Progressive Supranuclear palsy (PSP) disease progression, management, and healthcare resource utilization: a retrospective observational study in the US and Canada.

BACKGROUND: Progressive supranuclear palsy (PSP) is a rare neurodegenerative brain disease with rapid progression and currently limited treatment options. A comprehensive understanding of disease progression, management, and healthcare resource utilization is limited, and further research is challenging due to the small population of patients. To address these challenges in conducting PSP research, individuals with PSP were recruited using a multichannel approach tailored specifically to the PSP community. We performed a retrospective observational study using data abstracted from participant medical records collected from multiple patient care centers. RESULTS: Seventy-two individuals with PSP were eligible for inclusion. On average, 144 medical documents per participant were collected from an average of 2.9 healthcare centers per participant, with a mean study period of 7.9 years. Among participants with a date of symptom onset documented in the medical records, the median time for the onset of the first fall was 2.0 years (IQR 3.2) before diagnosis, the median onset of unsteady gait or gait impairment was 1.2 years (IQR 1.8) before diagnosis, and the median onset of mobility problems was 0.8 years (IQR 1.8) before diagnosis. The most widely utilized healthcare resources, with at least 85% of participants using each of these resources at some point during the disease course, were medications (100%), imaging (99%), assistive devices (90%), supportive care (86%), and surgeries and procedures (85%). CONCLUSIONS: This retrospective study adds to the current understanding of PSP symptoms, comorbidities, and healthcare resource utilization (HRU) across the disease journey. By involving individuals with PSP and their caregivers or legally authorized representatives in the research process, this study was unique in its approach to participant recruitment and enabled individuals to participate in research without the need for travel. We collected medical documents from multiple healthcare centers, allowing for broad data collection covering the entire disease journey. This approach to the collection of real-world data may be used to generate valuable insights into many aspects of disease progression and management in PSP and many other rare diseases.

The association between blood metals and hypertension in the GuLF study.

BACKGROUND: Both essential and non-essential metals come from natural and anthropogenic sources. Metals can bioaccumulate in humans and may impact human health, including hypertension. METHODS: Blood metal (cadmium, lead, mercury, manganese, and selenium) concentrations were measured at baseline for a sample of participants in the Gulf Long-Term Follow-up (GuLF) Study. The GuLF Study is a prospective cohort study focused on potential health effects following the 2010 Deepwater Horizon oil spill. Hypertension was defined as high systolic (≥140 mm Hg) or diastolic (≥90 mm Hg) blood pressure or taking anti-hypertensive medications. A total of 957 participants who had blood measurement for at least one metal, baseline blood pressure measurements, information on any anti-hypertensive medication use, and relevant covariates were included in this cross-sectional analysis. We used Poisson regression to explore the association between individual blood metal levels and hypertension. Quantile-based g-computation was used to investigate the association between the metal mixture and hypertension. We also explored the association between individual blood metal levels and continuous blood pressure measurements using general linear regression. RESULTS: Comparing the highest quartile of blood metals with the lowest (Q4vs1), the hypertension prevalence ratio (PR) was 0.92 (95 % confidence interval (CI) = 0.73,1.15) for cadmium, 0.86 (95%CI = 0.66,1.12) for lead, 0.89 (95%CI = 0.71,1.12) for mercury, 1.00 (95%CI = 0.80,1.26) for selenium, and 1.22 (95%CI = 0.95,1.57) for manganese. We observed some qualitative differences across race and BMI strata although none of these differences were statistically significant. In stratified analyses, the PR (Q4vs1) for mercury was 0.69 (95%CI = 0.53, 0.91) in White participants and 1.29 (95%CI = 0.86,1.92) in Black participants (p for interaction = 0.5). The PR (Q4vs1) for manganese was relatively higher in Black participants (PR = 1.37, 95%CI = 0.92,2.05) than in White participants (PR = 1.15, 95%CI = 0.83,1.60, p for interaction = 0.5), with a suggestive dose-response among Blacks. After stratifying by obesity (BMI ≥30 and < 30), positive associations of of hypertension with cadmium (PR [Q4vs1] = 1.19, 95%CI = 0.91,1.56, p for interaction = 0.5), lead (PR [Q4vs1] = 1.14, 95%CI = 0.84,1.55, p for interaction = 1.0) and manganese (PR = 1.25, 95%CI = 0.93,1.68, p for interaction = 0.8) were observed in participants with BMI≥30, but not in participants with BMI<30. The joint effect of the metal mixture was 0.96 (95%CI = 0.73,1.27). We did not observe clear associations between blood metal levels and continuous blood pressure measurements. CONCLUSION: We did not find overall cross-sectional associations between blood cadmium, lead, mercury, selenium levels and hypertension or blood pressure. We found some evidence suggesting that manganese might be positively associated with risk of hypertension. Associations varied somewhat by race and BMI.