Bleomycin Electrosclerotherapy (BEST) for Slow-flow Vascular Malformations

Обзор

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.

Вмешательства

• Процедура Bleomycin electrosclerotherapy (BEST)
  Bleomycin electrosclerotherapy combines intralesional administration of bleomycin with local electroporation, which transiently increases cell membrane permeability and enhances intracellular drug uptake within the vascular malformation.

Первичные конечные точки

• Change from baseline in OVAMA (Outcome Measures for Vascular Malformations) questionnaire scale scores [Срок оценки: Baseline and 6 months after BEST]

Вторичные конечные точки (4)

• Change from baseline in PROMIS-29 (Patient-Reported Outcomes Measurement Information System) domain scores [Срок оценки: Baseline and 6 months after BEST]
• Pain intensity measured using a visual analogue scale (VAS) [Срок оценки: Baseline, 2 weeks, 1 month, 3 months, and 6 months after BEST]
• Change from baseline in vascular malformation size assessed by MRI [Срок оценки: Baseline and 6 months after BEST]
• Number of participants with adverse events following BEST [Срок оценки: From the day of the BEST procedure to the 6-month follow-up]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• Invasive treatment of the vascular malformation within 6 months before enrollment, including surgery, sclerotherapy, or laser treatment.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Дания · 1 центр

• Copenhagen University Hospital - Rigshospitalet — Copenhagen

Публикации

• Gehr J, Hindso L, Taudorf M, Eiberg JP, Frevert SC, Jensen RJ, Kaltoft M, Lonn LB, de la Motte LR, Strom M, Clausen C. Effectiveness and Safety of Bleomycin Electrosclerotherapy for Slow-Flow Vascular Malformations: A Systematic Review. Cardiovasc Intervent Radiol. 2026 Jun;49(6):1105-1116. doi: 10.1007/s00270-026-04441-3. Epub 2026 Apr 27. PMID 42045624

Идентификаторы

NCT: NCT07720570 · p-2026-21479

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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