow-up.

Sexual well-being (SWB) is an important aspect of overall quality of life and should therefore be considered when measuring the effect of breast cancer on daily life. To identify positive and negative predictive factors associated with change in SWB 1 year after diagnosis (T12; hereafter, ∆SWB) and whether SWB changes the year after. All data were derived from an online patient-reported outcome measure that included patients aged >18 years who were treated for breast cancer between October 2015 and March 2022 at the Erasmus University Medical Center. Multivariable linear regression was used to analyze the association between demographic- and disease-specific variables and change in SWB between time of diagnoses (T0) and one year after (T12) (∆SWB). For defining the clinical relevance of ∆SWB, patients were divided into 3 groups based on their SWB score at T12: decreased, stable, and improved. Wilcoxon signed rank test was used to test the difference in SWB between T12 and T24 (2 years after diagnosis) in all 3 groups. Outcomes included the associations between demographic- and disease-specific variables and ∆SWB (T0 vs T12) and change in SWB the year after (T12 vs T24). An overall 204 patients were included, with a mean age of 51.7 years (SD, 12.8) and a mean SWB score of 64.3 (SD, 20.9) at T0. Body mass index >30 kg/m2 at T0 had a significant negative association (β = -8.369, P = .019) with ∆SWB. Reconstruction (β = 20.136, P < .001) and mastectomy (β = 11.157, P < .001) had a significant positive association with ∆SWB vs lumpectomy. Change in psychological well-being had a significant positive relation to ∆SWB (β = 0.349, P < .001). Patients with decreased SWB at T12 did not improve the year after (P = .376). By identifying the variables that are associated with decreased SWB during the trajectory of breast cancer treatment and by defining the clinical relevance of decreased SWB, patient groups can be targeted and offered extra support. This study is one of the first to analyze the development of SWB, instead of sexual function, over time in patients with breast cancer, and it uses data over a longer period. However, only one-third of the patients responded to the SWB domains at both time points. Type of operation, body mass index >30, and change in psychological well-being were associated with ∆SWB. Patients with decreased SWB 1 year after diagnosis tended not to improve or normalize the year after, indicating that intervention is needed to restore SWB in this specific group.

Abstract A020: Patients with pancreatic cancer reporting prediagnosis pain have greater pain intensity scores, symptom burden, and ER visits: need for improved, ongoing management in pancreatic pain and associated symptoms

Abstract Objective: This study assessed the pancreatic cancer (PC) patient-reported burdens of PC pain from 2016-2020 in an online patient-reported outcomes (PRO) survey supported by the Pancreatic Cancer Action Network (PanCAN). Assessments of PC symptoms, PC pain intensity, physical and mental functioning, associated symptom management and resource utilization were analyzed between PC patients reporting prediagnosis PC pain vs those who did not. Methods: Through years 2016 - 2020, PC patient volunteers filled out an online PRO survey composed of independent surveys and standalone items in this cross-sectional study. PC patient responses to Patient Information, PC Experience, General Health Assessment and Pain Management surveys were collected to assess PC pain and related factors in activities and associated symptoms. Comparisons between PC patient group responses to two survey items were assessed: 1) presence or absence of prediagnosis PC pain; 2) pain intensity, scored by an 11-point numeric rating scale (NRS). Patient groups: NRS=0-3 (none to mild pain) and NRS=5-8 (moderate to severe pain). Descriptive statistics were used for numbers and frequencies and all bivariate analyses were performed using Chi-square or Fisher’s Exact tests. Results: Survey responses to 108 items ranging from 5-100% participation were collected and analyzed from 1978 PC patient volunteers. Significant differences were noted in postdiagnosis average pain intensities based on presence or absence of prediagnosis pain: NRS 2.64+2.54 vs NRS 1.56+2.01, respectively, p=0.0039. The presence of PC pain (abdominal and/or back pain) was the most frequent prediagnosis symptom reported (62%, N=1222) followed by digestive problems (55.32%, p=0.0001), weight loss and/or loss of appetite (54.88%, p=0.0001) and fatigue (46.54%, p=0001). Significantly greater impairment of those with prediagnostic pain was noted in 45% of 20 health assessment statements, including pain interference&amp;gt;physical functioning &amp;gt;anxiety &amp;gt;depression &amp;gt;fatigue, and increased resource utilization for PC pain management was noted (ER visits N=42 vs N=8 respectively, p=0.018 and/or hospitalizations, N=30 vs N=4, respectively, p=NS). Patient responses to pain ranged from NRS 0-8 (N=155), and group NRS 5-8 scores reported had significantly greater impairment in 85% of 20 health assessment statements. No improvements in average pain intensity scores were noted between yrs 2018-2020 vs yrs prior to 2018. Conclusions: PC pain is the most frequent prediagnostic PC symptom and is associated with an increase in postdiagnosis average pain intensity, greater impairment in physical function, and an increase in ER visits compared to those who do not report prediagnostic pain. The results suggest that PC pain is not adequately managed early in the course of the disease. There is a need to better assess symptoms, QOL, and therapeutic interventional outcomes to determine the contribution of improvements in tumor reduction and survival with patient symptom reduction and better physical and mental functioning. Citation Format: Terry A. McNearney, Biai D.E. Digbeu, Dennis Ladnier, Kawther Abdilleh, Sudheer Doss, Lynn M. Matrisian, Jacques G. Baillargeon. Patients with pancreatic cancer reporting prediagnosis pain have greater pain intensity scores, symptom burden, and ER visits: need for improved, ongoing management in pancreatic pain and associated symptoms [abstract]. In: Proceedings of the AACR Special Conference on Pancreatic Cancer; 2022 Sep 13-16; Boston, MA. Philadelphia (PA): AACR; Cancer Res 2022;82(22 Suppl):Abstract nr A020.

