Objectives To characterize the rate of decline of forced expiratory volume in 1 second (FEV1 ) in children and adolescents with cystic fibrosis and to identify and compare risk factors associated ...with FEV1 decline. Study design The rate of decline in FEV1 % predicted over 3 to 6 years in 3 different age groups was determined. Risk factors for decline were identified and compared among and within age groups as a function of disease severity with repeated-measures, mixed-model regression. Results Mean (±SD) baseline FEV1 % predicted was 88.4% ± 20.5% for 6- to 8-year-olds (n = 1811), 85.3% ± 20.8% for 9- to 12-year-olds (n = 1696), and 78.4% ± 22.0% for 13- to 17-year-olds (n = 1359). Decline in FEV1 % predicted/year was −1.12, −2.39, and −2.34, respectively. High baseline FEV1 and persistent crackles were significant independent risk factors for decline across all age groups. Female sex, Pseudomonas aeruginosa infection, low weight-for-age, sputum, wheezing, sinusitis, pulmonary exacerbations treated with intravenous antibiotics, elevated liver test results, and pancreatic insufficiency were also identified as independent risk factors in some age groups. Conclusions This study identifies risk factors for FEV1 decline in children and adolescents with cystic fibrosis. Clinicians should not be reassured by high lung function, particularly in young children, because this factor, among others, is independently associated with steeper decline in FEV1.
Objective To determine the relation of growth and nutritional status to pulmonary function in young children with cystic fibrosis (CF). Study design The relation of weight-for-age (WFA), ...height-for-age (HFA), percent ideal body weight (%IBW), and signs of lung disease at age 3 years with pulmonary function at age 6 years was assessed in 931 patients with CF. Associations of changes in WFA from age 3 to 6 on pulmonary function were also assessed. Results WFA, HFA, and %IBW were poorly associated with lung disease at age 3 years, but all were strongly associated with pulmonary function at age 6 years. Those with WFA below the 5th percentile at age 3 had lower pulmonary function at age 6 compared with those above the 75th percentile (FEV1: 86 ± 20 SD versus 102 ± 18 % predicted, respectively). Pulmonary function was highest in those whose WFA remained >10th percentile from age 3 to 6 (FEV1: 100 ± 19 % predicted) and lowest in those who remained <10th percentile (84 ± 21 % predicted). Patients with signs and symptoms of lung disease at age 3 years had lower pulmonary function at age 6 years. Conclusions Aggressive intervention early in life aimed at growth and nutrition and/or lung disease may affect pulmonary function. (J Pediatr 2003;142:624-30)
Guidelines for managing cystic fibrosis (CF) patients have been widely circulated, but little is known about the variations in practice between sites and their association with outcomes.
To determine ...whether differences in lung health existed between groups of patients attending different CF care sites and to determine whether these differences are associated with differences in monitoring and intervention.
The analysis was conducted using data from the Epidemiologic Study of Cystic Fibrosis from 1995 through 1996.
This was an observational database collecting prospective information from a large number of CF patients undergoing routine care in North America.
Participating sites that had at least 50 CF patients who had each made at least one visit to a center during the 2-year study period were ranked on the basis of median values for FEV(1) within each of three age groups (6 to 12 years, 13 to 17 years, and >or= 18 years).
There were no prespecified interventions in this observational study.
The frequency of patient monitoring and the use of therapeutic interventions were compared between sites in the upper and lower quartiles after stratification within the site for disease severity.
Within-site rankings tended to be consistent across the three age groups. Patients who were treated at higher ranking sites had more frequent monitoring of their clinical status, measurements of lung function, and cultures for respiratory pathogens. These patients also received more interventions, particularly IV antibiotics for pulmonary exacerbations.
We found substantial differences in lung health across different CF care sites. We found that frequent monitoring and increased use of appropriate medications in the management of CF are associated with improved outcomes.
Pulmonary exacerbations in cystic fibrosis Rabin, Harvey R.; Butler, Steven M.; Wohl, Mary Ellen B. ...
Pediatric pulmonology,
20/May , Volume:
37, Issue:
5
Journal Article
Treatment of Acute Bronchiolitis Wohl, Mary Ellen B; Chernick, Victor
The New England journal of medicine,
07/2003, Volume:
349, Issue:
1
Journal Article
Peer reviewed
In this issue of the
Journal,
Wainwright et al.
1
provide data from a clinical trial in which 194 infants hospitalized with bronchiolitis were treated with inhaled epinephrine or placebo. In this age ...of commercial support for clinical trials, funding is available only when commercial sponsors stand to make a profit from the manufacture of a drug. Thus, Wainwright et al. are to be congratulated for having tackled an important question using a multicenter, randomized, blinded clinical trial that does not involve a new drug. The authors' findings overturn the popular notion that treatment of bronchiolitis with inhaled epinephrine, as compared . . .
To determine reproducibility of inflammatory marker concentrations in induced sputum from subjects with cystic fibrosis (CF), 15 nonexpectorating children, 6 to 13 years of age with mild CF lung ...disease, underwent 3 weekly sputum inductions with 3% saline. Neutrophil elastase concentration and bacterial cultures were reproducible. This study provides useful information for investigators designing trials of anti-inflammatory therapies in CF involving sputum induction.
