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ABSTRACT: Cystic fibrosis (CF) specific patient-derived and reported symptom tools are critical steps toward evaluating the outcomes of new therapies for CF.
We conducted 25 in-depth qualitative interviews using the Day Reconstruction Method and 9 cognitive interviews at two CF programs, the University of Washington and Seattle Children's Hospital and Regional Medical Center. The interviews were audio-recorded and transcribed, and then coded and analyzed for themes relating to pulmonary symptoms and related psychosocial impacts.
Six pulmonary symptoms were identified as central to CF: cough, sputum production, wheeze, chest tightness, difficulty breathing/shortness of breath, and fever. Emotional impacts included frustration, sadness/depression, irritability, worry, difficulty sleeping; while activity impacts included time spent sitting or lying down, reduction of usual activities, and missing school or work. In all, 8 symptom items, 4 emotional impacts items, and 4 activity impacts were selected for inclusion on a new daily diary. We also assessed triggers for seeking care.
Using a qualitative inductive methodology, we have obtained patient centered data regarding pulmonary symptoms and burdens and have created a novel patient reported outcome measure for CF. Future studies will assess the validity of the instruments.
Journal of cystic fibrosis: official journal of the European Cystic Fibrosis Society 06/2009; 8(4):245-52. · 3.19 Impact Factor
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ABSTRACT: Centralized spirometry may significantly improve quality of spirometry and reduce variability of this outcome measure in clinical trials in cystic fibrosis (CF).
Spirometry was performed during the phase 2 randomized, placebo-controlled, double-blind clinical trial of denufosol in patients with mild to moderate CF using American Thoracic Society guidelines. Uniform spirometers were used with electronic data transmission of all the data to a reading center. Spirometry was evaluated for quality by a central reader based on start of test, cough during the test, and evidence of a plateau.
A total of 1418 spirometry values were assessed in 89 subjects during the trial. In only 5 instances did the central reading center need to give feedback to sites regarding the quality of spirometry. The study site data matched the central reading center's data for all but 78 (6%) spirometry values in 33 patients. Many of these differences were small with only 35 (3%) values differing by more than 50 mL in 26 patients.
Spirometry in this clinical trial was of high quality with low rate of significant centralized over-read.
Journal of Cystic Fibrosis 04/2008; 7(2):147-53. · 3.19 Impact Factor
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ABSTRACT: Stenotrophomonas maltophilia (SM) is a Gram-negative non-fermenting bacteria cultured from the sputum of patients with cystic fibrosis (CF). To date, no information is available regarding the effect of this organism on lung function in CF.
A cohort study was conducted to assess the effect of SM on lung function among CF patients aged > or =6 years in the CF Foundation National Patient Registry from 1994 to 1999. Repeated measures regression was used to assess the association between SM and lung function.
The cohort consisted of 20 755 patients with median age at entry of 13.8 years and median follow up time of 3.8 years; 2739 patients (13%) were positive at least once for SM and 18 016 (87%) were never positive. After adjusting for sex, height and age, patients with SM had a mean forced expiratory volume in 1 second which was 0.09 l less (95% CI 0.05 to 0.14) than those without SM. The mean rate of decline associated with SM positivity was 0.025 l/year (95% CI 0.012 to 0.037) but, after adjusting for confounders (sex, height, weight, intravenous antibiotic courses, hospital admissions, pancreatic insufficiency, and Pseudomonas aeruginosa and Burkholderia cepacia status), the mean rate of decline decreased to 0.008 l/year (-0.008, 95% CI -0.019 to 0.003).
Although CF patients with SM have worse lung function at the time of positivity, no association was found between SM and increased rate of decline after controlling for confounders.
Thorax 12/2004; 59(11):955-9. · 6.84 Impact Factor
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ABSTRACT: Clinical trials have become critical to the advancement of medical science and to the evolution of patient care in medicine. The science of clinical research has advanced from early studies in which treatment was assessed without controls to sophisticated multinational collaborative randomized, double-blind, placebo controlled trials of therapeutic interventions. To facilitate the advancement of clinical research, clinical trials networks have been developed to conduct multicenter studies. This review describes the history of clinical trials, clinical trials networks, and the goals of such networks in the United States. The Cystic Fibrosis Therapeutics Development Network, a network that represents the paradigm for genetic and orphan diseases, is described in detail. This network has been extremely successful in its first 3.5 years of existence conducting 18 different clinical trials in patients with Cystic Fibrosis. Unique aspects of the network include the use of internet applications for study conduct and communication, the development of statistical methodology to enhance the efficiency of clinical trial design, the development of outcome measures specific to Cystic Fibrosis, and the development of infrastructure necessary for expediting protocol development. In the current environment, clinical research faces significant challenges related to ensuring the safe and ethical conduct of clinical research while promoting fast and efficient clinical trials. To succeed and move forward to provide treatments and find cures for diseases, clinical trials networks must continue to evolve. The Cystic Fibrosis Therapeutics Development Network represents a network that has met this challenge and will continue to provide a venue for the safe and efficient conduct of clinical trials in Cystic Fibrosis.
Advanced Drug Delivery Reviews 01/2003; 54(11):1505-28. · 11.50 Impact Factor
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ABSTRACT: Certain family structures have been identified as putting children at high risk for injury. To further define children at highest risk, we set out to explore the effect of an older sibling and birth interval on the risk of injury related hospital admission or death.
Data were analyzed using a case-control design. Cases and controls were identified by linking longitudinal birth data from Washington state (1989-96) to death certificate records and hospital discharge data obtained from the Washington State Comprehensive Hospital Abstract Reporting System and frequency matched in a 1:2 ratio on year of birth. Cases consisted of singleton children 6 years of age or younger who were hospitalized or died as a result of injury during the years 1989-96. Multivariate logistic regression was used to identify and adjust for confounding variables.
There were 3145 cases and 8371 controls. The adjusted odds ratio for injury in children with an older sibling was 1.50 (95% confidence interval 1.37 to 1.65). The effect was greatest in children under 2 years of age, and in those with a birth interval of less than two years. As the number of older siblings increased, so did the risk of injury, with the highest risk in children with three or more older siblings.
These data suggest that the presence of an older sibling is associated with an increased risk of injury. The risk is highest in those with very short birth intervals. Potential mechanisms for this increased risk may relate to inadequate parental supervision. Pediatricians and other care providers need to be alert to these identifiable risk factors and then direct preventive strategies, such as home visits and educational programs, toward these families.
Injury Prevention 10/2000; 6(3):219-22. · 1.39 Impact Factor
