Novel Optical Treatment Approach for Vascular Birthmarks

描述（由申请人提供）：每年有40万名儿童出生时带有葡萄酒色斑（PWS）胎记。他们和他们的家人面临着这种疾病造成的毁灭性的心理和身体后果。该提案是基础科学家和医生科学家之间的多学科合作，旨在改善儿科PWS的治疗，并提供一种新型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，联合方案可以提高目标微血管的去除。我们的长期目标是消除与这些病变相关的身体和心理创伤。本申请旨在研究PDT+PDL的安全性和有效性。中心假设是，PDT+PDL将实现增强的PWS皮肤变白，相比传统的PDL单独。我们的具体目标是：1）识别具有临床适用性的PDT+PDL治疗方案; 2）识别PDT+PDL方案的候选作用机制; 3）识别具有治疗功效的临床相关光剂量;以及4）确定所选PDT+PDL方案对青少年和成人患者的临床结果。拟议的转化研究是创新的，因为它将为儿科皮肤病治疗领域引入一种新的治疗方法。预期的结果是PWS胎记的临床管理的范式转变，以显着提高治疗效果。成功地去除PWS胎记将消除这些病变造成的心理社会损害，并将对受影响的个人及其家庭的生活产生重大而积极的影响。这样的结果将有助于国家卫生研究院的方法学发展的使命，从根本上改善疾病的治疗。 公共卫生相关性：评估PDT+PDL方案的拟议研究预计将产生重要的积极影响，为去除葡萄酒色斑胎记（PWS）提供更有效和更安全的方法。成功地去除PWS胎记将消除这些病变造成的心理社会损害，并将对受影响的个人及其家庭的生活产生重大而积极的影响。

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.