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
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