Church Chris, Patil Sana, Butler Stephanie, Miller Freeman, Salazar-Torres Jose J, Lennon Nancy, Shrader M Wade, Donohoe Maureen, Kalisperis Faithe, Mackenzie W G Stuart, Nichols Louise Reid
Department of Orthopaedics, Nemours Children's Health, Wilmington, DE 19803, USA.
Children (Basel). 2025 Mar 15;12(3):365. doi: 10.3390/children12030365.
: The use of patient-reported outcomes is essential to understand and manage health-related quality of life (HRQOL) in youth with lifelong disabilities. This study evaluated HRQOL in youth with physical disorders and examined its relationship with mobility. We conducted an IRB-approved retrospective study in which we administered the parent-reported Pediatric Outcomes Data Collection Instrument (PODCI) and Gross Motor Function Measure section D (GMFM-D) to ambulatory youth aged 2-18 years with cerebral palsy (CP; Gross Motor Function Classification System II; = 258), arthrogryposis ( = 138), achondroplasia ( = 102), and Morquio syndrome ( = 52) during clinical visits to a gait lab. The PODCI has two validated versions, child and adolescent, that assess perceptions about mobility, happiness, and pain. Differences in HRQOL between diagnostic groups, between age groups, and compared with non-disabled youth were examined using non-parametric tests. The relationship between GMFM-D and PODCI scores was analyzed with Pearson's correlations. Both age cohorts within all diagnosis groups demonstrated higher pain and lower mobility compared with non-disabled youth ( < 0.015). Happiness was lower for both age groups with CP and arthrogryposis, and for the child group with Morquio syndrome compared with non-disabled youth ( < 0.002). In diagnostic groups in both age spans, Global Function was higher ( < 0.0001) for those with achondroplasia compared with other groups. Despite functional differences, there were no significant differences between diagnostic groups in pain scores ( > 0.10). Happiness was lower in the group with CP compared with that with achondroplasia ( = 0.01). GMFM-D was related to PODCI mobility scores for all diagnoses = 0.31 to 0.79, < 0.03) but was not correlated with happiness ( = -0.16 to 0.092; > 0.14); GMFM-D and PODCI pain scores were associated only for the child group with achondroplasia ( = 0.355; < 0.001). Significant limitations in HRQOL are present in youth with physical disabilities. Pain levels were higher than those of non-disabled youth, but pain was not related to lower motor function. Happiness was not related to gross motor function, suggesting the need to examine other factors when mental health concerns are present in youth with disabilities.
使用患者报告的结果对于了解和管理患有终身残疾的青少年的健康相关生活质量（HRQOL）至关重要。本研究评估了患有身体疾病的青少年的HRQOL，并研究了其与运动能力的关系。我们进行了一项经机构审查委员会（IRB）批准的回顾性研究，在步态实验室的临床就诊期间，对2至18岁能够行走的患有脑瘫（CP；粗大运动功能分类系统II；n = 258）、先天性多发性关节挛缩症（n = 138）、软骨发育不全（n = 102）和莫尔基奥综合征（n = 52）的青少年，采用家长报告的儿科结果数据收集工具（PODCI）和粗大运动功能测量D部分（GMFM-D）进行评估。PODCI有两个经过验证的版本，儿童版和青少年版，用于评估对运动能力、幸福感和疼痛的认知。使用非参数检验来检查诊断组之间、年龄组之间以及与非残疾青少年相比的HRQOL差异。GMFM-D与PODCI分数之间的关系采用Pearson相关性分析。与非残疾青少年相比，所有诊断组中的两个年龄队列均表现出更高的疼痛和更低的运动能力（P < 0.015）。与非残疾青少年相比，患有CP和先天性多发性关节挛缩症的两个年龄组以及患有莫尔基奥综合征的儿童组的幸福感较低（P < 0.002）。在两个年龄跨度的诊断组中，与其他组相比，患有软骨发育不全的青少年的整体功能更高（P < 0.0001）。尽管存在功能差异，但诊断组之间的疼痛评分没有显著差异（P > 0.10）。与患有软骨发育不全的组相比，患有CP的组的幸福感较低（P = 0.01）。对于所有诊断，GMFM-D与PODCI运动能力分数相关（r = 0.31至0.79，P < 0.03），但与幸福感无关（r = -0.16至0.092；P > 0.14）；GMFM-D与PODCI疼痛评分仅在患有软骨发育不全的儿童组中相关（r = 0.355；P < 0.001）。身体残疾的青少年存在明显的HRQOL限制。疼痛水平高于非残疾青少年，但疼痛与较低的运动功能无关。幸福感与粗大运动功能无关，这表明在存在心理健康问题的残疾青少年中，需要检查其他因素。
