PURPOSE: There are no existing patient-reported outcomes (PROs) for non-CF bronchiectasis that meet the criteria in the FDA Guidance. PROs measure how a patient “feels or functions with respect to his or her health condition.” Increasingly, PROs are utilized as primary or secondary outcomes in drug registration trials and must follow the steps outlined in the Guidance (2009). The current study presents the content validity of a new, disease-specific PRO for bronchiectasis (Quality of Life Questionnaire-Bronchiectasis; QOL-B).
METHODS: Twenty-eight participants diagnosed with bronchiectasis were recruited from 3 sites across the United States; 82 % were women, mean age was 60.1 years (SD= 16.8), and mean FEV1% predicted was 65.0% (SD = 23.1). Participants were interviewed by doctoral-level psychologists and were asked open-ended questions about how bronchiectasis affects their daily lives. Interviews were audio-recorded, transcribed and then content analyzed using Atlas.ti, a software program designed for qualitative data analysis. Across participants, the frequency of specific symptoms and other effects of the disease were analyzed to produce saturation grids. The results were utilized to generate and confirm items on the QOL-B.
RESULTS: Overall, 73 disease-related topics were mentioned by participants during the open-ended interviews. 71 out of the 73 topics were mentioned by the first 14 adults, with no new topics reported by the last 5 patients. The top 3 most commonly reported symptoms were: 1) coughing, 2) fatigue, and 3) shortness of breath with exertion, endorsed by 89% of participants. Saturation of content was achieved across all dimensions of functioning.
CONCLUSIONS: The content validity of the QOL-B was supported. A cognitive testing study is in process to confirm adequacy of items and response options not previously tested. The final iteration of the QOL-B Version 3.0 has 37 items across 8 domains (Respiratory Symptoms, Physical Functioning, Vitality, Role Functioning, Health Perceptions, Emotional Functioning, Social Functioning, and Treatment Burden).
CLINICAL IMPLICATIONS: The development of the QOL-B will result in a tool that can be used to evaluate the efficacy of new medications, as well as the functioning of individual patients during routine or acute care.
DISCLOSURE: Alexandra Quittner: Consultant fee, speaker bureau, advisory committee, etc.: North American Scientific Advisory Group, Consultant fee, speaker bureau, advisory committee, etc.: Gilead Sciences, Vertex, Bayer, Grant monies (from sources other than industry): NIH SBIR and R01 grants, Grant monies (from industry related sources): Gilead Sciences, Novartis Pharmaceuticals
Kristen Marciel: Grant monies (from industry related sources): Urand, Consultant fee, speaker bureau, advisory committee, etc.: Gilead Sciences
Cara Kimberg: Consultant fee, speaker bureau, advisory committee, etc.: GIlead Sciences
The following authors have nothing to disclose: Alan Barker
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