Prognostic Value of Functional Exercise Test (EFX) in Cystic Fibrosis (EFX)

The objective of study is to prospectively determine if CPET with blood gas analysis should have a prognostic value in CF. The study plans to include 300 cystic fibrosis patients. Inclusion criteria will be: age >15 years, cystic fibrosis confirmed by chloride sweat test or genetic analysis, clinical and functional stability in the 2 month before CPET. Patients will perform a maximal exercise test on a cycloergometer during the inclusion visit, with pulmonary function testing and a six-minute walk test. The study will also include a visit every 6 months with: body mass index calculation, pulmonary function testing with DLCO (diffusing lung capacity for carbon monoxide), a six minute walk test, and antibacteriological study of sputum. The results of this study could help identify earlier the patients for referral to a lung transplantation centre, by using the usual criteria and the CPET abnormalities.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Other: cardiopulmonary exercise testing

Detailed Description

Current guidelines for referring cystic fibrosis (CF) patients for lung transplantation, based on clinical and functional resting parameters, are insufficient to predict 3-year mortality. Previous studies have shown that sex, impaired pulmonary function, undernutrition and colonization of the respiratory tract by Pseudomonas aeruginosa are associated with a poor prognosis. Current guidelines for referral to a lung transplant center include age, sex, forced expiratory volume at one second (FEV1) below 30% predicted or a rapid decline of FEV1, in particular in young female patients, increasing frequency of exacerbations requiring antibiotic therapy, refractory or recurrent pneumothorax, recurrent hemoptysis not controlled by embolization. But, despite these criteria, near that 30% of patients are still dying while on the lung transplant waiting list, or are transplanted in high emergency. Cardiopulmonary exercise testing (CPET) in CF patients would have an interest in the following of cystic fibrosis patients. A previous study, carried out on fifty one adult patients, showed that CPET with blood gas analysis may have a prognosis value in cystic fibrosis. The authors found that a BMI < 19.8 and P(A-a)O2 peak > 43 mmHg were independently associated with a lower chance of survival.

• Study Type: Interventional
• Enrollment (Actual): 287
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Anne Prevotat, MD; Email: anne.prevotat@chru-lille.fr

Study Locations

• France
  • Lille, France
    • Hôpital Calmette, CHRU

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 15 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Age more than 15 years
• Cystic fibrosis diagnosed by positive sweat chloride test or genetic test

Exclusion Criteria:

• Pregnant or breastfeeding woman
• waiting on transplantation list

Study Plan

How is the study designed?

Design Details

• Primary Purpose: SUPPORTIVE_CARE
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

OTHER: cystic fibrosis patients

Other: cardiopulmonary exercise testing; The Cardiopulmonary exercise testing (CPET) was performed at the beginning of the study, the same at each center. Each patient underwent a symptom-limited incremental exercise test on an ergometric bicycle (Ergoline-Ergometrics 800®). The protocol included a warm-up period of 3 min at 20 W followed by a progressively increasing work rate (WR) in a ramp fashion and then 3 min recovery. The ramped WR increment was individualized (range,8-30 W/min). during exercise, heart rate (HR) was monitored continuously by 12-lead ECG, and arterial oxygen saturation (SpO2) was measured by pulse oximetry (Nellcor N-395). The expired gases were analyzed with an Ergocard®, focusing on oxygen consumption(VO2), carbon dioxide production (VCO2), minute ventilation (VE), and tidal volume (VT).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Survival	the survival is defined by the occurrence of death or lung transplantation.	at five years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
6 minute walking distance		Every 12 months during 5 years
body mass index calculation		Every 12 months during 5 years
sputum sample culture	(a descriptive analysis of Haemophilus influenzae, Staphylococcus aureus, Pseudomonas aeruginosa,Burkholderia cepacia streptococci in the bacterial flora of sputum)	Every 12 months during 5 years
pulmonary function testing with DLCO	diffusing lung capacity for carbon monoxide.	Every 12 months during 5 years

Collaborators and Investigators

• Sponsor: University Hospital, Lille

Study record dates

Study Major Dates

• Study Start (ACTUAL): March 5, 2012
• Primary Completion (ACTUAL): June 16, 2019
• Study Completion (ACTUAL): June 16, 2019

Study Registration Dates

• First Submitted: August 16, 2016
• First Submitted That Met QC Criteria: December 13, 2016
• First Posted (ESTIMATE): December 15, 2016

Study Record Updates

• Last Update Posted (ACTUAL): February 8, 2023
• Last Update Submitted That Met QC Criteria: February 3, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Keywords: cystic fibrosis; Cardiopulmonary exercise testing; prognostic
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: 2011_31; 2011-A01532-39 (OTHER: ID-RCB number, ANSM)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO