Biomarkers of Injury and Destruction in the Cystic Fibrosis Lung
| Status: | Completed |
|---|---|
| Conditions: | Hospital, Pulmonary |
| Therapuetic Areas: | Pulmonary / Respiratory Diseases, Other |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 1/14/2018 |
| Start Date: | December 2012 |
| End Date: | December 2017 |
Cystic fibrosis (CF) is the most common autosomal recessive genetic disease in Caucasians. It
results in lung disease that affects quality of life and causes early death. Lung damage from
CF starts in infancy and continues over time. Lung damage can negatively affect how the lung
functions. It would be ideal to measure lung damage in CF patients in three instances: (1)
During the first year of life after diagnosis by state newborn screening programs, (2) In
children and adults over long periods of time (years), and (3) During times of illness
(pulmonary exacerbation), to allow for better treatment and therapy to prevent loss of lung
function. The lung is made of elastin, collagen and cartilage. When the lung is damaged by
CF, these components break down into pieces that can be measured in urine, sputum and blood.
These products may represent markers of lung injury. We believe that the levels of these
markers will be increased over time in CF patients and even higher in patients who are sick
with lung symptoms. The goal of my research is to measure the amount of lung breakdown
products in urine, sputum and blood in infants, children and adults with CF during times when
well and also during times of illness. I also hope to use new technologies involving the
study of proteins and metabolites in samples like sputum, urine and blood to help provide new
information regarding CF lung disease. These studies will help us to better treat CF lung
disease.
results in lung disease that affects quality of life and causes early death. Lung damage from
CF starts in infancy and continues over time. Lung damage can negatively affect how the lung
functions. It would be ideal to measure lung damage in CF patients in three instances: (1)
During the first year of life after diagnosis by state newborn screening programs, (2) In
children and adults over long periods of time (years), and (3) During times of illness
(pulmonary exacerbation), to allow for better treatment and therapy to prevent loss of lung
function. The lung is made of elastin, collagen and cartilage. When the lung is damaged by
CF, these components break down into pieces that can be measured in urine, sputum and blood.
These products may represent markers of lung injury. We believe that the levels of these
markers will be increased over time in CF patients and even higher in patients who are sick
with lung symptoms. The goal of my research is to measure the amount of lung breakdown
products in urine, sputum and blood in infants, children and adults with CF during times when
well and also during times of illness. I also hope to use new technologies involving the
study of proteins and metabolites in samples like sputum, urine and blood to help provide new
information regarding CF lung disease. These studies will help us to better treat CF lung
disease.
This is a prospective, observational cohort study of two CF populations (and appropriate
control populations):
Infants with CF Diagnosed by Newborn Screen: The purpose of this prospective cohort study is
to measure concentrations of biomarkers of lung injury in urine, blood and bronchial alveolar
lavage fluid (BALF) from CF infants. We plan to characterize these concentrations during the
first two years of life with the goal of determining if their profiles alone or in
combination are potentially useful biomarkers of early lung disease in CF.
Each CF infant enrolled has blood, urine and BALF samples, along with an oropharyngeal swab
(throat culture) in conjunction with infant lung function testing at approximately 6 months
of age and again at one year. Our CF Center performs infant Pulmonary Function Tests (PFT's),
throat culture and a blood draw as part of routine care at 6 months and at one year of age,
so the bronchoscopy and urine collection are obtained for research purposes only. Urine is
collected quarterly during routine outpatient clinic visits to the CF Center (4 samples
total/year for two years) which is also for research purposes only. During the second year of
the study, urine, blood and throat swab sample collection plus lung function testing continue
as per year one. In the event of a hospitalization for pulmonary exacerbation, blood and
urine samples are obtained at three time points during hospitalization and BALF samples are
obtained via bronchoscopy upon admission. An oropharyngeal culture is also obtained upon
admission. Subsequent hospitalizations for pulmonary exacerbation are recorded with the same
samples collected as described above. In the event of an outpatient exacerbation requiring
antibiotic therapy, the patient will be asked to provide a urine sample at the onset of
exacerbation. Normal CF treatment or care is not altered in any way by participation in this
study.
We collect urine only from age-matched healthy infants as a control group for comparison.
Subjects with CF ≥ 8 Years of Age: The purpose of this prospective cohort study is to measure
concentrations of biomarkers of lung injury in urine, blood and sputum from CF patients. This
is an observational study that does not involve a specific treatment or intervention. We plan
to characterize these concentrations during two years of both clinically stable time points
as well during hospitalizations and outpatient treatments for pulmonary exacerbations. The
study goal is determining if these profiles alone or in combination are potentially useful
biomarkers of early lung disease in CF.
