Published Studies in PAH

Study design

• Updated guidelines based on close review of published data, including new values and severity gradings, with emphasis on right heart health as it relates to PVD at risk for PH
• Replaces previous 2010 ASE right heart guidelines

Key insights

• Describes how the right heart adapts to PVD, particularly functional and structural changes because of increased afterload
• Defines acoustic windows and imaging views and parameters essential for right heart evaluation, including reference values for normal and abnormal, while recommending TV, PV, and PH disease considerations
• Standardizes right heart imaging and reporting for clinicians
• Emphasizes the crucial role of Echo in screening, detection, monitoring of treatment, and prognosis of PH

Study design

• Retrospective review of initial cohort of 96 patients with PH who received a RHC and Echo within 1 year of each other and validation cohort of 30 patients with PH
• Patients were scored on presence/absence of VEST parameters, including mitral E:e’, qualitative LA size, and presence of systolic intraventricular septal flattening

Key insights

• Positive VEST scores were both sensitive (80.0%) and specific (75.6%) for presence of PAH, separating potential PAH from left heart disease; a +3 score was highly specific (92.7%) and predicted PAH at 88.0%
• Information from standard Echo reports applied to VEST scoring supported consistent clear screening of patients with potential for PAH, allowing for a timely referral to PH Expert Centers and precise use of invasive RHCs for patients with greater potential for having PAH

Study design

• Analysis of 1655 patients from the COMPERA database to evaluate a modified risk stratification using 4-strata model (COMPERA 2.0) with refined cutoff levels for FC, 6MWD, and BNP/NT-pro-BNP
• Patients selected in this study were newly diagnosed with any form of PAH and had at least 1 follow-up assessment available

Key insights

• Most patients were categorized as intermediate risk with the 3-strata model; this group was further divided into intermediate-low and intermediate-high risk groups according to the 4-strata model
• Changes in risk from baseline to first follow-up occurred in 49.2% of patients categorized by the 4-strata model compared with 31.1% with the 3-strata model
• A 4-strata risk model yielded more nuanced separation and was able to predict differentiated long-term survival compared with the 3-strata model

Study design

• Retrospective analysis of prospectively collected data from 2879 patients with Group 1 PAH who were enrolled in the French PAH Registry between 2009 and 2020 to validate a 4-strata risk assessment approach
• Patients were categorized as low, intermediate-low, intermediate-high, or high risk per COMPERA Registry investigators

Key insights

• The 4-strata approach performed better than a 3-strata method for predicting mortality
• Applying the 4-strata approach, 53% of patients changed risk category from baseline compared with 39% of patients when using the 3-strata approach
• In comparison with the 3-strata risk assessment, the 4-strata approach was more sensitive to change, refined risk prediction, and had better survival predictions

Study design

• Literature review on the use of Echo parameters in PAH and their current and future roles in determining patient prognosis

Key insights

• Echo-derived parameters show how a patient’s RV is adapting to increased afterload and can indicate disease progression before worsening symptoms or 6MWD
• Many Echo parameters have proven to be prognostic in PAH, including right heart reverse remodeling, a recently identified key parameter in evaluating a patient’s response to PAH therapy
• Adding Echo parameters to current risk assessment tools could allow for improved prognostication and optimized therapeutic management

Study design

• Retrospective chart analysis of 153 FC II patients on mono- or dual therapy (ERA/PDE-5i)
• PAH-experienced physicians were surveyed for their gestalt assessment of risk; then risk was calculated independently using COMPERA, the French Noninvasive method, and REVEAL 2.0

Key insights

• More than half of FC II patients were classified as intermediate- or high-risk status when risk was formally calculated
• Factors that led to incongruent risk assessments included less frequent Echos at follow-up, higher physician-reported patient activity level, and HCP-reported patient symptomatic stability