Enabling self-reported outcomes for youth with developmental disabilities: The Pediatric Evaluation of Disability Inventory - Patient Reported Outcome (PEDI-PRO) - Phase II

缺乏适合患有神经发育障碍（DD）和相关认知障碍（包括唐氏综合症（DS））的青少年的患者报告的结果测量（PROMs），这对医疗保健研究和实践提出了一个重大问题。父母和其他专业人员不能确定与青少年相同的需求，未能让患有DD的青少年参与医疗保健评估，会影响医疗保健质量和结果。鉴于DD的流行和该人群的显著医疗保健费用，该人群对PROM的市场需求很高，其中遗传病因的最大比例是DS。在第一阶段，我们证明了我们的技术可以帮助患有DD和相关认知障碍的青少年完成PROMs。PEDI-PRO原型显示出很强的内部信度，并从56名14-22岁的患有DD和认知障碍的青少年中获得一致的分数。事实上，第一阶段的奖励证明了该调查在智商范围为47-75的年轻人（其中14%的样本具有DS）中的易用性，以及在智商范围为30-110的年轻人（其中12.7%的样本具有DS）中的重测信度和内部效度。全国的临床医生（n =14）建立了PEDI-PRO原型的可用性。该二期STTR将建立一个临床可靠的PEDI-PRO评估软件，以解决包括DS在内的青少年DD患者的prom市场缺口。具体目标：1)将I期PEDI-PRO概念原型转化为一个强大的，功能齐全的康复评估软件系统。2)对PEDI-PRO题库的心理测量特性进行测试和优化，确保测量结果有效可靠。3)设计临床可解释的评分报告，准确描述青少年的功能表现。

The lack of patient reported outcome measures (PROMs) appropriate for youth with neurodevelopmental disabilities (DD) and related cognitive impairments including Down syndrome (DS) poses a significant problem for healthcare research and practice. Parents and other professionals do not identify the same needs as youth, and failure to engage youth with DD in healthcare evaluation compromises healthcare quality and outcomes. The market demand for a PROM for this population is high given the prevalence of DD and the significant healthcare costs for this population, of which the largest proportion with a genetic etiology is DS. In Phase I, we demonstrated that our technology can help youth with DD and related cognitive impairments complete PROMs. The PEDI-PRO prototype demonstrated strong internal reliability and elicited consistent scores from 56 youth ages 14-22 with DD and cognitive impairments. In fact, the Phase I award demonstrated ease-of-use of the survey in youth with an IQ range of 47-75 (of which 14% of the sample had DS), and the test-retest reliability and internal validity in those with an IQ range of 30-110 (of which 12.7% had DS). Clinicians across the country (n =14) established the usability of the PEDI-PRO prototype. This Phase II STTR will build a clinically robust PEDI-PRO Assessment Software to address the market gap in PROMs for youth with DD including DS. Specific aims: 1) Transform the Phase I PEDI-PRO concept prototype into a robust, fully functioning assessment software system for use in rehabilitation. 2) Test and optimize the psychometric properties of the PEDI-PRO item bank to ensure valid and reliable measurement. 3) Design a clinically interpretable score report that accurately characterizes youth’s functional performance. The Phase II study design adheres to the Consensus-based Standards for the Selection of Measurement Instruments (COSMIN) framework to establish the validity and reliability of the PEDI-PRO. We will continue our partnership with a Youth Panel (n = 5) and Advisory Board (n = 8). We will design the PEDI-PRO Clinical Management System (PCMS: a HIPPA compliant clinician administrative dashboard) and incorporate 45 additional items (15 per domain, selected by the Youth Panel and Advisory Board) into the PEDI-PRO. Second, 300 youth with DD will complete the PEDI-PRO, including 50 individuals with DS, thus exceeding the prevalence estimates that individuals with DS make up 15-20% of those with an intellectual disability. We will adhere to COSMIN standards and examine the structural validity and internal reliability using both classical test theory (CTT) and item response theory (IRT). We will also use hypothesis testing to establish known groups and convergent validity. Third, we will incorporate automatic scoring into the PEDI-PRO Assessment Software; we will use a mixed methods to design and evaluate two score report prototypes. This Phase II will enable AbleLink Technologies to aggressively pursue commercialization through a 9 month to market timeline and distribute the PEDI-PRO Assessment Software for a variety of platforms.