Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt Universität zu Berlin, and Berlin Institut of Health, SPZ Interdisziplinär, Kinderendokrinologie und -diabetologie, Augustenburger Platz 1, 13353, Berlin, Germany.
Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin, Humboldt-Universität zu Berlin, and Berlin Institute of Health, Institute of Social Medicine, Epidemiology and Health Economics, Berlin, Germany.
BMC Endocr Disord. 2022 Jun 27;22(1):166. doi: 10.1186/s12902-022-01079-3.
Differences in sexual development (DSD) are rare diseases, which affect the chromosomal, anatomical or gonadal sex differentiation. Although patient education is recommended as essential in a holistic care approach, standardised programmes are still lacking. The present protocol describes the aims, study design and methods of the Empower-DSD project, which developed an age-adapted multidisciplinary education programme to improve the diagnosis-specific knowledge, skills and empowerment of patients and their parents.
The new patient education programme was developed for children, adolescents and young adults with congenital adrenal hyperplasia, Turner syndrome, Klinefelter syndrome or XX-/or XY-DSD and their parents. The quantitative and qualitative evaluation methods include standardised questionnaires, semi-structured interviews, and participatory observation. The main outcomes (assessed three and six months after the end of the programme) are health-related quality of life, disease burden, coping, and diagnosis-specific knowledge. The qualitative evaluation examines individual expectations and perceptions of the programme. The results of the quantitative and qualitative evaluation will be triangulated.
The study Empower-DSD was designed to reduce knowledge gaps regarding the feasibility, acceptance and effects of standardised patient education programmes for children and youth with DSD and their parents. A modular structured patient education programme with four generic and three diagnosis-specific modules based on the ModuS concept previously established for other chronic diseases was developed. The topics, learning objectives and recommended teaching methods are summarised in the structured curricula, one for each diagnosis and age group. At five study centres, 56 trainers were qualified for the implementation of the training programmes. A total of 336 subjects have been already enrolled in the study. The recruitment will go on until August 2022, the last follow-up survey is scheduled for February 2023. The results will help improve multidisciplinary and integrated care for children and youth with DSD and their families.
German Clinical Trials Register, DRKS00023096 . Registered 8 October 2020 - Retrospectively registered.
性发育差异（DSD）是罕见疾病，影响染色体、解剖结构或性腺性别分化。尽管推荐以整体护理方法为患者提供教育，但仍缺乏标准化的方案。本方案描述了 Empower-DSD 项目的目标、研究设计和方法，该项目开发了一个适合年龄的多学科教育方案，以提高患者及其父母的诊断特异性知识、技能和赋权。
新的患者教育方案针对患有先天性肾上腺皮质增生症、特纳综合征、克莱恩费尔特综合征或 XX-/或 XY-DSD 的儿童、青少年和年轻人及其父母而制定。定量和定性评估方法包括标准化问卷、半结构化访谈和参与性观察。主要结局（在方案结束后 3 个月和 6 个月评估）是健康相关生活质量、疾病负担、应对方式和诊断特异性知识。定性评估检查了方案的个体期望和认知。将对定量和定性评估的结果进行三角分析。
Empower-DSD 研究旨在减少关于 DSD 儿童和青少年及其父母的标准化患者教育方案的可行性、接受程度和效果的知识差距。基于先前为其他慢性疾病建立的 ModuS 概念，开发了一种模块化结构的患者教育方案，包含四个通用模块和三个诊断特异性模块。每个诊断和年龄组都有一个结构化课程，其中总结了主题、学习目标和推荐的教学方法。在五个研究中心，有 56 名培训师具备实施培训方案的资格。该研究共招募了 336 名受试者。招募工作将持续到 2022 年 8 月，最后一次随访调查计划于 2023 年 2 月进行。研究结果将有助于改善 DSD 儿童和青少年及其家庭的多学科综合护理。
德国临床试验注册处，DRKS00023096。于 2020 年 10 月 8 日注册-回顾性注册。
