Use of sirolimus in pediatric heart transplant patients: A multi-institutional study from the Pediatric Heart Transplant Study Group

J Heart Lung Transplant. 2017 Apr;36(4):427-433. doi: 10.1016/j.healun.2016.09.009. Epub 2016 Oct 4.


Background: Proliferation signal inhibitors, such as sirolimus, are increasingly used in solid-organ transplantation. However, limited data exist on sirolimus-treated pediatric patients. We aimed to describe sirolimus use in pediatric heart transplant patients and test the hypothesis that sirolimus use is associated with improved outcomes.

Methods: A retrospective review and propensity-matched analysis of the Pediatric Heart Transplant Study database was performed on patients undergoing primary heart transplantation from 2004 to 2013 with at least 1 year of follow-up comparing patients treated vs not treated with sirolimus at 1 year after transplant. The primary outcome of interest was patient survival, with secondary outcomes including cardiac allograft vasculopathy, rejection, malignancy, and renal insufficiency.

Results: Between 2004 and 2013, 2,531 patients underwent transplantation. At least 1 year of follow-up was available for 2,080 patients, of whom 144 (7%) were on sirolimus at 1 year post-transplant. Sirolimus-treated and non-treated patients had similar survival in the overall cohorts and in the propensity-matched analysis. The secondary outcomes measures were also similar, including a composite end point of all outcome measures. There was a trend toward increased time to cardiac allograft vasculopathy (p = 0.09) and decreased time to infection (p = 0.05) among sirolimus-treated patients in the overall cohort (p = 0.19) but not in the propensity-matched cohort (p = 0.17).

Conclusions: Sirolimus was used in less than 10% of patients at 1 year post-transplant. Overall outcomes of sirolimus treated and non-treated patients were similar with respect to survival and major transplant adverse events. Further study of sirolimus in pediatric heart transplant patients is needed.

Keywords: calcineurin inhibitor; cardiac allograft vasculopathy; drugs; pediatrics; sirolimus; transplantation.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Age Factors
  • Child
  • Child, Preschool
  • Female
  • Heart Failure / etiology
  • Heart Failure / mortality
  • Heart Failure / surgery*
  • Heart Transplantation*
  • Hospitalization
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Male
  • Retrospective Studies
  • Sirolimus / therapeutic use*
  • Survival Rate
  • Treatment Outcome


  • Immunosuppressive Agents
  • Sirolimus