Utility Of Patient-reported Outcomes Research Articles

Utilization of patient-reported outcomes in joint replacement care design.

Analysis finds health disparities among the elective surgery population.

Pediatric Patient-Reported Outcomes in Chronic Disease

Managing chronic disease is increasingly important in pediatric health care. In internal medicine, patient-reported, patient-important outcomes are a key part of assessment of programs. In pediatrics, there has been less focus on patient-reported outcomes. These outcomes are more complex in pediatrics because the utility of patient-reported outcomes may differ by age and, in most cases, parent-reported outcomes are also relevant. In some instances, parents serve as a surrogate for the pediatric patient. In other circumstances, parent-reported outcomes may differ from child-reported outcomes. In this volume of the Journal, Forrest et al report on self-reported child health outcomes across 10 chronic conditions. Across all these conditions, children reported more anxiety, fatigue, pain, and restriction of mobility than reported in the general pediatric population. Disease activity was associated with poorer self-reported outcomes. These results emphasize the need to incorporate patient-reported outcomes in pediatric chronic disease management. Programs will need to develop strategies to improve these patient-reported outcomes. Article page 207

Measuring Quality of Life Following Robot-Assisted Radical Prostatectomy.

BackgroundProstate cancer (PCa) represents the most common solid organ malignancy in men. Fortunately, at the time of diagnosis, the majority of cases are staged as localized or regional disease, conferring excellent 5- and 10-year cure rates. There are several first line treatment options including surgical approaches such as robot-assisted radical prostatectomy (RARP) and radiation therapy (RT) available to patients with localized disease that offer similar PCa oncologic outcomes but are associated with potentially significant side effects which may impact health-related quality of life (HRQOL) domains. Recently, clinicians and investigators have sought to better understand these changes in HRQOL metrics with the utilization of patient-reported outcomes (PRO). Given that RARP represents the most common surgical treatment for PCa in the United States, there has been a particular interest in assessing these outcomes derived by patient perspectives to more fully appreciate treatment-related impact on quality of life following RARP.ObjectiveThis narrative review sought to explore the instruments available to measure quality of life after RARP, a review of the PRO data after RARP, and future directions for assessing and improving quality of life outcomes following this surgery.Clinical UseThere are several treatment options for men diagnosed with local and regional prostate cancer with similar oncologic outcomes but differing patterns of side effects affecting post-treatment quality of life. Understanding data reported directly by patients following RARP about their side effects and quality of life gives providers additional information for appropriate preoperative counseling for patients choosing between treatment options for their prostate cancer.

QOL-31. USE OF PATIENT-REPORTED OUTCOMES TO IDENTIFY YOUTH AT RISK FOR IMPAIRED OVERALL HEALTH

Pediatric brain tumor survivors often experience persistent and clinically significant late-effects following treatment. Critical to understanding morbidity is utilization of patient-reported outcomes (PROs). The current study evaluated PROs of individuals previously diagnosed with a pediatric brain tumor and identified risk factors for less optimal overall health. Participants included 127 youth 10.59±4.81 (M±SD) years old at survey completion and 4.45±3.82 years from diagnosis of a brain tumor (34.6% Pilocytic Astrocytoma, 9.4% Medulloblastoma, 9.4% Ependymoma, 7.9% Craniopharyngioma, 38.6% Other). Outcomes were assessed via Patient-Reported Outcomes Measurement Information System (PROMIS) parent-proxy measures. Overall health was assessed via PROMIS Global Health (i.e., a measure of general, physical, mental, and social health). Univariate and logistic regression analyses examined potential demographic, medical, and psychosocial factors (e.g., age, race, diagnosis, treatment) related to poor global health. Initial descriptive analyses suggested that most youth experienced anxiety symptoms (T-score M±SD=50.71±11.54), depressive symptoms (47.96±10.34), cognitive functioning (46.52±9.10), and fatigue (55.14±10.62) similar to their peers. However, 31.0% of youth experienced impaired global health (T-score<40). After adjusting for other potential covariates, the final model suggested that youth with significant anxiety (OR=6.20, CI=1.56–24.65), youth with significant fatigue (OR=3.96, CI=1.26–12.41), and youth who did not undergo a gross total resection (OR=0.25, CI=0.07–0.96) were at risk for impaired global health. Identifying those at-risk for impaired health is essential to reducing survivor morbidity and optimizing overall quality of life following treatment. Current data suggest potentially modifiable factors that may improve long-term outcomes for survivors of pediatric brain tumors.

