Longitudinal CMV

本课题是关于伐更昔洛韦治疗长期听力和听力耐受性的纵向研究。 婴儿时期接受症状性先天性疾病治疗的青少年的神经发育结果 巨细胞病毒(CMV)疾病研究中心将由医学博士大卫·W·金伯林领导。它使用了一个随机队列，来自 婴儿症状性先天性巨细胞病毒病早期治疗研究 治疗对2岁以下儿童的听力和发育结果的益处在接下来的时间里持续存在 十年。这项名为CASG 112试验的研究是由合作抗病毒研究小组进行的 并在2008至2011年间招收了109名受试者。它记录了为期6个月的治疗 与6周相比，有症状的先天性巨细胞病毒可提供更好的听力和发育结果 这些好处一直持续到2岁。然而，尚不清楚的是， 这些益处是否持续更长时间，以及是否有任何长期毒性 伐更昔洛韦在生命的早期就提供了治疗。 之前参加CASG 112研究的受试者即将迎来他们的12岁生日。我们会把他们带来的 回到他们12岁时进行听力和发育评估的时候。返回的受试者也将 将青春期评估作为早期valganciclovir疗法潜在性腺毒性的标志， 并询问任何癌症的发展情况。 这项研究的结果将是重要的。如果这些对听力和发育的益处是持久的，它 建议疗程不超过6个月。另一方面，如果这些好处 6个月的治疗是不持久的(也就是说，如果它们随着时间的推移而消失)，这是同等的价值，因为它 阐明了需要进行的下一项研究，以评估可能12个月(或甚至是)的治疗 更长时间)，添加或不添加第二种抗病毒药物，如来特莫韦。此外，如果长期毒性 从早期的伐更昔洛韦疗法来看，这将对临床治疗产生直接影响 有症状的先天性巨细胞病毒病患者。最重要的是，我们将从中获得知识 遵循现在标准的长期听力和发育结果的纵向研究 治疗持续时间(6个月)，这将使接受该疗程的儿童受益。

This project, A Longitudinal Study of the Durability of Valganciclovir Therapy on Long-Term Hearing and Neurodevelopmental Outcomes in Adolescents Treated During Infancy for Symptomatic Congenital Cytomegalovirus (CMV) Diseases, will be led by David W. Kimberlin, MD. It uses a randomized cohort from an earlier study of the treatment of infants with symptomatic congenital CMV disease to ascertain whether the treatment benefits on hearing and developmental outcomes seen to 2 years of age persist over the ensuing decade. That study, known as the CASG 112 trial, was conducted by the Collaborative Antiviral Study Group and enrolled 109 subjects between 2008 and 2011. It documented that a 6 month duration of treatment of symptomatic congenital CMV provided superior hearing and developmental outcomes compared with 6 weeks of antiviral treatment, and that these benefits persisted to 2 years of age. What is not known, though, is whether these benefits persist longer than this, and whether there are any longer-term toxicities from valganciclovir therapy provided very early in life. Subjects previously enrolled in the CASG 112 study are approaching their 12th birthday. We will bring them back when they are 12 years of age for hearing and developmental assessments. Returning subjects also will have a pubertal assessment as a marker for potential gonadotoxicity from the earlier valganciclovir therapy, and for inquiry about development of any cancers. The results of this study will be important. If these benefits on hearing and development are durable, it suggests that treatment does not need to be longer than 6 months duration. On the other hand, if the benefits of 6 months of treatment are not durable (that is, if they wane over time), this is of equal value because it illumines the next study that needs to be conducted to assesses treatment of perhaps 12 months (or even longer), with or without the addition of a second antiviral drug such as letermovir. Also, if longer-term toxicities from the earlier valganciclovir therapy are identified, this will have a direct impact on the clinical treatment of patients with symptomatic congenital CMV disease. Most importantly, knowledge will be gained from this longitudinal study of long-term hearing and developmental outcomes following what is now the standard duration of treatment (6 months) that will benefit children who have received this course of therapy.