背景:葡萄酒胎记(PWB)是皮肤的先天性血管畸形。脉冲染料激光(PDL)是全球PWB治疗的“金标准”。血卟啉单甲醚(HMME或血泊芬)介导的光动力疗法(HMME-PDT)已成为PWB治疗的首选,特别是对于年幼的孩子,在过去的几十年里,中国的许多大医院。
目的:通过比较两种模式的临床疗效,评估HMME-PDT是否优于PDL。
方法:检索PubMed记录,查找使用PDL(1988-2023)或HMME-PDT(2007-2023)进行PWB治疗的所有相关研究。提取患者特征和临床疗效。包括具有四分位数百分比清除率或类似量表的研究。计算每个研究的平均颜色清除指数(CI),并在组间进行比较。总CI(C0),数据按队列大小加权,用于评估每种模式的最终疗效。
结果:共有18项HMME-PDT研究纳入了中国的3910例患者。同样,来自9个不同国家的5094名患者的40项PDL研究符合纳入本分析的条件。在HMME-PDT研究中,超过58%的患者是未成年人(<18岁)。很大一部分(21.3%)是幼儿(<3岁)。同样,PDL研究中33.2%的患者为未成年人。一小部分(9.3%)是幼儿。PDL的总体清除率略有下降,但并不重要,在所有年龄段的患者中,HMME-PDT高于HMME-PDT(C0,0.54vs.0.48,p=0.733),只有未成年人的亚群(C0,0.54vs.0.46,p=0.714),和年幼的孩子(C0,0.67vs.0.50,p=0.081)。遗憾的是,缺乏关于HMME-PDT对幼儿的疗效和影响的随访评估的长期数据,特别是中枢神经系统发育,因为与成年人相比,他们的血脑屏障具有更大的渗透性。
结论:PDL显示,在所有年龄段的患者中,总体清除率明显高于HMME-PDT;在幼儿中几乎达到了统计学意义。总的来说,目前的证据不足以支持HMME-PDT作为幼儿PWBs的首选治疗方案:(1)与PDL相比总体疗效较差;(2)手术期间脱靶暴露于脑膜脉管系统的风险;(3)给予药物缓解副作用;(4)缺乏关于HMME对幼儿中枢神经系统发育潜在影响的长期数据.
Port wine birthmark (PWB) is a congenital vascular malformation of the skin. Pulsed dye laser (PDL) is the \"gold standard\" for the treatment of PWB globally. Hematoporphyrin monomethyl ether (HMME or hemoporfin)-mediated photodynamic therapy (HMME-PDT) has emerged as the first choice for PWB treatment, particularly for young children, in many major hospitals in China during the past several decades.
To evaluate whether HMME-PDT is superior to PDL by comparing the clinical efficacies of both modalities.
PubMed records were searched for all relevant studies of PWB treatment using PDL (1988-2023) or HMME-PDT (2007-2023). Patient characteristics and clinical efficacies were extracted. Studies with a quartile percentage clearance or similar scale were included. A mean color clearance index (CI) per study was calculated and compared among groups. An overall CI (C0), with data weighted by cohort size, was used to evaluate the final efficacy for each modality.
A total of 18 HMME-PDT studies with 3910 patients in China were eligible for inclusion in this analysis. Similarly, 40 PDL studies with 5094 patients from nine different countries were eligible for inclusion in this analysis. Over 58% of patients in the HMME-PDT studies were minors (<18 years old). A significant portion (21.3%) were young children (<3 years old). Similarly, 33.2% of patients in the PDL studies were minors. A small proportion (9.3%) was young children. The overall clearance rates for PDL were slightly, but not significantly, higher than those for HMME-PDT in cohorts with patients of all ages (C0, 0.54 vs. 0.48, p = 0.733), subpopulations with only minors (C0, 0.54 vs. 0.46, p = 0.714), and young children (C0, 0.67 vs. 0.50, p = 0.081). Regrettably, there was a lack of long-term data on follow-up evaluations for efficacy and impact of HMME-PDT on young children in general, and central nervous system development in particular, because their blood-brain barriers have a greater permeability as compared to adults.
PDL shows overall albeit insignificantly higher clearance rates than HMME-PDT in patients of all ages; particularly statistical significance is nearly achieved in young children. Collectively, current evidence is insufficient to support HMME-PDT as the first choice of treatment of PWBs in young children given: (1) overall inferior efficacy as compared to PDL; (2) risk of off-target exposure to meningeal vasculature during the procedure; (3) administration of steriods for mitigation of side effects; -and (4) lack of long-term data on the potential impact of HMME on central nervous system development in young children.