Collection Of Patient-reported Outcomes Research Articles

Remote symptom monitoring with patient-reported outcomes and nudges during lung cancer immunotherapy in China (PRO-NET): protocol for a randomised controlled trial

IntroductionLung cancer is the leading cause of cancer-related mortality globally, with non-small cell lung cancer (NSCLC) comprising the majority of cases. For advanced NSCLC, immunotherapy offers substantial survival benefits but...

Improving Patient Reported Outcome Measure Collection Through Increased Patient Outreaches: Utilization of a Return-to-Work Program.

Patient reported outcome (PRO) measures are a critical tool to understand the patient's experience of their illness. Our institution collects PRO measures for patients receiving spine care through several modalities, including direct patient outreaches. We designed a quality project to increase the total number of patient outreaches within 1 year, without increasing program resources or costs, by utilizing a return-to-work program. Spine PRO collection rate data was extracted from the electronic health record by a data science analyst. This data included how many direct patient outreaches were completed each month and the average number of patient outreaches being completed by the patient outreach representative. For the balancing measure, full-time equivalent costs were monitored throughout the baseline and pilot periods. During the 6-month baseline period, 3477 patient outreaches were made, representing 14.5% of possible outreaches. During the first 3-month post-intervention remeasure period, 6272 patient outreaches were made, an increase of 55.44%. During the second 3-month post-intervention period, 9099 outreaches were made, an increase of 38.21% from the first remeasure. There was no increase in cost to the program related to the project. This increase in patient outreaches by return-to-work employees will translate to improvement in overall PRO collection rates, which may ultimately improve individual and population health care delivery.

P1050 Development of a patient-centred dashboard for routine IBD monitoring

Abstract Background A core outcome set (COS) for patients with inflammatory bowel disease (IBD) was recently developed through a multi-stakeholder consensus in the Health Outcomes Observatory (H2O) project.(1) Recognizing the importance of data visualisation in enhancing disease management, the H2O website now provides a population-level dashboard to track quality and key performance indicators.(2) Patient-level dashboards are designed to support both healthcare providers (HCPs) and patients by facilitating individual health monitoring, encouraging patient-clinician dialogue, and supporting shared decision-making. This work aimed to develop a patient-level dashboard for HCPs to routinely monitor IBD patients, visualising data collected in the H2O IBD COS. Methods In November 2023, a core team comprising three public and four private sector researchers, was formed to coordinate the project. To guide discussions with target users, the lead academic centre developed a static dashboard template including all IBD COS variables. On- and offline feedback was gathered from target users and incorporated iteratively to refine the dashboard until consensus was achieved. Six patients from six countries reviewed the template, identifying unclear or missing outcomes essential for discussion during an outpatient clinic. Results A total of 15 HCPs and 7 academic researchers from 8 countries reviewed dashboard templates, providing feedback over two improvement rounds. In March 2024, consensus among target users was reached on the third version of the static dashboard (Figure). Key improvements included integrating medication tracking with graphical displays of biomarkers and patient-reported outcomes (PROs) related to disease activity, adding pop-up information on hover, and introducing graph customisation options. All consulted patients approved the final dashboard template without requiring additional adjustments. Conclusion We developed a patient-level dashboard for HCPs to support routine monitoring of IBD patients. The static template offers flexibility, allowing implementation teams to add customized information. The template is currently being transformed into a dynamic version for integration into the electronic health records of four academic hospitals. Additionally, the patient-level dashboard is currently being adjusted in close collaboration with patients to integrate into existing PRO collection tools, enabling patients to actively track their health progress themselves, and ensuring that patients and HCPs speak a common language to drive shared decision making. References 1)Fierens L, Carney N, Novacek G, van der Woude CJ, Siegmund B, Casellas F, Borruel N, Huberts AS, Sonnenberg E, Gerold N, Primas C, Hedin CRH, Stamm T, Julsgaard M, Fiorino G, Radice S, Zini MLL, Gross E, Sander C, Arijs I, Vakouftsi VR, Koltai T, Health Outcomes Observatory H O Patient Advisory Board For Inflammatory Bowel Diseases, Health Outcomes Observatory H O Steering Committee, Charlafti I, Ferrante M. A Core Outcome Set for Inflammatory Bowel Diseases: Development and Recommendations for Implementation in Clinical Practice Through an International Multi-stakeholder Consensus Process. J Crohns Colitis. 2024 Oct 15;18(10):1583-1595. doi: 10.1093/ecco-jcc/jjad195. 2)https://health-outcomes-observatory.eu/h2o-insights-center/

Overcoming Barriers to Make Patient-Reported Outcome Collection the Standard of Care in Oncology

This Viewpoint outlines the importance of and known barriers to making patient-reported outcome (PRO) collection the standard of care in oncology.