Early Experience and Patient Characteristics from Opera: A Real-World Study of Pegcetacoplan Treatment

Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired life-threatening disease characterized by complement-mediated hemolysis and thrombosis. Chronic hemolysis leads to anemia, which can result in persistent fatigue and dependance on transfusions, affecting the overall quality of life (QOL) of patients with PNH. Pegcetacoplan (PEG; US brand name Empaveli®) is the first approved C3 inhibitor for US adults with PNH. Although clinical trials have assessed the efficacy of PEG, which include patient-reported outcomes, there is no information on the use of PEG in a real-world setting. Aims To provide the first report of trends in hemoglobin (Hb) values and baseline QOL (measured by patient-reported fatigue and cognitive impairment) from OPERA, a descriptive, observational exploratory outcomes study, presenting the first real-world data describing PEG treatment for adults with PNH post-approval. Methods This single site opt-in study recruited patients in the US, following a valid PEG prescription from a licensed medical professional. Patients ≥18 years of age, diagnosed with PNH and prescribed PEG, were electronically consented and then enrolled to participate in the OPERA study, as approved by the internal review board. OPERA collected information from routine care and did not direct or suggest any medical interventions. In this observational setting, Hb lab values were reported (by the patient/site) at varying timepoints when available. Participants self-reported QOL status from baseline over a 12-month period through monthly phone calls and quarterly online patient-reported outcome measures (PROMs). During monthly phone calls, participants were asked to report their most recent Hb levels (typical normal range: male, 13.6-18 g/dL; female, 12-16 g/dL) if available from their regular primary care visits in the previous month. Online PROMs collected data using validated QOL instruments including the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale (0-52 total score) and the Patient-Reported Outcomes Measurement Information System (PROMIS) scale for Cognitive Abilities (23.27-67.09 t-score). Lower scores on each measure represent greater levels of fatigue or cognitive impairment. Results Participants enrolled in OPERA with a 30-70% monthly range to opt-in. Of 17 participants enrolled with available baseline Hb values, mean (range) age was 45.76 (20-70) years, and 8 were female. No OPERA participants were previously enrolled in a PEG clinical trial. All participants were prescribed and received the label recommended dose of PEG (1080 mg, twice weekly). All participants reported prior treatment with either eculizumab, ravulizumab or both, before starting PEG. Additional dosing information and time since PNH diagnosis can be found in the Table. The mean (SD) baseline Hb levels from 17 OPERA participants was 8.90 (2.19) g/dL, which was similar to the mean (SD) baseline Hb levels (8.69 [1.08] g/dL) for 41 patients in the previously published phase 3 PEGASUS (NCT03500549) trial. Among 4 participants with available data after 1 month of PEG use, mean (SD) Hb levels increased to 11.95 (1.84) g/dL (mean change from baseline +2.83 (1.35) g/dL) which was similar to the mean (SD) Hb levels of 11.55 (1.62) g/dL seen in 40 PEGASUS participants after 1 month of treatment. QOL data was available at baseline in 12 participants and the mean (SD) baseline FACIT-Fatigue score was 30.25 (15.63), which was similar to the mean (SD) baseline FACIT-Fatigue score of 32.2 (11.4) from the PEGASUS trial, but below the reported mean FACIT-Fatigue population norm of 43.6 (Cella, et al. Cancer. 2002). Additionally, the mean (SD) PROMIS-Cognitive Abilities t-score for patients with available QOL data (n=12) was 43.57 (9.11), which is lower than the published population norm of 50 (Saffer, et al. Psychiatry Research 2015). Conclusions This first real-world study of adults with PNH receiving PEG indicates a positive trend in Hb levels in participants after 1 month of use. These early experiences are similar to previously reported changes in Hb levels after PEG treatment in phase 3 clinical trials (Hillmen et al, NEJM 2021). At baseline, participants with PNH report a considerable amount of fatigue and cognitive impairment, indicating poor QOL with previous C5 inhibitor treatment. More research is needed to corroborate the findings of this first analysis. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Abstract 11516: Heart Failure Patient Experiences With a Visualized Patient-Reported Outcome Monitoring Tool to Facilitate Beta-Blocker Decision-Making in an N-of-1 Trial

