MSGID: 1:317/3 62735342   
   PID: hpt/lnx 1.9.0-cur 2019-01-08   
   TID: hpt/lnx 1.9.0-cur 2019-01-08   
    Daily steroids safe and slows progression of duchenne muscular   
   dystrophy, study suggests    
      
     Date:   
         May 4, 2022   
     Source:   
         University of Rochester Medical Center   
     Summary:   
         New research recommends daily steroid doses for children with   
         Duchenne muscular dystrophy, marking a significant change in how   
         the disease is treated.   
      
      
      
   FULL STORY   
   ==========================================================================   
   New research published in JAMA recommends daily steroid doses for   
   children with Duchenne muscular dystrophy (DMD), marking a significant   
   change in how the disease is treated. University of Rochester Medical   
   Center (URMC) neurologist Robert Griggs, M.D., and Michela Guglieri,   
   M.D., with Newcastle University in the U.K., led the study, which was   
   conducted by a global team of researchers dedicated to improving care   
   for this fatal disease.   
      
      
   ==========================================================================   
   "Corticosteroids are likely to remain the main treatment for DMD   
   for the foreseeable future and worldwide so it is critical that we   
   establish a standard of care that is backed by scientific evidence,"   
   said Griggs. "This study shows that health concerns over the daily use of   
   corticosteroids are overstated and that there is a clear benefit in terms   
   of improved motor and pulmonary functions. These findings clearly support   
   the daily regimen over an intermittent one as an initial treatment for   
   boys with DMD."  DMD is a condition found almost exclusively in boys and   
   characterized by muscle weakness, which appear at age 3-4 and progresses   
   rapidly leading to significant disability. The symptoms eventually spread   
   to the heart and muscles responsible for breathing, and the disease is   
   often fatal by the time the boy reaches his late teens. An estimated   
   28,000 people in the U.S. suffer from the disease.   
      
   While corticosteroids prednisone and deflazacort are known to improve   
   muscle strength and function in patients with DMD and have been a   
   frontline treatment for years, there is currently no universally accepted   
   standard for steroid use in DMD. A global survey of physicians who treat   
   DMD found 29 different regimens, with the most common being ten days on   
   and ten days off. This intermittent dosing regimen was put in place in   
   an effort to limit the potential side effects associated with prolonged   
   steroid use in children, such as weight gain, stunted growth, and loss   
   of bone density.   
      
   The Finding the Optimum Regimen of Corticosteroids for DMD (FOR-DMD)   
   study was launched in 2013 to compare daily and intermittent steroid   
   use and establish, from clinical benefit and safety perspective, the   
   most beneficial regimen for DMD patients. Griggs and Kate Bushby, M.D.,   
   with Newcastle University initiated the phase 3 clinical trial conducted   
   through the Muscle Study Group, an international network of muscular   
   dystrophy researchers that Griggs helped create in 1997 to advance   
   clinical research in neuromuscular disorders, including DMD.   
      
   The new study recruited 196 boys with DMD at 32 research sites across   
   North American and Europe and followed them for three years. Participants   
   were assigned to three groups consisting of daily regimens of prednisone   
   or deflazacort, or intermittent prednisone, and followed for three   
   years. The researchers found that the daily regimens of both drugs   
   significantly slowed disease progression as measured by strength testing   
   and muscle function, as compared to the intermittent group. While the   
   daily regimen increased side effects overall, there were minimal serious   
   side effects.   
      
   The lead biostatistician for this study was Michael McDermott, Ph.D.,   
   and other URMC investigators include Kimberly Hart, Rabi Tawil, William   
   B. Martens, Barbara E. Herr, and Mary Brown. Other investigators involved   
   in study include Elaine McColl, Chris Speed, Jennifer Wilkinson and   
   Michelle Eagle with Newcastle University, Janbernd Kirschner with   
   University Hospital Frieburg, Germany, Wendy King with Ohio State   
   University, Tracey Willis with the Robert Jones and Agnes Hunt Orthopaedic   
   Hospital in the U.K. The FOR-DMD study was supported with funding from   
   National Institute of Neurological Diseases and Stroke, the Muscular   
   Dystrophy Association, the Parent Project for Muscular Dystrophy, PTC   
   Therapeutics, Sarepta Therapeutics, and Santhera Pharmaceuticals.   
      
      
   ==========================================================================   
   Story Source: Materials provided by   
   University_of_Rochester_Medical_Center. Original written by Mark   
   Michaud. Note: Content may be edited for style and length.   
      
      
   ==========================================================================   
   Journal Reference:   
      1. Michela Guglieri, Kate Bushby, Michael P. McDermott, Kimberly   
      A. Hart,   
         Rabi Tawil, William B. Martens, Barbara E. Herr, Elaine McColl,   
         Chris Speed, Jennifer Wilkinson, Janbernd Kirschner, Wendy M. King,   
         Michelle Eagle, Mary W. Brown, Tracey Willis, Robert C. Griggs,   
         Volker Straub, Henriette van Ruiten, Anne-Marie Childs, Emma   
         Ciafaloni, Perry B. Shieh, Stefan Spinty, Lorenzo Maggi, Giovanni   
         Baranello, Russell J. Butterfield, I. A. Horrocks, Helen Roper,   
         Zoya Alhaswani, Kevin M. Flanigan, Nancy L.   
      
         Kuntz, Adnan Manzur, Basil T. Darras, Peter B. Kang, Leslie   
         Morrison, Monika Krzesniak-Swinarska, Jean K. Mah, Tiziana   
         E. Mongini, Federica Ricci, Maja von der Hagen, Richard S. Finkel,   
         Kathleen O'Reardon, Matthew Wicklund, Ashutosh Kumar, Craig   
         M. McDonald, Jay J. Han, Nanette Joyce, Erik K. Henricson, Ulrike   
         Schara-Schmidt, Andrea Gangfuss, Ekkehard Wilichowski, Richard   
         J. Barohn, Jeffrey M. Statland, Craig Campbell, Giuseppe Vita,   
         Gian Luca Vita, James F. Howard, Imelda Hughes, Hugh J.   
      
         McMillan, Elena Pegoraro, Luca Bello, W. Bryan Burnette, Mathula   
         Thangarajh, Taeun Chang. Effect of Different Corticosteroid Dosing   
         Regimens on Clinical Outcomes in Boys With Duchenne Muscular   
         Dystrophy.   
      
         JAMA, 2022; 327 (15): 1456 DOI: 10.1001/jama.2022.4315   
   ==========================================================================   
      
   Link to news story:   
   https://www.sciencedaily.com/releases/2022/05/220504135635.htm   
      
   --- up 9 weeks, 2 days, 10 hours, 51 minutes   
    * Origin: -=> Castle Rock BBS <=- Now Husky HPT Powered! (1:317/3)   
   SEEN-BY: 15/0 106/201 114/705 123/120 129/330 331 153/7715 218/700   
   SEEN-BY: 229/110 111 317 400 426 428 470 664 700 292/854 298/25 305/3   
   SEEN-BY: 317/3 320/219 396/45   
   PATH: 317/3 229/426