Assessment Of Patient-reported Outcomes Research Articles

Patient-Reported Outcomes From the Phase III HIMALAYA Study of Tremelimumab Plus Durvalumab in Unresectable Hepatocellular Carcinoma.

In the phase III HIMALAYA study (ClinicalTrials.gov identifier: NCT03298451) in unresectable hepatocellular carcinoma (uHCC), the Single Tremelimumab Regular Interval Durvalumab (STRIDE) regimen significantly improved overall survival versus sorafenib, and durvalumab monotherapy was noninferior to sorafenib. Patient-reported outcomes (PROs), a secondary outcome from HIMALAYA, are reported here. Participants were randomly assigned to receive STRIDE, durvalumab, or sorafenib. PROs were assessed (preplanned secondary outcome) using the European Organization for Research and Treatment of Cancer 30-item Quality of Life Questionnaire and the 18-item HCC module. Time to deterioration (TTD), change from baseline and improvement rate in global health status/quality of life (GHS/QoL), functioning, and disease-related symptoms were analyzed. In total, 1,171 participants were randomly assigned to STRIDE (n = 393), durvalumab (n = 389), or sorafenib (n = 389) and were evaluable for PRO assessments. Across treatment arms, compliance rates for PROs were >77% at baseline and >70% overall. Baseline scores were comparable across treatment arms. TTD in GHS/QoL, physical functioning, fatigue, appetite loss, and abdominal pain was numerically longer for both STRIDE and durvalumab versus sorafenib. Clinically meaningful deterioration in PROs was not observed in any treatment arm. However, TTD in nausea and abdominal swelling was numerically longer for STRIDE versus sorafenib, and the likelihood of clinically meaningful improvement in GHS/QoL, role, emotional and social functioning, and disease-related symptoms was greater with STRIDE and durvalumab versus sorafenib. PROs with STRIDE and durvalumab were generally similar. Compared with sorafenib, STRIDE and durvalumab were associated with clinically meaningful, patient-centered GHS/QoL, functioning, and symptom benefits in people with uHCC. These findings support the benefits of the STRIDE regimen compared with sorafenib for a diverse population reflective of the global uHCC population.

#2528 Comparing patient-reported and performance-based physical function assessments in hemodialysis: insights from the CONVINCE trial

Abstract Background and Aims Physical function is a crucial outcome in nephrology. In alignment with the Patient-Reported Outcomes Measurement Information System (PROMIS) Initiative, physical function is characterized as the ability to execute activities involving physical actions, ranging from self-care to more complex activities, such as social activities. This definition of physical function spans various domains of the International Classification of Functioning, Health, and Disability (ICF). The assessment of physical function commonly employs two distinct approaches, patient-reported outcomes (PRO) and observed performance outcomes (PerfO). PRO and PerfO assessments have distinct advantages and limitations and were found to correlate only moderately, adding complexity to cross-instrument outcome comparisons and interpretation. Despite this, there is limited understanding of the factors contributing to the discordance between PRO and PerfO assessments in patients with kidney disease. In this study, we applied a previously established common T-score metric for patient-reported and performance-based physical function to compare patterns of associations with potential laboratory-based, psychosocial, sociodemographic, and health-related determinants in hemodialysis patients. Method Cross-sectional data from the baseline assessment of the CONVINCE trial (N = 1,132), a multinational randomized controlled trial comparing high-dose hemodiafiltration with high-flux hemodialysis, were analysed. To investigate the relationships between potential determinants and performance-based versus patient-reported physical function, we performed multiple linear regression analysis. Dependent variables were standardized T-scores derived from the PROMIS physical function short-form 4a (PRO) and the Physical Performance Test (PerfO). Both measures assess a generic concept of physical function. We compared the effect sizes of each potential determinant in the multiple regression model using partial f2 values, with thresholds of 0.02, 0.15, and 0.35 denoting small, medium, and large effects. Results Both, PerfO and PRO exhibited significant associations with a laboratory marker-based indicator of muscle mass (simplified creatinine index), albeit with relatively small effects (partial f² of 0.04 and 0.05, respectively). None of the other laboratory measures investigated in this study showed any relevant associations with physical functioning. Age demonstrated a negative association with physical function, with a larger effect size observed for performance-based (partial f² = 0.11) compared to patient-reported physical function (partial f² = 0.04). In contrast to performance-based physical function, patient-reported physical function displayed a stronger association with self-reported health domains, particularly pain interference and fatigue. Exploring the differences between patient-reported and performance-based T-scores at the individual level, we found that younger age and more cognitive/emotional fatigue were associated with lower patient-reported compared to performance-based physical function (small effect sizes). Conclusion PRO and PerfO assessments of physical function in hemodialysis patients had similar associations with the simplified creatinine index, suggesting comparable links to objective physical impairment. However, PRO-based physical function exhibited a notably stronger association with other patient-reported variables, suggesting that patient-reported constructs of physical function might represent more than just capability, but ‘capability free from symptomatic constraints’. This distinction should be taken into account when directly comparing or aggregating PRO and PerfO scores. A noteworthy aspect of our study is the utilization of a common T-score metric for both PRO and PerfO measures. This method enhances interpretability of results and facilitates comparisons across diverse assessment types, thereby offering valuable insights into the determinants of physical function in patients undergoing hemodialysis.

