Objective To compare outcomes of infants and children who underwent lung transplantation for genetic disorders of surfactant metabolism (SFTPB, SFTPC, ABCA3, and NKX2-1) over 2 epochs (1993-2003 and 2004-2015) at St Louis Children's Hospital. Study design We retrospectively reviewed clinical characteristics, mortality, and short- and long-term morbidities of infants (transplanted at <1 year; n=28) and children (transplanted >1 year; n=16) and compared outcomes by age at transplantation (infants vs children) and by epoch of transplantation. Results Infants underwent transplantation more frequently for surfactant protein-B deficiency, whereas children underwent transplantation more frequently for SFTPC mutations. Both infants and children underwent transplantation for ABCA3 deficiency. Compared with children, infants experienced shorter times from listing to transplantation (P=.014), were more likely to be mechanically ventilated at the time of transplantation (P<.0001), were less likely to develop bronchiolitis obliterans post-transplantation (P=.021), and were more likely to have speech and motor delays (P≤.0001). Despite advances in genetic diagnosis, immunosuppressive therapies, and supportive respiratory and nutritional therapies, mortality did not differ between infants and children (P=.076) or between epochs. Kaplan-Meier analyses demonstrated that children transplanted in epoch 1 (1993-2003) were more likely to develop systemic hypertension (P=.049) and less likely to develop post-transplantation lymphoproliferative disorder compared with children transplanted in epoch 2 (2004-2015) (P=.051). Conclusion Post-lung transplantation morbidities and mortality remain substantial for infants and children with genetic disorders of surfactant metabolism.

Original languageEnglish
Pages (from-to)157-164.e2
JournalJournal of Pediatrics
StatePublished - May 2017


  • ABCA3
  • NKX2.1
  • RDS
  • chILD
  • childhood interstitial lung disease
  • neonatal respiratory distress syndrome
  • pediatric lung transplantation
  • surfactant protein B
  • surfactant protein C


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