Treatment of Resistant Port-wine Stains With Bosentan and Pulsed Dye Laser: a Pilot Study

Centre Hospitalier Universitaire de Nice

Status and phase: Completed; Phase 2

Conditions: Port-wine Stains

Treatments: Drug: Bosentan; Device: Pulsed dye laser (PDL)

Study type: Interventional

Funder types: Other

Identifiers

NCT02317679

13-PP-10

Details and patient eligibility

About

Pulsed dye laser (PDL) is the gold standard treatment for port-wine stains (PWS). However, the outcomes are highly variable due to the new angiogenesis occurring after laser irradiation. Studies suggest that endothelin is involved in the neoangiogenesis that occurred after treatment of port-wine stains by PDL. The main objective of this pilot clinical trial is to evaluate the effectiveness and safety of an inhibitor of endothelin orally taken, the Bosentan, following PDL treatment. Four patients with facial port-wine stain resistant to the PDL treatment will be included. The treatment by Bosentan (2 mg/kg twice daily, maximum 62,5 mg twice daily) will be given one day before the PDL irradiation and continued for 14 days. Only one test area of PWS will be treated with PDL. The primary outcome measure will be an important or complete improvement (Investigator Global Assessment 3 or 4) between treated area and non treated one, 14 days after the end of the treatment which corresponds to one month after the laser PDL session. The evaluation will been performed on standardized pictures by 2 independent physicians blinded to the region treated or not.

Full description

Pulsed dye laser (PDL) is the gold standard treatment for port-wine stains (PWS). However, the outcomes are highly variable due to the new angiogenesis occurring after laser irradiation. Studies suggest that endothelin is involved in the neoangiogenesis that occurred after treatment of port-wine stains by PDL . The main objective of this pilot clinical trial is to evaluate the effectiveness and safety of an inhibitor of endothelin orally taken, the Bosentan, following PDL treatment. Four patients with facial port-wine stain resistant to the PDL treatment will be included. The treatment by Bosentan (2 mg/kg twice daily, maximum 62,5 mg twice daily) will be given one day before the PDL irradiation (maximum surface 100 cm²) and continued for 14 days. Only one test area of PWS will be treated with PDL. The primary outcome measure will be an important or complete improvement (Investigator Global Assessment 3 or 4) between treated area and non treated one, 14 days after the end of the treatment which corresponds to one month after the laser PDL session. The evaluation will been performed on standardized pictures by 2 independent physicians blinded to the region treated or not.

Enrollment: 4 patients

Sex: All

Ages: 7 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Children of 7 years old and over or adults aged under 60
• with a resistant port-wine stain after treatment by PDL will be included in this pilot study.
• The agreement of the parents and the child or the patient alone if major will be required.
• Subjects have to be registered to the social security.
• An informed consent will have to be signed by the parents or the patient if of a suitable age or the patient alone if major.
• An efficient contraception will be mandatory if the patient is female and an age to give birth.

Exclusion criteria

• Hypersensitivity to the Bosentan or to one of its excipients.
• Mild to severe liver disease corresponding to the Child-Pugh Score B or C.
• Serum levels of ASAT and/or ALAT greater three times the upper limit of normal. - Concurrent use of cyclosporine.
• Pregnancy.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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4 participants in 1 patient group

Bosentan and laser

Experimental group

Description:

Patients with PWS resistant to PDL treatment will be included. A test area of the PWS will be treated by pulsed dye laser (PDL) (λ= 595 nm, 7 mm spot diameter, τp= 1.5 ms, same energy density used at the last session for each subject). The treatment by Bosentan (twice daily :2 mg/kg and maximum 62,5 mg) will be given 1 day before the PDL irradiation (maximum area treated 100 cm2) and continued for 14 days. The clinical improvement of the lesions will be evaluated by comparing standardized pictures, 14 days after the end of the treatment by Bosentan which corresponds to 1 month after the laser PDL irradiation. The evaluation will be realized by 2 independent physicians blinded to the area treated or not. Hemoglobin and SGOT/SGPT will be controlled before and after the treatment by Bosentan.

Treatment:

Drug: Bosentan

Device: Pulsed dye laser (PDL)