Patient-reported symptoms and functioning as indicators of mortality in advanced cystic fibrosis: A new tool for referral and selection for lung transplantation

Background: Despite well-known risk factors and predictive survival models, many patients with cystic fibrosis (CF) die while on the waiting list for lung transplant. We evaluated whether specific Cystic Fibrosis Questionnaire (CFQ-R) scales provide additional benefit to conventional tools in identifying referral timing and waitlist mortality.

Methods: From January 2010 to January 2015, 152 patients (34% on the waitlist) were evaluated with the CFQ-R and standard protocol quarterly. Data were used to explore the prognostic association of health-related quality of life.

Results: The Physical Functioning domain (PFD) of the CFQ-R predicted mortality in advanced CF disease better than habitual parameters (p = 0.005). For patients with the same forced expiratory volume in 1 sec (FEV1), a low score categorized patients with an increased risk of death. For patients with CF and FEV1 <30% predicted and a low Physical score, mortality rate was ~35% at 2 years. The best model for probability of inclusion on the waitlist was FEV1 % (p < 0.001, hazard ratio [HR] = 0.94; 95% confidence interval [CI] [0.90, 0.97]) and Physical Functioning (p = 0.013, HR = 0.96; 95% CI [0.95, 0.99]). The best model for probability of death similarly included FEV1 % (p = 0.09, HR = 0.97; 95% CI [0.94, 1.00]) and CFQ-R Physical Functioning score (p = 0.005, HR = 0.97; 95% CI [0.95, 0.99]). The Health Perception score showed similar results. A low Health Perception score combined with a high resting heart rate showed a trend for mortality.

Conclusions: The CFQ-R may be an additional tool for guiding decisions to place a patient with CF on the waiting list for lung transplantation. The CFQ-R Physical Functioning and Health Perception scales were more accurate than conventional tools in predicting death before transplant.