Surgery Versus Intra-lesional Sclerosis With Polidocanol (Aetoxisclerol) in Labial Venous Malformations in Children and Adults (SASOV)

Experimental group: Surgical strategy for the excision of the superficial venous malformation (SVM). The surgery involves either partial reconstructive excision or complete excision of the SVM, depending on its size. The aim is to restore symmetry to the affected side of the lip in order to correct functional impairments and aesthetic effects.

The surgery is performed under local or general anaesthesia (depending on the size of the SVM and the patient's age). Various coagulation devices (monopolar forceps with a Colorado tip, bipolar scissors, etc.) are used to section and dissect the SVM and to control haemostasis.

Control group: Sclerotherapy strategy comprising one to two sessions of intra-lesional sclerotherapy with 3% polidocanol (Aetoxisclerol).

The procedure may be carried out under local or general anaesthesia, depending on the patient's age, anticipated tolerance and in accordance with standard practice at the centre. The patient is positioned in the supine position. Following manual and visual localisation of the target lesion, one or more needles of 30/100th to 18G gauge are inserted, and their position within the lesion is confirmed by gentle aspiration, which should yield blood without resistance. The injection of an iodinated contrast medium suitable for intravenous use, to characterise the drainage and under fluoroscopic guidance in the angiography suite, is recommended but not mandatory. Once in place, ≤ 2 mg/kg of polidocanol is injected per session, i.e. ≤ 0.067 mL/kg of 3% solution distributed amongst the needles (1/2 to 2 ampoules), until thrombosis occurs, significant tension is felt in the lesion, or pain develops.

Sclerotherapy may be repeated if necessary between 3 and 6 months after the first session.

Global change will be assessed 12 months after the intervention using a Likert scale ranging from -5 to +5 (0 = no improvement or worsening, higher scores mean better outcome), based on standardized pre- and post-intervention photographs. Assessments will be performed by an independent review panel comprising three physicians (one surgeon, one dermatologist, and one interventional radiologist) and one patient with a labial VM who did not participate in the study. The evaluation will be performed blinded to treatment allocation and will encompass changes in lesion volume, scar quality, and lip symmetry. A standardized photography protocol will be used. This primary endpoint (photograph-based assessment by an independent review panel) was selected to limit bias related to the open-label nature of the study, consistent with a PROBE design (Prospective Randomized Open, Blinded Endpoint).

Assessment of aesthetic burden using a 0 to 10 visual analogue scale (VAS) with higher scores mean worse outcome, measured at baseline and 12 months after the intervention.

Functional impact assessed using a 0 to 10 Visual Analogue Scale (VAS) for overall mouth function, with higher scores mean worse outcome

Quantification of lip continence using a self-administered drooling questionnaire (Drooling Severity and Frequency Scale [DSFS]), score ranging from 2 to 9 with higher scores mean worse outcome

Speech intelligibility assessed using a self-administered questionnaire measuring speech-related intelligibility, the Intelligibility in Context Scale (ICS). Score ranging from 7 to 35, with higher scores mean better outcome

Overall patient satisfaction assessed with a Likert scale ranging from -5 to +5 (0 = no improvement and no worsening), higher scores mean better outcome

Using achievement of personalized goals (maximum of three), predefined by the patient together with the investigator, assessed with the Goal Attainment Scale (GAS; scores ranging from -2 to +2, with higher scores mean better outcome). The Goal Attainment Scale (GAS) is a method for measuring treatment effectiveness by creating patient-specific outcome scales that are predefined before study participation. GAS aims to identify goals that are meaningful to the patient and related to their daily activities and individual priorities (Steinbook RM, 1977; DOI: 10.1001/archpsyc.1977.01770200061005).

Total number of painful days assessed using either a patient diary or the online ePRO portal.

Number of painful days with a VAS pain score > 3/10 assessed using either a patient diary or the online ePRO portal.

Venous malformation volume assessed before the intervention (within 6 months prior to treatment) and 12 months after the intervention (performed as part of routine care). Volumetric assessment will be conducted using a semi-automated thresholding method. Image analysis will be centralized and performed by an independent radiologist blinded to treatment allocation.

D-dimer

Fibrinogen level

Safety including adverse events and complications, recorded using a patient diary or the online ePRO portal

Acceptability of the interventions for the patient (or for parents in children aged < 6 years), assessed using a 0 to 10 Visual Analogue Scale (VAS), with higher scores mean worse outcome

In each arm, patients' healthcare utilization related to the procedure and outpatient care will be measured using hospital databases and a patient diary, then valued in euros from the perspective of the healthcare system.