Fibrosis quistica

Cystic Fibrosis Diagnosed After 2 Months of Age Leads to Worse Outcomes and Requires More Therapy Erika J. Sims, Allan Clark, Jonathan McCormick, Gita Mehta, Gary Connett, Anil Mehta and on behalf of the United Kingdom Cystic Fibrosis Database Steering Committee Pediatrics 2007;119;19-28 DOI: 10.1542/peds.2006-1498

The online version of this article, along with updated information andservices, is located on the World Wide Web at: http://www.pediatrics.org/cgi/content/full/119/1/19

PEDIATRICS is the official journal of the American Academy of Pediatrics. A monthly publication, it has been published continuously since 1948. PEDIATRICS is owned, published, and trademarked by the American Academy of Pediatrics, 141 Northwest Point Boulevard, Elk Grove Village, Illinois, 60007.Copyright © 2007 by the American Academy of Pediatrics. All rights reserved. Print ISSN: 0031-4005. Online ISSN: 1098-4275.

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ARTICLE

Cystic Fibrosis Diagnosed After 2 Months of Age Leads to Worse Outcomes and Requires More Therapy
Erika J. Sims, PhDa,b, Allan Clark, PhDb, Jonathan McCormick, MDc, Gita Mehta, MPhila, Gary Connett, FRCPCHd,Anil Mehta, FRCP(Edin)a, on behalf of the United Kingdom Cystic Fibrosis Database Steering Committee United Kingdom Cystic Fibrosis Database, Division of Maternal and Child Health Sciences, Ninewells Hospital and Medical School, University of Dundee, Dundee, Scotland, United Kingdom; bDepartment of Population Health, School of Medicine, Health Policy and Practice, University of East Anglia,Norwich, United Kingdom; cRespiratory Unit, Royal Hospital for Sick Children, Yorkhill National Health Service Trust, Glasgow, United Kingdom; dDepartment of Paediatrics, Southampton University Hospitals National Health Service Trust, Southampton, Hampshire, United Kingdom
Financial Disclosure: Dr Sims and Ms G. Mehta were funded by a grant awarded by the Cystic Fibrosis Trust at the time this studywas performed. Dr A. Mehta was the director of the United Kingdom Cystic Fibrosis Database and received grants from the Cystic Fibrosis Trust. Drs Clark, McCormick, and Connett have indicated they have no financial relationships relevant to this article to disclose.
a

ABSTRACT
OBJECTIVE. Newborn screening for cystic fibrosis remains controversial because improved pulmonary function has not beenestablished. Studies to date have not accounted for differences in treatments delivered to clinically diagnosed children and newborn-screened controls. Here, we compare outcomes and treatment of patients clinically diagnosed within the newborn-screening reporting window (early-clinically diagnosed), those presenting after this period (late-clinically diagnosed), and patients diagnosed by newbornscreening. PATIENTS AND METHODS. In a cross-sectional analysis of cohorts retrospectively ascer-

www.pediatrics.org/cgi/doi/10.1542/ peds.2006-1498 doi:10.1542/peds.2006-1498
Key Words cystic fibrosis, newborn screening, clinical diagnosis Abbreviations CF— cystic fibrosis CD— clinical diagnosis MI—meconium ileus NBS—newborn screening UKCFD—United Kingdom Cystic Fibrosis Database YDC—most recentyear of data collection SK—Shwachman-Kulczyki morbidity NEBS—nebulized therapies FEV1—forced expiratory volume in 1 second RR—relative risk CI— confidence interval rhDNase—recombinant human deoxyribonuclease IRT—immunoreactive trypsinogen
Accepted for publication Sep 25, 2006 Address correspondence to Erika J. Sims, PhD, School of Medicine, Health Policy and Practice, University of East Anglia,University Plain, Norwich NR4 7TJ, United Kingdom. E-mail: e.sims@uea.ac.uk PEDIATRICS (ISSN Numbers: Print, 0031-4005; Online, 1098-4275). Copyright © 2007 by the American Academy of Pediatrics

tained, patients who were homozygous F508 with cystic fibrosis, attending specialist cystic fibrosis centers, and 1 to 10 years of age between 2000 and 2002 were identified from the United Kingdom Cystic...