Original ArticleNewborn Screening for Cystic Fibrosis is Associated with Reduced Treatment Intensity
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
References (31)
- et al.
Neonatal screening for cystic fibrosis: long-term clinical balance
Pancreatology
(2001) - et al.
Relationship between socioeconomic status and disease severity in cystic fibrosis
J Pediatr
(1998) - et al.
Improved respiratory prognosis in patients with cystic fibrosis with normal fat absorption
J Pediatr
(1982) Evidence for newborn screening for cystic fibrosis
Paediatr Respir Rev
(2003)- et al.
Cost of care and clinical condition in paediatric cystic fibrosis patients
J Cystic Fibrosis
(2003) - et al.
Cystic fibrosis
Lancet
(2003) - et al.
Newborn screening for cystic fibrosis: ensuring more good than harm
J Pediatr
(2003) Maintaining the horizontal line: early intervention and prevention of CF lung disease
J Cystic Fibrosis
(2004)- et al.
The incidence of cystic fibrosis
Stat Med
(1996) - et al.
Neonatal screening for cystic fibrosis is beneficial even in the context of modern treatment
J Pediatr
(2005)
Nutritional benefits of neonatal screening for cystic fibrosis: Wisconsin Cystic Fibrosis Neonatal Screening Study Group
N Engl J Med
Early diagnosis of cystic fibrosis through neonatal screening prevents severe malnutrition and improves long-term growth: Wisconsin Cystic Fibrosis Neonatal Screening Study Group
Pediatrics
Bronchopulmonary disease in children with cystic fibrosis after early or delayed diagnosis
Am J Respir Crit Care Med
Long term prognosis of patients with cystic fibrosis in relation to early detection by neonatal screening and treatment in a cystic fibrosis centre
Thorax
Influence of neonatal screening and centralized treatment on long-term clinical outcome and survival of CF patients
Eur Respir J
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.