MCPHS University, Worcester, MA, USA.
Ann Pharmacother. 2020 Aug;54(8):788-794. doi: 10.1177/1060028019900500. Epub 2020 Feb 4.
The objective of this article is to review the pharmacology, pharmacokinetics, efficacy, safety, dosage and administration, and formulary considerations of deflazacort. A search of MEDLINE and EMBASE (1946 to December 31, 2019) was conducted using the terms and (DMD). Results were limited to clinical trials, humans, and English. Additional sources and data were obtained from the references of included articles and prescribing information. All articles published after July 2014 related to pharmacology, pharmacokinetics, efficacy, or safety of the therapy in human subjects were included. Deflazacort 0.9 mg/kg/d is a once-daily oral corticosteroid and is the first drug of its class to be Food and Drug Administration (FDA) approved for DMD. Studies with deflazacort show improved functional outcomes, delayed onset of cardiomyopathy, reduction in scoliosis surgery, and improved survival, but these improvements are supported by relatively weak evidence. This review presents data from studies published after the most recent DMD 2016 treatment guidelines and offers prescribing considerations, including pharmacology, pharmacokinetics, adverse effects, formulary considerations, and areas of uncertainty. Deflazacort presents an additional, FDA-approved corticosteroid option for patients that offers improved quality of life for DMD patients. However, there is weak evidence to support these benefits; a full risk-benefit analysis considering adverse events, efficacy, cost, and previous trials of steroid therapy is necessary when selecting therapy. Further research will help clarify deflazacort's optimal dose, duration of treatment, and impact on quality of life.
本文旨在回顾地夫可特的药理学、药代动力学、疗效、安全性、剂量和给药以及处方考虑因素。通过使用术语和（DMD）对 MEDLINE 和 EMBASE（1946 年至 2019 年 12 月 31 日）进行了搜索。结果仅限于临床试验、人类和英语。还从纳入文章的参考文献和处方信息中获得了其他来源和数据。所有 2014 年 7 月后发表的与人类受试者中该疗法的药理学、药代动力学、疗效或安全性相关的文章均包括在内。地夫可特 0.9 mg/kg/d 是一种每日一次的口服皮质类固醇，是第一个获得美国食品和药物管理局（FDA）批准用于 DMD 的同类药物。地夫可特的研究表明，其可改善功能结局、延迟心肌病的发病、减少脊柱侧凸手术，并提高生存率，但这些改善的证据相对较弱。本综述介绍了自最近的 DMD 2016 治疗指南发布以来发表的研究数据，并提供了处方考虑因素，包括药理学、药代动力学、不良反应、处方考虑因素和不确定领域。地夫可特为 DMD 患者提供了另一种经 FDA 批准的皮质类固醇选择，可提高患者的生活质量。然而，支持这些益处的证据较弱；在选择治疗方法时，需要充分考虑不良事件、疗效、成本和以前的类固醇治疗试验，进行风险-效益分析。进一步的研究将有助于阐明地夫可特的最佳剂量、治疗持续时间以及对生活质量的影响。
