Background Pulmonary vascular (PV) distensibility, defined as the percent increase in pulmonary vessel diameter per mmHg increase in pressure, permits the pulmonary arteries to increase in size to accommodate increased blood flow. We hypothesized that PV distensibility is abnormally low in patients with heart failure (HF) and serves as an important determinant of right ventricular performance and exercise capacity. Methods and Results Patients with HF and preserved ejection fraction (HFpEF, n=48), HF and reduced ejection fraction (HFrEF, n=55), pulmonary hypertension without left-heart failure (PAH, n=18), and control subjects (n=30) underwent cardiopulmonary exercise testing with invasive hemodynamic monitoring and first-pass radionuclide ventriculography. PV distensibility was derived from 1257 matched measurements (mean±SD, 8±2 per subject) of PA pressure, PA wedge pressure and cardiac output. PV distensibility was lowest in the PAH group (0.40±0.24% per mmHg) and intermediate in the HFpEF and HFrEF groups (0.92±0.39 and 0.84±0.33% per mmHg, respectively) compared to the control group (1.39±0.32% per mmHg, P<0.0001 for all three). PV distensibility was associated with change in RVEF (ρ=0.39, P<0.0001) with exercise and was an independent predictor of peak VO2. PV distensibility also predicted cardiovascular mortality independent of peak VO2 in HF patients (n=103, Cox HR 0.30, 95% CI 0.10–0.93, P=0.036). In a subset of HFrEF patients (n=26), 12 weeks of treatment with the pulmonary vasodilator sildenafil or placebo led to a 24.6% increase in PV distensibility (P=0.015) in the sildenafil group only. Conclusions PV distensibility is reduced in patients with HF and PAH and is closely related to RV systolic function during exercise, maximal exercise capacity, and survival. Furthermore, PV distensibility is modifiable with selective pulmonary vasodilator therapy and may represent an important target for therapy in selected HF patients with pulmonary hypertension. Clinical Trial Registration URL: http://www.clinicaltrials.gov. Unique identifier: NCT00309790.

Aim: This research was conducted to collect patient-reported data on the experience of living with Wilson disease and to broaden the existing knowledge of a rare neurometabolic disease with varied clinical manifestations. Materials & methods: Adult patients with Wilson disease or caregivers were recruited through a Wilson disease association or advocacy group, and asked to complete an online survey that assessed various aspects of living with Wilson disease. Survey data were analyzed descriptively. Results: 21 adults with Wilson disease completed the survey. Respondents reported experiencing signs, symptoms and diagnoses related to movement (e.g., involuntary muscle contractions [n = 9, 42.9%]), cognition (e.g., anxiety [n = 15, 71.4%]) and liver problems. Respondents most frequently reported medication regimen and financial burden as the most bothersome impacts of Wilson disease. Conclusion: The data expand the existing knowledge of this rare neurometabolic disease with heterogeneous clinical manifestations.

PRO instrument and the only instrument identified from all sources: clinical trials (n=30/971), HTA reports (n=11/13), published literature (n=8/29), and PROQOLID/PROLABELS (n=2/2). Use of other instruments in the literature and clinical trials was fragmented and limited to few sources. Many identified instruments measured immediate impacts associated with dry AMD (eg, dependency on others; poor spatial perception and mobility; difficulty reading, driving, and completing activities of daily living). However, no identified instrument adequately covered symptoms of dry AMD (eg, blurred vision, restricted visual fields). Instruments were also evaluated for their content validity and psychometrics. The PRO instruments with the strongest properties in the AMD population were the NEI-VFQ-25, Low Luminance Questionnaire, Macular Disease-Dependent Quality of Life, and Impact of Vision Impairment-Very Low Vision. Conclusions: These findings show that currently available/published instruments adequately measure disease impacts, but have limited inclusion of dry AMD symptoms. To fully capture the experience of patients with dry AMD, future clinical outcome assessment measures should include both symptoms and impacts.

The aim of the present study was to determine the effect of different pretest pedaling cadences on power outcomes obtained during the Wingate Anaerobic Test (WAnT). Vigorously exercising adult men (n = 14, 24.9 ± 1.2 years) and women (n = 14, 20.4 ± 0.6 years) participated in a randomized crossover study during which they performed the 30-second WAnT on a mechanically braked cycle ergometer (0.075 kg·kg(-1) body weight) under 2 conditions. Participants pedaled maximally with an unloaded flywheel during 5 seconds before resistance was applied and the test began (FAST). In another trial, participants maintained a moderate cadence (80 revolutions per minute [rpm]) during 5 seconds before the test began (MOD). All other components of the WAnT were identical. Peak power (PP), mean power (MP), minimum power (MinP), fatigue index (%FAT), and maximum cadence during test were recorded. Comparisons were made using a 2 × 2 factorial repeated-measures analysis of variance. Regardless of gender, the FAST condition resulted in 22.2% lower PP (612.6 ± 33.0 W vs. 788.3 ± 43.5 W), 13.3% lower MP (448.4 ± 22.2 W vs. 517.2 ± 26.4 W), 11.7% lower MinP (280.9 ± 14.8 W vs. 318.3 ± 17.2 W), and 9.0% lower %FAT (53.5 ± 1.3% vs. 58.8 ± 1.5%) than MOD condition (p < 0.01; mean ± SD). Similar outcomes were observed within gender. The authors conclude that practitioners of the WAnT should instruct participants to maintain a moderate pedal cadence (∼80 rpm) during 5 seconds before the test commences to avoid bias from software sampling and peripheral fatigue. Standardizing the pretest pedal cadence will be important to exercise testing professionals who compare data with norms or generate norms for specific populations.