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

https://doi.org/10.1016/j.healun.2016.01.1233Get rights and content

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.

Section snippets

Study design and population

The Research Ethics Board of the University Hospital la Fe approved this study. A prospective study of HRQoL was conducted in patients with CF either on a waitlist for LTX or not yet waitlisted. The sample included 152 patients who completed the CFQ-R every 3 months along with standard clinical measures. All patients with CF seen in our CF and LTX Unit from January 2010 to January 2015 were eligible for inclusion. Patients were followed from enrollment through the follow-up period (2–4 years).

Results

The study sample included 152 patients with no missing data or loss to follow-up. Baseline demographic characteristics are shown in Table 1. In general, female patients had worse scores than male patients on the following CFQ-R scales: Health Perceptions (p < 0.03), Social Functioning (p < 0.001), and Vitality (p < 0.006), regardless of age or lung function.

Of the 152 patients, 37 (24.34%) were activated on the waiting list for transplant during the study and 15 patients (9.87%) died (12 on the

Discussion

Existing guidelines on specific selection criteria for LTX in patients with CF have been primarily based on single-center data,8 which identified FEV1 <30% predicted as a useful survival marker. Numerous authors have attempted to develop models incorporating several variables to predict long-term survival in larger CF cohorts, but no model is widely used owing to lack of predictive ability. One model based on data from the US Cystic Fibrosis Foundation considered 9 parameters to assess 5-year

Disclosure statement

None of the authors has a financial relationship with a commercial entity that has an interest in the subject of the presented manuscript or other conflicts of interest to disclose.

This study was supported by a Pablo Motos grant from the Spanish Federation of Cystic Fibrosis and an International Traveling Scholarship from the International Society for Heart and Lung Transplantation (A.S.).

The authors thank all patients who participated in this study, Dr. Vincent Valentine for his initial help

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