Objective: Our objective was to determine whether long-term treatment of young patients with cystic fibrosis (CF) with dornase alfa maintains lung function and reduces respiratory tract ...exacerbations.
Study design: This was a 96-week, randomized, double-blind, placebo-controlled trial involving 49 CF centers. Inclusion criteria were age 6 to 10 years and forced vital capacity ≥ 85% predicted. Patients were excluded for hospitalization for complications of CF within 2 months and use of dornase alfa within 6 months. Patients were treated with dornase alfa 2.5 mg or placebo once daily with a jet nebulizer and a compressor.
Results: Patients were randomized, 239 to dornase alfa and 235 to placebo. At baseline the mean age was 8.4 years, the mean forced expiratory volume in 1 second 95% predicted, the mean forced expiratory flow, midexpiratory phase 85% predicted, and the mean forced vital capacity 102% predicted. At 96 weeks the treatment benefit for dornase alfa compared with placebo in percent predicted (mean ± SE) was 3.2 ± 1.2 for forced expiratory volume in 1 second (
P = .006), 7.9 ± 2.3 for forced expiratory flow between 25% and 75% of vital capacity (
P = .0008), and 0.7 ± 1.0 for forced vital capacity (
P = .51). The risk of respiratory tract exacerbation was reduced by 34% in patients who received dornase alfa (relative risk 0.66,
P = .048). There was no statistically significant difference between the groups in changes in weight-for-age percentile. Adverse event profiles for the treatment groups were similar.
Conclusions: Treatment of young patients with CF with dornase alfa maintains lung function and reduces the risk of exacerbations over a 96-week period. (J Pediatr 2001;139:813–20)
Asthma, Steroids, and Growth Wohl, Mary Ellen B; Majzoub, Joseph A
The New England journal of medicine,
10/2000, Volume:
343, Issue:
15
Journal Article
Peer reviewed
Long-term administration of systemic corticosteroids is a cause of impaired growth.
1
Trials comparing inhaled corticosteroid preparations with other treatment regimens in nearly 600 children with ...asthma found that inhaled beclomethasone had greater therapeutic effect than did other regimens.
2
–
5
In the studies, each of which lasted about one year, children treated with inhaled corticosteroids had less growth in height (1 to 1.6 cm 23 to 27 percent less) than those assigned to other treatments. The mechanisms by which this delay in growth occurs are unknown. If inhaled corticosteroids are not discontinued, does growth suppression continue, so that children with asthma . . .
The Pediatric Pulmonary and Cardiovascular Complications of Vertically Transmitted HIV (P(2)C(2) HIV) Study is a multicenter study examining pulmonary and cardiac outcomes in offspring of ...HIV-infected mothers. This portion of the P(2)C(2) study tests the hypothesis that infants exposed to, but uninfected by, maternal HIV have normal maximal expiratory flow at functional residual capacity (V'max,(FRC)). We obtained 500 measurements of V'max,(FRC) by rapid thoracic compression in 285 children ages 6-30 mo in five U.S. centers. The data were compared with those from a healthy cohort of children described elsewhere. V'max,(FRC) rose with height in a linear relationship. The slope of the regression line in the exposed infants did not differ statistically from the slope in the comparison group, but the intercept was about 20% lower (p < 0.001). Height and weight were comparable in the two cohorts, and the differences between intercepts persisted after adjusting for birth weight and gestational age. However, maternal HIV infection cannot be assumed to be the cause as the cohorts may have differed in other variables, such as socioeconomic status and frequency of maternal smoking and drug use. Also, measurements varied substantially within and between our five centers, probably in part because of different racial and ethnic distributions. In summary, maternal HIV infection probably has only a modest effect, if any, on maximal expiratory flow at functional residual capacity in uninfected infants.
Factors Influencing Outcomes in Cystic Fibrosis Johnson, Charles; Butler, Steven M.; Konstan, Michael W. ...
Chest,
January 2003, 20030101, 2003-01-00, Volume:
123, Issue:
1
Journal Article
Peer reviewed
Open access
Guidelines for managing cystic fibrosis (CF) patients have been widely circulated, but little is known about the variations in practice between sites and their association with outcomes.
To determine ...whether differences in lung health existed between groups of patients attending different CF care sites and to determine whether these differences are associated with differences in monitoring and intervention.
The analysis was conducted using data from the Epidemiologic Study of Cystic Fibrosis from 1995 through 1996.
This was an observational database collecting prospective information from a large number of CF patients undergoing routine care in North America.
Participating sites that had at least 50 CF patients who had each made at least one visit to a center during the 2-year study period were ranked on the basis of median values for FEV1 within each of three age groups (6 to 12 years, 13 to 17 years, and ≥ 18 years).
There were no prespecified interventions in this observational study.
The frequency of patient monitoring and the use of therapeutic interventions were compared between sites in the upper and lower quartiles after stratification within the site for disease severity.
Within-site rankings tended to be consistent across the three age groups. Patients who were treated at higher ranking sites had more frequent monitoring of their clinical status, measurements of lung function, and cultures for respiratory pathogens. These patients also received more interventions, particularly IV antibiotics for pulmonary exacerbations.
We found substantial differences in lung health across different CF care sites. We found that frequent monitoring and increased use of appropriate medications in the management of CF are associated with improved outcomes.