An Overview of Patient-reported Outcomes for Men with Prostate Cancer: Results from the PIONEER Consortium.

Patient-reported outcome measures (PROMs) are increasingly being used to capture the patients' perspective of their functional status and quality of life (QoL). Big data can help us better understand patient-reported outcomes (PROs). Using prospectively collected data from the Prostate Cancer Diagnosis and Treatment Enhancement Through the Power of Big Data in Europe (PIONEER) consortium, we aimed to describe the functional status and QoL in men with prostate cancer (PCa) treated with active surveillance (AS), radical prostatectomy (RP), and radiotherapy (RT), and to demonstrate the applicability of PROM data on a large scale and at a European level. We identified data sources that collected QoL data using the European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30, EORTC QLQ-PR25, or Expanded Prostate Cancer Index Composite (EPIC)-26/50 questionnaires. Aggregated summary scores for urinary, bowel, and sexual dysfunction, global health status, and QoL were shared for each data source. We identified eight data sources originating from various settings: routine hospital data, embedded research PRO collection, survey data collected by a patient organization, multi-institutional prospective cohort study, and registry data. PRO data were available for 709 men on AS, 20 508 on RP, and 3417 on RT, with a median time between diagnosis and PROM assessment ranging from 1 to 8.7yr. Most men were diagnosed with Gleason ≤7 disease, and T1 or T2 PCa. We observed that sexual dysfunction was the most affected PRO and found large differences between data sources. Our results support the feasibility of PRO assessment using big data in Europe. Implementation of PROMs in clinical practice and the use of standardized methods could improve value-based health care provision. In this study, we combined several data sources that reported urinary, bowel, and sexual dysfunction, global health status, and quality of life. We identified eight data sources and show that sexual function is the most affected domain after treatment.

Enhancing representativeness of patient-reported outcomes in routine radiation oncology care: a quality improvement protocol to address non-response

IntroductionNon-response significantly undermines the representativeness of patient-reported outcome (PRO) data, thereby compromising its utility for facilitating high-value, equitable, patient-centred care in cancer clinics. Quality improvement studies are needed to assess...

A high proportion of patients demonstrate recall bias in the retrospective collection of patient-reported outcomes following hip arthroscopy.

The aim of this study is to assess agreement between retrospectively and prospectively collected patient-reported outcome measures (PROMs) following hip arthroscopy for femoroacetabular impingement syndrome (FAIS). Patients undergoing hip arthroscopy from 2021 to 2023 for FAIS completed preoperative PROMs, including the modified Harris Hip Score (mHHS) and the Non-Arthritic Hip Score (NAHS). Post-operatively, patients were surveyed and asked to recall their preoperative hip function. Paired two-sample t tests were used to compare baseline and recalled baseline PROMs and the difference between scores was compared to previously published minimally clinically important difference (MCID) values. Intraclass correlation coefficients (ICCs) were calculated to test the reliability between scores based on a single-rater, two-way mixed-effects model. Multivariable regression, accounting for age, sex and preoperative baseline scores, was used to evaluate the relationship of time elapsed since surgery with recall accuracy. A total of 116 patients (age: 37.6 ± 11.8 years; 61.2% female) were included. The mean time elapsed for recalled data was 13.1 months (range: 1-27 months). Overall, patients' recalled scores were significantly lower than those prospectively collected (mHHS: 52.9 ± 20.1 vs. 61.5 ± 18.5, p < 0.0001; NAHS: 54.7 ± 20.0 vs. 58.8 ± 19.1, p < 0.0001). Frequency distribution found 68.1% of recalled mHHS and 61.2% of NAHS scores to have a greater difference (between baseline and recalled scores) than the MCID. The ICC was moderate for both mHHS (ICC = 0.559, 95% confidence interval [CI] = [0.420-0.672], p < 0.001) and NAHS (ICC = 0.612, 95% CI = [0.484-0.714], p < 0.001). Multivariate regression analysis did not find time elapsed since surgery to be associated with the difference between baseline and recalled mHHS (n.s.) or NAHS (n.s.). There are significant differences between retrospective and prospectively collected PROMs in patients undergoing hip arthroscopy that are not predicted by time to recall. These findings should impact the interpretation of the existing literature, support the routine collection of prospective data and inform patient counsel regarding their perceived post-operative outcomes. Level IV.

