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Duchenne 型肌营养不良症长期皮质类固醇治疗的药物选择、剂量和副作用处理:法国儿科神经病学会神经肌肉委员会指南。

Choice of compound, dosage, and management of side effects for long-term corticosteroid treatment in Duchenne muscular dystrophy: Guidelines from the Neuromuscular Commission of the French Society of Pediatric Neurology.

机构信息

Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, Hospices Civils de Lyon, 59 Boulevard Pinel, Bron F-69677, CEDEX, France.

UNIROUEN, Inserm U1245, Department of Neonatology, Pediatric Intensive Care and Neuropediatrics, Normandie University, CHU Rouen F-76000, Rouen, France.

出版信息

Arch Pediatr. 2024 Oct;31(7):410-418. doi: 10.1016/j.arcped.2024.05.003. Epub 2024 Sep 27.

Abstract

The French Society of Pediatric Neurology and the FILNEMUS network created a working group on corticosteroid therapy in children with Duchenne muscular dystrophy in order to analyze the literature review and current French practices. The aim of this work was to produce guidelines regarding treatment initiation, pre-therapeutic interventions, choice between available compounds, and treatment monitoring (dosage, duration, and discontinuation). The treatment side effects and their management are also detailed: osteoporosis, endocrinological anomaly (growth delay, weight gain, pubertal delay), cataract, arterial hypertension, behavioral disorders, management of immunosuppression and vaccines, and management of gastrointestinal and metabolic complications.

摘要

法国儿童神经病学学会和 FILNEMUS 网络成立了一个工作组,研究儿童杜氏肌营养不良症的皮质类固醇治疗,以分析文献综述和法国当前的实践。这项工作的目的是制定关于治疗启动、治疗前干预、现有化合物选择以及治疗监测(剂量、持续时间和停药)的指南。还详细介绍了治疗的副作用及其管理:骨质疏松症、内分泌异常(生长迟缓、体重增加、青春期延迟)、白内障、动脉高血压、行为障碍、免疫抑制和疫苗管理,以及胃肠道和代谢并发症的管理。

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