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 Laura Marqu es, MD, a Jorge M. N u~ nez-C ordoba, MD, MPH, PhD, b Leyre Aguado, MD, PhD, a Maider Pretel, MD, PhD, a Pablo Boixeda, MD, PhD, c Eduardo Nagore, MD, PhD, d Eulalia Baselga, MD, PhD, e and Pedro Redondo, MD, PhD a Navarra, Madrid, Valencia, and Barcelona, Spain 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 1 placebo, rapamycin, and PDL 1 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 1 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. Limitations: PDL was only applied to the lateral parts of the PWS area. Conclusion: Topical rapamycin associated with PDL seems to be an effective treatment for PWS in patients with SWS. ( J Am Acad Dermatol 2015;72:151-8.) Key words: capillary vascular malformation; pulsed dye laser; rapamycin; Sturge-Weber syndrome. S turge-Weber syndrome (SWS) is a congenital multisystem disorder characterized by ipsila- teral capillary-venous malformation of the face (port-wine stain [PWS] birthmark), choroidal and episcleral vascular malformation, and leptome- ningeal malformation. 1,2 From the Department of Dermatology a and Department of Preventive Medicine and Public Health, Medical School, b University Clinic of Navarra; Department of Dermatology, Hospital Ram on y Cajal, Madrid c ; Department of Dermatology, Instituto Valenciano de Oncolog  ıa, Universidad Cat olica de Valencia d ; and Department of Dermatology, Hospital de Sant Pau i de la Santa Creu, Barcelona. e Supported by grant Investigaci on Cl  ınica Independiente EC10-322 from Ministerio de Sanidad, Spain. Conflicts of interest: None declared. Accepted for publication October 8, 2014. Reprint requests: Pedro Redondo, MD, PhD, Department of Dermatology, University Clinic of Navarra, 31008 Pamplona, Navarra, Spain. E-mail: predondo@unav.es. Published online November 11, 2014. 0190-9622/$36.00 Ó 2014 by the American Academy of Dermatology, Inc. http://dx.doi.org/10.1016/j.jaad.2014.10.011 Abbreviations used: HIF: hypoxia-inducible factor OV: office visit PDL: pulsed dye laser PWS: port-wine stain SWS: Sturge-Weber syndrome 151