The OPTIMIST-A trial: evaluation of minimally-invasive surfactant therapy in preterm infants 25-28 weeks gestation

BMC Pediatr. 2014 Aug 27:14:213. doi: 10.1186/1471-2431-14-213.

Abstract

Background: It is now recognized that preterm infants ≤28 weeks gestation can be effectively supported from the outset with nasal continuous positive airway pressure. However, this form of respiratory therapy may fail to adequately support those infants with significant surfactant deficiency, with the result that intubation and delayed surfactant therapy are then required. Infants following this path are known to have a higher risk of adverse outcomes, including death, bronchopulmonary dysplasia and other morbidities. In an effort to circumvent this problem, techniques of minimally-invasive surfactant therapy have been developed, in which exogenous surfactant is administered to a spontaneously breathing infant who can then remain on continuous positive airway pressure. A method of surfactant delivery using a semi-rigid surfactant instillation catheter briefly passed into the trachea (the "Hobart method") has been shown to be feasible and potentially effective, and now requires evaluation in a randomised controlled trial.

Methods/design: This is a multicentre, randomised, masked, controlled trial in preterm infants 25-28 weeks gestation. Infants are eligible if managed on continuous positive airway pressure without prior intubation, and requiring FiO2 ≥ 0.30 at an age ≤6 hours. Randomisation will be to receive exogenous surfactant (200 mg/kg poractant alfa) via the Hobart method, or sham treatment. Infants in both groups will thereafter remain on continuous positive airway pressure unless intubation criteria are reached (FiO2 ≥ 0.45, unremitting apnoea or persistent acidosis). Primary outcome is the composite of death or physiological bronchopulmonary dysplasia, with secondary outcomes including incidence of death; major neonatal morbidities; durations of all modes of respiratory support and hospitalisation; safety of the Hobart method; and outcome at 2 years. A total of 606 infants will be enrolled. The trial will be conducted in >30 centres worldwide, and is expected to be completed by end-2017.

Discussion: Minimally-invasive surfactant therapy has the potential to ease the burden of respiratory morbidity in preterm infants. The trial will provide definitive evidence on the effectiveness of this approach in the care of preterm infants born at 25-28 weeks gestation.

Trial registration: Australia and New Zealand Clinical Trial Registry: ACTRN12611000916943; ClinicalTrials.gov: NCT02140580.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Video-Audio Media

MeSH terms

  • Biological Products / therapeutic use*
  • Bronchopulmonary Dysplasia / etiology
  • Bronchopulmonary Dysplasia / prevention & control
  • Clinical Protocols
  • Combined Modality Therapy
  • Continuous Positive Airway Pressure
  • Humans
  • Infant, Newborn
  • Infant, Premature
  • Intubation, Intratracheal
  • Phospholipids / therapeutic use*
  • Pulmonary Surfactants / therapeutic use*
  • Respiratory Distress Syndrome, Newborn / complications
  • Respiratory Distress Syndrome, Newborn / drug therapy*
  • Respiratory Distress Syndrome, Newborn / therapy
  • Single-Blind Method
  • Treatment Outcome

Substances

  • Biological Products
  • Phospholipids
  • Pulmonary Surfactants
  • poractant alfa

Associated data

  • ANZCTR/ACTRN12611000916943
  • ClinicalTrials.gov/NCT02140580