Prognostic factors for outcomes in patients with cystic fibrosis who are observed at the Federal Clinical Center

Abstract

Objective. To identify the factors significantly affecting clinical outcomes in patients with cystic fibrosis who are observed at the Federal Clinical Center based on a multivariate analysis of the disease course. Patients and methods. A total of 657 medical records of patients with cystic fibrosis were examined. The survival rate was analyzed depending on the type of genetic mutation and sex of patients. The dynamic model for predicting 2-year survival outcomes based on 36 parameters from the National Registry was developed. Results. The multivariate Cox proportional hazard model applied to dynamic data by a landmarking method revealed a statistically significant association between mortality over a 2-year lifetime with the values of the following factors: FEV1 (relative risk (RR): 0.95; 95% confidence interval (CI): 0.94–0.97); cystic fibrosis-related diabetes (RR: 2.36; CI: 1.47–3.80); Burkholderia cepacia complex infection (RR: 3.22; CI: 2.12–4.91); need for oxygen therapy (RR: 1.9; CI: 1.15–3.12); previous pneumothorax (RR: 2.72; CI: 1.19-6.20); vitamin therapy (RR: 2.72; CI: 1.09–6.81); pancreatic enzyme intake (RR: 0.38; CI: 0.17–0.86). Conclusion. The awareness of prognostic survival factors will allow to optimize treatment and rehabilitation programs for patients with cystic fibrosis, which will lead to an increase in patients’ life expectancy. Key words: cystic fibrosis, children, survival rate