Introduction: Emerging evidence suggests that beta-blockers may not be efficacious in preventing mortality in patients with heart failure with preserved ejection fraction (HFpEF) and could worsen exercise capacity. HFpEF patients’ symptoms often overlap with medication side effects, making precise decision-making about the need for beta-blockers a challenge. Objective: Evaluate patient attitudes toward using a patient-reported outcome (PRO) monitoring tool to facilitate shared decision-making about beta-blocker use in an N-of-1 clinical trial. Methods: Eligible HFpEF patients taking a beta-blocker were recruited from a heart failure clinic. Patients alternated between taking and not taking their beta-blocker for 2-week periods, with dedicated titration time in-between. Data were ascertained through exercise assessments, weekly/bi-weekly online PRO surveys, and remote vital measurements. Average symptom scores on and off beta-blocker were presented in a visualized report to patients, intended to facilitate a discussion about continuing vs. discontinuing their beta-blocker. We interviewed patients about feedback on study procedures and agency for decision-making at baseline and study visits. Interviews were recorded and transcribed. Three study team members analyzed interviews using directed content analysis. Results: To date, 9 patients completed the intervention, and 28 interviews were completed. Most participants were female and White (89%) with an average age of 78. Participants reported that their visualized PRO results were aligned their experiences, which reinforced their use of the tool and agency in beta-blocker decision-making (Table). Conclusions: Patients found that the visualized report helped to facilitate beta-blocker shared decision-making. Future research should evaluate the utility of integrating longitudinal PRO monitoring tools into usual care to facilitate individualized decisions about medications.

Orthopaedic Foot and Ankle Post-Operative Outcomes: The University of Colorado Orthopaedic Surgery Monitoring of Outcomes System (U-COSMOS)

Category: Other; Ankle; Hindfoot; Lesser Toes; Midfoot/Forefoot Introduction/Purpose: The automation and integration of patient reported outcomes (PROs) into standard of care practice and electronic health systems is becoming increasingly viable. While often collected as a quality assurance measure for a practice and surgical decision-making, PROs are also valuable to assess the relative treatment outcomes. In order for physicians to gain the most insight from PROs, more baseline outcomes are needed to serve as a benchmark of anticipated patient improvement following surgical interventions. The goal of this study is to evaluate differences in patient improvement among validated physical and mental health PROs in a foot and ankle surgery practice. Methods: Consecutive patients undergoing foot and ankle surgery were enrolled into the U-COSMOS pathway. All PROs are automatically distributed and tracked through a secure, HIPAA-compliant, online PRO data capture tool. All patients are invited to complete the PROMIS Physical Function (PF) Computer Adaptive Test (CAT), PROMIS Pain Interference CAT (PI), Foot and Ankle Single Assessment Numeric Evaluation (SANE), PROMIS Global Physical Health (GPH), PROMIS Global Mental Health (GMH), PROMIS Depression, and a brief resiliency scale (BRS). PROs are collected pre-operatively, and post-operatively at 3- months, 6-months, 12-months, and 24-months. In this study, differences between pre- and post-operative scores for each PRO are evaluated using an unpaired Mann-Whitney U Test. Results: To date, 3154 patients have been enrolled into the U-COSMOS foot and ankle surgery pathway. Overall survey compliance was 77%. A significant improvement was seen in PF, PI, SANE, and GPH at 6-months, 12-months, and 24-months (p &lt; 0.0001, see Table 1). There was no significant improvement in PF or GPH at the 3-month mark, supporting reduction of pain interference may be observed before return of physical function. All PROs measuring changes in mental health or resilience (Depression, GMH, BRS) did not significantly change at any post-operative visit relative to baseline. We found a mild positive correlation between BRS and improved 12-month PF and PI outcomes and a mild inverse correlation between pre-operative depression and 12-month PI and PF scores. Conclusion: Our data suggests that foot and ankle surgery leads to significant improvement in physical outcomes such as function or pain interference, but mental health and resilience outcomes remain somewhat unchanged throughout treatment on average. Within physical outcomes, we also find significant improvement in pain interference precedes functional and general measures. Early findings from the U-COSMOS outcomes pathway demonstrates greater overall improvement in physical health PROs, and a sustained improvement up to 24 months post-op. Future investigation of outcomes specific to procedures and conditions within the U-COSMOS pathway will help elucidate anticipated variation from general trends.

Safety and tolerability of pirfenidone in asbestosis: a prospective multicenter study

BackgroundPirfenidone slows down disease progression in idiopathic pulmonary fibrosis (IPF). Recent studies suggest a treatment effect in progressive pulmonary fibrosis other than IPF. However, the safety and effectiveness of pirfenidone in asbestosis patients remain unclear. In this study, we aimed to investigate the safety, tolerability and efficacy of pirfenidone in asbestosis patients with a progressive phenotype.MethodsThis was a multicenter prospective study in asbestosis patients with progressive lung function decline. After a 12-week observational period, patients were treated with pirfenidone 801 mg three times a day. Symptoms and adverse events were evaluated weekly and patients completed online patient-reported outcomes measures. At baseline, start of therapy, 12 and 24 weeks, in hospital measurement of lung function and a 6 min walking test were performed. Additionally, patients performed daily home spirometry measurements.ResultsIn total, 10 patients were included of whom 6 patients (66.7%) experienced any adverse events during the study period. Most frequently reported adverse events were fatigue, rash, anorexia and cough, which mostly occurred intermittently and were reported as not very bothersome. No significant changes in hospital pulmonary function (forced vital capacity (FVC), diffusion capacity of the lung for carbon monoxide (DLCO), 6 min walking test or patient-reported outcomes measures before and after start of pirfenidone were found. Home spirometry demonstrated a FVC decline in 12 weeks before start of pirfenidone, while FVC did not decline during the 24 week treatment phase, but this difference was not statistically significant.ConclusionsTreatment with pirfenidone in asbestosis has an acceptable safety and tolerability profile and home spirometry data suggest this antifibrotic treatment might attenuate FVC decline in progressive asbestosis.Trial registration MEC-2018-1392; EudraCT number: 2018-001781-41

User-Centered Development and Testing of the Online Patient-Reported Outcomes, Burdens, and Experiences (PROBE) Survey and the myPROBE App and Integration With the Canadian Bleeding Disorder Registry: Mixed Methods Study.

