Outcomes of Lung Transplantation for Infants and Children with Genetic Disorders of Surfactant Metabolism

Whitney B. Eldridge, Qunyuan Zhang, Albert Faro, Stuart C. Sweet, Pirooz Eghtesady, Aaron Hamvas, F. Sessions Cole, Jennifer A. Wambach*

*Corresponding author for this work

Research output: Contribution to journalArticle

20 Scopus citations

Abstract

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 (US)
Pages (from-to)157-164.e2
JournalJournal of Pediatrics
Volume184
DOIs
StatePublished - May 2017

Keywords

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

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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    Eldridge, W. B., Zhang, Q., Faro, A., Sweet, S. C., Eghtesady, P., Hamvas, A., Cole, F. S., & Wambach, J. A. (2017). Outcomes of Lung Transplantation for Infants and Children with Genetic Disorders of Surfactant Metabolism. Journal of Pediatrics, 184, 157-164.e2. https://doi.org/10.1016/j.jpeds.2017.01.017