Abstract：
BACKGROUND: Clinical medical management guidelines of Duchenne muscular dystrophy (DMD) emphasize prevention and early identification and treatment.
OBJECTIVE: The objective of our study was to review, synthesize, and grade published evidence of the impact of the timing of clinical interventions in DMD.
METHODS: We searched PubMed, Embase, and the Cochrane Library for records published from inception up until November 19, 2021, reporting evidence of the impact of the timing of clinical interventions in DMD. We assessed the quality of evidence using the Grading of Recommendations, Assessment, Development and Evaluations (GRADE) framework.
RESULTS: We included 12 publications encompassing 1,623 patients with DMD from seven countries (Australia, France, Germany, Italy, Japan, the United Kingdom, and the United States of America). Six (50%) studies reported evidence of an impact of the timing of initiation of glucocorticoids on loss of ambulation, cardiomyopathy, fractures, forced vital capacity, and height and BMI; four (33%) of cardiac medication (i.e., angiotensin-converting enzyme inhibitors, β-blockers, and eplerenone) on left ventricular size and function and survival; one (8%) of lower limb surgery on motor quotient and loss of ambulation; and one (8%) of ataluren on lower extremity and motor function. The overall quality of the body of evidence was low.
CONCLUSIONS: While there is a clinical rationale for anticipatory diagnostic and therapeutic strategies, evidence of the impact of the timing of initiation of treatments in patients with DMD is still emerging. Further research of this topic is warranted to inform treatment guidelines in this indication.
OBJECTIVE: The objective of our study was to review, synthesize, and grade published evidence of the impact of the timing of clinical interventions in DMD.
METHODS: We searched PubMed, Embase, and the Cochrane Library for records published from inception up until November 19, 2021, reporting evidence of the impact of the timing of clinical interventions in DMD. We assessed the quality of evidence using the Grading of Recommendations, Assessment, Development and Evaluations (GRADE) framework.
RESULTS: We included 12 publications encompassing 1,623 patients with DMD from seven countries (Australia, France, Germany, Italy, Japan, the United Kingdom, and the United States of America). Six (50%) studies reported evidence of an impact of the timing of initiation of glucocorticoids on loss of ambulation, cardiomyopathy, fractures, forced vital capacity, and height and BMI; four (33%) of cardiac medication (i.e., angiotensin-converting enzyme inhibitors, β-blockers, and eplerenone) on left ventricular size and function and survival; one (8%) of lower limb surgery on motor quotient and loss of ambulation; and one (8%) of ataluren on lower extremity and motor function. The overall quality of the body of evidence was low.
CONCLUSIONS: While there is a clinical rationale for anticipatory diagnostic and therapeutic strategies, evidence of the impact of the timing of initiation of treatments in patients with DMD is still emerging. Further research of this topic is warranted to inform treatment guidelines in this indication.
摘要：
背景：杜氏肌营养不良症（DMD）的临床医疗管理指南强调预防和早期识别和治疗。
目的：我们研究的目的是回顾，合成，并对已发表的DMD临床干预时机影响的证据进行分级。
方法：我们搜索了PubMed，Embase,和Cochrane图书馆从开始到2021年11月19日发布的记录，报告了DMD临床干预时机影响的证据。我们使用建议分级来评估证据的质量，评估,发展和评价（等级）框架。
结果：我们纳入了12篇出版物，包括来自七个国家的1,623名DMD患者（澳大利亚，法国,德国,意大利,Japan,联合王国,和美利坚合众国)。六项（50％）研究报告了糖皮质激素开始时间对步行丧失的影响的证据，心肌病,骨折,强制肺活量,身高和BMI；四种(33%)心脏药物(即，血管紧张素转换酶抑制剂，β-受体阻滞剂,和依普利酮）对左心室大小，功能和存活率的影响；一项（8％）下肢手术的运动商和步行能力丧失；一项（8％）的ataluren的下肢和运动功能。整体证据质量较低。
结论：虽然有一个临床理由的预期诊断和治疗策略，关于DMD患者开始治疗时机的影响的证据仍在出现.有必要对该主题进行进一步研究，以告知该适应症的治疗指南。
目的：我们研究的目的是回顾，合成，并对已发表的DMD临床干预时机影响的证据进行分级。
方法：我们搜索了PubMed，Embase,和Cochrane图书馆从开始到2021年11月19日发布的记录，报告了DMD临床干预时机影响的证据。我们使用建议分级来评估证据的质量，评估,发展和评价（等级）框架。
结果：我们纳入了12篇出版物，包括来自七个国家的1,623名DMD患者（澳大利亚，法国,德国,意大利,Japan,联合王国,和美利坚合众国)。六项（50％）研究报告了糖皮质激素开始时间对步行丧失的影响的证据，心肌病,骨折,强制肺活量,身高和BMI；四种(33%)心脏药物(即，血管紧张素转换酶抑制剂，β-受体阻滞剂,和依普利酮）对左心室大小，功能和存活率的影响；一项（8％）下肢手术的运动商和步行能力丧失；一项（8％）的ataluren的下肢和运动功能。整体证据质量较低。
结论：虽然有一个临床理由的预期诊断和治疗策略，关于DMD患者开始治疗时机的影响的证据仍在出现.有必要对该主题进行进一步研究，以告知该适应症的治疗指南。
