Health-Related Quality of Life Instruments for Children with Cochlear Implants: Development of Child and Parent Proxy Measures

 描述(由申请人提供)：严重到严重的听力损失与健康相关生活质量(HRQOL)的可测量缺陷有关，反映出其对口语、社交和情感功能以及学习表现的伴随影响。然而，对人工耳蜗术的研究主要集中在与语言和交流(如听觉、言语、语言技能)相关的疗效的临床测量上，而没有捕捉到植入物对日常功能的影响。从儿童和家庭的角度，HRQOL测量提供了对CI对广泛结果的影响的独特评估。FDA现在要求这些患者报告的结果(PRO)来衡量临床上有意义的效果(FDA指南，2009年)。到目前为止，还没有针对幼儿及其父母的针对CI的HRQOL测量(Morettin等人，2013年)。本研究的目标是为6至12岁的顺位儿童及其父母开发第一个HRQOL量表。建议的仪器将使用FDA的测量过程。第一阶段包括系统的文献回顾和与关键利益相关者(例如，种植外科医生、听力学专家)的焦点小组，以确定相关内容并创建可测试的概念性框架。我们还将对患有顺式耳聋的儿童及其父母(n=24例)进行开放式访谈，以了解儿童耳聋对日常功能的影响。在第二阶段，访谈数据将被用来创建临时问卷，这些问卷将通过对24名儿童及其父母的认知访谈进行修订和完善，这些儿童和他们的父母从两个人工耳蜗植入中心(迈阿密大学、费城儿童医院)招募。为了增加普适性，将使用来自不同族裔/种族背景的家庭和口述/手语的独联体儿童来创建这些文书。这些措施将用英语和西班牙语制定，利用对文化敏感的方法，以确保这些措施对不将英语口语作为第一语言的家庭有效。在第三阶段，最后的工具将被用来收集人口统计学不同样本(n=80对独联体儿童及其父母)的初步可靠性和有效性数据。收敛效度将用Kindl的儿童和父母版本(先前研究中使用的一般HRQOL测量)进行测试。假设这两种特定于CI的测量方法都将显示出很强的信度和效度。这些数据将用于提交后续建议，以资助多地点心理测量验证，包括对可靠性(即内部一致性、重测)、预测和收敛有效性以及对变化的响应性的测试。为顺位感染儿童及其父母开发的HRQOL测量方法将使临床医生能够：1)确定患者和家庭需求的特定领域；2)衡量人工耳蜗植入对患者/家庭功能的影响；3)比较单侧和双侧顺位感染儿童的语言能力；4)评估改善植入物使用的干预措施(例如，AV治疗、社交技能)的有效性；以及5)提供以患者为中心的临床信息，以改善护理。

 DESCRIPTION (provided by applicant): Severe to profound hearing loss is associated with measurable deficits in health-related quality of life (HRQoL), reflecting its concomitant effects o oral language, social and emotional functioning, and academic performance. However, studies of cochlear implantation (CI) have focused on clinical measures of efficacy related to language and communication (e.g., auditory, speech, language skills), and have not captured the implant's effects on daily functioning. HRQoL measures provide a unique assessment of the effects of a CI on a broad range of outcomes, from the child and family's perspective. These patient-reported outcomes (PROs) are now required by the FDA to measure clinically meaningful effects (FDA Guidance, 2009). To date, there are no CI-specific HRQoL measures for young children and their parents (Morettin et al., 2013). The goal of this study is to develop the first HRQoL instruments for children with CIs, ages 6 to 12, and their parents. The proposed instruments will use the FDA measurement process. Phase I includes a systematic literature review and focus groups with key stakeholders (e.g., implant surgeons, audiologists) to identify relevant content and create a testable, conceptual framework. We will also conduct open-ended interviews with children with CIs and their parents (n = 24 dyads) to understand the impacts of childhood deafness on daily functioning. In Phase II, data from the interviews will be used to create provisional questionnaires that will be revised and refined through cognitive interviews with 24 children and their parents recruited from two cochlear implant centers (The University of Miami, Children's Hospital of Philadelphia). To increase generalizability, the instruments will be created using families from varied ethnic/racial backgrounds and oral/signing children with CIs. The measures will be developed in both English and Spanish, utilizing a culturally sensitive approach to ensure the measures are valid for families who do not use spoken English as a first language. In Phase III, the final instruments will be used to collect initial reliability and validty data in a demographically diverse sample (n = 80 dyads of children with CIs and their parents). Convergent validity will be tested with the child and parent versions of the KINDL (a generic HRQoL measure used in previous research). It is hypothesized that both CI- specific measures will demonstrate strong reliability and validity. These data will be used to submit a follow-up proposal to fund a multi-site psychometric validation, including tests of reliability (i.e., internl consistency, test- retest), predictive and convergent validity, and responsivity to change. Development of HRQoL measures for children with CIs and their parents will enable clinicians to: 1) identify specific areas of patient and family need; 2) measure the effects of cochlear implantation on patient/family functioning; 3) compare language performance in children with unilateral vs. bilateral CIs; 4) assess the efficacy of interventions (e.g., AV therapy, social skils) to improve implant use; and 5) provide patient-centered clinical information to improve care.