The Oswestry Disability Index in Elective Single level Lumbar Fusion: Is 3 month follow up enough?

BackgroundProspective, longitudinal collection of patient reported outcomes (PRO) has become an essential metric in orthopedics. Despite the utility of PROs, data collection presents a significant challenge to the healthcare system. There is a need to better understand if serial data collection over a 1 to 2 year postoperative period is truly warranted. The purpose of this study was to determine if continued PRO collection after 3 months postop is needed in patients that underwent lumbar fusion. MethodsThis retrospective study utilized 239 patients that underwent an elective single level lumbar fusion between April 1, 2020 and February 1, 2023. Changes in the Oswestry Disability Index (ODI) scores over a one year period were assessed for all patients. Patients were placed into one of three study groups, those that improved by 10 points, those that worsened by 10 points, and those that did not change (score change less than 10 points in either direction). Movement between study groups, the minimal clinical important difference (MCID), and patient satisfaction were analyzed for each patient. ResultsImprovement between preoperative and the 3 month postoperative evaluations was noted for most patients. There was limited change in scores after 3 months. Patients in the improved group continued to improve through 1 year post-operative. Patients in the worsened group continued to decline by 1 year postoperative. Patients in the no change group demonstrated the highest potential to change groups. ConclusionsPatients with substantial improvement or worsening at 3 months have a low likelihood of substantial clinical change thereafter. This subset of patients may not need further evaluation with PROs, but those in the worsened group may benefit from other interventions to potentially alter their course.

Toward facilitating the collection and utilization of patient-reported outcomes in the Military Health System: Lessons learned from a pragmatic clinical trial on physical therapy management for low back pain.

Toward facilitating the collection and utilization of patient-reported outcomes in the Military Health System: Lessons learned from a pragmatic clinical trial on physical therapy management for low back pain.

Use of Patient-Reported Outcomes in Risk Prediction Model Development to Support Cancer Care Delivery: A Scoping Review.

The integration of patient-reported outcomes (PROs) into electronic health records (EHRs) has enabled systematic collection of symptom data to manage post-treatment symptoms. The use and integration of PRO data into routine care are associated with overall treatment success, adherence, and satisfaction. Clinical trials have demonstrated the prognostic value of PROs including physical function and global health status in predicting survival. It is unknown to what extent routinely collected PRO data are used in the development of risk prediction models (RPMs) in oncology care. The objective of the scoping review is to assess how PROs are used to train risk RPMs to predict patient outcomes in oncology care. Using the scoping review methodology outlined in the Joanna Briggs Institute Manual for Evidence Synthesis, we searched four databases (MEDLINE, CINAHL, Embase, and Web of Science) to locate peer-reviewed oncology articles that used PROs as predictors to train models. Study characteristics including settings, clinical outcomes, and model training, testing, validation, and performance data were extracted for analyses. Of the 1,254 studies identified, 18 met inclusion criteria. Most studies performed retrospective analyses of prospectively collected PRO data to build prediction models. Post-treatment survival was the most common outcome predicted. Discriminative performance of models trained using PROs was better than models trained without PROs. Most studies did not report model calibration. Systematic collection of PROs in routine practice provides an opportunity to use patient-reported data to develop RPMs. Model performance improves when PROs are used in combination with other comprehensive data sources.

MyMS: An Interface for Patient-Reported Outcomes for Finnish Individuals With Multiple Sclerosis.

