A Clinical Prediction Rule for the Severity of Congenital Diaphragmatic Hernias in Newborns WHAT’S KNOWN ON THIS SUBJECT: Predicting high-risk populations in congenital diaphragmatic hernia (CDH) can help target care strategies. Prediction rules for infants with CDH often lack validation, are aimed at a prenatal population, and are of limited generalizability. We cannot currently discriminate the highest risk neonates during the crucial period shortly after birth. WHAT THIS STUDY ADDS: This clinical prediction rule was developed and validated on an international database. It discriminates patients and high, intermediate, and low risk of mortality; is easy to apply; and is generalizable to most infants with CDH. abstract BACKGROUND: Congenital diaphragmatic hernia (CDH) is a condition with a highly variable outcome. Some infants have a relatively mild disease process, whereas others have significant pulmonary hypopla- sia and hypertension. Identifying high-risk infants postnatally may allow for targeted therapy. METHODS: Data were obtained on 2202 infants from the Congenital Diaphragmatic Hernia Study Group database from January 2007 to Oc- tober 2011. Using binary baseline predictors generated from birth weight, 5-minute Apgar score, congenital heart anomalies, and chro- mosome anomalies, as well as echocardiographic evidence of pulmo- nary hypertension, a clinical prediction rule was developed on a randomly selected subset of the data by using a backward selection algorithm. An integer-based clinical prediction rule was created. The performance of the model was validated by using the remaining data in terms of calibration and discrimination. RESULTS: The final model included the following predictors: very low birth weight, absent or low 5-minute Apgar score, presence of chro- mosomal or major cardiac anomaly, and suprasystemic pulmonary hy- pertension. This model discriminated between a population at high risk of death (∼50%) intermediate risk ( ∼20%), or low risk (,10%). The model performed well, with a C statistic of 0.806 in the derivation set and 0.769 in the validation set and good calibration (Hosmer-Lemeshow test, P = .2). CONCLUSIONS: A simple, generalizable scoring system was developed for CDH that can be calculated rapidly at the bedside. Using this model, intermediate- and high-risk infants could be selected for transfer to high-volume centers while infants at highest risk could be considered for advanced medical therapies. Pediatrics 2014;134:e413–e419 AUTHORS: Mary Elizabeth Brindle, MD, MPH, a Earl Francis Cook, ScD, b Dick Tibboel, MD, PhD, c Pamela A. Lally, MD, d and Kevin P. Lally, MD, MSc, d on behalf of the Congenital Diaphragmatic Hernia Study Group a Department of Surgery, University of Calgary, Calgary, Alberta, Canada; b Division of General Internal Medicine and Primary Care, Brigham and Women’ s Hospital, Boston, Massachusetts; c Erasmus MC–Sophia Children’ s Hospital, Rotterdam, Netherlands; and d Department of Pediatric Surgery, Utah Health Medical School and Children’ s Memorial Hermann Hospital, Houston, Texas KEY WORDS clinical prediction, congenital diaphragmatic hernia, population- based, survival ABBREVIATIONS CDH—congenital diaphragmatic hernia CDHSG—Congenital Diaphragmatic Hernia Study Group Dr Brindle conceptualized and designed the study, and drafted the initial manuscript; Dr Cook provided guidance with the statistical design of the study, analyses, and interpretation of the data and critically reviewed the manuscript; Dr Tibboel assisted with interpretation of data and manuscript revisions; Dr P. Lally coordinated and supervised data collection in the Congenital Diaphragmatic Hernia Study Group database, provided insight and feedback in terms of interpretation of the data, and critically reviewed the manuscript; and Dr K. Lally provided oversight in terms of study design and interpretation and critically reviewed and revised the manuscript. All authors approved the final manuscript as submitted. www.pediatrics.org/cgi/doi/10.1542/peds.2013-3367 doi:10.1542/peds.2013-3367 Accepted for publication May 15, 2014 Address correspondence to: Mary Elizabeth Brindle, MD, MPH, 2888 Shaganappi Trail NW, Calgary, AB T3B6A8 Canada. E-mail: maryebrindle@gmail.com PEDIATRICS (ISSN Numbers: Print, 0031-4005; Online, 1098-4275). Copyright © 2014 by the American Academy of Pediatrics FINANCIAL DISCLOSURE: The authors have indicated they have no financial relationships relevant to this article to disclose. FUNDING: This study was funded by the Alberta Children’ s Hospital Foundation Professorship. POTENTIAL CONFLICT OF INTEREST: The authors have indicated they have no potential conflicts of interest to disclose. PEDIATRICS Volume 134, Number 2, August 2014 e413 ARTICLE by guest on August 31, 2017 Downloaded from