Advancing Symptom Science and Management in Cystic Fibrosis: Biological, Social, and Clinical Mechanisms

NIH Pandemic-Era Grants

Pandemic Era Grants

2024

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Principal Investigator: Dionysios  Kavalieratos
Organization: EMORY UNIVERSITY
Fiscal Year: 2024
Award: $590,820
Funding agency: National Heart Lung and Blood Institute

Project Summary
Therapeutic advances have doubled life expectancy for many people living with cystic fibrosis (PLWCF), yet
multisystem symptom burden (e.g., pain, dyspnea, fatigue) continues to impair quality of life. Evidence also
suggests that quality of life and mortality in CF are influenced by social determinants of health and race. The
goal of this observational study is to identify overlapping biopsychosocial drivers of symptom experience in
PLWCF. Specific Aim 1: We will use patient-reported outcomes to identify clusters of co-occurring, plausibly
interrelated symptoms in 140 adults with CF. We will also determine the extent to which social determinants of
health interrelate with symptom clusters to form clinically meaningful phenotypes of CF symptom experience.
Specific Aim 2: We will perform untargeted metabolomic analysis of plasma and exhaled breath condensate to
identify metabolic signatures (e.g. inflammation and oxidative stress) and clustering techniques to propose
metabolic endotypes and determine their relationships with symptom clusters and phenotypes. Specific Aim 3:
We will use sequential mixed methods incorporating surveys and interviews to identify PLWCF experience of
CF clinical care and relationships between symptom clusters, social determinants of health, and care
experience. Cumulatively, these complementary yet independent approaches will create a robust, person-
centered understanding of CF symptoms, their correlates and interactions, and their effect on quality of life.
The expected outcome of this research is identification of new, actionable targets for interventions to prevent or
reduce symptoms at the biological, clinical, and systems levels.

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