Researchers from the U.S., Germany, Switzerland, and the U.K. have developed a new questionnaire, to help assess symptoms of pulmonary arterial hypertension (PAH) and the disease’s impact on patients’ lives.
The questionnaire, called PAH-SYMPACT, was created following guidance from the U.S. Food and Drug Administration (FDA) to include patient reported outcomes as secondary endpoints in clinical trials.
The questionnaire could be a practical and convenient tool to identify PAH symptoms and its impact, according to authors of the study “Development of the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT®) questionnaire: a new patient-reported outcome instrument for PAH,” published in Respiratory Research.
Researchers conducted the study at five U.S. centers between 2011 and 2103. The three-phase study included 55 PAH patients 18-80 years old.
In the first phase, 25 participants discussed symptoms and the impact of the condition on daily lives; 16 symptoms and several impacts were selected for inclusion in the draft questionnaire.
Common symptoms included:
- shortness of breath
- weakness/lack of energy
- swelling (in ankles/legs/hands)
- rapid heartbeat/heart fluttering
- chest pain/tightness
- fainting/passing out
Common impacts included:
- taking stairs
- doing exercise
- walking slowly or with difficulty
- doing housework
- running errands
- sleeping difficulties
- needing help from others
- effects on work or school
In the second phase, researchers interviewed ten new participants and asked them to assess the questionnaire. Participants generally found the questionnaire to be clear, comprehensive, and relevant. Minor adjustments were made following comments from the phase 2 participants.
In the third phase, researchers interviewed ten new participants face to face to confirm the validity of questionnaire content. Ten other participants also tested an electronic version for usability. All participants found it easy and convenient to complete using the electronic format.
PAH-SYMPACT is the first patient-reported outcome instrument developed for people with PAH following the FDA’s patient reported outcome guidance.
Upon final validation steps, PAH-SYMPACT could be used for PAH clinical trials and clinical practice in order to assess patients’ symptoms, the impact of the condition in daily life, and how well a drug is working.
A Phase 3b clinical trial, called Symphony, for PAH patients is currently underway in the U.S. to validate the PAH-SYMPACT tool. For more information about the trial, please visit the link.