Background: The first clinical trial of A1PI supplementation for the prevention of chronic lung disease of prematurity (CLD) was recently completed and short term outcome reported. 106 infants, with birth weights of 600-1250g who required respiratory support were enrolled (53 A1P1 (Rx), 53 controls (C)). A1PI appeared to protect against developing CLD but there was a trend towards increased total but not severe IVH in the Rx group. Aim: To determine, as part of a safety assessment, the neurodevelopmental outcome of the above study infants. Methods: Study infants were enrolled prospectively into the neonatal follow-up program and assessed at standard intervals to >18 months of age for evidence of neurological, cognitive, visual or auditory deficits. Infants were classified as normal, mildly impaired (motor abnormality without significant functional limitation,±mild hearing loss,±severe myopia,±developmental quotient (DQ)>1<2sd below mean) or severely impaired (motor abnormality with significant functional limitation,±deaf,±blind,±DQ>2sd below mean). Outcome is presented for all babies followed but 5 have not been assessed at more than 12 months. Results: 83 of 94 surviving infants (88%) returned for assessment (40Rx, 43 C). There were no differences between Rx and C groups with respect to gestational age, birth weight, sex ratio, RDS or IVH. The incidence of CLD was lower in the Rx group followed. There were no differences in neurodevelopmental outcome between the two groups (p=.85)(see table). Conclusion: Despite a trend towards an increased incidence of IVH in babies treated with A1PI in a RCT, there was no increase in adverse neurodevelopmental outcome in survivors. A1PI therapy in very preterm neonates appears to be safe and may protect against the development of CLD. Further trials are warranted.

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