title: Topical rapamycin combined with pulsed dye laser in the treatment of capillary vascular malformations in Sturge-Weber syndrome: phase II, randomized, double-blind, intraindividual placebo-controlled clinical trial.
abstract: BACKGROUND
  Sturge-Weber syndrome (SWS) is characterized by port-wine stains (PWS) affecting the face, eyes, and central nervous system. Pulsed dye laser (PDL) is the standard treatment for PWS. Unfortunately, recurrence is frequent because of reformation and reperfusion of blood vessels.
  
  
  OBJECTIVE
  We sought to assess the clinical efficacy of topical rapamycin combined with PDL in PWS of patients with SWS.
  
  
  METHODS
  We conducted a phase II, randomized, double-blind, intraindividual placebo-controlled, clinical trial. We recruited 23 patients with SWS and facial PWS (12 women; median age 33 years, age range 17-65 years) from the University Clinic of Navarra, Spain. Four interventions were evaluated: placebo, PDL + placebo, rapamycin, and PDL + rapamycin. Clinical and histologic responses were evaluated using a chromatographic computerized system, spectrometry, and histologic analyses at 6, 12, and 18 weeks after the intervention.
  
  
  RESULTS
  PDL + rapamycin yielded the lowest digital photographic image score and the lowest percentage of vessels in histologic analysis, and showed a statistically significant improvement compared with the other interventions. The treatment was generally well tolerated.
  
  
  CONCLUSIONS
  PDL was only applied to the lateral parts of the PWS area.
  
  
  CONCLUSIONS
  Topical rapamycin associated with PDL seems to be an effective treatment for PWS in patients with SWS.
authors.affiliation: Department of Dermatology, University Clinic of Navarra, Navarra, Spain.
authors.forename: Laura
authors.initials: L
authors.lastname: Marqués
authors.affiliation: Department of Preventive Medicine and Public Health, Medical School, University Clinic of Navarra, Navarra, Spain.
authors.forename: Jorge M
authors.initials: JM
authors.lastname: Núñez-Córdoba
authors.affiliation: Department of Dermatology, University Clinic of Navarra, Navarra, Spain.
authors.forename: Leyre
authors.initials: L
authors.lastname: Aguado
authors.affiliation: Department of Dermatology, University Clinic of Navarra, Navarra, Spain.
authors.forename: Maider
authors.initials: M
authors.lastname: Pretel
authors.affiliation: Department of Dermatology, Hospital Ramón y Cajal, Madrid, Spain.
authors.forename: Pablo
authors.initials: P
authors.lastname: Boixeda
authors.affiliation: Department of Dermatology, Instituto Valenciano de Oncología, Universidad Católica de Valencia, Valencia, Spain.
authors.forename: Eduardo
authors.initials: E
authors.lastname: Nagore
authors.affiliation: Department of Dermatology, Hospital de Sant Pau i de la Santa Creu, Barcelona, Spain.
authors.forename: Eulalia
authors.initials: E
authors.lastname: Baselga
authors.affiliation: Department of Dermatology, University Clinic of Navarra, Navarra, Spain. Electronic address: predondo@unav.es.
authors.forename: Pedro
authors.initials: P
authors.lastname: Redondo
mesh_terms: D000287:Administration, Topical; D000293:Adolescent; D000328:Adult; D000368:Aged; D002196:Capillaries / Q000002:abnormalities*; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*; D054025:Lasers, Dye / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D019339:Port-Wine Stain / Q000150:complications; D020123:Sirolimus / Q000008:administration & dosage*; D013341:Sturge-Weber Syndrome / Q000150:complications; D054079:Vascular Malformations / Q000209:etiology / Q000628:therapy*; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D020123:Sirolimus
keywords: Sturge-Weber syndrome; capillary vascular malformation; pulsed dye laser; rapamycin