Patient-generated data are a cornerstone of individualized multiple sclerosis (MS) treatment. MyMS, an interface for patient-reported outcomes (PROs) was developed by the Finnish MS Register to enable systematic collection of PROs. MyMS collects data on demographics, lifestyle factors, disease-related factors, and validated questionnaires, including the Quality of Life Questionnaire (15D), the Multiple Sclerosis Impact Scale (MSIS-29), and the Fatigue Severity Scale (FSS). At the end of 2020, the patient-reported Expanded Disability Status Scale (PREDSS), the EuroQOL-5 Dimension (EQ-5D), the Fatigue Scale for Motor and Cognitive Functions (FSMC), and the Multiple Sclerosis Neuropsychological Questionnaire (MSNQ) were added. As of January 1, 2023, 1201 individuals with MS (79% female) have added data to MyMS. Of the validated PRO measures (PROMs), the 15D, MSIS-29, and FSS are the most used. The mean PREDSS score is 3.0 and median disease duration is 6.4 years. According to the existing PROMs, patients report mildly compromised quality of life and problems with fatigue and cognition. The patient interface of the Finnish MS Register consists of data from 17 of 21 counties with well-being services. The interface is used by 10% of Finnish individuals with MS. The addition of the PREDSS, EQ-5D, FSMC, and MSNQ to the interface has increased health care professional and patient interest in the use of PROMs. We suggest that PROs should be integrated into electronic health records to improve shared decision-making and diminish documentation burden.

Increasing Completion of Daily Patient-Reported Outcomes in Psychotherapies for Late-Life Depression through User-Centered Design.

Treatment of depressive symptoms in older adults is a growing public health concern. Collecting patient-reported outcomes (PROs) may facilitate efficiently scaling psychotherapy for older adults but user-specific tailoring is needed to improve completion. This study investigates (1) the effect of updating PRO collection tools for middle-aged and older adults with depressive symptoms through a user-centered design process on user completion of PRO questions, (2) what sociodemographic factors correspond with participant completion, and (3) how completion of PRO questions change during the course of a psychotherapy intervention. Analysis was conducted on 139 middle-aged and older adults with depressive symptoms from three clinical trials at the Weill Cornell ALACRITY Center. Overall response percentages to daily PRO questionnaires were compared before and after the implementation of findings from a multiphase user-centered design process. Grouped least absolute shrinkage and selection operator (LASSO) was employed to examine which baseline factors correspond with patient completion and linear regression was conducted to explore the association. Changes in daily dichotomized completion over time were analyzed with mixed-effect logistic regression. After user-centered updates, there was a significantly higher (p < 0.001) percentage of completion (mean [standard deviation (SD)] percentage, 67.0 [35.6]%) than before (mean [SD] percentage, 24.9 [28.9]%). Additional years of education, age, and total annual household income greater than $25,000 were significant with completion percentage. Mixed-effects logistic regression showed that the odds of high completion increased each day (OR = 1.019 [95% CI: 1.014, 1.023; p < 0.001]). This study has shown that user-centered technology tailoring may be associated with increased PRO completion among middle-aged and older adults with depressive symptoms. PRO-supported psychotherapies are promising for middle-aged and older adults with depressive symptoms. Likewise, this study has demonstrated the potential benefits of employing a rigorous user-centered design process with PRO technology.

Impact of Implant Angulation on Stress Distribution and Survival Rate of Implant-Supported Fixed Dental Prostheses: A Prospective Clinical Study

ABSTRACT Background: Dental implantology has witnessed substantial progress, particularly with the widespread use of implant-supported fixed dental prostheses (FDPs) for edentulous patients. Methods: In this prospective clinical study involving 146 adult participants requiring implant-supported FDPs, a randomized allocation placed individuals into either the control group (ideal angulation) or the experimental group (deviated angulation). A comprehensive investigation unfolded through finite element analysis (FEA), examining biomechanical aspects. This was complemented by thorough clinical assessments and the collection of patient-reported outcomes. Results: The application of FEA unveiled a statistically significant increase in the maximum von Mises stress within the experimental group (P = 0.032), suggesting a tangible impact of deviated angulation on stress distribution. However, when transitioning to the clinical realm, the evaluation of implant stability, osseointegration, and prosthetic fit demonstrated no significant differences between the control and experimental groups. Patient-reported outcomes demonstrated no statistically significant differences in comfort, aesthetic satisfaction, or overall satisfaction between the control and experimental groups (all P &gt; 0.05). Conclusion: While finite element analysis identified biomechanical differences associated with deviated implant angulation, these distinctions did not consistently translate into clinically significant variations.

