The Natural History of Pediatric Onset Cutaneous and Systemic Mastocytosis

儿童发病的皮肤和系统性肥大细胞增多症的自然史

• 批准号: 8157107
• 负责人: melody c carter
• 金额: $ 12.16万
• 依托单位: NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/8157107
• 关键词: Childhood; Cutaneous; Natural History; Systemic Mastocytosis

The protocol is designed to evaluate children with more extensive disease or systemic mastocytosis and to better understand those children that do not resolve their disease or progression of systemic mastocytosis. Classification of their disease will augment their current management and more clearly define the prognosis. The protocol also follows children with cutaneous disease from prior LAD protocols and families with more than one affected child to identify possible markers of progression. All patients are currently stable and have not had any adverse events. A total of 41 subjects were accrued since protocol approval with all variants of pediatric-onset mastocytosis: urticaria pigmentosa, diffuse cutaneous mastocytosis, mastocytoma, and indolent systemic mastocytosis. The information we are collecting from subjects enrolled in the protocol provides much needed information for the community at large. This protocol will help to identify factors that may be associated with disease that does not resolve. Moreover, in those children with indolent systemic mastocytosis, this protocol will identify prognostic markers for disease progression into adulthood.

该方案旨在评估患有更广泛疾病或全身性肥大细胞增多症的儿童，并更好地了解那些疾病无法解决或全身性肥大细胞增多症进展的儿童。对他们的疾病进行分类将加强他们目前的治疗，并更清楚地确定预后。该方案还跟踪既往LAD方案中患有皮肤病的儿童以及有不止一个受影响儿童的家庭，以确定可能的进展标志。所有患者目前情况稳定，未发生任何不良事件。自方案批准以来，共有41名患有各种儿科发病肥大细胞增多症的受试者：荨麻疹色素性肥大细胞增多症、弥漫性皮肤肥大细胞增多症、肥大细胞瘤和惰性全身性肥大细胞增多症。