Current clinical evidence is insufficient to support HMME–PDT as the first choice of treatment for young children with port wine birthmarks

IF 2.2 3区医学 Q2 DERMATOLOGY

Lasers in Surgery and Medicine Pub Date : 2024-03-20 DOI:10.1002/lsm.23779

Chao Gao MSc, Vi Nguyen BA, Marcelo L. Hochman MD, Lin Gao MD, Elliott H. Chen MD, Harold I. Friedman MD, PhD, John Stuart Nelson MD, PhD, Wenbin Tan PhD

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Abstract

Background

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.

Aim

To evaluate whether HMME–PDT is superior to PDL by comparing the clinical efficacies of both modalities.

Method

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.

Result

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.

Conclusion

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.

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目前的临床证据不足以支持将 HMME-PDT 作为治疗患有葡萄酒胎记的幼儿的首选方法。

背景：葡萄酒港胎记（PWB）是一种先天性皮肤血管畸形。脉冲染料激光（PDL）是全球治疗葡萄酒胎记的 "金标准"。目的：通过比较HMME-PDT和PDL的临床疗效，评估HMME-PDT是否优于PDL：方法：在PubMed上检索所有使用PDL（1988-2023年）或HMME-PDT（2007-2023年）治疗PWB的相关研究。提取患者特征和临床疗效。纳入了具有四分位数百分比清除率或类似比例的研究。计算每项研究的平均颜色清除指数（CI），并在各组间进行比较。根据组群规模对数据进行加权后得出的总CI（C0）用于评估每种方法的最终疗效：结果：中国共有 18 项 HMME-PDT 研究、3910 名患者符合纳入分析的条件。同样，来自 9 个不同国家的 40 项 PDL 研究（5094 名患者）也符合纳入本分析的条件。HMME-PDT研究中超过58%的患者为未成年人（0 ，0.54 vs. 0.48，p = 0.733）、仅有未成年人的亚人群（C0 ，0.54 vs. 0.46，p = 0.714）和幼儿（C0 ，0.67 vs. 0.50，p = 0.081）。遗憾的是，由于幼儿的血脑屏障与成人相比具有更大的通透性，因此缺乏关于 HMME-PDT 对幼儿，尤其是中枢神经系统发育的疗效和影响的长期跟踪评估数据：结论：在所有年龄段的患者中，PDL 的清除率总体上高于 HMME-PDT，尽管微不足道；尤其是在幼儿中，几乎达到了统计学意义。总之，目前的证据不足以支持将 HMME-PDT 作为治疗幼儿血管内皮瘤的首选方法，因为：（1）总体疗效不如 PDL；（2）治疗过程中存在脑膜血管脱靶的风险；（3）需要使用类固醇来减轻副作用；以及（4）缺乏有关 HMME 对幼儿中枢神经系统发育潜在影响的长期数据。

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来源期刊

Lasers in Surgery and Medicine 医学-外科

CiteScore

5.40

自引率

12.50%

发文量

119

审稿时长

1 months

期刊介绍： Lasers in Surgery and Medicine publishes the highest quality research and clinical manuscripts in areas relating to the use of lasers in medicine and biology. The journal publishes basic and clinical studies on the therapeutic and diagnostic use of lasers in all the surgical and medical specialties. Contributions regarding clinical trials, new therapeutic techniques or instrumentation, laser biophysics and bioengineering, photobiology and photochemistry, outcomes research, cost-effectiveness, and other aspects of biomedicine are welcome. Using a process of rigorous yet rapid review of submitted manuscripts, findings of high scientific and medical interest are published with a minimum delay.