Associations between Patient-reported Outcomes and Death or Lung Transplant in Idiopathic Pulmonary Fibrosis. Data from the Idiopathic Pulmonary Fibrosis Prospective Outcomes Registry.

Rationale: Progression of idiopathic pulmonary fibrosis (IPF) is accompanied by worsening of symptoms, exercise capacity, and health-related quality of life. However, the utility of patient-reported outcomes as predictors of mortality remains uncertain.Objectives: To assess whether patient-reported outcomes are independently associated with mortality beyond clinical risk factors in patients with IPF.Methods: Data from the observational IPF Prospective Outcomes Registry were used to examine associations between patient-reported outcomes at enrollment and the composite outcome of death or lung transplant in the following year. Associations were examined using univariable models and models adjusted for age and clinical variables that have been associated with death or lung transplant in patients with IPF in this cohort (oxygen use, forced vital capacity % predicted, and diffusing capacity of the lungs for carbon monoxide % predicted at enrollment).Results: Among 662 patients, 45 died and 12 underwent lung transplant over 1 year. In the model adjusted for age and clinical variables that were associated with death or lung transplant, worse scores on the St. George’s Respiratory Questionnaire (SGRQ) total score (hazard ratio [HR], 1.22 [95% confidence interval (CI), 1.01–1.48] per 10-point increase), SGRQ activity score (HR, 1.25 [95% CI, 1.02–1.54] per 10-point increase) and SGRQ symptoms score (HR, 1.17 [95% CI, 1.01–1.36] per 10-point increase) were associated with death or lung transplant over 1 year.Conclusions: Patient-reported outcomes that assess symptoms and physical activity are independently associated with mortality in patients with IPF.

Emerging trends and utilization of patient-reported outcomes (PROs) in clinical trials of chimeric antigen receptor (CAR) T-cell therapies.

e19142 Background: Patient-reported outcomes (PROs) are an important tool to assess the impact of new therapies on health-related quality of life (HRQoL). This study aimed to describe if and what PRO instruments are currently being utilized in CAR-T cell therapy studies in solid and hematological malignancies while assessing the patterns of inclusion and trends of HRQoL data reporting. Methods: We used Citeline to search for clinical trials between Jan 2008 - Jan 2020, excluding planned or terminated studies, non-oncology, non-treatment, and duplicates. Reviewers extracted various parameters for included trials, then cross-matched data with EU Clinical Trials Register, Clinical trials.gov, trial protocols (when available), and Google. The reporting of PRO data was then assessed for those Closed/Completed trials that included a PRO via PubMed/MEDLINE, Sponsor, and Google. Results: A sample of 664 CAR-T trials was identified. PROs were included in only 6.17% (41/664) studies. Of the 41 trials that included a PRO, 63.41% (26/41) utilized more than one PRO, with the generic EORTC QLQ-C30 and the EQ-5D being used predominately. Median HRQoL follow-up was 5-years on most trials. No studies used PROs as primary endpoints. The majority of PROs were observed to be utilized in early phase trials (phase I, 12; Phase I/II, 17). PROs were first incorporated in CAR-T trials beginning in 2014, and the utilization rate has increased steadily, except for 2019. PROs were included in 3 first line trials, 22 second line, 5 third line, and 11 fourth line or greater. PRO utilization between solid tumor trials and hematologic malignancies was comparable (6.04% [9/149], and 6.26% [32/511]). Of the completed/closed trials, 28.57% (3/14) published PRO data and met at least eight of the CONSORT-PRO quality indicators. Conclusions: The utilization of PROs in CAR-T trials (6.17%) is under the industry average of 27%, despite the growing importance of HRQoL and its impact on value-based care. The findings from this review reflect the overall increased attention to CAR-T as a new therapeutic entity and the continued deficiency of including and reporting of PROs in trial designs.

Systematic Review of Patient-Reported Outcomes following Surgical Treatment of Lymphedema.

