Elsevier

The Journal of Pediatrics

Volume 147, Issue 3, September 2005, Pages 306-311
The Journal of Pediatrics

Original Article
Newborn Screening for Cystic Fibrosis is Associated with Reduced Treatment Intensity

https://doi.org/10.1016/j.jpeds.2005.05.034Get rights and content

Objectives

To determine whether the improved clinical status after newborn screening (NBS) for cystic fibrosis (CF) segregates with increased therapeutic intervention compared with presentation by clinical diagnosis (CD).

Study design

In 2002, two populations (1 to 9 years of age) who presented (excluding meconium ileus) by NBS ≤3 months of age or by CD were compared in an observational, cross-sectional design. NBS and CD populations (184 and 950 patients, respectively) were divided into 3-year age groups (1 to 3, 4 to 6, and 7 to 9 years). Therapies of duration >3 months were compared together with Pseudomonas aeruginosa infection status.

Results

NBS patients ≤6 years of age received significantly fewer and less demanding therapies not explained by age, genotype, geography, or social deprivation. In 7- to 9-year-olds, significantly fewer NBS patients received intravenous antibiotics. NBS patients without P aeruginosa infection received significantly fewer therapies, but no differences were found between intermittently or chronically infected NBS and CD populations. Comparable results were found in ΔF508/ΔF508 subpopulations.

Conclusions

CF populations diagnosed by NBS are associated with reduced treatment compared with age- and genotype-matched CD control subjects.

Section snippets

Methods

UK CF Database (UKCFD) data were collected, verified, and error-checked from 41 CF centers and 12 smaller CF clinics in the years 2001 to 2002, as described recently,2, 13 and at www.cystic-fibrosis.org.uk. All procedures were compliant with multicenter research ethics protocols and UK legislation on patient confidentiality. The study population was identical to that detailed in Sims et al2 and is summarized in the flow diagram in Figure 1 (available online at www.jpeds.com). Study patients

Study Population

Our study populations of NBS and CD patients (of all genotypes, ages 1 to 9 years and with clinical data) contained 184 and 950 patients, respectively (Figure 1; available online at www.jpeds.com). Of these, 98 and 531, respectively, were homozygous for ΔF508. We tested whether these study populations were representative, and there was no significant difference in median age at diagnosis for our NBS (n = 184) and CD (n = 950) study populations when compared with their respective parent registered

Discussion

Whether screening for CF provides long-term benefit remains controversial, given that no improvement in pulmonary outcome has been observed. We recently demonstrated that NBS for CF segregates with some clinical benefit with better median height and lower chest radiography scores as surrogate measures of disease severity for up to 6 years.2 Further, in 3-year, age-matched and genetically homogeneous (for ΔF508/ΔF508 genotype) subpopulations, we also found better clinical outcomes in NBS

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  • Cited by (0)

    Supported by the UK Cystic Fibrosis Trust, the National Services Division of the National Health Service (Scotland), and the Scottish Higher Education Funding Council (J.M.C. and A.M.).

    E.J.S. and G.M. are employed by the University of Dundee.

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