Patient-reported Outcomes Data Research Articles

Impact of tislelizumab + chemotherapy versus placebo + chemotherapy on patient-reported symptoms and disease progression by programmed death-ligand 1 expression in gastroesophageal adenocarcinoma: A post hoc analysis of the RATIONALE-305 trial.

365 Background: The relationship between patient-reported outcome (PRO)-based symptom scores, recurrent PRO-based symptomatic deterioration events (RDEs), and terminal events such as progression-free survival (PFS) are rarely examined in the oncology therapeutic domain. Thus, we applied a 3-component joint model (JM) framework aiming to illuminate more clinically interpretable associations between PRO-based treatment effects, RDEs, and PFS among subgroups of patients with programmed death-ligand 1 (PD-L1) expression of ≥1%, ≥5%, and ≥10%. Methods: The final analytic sample included 378 patients in the tislelizumab + chemotherapy arm (T+C) vs 401 in the placebo + chemotherapy arm (P+C) for the PD-L1 ≥1% subgroup, 238 in the T+C arm vs 237 in the P+C for the PD-L1 ≥5% subgroup, and 118 in the T+C arm vs 125 in the P+C arm for the PD-L1 ≥10% subgroup. Symptom domain scores from the EORTC QLQ-C30 and QLQ-STO22 symptom were modeled. Change from baseline (CFBL) in each domain was analyzed every cycle up to cycle 6, then every other cycle thereafter (up to cycle 25). The joint model was applied to all PD-L1 subgroups and comprised three components: 1) linear mixed model (LMM) predicting CFBL symptom scores, 2) Cox proportional hazard (CPH) model for disease progression (PFS as terminal event), and 3) frailty (random effects for RDEs) CPH model for time to PRO-based RDEs. Osoba’s 10-point threshold was used to define RDEs. Results: In the LMM, significant treatment efficacy for the T+C arm compared with the P+C arm was observed for the GHS/QoL ( P =0.0080) and dietary restriction scores ( P =0.0475) in the PD-L1 ≥5% subgroup. In the PD-L1 ≥1% subgroup, compared with the P+C arm, the T+C arm was associated with less worsening in pain/discomfort ( P =0.0376), upper gastrointestinal symptoms ( P =0.0088), and dietary restrictions ( P =0.0352). In the CPH model, the average hazard ratio indicated that compared with the P+C arm, the T+C arm was associated with a reduction in risk of disease progression across all PRO domains and PD-L1 subgroups. Lastly, the PRO-based RDE frailty predictions were strongly associated with PFS risk in the PD-L1 ≥1% and ≥5% subgroups for GHS/QoL, and in the PD-L1 ≥1% subgroup for physical functioning, fatigue, dysphagia-odynophagia, pain/discomfort, and dietary restrictions, as well as for pain/discomfort and dietary restrictions in the PD-L1 ≥5% subgroup. Conclusions: Through the 3-component JM, PRO-based effects were detected and demonstrated strong associations between PRO-based RDEs, and investigator-assessed PFS among varying PD-L1 expression levels. This framework may be a promising alternative for analyzing and interpreting PRO data in the context of PFS.

P0420 Interim analysis of real-world-data on PROs and biomarker remission collected through the Health Outcome Observatory (H2O) consortium in IBD patients

