Novel Optical Treatment Approach for Vascular Birthmarks

描述(申请人提供)：每年有400,000名儿童出生时会有葡萄酒色斑(PWS)胎记。他们及其家人面临着这种疾病造成的毁灭性的心理和身体后果。这项建议是基础科学家和内科科学家之间的多学科合作，旨在改善儿童PWS的治疗，并提供一种新的治疗方案的临床翻译，即光动力疗法(PDT)和脉冲染料激光(PDL)治疗(以下称为PDT+PDL)。我们的初步数据表明，联合方案可以增强靶向微血管的去除。我们的长期目标是消除与这些损伤相关的身体和心理社会创伤。本应用的目的是研究PDT+PDL的安全性和有效性。中心假设是，与传统的PDL相比，PDT+PDL将实现更好的PWS皮肤漂白。我们的具体目标是：1)确定具有临床适用性的PDT+PDL治疗方案；2)确定PDT+PDL方案的候选作用机制；3)确定临床上相关的光剂量与治疗效果；4)确定选定的PDT+PDL方案在青少年和成人患者中的临床结果。这项拟议的转化性研究具有创新性，因为它将把一种新的治疗方法引入儿童皮肤病治疗领域。预期的结果是PWS胎记临床管理的范式转变，以显著改善治疗结果。成功地去除儿童的PWS胎记将消除这些损害造成的心理社会损害，并将对受影响个人及其家人的生活产生重大和积极的影响。这样的结果将有助于NIH从根本上改进疾病治疗的方法学发展使命。 公共卫生相关性：评估PDT+PDL方案的拟议研究有望产生重要的积极影响，为去除葡萄酒胎记(PWS)提供一种更有效、更安全的方法。成功地去除儿童的PWS胎记将消除这些损害造成的心理社会损害，并将对受影响个人及其家人的生活产生重大和积极的影响。

DESCRIPTION (provided by applicant): Each year 400,000 children are born with port wine stain (PWS) birthmarks. They and their families are confronted with the devastating psychological and physical consequences of this disease. This proposal is a multidisciplinary collaboration between basic and physician scientists designed to improve treatment of pediatric PWS and provide clinical translation of a novel therapeutic regimen, combined photodynamic therapy (PDT) and pulsed dye laser (PDL) treatment (hereafter termed PDT+PDL). Our preliminary data indicate that the combined protocol can enhance the removal of targeted microvasculature. Our long-term goal is to eliminate the physical and psychosocial trauma associated with these lesions. The objective of this application is to investigate the safety and efficacy of PDT+PDL. The central hypothesis is that PDT+PDL will achieve enhanced PWS skin blanching, compared to conventional PDL alone. Our specific aims are to: 1) Identify PDT+PDL therapeutic protocols with clinical applicability; 2) Identify candidate mechanisms of action of the PDT+PDL protocol; 3) Identify clinically relevant light doses with therapeutic efficacy and 4) Determine clinical outcomes of the selected PDT+PDL protocol on adolescent and adult patients. The proposed translational research is innovative because it will introduce a novel treatment method to the field of pediatric dermatological therapy. The expected outcome is a paradigm shift in the clinical management of PWS birthmarks in order to dramatically improve treatment results. Successfully removing PWS birthmarks in children will eliminate the psychosocial damage these lesions inflict, and will significantly and positively impact the life of affected individuals and their families. Such an outcome will contribute to the NIH mission of methodology development to improve fundamentally the treatment of disease. PUBLIC HEALTH RELEVANCE: The proposed studies evaluating the PDT+PDL protocol are expected to have an important positive impact, providing a more effective and safer method for removal of port wine stain birthmarks (PWS). Successfully removing PWS birthmarks in children will eliminate the psychosocial damage these lesions inflict, and will significantly and positively impact the life of affected individuals and their families.