#2502 The CONVINCE randomized trial found positive effects of hemodiafiltration on quality of life in patients with kidney disease on dialysis

Abstract Background and Aims Health-related quality of life of ESKD patients is negatively affected by disease-specific symptoms and can additionally be impaired by renal replacement therapy. Previous studies indicate that hemodiafiltration (HDF) may have a positive impact on the patients’ perceived health status compared to hemodialysis (HD), but overall evidence has been inconclusive. We investigated whether HDF is beneficial with respect to the patients’ perceived health status in the CONVINCE Trial. Method The CONVINCE Trial is an international multi-center, prospective, randomized, open label, controlled trial, comparing benefits and safety high-dose HDF versus high-flux HD. All-cause mortality was the primary outcome of the trial and a broad range of Patient-Reported Outcomes (PROs) were investigated as secondary outcomes. Eight generic health domains (Physical Function, Pain Intensity, Pain Interference, Fatigue, Sleep Disturbance, Depression, Anxiety, Ability to Participate in Social Roles and Activities) were assessed using the Patient-Reported Outcome Measurement Information System, PROMIS®-29 v2.0 profile, and an additional PROMIS short-form assessing Cognitive Function. PROs were collected every three months over the course of the study. As the perceived health status is likely related to mortality, we estimated the effect of HDF on PROs using a joint modelling approach to account for potential bias by selective dropout due to death. Results A total of n = 1 360 patients have been randomized to HD (n = 677) and HDF (n = 683). Both groups were comparable at baseline, with physical function (44.0 on the PROMIS T-score metric) considerably lower than the general population. Median follow-up was 30 months. We could obtain 84% of the planned PRO follow-up assessments on average. Overall, we found a steady decline in Health-Related Quality of Life (HRQoL) over the observation period; with statistically significant group differences for Physical Function (P = .047), Cognitive Function (P = .049), Pain Interference (P = .027) and the Ability to Participate in Social Roles and Activities (P = .023), all favouring HDF over HD. Conclusion Within the CONVINCE trial we have applied a comprehensive assessment of PROs comparing HDF with HD. Along with a beneficial effect on survival rates, we observed a slower decline in patients perceived health status when treated with HDF compared to HD. CONVINCE provides further evidence that HDF is beneficial in terms of quality of life.

Return to work and activity after rib-fixation for acute chest trauma: first application of a validated patient-reported outcomes assessment tool.

Rib fractures present a heavy pain and functional burden in trauma. Our primary aim was to determine return to work in patients with acute rib fractures requiring surgical stabilisation of rib fractures. Our secondary outcomes were pain and quality of life. We also document the first application of the Work Productivity and Activity Impairment Instrument, a validated injury-specific patient-reported outcome measure, for chest wall injury in the literature. A retrospective review was conducted of patients with rib fractures requiring surgical fixation in a single centre between 2008-2020. After applying inclusion and exclusion criteria to ensure relevance, all eligible patients were asked to complete patient reported outcome measure questionnaires. Of 1841 trauma patients with rib fractures, 66 underwent surgical fixation. Thirty-nine patients were eligible and thirty-one completed the questionnaires. Pre-injury and post-injury answers were compared. The number of patients in employment decreased post-operatively from 22 to 16 (p = 0.006). For those that returned to work there was no difference in hours missed but reduced weekly hours and productivity scores. There were significantly more patients with pain and on pain relief. There was a lower quality of life score post-operatively. Approximately 1-in-5 patients who require surgical fixation for rib fractures will not return to work. This is the first chest wall trauma study that uses the Work Productivity and Activity Impairment Instrument, a validated tool for work productivity outcomes. We recommend this instrument as a reliable tool for investigating return to work outcomes in trauma patients.