BackgroundThe Patient-Reported Outcomes, Burdens, and Experiences (PROBE) questionnaire is a tool for assessing the quality of life and disease burden in people living with hemophilia.ObjectiveThe objectives of our study were (1) to assess the needs of relevant stakeholders involved in the use of PROBE, (2) to develop the software infrastructure needed to meet these needs, and (3) to test the usability of the final product.MethodsWe conducted a series of semistructured interviews of relevant stakeholders, including PROBE investigators, people with hemophilia, and representatives of the sponsor. Based on these, we developed an online survey and a mobile app for iOS and Android. A user group evaluated the final product using the System Usability Scale (SUS) and an open feedback framework.ResultsThe online survey was updated, and the myPROBE app for mobile devices and a new application programming interface were developed. The app was tested and modified according to user feedback over multiple cycles. The final version of the app was released in July 2019. Seventeen users aged 23 to 67 years evaluated the final version of the app using the SUS. The median (first, third quartile) SUS score for the app was 85 (68, 88) out of 100. The newly introduced functionalities were as follows: (1) capability to longitudinally track repeated fillings of the questionnaire at different time points by the same participant (as opposed to anonymous completion); (2) linking of the questionnaire with hemophilia registries, starting with the Canadian Bleeding Disorders Registry as a proof of concept; (3) removing or adding questions as needed; and (4) sending notifications to the users (eg, reminders). A new secure database was built for securely storing personal information separately from the questionnaire data. The PROBE online survey is currently available in 96 countries and 34 languages.ConclusionsThe online survey was updated successfully, and the myPROBE app was developed, with a SUS score of 85 (out of 100). The app has been released in 81 countries and 34 languages. This will facilitate data collection for research and advocacy purposes, and the use of this tool in everyday clinical practice.

Preferences for Accessing Patient Reported Outcomes and Health Information Among Thrombotic Thrombocytopenic Purpura Survivors

Introduction: Our previous qualitative studies of immune-mediated thrombotic thrombocytopenic purpura (iTTP) survivors revealed that patients did not communicate to their hematologists about residual cognitive and fatigue issues. A way to overcome this communication barrier is implementation of patient-reported outcomes (PROs) in routine care. Integration of PROs into the clinical care of other diseases has resulted in improved patient-provider communication, symptom management, quality of life and overall patient satisfaction. However, decisions about the mode of administration are challenging. Although 92% of the United States population has access to the internet, studies have shown minorities prefer alternative modes of administration. Given that iTTP disproportionately affects Black women, understanding patient preference is critical for integrating PRO instruments into routine care. The primary study goal was to determine the preferred mode of administration of PRO instruments in iTTP. Furthermore, many individuals use the internet as a source of medical information/advice. A study of iTTP literacy reported that only 34% of survivors correctly identified disease relapse risk factors suggesting a critical knowledge gap. A secondary goal was to describe iTTP survivors' behaviors regarding using the internet for medical information or support.Methods: We utilized a cross-sectional study design. iTTP survivors were recruited from August 2019 until present. Eligibility included: 1) age >18 years, 2) documented ADAMTS13 deficiency (< 10% activity) at diagnosis or during a relapse and 3) > 1-year clinical remission. Multi-center recruitment of survivors included: Oklahoma University, Ohio State University, University of Minnesota, Johns Hopkins University, University of Rochester, University of Pennsylvania, University of Alabama at Birmingham, University of Utah and the University of Vermont. Following informed consent, survivors were administered the PROMIS ® cognitive function ability, anxiety and fatigue instruments via their preferred mode (online, telephone, or self-administered). Typical internet usage, behaviors regarding searching for health information online and demographics were also obtained.Results: To date, 94 survivors have completed the study (83% female; 54% White; 34% Black; median age 49 years [range 26-85 years]). A majority (54%) preferred completing PROMIS ® surveys online vs. self-administered or telephone administered. However, among Black survivors, only 38% preferred online administration and among survivors aged ≥65 years only 22% preferred online administration. Interestingly, there was an overall shift in a preference toward online administration following the onset of the COVID-19 pandemic (45% (21/47) preferred online pre vs 66% (31/47) preferred online post).Ninety-one percent (86/94) of survivors reported at least occasional internet use vs 8 (9%) reported none. Similarly, 82% (70/85) had searched the internet for health/medical information for themselves in the past year. Also 62% (53/86) of the survivors selected ‘strongly agree’ or ‘agree’ to the statement that the internet helps them determine if symptoms are important enough to see a doctor. Likewise, 62% used the internet to interpret doctor's recommendations. Additionally, 47% (40/86) ‘strongly agree/agree’ the internet helped determine if they would take a medication/seek alternative treatment.Moreover, 64% (55/86) agreed the internet was a good way to find others experiencing similar health problems. However, when asked about behaviors over the past year, only 42% (36/85) used online social networking sites like Facebook to look for health information or find others with iTTP and only 36% (31/85) had actually participated in online iTTP support groups.Conclusions: Overall iTTP survivors preferred online PRO administration; however, Black and older survivors preferred other methods. Recognizing these preferences is a vital step toward integrating PROs into routine care. Furthermore, iTTP survivors are using the internet as a source of medical support and information. Therefore, it is critical to not only educate iTTP survivors about credible online resources but also to create additional content. Also, future studies are needed to further explore the impact of the COVID-19 pandemic on online health behaviors. DisclosuresTerrell: Sanofi: Consultancy; Takeda: Consultancy. Journeycake: HEMA Biologics: Honoraria; LFB: Honoraria. Mazepa: Answering TTP Foundation: Research Funding; Sanofi Aventis: Other. Cuker: Spark Therapeutics: Research Funding; Sanofi: Research Funding; Pfizer: Research Funding; Takeda: Research Funding; Novo Nordisk: Research Funding; Novartis: Research Funding; Bayer: Research Funding; Alexion: Research Funding; UpToDate: Patents & Royalties; Synergy: Consultancy. Chaturvedi: Dova: Other: Advisory board member; UCB: Other: Advisory board participation; Argenx: Other: Advisory board member; Alexion: Other: Advisory board member; Sanofi Genzyme: Other: Advisory board member. Lim: Hema Biologics: Honoraria; Sanofi Genzyme: Honoraria; Dova Pharmaceuticals: Honoraria. Gangaraju: Alexion: Consultancy; Sanofi Genzyme: Consultancy. Cataland: Alexion: Consultancy, Research Funding; Sanofi Genzyme: Consultancy; Ablynx/Sanofi: Consultancy, Research Funding; Takeda: Consultancy.

