PAH patient-reported outcome instrument developed
medwireNews: Researchers have developed the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT®) questionnaire, which they say captures symptoms and impacts relevant to the patient population.
Deborah McCollister (University of Colorado Denver, Aurora, USA) and co-authors stress that the development process strictly followed the US Food and Drug Administration’s guidance on patient-reported outcomes (PROs), and that the resulting survey is the first for use in PAH patients to meet these criteria.
To generate a disease-specific PRO instrument, they recruited 55 PAH patients with an average age of 53.1 years, the majority of whom were female (93%). Just over half (56%) of the participants had idiopathic disease, while 42% and 2% had associated and familial PAH, respectively.
In the first phase of the study, 25 patients (five per group) participated in focus group discussions to identify PAH symptoms and their impact on patients’ lives. Once saturation was reached – ie, no new relevant or important information emerged – a draft questionnaire comprising 16 symptoms and 23 impact items was developed with input from an international steering committee of experts, including on whether the symptoms mentioned by discussants were related to the disease or to treatment.
The questionnaire was assessed in the second phase, which involved two rounds of one-to-one cognitive interviews with 10 patients per round. Participants generally found the draft questionnaire to be “clear, comprehensive, and relevant”, says the team, and only a few modifications were made after each round of interviews.
Finally, interviews with an additional 10 patients demonstrated that the revised PAH-SYMPACT® questionnaire was not missing any important items and that the patients understood the wording as intended.
These participants also tested the usability of an electronic version of the survey on tablet computers, and found it “an easy and convenient way to complete the questionnaire”.
The questionnaire underwent translatability and lexibility evaluation at each step, which the team says should help facilitate the translation of the instrument into different languages and ensure that it will be understood even by patients with low education levels, respectively.
The final version of the questionnaire contains 16 symptoms grouped into four domains, namely respiratory and cardiovascular symptoms, tiredness, and other symptoms; and 25 impact items in five domains, ie, physical and daily activities, social and emotional impact, and cognition. For symptoms, the recall period is the past 24 hours, while it is the past 7 days for the impact items.
“The PAH-SYMPACT® is designed to be a practical tool both for use in general practice and in future trials to assess the effect of PAH and PAH-specific therapies on patients’ symptoms and their impacts”, McCollister et al write in Respiratory Research.
They add that the “[f]inal content and psychometric validation of the instrument will be based on the results of an ongoing Phase IIIb clinical trial in PAH patients.”
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