All study participants (CF Exacerbation Group and CF Non-Exacerbation Group) are asked to
provide urine, blood and sputum samples, as well as pulmonary function tests, during their
regularly scheduled quarterly CF Clinic visits over the study period. Quarterly lung function
testing is standard of care for all CF patients. For those patients in the CF Exacerbation
group, subjects are asked to provide three urine, blood and sputum samples as well as three
sets of pulmonary function before during and after an inpatient hospitalization for a
pulmonary exacerbation. We incorporate PFT's, and labs done as part of their hospital
admission whenever possible, to avoid duplicate testing. All hospitalized patients will
receive their routine, standard-of-care therapies including airway clearance, nutritional
support, IV antibiotics and other pulmonary medications. Treatment or care is not be altered
in any way by participation in this study. If a patient in the CF Non-Exacerbation Group
experiences a pulmonary exacerbation during the study period, samples will be obtained as
described for the CF Exacerbation Group.
For a control group comparison we collect sputum and urine only from volunteers > 8 years of
age who do not have CF.
control populations):
Infants with CF Diagnosed by Newborn Screen: The purpose of this prospective cohort study is
to measure concentrations of biomarkers of lung injury in urine, blood and bronchial alveolar
lavage fluid (BALF) from CF infants. We plan to characterize these concentrations during the
first two years of life with the goal of determining if their profiles alone or in
combination are potentially useful biomarkers of early lung disease in CF.
Each CF infant enrolled has blood, urine and BALF samples, along with an oropharyngeal swab
(throat culture) in conjunction with infant lung function testing at approximately 6 months
of age and again at one year. Our CF Center performs infant Pulmonary Function Tests (PFT's),
throat culture and a blood draw as part of routine care at 6 months and at one year of age,
so the bronchoscopy and urine collection are obtained for research purposes only. Urine is
collected quarterly during routine outpatient clinic visits to the CF Center (4 samples
total/year for two years) which is also for research purposes only. During the second year of
the study, urine, blood and throat swab sample collection plus lung function testing continue
as per year one. In the event of a hospitalization for pulmonary exacerbation, blood and
urine samples are obtained at three time points during hospitalization and BALF samples are
obtained via bronchoscopy upon admission. An oropharyngeal culture is also obtained upon
admission. Subsequent hospitalizations for pulmonary exacerbation are recorded with the same
samples collected as described above. In the event of an outpatient exacerbation requiring
antibiotic therapy, the patient will be asked to provide a urine sample at the onset of
exacerbation. Normal CF treatment or care is not altered in any way by participation in this
study.
We collect urine only from age-matched healthy infants as a control group for comparison.
Subjects with CF ≥ 8 Years of Age: The purpose of this prospective cohort study is to measure
concentrations of biomarkers of lung injury in urine, blood and sputum from CF patients. This
is an observational study that does not involve a specific treatment or intervention. We plan
to characterize these concentrations during two years of both clinically stable time points
as well during hospitalizations and outpatient treatments for pulmonary exacerbations. The
study goal is determining if these profiles alone or in combination are potentially useful
biomarkers of early lung disease in CF.
All study participants (CF Exacerbation Group and CF Non-Exacerbation Group) are asked to
provide urine, blood and sputum samples, as well as pulmonary function tests, during their
regularly scheduled quarterly CF Clinic visits over the study period. Quarterly lung function
testing is standard of care for all CF patients. For those patients in the CF Exacerbation
group, subjects are asked to provide three urine, blood and sputum samples as well as three
sets of pulmonary function before during and after an inpatient hospitalization for a
pulmonary exacerbation. We incorporate PFT's, and labs done as part of their hospital
admission whenever possible, to avoid duplicate testing. All hospitalized patients will
receive their routine, standard-of-care therapies including airway clearance, nutritional
support, IV antibiotics and other pulmonary medications. Treatment or care is not be altered
in any way by participation in this study. If a patient in the CF Non-Exacerbation Group
experiences a pulmonary exacerbation during the study period, samples will be obtained as
described for the CF Exacerbation Group.
For a control group comparison we collect sputum and urine only from volunteers > 8 years of
age who do not have CF.
Infant Study Inclusion Criteria
- Age < 6 months
- Cystic Fibrosis Diagnosed by Newborn Screen
Exclusion:
Age over 6months
Child Study Inclusion Criteria
- FEV1 < 35% predicted A
- Use of IV antibiotics for a pulmonary exacerbation or respiratory symptoms 12 months
prior to enrollment.
- Hospitalization for a pulmonary exacerbation 12 months prior to enrollment.
- Use of oral antibiotic for respiratory symptoms 28 days prior to enrollment.
- Any changes in medical regimen for treatment of CF (e.g. no addition of or elimination
of therapies such as hypertonic saline, inhaled corticosteroids or mucolytic therapy)
within 28 days of enrollment visit.
Exclusion Criteria:
- FEV1 < 35% predicted A
- Use of IV antibiotics for a pulmonary exacerbation or respiratory symptoms 12 months
prior to enrollment.
- Hospitalization for a pulmonary exacerbation 12 months prior to enrollment.
- Use of oral antibiotic for respiratory symptoms 28 days prior to enrollment.
- Any changes in medical regimen for treatment of CF (e.g. no addition of or elimination
of therapies such as hypertonic saline, inhaled corticosteroids or mucolytic therapy)
within 28 days of enrollment visit.
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