A Mixed Methods Study of Hysterectomy in a U.S. Sample of Deaf Women Who Use American Sign Language.

Background: Hysterectomy is one of the most common procedures performed in the United States. Yet, we know nothing about deaf women's experiences with hysterectomy. The study aims to establish a prevalence of hysterectomy among deaf women and provide insight into the experiences of those who have undergone hysterectomy.Materials and Methods: Quantitative data (n = 195; 27% Black, Indigenous, People of Color) were collected through a bilingual online patient-reported outcomes survey and reproductive health questions from the National Health and Nutrition Examination Survey (NHANES) between November 2019 and March 2020. Semistructured interviews were conducted between March and April 2021 with a smaller sample of deaf women who underwent hysterectomy. A multivariable logistic regression model identified the relationship between health care history and sociodemographic factors, while qualitative interview data were used to understand deaf women's experiences with hysterectomy.Results: Of the 195 deaf respondents, 34% underwent hysterectomy (n = 67). Results indicated that the odds of hysterectomy increased for higher age (per year), being African American/Black or Latinx, being married or living with a partner, being overweight or obese, and if communicating with the doctor through English writing or others. Qualitative interviews were conducted with eight women who provided consent to participate. Although all women reported improved quality of life posthysterectomy, patient-centered experience and decision making before hysterectomy were highly dependent on access to communication, information sources, and social support.Conclusions: Prioritizing the needs of deaf women leading up to, during, and after hysterectomy has the potential to improve overall experience with hysterectomy and patient–clinician communication.

Leveraging patient-reported outcomes (PROs) in patients with pancreatic cancer: The Pancreatic Cancer Action Network (PanCAN) online patient registry experience.

BackgroundThe Pancreatic Cancer Action Network (PanCAN) Patient Registry is an online, pancreatic cancer‐specific, global registry enabling patients to self‐report sociodemographics, disease/management characteristics, and patient‐reported outcomes (PROs). We sought to describe the creation, user experience, and research potential of the PanCAN Registry.MethodsWe obtained data to describe (1) the creation of the Registry (questionnaire development, marketing efforts, and regulatory considerations); (2) the user experience (user characteristics and interactions with the registry following inception); and (3) the research potential of the registry (comparing PROs and treatment patterns by age [±65 years] and treatment site [community or academic] for users with de novo metastatic disease).ResultsThe Registry was conceived as part of PanCAN’s strategic plan for a personalized therapy initiative. PanCAN staff and disease expert consultants developed questionnaires hosted on an electronic PRO platform. Users had the option to include their data in research efforts, and the Registry platform received institutional review board approval. From 7/2015 to 12/2020, 2187 patients visited the registry and 1697 (77.6%) completed at least one survey (median age = 64 years [range: 24–90], 47.9% women, 88.7% White, 34.0% metastatic disease). Among patients with metastatic disease (N = 567), 46.0% were ≥65 years old and 67.5% received treatment at community sites. Patients ≥65 years reported feeling less hopeful about the treatment plan (12.4% vs. 24.3%, p = 0.003), and patients treated at community sites reported more frequent treatment breaks of >2 weeks (58.2% vs. 28.1%, p < 0.001).ConclusionsOur findings demonstrate the feasibility, usability, and research potential of an online PRO registry for patients with cancer. This description of the PanCAN Registry should inform future registry‐building efforts to facilitate standardized PRO reporting and provide a valuable research database. Clinical trial registration number: Not applicable.

