Geographic variations in cystic fibrosis: An analysis of the U.S. CF Foundation Registry

Kopp, Benjamin T.; Nicholson, Lisa; Paul, Grace; Tobias, Joseph D.; Ramanathan, Chandar; Hayes, Don

doi:10.1002/ppul.23185

Cited by 24 publications

(14 citation statements)

References 22 publications

(33 reference statements)

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“…There are approximately 1000 new cases of Cystic Fibrosis (CF) diagnosed annually in North America with recent incidence estimates in the US state of Wisconsin estimating incidence at between 1.67 and 2.98 cases per 10 000 births per year, making it one of the most common life shortening genetic disorders in Caucasians. There are significant regional variations in many CF related markers, and newborn screening (NBS) for CF became universal in the United States in 2010 when Texas became the final state to screen all newborns for this disease . Prior to the implementation of NBS (and in countries where NBS is not performed), many children were diagnosed after presenting with failure to thrive, recurrent respiratory infections and chronic cough, rectal prolapse, and electrolyte abnormalities .…”

Section: Introductionmentioning

confidence: 99%

“…There are approximately 1000 new cases of Cystic Fibrosis (CF) diagnosed annually in North America 1 with recent incidence estimates in the US state of Wisconsin estimating incidence at between 1.67 and 2.98 cases per 10 000 births per year, 2 making it one of the most common life shortening genetic disorders in Caucasians. There are significant regional variations in many CF related markers, 3…”

Section: Introductionmentioning

confidence: 99%

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Clinical outcomes in U.S. infants with cystic fibrosis from 2001 to 2012

Hoch

Sontag

Scarbro

et al. 2018

Pediatric Pulmonology

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Background All 50 United States implemented newborn screening (NBS) for cystic fibrosis (CF) by 2010. The purpose of this study was to evaluate trends over the decade when NBS became universal to determine current rates of malnutrition, stunting, and infection rates in U.S. infants with CF. Methods Annual data were obtained on infants with CF up to 24 months of age diagnosed between 2001 and 2010 in the CF Foundation Patient Registry (CFFPR), in both the years of and after diagnosis, including method of diagnosis, demographics, and growth parameters and microbiology. Results Data were obtained on 8178 infants diagnosed with CF. The percentage of infants diagnosed by NBS increased from 15% in 2001‐83% in 2012 (P < 0.001). Mean weight, length, and weight‐for‐length z‐scores in the year of diagnosis increased from 2001 to 2012 (Wt z‐score 2001: −1.32 (SD 1.41), 2012: −0.72 (SD 1.12); Ht z‐score 2001: −1.32 (SD 1.57), 2012 −0.60 (SD 1.21); Wt/Ht Z score 2001: −0.54 (SD 1.18), 2012: 0.06 (SD 1.05); P < 0.001 for each). The proportion of infants on pancreatic enzymes decreased from 94% in 2001‐83% in 2012 (P < 0.0001). Pseudomonas aeruginosa culture positivity in the diagnosis year decreased significantly (27% in 2001, 15% in 2012, P < 0.001). Conclusions Nationwide implementation of CF NBS is temporally associated with significant improvements in growth outcomes and reductions in P. aeruginosa infections. Current rates of malnutrition, stunting, and airway infection present a target for early intervention and quality improvement efforts.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Clinical outcomes in U.S. infants with cystic fibrosis from 2001 to 2012

Hoch

Sontag

Scarbro

et al. 2018

Pediatric Pulmonology

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“…The single center represents a small sample of the CF population and has a small number of patients receiving CFTR modulators. However, focusing on a single center allows for common exposures that may be risk factors for acquisition and transmission of infections . We selected fixed time intervals for both eras since infections may fluctuate with time .…”

Section: Limitationsmentioning

confidence: 99%

“…However, focusing on a single center allows for common exposures that may be risk factors for acquisition and transmission of infections. 39 We selected fixed time intervals for both eras since infections may fluctuate with time. 40 We were not able to control for some potential confounding variables.…”

Section: Limitationsmentioning

confidence: 99%

Pathogen acquisition in patients with cystic fibrosis receiving ivacaftor or lumacaftor/ivacaftor

Singh

McLearn‐Montz

Milavetz

et al. 2019

Pediatric Pulmonology

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Background The cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor and lumacaftor/ivacaftor improve the status of existing infections in patients with cystic fibrosis (CF). It is unknown how well these drugs protect patients against incident infections. We hypothesized that CFTR modulator treatment would decrease new infections with Pseudomonas aeruginosa or Staphylococcus aureus. Methods We retrospectively studied a single‐center cohort of patients with CF during two time periods (2008‐2011, Era 1) and (2012‐2015, Era 2) based on the January 2012 approval of ivacaftor. Using Kaplan–Meier analysis, we compared the time to any new infection with P. aeruginosa, methicillin‐resistant S. aureus (MRSA), or methicillin‐sensitive S. aureus (MSSA) that was absent during a 2‐year baseline. We stratified the analysis based on whether patients received ivacaftor or lumacaftor/ivacaftor during Era 2. We used the log‐rank test and considered P < 0.05 statistically significant. Results For patients receiving ivacaftor or lumacaftor/ivacaftor in Era 2, there was a statistically significant delay in the time to new bacterial acquisition in Era 2 vs. Era 1 ( P = 0.008). For patients who did not receive CFTR modulators, there was a trend toward slower acquisition of new bacterial infections in Era 2 compared to Era 1, but this was not statistically significant ( P = 0.10). Conclusions Patients receiving ivacaftor or lumacaftor/ivacaftor for CF had significantly delayed acquisition of P. aeruginosa and S. aureus after these drugs were released. This method for analyzing incident infections may be useful for future studies of CFTR modulators and bacterial acquisition in CF registry cohorts.

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“…An analysis of the US CFFPR grouped geographically 30,896 subjects with CF reported 2007–2012 based upon the Nationwide Inpatient Sample classification . Significant differences in racial distribution were seen; half of the total African American CF population resides in the South.…”

Section: Epidemiologymentioning

confidence: 99%