Early Life Growth Trajectories in Cystic Fibrosis are Associated with Pulmonary Function at Age 6 Years.

OBJECTIVE: To determine whether severity of lung disease at age 6 years is associated with changes in nutritional status before age 6 within individual children with cystic fibrosis (CF). STUDY DESIGN: Children with CF born between 1994 and 2005 and followed in the CF Foundation Patient Registry from age ≤2 through 7 years were assessed according to changes in annualized weight-for-length (WFL) percentiles between ages 0 and 2 years and body mass index (BMI) percentiles between ages 2 and 6 years. The association between growth trajectories before age 6 and forced expiratory volume in 1 second (FEV1)% predicted at age 6-7 years was evaluated using multivariable linear regression. RESULTS: A total of 6805 subjects met inclusion criteria. Children with annualized WFL-BMI always >50th percentile (N = 1323 [19%]) had the highest adjusted mean (95% CI) FEV1 at 6-7 years (101.8 [100.1, 103.5]). FEV1 at 6-7 years for children whose WFL-BMI increased >10 percentile points by age 6 years was 98.3 (96.6, 100.0). This was statistically significantly higher than FEV1 for children whose WFL-BMI was stable (94.4 [92.6, 96.2]) or decreased >10 percentile points (92.9 [91.1, 94.8]). Among children whose WFL-BMI increased >10 percentile points, achieving and maintaining WFL-BMI >50th percentile at younger ages was associated with significantly higher FEV1 at 6-7 years. CONCLUSIONS: Within-patient changes in nutritional status in the first 6 years of life are significantly associated with FEV1 at age 6-7 years. The establishment of a clear relationship between early childhood growth measurements and later lung function suggests that early nutritional interventions may impact on eventual lung health.

The National Spina Bifida Patient Registry: profile of a large cohort of participants from the first 10 clinics.

OBJECTIVE: To use data from the US National Spina Bifida Patient Registry (NSBPR) to describe variations in Contexts of Care, Processes of Care, and Health Outcomes among individuals with spina bifida (SB) receiving care in 10 clinics. STUDY DESIGN: Reported here are baseline cross-sectional data representing the first visit of 2172 participants from 10 specialized, multidisciplinary SB clinics participating in the NSBPR. We used descriptive statistics, the Fisher exact test, χ(2) test, and Wilcoxon rank-sum test to examine the data. RESULTS: The mean age was 10.1 (SD 8.1) years with slightly more female subjects (52.5%). The majority was white (63.4%) and relied upon public insurance (53.5%). One-third had sacral lesions, 44.8% had mid-low lumbar lesions, and 24.9% had high lumbar and thoracic lesions. The most common surgery was ventricular shunt placement (65.7%). The most common bladder-management technique among those with bladder impairment was intermittent catheterization (69.0%). Almost 14% experienced a pressure ulcer in the last year. Of those ages 5 years or older with bowel or bladder impairments, almost 30% were continent of stool; a similar percentage was continent of urine. Most variables were associated with type of SB diagnosis. CONCLUSION: The NSBPR provides a cross section of a predominantly pediatric population of patients followed in specialized SB programs. There were wide variations in the variables studied and major differences in Context of Care, Processes of Care, and Health Outcomes by type of SB. Such wide variation and the differences by type of SB should be considered in future analyses of outcomes.

Pancreatic enzyme replacement therapy dosing and nutritional outcomes in children with cystic fibrosis.

OBJECTIVES: To utilize the Cystic Fibrosis Foundation Patient Registry to evaluate whether pancreatic enzyme dose is associated with better nutritional status as measured by average body mass index (BMI) percentile. STUDY DESIGN: A retrospective analysis of the Cystic Fibrosis Foundation Patient Registry from 2005-2008 was performed. The final analysis included 42 561 patient visits from 14 482 patients 2-20 years of age taking pancreatic enzyme replacement therapy from 179 programs. Cystic fibrosis care programs were assigned to quartiles based on adjusted mean patient BMI percentiles. Differences in median lipase dose between programs in the highest and lowest BMI quartiles were examined using a mixed effects model that adjusted for individual patient BMI, age, race, ethnicity, forced expiratory volume in 1 second percent, acid-blocker use, presence of Pseudomonas aeruginosa, nutritional supplement use, growth hormone use, and diagnosis of cystic fibrosis-related diabetes. RESULTS: A significant difference in median enzyme dose existed between the highest and lowest BMI quartiles. Multivariable analysis demonstrated the effect persisted after adjustment for covariates. Highest quartile programs had a median enzyme dose of 1755 lipase units/kg/meal compared with 1628 lipase units/kg/meal for lowest quartile programs. CONCLUSION: Patients attending US cystic fibrosis programs achieving highest nutritional outcomes, measured by mean BMI percentile, have higher enzyme dosing than those attending programs at lower performance levels. Further randomized clinical trials are necessary to determine the role of enzyme dose in improving nutritional outcomes.

