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Long Term Outcomes and Safety Profile of Sirolimus in Post Liver Transplant Children
Sandra Fernandes Lucas1, Suzan Warner1, Penny North-Lewis1
1Leeds Children's Hospital, Leeds, UK.
Insights
Sirolimus is a safe and effective alternative immunosuppressant for pediatric liver transplant recipients, particularly those with post-transplant lymphoproliferative disorder. This study shows sirolimus is well-tolerated, with most patients remaining on treatment long-term.
Area of Science:
- Pediatric transplantation
- Immunosuppression therapy
- Pharmacology
Background:
- Tacrolimus is the standard maintenance immunosuppression for pediatric liver transplant (LT) recipients.
- Sirolimus is used selectively as an alternative immunosuppressive treatment in specific pediatric LT cases.
Purpose of the Study:
- To review a single center's experience with sirolimus as an alternative to tacrolimus in pediatric LT recipients.
- To evaluate the safety and efficacy of sirolimus in this selected pediatric population.
Main Methods:
- Retrospective study of pediatric LT recipients (<16 years) initiated on sirolimus between May 2000 and May 2024.
- Analysis of reasons for switching from tacrolimus to sirolimus, patient outcomes, and adverse events.
Main Results:
- 19 children received sirolimus post-LT, with a median follow-up of 4 years.
- Post-transplant lymphoproliferative disorder (PTLD) was the primary reason for conversion (n=14), followed by adverse effects (n=4) and recurrence (n=1).
- No PTLD recurrence or biopsy-confirmed rejection occurred on sirolimus; proteinuria and hyperlipidemia were common side effects. 18 children remain on sirolimus, with no discontinuations due to side effects.
Conclusions:
- Sirolimus is a safe and effective alternative to tacrolimus in selected pediatric LT recipients.
- Further research with larger sample sizes is needed to confirm long-term safety and efficacy.
Background:
Tacrolimus is the maintenance immunosuppression of choice in pediatric liver transplant (LT) recipients. However, in selective cases, sirolimus is used as an alternative immunosuppressive treatment. We aimed to review a single centre's experience of using sirolimus as an alternative to tacrolimus in pediatric LT recipients.
Methods:
Single centre retrospective study of pediatric LT recipients who were started on sirolimus as an alternative immunosuppressant to tacrolimus. Children (< 16 years) who were started on sirolimus between May 2000 and May 2024 were included in the study.
Results:
A total of 19 children were started on sirolimus following LT and followed up for a median of 4 years (range 0.4-12.8). Post-transplant lymphoproliferative disorder (PTLD) was the most common reason for tacrolimus discontinuation and conversion to sirolimus (n = 14), followed by tacrolimus-related adverse effects (n = 4) and disease recurrence (n = 1). There were no cases of PTLD recurrence or biopsy-confirmed rejection whilst on sirolimus. Proteinuria and hyperlipidaemia were the most common sirolimus side effects observed. Eighteen children remain on sirolimus to date, and none required discontinuation from side effects. In those with PTLD, 4 episodes of rejection occurred between the period of tacrolimus discontinuation and starting sirolimus (median immunosuppression-free time of 5 months), with one child requiring regrafting due to chronic rejection.
Conclusion:
The experience from our centre demonstrates sirolimus to be a safe and effective alternative to tacrolimus in a selected population of pediatric LT recipients. Further research with a larger sample size is required to confirm these findings and evaluate the long-term safety of sirolimus in this population.
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