Hemoporfin Photodynamic Therapy for Port-Wine Stain: A Randomized Controlled Trial

Background and ObjectivesPhotodynamic 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.Patients and MethodsThis 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: ResultsCompared 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 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.ConclusionsHemoporfin-mediated PDT is an effective and safe treatment option for adolescent and adult patients with port-wine stain.Trial RegistrationChinese Clinical Trial Registry ChiCTR-TRC-08000213

背景与目的
光动力疗法（Photodynamic Therapy, PDT）在治疗鲜红斑痣（port-wine stain）方面已展现出潜在获益，但其获益-风险比仍未明确。本研究旨在临床评估应用海姆泊芬（hemoporfin）联合532nm连续波激光实施光动力疗法治疗鲜红斑痣的疗效与安全性。

患者与方法
本随机临床试验在中国8家医院开展。受试者为14~65岁的青少年及成年鲜红斑痣患者。在第一阶段（第1日至第8周），所有患者以3:1的比例随机分配至治疗组（给予海姆泊芬[5mg/kg]联合532nm激光照射，照射剂量为96~120 J/cm²，即PDT-海姆泊芬组，n=330）或安慰剂组（仅给予安慰剂联合激光照射，即PDT-安慰剂组，n=110）；第二阶段（第8周至第16周），两组患者均接受治疗。设盲的临床评估者依据病灶区域治疗前后的标准化影像，采用以下四级量表对每例病例进行疗效分级：无改善：

结果
与PDT-安慰剂组相比，PDT-海姆泊芬组在第8周时达到至少部分改善的患者比例显著更高（89.7%[n=295；95%CI：85.9%~92.5%] vs. 24.5%[n=27；95%CI：17.4%~33.3%]）。PDT-海姆泊芬组共完成735例次治疗，其中731例次（95%CI：98.7%~99.8%）治疗成功。PDT-海姆泊芬治疗患者中，色素沉着过度的发生率为每100患者-治疗次22.9例（n=168；95%CI：20.0~26.0）。

结论
海姆泊芬介导的光动力疗法是青少年及成年鲜红斑痣患者安全有效的治疗选择。

试验注册
中国临床试验注册中心ChiCTR-TRC-08000213