Badiu Corin, Bonomi Marco, Borshchevsky Ivan, Cools Martine, Craen Margarita, Ghervan Cristina, Hauschild Michael, Hershkovitz Eli, Hrabovszky Erik, Juul Anders, Kim Soo-Hyun, Kumanov Phillip, Lecumberri Beatriz, Lemos Manuel C, Neocleous Vassos, Niedziela Marek, Djurdjevic Sandra Pekic, Persani Luca, Phan-Hug Franziska, Pignatelli Duarte, Pitteloud Nelly, Popovic Vera, Quinton Richard, Skordis Nicos, Smith Neil, Stefanija Magdalena Avbelj, Xu Cheng, Young Jacques, Dwyer Andrew A
Department of Endocrinology, National Institute of Endocrinology, C. Davila University of Medicine and Pharmacy, Bucharest, 050474, Romania.
Deptartment of Clinical Sciences & Community Health and the Division of Endocrine and Metabolic Diseases & Laboratory of Endocrine and Metabolic Research, University of Milan, Milan, Italy.
Orphanet J Rare Dis. 2017 Mar 20;12(1):57. doi: 10.1186/s13023-017-0608-2.
Patients with rare diseases face health disparities and are often challenged to find accurate information about their condition. We aimed to use the best available evidence and community partnerships to produce patient education materials for congenital hypogonadotropic hypogonadism (CHH) and the olfacto-genital (Kallmann) syndrome (i.e., CHH and defective sense of smell), and to evaluate end-user acceptability. Expert clinicians, researchers and patients co-created the materials in a multi-step process. Six validated algorithms were used to assess reading level of the final product. Comprehensibility and actionability were measured using the Patient Education Materials Assessment Tool via web-based data collection. Descriptive statistics were employed to summarize data and thematic analysis for analyzing open-ended responses. Subsequently, translation and cultural adaption were conducted by clinicians and patients who are native speakers.
Co-created patient education materials reached the target 6 grade reading level according to 2/6 (33%) algorithms (range: grade 5.9-9.7). The online survey received 164 hits in 2 months and 63/159 (40%) of eligible patients completed the evaluation. Patients ranged in age from 18 to 66 years (median 36, mean 39 ± 11) and 52/63 (83%), had adequate health literacy. Patients scored understandability at 94.2% and actionability at 90.5%. The patient education materials were culturally adapted and translated into 20 languages (available in Additional file 1).
Partnering with patients enabled us to create patient education materials that met patient- identified needs as evidenced by high end-user acceptability, understandability and actionability. The web-based evaluation was effective for reaching dispersed rare disease patients. Combining dissemination via traditional healthcare professional platforms as well as patient-centric sites can facilitate broad uptake of culturally adapted translations. This process may serve as a roadmap for creating patient education materials for other rare diseases.
罕见病患者面临健康差异，往往难以找到有关自身病情的准确信息。我们旨在利用现有最佳证据和社区伙伴关系，制作先天性低促性腺激素性性腺功能减退（CHH）和嗅觉生殖器（卡尔曼）综合征（即CHH和嗅觉缺陷）的患者教育材料，并评估最终用户的可接受性。专家临床医生、研究人员和患者通过多步骤过程共同创建了这些材料。使用六种经过验证的算法评估最终产品的阅读水平。通过基于网络的数据收集，使用患者教育材料评估工具来衡量可理解性和可操作性。采用描述性统计来汇总数据，并进行主题分析以分析开放式回答。随后，由以母语为英语的临床医生和患者进行翻译和文化适应。
根据2/6（33%）的算法，共同创建的患者教育材料达到了目标六年级阅读水平（范围：5.9 - 9.7年级）。在线调查在两个月内收到164次点击，63/159（40%）符合条件的患者完成了评估。患者年龄在18至66岁之间（中位数36岁，平均39±11岁），52/63（83%）具备足够的健康素养。患者对可理解性的评分是94.2%，对可操作性的评分是90.5%。患者教育材料进行了文化适应，并翻译成20种语言（可在补充文件1中获取）。
与患者合作使我们能够创建满足患者确定需求的患者教育材料，高端用户的高接受度、可理解性和可操作性证明了这一点。基于网络的评估对于接触分散的罕见病患者是有效的。通过传统医疗专业平台以及以患者为中心的网站进行传播相结合，可以促进文化适应翻译的广泛采用。这个过程可以作为为其他罕见病创建患者教育材料路线图。