Age at surgery is correlated with pain scores following trochlear osteotomy in lateral patellar instability: a cross-sectional study of 113 cases

BackgroundA trochlear osteotomy aims to restore patellar stability in patients with recurrent patellar instability and trochlear dysplasia. The age of patients at time of surgery could be a relevant factor which influences outcome. We hypothesized that lower age at time of surgery is associated with better patient-reported outcomes.MethodsA retrospective study was conducted on patients with patellar instability and trochlear dysplasia. Patients were contacted by phone for informed consent and were then asked to complete online patient-reported outcome measurements (PROMs). The PROMs consisted of the Kujala Knee Score (KKS) (Kujala et al., Arthroscopy 9(2):159-63, 1993; Kievit et al. Knee Surg Sports Traumatol Arthrosc. 21(11):2647-53, 2013), the Short Form 36-item health survey (SF-36v1) (Ware, Med Care 73-83, 1992; Aaronson et al., J Clin Epidemiol. 51(11):1055-68, 1998), and visual analog scale (VAS) scoring pain, instability, disability, and satisfaction on a 0–100 scale. Multivariable linear regression models were used to study the effect of age on the PROM scores.ResultsFor this study, 125 surgical procedures in 113 patients were included. Mean VAS pain at rest was 19 and at activity 38; mean Kujala score was 73. Multivariable regression analysis revealed that age at the time of surgery was correlated with VAS pain at rest, with a 0.95 increase of VAS score (scale 0–100) for every year of age. Recurrence of instability was observed in 13 (10%) knees.ConclusionIn this cross-sectional study, pain scores of 113 patients who have undergone a lateral facet elevating trochlear osteotomy for patellar instability were reported. Age at time of surgery was correlated with an increased pain score at rest with an average of 9.5 points (scale 0–100) for every 10 years of age. Age at time of surgery was not correlated with overall satisfaction.

Five-Year Outcome of 1-Stage Cell-Based Cartilage Repair Using Recycled Autologous Chondrons and Allogenic Mesenchymal Stromal Cells: A First-in-Human Clinical Trial

Background: Long-term clinical evaluation of patient outcomes can steer treatment choices and further research for cartilage repair. Using mesenchymal stromal cells (MSCs) as signaling cells instead of stem cells is a novel approach in the field. Purpose: To report the 5-year follow-up of safety, clinical efficacy, and durability after treatment of symptomatic cartilage defects in the knee with allogenic MSCs mixed with recycled autologous chondrons in first-in-human study of 1-stage cartilage repair. Study Design: Case series; Level of evidence, 4. Methods: This study is an investigator-driven study aiming at the feasibility and safety of this innovative cartilage repair procedure. Between 2013 and 2014, a total of 35 patients (mean ± SD age, 36 ± 8 years) were treated with a 1-stage cartilage repair procedure called IMPACT (Instant MSC Product Accompanying Autologous Chondron Transplantation) for a symptomatic cartilage defect on the femoral condyle or trochlear groove. Subsequent follow-up after initial publication was performed annually using online patient-reported outcome measures with a mean follow-up of 61 months (range, 56-71 months). Patient-reported outcome measures included the KOOS (Knee injury and Osteoarthritis Outcome Score), visual analog scale for pain, and EuroQol–5 Dimensions. All clinical data and serious adverse events, including additional treatment received after IMPACT, were recorded. A failure of IMPACT was defined as a chondral defect of at least 20% of the index lesion with a need for a reintervention including a surgical procedure or an intra-articular injection. Results: Using allogenic MSCs, no signs of a foreign body response or serious adverse reactions were recorded after 5 years. The majority of patients showed statistically significant and clinically relevant improvement in the KOOS and all its subscales from baseline to 60 months: overall, 57.9 ± 16.3 to 78.9 ± 17.7 (P < .001); Pain, 62.3 ± 18.9 to 79.9 ± 20.0 (P = .03); Function, 61.6 ± 16.5 to 79.4 ± 17.3 (P = .01); Activities of Daily Living, 69.0 ± 19.0 to 89.9 ± 14.9 (P < .001); Sports and Recreation, 32.3 ± 22.6 to 57.5 ± 30.0 (P = .02); and Quality of Life, 25.9 ± 12.9 to 55.8 ± 26.8 (P < .001). The visual analog scale score for pain improved significantly from baseline (45.3 ± 23.6) to 60 months (15.4 ± 13.4) (P < .001). Five cases required reintervention. Conclusion: This is the first study showing the midterm safety and efficacy of the proof of concept that allogenic MSCs augment 1-stage articular cartilage repair. The absence of serious adverse events and the clinical outcome support the longevity of this unique concept. These data support MSC-augmented chondron transplantation (IMPACT) as a safe 1-stage surgical solution that is considerably more cost-effective and a logistically advantageous alternative to conventional 2-stage cell-based therapy for articular chondral defects in the knee.

Design of a virtual longitudinal observational study in Parkinson's disease (AT-HOME PD).

