(193) Cystic fibrosis-related liver disease is associated with an increased risk of mortality and a higher disease burden in pediatric patients with cystic fibrosis

Background: Cystic fibrosis-related liver disease (CFLD) is the third-leading cause of mortality in cystic fibrosis (CF) after end-stage pulmonary disease and lung transplant-related complications. CFLD is increasingly recognized due to advancing CF management and improved awareness among clinicians. There is lack of population-level data on the prevalence and impact of CFLD on mortality and hospital outcomes. The aim of this study is to evaluate the mortality and disease burden associated with CFLD in pediatric patients.

Methods: We analyzed all patients with CF who were enrolled in any of the 50 children’s hospitals participating in the Children’s Hospital Association (CHA). We interrogated the Pediatric Health Information System® (PHIS) database of CHA to include all patients < 21 years of age who were diagnosed with CF using the International Classification of Diseases (ICD) diagnostic codes. For this study, we defined CFLD if patients with CF had a concomitant diagnosis of at least one of the following: cirrhosis, liver fibrosis, chronic liver disease, esophageal/gastric varices, ascites, portal hypertension, liver transplant, and hypersplenism. Patients with CFLD were then compared with CF patients without any of the above concomitant diagnoses for demographics, and other comorbid conditions. The primary outcome variable was mortality.

Results: We analyzed a total of 21,827 patients between 2004 and 2021. The prevalence of CFLD was 6.4% (1,399) (Table 1). The prevalence of CFLD increased with age, with a peak rate of 16.7% at 21 years of age, P< 0.01. Patients with CFLD were older (median 14.7 vs 10.6 years) and more prevalent in males, P< 0.001. Patients with CFLD had an increased prevalence of significant comorbidities such as respiratory failure, admission to intensive care unit, mechanical ventilation, pulmonary hypertension, lung transplantation, diabetes mellitus, malnutrition, increased need for parenteral nutrition, and malignancies, P< 0.001. After adjusting for various factors, the multivariate regression model showed CFLD was associated with 1.72 times increased risk of mortality (CI: 1.35 to 2.18, P< 0.001).

Conclusions: CFLD is associated with an increased risk of mortality and also associated with multiple comorbidities representing increased disease severity. Prospective longitudinal studies are required to further delineate the impact of CFLD in CF patients.

Acknowledgements: SS is a recipient of the DIGEST award from the Cystic Fibrosis Foundation.

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