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

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.

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.

Conflict of interest statement

The authors declare no conflicts of interest.

Copyright © 2017 Elsevier Inc. All rights reserved.

Figures

Figure 1
Figure 1
Kaplan-Meier Analysis of Mortality: Infants, Epoch 1 (1993–2003) compared to Epoch 2 (2004–2015).
Figure 2
Figure 2
Kaplan-Meier analysis of mortality: children, epoch 1 (1993–2003) compared with epoch 2 (2004–2015).
Figure 3
Figure 3
A, Kaplan-Meier analysis of freedom from hypertension: children, epoch 1 (1993–2003) compared with epoch 2 (2004–2015). B, Kaplan-Meier analysis of freedom from PTLD: children, epoch 1 (1993–2003) compared with epoch 2 (2004–2015)
Figure 4
Figure 4
A, Kaplan-Meier analysis of mortality: infants compared with children, 1993–2015. B, Kaplan-Meier analysis of bronchiolitis obliterans: infants compared with children, 1993–2015.

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