ObjectiveThe expanding power and accessibility of personal technology provide an opportunity to reduce burdens and costs of traditional clinical site‐centric therapeutic trials in Parkinson’s disease and generate novel insights. The value of this approach has never been more evident than during the current COVID‐19 pandemic. We sought to (1) establish and implement the infrastructure for longitudinal, virtual follow‐up of clinical trial participants, (2) compare changes in smartphone‐based assessments, online patient‐reported outcomes, and remote expert assessments, and (3) explore novel digital markers of Parkinson’s disease disability and progression.MethodsParticipants from two recently completed phase III clinical trials of inosine and isradipine enrolled in Assessing Tele‐Health Outcomes in Multiyear Extensions of Parkinson’s Disease trials (AT‐HOME PD), a two‐year virtual cohort study. After providing electronic informed consent, individuals complete annual video visits with a movement disorder specialist, smartphone‐based assessments of motor function and socialization, and patient‐reported outcomes online.ResultsFrom the two clinical trials, 226 individuals from 42 states in the United States and Canada enrolled. Of these, 181 (80%) have successfully downloaded the study’s smartphone application and 161 (71%) have completed patient‐reported outcomes on the online platform.InterpretationIt is feasible to conduct a large‐scale, international virtual observational study following the completion of participation in brick‐and‐mortar clinical trials in Parkinson’s disease. This study, which brings research to participants, will compare established clinical endpoints with novel digital biomarkers and thereby inform the longitudinal follow‐up of clinical trial participants and design of future clinical trials.

Severity of Anosmia as an Early Symptom of COVID-19 Infection May Predict Lasting Loss of Smell.

Introduction: To evaluate the recovery rate of loss of smell (LOS) with objective olfactory testing in COVID-19 patients.Methods: Adults with confirmed COVID-19 and self-reported sudden LOS were prospectively recruited through a public call from the University of Mons (Belgium). Epidemiological and clinical data were collected using online patient-reported outcome questionnaires. Patients benefited from objective olfactory evaluation (Sniffin-Sticks-test) and were invited to attend for repeated evaluation until scores returned to normal levels.Results: From March 22 to May 22, 2020, 88 patients with sudden-onset LOS completed the evaluations. LOS developed after general symptoms in 44.6% of cases. Regarding objective evaluation, 22 patients (25.0%) recovered olfaction within 14 days following the onset of LOS. The smell function recovered between the 16th and the 70th day post-LOS in 48 patients (54.5%). At the time of final assessment at 2 months, 20.5% of patients (N = 18) had not achieved normal levels of olfactory function. Higher baseline severity of olfactory loss measured using Sniffin-Sticks was strongly predictive of persistent loss (p < 0.001).Conclusion: In the first 2 months, 79.5% of patients may expect to have complete recovery of their olfactory function. The severity of olfactory loss, as detected at the first Sniffin-Sticks-test, may predict the lack of mid-term recovery.

Feasibility of online home spirometry in systemic sclerosis-associated interstitial lung disease: a pilot study.

ObjectivesFrequent monitoring of forced vital capacity at home may be of added value in patients with SSc-associated interstitial lung disease (SSc-ILD) to monitor disease progression and guide treatment decisions. The aim of this study was to evaluate the feasibility and optimal frequency of online home spirometry using a home monitoring application in patients with SSc-ILD.MethodsThis was a prospective, observational study in patients with SSc-ILD. Patients evaluated for 3 months the online home monitoring application ILD-online integrated with a Bluetooth-connected spirometer. Patients performed daily home spirometry for 6 weeks and weekly home spirometry for 6 weeks. In addition, patients completed an evaluation questionnaire after 3 months and online patient-reported outcomes at baseline and 3 months.ResultsTen consecutive patients participated. Mean adherence to home spirometry was 98.8% (s.d. 1.5). Home and hospital spirometry were highly correlated. The mean coefficient of variation was lower for weekly [2.45% (s.d. 1.19)] than daily [3.86% (s.d. 1.45)] forced vital capacity measurements (P = 0.005). All patients considered the home monitoring application and spirometer easy to use and no patients considered home spirometry burdensome. All patients would recommend home monitoring to other patients with SSc.ConclusionsHome spirometry using an online home monitoring application is feasible in patients with SSc-ILD, with high adherence and patient satisfaction. Larger long-term studies are needed to assess whether home spirometry can detect the progression of ILD in patients with SSc.

Leveraging patient-reported outcomes (PROs) in patients with pancreatic cancer: The Pancreatic Cancer Action Network (PanCAN) online patient registry experience.

