title: Summary proceedings from the bronchopulmonary dysplasia group.
abstract: Despite improvements in neonatal care, bronchopulmonary dysplasia (BPD) continues to occur in approximately one third of newborns who have birth weights of <1000 g and contributes to significant morbidity in this population. Gaps in knowledge about the prevention and treatment of BPD remain, resulting in unintended short- and long-term sequelae. In addition to chronic lung disease, preterm newborns with BPD are more likely to develop language delay, cerebral palsy, and cognitive impairments compared with preterm newborns without BPD. The pulmonary group identified 3 critical needs to enhance the design of clinical trials in neonates with BPD: (1) identify the stages of BPD; (2) define BPD more clearly; and (3) identify subtypes of BPD patients. The group determined that trials are needed for 3 areas of BPD: (1) prevention of BPD; (2) treatment of evolving BPD; and (3) treatment of established BPD. The severity of BPD is defined as mild, moderate, and severe, and subgroups among those with BPD are described. Here we identify gaps in basic science and pharmacologic knowledge that hamper investigators' ability to conduct effective BPD clinical trials and provide a list of drugs to be studied in BPD trials. Priorities for drug-class evaluation by stage of BPD are given. The pulmonary group proposes a BPD clinical-trials framework that varies according to the different stages of BPD and describes characteristics of the overall design for BPD clinical trials. Finally, we discuss trial-design issues that are common to all neonatal studies.
authors.affiliation: Division of Neonatology, Rainbow Babies & Children's Hospital, Department of Pediatrics, Case Western Reserve University, 11100 Euclid Ave, Mailstop 6010, Cleveland, Ohio 44106-6010, USA. msw3@po.cwru.edu
authors.forename: Michele C
authors.initials: MC
authors.lastname: Walsh
authors.forename: Stanley
authors.initials: S
authors.lastname: Szefler
authors.forename: Jonathan
authors.initials: J
authors.lastname: Davis
authors.forename: Marilee
authors.initials: M
authors.lastname: Allen
authors.forename: Linda
authors.initials: L
authors.lastname: Van Marter
authors.forename: Steve
authors.initials: S
authors.lastname: Abman
authors.forename: Lillian
authors.initials: L
authors.lastname: Blackmon
authors.forename: Alan
authors.initials: A
authors.lastname: Jobe
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D001997:Bronchopulmonary Dysplasia / Q000145:classification / Q000188:drug therapy* / Q000517:prevention & control; D006801:Humans; D007231:Infant, Newborn; D007234:Infant, Premature; D019102:Infant, Very Low Birth Weight; D016032:Randomized Controlled Trials as Topic / Q000379:methods; D012720:Severity of Illness Index
chemical_list: D000305:Adrenal Cortex Hormones