Schwartz Carolyn E, Biletch Elijah, Stuart Richard B B, Rapkin Bruce D
DeltaQuest Foundation, Inc., 31 Mitchell Road, Concord, MA, 01742, USA.
Department of Medicine and Orthopaedic Surgery, Tufts University Medical School, Boston, MA, USA.
J Patient Rep Outcomes. 2022 Sep 10;6(1):96. doi: 10.1186/s41687-022-00501-7.
The inevitable and progressive loss of independence in Duchenne Muscular Dystrophy (DMD) patients may have an impact on their siblings' life aspirations. The present cross-sectional case-control study investigated how aspirations differed between brothers and sisters of people with DMD and a stratified comparison group of nationally representative children/adults.
A web-based survey was administered October through December 2020. Aspirations were measured using qualitative (open-ended) and quantitative (closed-ended) questions. Qualitative prompts asked participants about wishes, goals, and how they define quality of life (QOL) and were coded by six trained raters. Quantitative questions included 29 closed-ended goal-delineation items from the QOL Appraisal Profile. These data were analyzed using multivariate models adjusting for propensity scores (demographic differences) and testing for the effects of role (sibling or comparison), age, and role-by-age interactions.
The study sample of DMD sibling (n = 349) and comparison (n = 619) participants provided open-ended data on 968 wishes statements, 390 QOL-definition statements, and 328 goals statements. Inter-rater reliability (kappa = 0.77) reflected good agreement between raters. Results of both open-ended and closed-ended data, and of both unadjusted and adjusted analyses suggested that DMD siblings, with age, were more focused on DMD-related, family/community, intimacy, and work concerns than their peers. They were less focused on improving mood, independence, pragmatics, or subtle fine-tuning of problem-solving in life. In contrast, the comparison group was more focused on goals related to growth, purpose, and reflection. Some group differences were amplified amongst older siblings.
This is the first study to evaluate DMD siblings' aspirations in comparison to their peers. While there were many similarities between groups, the differences in aspirations between DMD siblings and their peers encompassed not just DMD, family/community, and intimacy, but also more work concerns. Directions for future quantitative and qualitative research are discussed.
杜氏肌营养不良症（DMD）患者不可避免且逐渐丧失独立能力，这可能会影响其兄弟姐妹的生活志向。本横断面病例对照研究调查了DMD患者的兄弟姐妹与具有全国代表性的儿童/成人分层比较组在志向上的差异。
2020年10月至12月进行了一项基于网络的调查。使用定性（开放式）和定量（封闭式）问题来衡量志向。定性提示询问参与者的愿望、目标以及他们如何定义生活质量（QOL），并由六名经过培训的评分者进行编码。定量问题包括来自生活质量评估概况的29个封闭式目标描述项目。使用多变量模型对这些数据进行分析，该模型调整了倾向得分（人口统计学差异），并测试了角色（兄弟姐妹或比较组）、年龄以及角色与年龄的相互作用的影响。
DMD患者兄弟姐妹（n = 349）和比较组（n = 619）参与者的研究样本提供了关于968条愿望陈述、390条生活质量定义陈述和328条目标陈述的开放式数据。评分者间信度（kappa = 0.77）反映了评分者之间的良好一致性。开放式和封闭式数据以及未调整和调整分析的结果均表明，随着年龄的增长，DMD患者的兄弟姐妹比同龄人更关注与DMD相关、家庭/社区、亲密关系和工作方面的问题。他们不太关注改善情绪、独立性、语用能力或对生活中解决问题的细微调整。相比之下，比较组更关注与成长、目标和反思相关的目标。一些组间差异在年龄较大的兄弟姐妹中更为明显。
这是第一项将DMD患者的兄弟姐妹的志向与其同龄人进行比较评估的研究。虽然两组之间有许多相似之处，但DMD患者的兄弟姐妹与其同龄人在志向上的差异不仅包括DMD、家庭/社区和亲密关系，还包括更多工作方面的问题。讨论了未来定量和定性研究的方向。
