Interdisciplinary Workshop on Vascular Anomalies Associated with Coagulopathies

描述（由申请人提供）：该资助申请要求支持多学科临床医生和科学家会议，重点关注与高发病率和死亡率相关的两种血管异常，Kaposiform血管内皮瘤（KHE）和多灶性淋巴管内皮瘤病伴血小板减少症（MLT）。KHE和簇状血管瘤是罕见的血管肿瘤，通常与Kasabach Merritt现象（KMP）有关，这是一种消耗性凝血病。MLT是另一种罕见的血管异常，与凝血病和危及生命的出血事件相关。由于这两种疾病都涉及多个器官，这些疾病本质上需要跨学科的方法来护理和研究。对这些疾病的婴儿和儿童的临床管理受到这些病变认识不足，治疗无效以及多学科护理提供者之间缺乏沟通的阻碍。我们小组的调查已经为这两种疾病提供了必要的基础知识，因此这样一个研讨会提供的额外专业知识将迅速提供翻译活动，从而改善临床实践。拟议的研讨会将汇集血管异常领域的专家临床医生和研究人员，努力交流机构经验，并促进为这些罕见患者群体集体创建创新治疗方案和算法。本次研讨会的总体目标是为临床专家、临床科学家和具有KHE/KMP或MLT患者经验的基础科学研究人员提供一个开放和透明的交流论坛。从这些讨论中出现，我们期望：（1）确定机构的不一致性和知识差距，（2）产生临床护理指南，（3）加速研究指令和科学发现的儿童与KHE与和没有KMP和MLT。多名医生在这一领域的集体经验将提供统一和商定的准确诊断标准和最佳治疗做法，并将通过医学期刊上的出版物向整个科学界传播。 公共卫生相关性：拟议的会议将汇集来自全国各地的血管异常领域的专家医生和研究人员，以进一步促进科学界对两个领域的理解：（1）卡波西样血管内皮瘤和簇状血管瘤伴或不伴Kasabach Merritt现象和（2）多灶性淋巴管内皮瘤病伴血小板减少症。在这次多学科研讨会结束时，医生将更好地了解如何照顾患有这两种疾病的婴儿和儿童，研究人员将能够优先考虑未来的研究领域，所有信息将通过在医学期刊上发表结果与世界各地的科学家分享。

DESCRIPTION (provided by applicant): This grant application requests support for a meeting of multidisciplinary clinicians and scientists focusing on two vascular anomalies associated with high morbidity and mortality, Kaposiform hemangioendothelioma (KHE) and multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT). KHE and tufted angioma are rare vascular tumors often associated with Kasabach Merritt Phenomenon (KMP), a form of consumptive coagulopathy. MLT is another rare vascular anomaly associated with coagulopathy and life-threatening bleeding events. Since both of these disorders involve multiple organs, the disorders inherently require an interdisciplinary approach to care and research. Clinical management of infants and children with these disorders is hampered by under-recognition of these lesions, ineffective therapies, and lack of communication between multidisciplinary care providers. Investigations by our group have already contributed essential fundamental knowledge about these two disorders so that the additional expertise provided by such a workshop will quickly provide translational activities that will improve clinical practice. The proposed workshop will bring together expert clinicians and investigators in the field of vascular anomalies in an effort to exchange institutional experience and foster the collective creation of innovative treatment protocols and algorithms for these rare patient populations. The overall goal of this workshop is to provide a forum for open and transparent communication between expert clinicians, clinician scientists, and basic science researchers with experience with KHE/KMP or MLT patients. Emergent from these discussions, we expect: (1) identification of institutional inconsistencies and knowledge gaps, (2) generation of clinical care guidelines, and (3) acceleration of research directives and scientific discoveries for children with KHE with and without KMP and MLT. The collective experience of multiple physicians in this area will provide uniform and agreed upon criteria for accurate diagnosis and best practices for treatment that will be disseminated to the entire scientific community through publications in medical journals. PUBLIC HEALTH RELEVANCE: The proposed conference will bring together expert physicians and researchers in the field of vascular anomalies from across the country to further the scientific community's understanding in two areas: (1) kaposiform hemangioendothelioma and tufted angioma with and without Kasabach Merritt Phenomenon and (2) multifocal lymphangioendotheliomatosis with thrombocytopenia. At the conclusion of this multidisciplinary workshop, doctors will have a better understanding of how to care for infants and children with these two diseases, researchers will be able to prioritize future areas of research, and all information will be shared with scientists worldwide through the publications of results in medical journals.