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

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

Patient-reported outcomes after oncologic hepatic resection predict the risk of delayed readiness to return to intended oncologic therapy (RIOT)

BackgroundOptimal surgical recovery is critical to readiness to return to intended oncologic therapy (RIOT). The current study defined the value of patient-reported outcomes (PROs) in predicting the risk for delayed RIOT after oncologic hepatic resection. MethodsIn a prospective longitudinal study, perioperative symptoms were assessed using a valid PRO assessment tool, the MD Anderson Symptom Inventory module for hepatectomy perioperative care (MDASI-PeriOp-Hep), for 4 weeks after surgery. The timed up and go test (TUGT) was administered before surgery, by discharge day, and at the first postoperative follow-up visit. Multivariate logistic regression analysis assessed the predictive value of PROs for delayed RIOT. ResultsWe enrolled 210 patients and analyzed 148 patients who received adjuvant chemotherapy and contributed more than 3 PRO assessments postoperatively. About 36 percent of the patients had delayed RIOT (>5 weeks, range 1-14 weeks). MDASI scores for drowsiness, fatigue, dry mouth, and interference with general activity, walking, and work on day 7 after discharge and MDASI scores for incisional tightness, fatigue, dry mouth, shortness of breath, and interference with work on day 14 after discharge were associated with delayed RIOT (all P<0.05). Walking and general activity items on the MDASI-Interference subscale on day 7 after discharge were highly correlated with prolonged TUGT scores at discharge (P<0.01). ConclusionWe defined clinically meaningful PROs on MDASI-PeriOp-Hep after hepatic resection that predicted increased risk of delayed RIOT. These findings highlight the importance PROs for monitoring symptoms and functioning 1-2 weeks after discharge to be implementing into perioperative care.

Quality of Life and Patient-Reported Outcomes Assessments in the Field of Oncology

Quality of Life and Patient-Reported Outcomes Assessments in the Field of Oncology

Clinical Impact of Routine Assessment of Patient-Reported Health Status in Heart Failure Clinic: The PRO-HF Trial.

The impact of routine clinic use of patient-reported outcome (PRO) measures on clinical outcomes in patients with heart failure (HF) has not been well-characterized. We tested if clinic-based use of a disease-specific PRO improves patient-reported quality of life at 1 year. The PRO-HF trial (Patient-Reported Outcome Measurement in Heart Failure Clinic) was an open-label, parallel, patient-level randomized clinical trial of routine PRO assessment or usual care at an academic HF clinic between August 30, 2021, and June 30, 2022, with 1 year of follow-up. In the PRO assessment arm, participants completed the Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12) at each HF clinic visit, and results were shared with their treating clinician. The usual care arm completed the KCCQ-12 at randomization and 1 year later, which was not shared with the treating clinician. The primary outcome was the KCCQ-12 overall summary score (OSS) between 12 and 15 months after randomization. Secondary outcomes included domains of the KCCQ-12, hospitalization and emergency department visit rates, HF medication therapy, clinic visit frequency, and testing rates. Across 17 clinicians, 1248 participants were enrolled and randomized to PRO assessment (n=624) or usual care (n=624). The median age was 63.9 years (interquartile range [IQR], 51.8-72.8), 38.9% were women, and the median baseline KCCQ-12 OSS was 82.3 (IQR, 58.3-94.8). Final KCCQ-12 (available in 87.9% of the PRO arm and 85.1% in usual care; P=0.16) median OSS were 87.5 (IQR, 68.8-96.9) in the PRO arm and 87.6 (IQR, 69.7-96.9) in the usual care arm with a baseline-adjusted mean difference of 0.2 ([95% CI, -1.7 to 2.0]; P=0.85). The results were consistent across prespecified subgroups. A post hoc analysis demonstrated a significant interaction with greater benefit among participants with a baseline KCCQ-12 OSS of 60 to 80 but not in less or more symptomatic participants. No significant differences were found in 1-year mortality, hospitalizations, emergency department visits, medication therapy, clinic follow-up, or testing rates between arms. Routine PRO assessment in HF clinic visits did not impact patient-reported quality of life or other clinical outcomes. Alternate strategies and settings for embedding PROs into routine clinical care should be tested. URL: https://www.clinicaltrials.gov; Unique identifier: NCT04164004.