Abstract Background The Health Outcome Observatory (H2O) project aims to standardize and facilitate the collection of patient reported outcomes (PROs) and clinical outcomes by Core Outcome Sets (COS) in patients with inflammatory bowel disease (IBD).1 The present study describes preliminary data on clinical and biomarker remission as well as quality of life. Methods This is a real-world interim analysis of IBD patients included in the H2O project at the Medical University of Vienna, Austria, and the Hospital Universitari Vall d’Hebron, Barcelona, Spain. The variables analyzed were part of the COS. Clinical remission was defined by PRO-2 (for Crohn’s disease (CD): number of liquid or very soft stools ≤3, abdominal pain score ≤1; for ulcerative colitis (UC): rectal bleeding score=0, stool frequency score ≤1), biomarker remission by faecal calprotectin (<150g/g) and C-reactive protein (<0.5mg/dl), and quality of life by PROMIS_10_Q02 (quality of life answered as very good or excellent). Co-primary endpoints were clinical and biomarker remission and a very good or excellent quality of life. A descriptive analysis was performed. Results 532 patients were included in Austria (221 female [42%]; 298 CD, 178 UC, 6 IBDU, 50 with missing diagnosis) and 30 in Spain (14 female [47%]; 18 CD, 12 UC). 138 of 532 patients in Austria (26%) did not provide PRO data. The majority of patients were on advanced therapies (CD: 221/298; 77%; UC: 118/178; 66%). Among CD patients 175 were in PRO-2 remission in Austria (80%) and 13 in Spain (72%), the corresponding numbers of patients in UC PRO-2 remission were 86 in Austria (62%) and 11 in Spain (92%). Biomarker remission was observed in 128/240 CD patients (53%) and 84/135 UC patients (62%). Detailed results of the PRO-2 and of the biomarker values of the Austrian IBD patients are given in Table 1. Very good or excellent quality of life was reported by 97/224 (43.3%) CD patients and 69/140 (49.2%) UC patients. 77/240 CD patients (32%) and 58/135 UC patients (43%) were in clinical PRO-2 as well as biomarker remission and had a very good or excellent quality or life. Conclusion While a large number of IBD patients in tertiary university centers were in clinical PRO-2 remission a lower number of patients had a very good or excellent quality of life. Biomarker remission was achieved almost equally often as clinical PRO-2 remission in UC patients but less often in CD patients. About one third of the patients were in clinical PRO-2 and biomarker remission and had a very good or excellent quality of life. Despite the frequent use of advanced therapies there is a need for treatment optimizing to reach all treatment goals.

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.

Clinical Outcomes for Workers' Compensation versus Commercially Insured Patients Following Ambulatory Lumbar Decompression: A Cohort Matched Analysis with Two-Year Follow-Up.

To evaluate outcomes for workers' compensation (WC) versus commercially insured (CI) patients undergoing lumbar decompression (LD) at an ambulatory surgical center (ASC). This is a retrospective cohort study utilizing propensity score matched groups. Patients undergoing elective LD at an ASC with two-year follow-up were identified and grouped based on insurance type (WC or CI). Other demographic, diagnostic, and patient-reported outcome (PRO) data were obtained as well. PROs included Patient-Reported Outcome Measurement Information System-Physical Function (PROMIS-PF), Visual Analog Scale-Back (VAS-B), VAS-Leg, Oswestry Disability Index (ODI), 12-Item Veterans Rand Physical Composite Score (VR-12 PCS), VR-12 Mental Composite Score (VR-12 MCS) and 9-Item Patient Health Questionnaire (PHQ-9). PRO scores were obtained preoperatively and up to two years postoperatively. Rates of minimum clinically important difference (MCID) were obtained. Multivariate regression was utilized to compare postoperative PROs. Following propensity score matching, 153 patients remained in the CI group and 71 were in the WC group. At baseline through one year postoperatively, WC patients reported worse ODI, PHQ-9, PROMIS-PF, VR-12 PCS, and VR-12 MCS (p≤0.041, all). At two years, WC patients reported similar outcomes as CI patients. WC patients reported significantly greater MCID achievement for PHQ-9 (73.1% versus 37.7% for CI patients, p=0.008). WC patients report inferior pre- and postoperative outcomes in disability, mental health, and physical function compared to CI patients. However, WC patients were more likely to report clinically significant improvements in depressive burden. Surgeons can counsel WC patients on expected mental health improvements and recommend psychosocial and physical rehabilitation.

Concurrent preoperative eribulin and radiation for resectable retroperitoneal liposarcoma: a phase 1B study.

