Objective: To determine the impact of age and condition at the time of diagnosis on survival of patients with cystic fi-brosis (CF). Study design: By mode of diagnosis, 27,692 patients documented in the 1986-2000 CF F...Objective: To determine the impact of age and condition at the time of diagnosis on survival of patients with cystic fi-brosis (CF). Study design: By mode of diagnosis, 27,692 patients documented in the 1986-2000 CF Foundation Registry were segregated into meconium ileus (MI), prenatal or neonatal screening (SCREEN), positive family history only (FH), and symptoms other than MI (SYMPTOM). Patients in the MI, SCREEN, and SYMPTOM groups were further categorized by initial presenting symptoms into combined respiratory symptoms and malnutrition (RESP + NUTR), RESP, NUTR, other less common symptoms (OTHER), and OTHER + RESP/NUTR. Results: Fifty-five percent of patients in the SCREEN group and 59% of patients in the MI group were diagnosed within age 1 month, as contrasted with 5% in patients in the SYMPTOM group (P < .001). Compared with patients in the SCREEN group, patients in the MI and SYMPTOM groups had significantly greater risks of shortened survival. Patients in the SYMPTOM group presenting with RESP + NUTR had significantly greater risk of shortened survival than the SCREEN group (P < .05). Survival of patients in the SYMPTOM group diagnosed “ early,” that is, within 1 month of age, did not differ from patients in the SCREEN group but was significantly better than patients in the SYMPTOM group diagnosed beyond age 1 month to 10 years. Conclusions: Early diagnosis through screening is associated with better survival compared with delayed diagnosis through non-MI symptoms beyond the age of 1 month.展开更多
Context: Although Pseudomonas aeruginosa is the most common virulent respiratory pathogen in cystic fibrosis (CF), the longitudinal development of P aeruginosa infection and its effect on antibody responses and lung d...Context: Although Pseudomonas aeruginosa is the most common virulent respiratory pathogen in cystic fibrosis (CF), the longitudinal development of P aeruginosa infection and its effect on antibody responses and lung disease progression in children with CF remain unclear. Objective: To prospectively examine the epidemiology of P aeruginosa infection and its impact on CF pulmonary morbidity. Design, Setting, and Patients: We prospectively evaluated 56 CF patients at 2 CF centers in Madison and Milwaukee, Wis, from birth up to age 16 years between April 15, 1985, and April 15, 2004, with diagnoses made through the Wisconsin CF Neonatal Screening Project. Main Outcome Measures: Timing of nonmucoid P aeruginosa and mucoid P aeruginosa acquisition was assessed by first positive result. Longitudinal development from no P aeruginosa to nonmucoid P aeruginosa and from nonmucoid P aeruginosa to mucoid P aeruginosa was examined. Outcome measurements included antibody titers, respiratory symptoms, quantitative chest radiography, and pulmonary function tests. Results: Sixteen patients (29%) acquired nonmucoid P aeruginosa in the first 6 months of life. The age-specific prevalence of mucoid P aeruginosa increased markedly from age 4 to 16 years. Nonmucoid and mucoid P aeruginosa were acquired at median ages of 1.0 and 13.0 years, respectively. In contrast with the short transition time from no P aeruginosa to nonmucoid P aeruginosa, the transition time from nonmucoid to mucoid P aeruginosa was relatively long (median, 10.9 years) and could be slightly extended by brief/low anti-P aeruginosa antibiotic treatment. Antibody titers increased with both transitions, but the deterioration in cough scores, chest radiograph scores, and pulmonary function correlated best with transition from nonmucoid to mucoid P aeruginosa. Conclusions: Early prevention and detection of nonmucoid andmucoid P aeruginosa are critical because of early acquisition and prevalence. There is a window of opportunity for suppression and possible eradication (by aggressive anti-P aeruginosa treatment) of initial nonmucoid P aeruginosa. Mucoid P aeruginosa plays a much greater role in CF lung disease progression than nonmucoid P aeruginosa. Antibody titers, cough scores, and chest radiographs are early signs of nonmucoid P aeruginosa and especially mucoid P aeruginosa stages.展开更多
