Treatment of Low-flow Vascular Malformations With Bleomycin Electrosclerotherapy (BEST)
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 140
- 主要终点
- MRI-measured mean change in lesion volume from baseline to 3 months after BEST treatment
研究概览
简要总结
In biomedical applications, electroporation is used not only for cancer treatment but also for vaccinations, treatment of cardiac arrhythmias and, more recently, for the treatment of vascular malformations. Bleomycin is a frequently used sclerosing agent in the treatment of various vascular malformations. The use of electrical pulses in addition to bleomycin increases the effectiveness of the treatment, similar to electrochemotherapy. Bleomycin electrosclerotherapy (BEST) is a new treatment modality that is effective in the treatment of low-flow malformations (venous and lymphatic malformations) and potentially also high-flow malformations (arteriovenous malformations). Although a limited number of reports have been published to date, more and more centers are using BEST for the treatment of vascular malformations. As part of the International Network for Sharing Practices on Electrochemotherapy (InspECT) consortium, a dedicated working group has been set up to develop standard operating procedures for BEST. Current Operating Procedures have been prepared and will be used in this clinical trial.
详细描述
Vascular malformations are rare conditions caused by abnormally developed blood vessels. They can occur anywhere in the body and range from simple and benign lesions to complex conditions.
The latest and most commonly used categorization is the International Society for the Study of Vascular Anomalies (ISSVA) classification. This classification divides vascular anomalies into two main categories: tumors, defined as true proliferative neoplasms, and malformations, defined as morphogenetic defects. These two categories are further subcategorized: tumors are divided into benign, locally aggressive/borderline, and malignant tumors, whereas malformations are subdivided into simple, combined, or associated with other anomalies. Clinically, vascular anomalies can also be divided into low-flow and high-flow malformations.
Current treatment of vascular malformations varies depending on the type and anatomical location of the vascular malformation. Treatment options include observation, sclerotherapy, laser therapy, embolization, and surgery. Sclerotherapy involves the injection of sclerosing agents, such as bleomycin, pingyangmycin, absolute ethanol, ethanolamine oleate, polidocanol, doxycycline, cyanoacrylate, sodium morrhuate, and sodium tetradecyl sulfate (STS).
Current treatment options for low-flow vascular malformations remain suboptimal. Current therapies demonstrate limited clinical efficacy. Ethanol is widely used as a sclerosing agent and can induce substantial lesion regression; however, its clinical utility is limited by considerable safety concerns. In particular, ethanol may induce extensive tissue necrosis and damage to surrounding healthy structures, which limits its therapeutic use.
Another agent frequently employed for sclerotherapy is bleomycin. However, when administered as monotherapy, without the use of electroporation, bleomycin often shows insufficient therapeutic effectiveness. In many cases, treatment results only in minor reduction of lesion volume and fails to adequately alleviate patient-reported symptoms, including pain and functional discomfort.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patients with low-flow vascular malformations (venous, lymphatic, capillary or mixed type malformations),
- •patients with a low-flow vascular malformation poorly responding or recurring after previous treatment(s),
- •longer lesion diameter not exceeding 25 cm.
- •more than one lesion can be treated. The limiting factor is the maximal dose per patient per treatment session; 10 000 IU in adults,
- •skin or mucosal, superficial or deep-seated lesions can be treated,
- •technical feasibility of the BEST procedure, i.e.: injection of bleomycin and safe placement of electrodes into the vascular malformation are technically feasible.
排除标准
- •pregnancy and lactation,
- •women of childbearing potential and men not using reliable contraception,
- •in adults, previous bleomycin exposure with a cumulative dose greater than 100 000 IU. In case of abnormal respiratory results/chest pathology (including previous severe or long COVID) in consultation with a pulmonologist, special care is required, and bleomycin exposure may be contraindicated,
- •known allergy or hypersensitivity to bleomycin,
- •presence of significant central venous drainage precluding sclerotherapy,
- •acute lung infection or severely reduced lung function,
- •bleomycin-related lung toxicity or reduced lung function which can indicate bleomycin-related lung toxicity,
- •ataxia telangiectasia,
- •chronic renal dysfunction.
研究组 & 干预措施
Bleomycin electrosclerotherapy
Participants with low-flow vascular malformations will receive intralesional bleomycin combined with local application of electrical pulses according to the BEST procedure.
干预措施: Bleomycin Sulfate (Drug)
结局指标
主要结局
MRI-measured mean change in lesion volume from baseline to 3 months after BEST treatment
时间窗: Baseline and 3 months after treatment
Lesion volume will be assessed by magnetic resonance imaging at baseline and 3 months after treatment. Lesion volume will be calculated from MRI images using the ellipsoid formula: a x b x c x π/6. The outcome measure will report the mean change in lesion volume from baseline to 3 months after BEST treatment.
次要结局
- MRI-measured mean change in lesion volume from baseline to 12 months after BEST treatment(Baseline and 12 months after treatment)
- Number of participants who complete protocol-defined BEST treatment and follow-up visits(Up to 12 months after treatment)
- Number of participants with treatment-emergent adverse events as assessed by CTCAE version 5.0(Up to 12 months after treatment)
- Change in quality of life from baseline to 3 and 12 months after BEST treatment as assessed by the OVAMA questionnaire(Baseline, 3 months, and 12 months after treatment)