Prediction of Survival in Children With Cystic Fibrosis Using the Multiple-breath Washout

Multiple-breath Washout for Lung Function Testing in Children With Cystic Fibrosis: Predictive for Survival?

Retrospective cohort study using routinely collected annual data on lung clearance index (LCI) in combination with clinical data to predict survival in patients with Cystic Fibrosis. The primary study endpoint is the association of LCI with the compound outcome survival or lung transplantation in patients with CF.

Study Overview

• Status: Completed
• Conditions: Death; Cystic Fibrosis; Lung Transplantation
• Intervention / Treatment: Diagnostic test: Lung function testing

Detailed Description

Retrospective analysis of longitudinal data collected during clinically routine from approximately 200 patients with CF aged 3 years or more between 01.01.1980 and 31.12.2005. The overall aim of this project is to retrospectively assess whether LCI measured for clinical purposes during school age is predictive for survival or lung transplantation in adulthood. For this, the investigators will assess in a systematic way clinically measured LCI and relate them with the compound outcome survival or lung transplantation retrospectively.

• Study Type: Observational
• Enrollment (Actual): 200

Contacts and Locations

Study Locations

• Switzerland
  • Bern, Switzerland, 3007
    • University Children's hospital Bern
  • Bern, Switzerland, 3012
    • Lindenhof Spital, Quartier Bleu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Non-Probability sample

Description

Inclusion Criteria:

• Patients treated at the Children's Hospital Bern
• Confirmed CF Diagnosis
• Age >= 3 years

Exclusion Criteria:

• Uncertain diagnosis of CF according to current standards
• Existing written or otherwise documented refusal to participate in research

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lung clearance index (LCI)	Lung function outcome derived by MBW and the compound Outcomes mortality or lung transplantation	Lung function is collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Functional residual capacity (FRC)	Lung function outcome derived by MBW	Lung function is collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached
Forced expired Volume in 1 second (FEV1)	Lung function outcome derived by Spirometry	Lung function is collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached
Body weight	Anthropometric characteristics	Characteristics are collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached
Body length	Anthropometric characteristics	Characteristics are collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached
Gender	Anthropometric characteristics	Characteristics are collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached
Presence of Pseudomonas aeruginosa infection	Clinical characteristics	Characteristics are collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached
Presence of Staphylococcus aureus infection	Clinical characteristics	Characteristics are collected quarterly until outcome (death or lung transplantation) or end of Study (12/2005) is reached

Collaborators and Investigators

• Sponsor: University Hospital Inselspital, Berne
• Collaborators: Lindenhofspital
• Investigators: Principal Investigator: Florian Singer, University Hospital Inselspital, Berne

Publications and helpful links

General Publications

• Kurz JM, Ramsey KA, Rodriguez R, Spycher B, Fischer Biner R, Latzin P, Singer F. Association of lung clearance index with survival in individuals with cystic fibrosis. Eur Respir J. 2022 Mar 3;59(3):2100432. doi: 10.1183/13993003.00432-2021. Print 2022 Mar.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 1990
• Primary Completion (Actual): December 1, 2005
• Study Completion (Actual): December 1, 2005

Study Registration Dates

• First Submitted: May 7, 2019
• First Submitted That Met QC Criteria: July 9, 2019
• First Posted (Actual): July 11, 2019

Study Record Updates

• Last Update Posted (Actual): July 11, 2019
• Last Update Submitted That Met QC Criteria: July 9, 2019
• Last Verified: July 1, 2019

More Information

Terms related to this study

• Keywords: Survival; Prediction; Cystic Fibrosis, CF; Multiple-breath washout, MBW; Lung clearance index, LCI
• 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: 3840

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: IPD will be shared with researchers involved in the data collection and analysis.
• IPD Sharing Time Frame: Until 12/2020
• IPD Sharing Access Criteria: Researchers involved in data collection
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No