title: Hemoporfin Photodynamic Therapy for Port-Wine Stain: A Randomized Controlled Trial.
abstract: OBJECTIVE
  Photodynamic therapy (PDT) has shown potentially beneficial results in treating port-wine stain, but its benefit-risk profile remains undefined. This study aimed to evaluate the efficacy and safety of PDT conducted with hemoporfin and a 532 nm continuous wave laser to treat port-wine stain clinically.
  
  
  METHODS
  This randomized clinical trial was conducted in eight hospitals in China. Participants were adolescent and adult patients (age range: 14-65 years old) with port-wine stain. During stage 1 (day 1 to week 8) all patients were randomized at a 3:1 ratio to treatment (532 nm laser irradiation (96-120 J/cm2) with hemoporfin (5mg/kg; PDT-hemoporfin, n = 330)) or placebo groups (irradiation with placebo (PDT-placebo, n = 110)); during stage 2 (week 8 to 16) patients in both groups were offered treatment. Clinician-evaluators, who were blind to the study, classified each case on the following four-level scale according to assessment of before and after standardized pictures of the lesion area: no improvement: <20%; some improvement: 20-59%; great improvement: 60-89%; or nearly completely resolved: ≥90%. The primary efficacy endpoint was proportion of patients achieving at least some improvement at week 8. The secondary efficacy endpoints were proportion of patients achieving nearly completely resolved or at least great improvement at week 8, proportion of patients achieving early completely resolved, at least great improvement, or at least some improvement at week 16, and the corresponding satisfaction of the investigators and the patients (designated as 'excellent', 'good', 'moderate', or 'ineffective') at weeks 8 and 16.
  
  
  RESULTS
  Compared to the PDT-placebo group, the PDT-hemoporfin group showed a significantly higher proportion of patients that achieved at least some improvement (89.7% [n = 295; 95% CI, 85.9%-92.5%] vs. 24.5% [n = 27; 95% CI, 17.4%-33.3%]) at week 8 (P < 0.0001) and higher improvements for all secondary efficacy endpoints. Treatment reactions occurred in 99.5% (n = 731; 95% CI, 98.7%-99.8%) of the PDT-hemoporfin treatments (n = 735). Hyperpigmentation occurred in 22.9 per 100 patient-treatments (n = 168; 95% CI, 20.0-26.0) in the PDT-hemoporfin treated patients.
  
  
  CONCLUSIONS
  Hemoporfin-mediated PDT is an effective and safe treatment option for adolescent and adult patients with port-wine stain.
  
  
  BACKGROUND
  Chinese Clinical Trial Registry ChiCTR-TRC-08000213.
authors.affiliation: Department of Dermatology and Venereology, Peking University First Hospital, Beijing, China.
authors.forename: Yi
authors.initials: Y
authors.lastname: Zhao
authors.affiliation: Department of Dermatology and Venereology, Peking University First Hospital, Beijing, China.
authors.forename: Ping
authors.initials: P
authors.lastname: Tu
authors.affiliation: Department of Oral Maxillofacial Surgery, Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine. Shanghai, China.
authors.forename: Guoyu
authors.initials: G
authors.lastname: Zhou
authors.affiliation: Institute of Dermatology, Chinese Academy of Medical Sciences and Peking Union Medical College, Nanjing, China.
authors.forename: Zhanchao
authors.initials: Z
authors.lastname: Zhou
authors.affiliation: Department of Plastic and Reconstructive Surgery, Ninth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine. Shanghai, China.
authors.forename: Xiaoxi
authors.initials: X
authors.lastname: Lin
authors.affiliation: Department of Dermatology, The General Hospital of Guangzhou Military Command, Guangzhou, China.
authors.forename: Huilan
authors.initials: H
authors.lastname: Yang
authors.affiliation: Department of Dermatology, Huashan Hospital, Fudan University, Shanghai, China.
authors.forename: Zhong
authors.initials: Z
authors.lastname: Lu
authors.affiliation: Institute of Dermatology of Chinese PLA, Xijing Hospital, Fourth Military Medical University, Xi'an, China.
authors.forename: Tianwen
authors.initials: T
authors.lastname: Gao
authors.affiliation: Department of Dermatology, Union Hospital of Tongji Medical College of Huazhong University of Science and Technology, Wuhan, China.
authors.forename: Yating
authors.initials: Y
authors.lastname: Tu
authors.affiliation: Department of Dermatology, Xiangya Hospital Central South University, Changsha, China.
authors.forename: Hongfu
authors.initials: H
authors.lastname: Xie
authors.affiliation: Center for Drug Clinical Research, Shanghai University of Chinese Traditional Medicine, Shanghai, China.
authors.forename: Qingshan
authors.initials: Q
authors.lastname: Zheng
authors.affiliation: Department of Laser Medicine, Chinese PLA General Hospital, Beijing, China.
authors.forename: Ying
authors.initials: Y
authors.lastname: Gu
authors.affiliation: Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd., Shanghai, China.
authors.forename: Jining
authors.initials: J
authors.lastname: Tao
authors.affiliation: Department of Dermatology and Venereology, Peking University First Hospital, Beijing, China.
authors.forename: Xuejun
authors.initials: X
authors.lastname: Zhu
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002681:China; D004311:Double-Blind Method; D005260:Female; D006415:Hematoporphyrins / Q000627:therapeutic use*; D006801:Humans; D028022:Low-Level Light Therapy; D008297:Male; D008875:Middle Aged; D010778:Photochemotherapy; D017319:Photosensitizing Agents / Q000627:therapeutic use*; D019339:Port-Wine Stain / Q000188:drug therapy* / Q000473:pathology; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D006415:Hematoporphyrins; D017319:Photosensitizing Agents; C494379:hematoporphyrin monomethyl ether