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Long-term effects of sirolimus treatment for slow-flow vascular malformations: Real-world evidence from the French

Cécilia Maillet1,2, Olivia Boccara3, Stéphanie Mallet4

  • 1Unit of Pediatric Dermatology, Department of Dermatology, University Hospital Center (CHRU) of Tours, Tours, France.

Journal of the European Academy of Dermatology and Venereology : JEADV
|November 25, 2024
PubMed
Abstract

Insights

Oral sirolimus effectively manages slow-flow vascular malformations (SFVMs) symptoms like pain and bleeding long-term. While generally well-tolerated, its efficacy in reducing malformation volume is limited, and some patients may require dosage adjustments or alternative treatments.

Area of Science:

  • Vascular Anomalies
  • Pharmacology
  • Dermatology

Background:

  • Sirolimus is a key treatment for slow-flow vascular malformations (SFVMs).
  • Long-term management of SFVMs with sirolimus presents ongoing challenges.
  • Real-world data on sirolimus's long-term efficacy and safety in SFVMs is crucial.

Purpose of the Study:

  • To assess the long-term effects of oral sirolimus in patients with SFVMs.
  • To evaluate the real-life management strategies for SFVMs treated with sirolimus.
  • To investigate treatment goals, efficacy, safety, and withdrawal patterns in a large cohort.

Main Methods:

  • Retrospective analysis of 67 patients with SFVMs treated with sirolimus for at least 3 years.
  • Data collected from 15 French tertiary centers for vascular anomalies.
  • Evaluation of treatment goals, investigator-reported efficacy, safety, dosages, and withdrawal history.

Main Results:

  • Sirolimus demonstrated persistent efficacy in managing bleeding, ulceration, and pain associated with SFVMs.
  • Volume reduction efficacy was noted as slight, with a trend towards lower sirolimus concentrations over time.
  • The treatment was generally well-tolerated, with serious adverse events in 9.0% of patients, and 16.4% experienced temporary withdrawal due to symptom recurrence.

Conclusions:

  • This study provides valuable real-world insights into the long-term use of sirolimus for SFVMs.
  • Findings address common patient and parent concerns regarding efficacy persistence, long-term side effects, and recurrence after withdrawal.
  • Sirolimus remains a viable option for symptom management in SFVMs, though volume reduction is limited and careful monitoring is necessary.

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