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Experience with the mTOR Inhibitor Everolimus in Pediatric Liver Graft Recipients
Mathis Wehming1, Dorothée Krebs-Schmitt1, Andrea Briem-Richter1
1Department of Pediatric Gastroenterology and Hepatology, University Hospital Hamburg-Eppendorf, 20246 Hamburg, Germany.
Insights
Everolimus is a viable option for pediatric liver transplant patients facing graft dysfunction or side effects. This mTOR inhibitor demonstrated good efficacy and an acceptable safety profile in a 3-year study.
Area of Science:
- Pediatric Hepatology
- Transplantation Immunology
- Pharmacology
Background:
- Pediatric liver transplantation immunosuppression is challenging.
- MTOR inhibitors offer a promising strategy with reduced calcineurin inhibitors (CNI).
- Limited data exist on pediatric MTOR inhibitor use.
Purpose of the Study:
- To evaluate Everolimus efficacy and safety in pediatric liver transplant recipients.
- To assess outcomes in specific patient subgroups requiring alternative immunosuppression.
Main Methods:
- Retrospective analysis of 37 pediatric liver transplant patients (median age 10 years).
- Indications included chronic graft dysfunction, renal impairment, adverse effects, and malignancies.
- Median follow-up was 36 months.
Main Results:
- High patient (97%) and graft (84%) survival rates.
- Graft function stabilization in 59% of patients with chronic graft dysfunction.
- Acceptable safety profile with infections as the most common side effect (54.1%); no significant impact on growth.
Conclusions:
- Everolimus is a suitable treatment option for selected pediatric liver transplant recipients.
- It offers good efficacy and an acceptable side effect profile.
- Consideration for patients with specific indications where other regimens are unsuitable.
Introduction:
Immunosuppression after pediatric liver transplantation remains a major challenge. MTOR inhibitors provide a promising therapeutic approach in combination with reduced CNI after transplantation. However, there are still few data regarding their use in children.
Patients:
We analyzed 37 patients with a median age of 10 years, who received Everolimus for one or more of the following indications: I = chronic graft dysfunction (n = 22); II = progressive renal impairment (n = 5); III = non-tolerable side effects with previous immunosuppressive medication (n = 6); and IV = malignancies (n = 10). The median follow-up time was 36 months.
Results:
Patient survival was 97%, and graft survival 84%, respectively. Stabilization of graft function was observed in 59% in subgroup 1, with 18.2% ultimately requiring retransplantation. No patient in subgroup IV developed recurrence of his primary tumor or PTLD by the endpoint of the study. Side effects were observed in 67.5% of the study patients, with infections being the most frequent (n = 20; 54.1%). There were no relevant effects on growth and development.
Conclusion:
Everolimus seems to be a treatment option in selected pediatric liver graft recipients for whom other regimens are not suitable. Overall, the efficacy was good and the side effect profile appeared to be acceptable.
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