Delandistrogene moxeparvovec is a gene transfer therapy approved in the United States, United Arab Emirates, and Qatar for the treatment of ambulatory patients aged four through five years with a confirmed Duchenne muscular dystrophy (DMD)-causing mutation in the gene. This therapy was developed to address the underlying cause of DMD through targeted skeletal, respiratory, and cardiac muscle expression of delandistrogene moxeparvovec micro-dystrophin, an engineered, functional dystrophin protein. Drawing on clinical trial experience from Study 101 (NCT03375164), Study 102 (NCT03769116), and ENDEAVOR (Study 103; NCT04626674), we outline practical considerations for delandistrogene moxeparvovec treatment. Before infusion, the following are recommended: (1) screen for anti-adeno-associated virus rhesus isolate serotype 74 total binding antibody titers <1:400; (2) assess liver function, platelet count, and troponin-I; (3) ensure patients are up to date with vaccinations and avoid vaccine coadministration with infusion; (4) administer additional corticosteroids starting one day preinfusion (for patients already on corticosteroids); and (5) postpone dosing patients with any infection or acute liver disease until event resolution. Postinfusion, the following are recommended: (1) monitor liver function weekly (three months postinfusion) and, if indicated, continue until results are unremarkable; (2) monitor troponin-I levels weekly (first month postinfusion, continuing if indicated); (3) obtain platelet counts weekly (two weeks postinfusion), continuing if indicated; and (4) maintain the corticosteroid regimen for at least 60 days postinfusion, unless earlier tapering is indicated. Although the clinical safety profile of delandistrogene moxeparvovec has been consistent, monitorable, and manageable, these practical considerations may mitigate potential risks in a real-world clinical practice setting.

中文翻译：

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Delandistrogene Moxeparvovec 基因治疗杜氏肌营养不良症患者的实际考虑

Delandistrogene moxeparvovec 是一种基因转移疗法，已在美国、阿拉伯联合酋长国和卡塔尔获批，用于治疗 4 至 5 岁、确诊患有杜氏肌营养不良症 (DMD) 的基因突变的门诊患者。该疗法旨在通过 delandistrogene moxeparvovec 微肌营养不良蛋白（一种工程化的功能性肌营养不良蛋白）的靶向骨骼、呼吸和心肌表达来解决 DMD 的根本原因。借鉴研究 101 (NCT03375164)、研究 102 (NCT03769116) 和 ENDEAVOR（研究 103；NCT04626674）的临床试验经验，我们概述了 delandistrogene moxeparvovec 治疗的实际考虑因素。输注前建议：（1）筛查抗腺相关病毒恒河猴分离株血清型74总结合抗体滴度<1:400；(2)评估肝功能、血小板计数、肌钙蛋白-I；(3) 确保患者及时接种疫苗，避免疫苗与输注同时给药；(4) 从输注前一天开始给予额外的皮质类固醇（对于已经使用皮质类固醇的患者）；(5) 推迟对患有任何感染或急性肝病的患者给药直至事件解决。输注后，建议采取以下措施： (1) 每周监测肝功能（输注后三个月），如有需要，继续监测，直至结果不显着；(2) 每周监测肌钙蛋白-I 水平（输注后第一个月，如果有需要则继续监测）；(3) 每周（输注后两周）获取血小板计数，如果有指示则继续进行；(4) 输注后维持皮质类固醇治疗方案至少 60 天，除非需要提前逐渐减量。尽管 delandistrogene moxeparvovec 的临床安全性是一致的、可监测和可管理的，但这些实际考虑因素可能会减轻现实临床实践环境中的潜在风险。