Applying Patient-Reported Outcome Methodology to Capture Patient-Reported Health Data: Report From an NIH Collaboratory Roundtable

Patient-reported health data provide information for pragmatic clinical trials that may not be readily available from electronic health records or administrative claims data. In this report, we present key considerations for collecting patient-reported health information in pragmatic clinical trials, which are informed by best practices from patient-reported outcome research. We focus on question design and administration via electronic data collection platforms with respect to 3 types of patient-reported health data: medication use, utilization of health care services, and comorbid conditions. We summarize key scientific literature on the accuracy of these patient-reported data compared with electronic health record data. We discuss question design in detail, specifically defining the concept to be measured, patient understanding of the concept, recall periods of the question, and patient willingness to report. In addition, we discuss approaches for question administration and data collection platforms, which are key aspects of successful patient-reported data collection

Parents' reported preference scores for childhood atopic dermatitis disease states

BACKGROUND: We sought to elicit preference weights from parents for health states corresponding to children with various levels of severity of atopic dermatitis. We also evaluated the hypothesis that parents with children who had been diagnosed with atopic dermatitis would assign different preferences to the health state scenarios compared with parents who did not have a child with atopic dermatitis. METHODS: Subjects were parents of children aged 3 months to 18 years. The sample was derived from the General Panel, Mommies Sub-Panel, and Chronic Illness Sub-Panel of Harris Interactive. Participants rated health scenarios for atopic dermatitis, asthma, and eyeglasses on a visual analog scale, imagining a child was experiencing the described state. RESULTS: A total of 3539 parents completed the survey. Twenty-nine percent had a child with a history of atopic dermatitis. Mean preference scores for atopic dermatitis were as follows: mild, 91 (95% confidence interval [CI], 90.7 to 91.5); mild/moderate, 84 (95%CI, 83.5 to 84.4); moderate, 73 (95%CI, 72.5 to 73.6); moderate/severe, 61 (95%CI, 60.6 to 61.8); severe, 49 (95% CI, 48.7 to 50.1); asthma, 58 (95%CI, 57.4 to 58.8); and eyeglasses, 87(95%CI, 86.3 to 87.4). CONCLUSIONS: Parents perceive that atopic dermatitis has a negative effect on quality of life that increases with disease severity. Estimates of parents' preferences can provide physicians with insight into the value that parents place on their children's treatment and can be used to evaluate new medical therapies for atopic dermatitis

Sexual Dysfunction and Reproductive Concerns in Young Men Diagnosed With Testicular Cancer: An Observational Study

INTRODUCTION: The survival rates for testicular cancer are excellent; still, there is a lack of knowledge regarding important survivorship issues, such as sexual dysfunction and reproductive concerns. AIM: The aim of this study was to investigate the prevalence and predictors of sexual dysfunction and reproductive concerns and the potential association between these issues in young men ∼2 years after a diagnosis of testicular cancer. METHODS: Data were collected from 111 men (response rate = 50%) diagnosed with testicular cancer at age 16-39. Patients were identified via the Swedish National Quality Registry for Testicular Cancer and approached with a survey, including standardized measures of sexual function, reproductive concerns, body image, and health-related quality of life. The survey was sent to participants approximately 2 years after their cancer diagnosis. Clinical variables were collected from the registry. Predictors were identified by multivariable linear regression analyses. MAIN OUTCOME MEASURES: The main outcomes were sexual function, assessed with the Patient-Reported Outcomes Measurement Information System Sexual Function and Satisfaction measure version 2.0, and reproductive concerns, assessed with the Reproductive Concerns After Cancer scale. RESULTS: Sexual dysfunction was reported by 26% of men, and a high level of reproductive concerns was reported by 28%. Lower satisfaction with sex life was associated with older age (β = -0.41), negative body image (β = -0.42), not having a partner (β = 4.8), and dissatisfaction with sex life before cancer (β = 8.31). Negative body image was associated with reproductive concerns in the dimensions of fertility potential (β = 0.06), partner disclosure (β = 0.08), and child's health (β = 0.07), whereas having had fertility preservation predicted higher levels of concerns with regard to personal health (β = 0.52) and achieving pregnancy (β = 0.53). Clinical variables did not predict either sexual function or reproductive concerns. CLINICAL IMPLICATIONS: Our results show that the majority of young men diagnosed with testicular cancer do not report sexual dysfunction or reproductive concerns 2 years after diagnosis. A sizeable minority, however, does report dysfunction or reproductive concerns, which should be recognized in the follow-up care of this population. STRENGTHS & LIMITATIONS: A strength of the study is the use of high-quality registry data and validated instruments. The lack of Swedish norms for sexual function and reproductive concerns is a possible limitation. CONCLUSION: A subgroup of young men treated for testicular cancer report sexual dysfunction or reproductive concerns approximately 2 years after diagnosis. Factors associated with these issues seem to mainly be psychological, rather than medical, nature. Ljungman L, Eriksson LE, Flynn KE, et al. Sexual Dysfunction and Reproductive Concerns in Young Men Diagnosed With Testicular Cancer: An Observational Study. J Sex Med 2019;16:1049-1059

Consistency of Financial Interest Disclosures in the Biomedical Literature: The Case of Coronary Stents