Introduction: Analysis of quality of life (QOL) outcomes is an important aspect of lymphedema treatment since this disease can substantially impact QOL in affected individuals. There are a growing number of studies reporting patient-reported outcomes (PROMs) for patients with lymphedema. The purpose of this study was to conduct a systematic review of outcomes and utilization of PROMs following surgical treatment of lymphedema. Methods: A literature search of four databases was performed up to and including March, 2019. Studies included reported on QOL outcomes after physiologic procedures, defined as either lymphovenous bypass (LVB) or vascularized lymph node transplant (VLNT), to treat upper and/or lower extremity primary or secondary lymphedema. Results: In total, 850 studies were screened—of which, 32 studies were included in this review. Lymphovenous bypass was the surgical intervention in 16 studies, VLNT in 11 studies, and both in 5 studies. Of the 32 total studies, 16 used validated survey tools. The most commonly used PROM was the lymph quality of life measure for limb lymphedema (LYMQOL) (12 studies). In the remaining four studies, the upper limb lymphedema 27 scale (ULL27), the short form 36 questionnaire (SF-36), the lymphedema functioning, disability and health questionnaire (Lymph-ICF), and lymphedema life impact scale (LLIS) were each used once. QOL improvement following surgical treatment was noted in all studies. Conclusions: Physiologic surgical treatment of lymphedema results in improved QOL outcomes in most patients. The use of validated PROM tools is increasing but there is no current consensus on use. Future research to evaluate the psychometric properties of PROMs in lymphedema is needed to guide the development and use of lymphedema-specific tools.

Advances in pediatric psychooncology.

This article reviews the current literature on psychosocial care of children with cancer with particular focus on evidence-based standards of care, including developments in systematic distress screening, utilization of patient-reported outcomes, evidence-based interventions, survivorship, palliative care and bereavement. Although attention to the psychosocial needs of the child and family is increasingly recognized as an essential element of care for children with cancer, implementing evidence-based care remains suboptimal. Recent efforts have focused on utilizing technology to improve the reach of these interventions and to enhance engagement with special populations, such as adolescents and young adults. Increasing data elucidate the long-term psychological and physical late effects of childhood cancer survivorship and the impact of cancer on siblings and the family. Gaps in clinical care and important directions for future research include the needs of infants and toddlers, overlooked minorities, and patients with hereditary tumor predisposition syndromes, and attention to the psychosocial impact of exciting new treatments, such as autologous chimeric antigen receptor (CAR) T-cell therapy. The evidence base for the psychosocial standards of care for children with cancer and their families continues to grow, but more work is needed to successfully implement these standards across pediatric cancer centers.

Utilization of Patient-Reported Outcomes in Myeloproliferative Neoplasm Clinical Trials Registered at Clinicaltrials.Gov

