地夫可特治疗杜氏肌营养不良有效性及安全性研究进展

Research Progress on the Efficacy and Safety of Deflazacort in the Treatment of Duchenne Muscular Dystrophy

  • 摘要: 地夫可特作为一种糖皮质激素类药物,有利于改善杜氏肌营养不良(Duchenne muscular dystrophy, DMD)患者运动功能和肌力、延迟患者丧失自主活动时间,改善肺功能,降低脊柱侧凸风险,延缓心肌病进展及提高生存率。2017年2月,美国食品药品监督管理局批准地夫可特用于治疗DMD。2024年5月,地夫可特以“临时进口”途径进入北京协和医院定点使用。本文从作用机制、药动学、临床疗效及不良反应等方面对其进行介绍,以期为地夫可特在临床中合理、安全使用提供参考。

     

    Abstract: Deflazacort, as a glucocorticoid medication, is conductive to improving motor function and muscle strength, delaying the loss of ambulation, enhancing pulmonary function, reducing the risk of scoliosis, slowing the progression of cardiomyopathy, and increasing survival rates in patients with Duchenne muscular dystrophy (DMD). In February 2017, the U.S. Food and Drug Administration (FDA) approved deflazacort for the treatment of DMD. In May 2024, deflazacort entered Peking Union Medical College Hospital for designated use through the " temporary import" pathway.This article provides an overview of deflazacort from the perspectives of its mechanism of action, pharmacokinetics, clinical efficacy, and adverse effects, aiming to offer a reference for its rational and safe application in clinical practice.

     

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