Objective
To compare outcomes of infants and children who underwent lung transplantation for genetic disorders of surfactant metabolism (SFTPB, SFTPC, ABCA3, 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 were transplanted more frequently for surfactant protein-B or ABCA3 deficiency, while children were transplanted more frequently for SFTPC mutations or ABCA3 deficiency. Infants experienced shorter times from listing to transplant (p=0.014), were more likely to be mechanically ventilated at time of transplant (p<0.0001), were less likely to develop bronchiolitis obliterans post-transplant (p=0.021), and were more likely to have speech and motor delays (p = <0.0001) than children. Despite advances in genetic diagnosis, immunosuppressive therapies, and supportive respiratory and nutritional therapies, mortality did not differ between infants and children (p = 0.076) or between epochs. Kaplan-Meier analyses demonstrated that children transplanted in epoch 1 (1993–2003) were more likely to develop systemic hypertension (p=0.049) and less likely to develop post-transplant lymphoproliferative disorder than children transplanted in epoch 2 (2004–2015) (p=0.051).
Conclusion
Post-lung transplant mortality and morbidities remain substantial for infants and children with genetic disorders of surfactant metabolism.