IntroductionPatients with myeloproliferative neoplasms (MPN) typically experience debilitating symptom profiles and profound quality of life (QoL) decrements. Characterizing symptom burden in MPN clinical trials is vital to inform efficacy analyses. Previous studies have shown clinicians and researchers depend on patient-reported outcomes (PROs) to glean disease-related symptoms and signs of QoL decline. The current literature also shows that integrating PROs into the routine care of patients with cancer is associated with increased survival compared with usual care. However, the extent to which PROs are employed in MPN clinical trials is undescribed. ClincalTrials.gov is a registry of clinical trials mandated by the US Congress and implemented in 2000 by the National Institutes of Health. This study used data from ClinicalTrials.gov to evaluate the characteristics of MPN clinical trials over time and their use of PROs as primary and secondary outcomes.MethodsThe statistical software R and the rclinicaltrials package were used to download data from ClinicalTrials.gov. Trial data were included in this study if the following criteria were met: (1) the words essential thrombocythemia, polycythemia vera, myelofibrosis, or myeloproliferativeneoplasm (MPN) were used in the registered primary disease condition or official trial title; (2) the trial start date was between January 1, 2000 - July 1, 2018; (3) primary/secondary outcome data were available. Trial outcome data were then parsed for PRO text phrases including MPN-Symptom Assessment Form, quality of life, questionnaire, patient-reportedoutcome, and more specific PRO items (e.g., bone pain, concentration problems, and depression). Descriptive statistics and regression analysis were used to assess rates of primary/secondary outcomes over time.ResultsClinical trial characteristicsThere were 535 trials satisfying the inclusion criteria. Since 2000, the number of registered MPN-related clinical trials ranged from 11 to 36 trials per year with 28 trials registered annually on average (Figure 1). Of the 535 trials, 470 (88%) were interventional and 65 (12%) observational. Trials often employed multiple intervention types including: drug (77%); procedure (28%); biological (19%); radiation (13%); genetic (3%); behavioral (2%); device (2%); dietary supplement (1%); other (24%) [percentages do not sum to 100% as categories are not mutually exclusive]. Eligibility criteria varied widely, though most used 18 years old as the minimum age requirement (71%; 379/535) and nearly all included both genders (>99%; 533/535).PRO outcomes over timeOf the 535 MPN trials, the number of primary outcomes within MPN trials did not change over time (p=.50) with an average of 1.6 per trial. Around 8% of MPN clinical trials had PRO-related primary outcomes; this rate also did not change over time (Figure 1: solid red line; p=.97). However, the total number of secondary outcomes per trial has increased since 2000 (p<.001). From 2000-2005, 2006-2011, and 2012-2018 there was an average of 2.8, 3.3, and 5.2 secondary outcomes per trial, respectively. Also, the proportion of MPN clinical trials with PRO-related secondary outcomes has increased steadily over time (Figure 1: dashed blue line; p<.001). On average, from 2000-2005, 2006-2011, and 2012-2018 the proportion of MPN clinical trials with PRO-related secondary outcomes increased from 8% to 23%, to 33%.ConclusionAnalysis of ClinicalTrials.gov data suggest that patient-reported outcomes are being implemented into MPN clinical trials at increasing rates. While this suggests that MPN trials are becoming more patient-centered, PROs are included more often as secondary endpoints than primary endpoints. This analysis also suggests an increase in the number of secondary endpoints in recent years in MPN clinical trials, consistent with observed trends of growing complexity and increasing amount of data captured in clinical trials in general. DisclosuresMesa:Ariad: Consultancy; Galena: Consultancy; Gilead: Research Funding; Promedior: Research Funding; Incyte: Research Funding; Novartis: Consultancy; CTI: Research Funding; Celgene: Research Funding. Scherber:Orphan Pharmaceuticals: Honoraria; Incyte: Consultancy. Dueck:Bayer: Employment; Phytogine: Employment; Pfizer: Honoraria.

PMU60 - Utilization of Patient-Reported Outcomes in Asian Trial Literature

While overall use of patient-reported outcomes (PRO) measures has increased dramatically globally in the past 10 years, the inclusion of PRO data in controlled trials are still in its naissance, especially in the larger Asian region. PRO measures are still generally observed as a means of arriving at the current paragon of measuring the worth of a treatment - its cost-effectiveness. As the global trend in healthcare moves rapidly towards patient-centered medicine, it will become increasingly important to focus sincerely on accurate and systematic data capture in this realm. The current study seeks to track the use of PROs in clinical and controlled trials in Asia to summarize the current trends in utilization of standardized and non-standardized PRO instruments to pave the way for future investigation in systematically collecting this data alongside clinical data. A search strategy that includes terms to screen for prospective Asian clinical and controlled trials that have used survey instruments to measure PROs. The literature review will be conducted using databases like MEDLINE, PubMed, Cochrane Library and Asian registries and will be organized according to country population, sub-disease, standardized PROs used, outcome type, and purpose of study. A total of 179 studies were identified and further screened to 130 in the past 10 years by eliminating studies that were focusing on Asian patients in the Western world. Of these, 41 were based in China, 29 in Japan, 16 in Korea, 13 in India, and the rest in other South East Asian countries. The preliminary results show that the focus of PROs is mainly focused on cancer therapies, major psychoses such as Alzheimer's and schizophrenia, or degenerative diseases like Parkinson's disease. Secondary analysis will confirm the above results, identify the most common PROs utilized, and the purpose behind this type of data capture.

Utilization of Patient-Reported Outcomes to Guide Symptom Management During Stereotactic Body Radiation Therapy for Prostate Cancer

Utilization of Patient-Reported Outcomes to Guide Symptom Management During Stereotactic Body Radiation Therapy for Prostate Cancer