Wang Bingying, Zhou Linyuhan, Li Shuangru, Xu Huayan, Guo Yingkun, Hu Qin, Huang Min, Zhou Dan, Cai Xiaotang, Wang Qiu, Sun Xiaomei
Department of Rehabilitation Medicine, China Key Laboratory of Birth Defects and Related Diseases of Women and Children, Ministry of Education, West China Second University Hospital, Sichuan University, Chengdu, Sichuan, China.
Department of Radiology, China Key Laboratory of Birth Defects and Related Diseases of Women and Children, Ministry of Education, West China Second University Hospital, Sichuan University, Chengdu, Sichuan, China.
PLoS One. 2025 Jan 10;20(1):e0316938. doi: 10.1371/journal.pone.0316938. eCollection 2025.
Short stature is a frequent complication of DMD, and its pathomechanisms and influencing factors are specific to this disease and the idiosyncratic treatment for DMD.
To establish the height growth curve of early DMD, and evaluate the potential influencing markers on height growth, provide further evidence for pathological mechanism, height growth management and bone health in DMD.
A retrospective, cross-sectional study of 348 participants with DMD aged 2-12 years was conducted at West China Second Hospital of Sichuan University from January 2023 to October 2023.
The growth curve for 2-12 years old boys with DMD indicates a slower growth rate compared to the average population. At age two, children with DMD have a similar height to their peers, but gradually falls behind afterwards. Short stature was observed in children with DMD before and after GC exposure, and prolonged GC use exacerbated the retardation. BMI (β = -0.47, p = 0.007), BMD (β = -0.005, p = 0.014), β-CTX (β = 0.001, p = 0.002), delayed BA (β = 0.417, p < .001), GC duration (β = -0.006, p = 0.047) were independent influencing factors of height. Relevant bone health markers showed different sequential changing patterns.
The high proportion and progression of short stature are associated with the broad bone health status. Different bone indicators have different sensitivities and specificities and need to be considered together for clinical monitoring of bone health. This study provides evidence for the early monitoring of height development and relevant factors as part of bone health management in DMD, to minimize the occurrence of bone-related complications later in life.
身材矮小是杜氏肌营养不良症（DMD）的常见并发症，其发病机制和影响因素因该疾病及DMD的特殊治疗而异。
建立早期DMD患者的身高生长曲线，评估身高生长的潜在影响标志物，为DMD的病理机制、身高生长管理和骨骼健康提供进一步证据。
2023年1月至2023年10月，在四川大学华西第二医院对348名2至12岁的DMD患者进行了一项回顾性横断面研究。
2至12岁DMD男孩的生长曲线表明，其生长速度低于一般人群。两岁时，DMD患儿的身高与同龄人相似，但随后逐渐落后。在接受糖皮质激素（GC）治疗前后，DMD患儿均出现身材矮小，且GC使用时间延长会加剧生长迟缓。体重指数（β = -0.47, p = 0.007）、骨密度（β = -0.005, p = 0.014）、β-胶原交联羧基末端肽（β = 0.001, p = 0.002）、青春期延迟（β = 0.417, p <.001）、GC使用时间（β = -0.006, p = 0.047）是身高的独立影响因素。相关的骨骼健康标志物呈现出不同的顺序变化模式。
身材矮小的高比例发生率及其进展与广泛的骨骼健康状况相关。不同的骨骼指标具有不同的敏感性和特异性，在临床监测骨骼健康时需要综合考虑。本研究为DMD患者骨骼健康管理中身高发育及相关因素的早期监测提供了证据，以尽量减少后期生活中与骨骼相关并发症的发生。
