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）：重度至极重度听力损失与健康相关生活质量（HRQoL）的可测量缺陷相关，反映了其对口头语言、社交和情感功能以及学习成绩的伴随影响。然而，人工耳蜗植入（CI）的研究集中在与语言和交流相关的疗效的临床测量（例如，听觉、言语、语言技能），并且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来看，HRQoL措施提供了CI对广泛结局影响的独特评估。FDA现在要求这些患者报告结局（PRO）来衡量有临床意义的效果（FDA指南，2009）。到目前为止，还没有针对幼儿及其父母的CI特异性HRQoL测量（Morettin等人，2013年）。本研究的目标是为6至12岁的CI儿童及其父母开发第一个HRQoL工具。申报仪器将使用FDA测量过程。第一阶段包括系统的文献综述和与关键利益相关者（例如，植入外科医生、听力学家）来识别相关内容并创建可测试的概念框架。我们还将与CI儿童及其父母（n = 24对）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，访谈数据将用于创建临时问卷，通过对从两个人工耳蜗植入中心（迈阿密大学、费城儿童医院）招募的24名儿童及其父母进行认知访谈，对这些问卷进行修订和完善。为了提高普遍性，将使用来自不同民族/种族背景的家庭和具有CI的口头/签名儿童来创建工具。这些措施将以英语和西班牙语制定，采用文化敏感的方法，确保这些措施对不以英语为第一语言的家庭有效。在III期研究中，最终工具将用于收集人口统计学多样化样本（n = 80对CI儿童及其父母）的初始可靠性和有效性数据。收敛有效性将使用KINDL（以前研究中使用的通用HRQoL测量）的儿童和父母版本进行测试。据推测，这两个CI特定的措施将表现出很强的信度和效度。这些数据将用于提交一份后续提案，以资助多地点的心理测量验证，包括可靠性测试（即，内部一致性，重测），预测和收敛效度，以及对变化的反应。为CI儿童及其父母制定HRQoL指标将使临床医生能够：1）确定患者和家庭需求的特定领域; 2）测量人工耳蜗植入对患者/家庭功能的影响; 3）比较单侧与双侧CI儿童的语言表现; 4）评估干预措施的有效性（例如，AV治疗、社交技能），以改善植入物的使用;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.