Objective: To generate and examine evidence in support of diagnosing cystic fibrosis (CF) early through newborn screening (NBS). Study design: Using a randomized controlled trial with unique unblinding/surveillance,we...Objective: To generate and examine evidence in support of diagnosing cystic fibrosis (CF) early through newborn screening (NBS). Study design: Using a randomized controlled trial with unique unblinding/surveillance,we evaluated patients with CF receiving similar treatment after assignment to an early diagnosis (screened) group or to a control group. Outcomes studied at diagnosis and longitudinally included measures of nutritional status and lung disease. Results: Assessment of patients with CF without meconium ileus who had pancreatic insufficiency revealed marked differences in age and condition at diagnosis -screened patients had significantly better length-/height, weight, and head circumference. Follow-up evaluation for 16 years showed that height and weight differences persisted long term. Although screened patients had better chest x-ray scores at diagnosis, our trial suggests that the effects of confounders such as Pseudomonas aeruginosa infections led to deterioration of their scores after 10 years, but there were no significant differences between the 2 CF/pancreatic insufficiency subgroups. Conclusions: Early diagnosis of CF and aggressive nutritional management can prevent malnutrition and growth failure. Although CF NBS provides a potential opportunity for better pulmonary outcomes, it appears that other factors can predominate over time in pulmonary prognosis. Overall, the Wisconsin trial is positive and provides enough evidence for routine CF NBS.展开更多
Objectives: To extend previous evaluations of costs of cystic fibrosis (CF) diagnosis and examine key issues in assessing the CF cost of care. Study design: Costs for CF newborn screening (NBS) including CF multi-muta...Objectives: To extend previous evaluations of costs of cystic fibrosis (CF) diagnosis and examine key issues in assessing the CF cost of care. Study design: Costs for CF newborn screening (NBS) including CF multi-mutation testing are analyzed by using data from the Wisconsin State Laboratory of Hygiene. Electronic data from 2 Wisconsin CF centers are used to illustrate the complexity of analyzing CF health care utilization and costs. Results: The current cost-per-newborn of a CF multi-mutation test is 50% higher than testing for a single mutation. Data collection for the cost-of-care study requires a combination of electronic and manual data collection; modeling of cost data requires consideration of any censoring. Hospitalizations are shown to have a large impact on costs and show high variability at the individual level. Sixty-nine percent of children with meconium ileus had some hospitalization versus 56% of children without meconium ileus. Conclusion: A cost-benefit analysis of CF multi-mutation testing is warranted. The study of health care cost data is complex and utilization varies between children. Individual-level modeling of CF costs must include factors contributing to the severity of the disease and allow for consideration of individual-level utilization, such as the number of hospitalizations.展开更多
文摘Objective: To determine the impact of age and condition at the time of diagnosis on survival of patients with cystic fi-brosis (CF). Study design: By mode of diagnosis, 27,692 patients documented in the 1986-2000 CF Foundation Registry were segregated into meconium ileus (MI), prenatal or neonatal screening (SCREEN), positive family history only (FH), and symptoms other than MI (SYMPTOM). Patients in the MI, SCREEN, and SYMPTOM groups were further categorized by initial presenting symptoms into combined respiratory symptoms and malnutrition (RESP + NUTR), RESP, NUTR, other less common symptoms (OTHER), and OTHER + RESP/NUTR. Results: Fifty-five percent of patients in the SCREEN group and 59% of patients in the MI group were diagnosed within age 1 month, as contrasted with 5% in patients in the SYMPTOM group (P < .001). Compared with patients in the SCREEN group, patients in the MI and SYMPTOM groups had significantly greater risks of shortened survival. Patients in the SYMPTOM group presenting with RESP + NUTR had significantly greater risk of shortened survival than the SCREEN group (P < .05). Survival of patients in the SYMPTOM group diagnosed “ early,” that is, within 1 month of age, did not differ from patients in the SCREEN group but was significantly better than patients in the SYMPTOM group diagnosed beyond age 1 month to 10 years. Conclusions: Early diagnosis through screening is associated with better survival compared with delayed diagnosis through non-MI symptoms beyond the age of 1 month.
