Early Intervention in Cystic Fibrosis Exacerbation

Individuals with CF develop chronic lung infections and suffer intermittent exacerbations,
which require intensive treatment with antibiotics. The most common and useful objective
measure of CF lung disease is spirometry. Chronic treatment of CF lung disease requires
airway clearance, mucolytics and antibiotics. These treatments have been quite successful and
there is evidence that early, aggressive treatment of lung disease results in better
outcomes. Unfortunately, most exacerbations are not treated until they cause pronounced
deterioration in symptoms, which prompts patients to seek medical attention. Self-monitoring
of clinical status has improved outcomes in many other disorders such as asthma, diabetes
mellitus, and lung transplantation. This is an important, randomized trial of home lung
function and symptom monitoring in CF. Subjects will be assigned to one of two groups: 1)
Home monitoring, in which spirometry and symptoms are recorded daily; or 2) Standard Care.
The home monitoring data will be transmitted electronically twice weekly to the study center,
where the results will be reviewed. If spirometry or symptoms have deteriorated substantially
below baseline, treatment for a CF pulmonary exacerbation will be initiated. It is
anticipated that use of home monitoring will translate into better clinical outcomes. We will
test the hypothesis that if pulmonary exacerbations are identified and treated earlier than
the current standard of care, the progression of lung disease will be slowed.

Inclusion Criteria:

- CF diagnosis confirmed with sweat test, abnormal nasal potential difference and/or
genetic testing

- Age 14 and older

- Able to perform spirometry

- Clinically stable without antibiotic treatment for a pulmonary exacerbation in the two
weeks prior to the screening visit

- Forced expiratory volume in the first second (FEV1) greater than 25% of predicted at
screening

Exclusion Criteria:

- History of solid organ transplant

- Participation in any interventional trial within the last 30 days

- Inability to speak and read the English language well enough to complete
questionnaires

- Colonization with Burkholderia cepacia genomovar III within the last 24 months

- Currently receiving antimicrobial treatment specifically used to treat active
non-tuberculosis mycobacterium

- Confirmed diagnosis of allergic bronchopulmonary aspergillosis (ABPA) as defined by
the Cystic Fibrosis Foundation (CFF) guidance document that is being actively treated