{"id":21685,"date":"2022-05-27T04:53:00","date_gmt":"2022-05-26T20:53:00","guid":{"rendered":"http:\/\/csccm.org.cn\/?p=21685"},"modified":"2022-05-27T05:32:46","modified_gmt":"2022-05-26T21:32:46","slug":"jama%e5%8f%91%e8%a1%a8%e8%ae%ba%e6%96%87%ef%bc%9a%e4%b8%8d%e5%90%8c%e5%89%82%e9%87%8f%e7%9a%ae%e8%b4%a8%e6%bf%80%e7%b4%a0%e6%96%b9%e6%a1%88%e5%af%b9duchenne%e8%82%8c%e8%90%a5%e5%85%bb%e4%b8%8d","status":"publish","type":"post","link":"https:\/\/csccm.org.cn\/?p=21685","title":{"rendered":"[JAMA\u53d1\u8868\u8bba\u6587]\uff1a\u4e0d\u540c\u5242\u91cf\u76ae\u8d28\u6fc0\u7d20\u65b9\u6848\u5bf9Duchenne\u808c\u8425\u517b\u4e0d\u826f\u7537\u7ae5\u4e34\u5e8a\u9884\u540e\u7684\u5f71\u54cd"},"content":{"rendered":"\n

Original Investigation April 5, 2022<\/p>\n\n\n\n

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial<\/h1>\n\n\n\n

Michela\u00a0Guglieri,\u00a0Kate\u00a0Bushby,\u00a0Michael P.\u00a0McDermott,\u00a0et al<\/h3>\n\n\n\n

JAMA.\u00a0<\/em>2022;327(15):1456-1468. doi:10.1001\/jama.2022.4315<\/h3>\n\n\n\n

Key Points<\/p>\n\n\n\n

Question<\/strong>  What is the difference in clinical outcomes among 3 corticosteroid regimens (0.75 mg\/kg of daily prednisone, 0.90 mg\/kg of daily deflazacort, or 0.75 mg\/kg of intermittent prednisone for 10 days on and then 10 days off) as initial treatment for boys with Duchenne muscular dystrophy?<\/p>\n\n\n\n

Findings<\/strong>  This randomized clinical trial included 196 boys with Duchenne muscular dystrophy; the clinical outcome was a global outcome that incorporated a measure of rising from the floor, forced vital capacity, and global satisfaction with treatment assessed over 3 years. Daily prednisone and daily deflazacort resulted in significantly better outcomes compared with intermittent prednisone; there was no significant difference between the 2 daily regimens.<\/p>\n\n\n\n

Meaning<\/strong>  The findings support the use of a daily corticosteroid regimen over an intermittent prednisone regimen that alternates dosing for 10 days on and 10 days off as initial treatment for boys with Duchenne muscular dystrophy.<\/a>Abstract<\/p>\n\n\n\n

Importance<\/strong>  Corticosteroids improve strength and function in boys with Duchenne muscular dystrophy. However, there is uncertainty regarding the optimum regimen and dosage.<\/p>\n\n\n\n

Objective<\/strong>  To compare efficacy and adverse effects of the 3 most frequently prescribed corticosteroid regimens in boys with Duchenne muscular dystrophy.<\/p>\n\n\n\n

Design, Setting, and Participants<\/strong>  Double-blind, parallel-group randomized clinical trial including 196 boys aged 4 to 7 years with Duchenne muscular dystrophy who had not previously been treated with corticosteroids; enrollment occurred between January 30, 2013, and September 17, 2016, at 32 clinic sites in 5 countries. The boys were assessed for 3 years (last participant visit on October 16, 2019).<\/p>\n\n\n\n

Interventions<\/strong>  Participants were randomized to daily prednisone (0.75 mg\/kg) (n\u2009=\u200965), daily deflazacort (0.90 mg\/kg) (n\u2009=\u200965), or intermittent prednisone (0.75 mg\/kg for 10 days on and then 10 days off) (n\u2009=\u200966).<\/p>\n\n\n\n