文摘Context: Although Pseudomonas aeruginosa is the most common virulent respiratory pathogen in cystic fibrosis (CF), the longitudinal development of P aeruginosa infection and its effect on antibody responses and lung disease progression in children with CF remain unclear. Objective: To prospectively examine the epidemiology of P aeruginosa infection and its impact on CF pulmonary morbidity. Design, Setting, and Patients: We prospectively evaluated 56 CF patients at 2 CF centers in Madison and Milwaukee, Wis, from birth up to age 16 years between April 15, 1985, and April 15, 2004, with diagnoses made through the Wisconsin CF Neonatal Screening Project. Main Outcome Measures: Timing of nonmucoid P aeruginosa and mucoid P aeruginosa acquisition was assessed by first positive result. Longitudinal development from no P aeruginosa to nonmucoid P aeruginosa and from nonmucoid P aeruginosa to mucoid P aeruginosa was examined. Outcome measurements included antibody titers, respiratory symptoms, quantitative chest radiography, and pulmonary function tests. Results: Sixteen patients (29%) acquired nonmucoid P aeruginosa in the first 6 months of life. The age-specific prevalence of mucoid P aeruginosa increased markedly from age 4 to 16 years. Nonmucoid and mucoid P aeruginosa were acquired at median ages of 1.0 and 13.0 years, respectively. In contrast with the short transition time from no P aeruginosa to nonmucoid P aeruginosa, the transition time from nonmucoid to mucoid P aeruginosa was relatively long (median, 10.9 years) and could be slightly extended by brief/low anti-P aeruginosa antibiotic treatment. Antibody titers increased with both transitions, but the deterioration in cough scores, chest radiograph scores, and pulmonary function correlated best with transition from nonmucoid to mucoid P aeruginosa. Conclusions: Early prevention and detection of nonmucoid andmucoid P aeruginosa are critical because of early acquisition and prevalence. There is a window of opportunity for suppression and possible eradication (by aggressive anti-P aeruginosa treatment) of initial nonmucoid P aeruginosa. Mucoid P aeruginosa plays a much greater role in CF lung disease progression than nonmucoid P aeruginosa. Antibody titers, cough scores, and chest radiographs are early signs of nonmucoid P aeruginosa and especially mucoid P aeruginosa stages.
文摘Objective: To generate and examine evidence in support of diagnosing cystic fibrosis (CF) early through newborn screening (NBS). Study design: Using a randomized controlled trial with unique unblinding/surveillance,we evaluated patients with CF receiving similar treatment after assignment to an early diagnosis (screened) group or to a control group. Outcomes studied at diagnosis and longitudinally included measures of nutritional status and lung disease. Results: Assessment of patients with CF without meconium ileus who had pancreatic insufficiency revealed marked differences in age and condition at diagnosis -screened patients had significantly better length-/height, weight, and head circumference. Follow-up evaluation for 16 years showed that height and weight differences persisted long term. Although screened patients had better chest x-ray scores at diagnosis, our trial suggests that the effects of confounders such as Pseudomonas aeruginosa infections led to deterioration of their scores after 10 years, but there were no significant differences between the 2 CF/pancreatic insufficiency subgroups. Conclusions: Early diagnosis of CF and aggressive nutritional management can prevent malnutrition and growth failure. Although CF NBS provides a potential opportunity for better pulmonary outcomes, it appears that other factors can predominate over time in pulmonary prognosis. Overall, the Wisconsin trial is positive and provides enough evidence for routine CF NBS.
文摘Objectives: To extend previous evaluations of costs of cystic fibrosis (CF) diagnosis and examine key issues in assessing the CF cost of care. Study design: Costs for CF newborn screening (NBS) including CF multi-mutation testing are analyzed by using data from the Wisconsin State Laboratory of Hygiene. Electronic data from 2 Wisconsin CF centers are used to illustrate the complexity of analyzing CF health care utilization and costs. Results: The current cost-per-newborn of a CF multi-mutation test is 50% higher than testing for a single mutation. Data collection for the cost-of-care study requires a combination of electronic and manual data collection; modeling of cost data requires consideration of any censoring. Hospitalizations are shown to have a large impact on costs and show high variability at the individual level. Sixty-nine percent of children with meconium ileus had some hospitalization versus 56% of children without meconium ileus. Conclusion: A cost-benefit analysis of CF multi-mutation testing is warranted. The study of health care cost data is complex and utilization varies between children. Individual-level modeling of CF costs must include factors contributing to the severity of the disease and allow for consideration of individual-level utilization, such as the number of hospitalizations.
