进行性假肥大性肌营养不良的糖皮质激素治疗循证指南（2025版）

Evidence-based guidelines for the use of glucocorticoids in Duchenne muscular dystrophy (2025)

摘要:

Duchenne型肌营养不良（DMD）是儿童最常见的肌营养不良症，预后差，多数患者在30岁左右死于呼吸衰竭或心力衰竭。糖皮质激素（GC）常用于减缓DMD患者肌肉力量和功能下降，但长期使用GC不可避免会出现一些不良反应，且国内GC的使用仍欠规范。为指导儿科和神经科医师对DMD患者进行规范的GC治疗，该指南综合现有的循证医学证据，采用推荐意见评估、制订及评价（GRADE）分级体系，针对GC治疗DMD的时机、剂量方案、疗效评价、不良反应的监测及处理、疗程及过渡到成人时期的管理等临床问题形成了30条推荐意见，以期为广大医护人员在DMD患者的GC治疗及随访管理方面提供指导与决策依据。

Abstract:

Duchenne Muscular Dystrophy （DMD） is the most common form of muscular dystrophy in children， with a poor prognosis， and most patients die from respiratory or heart failure around the age of 30. Glucocorticoids （GC） are often used to slow down the decline of muscle strength and function in DMD patients. However， the long-term use of GC inevitably leads to some side effects， and the use of GC is still not standardized in China. To guide pediatricians and neurologists in standardizing the use of GC in DMD patients， the guidelines are developed based on existing evidence-based medicine， with the application of Grading of Recommendations Assessment， Development and Evaluation. To address clinical issues such as the timing， dosage regimens， efficacy evaluation， monitoring and management of side effects， course， and transition to adult care of GC treatment in DMD， 30 recommendations are formulated to provide guidance and a decision-making basis for healthcare workers in GC treatment and follow-up management of DMD patients.