Prevention of CF Exacerbation in Childhood: PREVEC Study

• Number of exacerbations [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  Definition of an exacerbation according to Treggiari MM et al.
• Lung function [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  FEV1 % predicted value

• Quality of life [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  Quality of life questionnaire
• Pulmonary imaging [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  High resolution computed tomography (HRCT) scan
• Cost-effectiveness [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  Incremental costs per exacerbation prevented

Pulmonary exacerbations of CF are an important cause for the experienced disability of patients, respiratory symptoms, and decreases in lung function, which require antibiotic therapy at home or in the hospital. Therefore, prevention of exacerbations in CF is important. In an earlier prospective study during one year, we have demonstrated that non-invasive inflammatory markers in exhaled breath (condensate) are able to predict clinical CF exacerbation before they are clinically manifest. The aim of the study is to assess the efficacy of an intervention directed towards prevention of clinical CF exacerbations by means of early recognition and early antibiotic treatment.

• Cystic Fibrosis
• Children
• Exhaled Breath Condensate
• Non-invasive Inflammatory Markers
• Volatile Organic Compounds

• Other: Diagnostic intervention without standard therapy
  Diagnostic intervention without standard therapy
• Other: Diagnostic intervention with standard therapy
  Diagnostic intervention with standard therapy

• Active Comparator: Diagnostic intervention with standard therapy
  Diagnostic assessments of non-invasive inflammatory markers in exhaled air and exhaled breath condensate in addition to symptoms/lung function to guide treatment (active intervention group) compared to usual care (guiding of treatment by symptoms and lung function only).
  Intervention: Other: Diagnostic intervention with standard therapy
• Diagnostic intervention without standard therapy
  Intervention: Other: Diagnostic intervention without standard therapy

Inclusion Criteria:

• CF disease is defined as the combination of:

  1. characteristic clinical features (persistent pulmonary symptoms, meconium ileus, failure to thrive, steatorrhoe);
  2. and/or abnormal sweat test (chloride > 60mM);
  3. and/or two CF mutations.

Exclusion Criteria:

• Exclusion criteria are:

  1. cardiac abnormalities;
  2. mental retardation;
  3. no technical satisfactory performance of measurements;
  4. on the waiting list for lung transplantation;
  5. non-compliance with the home-assessments;
  6. patients with Burkholderia Cepacia;
  7. participation in another intervention trial.

• NCFS
• Chiesie Pharmaceuticals B.V.