Stakeholder Perspectives on the Meaningful Integration of Clinical Informatics Interventions Using Patient-Reported Outcomes in Healthcare

Abstract Background Patient-reported outcomes (PROs) capture where patients are on their disease trajectory and can identify changes in health status from their perspective. Objectives This study applied the equity and sustainability-informed RE-AIM framework (Reach, Effectiveness, Adoption, Implementation, and Maintenance) to gain insights into clinical informatics interventions for collection and use of PROs across health systems. Methods A total of 14 health informatics and clinical professionals were interviewed about the development and use of PROs within their health systems and individual practices. Directed content analysis was performed to highlight patterns, similarities, and differences in stakeholder perspectives across RE-AIM domains. Results The reach of clinical informatics interventions using PROs varied across clinical practices and settings based upon institutional commitment and support, integration of clinical information systems, and engagement with patients and families. Although interventions using PROs were viewed as effective for enabling focused conversations with patients and facilitating shared decision-making, barriers to adoption included licensing requirements associated with PRO instruments, lack of incentives for their use, limited integration of PRO results into electronic medical record systems, and poor support for patients with low technology and/or health literacy. Implementation of interventions using PROs was facilitated through training and support staff who aided clinicians with clinical workflow integration, availability of questionnaires in multiple languages, identifying thresholds and strategies for action, and presenting interpretable visualizations showing changes over time alongside significant clinical events. Maintenance of interventions using PROs was enabled through multimodal data collection approaches and data governance groups that evaluated organizational requests to track new measures. Conclusion Initiatives to increase the reach of clinical informatics interventions using PROs will require health system investments into medical record system integration, education, and implementation support for clinicians and patients, and efforts to reach patient populations with language barriers or limited technology literacy.

Patient-centered clinical decision support challenges and opportunities identified from workflow execution models.

To use workflow execution models to highlight new considerations for patient-centered clinical decision support policies (PC CDS), processes, procedures, technology, and expertise required to support new workflows. To generate and refine models, we used (1) targeted literature reviews; (2) key informant interviews with 6 external PC CDS experts; (3) model refinement based on authors' experience; and (4) validation of the models by a 26-member steering committee. We identified 7 major issues that provide significant challenges and opportunities for healthcare systems, researchers, administrators, and health IT and app developers. Overcoming these challenges presents opportunities for new or modified policies, processes, procedures, technology, and expertise to: (1) Ensure patient-generated health data (PGHD), including patient-reported outcomes (PROs), are documented, reviewed, and managed by appropriately trained clinicians, between visits and after regular working hours. (2) Educate patients to use connected medical devices and handle technical issues. (3) Facilitate collection and incorporation of PGHD, PROs, patient preferences, and social determinants of health into existing electronic health records. (4) Troubleshoot erroneous data received from devices. (5) Develop dashboards to display longitudinal patient-reported data. (6) Provide reimbursement to support new models of care. (7) Support patient engagement with remote devices. Several new policies, processes, technologies, and expertise are required to ensure safe and effective implementation and use of PC CDS. As we gain more experience implementing and working with PC CDS, we should be able to begin realizing the long-term positive impact on patient health that the patient-centered movement in healthcare promises.

Patient engagement in hematopoietic stem cell transplantation and cell therapy: a survey by the EBMT patient engagement task force & transplantation complications working party.

The EBMT (European Blood and Marrow Transplantation Society) aims to connect patients, the scientific community, and other stakeholders to improve hematopoietic stem cell transplantation and cellular therapy outcomes. We performed a cross-sectional online survey to understand the perceptions regarding Patient Reported Outcomes (PROs) and Patient Active Involvement in Research (PAIR) in over 800 stakeholders (n = 813). Patients (n = 278) and health care professionals (HCPs) (n = 351) were compared. We observed high openness for EBMT PRO collection (n = 680, 84.5% across stakeholders' groups; patients n = 256, 93.1% versus HCPs n = 273, 78.4% [p < 0.001]) and PAIR (n = 702, 87.3% across stakeholder groups; patients n = 256, 92.4% versus HCPs n = 296, 85.8% [p = 0.009]), with a significantly higher proportion of patients expressing interest compared to HCPs. Priority domains for PROs data-collection identified were the assessment of symptom experience, psychosocial and cognitive functioning. The most important issues for patients specifically were the data-collection of PROs reflecting cognitive function, the option of reporting data at home, the importance of identifying actionable targets to improve their recovery, and receiving feedback on their input when participating in research projects. Our multistakeholder approach suggests an added value to embracing patient engagement in the development of meaningful research and service design within the transplantation and cellular therapy community.

International trends in shoulder replacement: a meta-analysis from 11 public joint registers.

