title: The feasibility of conducting clinical trials in infants and children with acute respiratory failure.
abstract: Designing robust clinical trials in critically ill, mechanically ventilated children requires an understanding of the epidemiology and course of pediatric respiratory failure. As part of a clinical trial, we screened all mechanically ventilated children in nine large pediatric intensive care units (ICUs) across North America for 6 consecutive months. Of 6,403 total ICU admissions, 1,096 (17.1%) required mechanical ventilator support for a minimum of 24 hours. Of these, 701 (64%) met one or more exclusion criteria for trial enrollment. Common reasons for exclusion were upper airway obstruction (13.5%) and cyanotic congenital heart disease (11.5%). Life support interventions were restricted for 9.7% of patients, and 5.5% were chronically ventilator dependent. In the patients who were eligible for respiratory failure studies, 62.4% had an acute primary diagnosis of pulmonary disease, 14.2% neurologic disease, and 8.9% cardiac disease. Chronic underlying conditions were present in 43.2% of the patients. The most common acute diagnosis was bronchiolitis in infants (43.6%) and pneumonia in children 1 year old and older (24.5%). Mortality was rare (1.6%), and the median duration of ventilation was 7 days. The design of clinical trials in critically ill children is feasible but must account for the diverse population, infrequent mortality, and short duration of mechanical ventilation.
authors.affiliation: Department of Anesthesia, Children's Hospital, Boston, MA 02115, USA. adrienne.randolph@tch.harvard.edu
authors.forename: Adrienne G
authors.initials: AG
authors.lastname: Randolph
authors.forename: Kathleen L
authors.initials: KL
authors.lastname: Meert
authors.forename: Mary E
authors.initials: ME
authors.lastname: O'Neil
authors.forename: James H
authors.initials: JH
authors.lastname: Hanson
authors.forename: Peter M
authors.initials: PM
authors.lastname: Luckett
authors.forename: John H
authors.initials: JH
authors.lastname: Arnold
authors.forename: Rainer G
authors.initials: RG
authors.lastname: Gedeit
authors.forename: Peter N
authors.initials: PN
authors.lastname: Cox
authors.forename: Joan S
authors.initials: JS
authors.lastname: Roberts
authors.forename: Shekhar T
authors.initials: ST
authors.lastname: Venkataraman
authors.forename: Peter W
authors.initials: PW
authors.lastname: Forbes
authors.forename: Ira M
authors.initials: IM
authors.lastname: Cheifetz
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D005240:Feasibility Studies; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D015278:Intensive Care Units, Pediatric; D008297:Male; D015337:Multicenter Studies as Topic; D011336:Probability; D012016:Reference Values; D012121:Respiration, Artificial / Q000592:standards* / Q000639:trends; D012128:Respiratory Distress Syndrome / Q000175:diagnosis* / Q000628:therapy*; D012127:Respiratory Distress Syndrome, Newborn / Q000175:diagnosis* / Q000628:therapy*; D018570:Risk Assessment; D012720:Severity of Illness Index; D016896:Treatment Outcome