Influence of neonatal screening and centralized treatment on long-term clinical outcome and survival of CF patients.
After an experimental neonatal screening program for cystic fibrosis (CF) from 1973-1979, a follow-up study took place from 1980-1997. Patients were treated at specialized centres (C) or at local hospitals (non-C). Aims of the study were: 1) to determine whether the previously reported benefits from screening persisted with time and after adjustment for confounding variables; and 2) to investigate whether centre treatment was associated with improved prognosis of CF patients. Prognosis of patients detected by screening (S; n=24) was compared with patients detected clinically, born during (non-S; n=29) and after the screening programme (post-S; n=39). In addition, prognosis was compared between 45 C and 47 non-C patients. Multivariable regression analysis was used to compare survival and mixed-effects model regression analysis was used to compare clinical outcome between patients. The analyses included the variables screening, centre treatment, sex, meconium ileus and genotype. S patients had a significantly smaller decline in forced expiratory volume in one second (FEVI) (difference +2.74% predicted) and significantly lower immunoglobulin-G (IgG) levels (difference -473.69 mg x dL(-1)) than non-S patients until 12 yrs of age. At 12 yrs of age, vital capacity was significantly higher in S patients than in non-S patients (difference +362.79 mL). Survival seemed to be best for S patients compared to both non-S and post-S patients. Post-S patients were significantly heavier (difference in SD weight +0.77), had a significantly smaller decline in FEV1 (difference +2.80% pred) and lower IgG levels (difference -453.04 mg x dL(-1)) than non-S patients until 12 yrs of age. C patients had a significantly improved survival (relative risk (RR) 0.18, 95% confidence interval 0.05-0.57) than non-C patients. Early diagnosis through neonatal screening leads to better preservation of lung function in the long term in cystic fibrosis patients. Management of cystic fibrosis patients in specialized centres improves survival.
Mérelle
M E
ME
Dept of Pediatrics, Medical Centre Vrije Universiteit, Amsterdam, The Netherlands.
Schouten
J P
JP
Gerritsen
J
J
Dankert-Roelse
J E
JE
D001827:Body Height; D001835:Body Weight; D003550:Cystic Fibrosis / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality* / Q000503:physiopathology; D005260:Female; D005500:Follow-Up Studies; D005541:Forced Expiratory Volume; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D015999:Multivariate Analysis; D015997:Neonatal Screening; D009426:Netherlands / Q000453:epidemiology; D011379:Prognosis; D012044:Regression Analysis; D012307:Risk Factors; D016019:Survival Analysis; D013997:Time Factors; D016896:Treatment Outcome