Study design (if review, criteria of inclusion for studies)
Retrospective analysis of the RCT cohort utilized longitudinal outcome measures obtained from the Cystic Fibrosis Foundation Patient Registry (CFFPR).
Participants
145 subjects who consented to the original study (The Wisconsin Cystic Fibrosis Neonatal Screening Project ), 104 subjects met inclusion criteria and had adequate data in the CFFPR.
Interventions
An early diagnosis of CF via newborn screening (NBS) vs diagnosis by clinical symptoms
Outcome measures
Clinical characteristics, percent predicted forced expiratory volume in 1 s (ppFEV(1)) and mortality. A random intercept model was used to compare the ppFEV(1) decline of subjects between the two groups up to age 26 years. Mortality was analyzed using the Kaplan-Meier method.
Main results
Of the 145 subjects who consented to the original study, 104 subjects met inclusion criteria and had adequate data in the CFFPR. Of 57 subjects in the screened group and 47 in the control group, the rates of ppFEV(1) decline were 1.76%/year (95% CI 1.62 to 1.91%) and 1.43%/year (95% CI 1.26 to 1.60%), respectively (p<0.0002). Pseudomonas aeruginosa acquired before 2 years was partially responsible. There was no difference in mortality between the two groups.
Authors' conclusions
NBS alone does not improve pulmonary outcomes in CF, particularly when other risk factors supervene. In an era prior to strict infection control and current therapies, NBS for CF may be associated with worse pulmonary outcomes.