DEVELOPMENT AND PRELIMINARY TESTING OF A PATIENT-REPORTED OUTCOME MEASURE TO ASSESS SYMPTOM MANAGEMENT DURING AN EXACERBATION IN ADULTS LIVING WITH CYSTIC FIBROSIS

UoM administered thesis: Phd

  • Authors:
  • Gabriela Schmid-Mohler

Abstract

Background: Adults with cystic fibrosis (CF) experience, on average, two to three pulmonary exacerbations per year. Exacerbations are associated with a decline in lung function and increased mortality, result in high symptom and treatment burden and present self-management challenges for patients. Little is known about the emotional impact of exacerbations and how it relates to self-management. There are no patient-reported outcomes measures (PROMs) currently available to assess symptom distress and self-management during a pulmonary exacerbation in CF, limiting the ability to evaluate interventions in support of exacerbation-related self-management. Aim and objectives: The aim was to develop and conduct preliminary validity testing of a PROM for measuring a concept of relevance for symptom management during pulmonary exacerbation in adult CF patients, guided by the Food and Drug Administration (FDA) framework. Objectives were to: i) critically review PROMs related to symptom management in CF and appraise their suitability of use during exacerbations (Phase 1); ii) identify and describe current gaps in knowledge regarding CF patients experience of a pulmonary exacerbation (Phase 2); iii) explore patients’ experience (Phase 3); iv) develop a conceptual framework for symptom management in exacerbations (Phase 4); and v) develop a draft item list and assess content validity and clarity (Phase 5). Methods: A multistage study was undertaken, comprising qualitative methods (Phase 2), and two convergent mixed-method studies (Phases 3 and 5) Results: Phase 1: Five PROMs were included in the review; none measured the concept of symptom distress or self-management during exacerbation. Phase 2: The thematic synthesis of qualitative studies identified a broad spectrum of physical and emotional symptoms experienced by patients but data relating to experiences during exacerbation were limited. Phase 3: The mixed-method study (n=18) revealed that pulmonary exacerbations are characterized by an increase in emotional distress, which was linked to the meaning associated with the exacerbation. Experiencing a pulmonary exacerbation meant being ‘thrust out of normality’ and indicated a period of threat and life domination by CF. Symptom and treatment burden consumed energy and placed restrictions on physical activity and daily life roles. Phase 4: Based on a review of relevant theoretical frameworks, a conceptual model describing illness-related emotional distress was developed. Phase 5: A list of 27 items were extracted from patient interviews (Phase 3) which were underpinned by the conceptual framework (Phase 3 & 4). Clinical experts (n=12) and patients (n=8) rated the concept ‘emotional distress’ as relevant and comprehensively covered by the item list, and agreed that the draft PROM layout and wording were easily understood. Following this consultation, three further items were added (giving a total of 30 items) and yielded a draft PROM which is ready for refinement and psychometric testing in future quantitative studies. Conclusion: During exacerbation, illness-related emotional distress increases and is multidimensional, acting as a driver for self-management. The preliminary item list with confirmed face validity provides a basis for guiding and evaluating interventions.

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Original languageEnglish
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Award date1 Aug 2018