Vascular Malformations
Conditions
Keywords
Bleomycin electrosclerotherapy, Electroporation, Sclerotherapy, Patient-reported outcome measures, Slow-flow vascular malformations, Vascular malformations
Brief summary
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.
Interventions
PROCEDUREBleomycin 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.
Sponsors
Rigshospitalet, Denmark
University of Copenhagen
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
• Invasive treatment of the vascular malformation within 6 months before enrollment, including surgery, sclerotherapy, or laser treatment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in OVAMA (Outcome Measures for Vascular Malformations) questionnaire scale scores | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in PROMIS-29 (Patient-Reported Outcomes Measurement Information System) domain scores | Baseline and 6 months after BEST | The Patient-Reported Outcomes Measurement Information System 29-item Profile (PROMIS-29) is a generic patient-reported outcome measure assessing physical, mental, and social health. For participants aged 18 years or older. |
| Pain intensity measured using a visual analogue scale (VAS) | Baseline, 2 weeks, 1 month, 3 months, and 6 months after BEST | Participants rate pain intensity using a VAS ranging from 0 (no pain) to 10 (worst imaginable pain). For participants aged 7 years or older. |
| Change from baseline in vascular malformation size assessed by MRI | Baseline and 6 months after BEST | MRI will be used to compare the size of the treated vascular malformation before BEST and 6 months after treatment. For participants aged 18 years or older. |
| Number of participants with adverse events following BEST | From the day of the BEST procedure to the 6-month follow-up | Adverse events occurring on the day of BEST or during follow-up will be recorded, including their nature, severity, management, and outcome. Events will be classified according to the CIRSE and Clavien-Dindo classification systems. |
Countries
Denmark
Contacts
PRINCIPAL_INVESTIGATORCaroline Clausen, MD, PhD
Rigshospitalet, Denmark
Outcome results
None listed