Management of retroperitoneal liposarcoma (RPLPS) is challenging and recurrence rates remain high despite aggressive surgical resections. Preoperative radiation alone lacks definitive benefit, thus we sought to evaluate combined chemoradiotherapy with the potential to enhance local efficacy of radiation as well as control micrometastatic disease. We assessed the safety and tolerability of preoperative eribulin, a cytotoxic microtubule inhibitor approved for the treatment of advanced liposarcoma, in combination with radiation in patients with RPLPS. In this open-label dose-finding study, patients with primary or recurrent resectable RPLPS received preoperative intensity-modulated radiation therapy (IMRT) with escalating doses of eribulin. Eribulin was administered for three 21-day cycles at a starting dose of 1.1 mg/m2. Concurrent radiation to 50.4 Gy began during cycle 1. Surgical resection occurred 3-10 weeks after completion of chemoradiation. The primary endpoint was determination of the recommended phase 2 doses (RP2D) of concurrent eribulin and radiation. Between 2018-2023, fifteen patients were enrolled. Thirteen patients were evaluable for dose-determination. Four patients treated at starting dose level had no dose-limiting toxicities (DLTs). Two of nine patients treated with escalated eribulin dose had DLTs. The RP2D was established as eribulin 1.4 mg/m2 and IMRT 50.4 Gy. Eleven patients were evaluable for secondary efficacy endpoints. The median recurrence-free survival was 30.4 months (95% CI 12.0-NR) and the median overall survival was 54.1 months (95% CI 9.5-NR). Patient reported outcome data did not show any significant changes over the study period. A preoperative chemoradiation protocol of eribulin in combination with IMRT showed a manageable safety profile and warrants additional prospective evaluation for treatment of resectable RPLPS.

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...

Improving Diversity in a Novel Psoriasis Study

Diverse racial and ethnic representation in clinical trials has been limited, not representative of the US population, and the subject of pending US Food and Drug Administration guidance. Psoriasis presentation and disease burden can vary by skin pigmentation, race and ethnicity, and socioeconomic differences. Overall, there are limited primary data on clinical response, genetics, and quality of life in populations with psoriasis and skin of color (SoC). The Varying Skin Tones in Body and Scalp Psoriasis: Guselkumab Efficacy and Safety trial (VISIBLE) is underway and uses strategies aimed at addressing this persistent gap. To assess the innovative strategies used in the VISIBLE trial to recruit and retain diverse participants in a randomized clinical trial of psoriasis in participants with SoC. This was an ad hoc quality improvement assessment of participant recruitment and retention approaches used by the VISIBLE trial. VISIBLE enrolled and randomized 211 participants (mean [SD] age, 43 [13] years; 75 females [36%] and 136 males [64%]) with SoC and moderate to severe plaque psoriasis from August 2022 to March 2023 to evaluate guselkumab treatment. The self-identified race and ethnicity of the participants was: 1 American Indian/Alaska Native (0.5%), 63 Asian (29.9%), 24 Black (11.4%), 94 Hispanic/Latino (44.5%), 13 Middle Eastern (6.2%), 1 Pacific Islander/Native Hawaiian (0.5%), 12 multiracial (5.7%), and 3 of other race and/or ethnicity (1.4%). Using a combination of objective (colorimetry to determine Fitzpatrick skin type) and self-reported (race and ethnicity consistent with SoC) parameters, VISIBLE sought to broaden inclusion of participants from various backgrounds. Observed improvements were that participant enrollment occurred approximately 7 times faster than anticipated (vs historical recruitment data for psoriasis studies); 211 participants (100%) self-identified themselves as a race or ethnicity other than White; and more than 50% had skin tone in the darker half of the Fitzpatrick skin type spectrum (type IV-VI). Innovations implemented by VISIBLE were (1) assessment of the natural history of postinflammatory pigment alteration and improvements over time using combined objective colorimetry and clinician- and patient-reported outcomes; (2) evaluation of genetic and comorbidity biomarkers relevant to participants with SoC; (3) a diverse demographic-driven approach to site selection (emphasizing investigator and staff diversity and experience with populations with SoC); (4) provision of cultural competency training to enhance participant enrollment and retention; (5) collection of patient-reported outcomes data in participants' primary language; and (6) periodic, blinded central review and feedback on investigator efficacy scoring to promote consistency and accuracy in evaluating psoriasis in participants with SoC. VISIBLE is a unique study focused on addressing important knowledge and data gaps in populations of patients with psoriasis and SoC, with the goal of generating data to help improve clinical care and inform future best practices in diversity within dermatology research. The rapid study enrollment demonstrates that intentional and strategic approaches to clinical trial design and conduct can speed recruitment and bolster participation and retention of diverse populations in a dermatologic setting. ClinicalTrials.gov Identifier: NCT05272150.

Patient-reported outcomes after CAR T-cell therapy in patients with hematological malignancies.

