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Single Center Experience of Sirolimus Therapy in Head and Neck Low-flow Vascular Malformations

Pang, C; Evans, N; Jethwa, P; Papadopoulou, A; Khalifa, M; Tsui, J; Hamilton, G; ... Brookes, J; + view all (2021) Single Center Experience of Sirolimus Therapy in Head and Neck Low-flow Vascular Malformations. Vascular and Endovascular Surgery 10.1177/15385744211010378. (In press). Green open access

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Abstract

OBJECTIVE: Recently, studies have shown that sirolimus is clinically efficacious in the treatment of some low-flow vascular malformations (LFVM). This study aimed to assess the efficacy and safety of sirolimus in treating complex head and neck (H&N) LFVM that were challenging and/or refractory to standard treatment. METHODS: Each patient had baseline and 6-months assessments consisting of clinical history and examination, quality of life (QoL) questionnaires, laboratory investigations, MRI and medical photography. Patients were followed up 1-week and then 1-monthly for 6-months. Wilcoxon signed-rank test was used to compare pre-and 6-months treatment in all 8 domains of RAND 36-Item Short Form Health Survey (SF-36), hospital anxiety and depression scale (HADS), and visual analog score for pain (VAS-P). P < 0.05 was considered significant. RESULTS: Seven patients (median age 43 years, range 23-65 years) were recruited. Six patients completed the six-months course of therapy with 1 patient withdrawing due to intolerable side effects. All six patients reported reduction of swelling with and without other symptom improvement related to the vascular malformations while on treatment. However, at 1-month review after discontinuation of sirolimus, 5 patients reported return of initial symptoms. Overall, patients demonstrated an improvement in QoL six-months treatment but there was no statistical significance (P > 0.05) in all 8 domains of SF-36, HADS and VAS-P. Five patients demonstrated a minimum 10% decrease in lesion size six-months treatment (median 21%, range 13-40%). A Wilcoxon signed-rank test showed that sirolimus treatment did elicit a statistically significant change in lesion size in either direction (Z = -1.992, P = 0.046). The most common side effects found were dyslipidaemia (n-4) and mouth ulcers (n = 2). CONCLUSIONS: In our preliminary experience, sirolimus is effective and safe in treating patients with complex H&N LFVM. This provides an alternative treatment where standard treatment is challenging and/or refractory.

Type: Article
Title: Single Center Experience of Sirolimus Therapy in Head and Neck Low-flow Vascular Malformations
Open access status: An open access version is available from UCL Discovery
DOI: 10.1177/15385744211010378
Publisher version: https://doi.org/10.1177/15385744211010378
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Vascular malformations, complex vascular malformation, head and neck, sirolimus, mTOR inhibitors
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Surgery and Interventional Sci
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Surgery and Interventional Sci > Department of Surgical Biotechnology
URI: https://discovery.ucl.ac.uk/id/eprint/10128377
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