Bleomycin Electrosclerotherapy (BEST) for Slow-flow Vascular Malformations
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Change from baseline in OVAMA (Outcome Measures for Vascular Malformations) questionnaire scale scores
研究概览
简要总结
Slow-flow vascular malformations consist of abnormally formed veins, capillaries, lymphatic vessels, or a combination of these. They can cause pain, swelling, functional limitations, and concerns about appearance and may substantially affect quality of life. Bleomycin electrosclerotherapy (BEST) is an emerging treatment in which the sclerosant bleomycin is injected into the malformation and brief electrical pulses are applied to increase its local effect.
As treatment of slow-flow vascular malformations primarily aims to alleviate symptoms and improve quality of life, patient-reported outcomes are central to assessing treatment benefit. However, the available evidence on patient-reported outcomes following BEST is sparse and heterogeneous, and prospective data are particularly limited.
This prospective observational study follows children and adults undergoing BEST as part of routine clinical care at Copenhagen University Hospital - Rigshospitalet.
The OVAMA questionnaire will be used to assess symptoms and appearance in all participants before treatment and at the 6-month follow-up. Depending on age, additional patient-reported measures will assess health-related quality of life (PROMIS-29) and pain (VAS) during follow-up. For adults, the study will assess changes in lesion size by comparing magnetic resonance imaging (MRI) scans performed before treatment and at 6 months. Adverse events will be recorded throughout follow-up.
The study aims to evaluate whether patient-reported outcomes improve during the 6 months after BEST and to provide further information about treatment safety.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Scheduled to undergo BEST as part of routine clinical care
- •Diagnosis of a slow-flow vascular malformation confirmed by contrast-enhanced MRI performed within 6 months before enrollment.
排除标准
- •Invasive treatment of the vascular malformation within 6 months before enrollment, including surgery, sclerotherapy, or laser treatment.
结局指标
主要结局
Change from baseline in OVAMA (Outcome Measures for Vascular Malformations) questionnaire scale scores
时间窗: Baseline and 6 months after BEST
The OVAMA questionnaire is a condition-specific patient-reported outcome measure assessing symptoms and appearance related to vascular malformations.
次要结局
- Change from baseline in PROMIS-29 (Patient-Reported Outcomes Measurement Information System) domain scores(Baseline and 6 months after BEST)
- Pain intensity measured using a visual analogue scale (VAS)(Baseline, 2 weeks, 1 month, 3 months, and 6 months after BEST)
- Change from baseline in vascular malformation size assessed by MRI(Baseline and 6 months after BEST)
- Number of participants with adverse events following BEST(From the day of the BEST procedure to the 6-month follow-up)
研究者
Caroline Clausen
MD, PhD
Rigshospitalet, Denmark