The remarkable improvement in survival among individuals with hematological malignancies receiving chimeric antigen receptor (CAR) T-cell therapy has highlighted the growing unmet need to incorporate patient-centered assessments in management guidelines for these patients. That CAR T-cell therapy is associated with unique toxicities and relatively high symptom burden in the first few weeks after cell infusion is well known. Magnifying the patient's voice by using patient-reported outcomes (PROs) might support personalized intervention in the acute-care setting, optimize the use of medical resources, improve satisfaction with therapy, and enhance survival benefit. However, various factors impede PRO use in routine patient care: (1) the feasibility of PRO assessment during the acute phase of treatment, especially in patients experiencing neurological toxicities, is not well established; (2) although PROs are widely used in drug- development trials, the assessment tools used in clinical trials primarily inform quality-of-life or safety comparisons among study arms and are rarely the proper tools for assessing and capturing clinically meaningful adverse events that should be monitored in routine patient care; (3) PRO data that could guide how best to monitor and capture the delayed effects of CAR T-cell therapy in long-term survivors are limited. There is a pressing need to overcome these barriers to integrating evidence-based PROs into standard-of-care guidelines for patients receiving CAR T-cell therapy. In this review, we present the current state of PRO utilization in CAR T-cell therapy. We also discuss practical approaches and future directions for successful implementation of PROs in the care of patients receiving CAR T-cell therapy.

Routine review of patient-reported outcome data influences radiotherapy care: IMPROVE study results.

Radiation oncologists closely monitor patients during weekly on-treatment visits (OTVs). This study examines whether routine patient-reported outcome measures (PROMs) during OTVs change physicians' perceptions of treatment-toxicity and inform symptom-management. IMPROVE is a single-arm prospective multicenter trial, conducted from 2020 to 2023. Patients with locally-advanced or oligometastatic thoracic or gastrointestinal cancers receiving definitive-intent radiation, with or without chemotherapy, and their physicians enrolled. Patients completed a 14-question disease-specific PROM in-clinic prior to OTVs. Physicians rated their patient's global toxicity-burden based on clinical data/assessments, then re-rated their patient's toxicity-burden and reported management-changes after PROM review. At radiotherapy end, physicians completed a Feedback Form. PROMs and outcome-data collection used electronic or paper forms. We report any change in physician-assessed burden-score and symptom-management due to PROMs. The 100 patients enrolled (49 academic, 51 community-based) were 70 years old (median), 51 % female, 81 % Caucasian, 95 % ECOG 0-1, and 94 % received concurrent chemotherapy. The median radiation dose was 60 Gy, delivered over 6 weeks. PROMs were available for review for 607/629 (97 %) OTVs: full 433/629 (69 %), partial 174/629 (28 %). For 75/100 patients (75 %; 95 % CI:65 %-83 %), PROM review resulted in any change in physician-reported burden-score, and for 50/100 patients (50 %; 95 % CI:40 %-60 %) any change in patients' on-treatment management. Rates of burden-score and management-changes were similar between academic and community-based practices (78 % vs. 73 %; 53 % vs. 47 %, respectively). For 78/100 patients with Feedback Forms, physicians agreed/strongly agreed that PROMs improved patients' quality-of-care (91 %). PROM review changes radiation oncologists' on-treatment toxicity assessment in 75 % and care delivery in 50 % of their patients.

A normalized quality of life approach for regulatory approval of medical devices

Abstract Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of treatment on patients’ symptoms, function and quality of life. High-quality PRO data from trials can inform shared decisionmaking, regulatory and economic analyses and health policy. The heterogeneity between the patient groups and the associated differences in morbidity and quality of life can be taken into account by standardization to the general population. As a result, our study shows that despite the retrospective design with the corresponding level of evidence, mesh repair of a ventral hernia leads to an equivalent result compared to tissue hernia surgery.

«Declared quality of life»: new perspectives for assessing patients’ quality of life in clinical medicine

In modern patient-oriented medical care, quality of life is an important criterion for a comprehensive assessment of the patient's health and evaluation of treatment effectiveness. This article is devoted to the current methodological issues of assessing quality of life and other patient-reported outcomes (PRO) and attempts to reveal the difficulties of interpreting PRO data obtained in real-world clinical practice. A new concept for identifying significant changes in the patient's general health condition in clinical practice is presented, and a new model of quality of life outcomes before and after treatment is considered. The concepts of “declared quality of life”, “index of achieved quality of life” and “declared clinical effect” have been declared. Assessment of the declared quality of life of the patient and determination of the index of the achieved quality of life can be used for practical purposes to implement the principle of patient-oriented care in clinical practice.

