Single Center Experience of Sirolimus Therapy in Head and Neck Low-flow Vascular Malformations. (July 2021)

• Record Type: Journal Article
• Authors: Pang, Calver; Evans, Nicholas; Jethwa, Poonam; Papadopoulou, Anthie; Khalifa, Mohamed; Tsui, Janice; Hamilton, George; Lim, Chung Sim; Brookes, Jocelyn
• Is Part Of: Vascular & endovascular surgery. Volume 55:Number 5(2021)
• Page Start: 482
• Page End: 490
• Publication Date: 2021-07
• Subjects: vascular malformations -- complex vascular malformation -- head and neck -- sirolimus -- mTOR inhibitors; Blood-vessels -- Surgery -- Periodicals; Blood-vessels -- Endoscopic surgery -- Periodicals; Vascular Surgical Procedures -- Periodicals; Angioplasty -- Periodicals; Surgical Procedures, Minimally Invasive -- Periodicals; Vascular Diseases -- surgery -- Periodicals; Vaisseaux sanguins -- Chirurgie -- Périodiques; Vaisseaux sanguins -- Chirurgie endoscopique -- Périodiques; 617.41
• DOI: 10.1177/15385744211010378
• Languages: English
• ISSNs: 1538-5744

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). Conclusion: 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.