Development and Validation of a Pierre Robin Sequence Quality of Life Instrument

皮埃尔罗宾序列生活质量仪器的开发和验证

• 批准号: 10274483
• 负责人: Jonathan Robert Skirko
• 金额: $ 20.17万
• 依托单位: UNIVERSITY OF ARIZONA
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-01-15 至 2023-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10274483
• 关键词: Address; Affect; Age; Agreement; Back; Breathing; Bypass; Caring; Characteristics; Child; Cleft Palate; Clinical; Clinical Research; Code; Craniofacial Abnormalities; Development; Disease; Emotional; Enteral Feeding; Event; Factor Analysis; Family; Focus Groups; Future; Gastrostomy; Goals; Group Interviews; Hospitalization; Infant; Jaw; Language; Life; Mandible; Measures; Micrognathism; Morbidity - disease rate; Obstruction; Operative Surgical Procedures; Outcome; Parents; Patients; Pierre Robin Syndrome; Polysomnography; Population; Positioning Attribute; Provider; Psychometrics; Public Health; Quality of life; Reporting; Research Personnel; Sampling; Sleep Apnea Syndromes; Structure; Symptoms; Syndrome; Techniques; Testing; Tongue; Tracheostomy procedure; Tracheotomy procedure; Transcript; Treatment Effectiveness; Tube; Validation; Validity and Reliability; Vulnerable Populations; Well in self; age group; airway obstruction; burden of illness; cognitive interview; comparative effectiveness study; contextual factors; craniofacial; craniofacial disorder; distraction; experience; feeding; functional status; improved; indexing; infancy; instrument; mortality; novel; nutrition; physical conditioning; preservation; psychosocial; recruit; response; tongue root; treatment comparison

PROJECT SUMMARY ABSTRACT Children with Pierre Robin Sequence (PRS) are born with micrognathia (small jaw), glossoptosis (tongue pushed back), and airway obstruction; many have cleft palates. PRS patients have breathing and feeding difficulties of varying degrees, which can profoundly affect their own and their families’ quality of life (QoL). Many infants with PRS have complicated and tenuous early lives requiring surgeries such as tracheostomy to bypass upper airway obstruction, surgical feeding tubes for nutrition, or major surgeries to improve their craniofacial anomaly. In addition to the child’s symptoms, parents often struggle to manage their own psychosocial and emotional response to their child’s disorder. Current QoL instruments are too broad in scope and focused on different populations to adequately measure QoL in this population. This not only limits understanding of these patients’ holistic disease burden, it constrains comparative effectiveness studies to outcomes that may be less relevant to patients and families. Our objective during the R21 period is to develop and perform preliminary validation of a PRS-specific QoL instrument measuring both child symptoms and family QoL. Specifically, we will develop PRS QoL items from from focus groups with parents of children with PRS. To achieve this, additional focus groups with PRS families will be conducted, allowing the inclusion of patients from different backgrounds, older age groups, and with treatments that were not included in our original sample. Transcripts will be coded for key themes and concepts. Items developed using the language, themes, and concepts from these focus groups will provide the PRS QoL content validity. Items will be evaluated through cognitive interviews with PRS families and iteratively revised and retested until a preliminary instrument is developed. This instrument will be administered to a representative sample of PRS parents. The instrument’s psychometric characteristics will be assessed with classic item analysis and qualitative techniques, and redundant or ineffective items will be eliminated. The resulting instrument will be tested for validity and reliability. Construct validity will be assessed using responses from PRS families with varying ages, whose scores on the PRS QoL instrument will be compared with scores on validated instruments assessing general QoL, the impact of sleep apnea, and the impact of children’s illness on families. Additionally, PRS QoL scores will be compared with objective measures of airway function and feeding, such as polysomnography reports and feeding tube status. Reliability will be assessed through test-retest assessment. The resulting, preliminarily validated QoL instrument will be a comprehensive measure of disease burden allowing providers and clinical researchers to measure the impact of PRS on children and families. This novel instrument will advance our understanding of the impact this disorder has on families, representing a substantial shift from previous assessments in this vulnerable population. It will also facilitate critically needed future studies comparing PRS treatments.

项目概要摘要 患有皮埃尔·罗宾序列 (PRS) 的儿童出生时患有小颌畸形（小下颌）、舌下垂（舌头） 推回）和气道阻塞；许多人患有腭裂。 PRS 患者有呼吸和进食 不同程度的困难，这会深刻影响他们自己和家人的生活质量（QoL）。 许多患有 PRS 的婴儿的早期生活复杂而脆弱，需要进行气管切开术等手术。 绕过上呼吸道阻塞、外科饲管获取营养或进行大手术以改善其症状 颅面异常。除了孩子的症状外，父母也常常难以控制自己的症状 对孩子疾病的心理社会和情感反应。当前的生活质量工具范围过于广泛 并关注不同人群，以充分衡量该人群的生活质量。这不仅限制 了解这些患者的整体疾病负担，它限制了比较有效性研究 结果可能与患者和家庭不太相关。我们在 R21 期间的目标是开发 并对 PRS 特定的 QoL 仪器进行初步验证，测量儿童症状和 家庭生活质量。具体来说，我们将根据患有以下疾病的儿童家长的焦点小组制定 PRS 生活质量项目： PRS。为了实现这一目标，将与 PRS 家庭进行额外的焦点小组讨论，从而将 来自不同背景、年龄较大的患者，以及接受未包括在我们的治疗范围内的治疗的患者 原始样本。脚本将针对关键主题和概念进行编码。使用该语言开发的项目， 这些焦点小组的主题和概念将提供 PRS 生活质量内容的有效性。物品将是 通过对 PRS 家庭进行认知访谈进行评估，并反复修改和重新测试，直到获得初步结果 仪器研制成功。该工具将针对 PRS 家长的代表性样本进行管理。这 仪器的心理测量特征将通过经典的项目分析和定性评估 技术，多余或无效的项目将被淘汰。所得仪器将进行测试 有效性和可靠性。将使用不同年龄的 PRS 家庭的回答来评估构建有效性， 其 PRS 生活质量工具的分数将与经过验证的工具评估的分数进行比较 一般生活质量、睡眠呼吸暂停的影响以及儿童疾病对家庭的影响。此外，PRS 生活质量 分数将与气道功能和喂养的客观测量进行比较，例如多导睡眠图 报告和饲管状态。可靠性将通过重测评估进行评估。由此产生的， 初步验证的生活质量工具将是疾病负担的综合衡量标准，使提供者能够 和临床研究人员来衡量 PRS 对儿童和家庭的影响。这种新颖的仪器将 增进我们对这种疾病对家庭影响的理解，代表着从 之前对这一弱势群体的评估。它还将促进未来急需的研究 比较 PRS 治疗。