Socioeconomic status and the likelihood of antibiotic treatment for signs and symptoms of pulmonary exacerbation in children with cystic fibrosis.

OBJECTIVE: To determine whether socioeconomic status (SES) influences the likelihood of antibiotic treatment of pulmonary exacerbations in patients with cystic fibrosis (CF). STUDY DESIGN: We used data on 9895 patients ≤ 18 years old from the Epidemiologic Study of CF. After establishing an individual baseline of clinical signs and symptoms, we ascertained whether antibiotics were prescribed when new signs/symptoms suggested a pulmonary exacerbation, adjusting for sex, presence of Pseudomonas aeruginosa, the number of new signs/symptoms, and baseline disease severity. RESULTS: In a 12-month period, 20.0% of patients <6 years of age, 33.8% of patients 6 to 12 years of age, and 41.4% of patients 13 to 18 years of age were treated with any (oral, intravenous (IV), or inhaled) antibiotics; the percentage receiving IV antibiotics was 7.3%, 15.2%, and 20.9%, respectively. SES had little effect on treatment for pulmonary exacerbation with any antibiotics, but IV antibiotics were prescribed more frequently for patients with lower SES. CONCLUSIONS: SES-related disparities in CF health outcomes do not appear to be explained by differential treatment of pulmonary exacerbations.

Association of socioeconomic status with the use of chronic therapies and healthcare utilization in children with cystic fibrosis.

OBJECTIVE: To determine whether previously reported socioeconomic status (SES)-related disparities in cystic fibrosis (CF) health outcomes vary by the indicator used (median household income by zip code [MIZ], maternal educational attainment [MEA], and state insurance coverage [MA]), and whether these disparities can be explained by differences in medical treatment. STUDY DESIGN: A cross-sectional analysis of data on patients age <18 years from the Epidemiologic Study of Cystic Fibrosis (ESCF). RESULTS: Disease severity showed a similar inverse correlation with all 3 SES measures. The number of stable clinic visits was unrelated to SES. Patients with MA had more sick outpatient visits and more courses of intravenous (IV) antibiotics for pulmonary exacerbations, and were more likely to be prescribed all chronic therapies. Low-MIZ patients had slightly fewer sick visits and more courses of IV antibiotics, and were more likely to receive oral nutrition supplements but less likely to receive macrolide prescriptions. Low-MEA patients were less likely to receive IV antibiotics at home, more likely to receive oral nutrition supplements, but less likely to receive macrolide prescriptions. CONCLUSIONS: CF health outcomes are correlated with the SES spectrum, but these disparities are not explained by differential use of health services or prescription of chronic therapy. Future investigations should focus on the possible impact of environmental exposures and differences in disease self-management.

Follow-up study of a randomized controlled trial of postnatal dexamethasone therapy in very low birth weight infants: effects on pulmonary outcomes at age 8 to 11 years.

OBJECTIVE: To determine whether postnatal dexamethasone (DEX) exposure affects pulmonary outcomes at school age in children born with very low birth weight (VLBW). STUDY DESIGN: Follow-up study of 68 VLBW children who participated in a randomized controlled trial of postnatal DEX. Pulmonary function was assessed by spirometry. Current asthma status was obtained from a parent. RESULTS: Sixty-eight percent of the placebo group had below-normal forced expiratory volume in 1 second (FEV1), compared with 40% of the DEX group (chi2 = 4.84; P = .03), with trends for lower forced vital capacity (FVC) and FEV1 values in the placebo group. Fifty percent of the placebo group and 34% of DEX group had below normal FEV1/FVC (chi2 =1.59; P =.21). Parent-reported prevalence of asthma did not differ between groups. Logistic regression analysis suggested that the positive effects of DEX on pulmonary function at follow-up were mediated in part by shortened exposure to mechanical ventilation. CONCLUSIONS: Postnatal DEX exposure was associated with higher expiratory flow with no adverse effects on pulmonary outcomes at school age. The prevalences of asthma and impaired pulmonary function underscore the influence of neonatal illness on health at school age, and stress the importance of repeated follow-up examinations of these children.