Electronic health intervention to manage symptoms of immunotherapy in patients with cancer (SOFIA): Results from a randomized controlled pilot trial.

For patients receiving immune checkpoint inhibitors, early detection of immune-related adverse events (irAEs) is critical for one's safety. To this end, a smartphone app (SOFIA) was developed that featured the assessment of electronic patient-reported outcomes (ePROs) focusing on irAEs as well as a set of comprehensive supportive information. Its feasibility and preliminary efficacywere evaluated in a randomized controlled trial (RCT). Patients who received immune checkpoint inhibition therapy were randomly assigned to an intervention group (IG) or a control group (CG; care as usual). During the 12-week intervention period, IG patients used SOFIA to report twice weekly ePROs and receive cancer- and immunotherapy-relevant contents. Before a patient's next clinical visit, the physician in charge was given the ePRO reports. The primary objective was to test the feasibility of SOFIA. Furthermore, the preliminary efficacy of SOFIA for health-related quality of life (HRQOL), psychosocial outcomes, and medical data was examined. Clinical outcomes were assessed at baseline (T0), post-intervention (T1), and a 3-month follow-up (T2). Seventy-one patients were randomized to the IG (n=34) or the CG (n=37). SOFIA showed high feasibility and acceptance. At T1, patients in the IG reported significantly better HRQOL and role functioning and less depression, distress, and appetite loss. No significant differences were revealed regarding medical data, the utilization of supportive care services, or survival. SOFIA showed high feasibility and acceptance and improved HRQOL and psychosocial outcomes. These results suggest further evaluation of efficacy in a large-scale confirmatory multicenter RCT.

Thresholds in PROMIS Scores Anchored to Subsequent Unscheduled Health Service Use Among People Diagnosed With Cancer.

To establish thresholds in the Patient-Reported Outcomes Measurement Information System (PROMIS) pain interference, physical function, fatigue, and depression scores on the basis of their association with subsequent use of the emergency department (ED) or urgent care by people diagnosed with cancer. Retrospective data from 952 people seen at Henry Ford Cancer and insured through the Health Alliance Plan were analyzed using generalized linear mixed-effects models. The log odds of ED or urgent care use during 14 or 30 days after each patient-reported outcome (PRO) assessment were related to PRO scores, while adjusting for comorbidity, sociodemographic, and tumor characteristics. Pain interference and physical function were associated with subsequent ED or urgent care visits, but fatigue and depression were not, and the results for 14- and 30-day visits were similar. Thresholds anchored in the likelihood of these visits differed according to cancer stage. For people with advanced cancer, a pain interference score of 60 or higher (odds ratio [OR] 3.75, [95% CI, 1.53 to 7.87]) and a physical function score lower than 40 (OR 2.94, [95% CI, 1.22 to 7.06]) produced the largest ORs with narrowest CIs for 30-day visits. For people with nonadvanced cancer, the thresholds of 65 for pain interference (OR 2.64, [95% CI, 1.40 to 5.01]) and 35 for physical function (OR 1.87, [95% CI, 1.01 to 3.45]) produced largest ORs with narrowest CIs for 30-day visits. These anchor-based thresholds in PROMIS scores can inform clinicians' actions with the goal of preventing ED or urgent care visits.

The GUTFIT Cohort: Understanding of different gastrointestinal symptoms score variation between Chinese and non-Chinese individuals with functional constipation