Patients and Spine Surgeons' Perspectives regarding Decision-Making and Outcomes in Lumbar Surgery: An Exploratory, Qualitative Study.

Decision-making in lumbar surgery for degenerative conditions is influenced by various factors, including patient expectations and empowerment. The role of patient-reported outcomes (PROs) in guiding these decisions is underexplored. This study aims to understand the perspectives of patients and spine surgeons in decision-making for lumbar surgery and explore their perspectives on the relevance and influence of pre- and post-surgery PROs. An exploratory qualitative study was conducted between February 2022 and November 2023. A total of 15 patients with degenerative lumbar conditions and 9 spine surgeons from 5 tertiary public hospitals in Singapore were recruited. Data were analyzed using a framework analysis approach, ensuring validity through member checking, reflexive journals, and data source triangulation. Three overarching categories emerged: "expectations and outcomes," "decision empowerment," and "surgical experiences." There is a shift toward shared decision-making, highlighting the importance of patient-centric approaches. Surgical decisions are primarily influenced by PROs, particularly health-related quality of life (HRQoL) and pain relief. Patients prioritize pain relief and improvements in daily functioning, while surgeons also emphasize avoiding postoperative complications. Decision support tools, including PRO data, are essential but need better accessibility and integration within clinical settings. Positive surgical experiences are driven by clear communication, trust with surgeons, swift recovery, and no regrets post-surgery. Our study emphasizes the importance of patient-centered approaches in lumbar surgery decision-making, particularly regarding pre- and post-surgery PROs. Implementing these approaches may enhance patient satisfaction and surgical outcomes, urging the surgical community to prioritize informed, empathetic decision-making to improve healthcare quality.

Integrating Clinical Data and Patient-Reported Outcomes for Analyzing Gender Differences and Progression in Multiple Sclerosis Using Machine Learning.

Multiple sclerosis (MS) is a complex neurodegenerative disease with a variable prognosis that complicates effective management and treatment. This study leverages machine learning (ML) to enhance the understanding of disease progression and uncover gender-based differences in MS by analyzing clinical data integrated with patient-reported outcomes (PROMs). We conducted a prospective cohort study involving 250 MS patients at a secondary care hospital in Spain over an 18-month period. Using REDCap for data management, we collected comprehensive demographic, clinical, and PROMs data. Our analysis utilized Decision Trees, Random Forest, and Support Vector Machine algorithms to classify patients based on disease evolution and infer Expanded Disability Status Scale (EDSS) levels. Additionally, we employed propensity score matching to analyze gender differences, focusing on clinical outcomes and quality of life measures. The results could indicate that integrating diverse data sets through ML would significantly improve the diagnostic accuracy and serve as a support for clinician's decision making. Our models achieved high accuracy in classifying MS types and predicting disability levels, demonstrating the potential of ML in personalized treatment planning. Furthermore, our findings suggest notable gender differences in disease progression and response to treatment. These insights advocate for a gender-specific approach in MS management and highlight the importance of personalized medicine. This study underscores the transformative potential of ML in enhancing the understanding and management of MS through integrated data analysis.

Does Discussing Patient-reported Outcome Measures Increase Pain Self-efficacy at an Orthopaedic Visit? A Prospective, Sequential, Comparative Series.

