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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.

DOI:10.1016/j.arcped.2024.05.003
PMID:39332946
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。

相似文献

1
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.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。
Arch Pediatr. 2024 Oct;31(7):410-418. doi: 10.1016/j.arcped.2024.05.003. Epub 2024 Sep 27.
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Corticosteroids for the treatment of Duchenne muscular dystrophy.用于治疗杜氏肌营养不良症的皮质类固醇
Cochrane Database Syst Rev. 2016 May 5;2016(5):CD003725. doi: 10.1002/14651858.CD003725.pub4.
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Glucocorticoid corticosteroids for Duchenne muscular dystrophy.用于杜氏肌营养不良症的糖皮质激素皮质类固醇
Cochrane Database Syst Rev. 2008 Jan 23(1):CD003725. doi: 10.1002/14651858.CD003725.pub3.
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Glucocorticoid corticosteroids for Duchenne muscular dystrophy.用于杜氏肌营养不良症的糖皮质激素皮质类固醇
Cochrane Database Syst Rev. 2004(2):CD003725. doi: 10.1002/14651858.CD003725.pub2.
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Use of corticosteroids in a population-based cohort of boys with duchenne and becker muscular dystrophy.在以人群为基础的杜氏和贝克型肌营养不良男孩队列中使用皮质类固醇。
J Child Neurol. 2010 Nov;25(11):1319-24. doi: 10.1177/0883073810362762. Epub 2010 Mar 5.
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Interventions to prevent and treat corticosteroid-induced osteoporosis and prevent osteoporotic fractures in Duchenne muscular dystrophy.预防和治疗杜氏肌营养不良症中皮质类固醇诱导的骨质疏松症以及预防骨质疏松性骨折的干预措施。
Cochrane Database Syst Rev. 2017 Jan 24;1(1):CD010899. doi: 10.1002/14651858.CD010899.pub2.
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The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy.加拿大在杜氏肌营养不良症长期使用地夫可特治疗方面的经验。
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Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in the Duchenne Registry.杜氏肌营养不良症登记处中美国男性参与者使用皮质类固醇的变异性和趋势。
BMC Neurol. 2019 May 2;19(1):84. doi: 10.1186/s12883-019-1304-8.
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Management of Adrenal Insufficiency Risk After Long-term Systemic Glucocorticoid Therapy in Duchenne Muscular Dystrophy: Clinical Practice Recommendations.Duchenne 型肌营养不良症患者长期全身糖皮质激素治疗后肾上腺皮质功能不全风险的管理:临床实践建议。
J Neuromuscul Dis. 2019;6(1):31-41. doi: 10.3233/JND-180346.
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Practice parameter: corticosteroid treatment of Duchenne dystrophy [RETIRED]: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society.实践参数:杜氏肌营养不良症的皮质类固醇治疗[已退休]:美国神经病学学会质量标准小组委员会和儿童神经病学协会实践委员会的报告。
Neurology. 2005 Jan 11;64(1):13-20. doi: 10.1212/01.WNL.0000148485.00049.B7.

引用本文的文献

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Improving the therapeutic efficacy of gene therapy for duchenne muscular dystrophy (DMD) by evaluating and managing inflammation.通过评估和控制炎症来提高杜氏肌营养不良症(DMD)基因治疗的疗效。
Front Genet. 2025 Jun 9;16:1569289. doi: 10.3389/fgene.2025.1569289. eCollection 2025.