Ru Liang, Wang Yanan, Yan Mei
Department of Pediatrics, The First Affiliated Hospital of Xinjiang Medical University, Urumqi, Xinjiang, China.
Transl Pediatr. 2021 Nov;10(11):3046-3057. doi: 10.21037/tp-21-461.
Hormonal drug therapy has been widely used in clinical practice for the treatment of progressive muscular dystrophy (PMD). Glucocorticoids, as a common drug in the clinical treatment of PMD, have been reported in several clinical studies.
Chinese and English databases were respectively searched using "randomized controlled trials", "Duchenne-type myotonic dystrophy", "glucocorticoids", Prednisone", "Prednisolone", and "Methylprednisolone", and "Defibrotide" were used as search terms. The meta-analysis was performed using the RevMan 5.3 and Stata 13 software provided by the Cochrane system.
this study included five randomized controlled trials, all of which described the correct randomization method. There were four detailed descriptions of hidden distribution schemes. There were four literatures using blind method. Heterogeneity analysis showed that there was some heterogeneity between the results of the mean prognostic muscle strength, walking time of 9 meters, and 4 flights of stairs climbing between the glucocorticoid-treated group (the experimental group) and the placebo group (the control group). There were no significant differences between the experimental group and the control group in average muscle strength level, walking time of 9 meters and climbing time of 4 flights of stairs (MD =1.77; 95% CI: -0.95 to 4.48; P=0.20>0.05), (MD =-12.27; 95% CI: -35.94 to 11.40; P=0.31>0.01), (MD =-3.09; 95% CI: -11.16 to 4.99; P=0.45>0.05). In addition, glucocorticoid treatment significantly increased creatine kinase level in patients with PMD (MD =-0.28, 95% CI: -0.57 to 0.00; P=0.05). In terms of the incidence of adverse reactions, glucocorticoid treatment significantly increased the prognostic probability of acne, rapid hair growth, and emotional irritability in PMD patients (OR =2.40; 95% CI: 1.09 to 5.27; P=0.03<0.05), (OR =3.05; 95% CI: 1.55 to 5.99; P=0.001<0.05), (OR =4.04; 95% CI: 1.82 to 10.63; P=0.001<0.05). There was no significant difference in the incidence of prognostic depression between the experimental group and the control group (OR =5.11; 95% CI: 0.80 to 32.79; P=0.09>0.05).
The results suggest that glucocorticoids have a significant effect on PMD patients, but to a certain extent they increase the incidence of adverse reactions in patients after treatment. However, due to the lack of complete clinical data in some ongoing studies, our conclusions may not be fully representative.
激素药物疗法已在临床实践中广泛用于治疗进行性肌营养不良（PMD）。糖皮质激素作为PMD临床治疗中的常用药物，已有多项临床研究报道。
分别使用“随机对照试验”“杜氏型肌强直性营养不良”“糖皮质激素”“泼尼松”“泼尼松龙”“甲泼尼龙”以及“去纤苷”作为检索词，对中英文数据库进行检索。使用Cochrane系统提供的RevMan 5.3和Stata 13软件进行荟萃分析。
本研究纳入五项随机对照试验，所有试验均描述了正确的随机化方法。有四项对隐藏分配方案进行了详细描述。有四项文献使用了盲法。异质性分析表明，糖皮质激素治疗组（实验组）和安慰剂组（对照组）之间，平均预后肌肉力量、9米步行时间以及爬4层楼梯时间的结果之间存在一定异质性。实验组和对照组在平均肌肉力量水平、9米步行时间和爬4层楼梯时间方面无显著差异（MD =1.77；95%CI：-0.95至4.48；P=0.20>0.05），（MD =-12.27；95%CI：-35.94至11.40；P=0.31>0.01），（MD =-3.09；95%CI：-11.16至4.99；P=0.45>0.05）。此外，糖皮质激素治疗显著增加了PMD患者的肌酸激酶水平（MD =-0.28，95%CI：-0.57至0.00；P=0.05）。在不良反应发生率方面，糖皮质激素治疗显著增加了PMD患者痤疮、毛发快速生长和情绪易怒的预后概率（OR =2.40；95%CI：从1.09至5.27；P=0.03<0.05），（OR =3.05；95%CI：1.55至5.99；P=0.001<0.05），（OR =4.04；95%CI：1.82至10.63；P=0.001<0.05）。实验组和对照组之间预后抑郁的发生率无显著差异（OR =5.11；CI：0.80至32.79；P=0.09>0.05）。
结果表明，糖皮质激素对PMD患者有显著疗效，但在一定程度上增加了治疗后患者的不良反应发生率。然而，由于一些正在进行的研究缺乏完整的临床数据，我们的结论可能不具有充分的代表性。
