title: Patient-reported symptoms and functioning as indicators of mortality in advanced cystic fibrosis: A new tool for referral and selection for lung transplantation.
abstract: 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.
  
  
  
  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.
  
  
  
  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.
  
  
  
  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.
authors.affiliation: Lung Transplant and Cystic Fibrosis Unit, University and Polytechnic Hospital La Fe, Valencia, Spain; Health Research Institute La Fe, Valencia, Spain. Electronic address: sole_amp@gva.es.
authors.forename: Amparo
authors.initials: A
authors.lastname: Solé
authors.affiliation: Lung Transplant and Cystic Fibrosis Unit, University and Polytechnic Hospital La Fe, Valencia, Spain; Health Research Institute La Fe, Valencia, Spain.
authors.forename: Inés
authors.initials: I
authors.lastname: Pérez
authors.affiliation: Lung Transplant and Cystic Fibrosis Unit, University and Polytechnic Hospital La Fe, Valencia, Spain; Respiratory Rehabilitation Department, University and Polytechnic Hospital La Fe, Valencia, Spain.
authors.forename: Isabel
authors.initials: I
authors.lastname: Vázquez
authors.affiliation: Lung Transplant and Cystic Fibrosis Unit, University and Polytechnic Hospital La Fe, Valencia, Spain.
authors.forename: Amparo
authors.initials: A
authors.lastname: Pastor
authors.affiliation: Lung Transplant and Cystic Fibrosis Unit, University and Polytechnic Hospital La Fe, Valencia, Spain.
authors.forename: Juan
authors.initials: J
authors.lastname: Escrivá
authors.affiliation: Lung Transplant and Cystic Fibrosis Unit, University and Polytechnic Hospital La Fe, Valencia, Spain.
authors.forename: Gabriel
authors.initials: G
authors.lastname: Sales
authors.affiliation: Biostatistics Unit, Health Research Institute La Fe, Valencia, Spain.
authors.forename: David
authors.initials: D
authors.lastname: Hervás
authors.affiliation: Lung Transplant Unit, St. Vincent's Hospital, Sydney, New South Wales, Australia.
authors.forename: Allan R
authors.initials: AR
authors.lastname: Glanville
authors.affiliation: Departments of Psychology and Pediatrics, University of Miami, Coral Gables, Florida.
authors.forename: Alexandra L
authors.initials: AL
authors.lastname: Quittner
mesh_terms: D003550:Cystic Fibrosis; D005541:Forced Expiratory Volume; D006801:Humans; D016040:Lung Transplantation; D011788:Quality of Life; D012017:Referral and Consultation
keywords: cystic fibrosis; health-related quality of life; lung transplantation; patient reported outcomes; survival; waiting list