Developing patient-reported outcome measures for children and teenagers for clinical trials of sight-restoring interventions in very low and ultra-low vision

为儿童和青少年制定患者报告的结果测量，用于极低和超低视力恢复视力干预措施的临床试验

• 批准号: 498046067
• 负责人: Professor Dr. Robert Patrick Finger, Ph.D.
• 金额: --
• 依托单位: Augenklinik
• 依托单位国家: 德国
• 项目类别: Priority Programmes
• 资助国家: 德国
• 项目状态: 未结题
• 来源: https://gepris.dfg.de/gepris/projekt/498046067?language=en
• 关键词: Developing; patient; reported; outcome; measures

Sight-restoring new technologies and treatments are rapidly evolving, including pharmaceutical treatments, gene therapy, stem cells and retinal visual prostheses. As many of the causes addressed by these interventions lead to early onset visual loss, children and teenagers participate in the respective clinical trials. At this stage, interventional trials are often limited to persons who have little or no vision, and any improvements in visual function are expected to be small and unlikely to be captured in conventional clinical measurements. Thus, the evaluation of therapeutic efficacy should include patient-reported outcome measures (PROMs) such as vision-related quality of life (VRQoL) and visual functioning. We know that PROMs for children and teenagers need to be age-appropriate in content and display, and that children below the age of eight years should be excluded from self-report for a number of reasons such need for proxy report. Thus, we will concentrate on children aged 8 to 18 in this project. Recently, robust PROM instruments targeting children and teenagers with severe visual impairment or blindness were developed by Prof. Jugnoo Rahi (collaborator in this project). To be age-appropriate two versions (children 8-12 and teenagers 13-18 years) were created for both the Functional Vision Questionnaire for Children and Young People (FVQ_CYP) and the Vision related Quality of Life instrument for Children and Young People (VQol_CYP). Both PROMs are theoretically suited as endpoints for clinical trials pending several hurdles being addressed:1. Additional language versions are needed: Any trials will be multi-national as only rare diseases cause visual impairment in these age groups. Thus we will create German PROM versions.2. PROM instruments must yield high-quality data, be psychometrically robust and allow for comparison across different language versions: We will assess psychometric performance for the UK English and German version including any calibration needed to allow for sample-independent comparisons using modern psychometric theory e.g. the Rasch model.3. Allow for continuous measurement across age-appropriate versions:As respondents naturally age whilst participating in clinical trials they may start with the child version but continue with the version for teenagers which requires calibration across both versions. This calibration has not been done for the FVQ_CYP nor the VQoL_CYP.This project will deliver ready to use PROMs for children and teenagers with visual impairment for use in clinical trials, allowing children and teenagers participating in the evaluation of tested interventions as well as enabling them to voice their preferences and share in the medical decision making.

恢复视力的新技术和治疗方法正在迅速发展，包括药物治疗、基因治疗、干细胞和视网膜视觉假体。由于这些干预措施解决的许多原因导致早发性视力丧失，儿童和青少年参加了相应的临床试验。在这个阶段，干预性试验通常仅限于视力很差或没有视力的人，预计视觉功能的任何改善都很小，不太可能在传统的临床测量中获得。因此，疗效评价应包括患者报告的结局指标（PROM），如视觉相关生活质量（VRQoL）和视觉功能。我们知道，儿童和青少年的PROM在内容和显示方面需要与年龄相适应，并且出于一些原因，例如需要代理报告，应将8岁以下的儿童排除在自我报告之外。因此，我们将在这个项目中集中关注8至18岁的儿童。最近，Jugnoo Rahi教授（该项目的合作者）开发了针对患有严重视力障碍或失明的儿童和青少年的强大PROM仪器。为了适合年龄，为儿童和青年人功能性视觉问卷（FVQ_Qs）和儿童和青年人视觉相关生活质量量表（VQol_Qs）创建了两个版本（8-12岁儿童和13-18岁青少年）。这两种PROM理论上都适合作为临床试验的终点，有待解决的几个障碍：1。 需要额外的语言版本：任何试验都将是多国的，因为只有罕见疾病才会导致这些年龄组的视力障碍。因此，我们将创建德国PROM版本。2. PROM仪器必须产生高质量的数据，在心理测量上是稳健的，并允许在不同的语言版本之间进行比较：我们将评估英国英语和德语版本的心理测量性能，包括使用现代心理测量理论（例如Rasch模型）进行样本独立比较所需的任何校准。 允许在适合年龄的版本之间进行连续测量：由于受访者在参与临床试验时自然老化，他们可以从儿童版本开始，但继续使用青少年版本，这需要在两个版本之间进行校准。该项目将为视力障碍儿童和青少年提供即用型PROM，用于临床试验，使儿童和青少年能够参与对测试干预措施的评估，并使他们能够表达自己的偏好，分享医疗决策。