Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Aug;81(2):167-176.
doi: 10.1002/jpn3.70073. Epub 2025 May 19.

Sirolimus utilization in pediatric liver transplantation: A large high-volume quaternary center experience

Wenly Ruan  1   2 , Dana N Cerminara  3 , Nhu Thao Nguyen Galvan  4   5 , Douglas S Fishman  1   2 , Sanjiv Harpavat  1   2 , Paula M Hertel  1   2 , Krupa R Mysore  1   2 , Mary Elizabeth Tessier  1   2 , Kelby Fuller  4   5 , Marielle Faraone  4   5 , Ronald T Cotton  4   5 , Christine A O'Mahony  4   5 , Abbas Rana  4   5 , John A Goss  4   5 , Daniel H Leung  1   2
Affiliations

Sirolimus utilization in pediatric liver transplantation: A large high-volume quaternary center experience

Wenly Ruan et al. J Pediatr Gastroenterol Nutr. 2025 Aug.

Abstract

Objectives: Optimization of liver transplant (LT) immunosuppression is essential for long-term graft function in children. Mammalian target of rapamycin (mTOR) inhibitors, such as sirolimus, have not been extensively studied in pediatric LT. We aimed to explore the usage of and indications for sirolimus at a quaternary pediatric LT center in a retrospective cohort analysis.

Methods: Pediatric LT patients who received sirolimus from 2010 to 2018 were included. Their electronic medical records were evaluated for demographic information, clinical data, and laboratory data before and after initiation of sirolimus.

Results: Sirolimus was initiated on 41 pediatric LT recipients at a median of 0.67 years post-transplant (interquartile range [IQR]: 0.13-1.83) at Texas Children's Hospital. The leading indications for initiating sirolimus were chronic rejection (CR) (n = 12, 29.3%), T-cell-mediated rejection (TCMR) on tacrolimus (n = 8, 19.5%), posterior reversible encephalopathy syndrome (PRES) (n = 8, 19.5%), and unresectable hepatic tumors/malignancies (n = 7, 17.1%). Among patients started on sirolimus for CR, 58% (n = 7/12) demonstrated histological or biochemical improvement. Among those started for TCMR augmentation, 62.5% experienced biochemical improvement. No patients who started on sirolimus for unresectable hepatic tumors/malignancies had malignancy recurrence 1-year post-LT. Median cholesterol and triglyceride levels 1-year post-initiation were higher than values 1-year pre-initiation (p < 0.0001); however, sirolimus-induced anemia, nephrotoxicity, and HAT were not observed.

Conclusions: This study is among the first to review the clinical experience of sirolimus usage in pediatric LT. Our experience suggests sirolimus can be used safely and effectively. Further research is warranted to evaluate long-term benefits, adverse effects, and optimal dosing of sirolimus in pediatric liver transplantation.

Keywords: immunosuppression; liver transplant; mTOR; post‐transplant; rejection.

PubMed Disclaimer

References

REFERENCES

    1. Enderby C, Keller CA. An overview of immunosuppression in solid organ transplantation. Am J Manag Care. 2015;21:12‐23.
    1. Hussaini T, Erb S, Yoshida EM. Immunosuppressive pharmacotherapy in liver transplantation. AME Med J. 2018;3:18.
    1. Neff GW, Montalbano M, Tzakis AG. Ten years of sirolimus therapy in orthotopic liver transplant recipients. Transplant Proc. 2003;35:S209‐S216.
    1. Sehgal SN. Sirolimus: its discovery, biological properties, and mechanism of action. Transplant Proc. 2003;35:S7‐S14.
    1. Pfizer. Pfizer Sirolimus [Package Insert] [Internet]. U.S. Food and Drug Administration; 2017:1‐56. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/021083s059,021...

Grants and funding

LinkOut - more resources