Differences in the Presentation and Progression of Parkinson's Disease by Sex.

BACKGROUND: Previous studies reported various symptoms of Parkinson's disease (PD) associated with sex. Some were conflicting or confirmed in only one study. OBJECTIVES: We examined sex associations to PD phenotypes cross-sectionally and longitudinally in large-scale data. METHODS: We tested 40 clinical phenotypes, using longitudinal, clinic-based patient cohorts, consisting of 5946 patients, with a median follow-up of 3.1 years. For continuous outcomes, we used linear regressions at baseline to test sex-associated differences in presentation, and linear mixed-effects models to test sex-associated differences in progression. For binomial outcomes, we used logistic regression models at baseline and Cox regression models for survival analyses. We adjusted for age, disease duration, and medication use. In the secondary analyses, data from 17 719 PD patients and 7588 non-PD participants from an online-only, self-assessment PD cohort were cross-sectionally evaluated to determine whether the sex-associated differences identified in the primary analyses were consistent and unique to PD. RESULTS: Female PD patients had a higher risk of developing dyskinesia early during the follow-up period, with a slower progression in activities of daily living difficulties, and a lower risk of developing cognitive impairments compared with male patients. The findings in the longitudinal, clinic-based cohorts were mostly consistent with the results of the online-only cohort. CONCLUSIONS: We observed sex-associated contributions to PD heterogeneity. These results highlight the necessity of future research to determine the underlying mechanisms and importance of personalized clinical management. © 2020 International Parkinson and Movement Disorder Society.

Impact of Remote Symptom Monitoring With Electronic Patient-Reported Outcomes on Hospitalization, Survival, and Cost in Community Oncology Practice: The Texas Two-Step Study.

PURPOSE: There is raising interest to implement electronic patient-reported outcomes (ePROs) for symptom monitoring to enhance the quality of cancer care. Step 1 of the Texas Two-Step Study demonstrated successful implementation of an ePRO system in >200 sites of service of a large community oncology practice. We now report step 2 of this study which evaluates the impact of ePROs on outcomes among patients enrolled in the Centers for Medicare & Medicaid Services' Oncology Care Model (OCM) program. METHODS: This observational study focused on patients with metastatic cancer enrolled in OCM at large community oncology practice located in Texas between July 2020 and December 2020. Patients who completed ≥1 survey via the ePRO tool were included in the study group and were propensity score matched with patients in a control group. Adverse events (AEs; hospitalizations, emergency department visits, deaths) and total cost of care were a priori study outcomes. Mann-Whitney U and chi-square tests compared continuous and categorical variables, respectively, with multivariable logistic regression for adjustment of covariates. RESULTS: Of 831 patients with metastatic cancer, 458 matched patients (229/group) were identified, with 52% male and a mean age of 74 years. Mean total AEs were lower in the study group compared with control (0.98 v 1.41; P = .007), with decreased hospitalizations (20% v 32.5%; P = .002), emergency visits (38.4% v 42.3%; P > .05), and deaths (11.8% v 16.6%; P > .05). Average number of hospitalizations was lower (0.28 v 0.52; P = .003) with reduced mean duration of hospitalizations (1.9 vs 3.2 d; P = .03). The total cost of care was reduced by an average of $1,146 per member per month. CONCLUSION: Symptom monitoring with ePROs improved quality and value of cancer care delivery by reducing hospitalizations, emergency visits, and deaths while lowering cost of care in a large oncology practice.

Differences in Receipt of Immunotherapy Treatment Among Patients With Head and Neck Cancer.

Importance: The US Food and Drug Administration approved immune checkpoint inhibitors (immunotherapy) for select cases of head and neck squamous cell carcinoma (HNSCC) in 2016. However, it is unclear whether there are clinical or sociodemographic differences among patients receiving immunotherapy as part of their care. Given the known disparities in head and neck cancer care, we hypothesized that there are differences in receipt of immunotherapy among patients with HNSCC based on clinical and nonclinical characteristics. Objective: To characterize clinical and nonclinical factors associated with receipt of immunotherapy among older patients with HNSCC. Design, Setting, and Participants: This retrospective cohort study included patients 65 years or older diagnosed with HNSCC (n = 4860) in a community oncology care setting. Electronic health records from Navigating Cancer were assessed from January 1, 2017, to April 30, 2022. Main Outcomes and Measures: Multivariable logistic regression was used to characterize clinical (tumor stage [localized vs advanced] and anatomical subsite [oropharyngeal vs nonoropharyngeal]) and nonclinical (age, smoking history, race and ethnicity, sex, and marital status) factors associated with receipt of immunotherapy. Results: In the study cohort of 4860 patients, 3593 (73.9%) were men; 4230 (87.0%) were White and 630 (13.0%) were of other races. A total of 552 patients (11.4%) had received immunotherapy. After adjusting for covariates, in the final model, White patients with HNSCC had 80% increased odds of receiving immunotherapy (adjusted odds ratio [AOR], 1.80 [95% CI, 1.30-2.48]) compared with patients of other races. There were no statistically significant differences in the odds of receiving immunotherapy based on age, sex, or smoking history. Patients with nonoropharyngeal disease were significantly more likely to receive immunotherapy than those with oropharyngeal cancer (AOR, 1.29 [95% CI, 1.05-1.59]), as were those with advanced compared with local disease (AOR, 2.39 [95% CI, 1.71-3.34]). Conclusions and Relevance: The findings of this cohort study suggest that among older patients with HNSCC, White patients may be more likely to receive immunotherapy as part of their care. Equitable access to immunotherapy and other treatment options will reduce cancer-related health disparities and improve survival of patients with HNSCC.

