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Systemic Sirolimus Monitoring in Pediatric Pulmonary Vein Stenosis

Heather Meluskey1, Bridget Blowey2, Anna B O'Brien2

  • 1Division of Cardiology, The Children's Hospital of Philadelphia and Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.

Pediatric Cardiology
|October 1, 2025
PubMed

Insights

Systemic sirolimus (SS) management for pediatric pulmonary vein stenosis (PVS) shows variable therapeutic levels but uncommon discontinuation due to adverse events. Dedicated team monitoring may improve patient compliance with toxicity surveillance.

Area of Science:

  • Cardiology
  • Pharmacology
  • Pediatric Pulmonology

Background:

  • Systemic sirolimus (SS) is an mTOR inhibitor utilized for pediatric intraluminal pulmonary vein stenosis (PVS).
  • Data on SS initiation, monitoring, patient compliance, and adverse events (AE) in PVS patients are limited.
  • Understanding SS management is crucial for optimizing treatment outcomes in this population.

Purpose of the Study:

  • To evaluate the initiation, monitoring, and safety of systemic sirolimus (SS) in pediatric patients with pulmonary vein stenosis (PVS).
  • To assess patient compliance with toxicity surveillance and identify potential adverse events (AE) associated with SS therapy.
  • To analyze the impact of a dedicated PVS team on SS management and compliance.

Main Methods:

  • A single-center retrospective cohort study included consecutive pediatric PVS patients initiated on SS.
  • Data were collected from January 1, 2020, to December 31, 2024.
  • Analysis focused on time to therapeutic levels, blood draws, dose adjustments, AE, and compliance rates.

Main Results:

  • Fifty pediatric patients with PVS received SS for a median of 18 months.
  • Median time to therapeutic SS level was 9 days, with two patients never achieving therapeutic values.
  • Toxicity surveillance compliance increased from 58% to 79% after transitioning management to a dedicated PVS team (p=0.22).
  • Eighteen percent of patients experienced AEs potentially related to SS, with discontinuation in three cases.
  • SS discontinuation due to AEs was uncommon.

Conclusions:

  • Pediatric PVS patients on systemic sirolimus exhibit variable achievement of therapeutic levels.
  • Adverse events leading to SS discontinuation are infrequent in this cohort.
  • Dedicated monitoring teams may enhance compliance with essential safety laboratory monitoring for SS therapy.

Keywords:
Congenital heart diseaseIn stent restenosisMedical therapymTOR inhibition

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