Pediatric Lung Transplant Outcomes Based on Immunosuppressive Regimen at Discharge: Retrospective Cohort Study Using Real-World Evidence From the US Scientific Registry of Transplant Recipients

• Published in: Transplantation proceedings Vol. 55; no. 7; pp. 1692 - 1705
• Main Authors: Erdman, Jay, Wolfram, Josephine, Nimke, David, Croy, Richard, Wang, Xuegong, Weaver, Tim, Schladt, David, Fitzsimmons, William E.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.09.2023
• ISSN: 0041-1345; 1873-2623
• DOI: 10.1016/j.transproceed.2023.05.022

•Seven hundred twenty-five pediatric lung transplant recipients were followed for up to 3 years.•Recipients were more likely to continue with a tacrolimus (TAC)+mycophenolate mofetil (MMF) discharge regimen at 1 year versus other regimens.•No significant difference in graft survival (1999-2005) of TAC+MMF vs other regimens.•Graft survival at 1 year post-transplant was 92% with TAC+MMF.•This real-world evidence supported the Food and Drug Administration's approval of tacrolimus in pediatric lung transplant recipients. [Display omitted] This retrospective analysis of the US Scientific Registry of Transplant Recipients was undertaken to obtain real-world evidence concerning the efficacy and safety of tacrolimus-based immunosuppression in pediatric lung transplant recipients to support a supplemental New Drug Application. Overall, 725 pediatric recipients of a primary deceased-donor lung transplant between January 1, 1999, and December 31, 2017, were followed for up to 3 years post-transplant based on an immunosuppressive regimen at hospital discharge: immediate-release tacrolimus (TAC)+mycophenolate mofetil (MMF), TAC+azathioprine (AZA), cyclosporine (CsA)+MMF, or CsA+AZA. The primary outcome was the composite endpoint of graft failure or death (all-cause) at 1 year post-transplant, calculated by Kaplan–Meier analysis. The use of TAC+MMF increased over time. During 2010 to 2017, 91.7% of pediatric lung transplant recipients were receiving TAC+MMF at the time of discharge. The proportion of recipients continuing their discharge regimen at 1 year post-transplant was 83.7% with TAC+MMF and 40.4% to 59.7% with the other regimens. Cumulative incidence of the composite endpoint of graft failure or death at 1 year post-transplant was 7.7% with TAC+MMF, 13.9% with TAC+AZA, 8.9% with CsA+MMF, and 9.1% with CsA+AZA. There was no significant difference in the risk of graft failure or death at 1 year post-transplant between groups from 1999 to 2005 (the only era when adequate numbers on each regimen allowed statistical comparison). No increase in hospitalization for infection or malignancy was seen with TAC+MMF. The real-world evidence from the US database of transplant recipients supported the Food and Drug Administration's approval of tacrolimus-based maintenance immunosuppression in pediatric lung transplant recipients.