Development of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT®) questionnaire: a new patient-reported outcome instrument for PAH

Abstract

BackgroundRegulators and clinical experts increasingly recognize the importance of incorporating patient-reported outcomes (PROs) in clinical studies of therapies for pulmonary arterial hypertension (PAH). No PAH-specific instruments have been developed to date in accordance with the 2009 FDA guidance for the development of PROs as endpoints in clinical trials. A qualitative research study was conducted to develop a new instrument assessing PAH symptoms and their impacts following the FDA PRO guidance.MethodsA cross-sectional study was conducted at 5 centers in the US in symptomatic PAH patients aged 18–80 years. Concept elicitation was based on 5 focus group discussions, after which saturation of emergent concepts was reached. A PRO instrument for PAH symptoms and their impacts was drafted. To assess the appropriateness of items, instructions, response options, and recall periods, 2 rounds of one-on-one cognitive interviews were conducted, with instrument revisions following each round. Additional interviews tested the usability of an electronic version (ePRO). PRO development considered input from an international Steering Committee, and translatability and lexibility assessments.ResultsFocus groups comprised 25 patients (5 per group); 20 additional patients participated in cognitive interviews (10 per round); and 10 participated in usability interviews. Participants had a mean ± SD age of 53.1 ± 15.8 years, were predominantly female (93 %), and were diverse in race/ethnicity, WHO functional class (FC I/II: 56 %, III/IV: 44 %), and PAH etiology (idiopathic: 56 %, familial: 2 %, associated: 42 %). The draft PRO instrument (PAH-SYMPACT®) was found to be clear, comprehensive, and relevant to PAH patients in cognitive interviews. Items were organized in a draft conceptual framework with 16 symptom items in 4 domains (respiratory symptoms, tiredness, cardiovascular symptoms, other symptoms) and 25 impact items in 5 domains (physical activities, daily activities, social impact, cognition, emotional impact). The recall period is the past 24 h for symptoms, and the past 7 days for impacts.ConclusionsThe PAH-SYMPACT® was shown to capture symptoms and their impacts relevant to PAH patients, demonstrating content saturation, concept validity, and ePRO usability. Final content and psychometric validation of the instrument will be based on the results of an ongoing Phase IIIb clinical trial in PAH patients.Electronic supplementary materialThe online version of this article (doi:10.1186/s12931-016-0388-6) contains supplementary material, which is available to authorized users.

Combining creative writing and narrative analysis to deliver new insights into the impact of pulmonary hypertension

“…However, the delivery of good clinical care is often centred in profoundly personal interactions that change outcomes for individuals very powerfully. 2 Quality of life can be assessed by clinically relevant and applicable scoring systems [3][4][5] and correlates with outcomes. 6 Study of these qualitative aspects of healthcare, though central to clinical practice, is rarely accorded high impact, perhaps both because of its perceived immediate failure to generate a new therapeutic target, and also in the difficulty there is in precisely quantifying the impact that it has.…”

Psychometric Validation of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Questionnaire

“…With the objective of developing a PRO measure that can be used both in clinical trials and in clinical practice, a draft version of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) questionnaire was developed based on qualitative interviews with patients with PAH. 13 The development of this questionnaire-which consists of two parts (one assessing PAH symptoms and the other assessing impacts)-has previously been described (Fig 1). 13 Here, we report results of the psychometric analyses performed to validate the PAH-SYMPACT based on A Study of Macitentan in Pulmonary Arterial Hypertension to Validate the PAH-SYMPACT (SYMPHONY).…”

“…13 The development of this questionnaire-which consists of two parts (one assessing PAH symptoms and the other assessing impacts)-has previously been described (Fig 1). 13 Here, we report results of the psychometric analyses performed to validate the PAH-SYMPACT based on A Study of Macitentan in Pulmonary Arterial Hypertension to Validate the PAH-SYMPACT (SYMPHONY). This psychometric validation study included patients with PAH who received macitentan 10 mg, an endothelin receptor antagonist approved for the treatment of PAH, 14,15 to enable change in symptoms and impacts.…”

Palliative care in pulmonary arterial hypertension: an underutilised treatment

“…The overall HRQoL is measured by assessing a variety of indicators, including functional status, physical, emotional, social and spiritual wellbeing. The Pulmonary Arterial Hypertension -Symptoms and Impact (PAH-SYMPACT) questionnaire assesses disease-specific patient-reported outcomes and has recently been validated for use in PAH patients [23,24]. This questionnaire assesses the symptoms of PAH in the previous 24 h and the impact PAH had on patients' lives in the preceding 7 days.…”