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Sirolimus is efficacious in treatment for extensive and/or complex slow-flow vascular malformations: a monocentric

Jennifer Hammer, Emmanuel Seront1, Steven Duez2

  • 1Center for Vascular Anomalies, Institut Roi Albert II, Department of Medical Oncology, Cliniques universitaires Saint Luc, University of Louvain, Brussels, Belgium.

Orphanet Journal of Rare Diseases
|October 31, 2018
PubMed
Abstract

Insights

Sirolimus effectively treats extensive vascular malformations, improving patient quality of life and symptoms. This drug is well-tolerated and offers a new therapeutic option for complex cases.

Area of Science:

  • Vascular Biology
  • Pharmacology
  • Medical Therapeutics

Background:

  • Extensive vascular malformations cause chronic pain and functional limitations.
  • Conventional treatments like surgery and sclerotherapy are often ineffective.
  • Sirolimus shows promise in inhibiting the PI3K/AKT/mTOR pathway to manage these malformations.

Purpose of the Study:

  • To evaluate the efficacy and safety of sirolimus for extensive or complex slow-flow vascular malformations.
  • To assess improvements in patient symptoms, quality of life, and treatment outcomes.

Main Methods:

  • Prospective study involving oral sirolimus administration with pharmacokinetic-guided dosing (10-15 ng/ml).
  • Monthly follow-up for three months, then quarterly.
  • Primary endpoints included safety and efficacy via clinical, biological, radiological, and quality of life assessments.

Main Results:

  • Nineteen patients (3-64 years) with refractory lymphatic (LM) or venous malformations (VM) received sirolimus.
  • After 12 months, 16 patients showed significant symptom and quality of life improvement.
  • Sirolimus enabled previously unfeasible surgery/sclerotherapy in two patients; it was well-tolerated with mucositis as the most common grade 3 adverse event.

Conclusions:

  • Sirolimus is effective for extensive LM, VM, and complex malformations resistant to conventional therapies.
  • The treatment demonstrates good tolerability.
  • Sirolimus represents a viable therapeutic option for challenging vascular malformations.
Keywords:
Complex vascular malformationExtensive vascular anomalyLymphatic malformationRapamycinSirolimusSlow-flow anomalyVenous malformation

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