154 Background: By allowing patients (pts) to self-report key issues related to their quality of life and symptoms, PROs have important clinical and research implications. The PanCAN Registry, which began collecting data in July 2015, is a pancreatic cancer-specific global online registry enabling pts to report sociodemographics, disease/management characteristics, and PROs via online surveys. We sought to describe pt experiences with the PanCAN Registry. Methods: We assessed individual characteristics and interactions with the registry (visits, survey completions, and longitudinal use) from 7/2015-10/2019 for pts who provided permission to use their data. The registry allows pts to complete surveys about their experience (e.g. basics of pancreatic cancer, general information), symptoms (e.g. fatigue, pain), diagnostics (e.g. labs, scans), and drug therapy (e.g. type, frequency). We validated PROs using the PanCAN Know Your Tumor database. For a subset of pts (those with de novo metastatic disease), we compared PROs, treatment patterns, and side effects by age (+/- 65 years) and treatment site (community or academic). Results: Of 2,836 pts who visited the registry, 2,076 (73%) completed at least one survey (median age = 64 [range: 18-97], 48% women, 92% white, 32% metastatic disease). Pts most commonly completed the basics (73%), general information (39%), and drug therapy (37%) surveys. Overall, 10% answered surveys longitudinally. We observed 95% concordance between PROs and the PanCAN Know Your Tumor database. Among the 667 pts with de novo metastatic disease, 34% were older (age 65+) and 50% were treated at academic sites. Younger pts were more hopeful about the treatment plan (strongly agree: 24% v 12%, p &lt; .01) and reported less constipation (moderate/severe: 33% v 48%, p &lt; .01) compared with older pts. Pts treated at academic sites reported less frequent treatment breaks of &gt; 2 weeks (28% v 58%, p = 0.01) and more frequent severe cytopenias (27% v 12%, p = 0.01) compared with those treated at community sites. Conclusions: With &gt; 2,800 pts visiting the PanCAN registry and &gt; 70% completing a survey, these findings demonstrate the feasibility, robustness, and research potential of an online PRO registry. We observed important differences by age and treatment site regarding pts’ outlook, symptoms, treatment patterns, and side effects. With increasing focus on PROs, registries like this can facilitate standardized PRO reporting and monitoring, while also providing a valuable research database.

Patient-reported outcomes integrated within electronic medical record in patients with head and neck cancer.

e14026 Background: Patient-reported outcome (PRO) tools have been shown to improve outcomes including overall survival for cancer patients. However, routine implementation of PROs in clinical practice within the electronic medical record (EMR) remains limited. We studied the use of a PRO tool for patients with head and neck cancer (HNC) integrated in an EMR. Methods: Between August 2017 and December 2019 patients with newly diagnosed HNC were surveyed at baseline, then every 3 months using the FACT-G7 and FACT H&amp;N (Version 4). A medical assistant (MA) performed a baseline survey around the time of diagnosis and then notified patients electronically every 3 months for two years and then every 6 months until patients completed their 5 years of surveillance. Patients who did not respond to online PRO surveys could complete them via telephone appointments with MAs, or in office pre-rooming. Abnormal findings on PRO surveys were referred to appropriate members of the care team or the treating ENT physicians. Results: 290 patients received baseline surveys. Patients received up to a maximum of 8 subsequent surveys (table). Out of a total of 597 electronic surveys, 585 (97.9%) were completed. The percentage of patient completing each survey ranged from 92-100%. Conclusions: PRO tools can be effectively deployed within the EMR for HNC patients with a high completion rate provided that there is strong engagement from a dedicated member of the care team. This has important implications for designing clinical trials and symptom monitoring in clinical practices that incorporate EMRs.

Impact of cancer on physical and mental activities of daily living in young adult (YA) survivors.

11517 Background: Young Adult (YA) survivors face challenges unique from those survivors of childhood cancer or of middle and older age adults. The potential impact of cancer, its treatment or the lasting effects on daily mental and physical tasks are not fully understood. Methods: Eligibility included diagnosis of malignancy between ages 18-39, 1-5 years from diagnosis and ≥1 year from therapy completion. Participants were randomly selected from tumor registries of 7 academic institutions. Enrolled participants were asked to complete an online patient reported outcomes (PRO) survey related to the effects of cancer and its treatment on daily functionality, finances, and cancer-related distress. All enrolled subjects had diagnostic and treatment information abstracted by a standardized protocol and entered into a database. Results: Enrollment and the online PRO survey was completed by 872 survivors (43.5% of eligible survivors). Survivors were 72.3% female, 90% non-Hispanic or Latino and the most common cancer for males and females were testicular and breast cancer respectively. Cancer, its treatment or the lasting effects limited the kind or amount of instrumental activities of daily living (IADLs) in 649 (76.3%) of survivors. Exposure to chemotherapy was associated with a 3.2-fold increased odds of interference with IADLs (CI 2.08-4.83, p &lt; 0.01) after controlling for diagnosis, age, gender, radiation and surgery exposure. This impairment lasted longer than 1 year in 268 (41.3%) of survivors. Cancer, its treatment or the lasting effects interfered with their ability to perform any mental tasks as part of their IADLs in 454 (53.3%) of survivors. Exposure to chemotherapy was associated with a 2.8-fold increased odds of interference with mental impairment of IADLs (CI 1.94-3.96, p &lt; 0.01) after controlling for diagnosis, age, gender, radiation and surgery exposure. Exposure to radiation was also associated with an increased odds of interference with mental impairment of daily activities (CI 1.05-1.91, p &lt; 0.01) after controlling for diagnosis, age, gender, chemotherapy and surgery exposure. Conclusions: In YA cancer survivors, cancer, its treatment or the lasting effects of treatment commonly impact the daily physical and mental IADLs and many survivors report ongoing limitations &gt; 1 year from therapy completion.