Pain self-efficacy, or the ability to carry out desired activities in the presence of pain, can affect a patient's ability to function before and after orthopaedic surgery. Previous studies suggest that shared decision-making practices such as discussing patient-reported outcome measures (PROMs) can activate patients and improve their pain self-efficacy. However, the ability of PROMs to influence pain self-efficacy in patients who have undergone orthopaedic surgery has yet to be investigated. (1) Is immediately discussing the results of a PROM associated with an increase pain self-efficacy in new patients presenting to the orthopaedic surgery clinic? (2) Is there a correlation between patient resilience or patient involvement in decision-making in changes in pain self-efficacy? This was a prospective, sequential, comparative series completed between February to October 2023 at a single large tertiary referral center at a multispecialty orthopaedic clinic. Orthopaedic subspecialties included total joint arthroplasty, spine, hand, sports, and trauma. The first 64 patients underwent standard care, and the following 64 had a conversation with their orthopaedic surgeon about their PROMs during the initial intake visit. We collected scores from the Pain Self-Efficacy Questionnaire (PSEQ), Brief Resilience Scale (BRS), and Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function form (PF-SF10a) and data on demographic characteristics before the visit. The PSEQ is a validated PROM used to measure pain self-efficacy, while the BRS measures the ability of patients to recover from stress, and the PROMIS PF-SF10a is used to assess overall physical function. PROMs have been utilized frequently for their ability to report the real-time physical and psychological well-being of patients. In the standard care group, the PROMIS PF-SF10a score was not discussed with the patient. In the PROMs group, the physician discussed the PROMIS PF-SF10a score using a script that gave context to the patient's score. Additional conversation about the patient's score was permitted but not required for all patients. Scores from the Observing Patient Involvement in Decision Making (OPTION-5) instrument were recorded during the visit as a measure of patient involvement in clinical decision-making. After the visit, both groups completed the PSEQ. The primary outcome was change in the PSEQ. Change in pain self-efficacy was recorded as greater or less than the minimum clinically important difference, previously defined at 8.5 points for the PSEQ [10]. The secondary outcomes were correlation between PSEQ change and the BRS or OPTION-5. Between the PROMs and standard care groups, there was no difference in the change in PSEQ scores from before the visit to after (mean ± SD change in control 4 ± 10 versus change in PROMs group 3 ± 7, mean difference 1 [95% confidence interval (CI) -2.0 to 4.0]; p = 0.29). Fifty-six percent (36 of 64) of patients in the standard care group demonstrated an increase in pain self-efficacy (of whom 22% [14 of 64] had clinically important improvements), and 59% (38 of 64) of patients in the PROMs group demonstrated an increase in pain self-efficacy (of whom 19% [12 of 64] had clinically important improvements). In the control group, there was no correlation between the change in PSEQ score and resiliency (BRS score r = -0.13 [95% CI -0.36 to 0.12]; p = 0.30) or patient involvement in decision-making (OPTION-5 r = 0.003 [95% CI -0.24 to 0.25]; p = 0.98). Similarly, in the PROMs group, there was no correlation between the change in PSEQ score and resiliency (BRS score r = -0.10 [95% CI -0.33 to 0.16]; p = 0.45) or patient involvement in decision-making (OPTION-5 r = -0.02 [95% CI -0.26 to 0.23]; p = 0.88). Discussing PROMs results (PROMIS PF-SF10a) at the point of care did not increase pain self-efficacy during one visit. Therefore, surgeons do not need to discuss pain self-efficacy PROM scores in order to influence patient pain self-efficacy. While PROMs remain valuable tools for assessing patient outcomes, further work may assess whether the collection of PROMs itself may increase pain self-efficacy or whether longitudinal discussion of PROMs with patients changes pain self-efficacy. Level II, therapeutic study.

Collecting Real-Time Patient-Reported Outcome Data During Latent Labor: Feasibility Study of the MyCap Mobile App in Prospective Person-Centered Research.

The growing emphasis on patient experience in medical research has increased the focus on patient-reported outcomes and symptom measures. However, patient-reported outcomes data are subject to recall bias, limiting reliability. Patient-reported data are most valid when reported by patients in real time; however, this type of data is difficult to collect from patients experiencing acute health events such as labor. Mobile technologies such as the MyCap app, integrated with the REDCap (Research Electronic Data Capture) platform, have emerged as tools for collecting patient-generated health data in real time offering potential improvements in data quality and relevance. This study aimed to evaluate the feasibility of using MyCap for real-time, patient-reported data collection during latent labor. The objective was to assess the usability of MyCap in characterizing patient experiences during this acute health event and to identify any challenges in data collection that could inform future research. In this descriptive cohort study, we quantified and characterized data collected prospectively through MyCap and the extent to which participants engaged with the app as a research tool for collecting patient-reported data in real time. Longitudinal quantitative and qualitative surveys were sent to (N=18) enrolled patients with term pregnancies planning vaginal birth at Oregon Health Sciences University. Participants were trained in app use prenatally. Then participants were invited to initiate the research survey on their personal smartphone via MyCap when they experienced labor symptoms and were asked to return to MyCap every 3 hours to provide additional longitudinal symptom data. Out of 18 enrolled participants, 17 completed the study. During latent labor, 13 (76.5%) participants (all those who labored at home and two-thirds of those who were induced) recorded at least 1 symptom report during latent labor. A total of 191 quantitative symptom reports (mean of 10 per participant) were recorded. The most commonly reported symptoms were fatigue, contractions, and pain, with nausea and diarrhea being less frequent but more intense. Four participants recorded qualitative data during labor and 14 responded to qualitative prompts in the postpartum period. The study demonstrated that MyCap could effectively capture real-time patient-reported data during latent labor, although qualitative data collection during active symptoms was less robust. MyCap is a feasible tool for collecting prospective data on patient-reported symptoms during latent labor. Participants engaged actively with quantitative symptom reporting, though qualitative data collection was more challenging. The use of MyCap appears to reduce recall bias and facilitate more accurate data collection for patient-reported symptoms during acute health events outside of health care settings. Future research should explore strategies to enhance qualitative data collection and assess the tool's usability across more diverse populations and disease states.