International variation exists in the types of shoulder replacement used for treatment of specific diseases. Implant choice continues to evolve without high-quality evidence. Our aim was to evaluate trends in incidence rates of shoulder replacement and assess any recent changes in practice between countries by using registry data. Patient characteristics, indication and year of surgery, type of replacement, and collection methods of patient-reported outcomes (PROMs) was extracted from 11 public joint registries. Meta-analyses examined use of reverse total shoulder replacement (RTSR) for osteoarthritis, cuff tear arthropathy, and acute fracture; use of anatomical total shoulder replacement (TSR) for osteoarthritis; and use of humeral hemiarthroplasty for fracture. The annual growth rate of shoulder replacements performed is 6-15% (2011-2019). The use of RTSR has almost doubled (93%). RTSR is now universally performed for cuff tear arthropathy (97.3%, 95% confidence interval [CI] 96.0-98.1). Its use for avascular necrosis, trauma, and inflammatory arthropathy is increasing. The use of RTSR was similar (43.1%, CI 30.0-57.2) versus TSR (44.7%, CI 31.1-59.1) for osteoarthritis. The types of PROMs used, collection time points, and response rates lack standardization. COVID-19 had a varying inter-registry impact on incidence rates. The incidence of shoulder replacements has grown. Use of RTSR has increased for all disease indications despite limited high-quality evidence driving this change in indications outside of cuff arthropathy. Consequently, less variation is observed in international practice. Existing differences now relate to use of newer implant types and methodology of PROMs collection, which prevents international comparison and outcome analysis.

Patient-Reported Outcomes in Phase 3 Clinical Trials for Blood Cancers: A Systematic Review

Published research suggests that patient-reported outcomes (PROs) are neither commonly collected nor reported in randomized clinical trials (RCTs) for solid tumors. Little is known about these practices in RCTs for hematological malignant neoplasms. To evaluate the prevalence of PROs as prespecified end points in RCTs of hematological malignant neoplasms, and to assess reporting of PROs in associated trial publications. All issues of 8 journals known for publishing high-impact RCTs (NEJM, Lancet, Lancet Hematology, Lancet Oncology, Journal of Clinical Oncology, Blood, JAMA, and JAMA Oncology) between January 1, 2018, and December 13, 2022, were searched for primary publications of therapeutic phase 3 trials for adults with hematological malignant neoplasms. Studies that evaluated pretransplant conditioning regimens, graft-vs-host disease treatment, or radiotherapy as experimental treatment were excluded. Data regarding trial characteristics and PROs were extracted from manuscripts and trial protocols. Univariable analyses assessed associations between trial characteristics and PRO collection or reporting. Ninety RCTs were eligible for analysis. PROs were an end point in 66 (73%) trials: in 1 trial (1%) as a primary end point, in 50 (56%) as a secondary end point, and in 15 (17%) as an exploratory end point. PRO data were reported in 26 of 66 primary publications (39%): outcomes were unchanged in 18 and improved in 8, with none reporting worse PROs with experimental treatment. Trials sponsored by for-profit entities were more likely to include PROs as an end point (49 of 55 [89%] vs 17 of 35 [49%]; P < .001) but were not significantly more likely to report PRO data (20 of 49 [41%] vs 6 of 17 [35%]; P = .69). Compared with trials involving lymphoma (18 of 29 [62%]) or leukemia or myelodysplastic syndrome (18 of 28 [64%]), those involving plasma cell disorders or multiple myeloma (27 of 30 [90%]) or myeloproliferative neoplasms (3 of 3 [100%]) were more likely to include PROs as an end point (P = .03). Similarly, compared with trials involving lymphoma (3 of 18 [17%]) or leukemia or myelodysplastic syndrome (5 of 18 [28%]), those involving plasma cell disorders or multiple myeloma (16 of 27 [59%]) or myeloproliferative neoplasms (2 of 3 [67%]) were more likely to report PROs in the primary publication (P = .01). In this systematic review, almost 3 of every 4 therapeutic RCTs for blood cancers collected PRO data; however, only 1 RCT included PROs as a primary end point. Moreover, most did not report resulting PRO data in the primary publication and when reported, PROs were either better or unchanged, raising concern for publication bias. This analysis suggests a critical gap in dissemination of data on the lived experiences of patients enrolled in RCTs for hematological malignant neoplasms.

