Interdisciplinary Workshop on Vascular Anomalies Associated with Coagulopathies

描述(由申请者提供)：这项拨款申请请求支持一次多学科临床医生和科学家的会议，重点关注与高发病率和死亡率相关的两种血管异常，卡波西样血管内皮瘤(KHE)和多灶性淋巴管内皮细胞瘤伴血小板减少症(MLT)。KHE和簇状血管瘤是一种罕见的血管肿瘤，常与Kasabach Merritt现象(KMP)有关，KMP是一种消耗性凝血障碍。MLT是另一种罕见的血管异常，与凝血障碍和危及生命的出血事件有关。由于这两种疾病都涉及多个器官，因此这些疾病本身就需要一种跨学科的方法来护理和研究。患有这些疾病的婴儿和儿童的临床管理受到对这些损害的认识不足、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提供了关于这两种疾病的基本基础知识，因此这样的研讨会提供的额外专业知识将迅速提供将改善临床实践的翻译活动。拟议的研讨会将把血管异常领域的专家临床医生和研究人员聚集在一起，努力交流机构经验，并促进为这些罕见的患者群体集体创建创新的治疗方案和算法。该研讨会的总体目标是为具有KHE/KMP或MLT患者经验的专业临床医生、临床科学家和基础科学研究人员之间提供一个公开和透明的交流论坛。从这些讨论中，我们期望：(1)识别机构的不一致和知识差距，(2)生成临床护理指南，以及(3)加速KHE儿童的研究指导和科学发现，无论有没有KMP和MLT。多名医生在这一领域的集体经验将为准确诊断和最佳治疗提供统一和商定的标准，并将通过医学期刊上的出版物向整个科学界传播。 与公共卫生相关：拟议中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，以促进科学界在两个领域的理解：(1)有或没有Kasabach Merritt现象的卡波西样血管内皮瘤和簇状血管瘤，(2)伴有血小板减少的多灶性淋巴管内皮瘤。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的结果与世界各地的科学家共享。

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.