Aguirre Alex S, Astudillo Moncayo Olga M, Mosquera Johanna, Muyolema Arce Veronica E, Gallegos Camila, Ortiz Juan Fernando, Andrade Andres F, Oña Sebastian, Buj Maja J
School of Medicine, Universidad San Francisco de Quito, Quito, ECU.
General Medicine, University of Cuenca, Cuenca, ECU.
Cureus. 2023 Jun 3;15(6):e39903. doi: 10.7759/cureus.39903. eCollection 2023 Jun.
Facioscapulohumeral muscular dystrophy (FSHD) is the third most common type of muscular dystrophy. This disease presents as a slowly progressive asymmetric muscle weakness that involves the facial, scapular, and upper arm muscles mainly. Currently, there is no established consensus on this disease treatment in terms of medications. We assessed the response to the treatment of the drugs utilized in clinical trials by performing a systematic literature review in English using the preferred reporting items for systematic reviews (PRISMA) and meta-analyses. We only used human clinical trials in patients diagnosed with FSHD that received consistent pharmacological treatment. We included 11 clinical trials that fulfilled our criteria. We concluded that albuterol had statistically significant results in three out of four clinical trials, with improved elbow flexors muscle strength. Vitamin C, vitamin E, zinc gluconate, and selenomethionine showed significant improvement in the maximal voluntary contraction and endurance limit time of quadriceps muscle. At the same time, diltiazem and MYO-029 demonstrate no improvement in function, strength, or muscle mass. Losmapimod, currently in phase I of the ReDUX4 trial, showed promising results. Peradventure, more clinical trials are still needed to address this subject. Nevertheless, this review provides a clear and concise update on the treatment for this disease.
面肩肱型肌营养不良症（FSHD）是第三常见的肌营养不良类型。这种疾病表现为缓慢进展的不对称肌无力，主要累及面部、肩胛和上臂肌肉。目前，在药物治疗方面，对于这种疾病尚无既定的共识。我们使用系统评价和Meta分析的首选报告项目（PRISMA），通过进行英文系统文献综述，评估了临床试验中使用的药物的治疗反应。我们仅纳入了被诊断为FSHD且接受一致药物治疗的患者的人体临床试验。我们纳入了11项符合我们标准的临床试验。我们得出结论，沙丁胺醇在四项临床试验中的三项中具有统计学显著结果，肱二头肌肌力有所改善。维生素C、维生素E、葡萄糖酸锌和硒代蛋氨酸在股四头肌的最大自主收缩和耐力极限时间方面显示出显著改善。同时，地尔硫䓬和MYO - 029在功能、力量或肌肉质量方面未显示出改善。目前处于ReDUX4试验I期的洛索匹莫显示出有前景的结果。或许仍需要更多的临床试验来研究这个问题。尽管如此，本综述为该疾病的治疗提供了清晰简洁的最新信息。