MSK Engage: A comprehensive program for collection of patient reported outcomes at scale.

e13666 Background: Collecting patient-reported outcomes (PROs) yields high value data for clinical care and research and is associated with improved survival in oncology. However, collection of PROs at scale requires a multifaceted program that in most institutions is not available. This study describes a comprehensive PRO program to support research and standard clinical care at scale. Methods: Memorial Sloan Kettering Cancer Center is a high-volume cancer center in New York. Beginning in 2016, we developed a comprehensive PRO program, MSK Engage, with the following capabilities: (i) A governance process to oversee the creation of questionnaires for both research and clinical care. This process includes alignment with the IRB and standardization of questions presented to patients. (ii) Technology to determine which patients should get an assessment (i.e., instance of a questionnaire) at what point in time. (iii) An interactive module in our patient portal that would present the assessment to the patient. (iv) Various methods to present the PRO data to providers to support clinical decision making. These methods included (a) alerts based on threshold logic that would send secure messages to the clinical team, (b) integration of the PRO data into the EHR to be viewed with other clinical data, and (c) the creation of specialized workflows. The results of assessments are also stored in an analytics database to support operations, research, and the creation of smart reports that compare a patient’s responses with other similar patients. Results: We analyzed the use of the program from 2016 to 2023 (Table). 189 questionnaires have been implemented, generating 3,098,574 completed assessments from 314,685 distinct patients. Completion rates varied. The median completion time for all questionnaires was 2 minutes. Examples of programs enabled by patient-reported outcomes include post-operative symptom monitoring, family history questionnaires to support clinical genetics decision making and operations, intake and follow up questionnaires that support clinical documentation and quality of life questionnaires to support goal concordant care. Conclusions: Adoption of PROs at scale for both research and standard of care in oncology is feasible and innovative programs of care can be supported while maintaining a centralized and acceptable patient experience. This model can be applied across institutions seeking to develop or scale PRO programs to assure patient data are meaningfully incorporated into decision-making. [Table: see text]

Randomized, placebo-controlled trial of atorvastatin to prevent hearing loss in patients with head and neck squamous cell carcinoma receiving cisplatin based chemoradiation (CRT).

TPS12153 Background: Cisplatin chemotherapy is associated with several adverse effects, including a permanent, high-frequency, sensorineural hearing loss in about 50% of patients. Preclinical studies suggest that statin drugs can mitigate cisplatin ototoxicity (Fernandez et al., Hearing Research 2020). In our prior combined retrospective/prospective dataset of patients with head and neck squamous cell carcinoma (HNSCC) treated with cisplatin-based CRT, the incidence of CTCAE grade ≥ 2 hearing loss was 29.4% in statin non-users, versus 9.7% in atorvastatin users (Fernandez et al., J Clin Invest 2021). An adjusted odds ratio analysis indicated that atorvastatin users are significantly less likely to develop hearing loss versus statin non-users (odds ratio 0.47), providing the rationale for a randomized, controlled, prospective study. Methods: This multi-center, randomized, placebo-controlled study is enrolling patients with previously-untreated, locally advanced head and neck squamous cell carcinoma (HNSCC) for which cisplatin-based chemoradiation is indicated, either in the definitive or adjuvant setting. Patients who will be treated with cisplatin (at 75-100 mg/m2 bolus every three weeks or 40 mg/m2 weekly) are eligible. Patients currently taking a statin or those without measurable hearing in at least one ear are excluded. A baseline audiogram is performed, and patients are randomized to atorvastatin (40 mg/day) or placebo, which is started 2-7 days before CRT and taken daily until 3 months after completion of CRT. Post-treatment audiometry is repeated at 3 months and 2 years after CRT, along with collection of patient-reported outcomes on hearing, tinnitus, and peripheral neuropathy. Patients are monitored for liver and muscle toxicity, with dose de-escalation or cessation of atorvastatin as needed. The primary endpoint is the incidence of CTCAE grade ≥2 hearing loss at 3 months after CRT. Secondary endpoints include other treatment related grade 3-4 adverse events, overall survival, and disease-free survival. Up to 214 subjects will be enrolled at 4 U.S. sites (NIH Clinical Center, Emory University, University of Maryland, University of Rochester). Funding: NIH/NIDCD, U01 DC020452 and intramural project ZIA DC000079. Clinical trial information: NCT04915183 .