Modeling Longitudinal Quality of Life Changes in Patients Diagnosed with Lymphomas: Heatmap Analysis of Electronic Patient-Reported Outcomes (ePRO) Data from the Bison-PRO Quality of Life Study

Modeling Longitudinal Quality of Life Changes in Patients Diagnosed with Lymphomas: Heatmap Analysis of Electronic Patient-Reported Outcomes (ePRO) Data from the Bison-PRO Quality of Life Study

Machine learning models including patient-reported outcome data in oncology: a systematic literature review and analysis of their reporting quality.

To critically examine the current state of machine learning (ML) models including patient-reported outcome measure (PROM) scores in cancer research, by investigating the reporting quality of currently available studies and proposing areas of improvement for future use of ML in the field. PubMed and Web of Science were systematically searched for publications of studies on patients with cancer applying ML models with PROM scores as either predictors or outcomes. The reporting quality of applied ML models was assessed utilizing an adapted version of the MI-CLAIM (Minimum Information about CLinical Artificial Intelligence Modelling) checklist. The key variables of the checklist are study design, data preparation, model development, optimization, performance, and examination. Reproducibility and transparency complement the reporting quality criteria. The literature search yielded 1634 hits, of which 52 (3.2%) were eligible. Thirty-six (69.2%) publications included PROM scores as a predictor and 32 (61.5%) as an outcome. Results of the reporting quality appraisal indicate a potential for improvement, especially in the areas of model examination. According to the standards of the MI-CLAIM checklist, the reporting quality of ML models in included studies proved to be low. Only nine (17.3%) publications present a discussion about the clinical applicability of the developed model and reproducibility and only three (5.8%) provide a code to reproduce the model and the results. The herein performed critical examination of the status quo of the application of ML models including PROM scores in published oncological studies allowed the identification of areas of improvement for reporting and future use of ML in the field.

Patients with Chronic Kidney Disease: Background Factors Associated with Experienced Health Status and Life Satisfaction.

The prevalence of chronic kidney disease (CKD) is increasing and CKD often goes undiagnosed and untreated until its later stages when irreversible damage has occurred. Patients with CKD have been reported to have lower quality of life than the general population, but the patient-reported outcome data on CKD patients in Finland are limited. The primary outcome of this structural, multiple-choice survey study was to assess life satisfaction and experienced health status in Finnish patients with CKD. The results were presented as numbers (n) and percentages (%). The secondary outcome was to identify patient groups using the K-means clustering method based on preselected response variables and to assess the associated background factors. In total, 558 patients with CKD responded to the electronic survey. Of the 395 patients who completed the whole survey, 39.7% reported their health status as good, pretty good, or excellent, and 59.9% were fairly or very satisfied with their life. Two clusters of patients could be identified based on their health status and life satisfaction: patients with (1) poorer or (2) better well-being. Patients with poorer well-being were more likely to have at least 3 comorbidities (66.8% vs 44.3%) and lack follow-up visits entirely (10.5% vs 1.9%), compared with patients with better well-being. The patients with poorer well-being were less often knowledgeable about the disease, its causes (35.5% vs 48.1%), and its care (30.7% vs 20.3%) than patients with better well-being, and they showed weaker adherence to lifestyle interventions such as following dietary instructions (30.3% vs 40.5%). Screening for CKD to enable early diagnosis, early commitment to treatment, and empowering the patient by providing education are key for improvement of health and life satisfaction in patients with CKD. Therefore, resources should be allocated to these measures of action.

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.

Analyzing Patient-Reported Outcome Data in Oncology Care

Analyzing Patient-Reported Outcome Data in Oncology Care