Pulmonary Arterial Hypertension—Symptoms and Impact Questionnaire: feasibility of utilizing one‐day versus seven‐day symptom reporting

Abstract

Patient-reported outcomes are important measures to include in pulmonary arterial hypertension clinical trials but are not widely utilized in clinical practice. Pulmonary Arterial Hypertension—Symptoms and Impact Questionnaire (PAH-SYMPACT) is the only pulmonary arterial hypertension-specific patient-reported outcomes instrument developed and validated in accordance with the US Food and Drug Administration guidance on patient-reported outcomes development. The PAH-SYMPACT tool measures pulmonary arterial hypertension-related symptoms and impact of pulmonary arterial hypertension on daily life. Symptoms are reported each day for seven consecutive days, and the impact of pulmonary arterial hypertension over one week is recalled and reported on day 7; however, daily symptom reporting may overburden patients and healthcare resources, limiting the practicality of PAH-SYMPACT outside of clinical trials. To determine the practicability of an abridged version of PAH-SYMPACT for which all reporting is completed on one day, symptom data from the SYMPHONY trial (NCT01841762; PAH-SYMPACT validation study) were retrospectively analyzed to assess whether symptoms reported on each day correlated with the weekly average and whether one-day symptom scores were sensitive to disease severity. Correlation coefficients comparing the weekly average and individual day symptom scores were mostly high or very high regardless of the day they were measured. Findings were similar when using either Spearman's rank correlation or weighted kappa method. One-day symptom scores differentiated well between World Health Organization functional classes II and III/IV pulmonary arterial hypertension and were sensitive to change in disease severity as measured by the Patient Global Assessment of Disease Severity. These data suggest that the one-day PAH-SYMPACT is feasible and appropriate for routine implementation in clinical practice.

The feasibility and value of assessing patient‐reported outcomes in pulmonary arterial hypertension

“…For practical use in the clinical setting, patients completed the 1‐day version of the PAH‐SYMPACT questionnaire. 10 The PAH‐SYMPACT questionnaire, licensed for study use by Mapi research trust, consists of 22 Likert‐scale questions that assess HRQOL across four domains: CV symptoms, CP symptoms, PI, and CE impact. The score of each question within a domain is added and then divided by the total number of questions within the domain to provide a mean domain score.…”

“…Patients were preferentially asked to complete the questionnaires before discussion of clinical test results when feasible. For practical use in the clinical setting, patients completed the 1‐day version of the PAH‐SYMPACT questionnaire 10 . The PAH‐SYMPACT questionnaire, licensed for study use by Mapi research trust, consists of 22 Likert‐scale questions that assess HRQOL across four domains: CV symptoms, CP symptoms, PI, and CE impact.…”

“… 8 , 9 PAH‐SYMPACT assesses HRQOL across four domains: cardiopulmonary (CP) symptoms, cardiovascular (CV) symptoms, physical impact (PI), and cognitive and emotional (CE) impact. 9 , 10 Importantly, the instrument is sensitive to change 9 and thus can detect the effect of specific interventions on HRQOL. Although the PAH‐SYMPACT tool is a validated disease‐specific instrument, there is limited literature regarding its use outside of clinical trials.…”

“…The PAH‐symptoms and impact (PAH‐SYMPACT) questionnaire is a PAH disease‐specific PRO instrument that was recently developed and validated in accordance with Food and Drug Administration guidelines to assess the impact of PAH and its treatment on an individual's HRQOL 8,9 . PAH‐SYMPACT assesses HRQOL across four domains: cardiopulmonary (CP) symptoms, cardiovascular (CV) symptoms, physical impact (PI), and cognitive and emotional (CE) impact 9,10 . Importantly, the instrument is sensitive to change 9 and thus can detect the effect of specific interventions on HRQOL.…”

Clinical application of risk assessment in PAH: Expert center APRN recommendations

“…Many of these devices capture the biometric data needed for risk assessments. 39 , 40 Consumer‐based accelerometers for 6MWD, 41 recently validated patient‐assessed functional class questionnaires, 42 blood pressure cuffs, 43 , 44 and satellite laboratory companies for assessing B‐type natriuretic peptide or N‐terminal prohormone brain natriuretic peptide can also be incorporated to facilitate telehealth care. Conversely, the lack of technological devices remains a limitation for a large percentage of the population, adding to the digital access divide and this limitation must be considered in PH program risk assessment.…”