Main Outcomes and Measures<\/strong>  The global primary outcome comprised 3 end points: rise from the floor velocity (in rise\/seconds), forced vital capacity (in liters), and participant or parent global satisfaction with treatment measured by the Treatment Satisfaction Questionnaire for Medication (TSQM; score range, 0 to 100), each averaged across all study visits after baseline. Pairwise group comparisons used a Bonferroni-adjusted significance level of .017.<\/p>\n\n\n\n

Results<\/strong>  Among the 196 boys randomized (mean age, 5.8 years [SD, 1.0 years]), 164 (84%) completed the trial. Both daily prednisone and daily deflazacort were more effective than intermittent prednisone for the primary outcome (P<\/em>\u2009<\u2009.001 for daily prednisone vs intermittent prednisone using a global test; P<\/em>\u2009=\u2009.017 for daily deflazacort vs intermittent prednisone using a global test) and the daily regimens did not differ significantly (P<\/em>\u2009=\u2009.38 for daily prednisone vs daily deflazacort using a global test). The between-group differences were principally attributable to rise from the floor velocity (0.06 rise\/s [98.3% CI, 0.03 to 0.08 rise\/s] for daily prednisone vs intermittent prednisone [P<\/em>\u2009=\u2009.003]; 0.06 rise\/s [98.3% CI, 0.03 to 0.09 rise\/s] for daily deflazacort vs intermittent prednisone [P<\/em>\u2009=\u2009.017]; and \u22120.004 rise\/s [98.3% CI, \u22120.03 to 0.02 rise\/s] for daily prednisone vs daily deflazacort [P<\/em>\u2009=\u2009.75]). The pairwise comparisons for forced vital capacity and TSQM global satisfaction subscale score were not statistically significant. The most common adverse events were abnormal behavior (22 [34%] in the daily prednisone group, 25 [38%] in the daily deflazacort group, and 24 [36%] in the intermittent prednisone group), upper respiratory tract infection (24 [37%], 19 [29%], and 24 [36%], respectively), and vomiting (19 [29%], 17 [26%], and 15 [23%]).<\/p>\n\n\n\n

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Conclusions and Relevance<\/strong>  Among patients with Duchenne muscular dystrophy, treatment with daily prednisone or daily deflazacort, compared with intermittent prednisone alternating 10 days on and 10 days off, resulted in significant improvement over 3 years in a composite outcome comprising measures of motor function, pulmonary function, and satisfaction with treatment; there was no significant difference between the 2 daily corticosteroid regimens. The findings support the use of a daily corticosteroid regimen over the intermittent prednisone regimen tested in this study as initial treatment for boys with Duchenne muscular dystrophy.<\/p>\n\n\n\n

Trial Registration<\/strong>  ClinicalTrials.gov Identifier: NCT01603407<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

Original Investigation April 5, 2022 Effect of Dif […]<\/p>\n","protected":false},"author":3,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[32,23],"tags":[],"_links":{"self":[{"href":"https:\/\/csccm.org.cn\/index.php?rest_route=\/wp\/v2\/posts\/21685"}],"collection":[{"href":"https:\/\/csccm.org.cn\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/csccm.org.cn\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/csccm.org.cn\/index.php?rest_route=\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/csccm.org.cn\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=21685"}],"version-history":[{"count":1,"href":"https:\/\/csccm.org.cn\/index.php?rest_route=\/wp\/v2\/posts\/21685\/revisions"}],"predecessor-version":[{"id":21686,"href":"https:\/\/csccm.org.cn\/index.php?rest_route=\/wp\/v2\/posts\/21685\/revisions\/21686"}],"wp:attachment":[{"href":"https:\/\/csccm.org.cn\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=21685"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/csccm.org.cn\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=21685"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/csccm.org.cn\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=21685"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}