Ostrow K L, Bergner A L, Blakeley J, Evans D G, Ferner R, Friedman J M, Harris G J, Jordan J T, Korf B, Langmead S, Leschziner G, Mautner V, Merker V L, Papi L, Plotkin S R, Slopis J M, Smith M J, Stemmer-Rachamimov A, Yohay K, Belzberg A J
Johns Hopkins University, Baltimore, Maryland.
University of Manchester, Manchester, England.
Am J Med Genet A. 2017 Feb;173(2):407-413. doi: 10.1002/ajmg.a.38024. Epub 2016 Oct 19.
Schwannomatosis is a tumor suppressor syndrome that causes multiple tumors along peripheral nerves. Formal diagnostic criteria were first published in 2005. Variability in clinical presentation and a relative lack of awareness of the syndrome have contributed to difficulty recognizing affected individuals and accurately describing the natural history of the disorder. Many critical questions such as the mutations underlying schwannomatosis, genotype-phenotype correlations, inheritance patterns, pathologic diagnosis of schwannomatosis-associated schwannomas, tumor burden in schwannomatosis, the incidence of malignancy, and the effectiveness of current, or new treatments remain unanswered. A well-curated registry of schwannomatosis patients is needed to facilitate research in field. An international consortium of clinicians and scientists across multiple disciplines with expertise in schwannomatosis was established and charged with the task of designing and populating a schwannomatosis patient registry. The International Schwannomatosis Registry (ISR) was built around key data points that allow confirmation of the diagnosis and identification of potential research subjects to advance research to further the knowledge base for schwannomatosis. A registry with 389 participants enrolled to date has been established. Twenty-three additional subjects are pending review. A formal process has been established for scientific investigators to propose research projects, identify eligible subjects, and seek collaborators from ISR sites. Research collaborations have been created using the information collected by the registry and are currently being conducted. The ISR is a platform from which multiple research endeavors can be launched, facilitating connections between affected individuals interested in participating in research and researchers actively investigating a variety of aspects of schwannomatosis. © 2016 Wiley Periodicals, Inc.
神经鞘瘤病是一种肿瘤抑制综合征,可导致沿周围神经出现多个肿瘤。正式的诊断标准于2005年首次公布。临床表现的变异性以及对该综合征相对缺乏认识,导致难以识别受影响个体并准确描述该疾病的自然史。许多关键问题,如神经鞘瘤病的潜在突变、基因型与表型的相关性、遗传模式、神经鞘瘤病相关神经鞘瘤的病理诊断、神经鞘瘤病中的肿瘤负荷、恶性肿瘤的发生率以及当前或新治疗方法的有效性等,仍未得到解答。需要一个精心策划的神经鞘瘤病患者登记处来促进该领域的研究。一个由多个学科的临床医生和科学家组成的国际联盟成立了,他们在神经鞘瘤病方面具有专业知识,并负责设计和填充神经鞘瘤病患者登记处的任务。国际神经鞘瘤病登记处(ISR)围绕关键数据点建立,这些数据点可用于确认诊断和识别潜在的研究对象,以推进研究,进一步拓展神经鞘瘤病的知识库。目前已建立了一个登记处,登记了389名参与者。另外23名受试者正在等待审核。已为科研人员建立了一个正式程序,以提出研究项目、确定符合条件的受试者,并从ISR站点寻找合作者。利用登记处收集的信息建立了研究合作关系,目前正在开展相关研究。ISR是一个平台,可在此发起多项研究工作,促进有兴趣参与研究的受影响个体与积极研究神经鞘瘤病各个方面的研究人员之间的联系。© 2016威利期刊公司