A Clinical Trial of Home Lung Function and Symptom Monitoring in Cystic Fibrosis (CF)
Summary
Cystic fibrosis (CF) is the most common life shortening inherited disease in Caucasians. Individuals with CF develop chronic lung infections and suffer intermittent acute exacerbations of their lung disease, which require intensive treatment with antibiotics. The most common objective measure of CF lung disease is spirometry, which is measured during physician visits. Early, aggressive treatment of lung disease improves outcomes, but most exacerbations are not treated until they cause increased symptoms, and patients seek medical attention. Another obstacle to CF care is poor adherence to therapy. Individuals with CF frequently take more than eight different medications, many of which are costly and difficult to administer. Self-monitoring of clinical status has improved outcomes in many other disorders such as asthma and diabetes mellitus, but has not been widely studied in CF. This proposal details a study of home lung function and symptom monitoring. Subjects will be randomly assigned to one of two groups: 1) home monitoring, in which spirometry and symptoms are recorded daily; or 2) CF education. The home monitoring data will be transmitted via phone modem 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. Subjects in the CF Education arm will contact the study nurse if they become symptomatic of an exacerbation. It is anticipated that use of home monitoring will lead to earlier, more reliable recognition and treatment of exacerbations, which will translate into better lung health.
Description
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. Another obstacle to CF care is poor adherence to therapy. Individuals with CF frequently take as many as eight different medications, many of which are difficult to administer and costly. Self-monitoring of clinical status has improved outcomes in many other disorders such as asthma, diabetes mellitus, and lung transplantation. This proposal details an important, randomized trial of home lung function and symptom monitoring. Subjects will be assigned to one of two groups: 1) home monitoring, in which spirometry and symptoms are recorded daily; or 2) CF education. The home monitoring data will be transmitted electronically 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 two main hypotheses: 1) home FEV1 and symptom monitoring will result in better lung function, and 2) home monitoring will improve medication adherence.
Study Design
Allocation: Randomized, Control: Active Control, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Conditions
Cystic Fibrosis
Intervention
Home lung function and symptom monitoring
Location
Johns Hopkins University CF Clinic
Baltimore
Maryland
United States
21205
Status
Not yet recruiting
Source
Johns Hopkins University
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT01104402
- Information obtained from ClinicalTrials.gov on July 15, 2010
Medical and Biotech [MESH] Definitions
Cystic Fibrosis
An autosomal recessive genetic disease of the EXOCRINE GLANDS. It is caused by mutations in the gene encoding the CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR expressed in several organs including the LUNG, the PANCREAS, the BILIARY SYSTEM, and the SWEAT GLANDS. Cystic fibrosis is characterized by epithelial secretory dysfunction associated with ductal obstruction resulting in AIRWAY OBSTRUCTION; chronic RESPIRATORY INFECTIONS; PANCREATIC INSUFFICIENCY; maldigestion; salt depletion; and HEAT PROSTRATION.
Cystic Fibrosis Transmembrane Conductance Regulator
A chloride channel that regulates secretion in many exocrine tissues. Abnormalities in the CFTR gene have been shown to cause cystic fibrosis. (Hum Genet 1994;93(4):364-8)
Mice, Inbred Cftr
A strain of mice widely studied as a model for cystic fibrosis. These mice are generated from embryonic stem cells in which the CFTR (cystic fibrosis transmembrane conductance regulator) gene is inactivated by gene targeting. As a result, all mice have one copy of this altered gene in all their tissues. Mice homozygous for the disrupted gene exhibit many features common to young cystic fibrosis patients, including failure to thrive, meconium ileus, and alteration of mucous and serous glands.
Stenotrophomonas Maltophilia
A species of STENOTROPHOMONAS, formerly called Xanthomonas maltophilia, which reduces nitrate. It is a cause of hospital-acquired ocular and lung infections, especially in those patients with cystic fibrosis and those who are immunosuppressed.
Drainage, Postural
A rehabilitation therapy for removal of copious mucus secretion from the lung of patients with diseases such as CHRONIC BRONCHITIS; BRONCHIECTASIS; PULMONARY ABSCESS; or CYSTIC FIBROSIS. The patient's head is placed in a downward incline (so the TRACHEA is inferior to the affected area) for 15- to 20-minute sessions.
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