Video-based Parkinson's disease assessments in a nationwide cohort of Fox Insight participants.

INTRODUCTION: Parkinson's disease (PD) research is hampered by slow, inefficient recruitment and burdensome in-person assessments that may be challenging to conduct in a world affected by COVID-19. Fox Insight is an ongoing prospective clinical research study that enables individuals to participate in clinical research from their own homes by completing online questionnaires. To date, over 45,000 participants with and without PD have enrolled. We sought to validate self-reported PD diagnosis in the Fox Insight cohort, assess the validity of other self-reported health information, and evaluate the willingness of participants to participate in video-based research studies. METHODS: Individuals with and without self-reported PD enrolled in Fox Insight were invited to participate in this virtual research study. Participants completed online questionnaires and two virtual visits, during which we conducted standard cognitive and motor assessments. A movement disorder expert determined the most likely diagnosis, which was compared to self-reported diagnosis. RESULTS: A total of 203 participants from 40 U.S. states, 159 with remote clinician-determined PD and 44 without, completed the study (59% male, mean (SD) age 65.7 (9.8)). Level of agreement between self-reported PD diagnosis in Fox Insight and clinician-determined diagnosis was very good ((kappa = 0.85, 95% CI 0.76-0.94). Overall, 97.9% of participants were satisfied with the study, 98.5% were willing to participate in a future observational study with virtual visits, and 76.1% were willing to participate in an interventional trial with virtual visits. CONCLUSION: Among the Fox Insight cohort, self-reported diagnosis is accurate and interest in virtual research studies is high.

Fox Insight collects online, longitudinal patient-reported outcomes and genetic data on Parkinson's disease.

Fox Insight is an online, longitudinal health study of people with and without Parkinson's disease with targeted enrollment set to at least 125,000 individuals. Fox Insight data is a rich data set facilitating discovery, validation, and reproducibility in Parkinson's disease research. The dataset is generated through routine longitudinal assessments (health and medical questionnaires evaluated at regular cycles), one-time questionnaires about environmental exposure and healthcare preferences, and genetic data collection. Qualified Researchers can explore, analyze, and download patient-reported outcomes (PROs) data and Parkinson's disease- related genetic variants at https://foxden.michaeljfox.org. The full Fox Insight genetic data set, including approximately 600,000 single nucleotide polymorphisms (SNPs), can be requested separately with institutional review and are described outside of this data descriptor.

Innovative Recruitment Strategies to Increase Diversity of Participation in Parkinson's Disease Research: The Fox Insight Cohort Experience.

BACKGROUND: Clinical research in Parkinson's disease (PD) faces practical and ethical challenges due to two interrelated problems: participant under-recruitment and lack of diversity. Fox Insight (FI) is a web-based longitudinal study collecting patient-reported outcomes and genetic data worldwide to inform therapeutic studies. FI's online platform provides an opportunity to evaluate online strategies for recruiting large, diverse research cohorts. OBJECTIVE: This project aimed to determine 1) whether FI's digital marketing was associated with increased enrollment overall and from under-represented patient groups, compared to traditional recruitment methods; 2) the clinical and demographic characteristics of samples recruited online, and 3) the cost of this online recruitment. METHOD: FI recruitment during a 6-week baseline period without digital promotion was compared to recruitment during several periods of digital outreach. Separate online recruiting intervals included general online study promotion and unique Facebook and Google ad campaigns targeting under-represented subgroups: early PD, late/advanced PD, and residents of underrepresented/rural geographic areas. RESULTS: Early PD, late PD, and geotargeting campaigns enrolled more individuals in their respective cohorts compared to baseline. All online campaigns also yielded greater total FI enrollment, attracting more participants who were non-White, Hispanic, older, female, and had lower educational attainment and income, and more medical comorbidities. Cost per new participant ranged from $21 (Facebook) to $108 (Google). CONCLUSION: Digital marketing may allow researchers to increase, accelerate, and diversify recruitment for PD clinical studies, by tailoring digital ads to target PD cohort characteristics.

Comparison of an Online-Only Parkinson's Disease Research Cohort to Cohorts Assessed In Person.

BACKGROUND: Online tools for data collection could be of value in patient-oriented research. The Fox Insight (FI) study collects data online from individuals with self-reported Parkinson's disease (PD). Comparing the FI cohort to other cohorts assessed through more traditional (in-person) observational research studies would inform the representativeness and utility of FI data. OBJECTIVE: To compare self-reported demographic characteristics, symptoms, medical history, and PD medication use of the FI PD cohort to other recent observational research study cohorts assessed with in-person visits. METHODS: The FI PD cohort (n = 12,654) was compared to 3 other cohorts, selected based on data accessibility and breadth of assessments: Parkinson's Progression Markers Initiative (PPMI; PD n = 422), Parkinson's Disease Biomarker Program (PDBP; n = 700), and PD participants in the LRRK2 consortium without LRRK2 mutations (n = 508). Demographics, motor and non-motor assessments, and medications were compared across cohorts. Where available, identical items on surveys and assessments were compared; otherwise, expert opinion was used to determine comparable definitions for a given variable. RESULTS: The proportion of females was significantly higher in FI (45.56%) compared to PPMI (34.36%) and PDBP (35.71%). The FI cohort had greater educational attainment as compared to all other cohorts. Overall, prevalence of difficulties with motor experiences of daily living and non-motor symptoms in the FI cohort was similar to other cohorts, with only a few significant differences that were generally small in magnitude. Missing data were rare for the FI cohort, except on a few variables. DISCUSSION: Patterns of responses to patient-reported assessments obtained online on the PD cohort of the FI study were similar to PD cohorts assessed in-person.