Steroid Treatment for Cardiac Function in Duchenne Muscular Dystrophy

Abstract

Cardiology & Cardiac Surgery| May 01 2006 Steroid Treatment for Cardiac Function in Duchenne Muscular Dystrophy AAP Grand Rounds (2006) 15 (5): 58–59. https://doi.org/10.1542/gr.15-5-58-a Views Icon Views Article contents Figures & tables Video Audio Supplementary Data Peer Review Share Icon Share Facebook Twitter LinkedIn MailTo Tools Icon Tools Get Permissions Cite Icon Cite Search Site Citation Steroid Treatment for Cardiac Function in Duchenne Muscular Dystrophy. AAP Grand Rounds May 2006; 15 (5): 58–59. https://doi.org/10.1542/gr.15-5-58-a Download citation file: Ris (Zotero) Reference Manager EasyBib Bookends Mendeley Papers EndNote RefWorks BibTex toolbar search toolbar search search input Search input auto suggest filter your search All PublicationsAll JournalsAAP Grand RoundsPediatricsHospital PediatricsPediatrics In ReviewNeoReviewsAAP NewsAll AAP Sites Search Advanced Search Topics: cardiac function, duchenne's muscular dystrophy, steroid therapy Source: Markham LW, Spicer RL, Khoury PR, et al. Steroid therapy and cardiac function in Duchenne muscular dystrophy. Pediatr Cardiol. 2005;26:768–771. A 7-year experience at 2 institutions, Cincinnati’s Children’s Hospital Medical Center, Ohio, and the University of Iowa, Iowa City, was reviewed to test the hypothesis that corticosteroid treatment for long-term Duchenne muscular dystrophy (DMD)1,2 stabilizes cardiac muscle function. One hundred eleven subjects under age 21 years with DMD were divided into treated (steroid treatment >6 months, n=48) and untreated (steroid treatment <6 months, n=63) groups. Of the 48 treated patients, 29 received prednisone, and 19 received deflazacort (a derivative of prednisolone reported to cause less weight gain).3 Steroid treatment was begun at a mean age of 6.7 years and continued, on average, for 3 years. Treatment dose was not evaluated owing to variability within the study group. Ten subjects had had a prior course of corticosteroids (mean duration 4 years) that had been discontinued. Shortening fraction (SF), derived from M-mode echocardiography, was obtained in all cases and compared between the treated and untreated groups. The mean SF in the treated group was 36% compared with 30% in the untreated group (P<.001). For each year of age, the odds of a depressed SF increased 30% in the untreated group relative to the treated group. There were no demonstrable differences in cardiac function between the prednisone and deflazacort treatment subgroups. The group of patients who had a prior course of steroids maintained a mean SF of 35%, which was significantly higher than that of the untreated group but not demonstrably different from those actively receiving corticosteroids. The authors conclude that the clinical benefits of steroid therapy in DMD extend to cardiac function, that these benefits appear to persist even after discontinuation of therapy, and that the benefits outweigh the risks of side effects from chronic steroid use. Dr. Danford has disclosed no financial relationship relevant to this commentary. This commentary does not contain a discussion of a commercial product/device. This commentary does not contain a discussion of an unapproved/investigative use of a commercial product/device. Pediatricians and cardiologists are taught as general dogma that chronic use of corticosteroids is to be avoided, in part because of the cardiovascular damage that might be done. Contrary to this widespread belief, the above report strongly suggests that in DMD these medications are actually helpful for the heart. The retrospective study design is a weakness that leaves room for other interpretations of the data. We wonder, of course, if the patients who received steroid treatment were the beneficiaries of more meticulous management of their disease apart from the use of steroids. The authors acknowledge the shortcomings of their retrospective study, but point out that the general noncardiac advantages of steroid treatment for DMD are so great that a placebo-controlled trial design, in which treatment is withheld from some subjects, would be unethical. The best evidence we have (and the best evidence we are likely to... You do not currently have access to this content.