The diagnosis of functional constipation (FC) relies on patient-reported outcomes evaluated as criteria based on the clustering of symptoms. Although the ROME IV criteria for FC diagnosis is relevant for a multicultural population(1), how an individual’s lifestyle, environment and culture may influence the pathophysiology of FC remains a gap in our knowledge. Building on insights into mechanisms underpinning disorders of gut-brain interactions (formerly functional gastrointestinal disorders) in the COMFORT Cohort(2), this study aimed to investigate the differences in gastrointestinal (GI) symptom scores among participants with FC in comparison to healthy controls between Chinese and non-Chinese New Zealanders. The Gastrointestinal Understanding of Functional Constipation In an Urban Chinese and Urban non-Chinese New Zealander Cohort (GUTFIT) study was a longitudinal cohort study, which aimed to determine a comprehensive profile of characteristics and biological markers of FC between Chinese and non-Chinese New Zealanders. Chinese (classified according to maternal and paternal ethnicity) or non-Chinese (mixed ethnicities) adults living in Auckland classified as with or without FC based on ROME IV were enrolled. Monthly assessment (for 3 months) of GI symptoms, anthropometry, quality of life, diet, and biological samples were assessed monthly over March to June 2023. Demographics were obtained through a self-reported questionnaires and GI symptoms were assessed using the Gastrointestinal Symptom Rating Scale (GSRS) and Structured Assessment of Gastrointestinal Symptoms Scale (SAGIS). This analysis is a cross-sectional assessment of patient-reported outcomes of GI symptoms. Of 78 enrolled participants, 66 completed the study (male, n = 10; female, n = 56) and were distributed across: Chinese with FC (Ch-FC; n = 11), Chinese control (Ch-CON; n = 19), non-Chinese with FC (NCh-FC; n = 16), non-Chinese control (NCh-CON; n = 20). Mean (SD) age, body mass index, and waist circumference were 40 ± 9 years, 22.7 ± 2.5 kg/m2, and 78.0 ± 7.6 cm, respectively. Ethnicity did not impact SAGIS domain scores for GI symptoms (Ethnicity x FC severity interaction p&gt;0.05). Yet, the constipation symptoms domain of the GSRS was scored differently depending on ethnicity and FC status (Ethnicity x FC interaction p&lt;0.05). In post hoc comparison, NCh-FC tended to have higher GSRS constipation severity scores than Ch-FC (3.4 ± 1.0 versus 3.8 ± 0.8 /8, p&lt;0.1) Although constipation symptom severity tended to be higher in NCh-FC, on the whole, ethnicity did not explain variation in this cohort. FC status was a more important predictor of GI symptoms scores. Future research will assess differences in symptom burden to explore ethnicity-specific characteristics of FC.

Early and Sustained Improvements in Symptoms and Quality of Life with Upadacitinib in Adults and Adolescents with Moderate-to-Severe Atopic Dermatitis: 52-Week Results from Two Phase III Randomized Clinical Trials (Measure Up 1 and MeasureUp2).

Atopic dermatitis is a chronic inflammatory disease characterized by increased itch, skin pain, poor sleep quality, and other symptoms that negatively affect patient quality of life. Upadacitinib, an oral selective Janus kinase (JAK) inhibitor with greater inhibitory potency for JAK1 than JAK2, JAK3, or tyrosine kinase 2, is approved to treat moderate-to-severe atopic dermatitis. We aimed to evaluate the effect of upadacitinib on patient-reported outcomes over 52weeks in adults and adolescents with moderate-to-severe atopic dermatitis. Data from two phase III monotherapy trials of upadacitinib (Measure Up 1, NCT03569293; Measure Up 2, NCT03607422) were integrated. Changes in pruritus, pain, other skin symptoms, sleep, quality of life, mental health, and patient impression were evaluated. Patient-reported outcome assessments included the Worst Pruritus Numerical Rating Scale, Patient-Oriented Eczema Measure, Dermatology Life Quality Index, Atopic Dermatitis Symptom Scale, Atopic Dermatitis Impact Scale, Hospital Anxiety and Depression Scale, SCORing Atopic Dermatitis index, Patient Global Impression of Severity, Patient Global Impression of Change, and Patient Global Impression of Treatment. Minimal clinically important differences, achievement of scores representing minimal disease burden, and the change from baseline were evaluated in patients who received upadacitinib through week 52 and in patients who received placebo through week16. This analysis included 1609 patients (upadacitinib 15 mg, N=557; upadacitinib 30 mg, N=567; placebo, N=485). Baseline demographics and disease characteristics were generally similar across all arms. The proportion of patients treated with upadacitinib reporting improvements in itch increased rapidly by week 1, increased steadily through week 8, and was sustained through week 52. Patients receiving upadacitinib also experienced improvements in pain and other skin symptoms by week 1, which continued through week 16; improvements were maintained through week 52. Patient reports of improved sleep increased rapidly from baseline to week 1, increased steadily through week 32, and were sustained through week 52. Patients experienced quality-of-life improvements through week 8, which were maintained through week 52. By week 1, patients in both upadacitinib groups experienced rapid improvements in emotional state, and by week 12, patients also achieved meaningful improvements in anxiety and depression. Improvements in mental health continued steadily through week 32 and were maintained through week 52. Patients treated with upadacitinib30mg generally experienced improvements in patient-reported outcomes earlier than those treated with upadacitinib 15 mg. Through week 16, patients receiving upadacitinib experienced greater improvements versus those receiving placebo in all assessed patient-reported outcomes. Adults and adolescents with moderate-to-severe atopic dermatitis treated with once-daily upadacitinib 15 or 30 mg experienced early improvements in itch, pain, other skin symptoms, sleep, quality of life, and mental health that were sustained through week52. ClinicalTrials.gov identifiers NCT03569293 (13 August 2018) and NCT03607422 (27 July 2018).

