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Oral sirolimus therapy for patients with complex low-flow vascular malformations
Rebecca Nisbet1, Calver Pang2, Nicholas Evans3
1Royal Free London Vascular Anomalies Centre, Royal Free London NHS Foundation Trust, London, UK; School of Medicine, University of Leeds, Leeds, UK.
Summary
Oral sirolimus effectively treated complex low-flow vascular malformations (LFVMs) when standard therapies failed, significantly reducing lesion size. While side effects were common, the treatment proved safe and beneficial for these challenging LFVM cases.
Area of Science:
- Vascular Biology
- Pharmacology
- Dermatology
Background:
- Low-flow vascular malformations (LFVMs) present significant challenges, particularly in extensive or complicated cases.
- Existing evidence suggests sirolimus may benefit patients with complex LFVMs.
- Standard treatments are often inadequate for severe LFVM presentations.
Purpose of the Study:
- To evaluate the efficacy and safety of oral sirolimus therapy.
- To assess treatment outcomes in adult patients with complex LFVMs where standard therapy was insufficient.
Main Methods:
- Retrospective study of 55 adult LFVM patients treated with oral sirolimus from May 2016 to April 2023.
- Data collected included patient-reported outcomes, pain scores, quality of life (SF-36), and adverse effects.
- Analysis compared outcomes between syndromic and nonsyndromic patients.
Main Results:
- 58.2% of patients showed some improvement, with a significant decrease in lesion size (P = .0004).
- Quality of life scores showed nonsignificant improvements in physical function, energy, and pain, but increased anxiety/depression.
- Common side effects included mouth ulcers (54.5%) and fatigue (29.1%); 9.1% reported no side effects.
Conclusions:
- Oral sirolimus is clinically effective and safe for complex LFVMs unresponsive to standard therapy.
- The treatment demonstrated a significant reduction in lesion size.
- Further long-term studies are warranted to fully elucidate the role of sirolimus in LFVM management.
