在接受皮质类固醇治疗的杜申肌肉发育不良症男孩中,增长和运动功能的联合建模
Georgia Stimpson1, Deborah Ridout2, Amy Wolfe1,3
1Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
European journal of neurology
|November 7, 2025
概括
杜申肌力发育不良 (DMD) 的男孩接受皮质类固醇治疗会影响生长和运动功能. 更轻微的体重增加或更高的身高衰减与更好的运动功能轨迹相关,为治疗的好处和缺点提供了见解.
科学领域:
- 儿科神经学 儿科神经学
- 杜恩肌肉衰竭研究研究
- 增长与发展研究 研究 研究 研究
背景情况:
- 杜申肌力发育不良症 (DMD) 的皮质类固醇 (CS) 治疗改善了运动功能,并延迟了行走能力的丧失,但导致身高迟缓和体重增加.
- 由于复杂的建模挑战,CS治疗的DMD患者的生长指标和运动功能的相互作用仍然未得到充分探索.
研究的目的:
- 在接受皮质类固醇治疗的DMD男孩中调查生长参数 (身高,体重) 和运动功能结果之间的复杂关系.
- 分析不同的皮质类固醇疗法 (例如,每日与间歇性,普雷迪尼索隆与德夫拉扎科特) 如何影响这些生长和运动功能动态.
主要方法:
- 利用新开发的运动功能的百分点进行北星门诊评估 (NSAA),walrFF和10米步行/跑步 (10MWR).
- 采用多变量回归模型来评估生长 (身高/体重Z分数) 和运动功能,控制CS治疗类型和时间表.
- 检查了对生长和运动功能结果的单独和综合影响,包括它们的相互关系.
主要成果:
- 在1643次评估中分析了559名患有DMD的男孩 (5-16岁) 的数据.
- 每日CS治疗,特别是每日除,与运动功能轨迹的改善有关 (例如,NSAA百分位数的积极变化).
- 在运动功能的年度变化 (NSAA,10MWR) 和增长 (身高,体重) Z-score之间观察到轻微的负相关性 (-0.25到-0.36),表明较少的体重增加或更高的身高缩与更好的运动功能的进展相关.
结论:
- 该研究阐明了在DMD中皮质类固醇治疗,生长和运动功能之间的复杂相互作用.
- 研究结果为临床讨论提供了有价值的见解,这些见解涉及皮质类固醇治疗对DMD治疗的益处和不良影响的平衡.
更多相关视频
06:52Behavioral and Locomotor Measurements Using an Open Field Activity Monitoring System for Skeletal Muscle Diseases
Published on: September 29, 2014
54.4K
14:10Isometric and Eccentric Force Generation Assessment of Skeletal Muscles Isolated from Murine Models of Muscular Dystrophies
Published on: January 31, 2013
33.8K
相关概念视频
01:21Satellite Stem Cells and Muscular Dystrophy
2.3K
Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
2.3K
01:27Growth of Cartilage and Bone Tissue
4.0K
Chondrocytes form a temporary cartilaginous model by dividing and secreting a thick gel-like extracellular matrix. Once the chondrocytes undergo programmed cell death, osteoblasts enter the site of the cartilaginous model. The process of replacing the temporary cartilaginous model with bone in an ordered manner is called endochondral ossification. In endochondral ossification, not all of the cartilage is replaced by bone tissue. Some cartilage that performs a protective and supportive function...
4.0K
01:28Disorders of the Skeletal Muscle
1.7K
The clinical conditions affecting the skeletal muscle tissue are broadly categorized as musculoskeletal and neuromuscular disorders.
Musculoskeletal disorders
Musculoskeletal disorders involve injuries and conditions affecting the skeletal muscles and associated connective tissues. These disorders can arise from acute biomechanical stresses or chronic overuse and can occur across different age groups. Common injuries include sprains, fractures, and muscular strains, often resulting from...
Musculoskeletal disorders
Musculoskeletal disorders involve injuries and conditions affecting the skeletal muscles and associated connective tissues. These disorders can arise from acute biomechanical stresses or chronic overuse and can occur across different age groups. Common injuries include sprains, fractures, and muscular strains, often resulting from...
1.7K
02:33Exponential Equations for Modeling Growth
196
Exponential models are essential for describing rapid, multiplicative changes in natural systems, such as population growth. When a population doubles at regular intervals, the process can be modeled using a suitable base. For instance, a bacterial culture that doubles every three hours follows the model n(t)=n0⋅2t/3, where n(t) is the population at the time t.A more general model uses the natural base e, especially for continuous growth. This takes the form n(t)=n0⋅ert, where r is...
196
01:09Changes in the Appendicular Skeleton with Age
3.3K
The upper and lower limb initially develops as a small bulge called a limb bud, which appears on the lateral side of the early embryo. The upper limb bud appears near the end of the fourth week of development, with the lower limb bud appearing shortly after.
Initially, the limb buds consist of a core of mesenchyme covered by a layer of ectoderm. The ectoderm at the end of the limb bud thickens to form a narrow crest called the apical ectodermal ridge. This ridge stimulates the underlying...
Initially, the limb buds consist of a core of mesenchyme covered by a layer of ectoderm. The ectoderm at the end of the limb bud thickens to form a narrow crest called the apical ectodermal ridge. This ridge stimulates the underlying...
3.3K
01:07Development of the Limb Synovial Joints
2.2K
Joints form during embryonic development in conjunction with the formation and growth of the associated bones. The embryonic tissue that gives rise to all bones, cartilage, and connective tissues of the body is called mesenchyme.
The mesenchymal stem cells differentiate into chondrocytes that form the hyaline cartilage, and later the cartilaginous model of the bone. This model further transforms into a bone. This process is known as endochondral ossification.
During development, the limbs...
The mesenchymal stem cells differentiate into chondrocytes that form the hyaline cartilage, and later the cartilaginous model of the bone. This model further transforms into a bone. This process is known as endochondral ossification.
During development, the limbs...
2.2K