Abstract 1302: Development and implementation of a systematic approach to measure patient-reported outcomes in early-phase cancer trials

Abstract Background: There has been a shift in the clinical trial landscape toward early-phase studies where data from well-designed and pivotal phase I/II trials is leading to accelerated drug approval. Our goal was to develop a standardized approach to the measurement and reporting of patient-reported outcomes (PROs) in early-phase trials to improve assessment of tolerability, address noted barriers to inclusion, and enhance patient-provider communication to inform treatment decisions and ultimately improve outcomes. Methods: The Office of Patient Centered Outcomes Research (OPCORe) was established in 2022 within the Center for Cancer Research at the National Cancer Institute (NCI) with the mission to advance understanding of the clinical benefit and tolerability of cancer therapies by integrating patient-centered approaches into CCR clinical trials by fostering education and collaboration with stakeholders. Implementation of regulatory/consortia guidelines related to use of core constructs, assessment frequency, and hypothesis-driven objectives and analysis plans through protocol template, development of data capture, email-based alerts and data visualization for clinical providers was developed and standardized templates for protocol language, research staff education, tools for workflow, and tracking implemented. Results: The OPCORe protocol template includes measurement of 3 constructs, including tolerability, clinical benefit and disease-specific impact, as well as anchors of meaningful change. Core symptomatic toxicities are measured to inform understanding across trials with agent/class-specific symptomatic toxicities added to assure relevance. Baseline and standardized weekly electronic capture of tolerability during early cycles with alerts to clinical research staff if moderate-severe toxicities reported helps facilitate patient check-ins. Repeat PROs assessments in later cycles are expanded to include disease-specific assessments and align with disease evaluation to further inform clinical impact. To date, we have collaborated with 7 research groups and developed approaches for first-in-human, phase I, and phase II studies with trial start-up and data collection in process. Conclusion: The value of including PROs in cancer trials is increasingly recognized by regulators, clinicians, patients, and advocates to advance understanding of the clinical benefit &amp; tolerability of emerging therapies. Standardization and incorporation of PROs in earlier phase trials may enable PROs to be optimized, improve later phase trial design, and help inform health policy, support drug approval, and promote shared decision making between providers and patients to inform treatment decisions. Citation Format: Amanda L. King, Jason E. Levine, Elizabeth Vera, Yuti Gandhi, Elaine Novakovich, Theresa Yu, Sefanit Berhanu, Ciara Locke, Kaelyn Julmeus, Tito Mendoza, Terri S. Armstrong. Development and implementation of a systematic approach to measure patient-reported outcomes in early-phase cancer trials [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 1302.

Electrical impedance tomography as a bedside assessment tool for COPD treatment during hospitalization.

For patients with chronic obstructive pulmonary disease (COPD), the assessment of the treatment efficacy during hospitalization is of importance to the optimization of clinical treatments. Conventional spirometry might not be sensitive enough to capture the regional lung function development. The study aimed to evaluate the feasibility of using electrical impedance tomography (EIT) as an objective bedside evaluation tool for the treatment of acute exacerbation of COPD (AECOPD). Consecutive patients who required hospitalization due to AECOPD were included prospectively. EIT measurements were conducted at the time of admission and before the discharge simultaneously when a forced vital capacity maneuver was conducted. EIT-based heterogeneity measures of regional lung function were calculated based on the impedance changes over time. Surveys for attending doctors and patients were designed to evaluate the ease of use, feasibility, and overall satisfaction level to understand the acceptability of EIT measurements. Patient-reported outcome assessments were conducted. User's acceptance of EIT technology was investigated with a five-dimension survey. A total of 32 patients were included, and 8 patients were excluded due to the FVC maneuver not meeting the ATS criteria. Spirometry-based lung function was improved during hospitalization but not significantly different (FEV1 %pred.: 35.8% ± 6.7% vs. 45.3% ± 8.8% at admission vs. discharge; p = 0.11. FVC %pred.: 67.8% ± 0.4% vs. 82.6% ± 5.0%; p = 0.15. FEV1/FVC: 0.41 ± 0.09 vs. 0.42 ± 0.07, p = 0.71). The symptoms of COPD were significantly improved, but the correlations between the improvement of symptoms and spirometry FEV1 and FEV1/FVC were low (R = 0.1 and -0.01, respectively). The differences in blood gasses and blood tests were insignificant. All but one EIT-based regional lung function parameter were significantly improved after hospitalization. The results highly correlated with the patient-reported outcome assessment (R > 0.6, p < 0.001). The overall acceptability score of EIT measurement for both attending physicians and patients was high (4.1 ± 0.8 for physicians, 4.5 ± 0.5 for patients out of 5). These results demonstrated that it was feasible and acceptable to use EIT as an objective bedside evaluation tool for COPD treatment efficacy.

