Published Studies in 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

• Review article on recommendations to overcome barriers for utilizing and implementing risk assessment tools routinely

Key Insights

• To overcome the challenge of time constraints for implementing risk assessments, use of technology-based solutions (ie, electronic alerts and reminders) is recommended and supporting staff members should be trained to add test results into a risk assessment tool
• Insufficient awareness and training of risk assessment is another barrier that could be resolved by training providers how to use these tools, educating patients on their results, increasing provider autonomy to allow them to use their clinical judgment to perform a risk assessment, and incorporating telehealth for follow-up visits with an abbreviated risk assessment

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 (RHRR), 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, and 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

Study Design

• Retrospective analysis of 181 treatment-naive patients initiated on ERA + PDE-5i therapy who received RHC at baseline and at 6-month follow-up

Key Insights

• Initial ERA + PDE-5i therapy decreased PVR by an average of 35%, and one-third of patients had a decrease in PVR <25%
• Low-risk status was achieved or maintained in only 35% of patients according to REVEAL 2.0 scores
• Patients who were able to achieve low-risk status had greater PVR reductions

Study Design

• Chart review of 90 PAH-experienced cardiologists and pulmonologists who managed ≥7 patients with PAH

Key Insights

• Clinical gestalt and formal risk calculations were compared for 365 patient charts
• When calculated, risk status aligned with gestalt in less than half (45%) the charts evaluated
• Of patients assessed to be low risk by gestalt, 80% were assigned to a higher risk category after formal risk assessment

Study Design

• In 2019, 121 treatment decision makers of the PHA providers’ network were surveyed on their use of risk assessment tools

Key Insights

• 59% of treatment decision makers reported using formal risk assessment tools
• Risk tools were most commonly used at the time of diagnosis (54%) and of worsening symptoms (42%)
• Fewer than 1 in 5 used formal risk tools at all of the following: the time of diagnosis, worsening symptoms, changing medications, repeat Echo, and repeat RHC