Potential Cardiovascular Events Avoided with Bempedoic Acid Plus Ezetimibe Fixed-Dose Combination Compared with Ezetimibe Alone in Patients with Atherosclerotic Cardiovascular Disease Taking Maximally Tolerated Statins.

BACKGROUND: Patients with atherosclerotic cardiovascular disease who require additional low-density lipoprotein cholesterol (LDL-C) lowering despite maximally tolerated statins have a significant unmet medical need and are at increased risk of future cardiovascular events and a reduced quality of life. OBJECTIVE: We aimed to estimate the percentage of cardiovascular events avoided following treatment with a fixed-dose combination of bempedoic acid plus ezetimibe (BA+EZE FDC) versus ezetimibe (EZE) in patients with atherosclerotic cardiovascular disease receiving maximally tolerated statins across a range of baseline LDL-C levels. METHODS: A Markov cohort simulation model estimated major adverse cardiovascular events avoided over a lifetime horizon among patients with atherosclerotic cardiovascular disease and baseline LDL-C levels from 80 to >200 mg/dL. BA+EZE FDC was compared with EZE based on mean percent LDL-C reductions versus placebo reported in a phase III trial. Health outcomes for the average patient were extrapolated to a US population of 100,000 persons using evidence on contemporary LDL-C levels from the National Health and Nutrition Examination Survey. RESULTS: Among patients with atherosclerotic cardiovascular disease not at the LDL-C goal with maximally tolerated statins, the addition of BA+EZE FDC compared with the addition of EZE was predicted to provide incremental absolute reductions in major adverse cardiovascular events dependent on baseline LDL-C levels at the population level. For those with baseline LDL-C of 101-110 mg/dL (n = 15,237), there were 4.9% (744) fewer events predicted, while for patients with baseline LDL-C of > 200 mg/dL (n = 1689), 10.9% (184) fewer events were predicted through the addition of BA+EZE FDC versus EZE. CONCLUSIONS: Further LDL-C reductions through the addition of BA+EZE FDC to maximally tolerated statins are predicted to reduce major adverse cardiovascular events compared with the addition of EZE. Benefits are potentially greater among those with higher starting LDL-C.

Development of the Impact of Nighttime Urination (INTU) questionnaire to assess the impact of nocturia on health and functioning.

AIMS: This study describes development of the Impact of Nighttime Urination (INTU) questionnaire to assess nocturia impacts on health and functioning. METHODS: Development of the questionnaire followed an iterative patient-directed process as recommended by current guidance for patient-reported outcome (PRO) measures. An initial 15-item questionnaire was devised based on reviewing the published literature, and then modified through four rounds of semi-structured interviews of 28 individuals with nocturia. In each round, open-ended concept elicitation, followed by cognitive debriefing, was used to assess the questionnaire. Items were modified based on participants' responses and incorporated into the next round of interviews. RESULTS: In all rounds, participants reported that their experiences were easy to recall and report on a daily basis and that the burden of completing the questionnaire was low. The final questionnaire has a same-day recall period. It includes six daytime impact items-having limited concentration, a sense of feeling tired, difficulty getting things done, irritability, not feeling rested, and drowsiness-and four items that measure the nighttime impact of nocturia-patient concern, waking up too early, difficulty getting enough sleep, and feeling bothered by having to get up at night to void. Responses follow a 5- or 4-point scale. The final INTU captures the key concepts associated with nocturia as confirmed by cognitive debriefing. CONCLUSIONS: Development of the 10-item INTU, a nocturia-specific PRO measure, was based on direct input and feedback from patients and has demonstrated that it captures the patient-reported impacts of nocturia.