A Randomized Trial to Determine the Role of Valganciclovir for Asymptomatic Cytomegalovirus Infected Hearing Impaired Infants

确定缬更昔洛韦对无症状巨细胞病毒感染听力障碍婴儿的作用的随机试验

• 批准号: 9976487
• 负责人: Albert H Park
• 金额: $ 63.18万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-07-06 至 2022-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9976487
• 关键词: Accounting; Address; Advocate; Affect; Age; Antiviral Agents; Antiviral Therapy; Area; Auditory Threshold; Brain; Child; Childhood; Chiroptera; Clinical Trials; Collaborations; Communication impairment; Comparative Effectiveness Research; Controlled Clinical Trials; Cytomegalovirus; Data; Data Coordinating Center; Development; Diagnosis; Double-Blind Method; Ear; Effectiveness; Enrollment; Equilibrium; Equipment and supply inventories; Evaluation; Fetus; Frequencies; Goals; Health Care Costs; Healthcare; Healthcare Systems; Hearing; Infant; Institution; Knowledge; Labyrinth; Language; Language Development; Life; Methods; Minority; Mission; Mothers; Multi-Institutional Clinical Trial; National Institute on Deafness and Other Communication Disorders; Nerve; Neutropenia; Newborn Infant; Otolaryngology; Outcome; Outcome Measure; Patient-Focused Outcomes; Patients; Placebos; Policies; Productivity; Public Health; Public Policy; Randomized; Research; Research Design; Research Personnel; Research Priority; Research Support; Risk; Role; Safety; Sensorineural Hearing Loss; Site; Societies; Speech; Strategic Planning; Testing; Time; Utah; Valganciclovir; Work; base; burden of illness; childhood hearing loss; clinical practice; cohort; congenital cytomegalovirus; cost; data management; global health; hearing impairment; hearing screening; improved; infant outcome; language outcome; neonatal infection; neutrophil; permanent hearing loss; prevent; primary outcome; programs; progressive hearing loss; randomized trial; response; screening; sound

PROJECT SUMMARY / ABSTRACT Cytomegalovirus (CMV) can be transmitted from the mother to the fetus and is a leading cause of sensorineural hearing loss (SNHL), which is a condition where the inner ear is unable to convert sound into nerve impulses to the brain. This hearing loss and its detrimental effect on language development contribute nearly $4 billion annually to the health care costs in the U.S. Unlike other types of SNHL, CMV induced hearing loss can be treated. Several clinical trials have demonstrated that antiviral therapy may prevent progressive hearing loss if administered early in life for severely affected (symptomatic CMV) infants. These promising findings have given rise to a debate regarding the best method for identifying and treating less affected but more numerous (asymptomatic CMV) infants with CMV. One approach is CMV testing only those newborns who fail their newborn hearing screening. This path has been advocated as a targeted option to identify those infants at greatest risk to develop progressive hearing loss. Utah is the first state to mandate this approach whereby infants under three weeks of age who fail their newborn hearing screening undergo CMV testing. Since we anticipate maximal benefit from antiviral therapy, we will compare the hearing, and language outcomes of asymptomatic CMV infected valganciclovir (VGC) treated and untreated infants identified via a hearinglosstargetedearlyCMVapproach(HT-CMV).Thisanalysiswillinformpublicpolicyandpotentiallyshift our current clinical practice regarding pediatric hearing loss evaluation. In the proposed work, the investigators will pursue two specific aims: (1) Compare the hearing and language outcomes of asymptomatic CMV-infected hearing-impaired treated to untreated infants via a multi-institutional double-blinded placebo controlled clinical trial. (2) To evaluate the safety of antiviral valganciclovir therapy for asymptomatic CMV-infected hearing impaired infants. This project is directly relevant to the mission of the NIDCD “to support research evaluating approaches to the identification and treatment of communication disorders and patient outcomes .” More specifically, in the 2012–2016 NIDCD Strategic Plan, in the Priority Area of Hearing and Balance Research, Priority Area 4 includes comparative effectiveness research which will inform health care decisions.

项目概要/摘要 巨细胞病毒（CMV）可以从母亲传染给胎儿，是导致胎儿畸形的主要原因。 感音神经性听力损失 (SNHL)，这是一种内耳无法将声音转换为声音的疾病 神经冲动传至大脑。这种听力损失及其对语言发展的不利影响有助于 美国每年花费近 40 亿美元的医疗保健费用。与其他类型的 SNHL 不同，CMV 诱发听力 损失是可以治疗的。多项临床试验表明，抗病毒治疗可以预防进行性进展 如果在生命早期对严重受影响（有 CMV 症状）的婴儿进行治疗，可能会导致听力损失。这些有前途的 研究结果引发了一场关于识别和治疗受影响较小但受影响程度较低的最佳方法的争论。 更多（无症状 CMV）婴儿患有 CMV。一种方法是仅对新生儿进行 CMV 检测 未通过新生儿听力筛查的人。这条路径被提倡作为一个有针对性的选择来识别那些 婴儿发生进行性听力损失的风险最大。犹他州是第一个强制采用这种方法的州 未通过新生儿听力筛查的三周以下婴儿将接受 CMV 测试。 由于我们预计抗病毒治疗会带来最大益处，因此我们将比较听力和语言能力 通过缬更昔洛韦 (VGC) 治疗和未治疗的无症状 CMV 感染婴儿的结果 针对听力损失的早期 CMV 方法 (HT-CMV)。此分析将为公共政策提供信息并可能发生转变 我们目前关于儿科听力损失评估的临床实践。在拟议的工作中，研究人员 将追求两个具体目标：（1）比较无症状 CMV 感染者的听力和语言结果 通过多机构双盲安慰剂对照临床对听力受损的婴儿进行治疗 审判。 (2) 评估抗病毒缬更昔洛韦治疗无症状CMV感染听力的安全性 受损婴儿。该项目与 NIDCD“支持研究评估”的使命直接相关 识别和治疗沟通障碍和患者结果的方法。”更多的 具体而言，在 2012-2016 年 NIDCD 战略计划中，在听力和平衡研究的优先领域中， 优先领域 4 包括比较有效性研究，为医疗保健决策提供信息。

项目成果

The Generalizability of the Clinical Assessment Score-15 for Pediatric Sleep-Disordered Breathing.

小儿睡眠呼吸障碍临床评估评分 15 的普遍性。

• DOI: 10.1002/lary.28428
• 发表时间: 2020
• 期刊: The Laryngoscope
• 作者: Goldstein,NiraA;Friedman,NormanR;Nardone,HeatherC;Aljasser,Abdullah;Tobey,AllisonBJ;Don,Debra;Baroody,FuadM;Lam,DerekJ;Goudy,Steven;Ishman,StaceyL;Arganbright,JillM;Baldassari,Cristina;Schreinemakers,JBS;Wine,ToddM

Brain Magnetic Resonance Imaging in Congenital Cytomegalovirus With Failed Newborn Hearing Screen.

• DOI: 10.1016/j.pediatrneurol.2020.05.006
• 发表时间: 2020-09
• 期刊: Pediatric neurology
• 影响因子: 3.8
• 作者: Hranilovich JA;Park AH;Knackstedt ED;Ostrander BE;Hedlund GL;Shi K;Bale JF Jr
• 通讯作者: Bale JF Jr