Background Disclosure of authors' financial interests has been proposed as a strategy for protecting the integrity of the biomedical literature. We examined whether authors' financial interests were disclosed consistently in articles on coronary stents published in 2006. Methodology/Principal Findings We searched PubMed for English-language articles published in 2006 that provided evidence or guidance regarding the use of coronary artery stents. We recorded article characteristics, including information about authors' financial disclosures. The main outcome measures were the prevalence, nature, and consistency of financial disclosures. There were 746 articles, 2985 authors, and 135 journals in the database. Eighty-three percent of the articles did not contain disclosure statements for any author (including declarations of no interests). Only 6% of authors had an article with a disclosure statement. In comparisons between articles by the same author, the types of disagreement were as follows: no disclosure statements vs declarations of no interests (64%); specific disclosures vs no disclosure statements (34%); and specific disclosures vs declarations of no interests (2%). Among the 75 authors who disclosed at least 1 relationship with an organization, there were 2 cases (3%) in which the organization was disclosed in every article the author wrote. Conclusions/Significance In the rare instances when financial interests were disclosed, they were not disclosed consistently, suggesting that there are problems with transparency in an area of the literature that has important implications for patient care. Our findings suggest that the inconsistencies we observed are due to both the policies of journals and the behavior of some authors

The hospital burden of disease associated with bone metastases and skeletal-related events in patients with breast cancer, lung cancer, or prostate cancer in Spain

POCKETT R.D., CASTELLANO D., MCEWAN P., OGLESBY A., BARBER B.L. & CHUNG K. (2010) European Journal of Cancer Care19, 755–760 The hospital burden of disease associated with bone metastases and skeletal-related events in patients with breast cancer, lung cancer, or prostate cancer in Spai

Pain outcomes in patients with bone metastases from advanced cancer: assessment and management with bone-targeting agents

Bone metastases in advanced cancer frequently cause painful complications that impair patient physical activity and negatively affect quality of life. Pain is often underreported and poorly managed in these patients. The most commonly used pain assessment instruments are visual analogue scales, a single-item measure, and the Brief Pain Inventory Questionnaire-Short Form. The World Health Organization analgesic ladder and the Analgesic Quantification Algorithm are used to evaluate analgesic use. Bone-targeting agents, such as denosumab or bisphosphonates, prevent skeletal complications (i.e., radiation to bone, pathologic fractures, surgery to bone, and spinal cord compression) and can also improve pain outcomes in patients with metastatic bone disease. We have reviewed pain outcomes and analgesic use and reported pain data from an integrated analysis of randomized controlled studies of denosumab versus the bisphosphonate zoledronic acid (ZA) in patients with bone metastases from advanced solid tumors. Intravenous bisphosphonates improved pain outcomes in patients with bone metastases from solid tumors. Compared with ZA, denosumab further prevented pain worsening and delayed the need for treatment with strong opioids. In patients with no or mild pain at baseline, denosumab reduced the risk of increasing pain severity and delayed pain worsening along with the time to increased pain interference compared with ZA, suggesting that use of denosumab (with appropriate calcium and vitamin D supplementation) before patients develop bone pain may improve outcomes. These data also support the use of validated pain assessments to optimize treatment and reduce the burden of pain associated with metastatic bone disease

Consulting Communities When Patients Cannot Consent: A Multi-Center Study of Community Consultation for Research in Emergency Settings

OBJECTIVE: To assess the range of responses to community consultation efforts conducted within a large network and the impact of different consultation methods on acceptance of exception from informed consent (EFIC) research and understanding of the proposed study. DESIGN: A cognitively pre-tested survey instrument was administered to 2,612 community consultation participants at 12 US centers participating in a multi-center trial of treatment for acute traumatic brain injury (TBI). SETTING: Survey nested within community consultation for a Phase III, randomized controlled trial of treatment for acute TBI conducted within a multi-center trial network and using EFIC. SUBJECTS: Adult participants in community consultation events. INTERVENTIONS: Community consultation efforts at participating sites. MEASUREMENTS AND MAIN RESULTS: Acceptance of EFIC in general, attitude toward personal EFIC enrollment, and understanding of the study content were assessed. 54% of participants agreed EFIC was acceptable in the proposed study; 71% were accepting of personal EFIC enrollment. Participants in interactive versus non-interactive community consultation events were more accepting of EFIC in general (63% vs. 49%) and personal EFIC inclusion (77% vs. 67%). Interactive community consultation participants had high-level recall of study content significantly more often than non-interactive consultation participants (77% vs. 67%). Participants of interactive consultation were more likely to recall possible study benefits (61% vs. 45%) but less likely to recall potential risks (56% vs. 69%). CONCLUSIONS: Interactive community consultation methods were associated with increased acceptance of EFIC and greater overall recall of study information but lower recall of risks. There was also significant variability in EFIC acceptance among different interactive consultation events. These findings have important implications for IRBs and investigators conducting EFIC research and for community engagement efforts in research more generally

Sexual Satisfaction and the Importance of Sexual Health to Quality of Life Throughout the Life Course of U.S. Adults

Discussions about sexual health are uncommon in clinical encounters, despite the sexual dysfunction associated with many common health conditions. Understanding of the importance of sexual health and sexual satisfaction among US adults is limited