TY - JOUR
T1 - Outcomes of Lung Transplantation for Infants and Children with Genetic Disorders of Surfactant Metabolism
AU - Eldridge, Whitney B.
AU - Zhang, Qunyuan
AU - Faro, Albert
AU - Sweet, Stuart C.
AU - Eghtesady, Pirooz
AU - Hamvas, Aaron
AU - Cole, F. Sessions
AU - Wambach, Jennifer A.
N1 - Funding Information:
Support by the National Institutes of Health (Grants K08 HL105891 [to J.W.], K12 HL120002 [to F.C.], R01 HL065174 [to F.C.], and R01 HL082747 [to F.C.]), the American Lung Association (to J.W.), the American Thoracic Society (to J.W.), Children's Discovery Institute (to F.C.), and the Saigh Foundation (to F.C.). The authors declare no conflicts of interest.
Publisher Copyright:
© 2017 Elsevier Inc.
PY - 2017/5
Y1 - 2017/5
N2 - 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.
AB - 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.
KW - ABCA3
KW - NKX2.1
KW - RDS
KW - SFTPB
KW - SFTPC
KW - chILD
KW - childhood interstitial lung disease
KW - neonatal respiratory distress syndrome
KW - pediatric lung transplantation
KW - surfactant protein B
KW - surfactant protein C
UR - http://www.scopus.com/inward/record.url?scp=85012880259&partnerID=8YFLogxK
U2 - 10.1016/j.jpeds.2017.01.017
DO - 10.1016/j.jpeds.2017.01.017
M3 - Article
C2 - 28215425
AN - SCOPUS:85012880259
SN - 0022-3476
VL - 184
SP - 157-164.e2
JO - Journal of Pediatrics
JF - Journal of Pediatrics
ER -