A Comprehensive Review of Total Hip Arthroplasty Outcomes in Post-traumatic Hip Arthritis: Insights and Perspectives.

Post-traumatic hip arthritis presents a challenging condition characterized by degenerative changes in the hip joint following traumatic injury. Total hip arthroplasty (THA) is a cornerstone in managing this condition, offering significant pain relief, functional improvement, and enhanced quality of life. This comprehensive review aims to synthesize existing literature to elucidate the outcomes of THA in post-traumatic hip arthritis, exploring factors influencing surgical success and identifying areas for further research. Key findings reveal favourable clinical outcomes associated with THA, though considerations such as patient characteristics, surgical techniques, and implant selection impact outcomes. Implications for clinical practice underscore the importance of tailored preoperative assessment and ongoing advancements in surgical approaches and implant technology. Furthermore, opportunities for future research lie in long-term durability studies, patient-reported outcomes assessment, and exploration of innovative surgical techniques. Overall, THA emerges as a promising intervention for post-traumatic hip arthritis, yet continual refinement through research and innovation remains imperative to optimize patient care in this population.

Utility of a Focused Patient Reported Outcome Assessment in a Head and Neck Cancer Radiation Oncology Clinic

Utility of a Focused Patient Reported Outcome Assessment in a Head and Neck Cancer Radiation Oncology Clinic

Health-related quality of life in patients with hematologic malignancies treated with chimeric antigen receptor T-cell therapy: review and current progress.

Chimeric antigen receptor (CAR) T-cell therapy has transformed the care of patients with relapsed/refractory B-cell derived hematologic malignancies. To date, six CAR T-cell therapies, targeting either CD19 or B-cell maturation antigen, have received regulatory approval. Along with the promising survival benefit, CAR T-cell therapy is associated with potentially lifethreatening adverse events (AE), including cytokine release syndrome and immune effector cellassociated neurotoxicity syndrome. While clinical trials evaluating CAR T-cell therapy consistently report the incidence of these AE, most trials do not collect health-related quality of life (HRQoL) data. As such, the impact of CAR T-cell therapy process and related AE on the physical and psychological well-being of patients remains uncertain. HRQoL and other patientreported outcome (PRO) assessments in patients with relapsed or refractory hematologic malignancies are of utmost importance, as individuals may have unmet needs and a high demand for tolerable therapy if a cure is not obtained. In addition, it is important to standardize methods of data collection to better assess the impact of CAR T-cell therapy on quality of life, optimize patient care and costs, and enable comparison between different studies. We conducted a literature search up to June 2023 to identify the HRQoL tools used in clinical trials and in realworld studies investigating CAR T-cell therapy in patients with lymphomas or leukemias. In the present comprehensive review, we summarize the most commonly used CAR T-cell specific and non-specific HRQoL tools and discuss how the use of HRQoL and other PRO tools may be optimized.

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

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

Recommendations to address respondent burden associated with patient-reported outcome assessment.

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient perspective and to inform regulatory decisions and health policy. The use of PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with potential impact on the completeness and quality of the data for decision-making. We therefore conducted an initial literature review to generate a list of candidate recommendations aimed at reducing respondent burden. This was followed by a two-stage Delphi survey by an international multi-stakeholder group. A consensus meeting was held to finalize the recommendations. The final consensus statement includes 19 recommendations to address PRO respondent burden in healthcare research and clinical practice. If implemented, these recommendations may reduce PRO respondent burden.

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

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