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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Corticosteroids in Duchenne muscular dystrophy: major variations in practice
Robert C Griggs1, Barbara E Herr, Allen Reha
1University of Rochester, Departments of Neurology and Biostatistics, 265 Crittenden Boulevard, CU 420669, Rochester, New York, 14642, USA. robert_griggs@urmc.rochester.edu
Muscle & Nerve
|March 14, 2013
Summary
Physicians show varied corticosteroid use in Duchenne muscular dystrophy (DMD) care, with no single regimen dominating despite established short-term efficacy of prednisone. This highlights uncertainty regarding optimal treatment risks and benefits.
Area of Science:
- Neurology
- Pediatrics
- Pharmacology
Background:
- Established short-term efficacy of prednisone (0.75 mg/kg/day) for Duchenne muscular dystrophy (DMD) was noted in 2004.
- Subsequent standardization efforts revealed significant variability in corticosteroid prescribing for DMD patients.
Purpose of the Study:
- To investigate current corticosteroid prescribing patterns in Duchenne muscular dystrophy (DMD) care.
- To document the diversity of corticosteroid regimens used by physicians treating DMD.
Main Methods:
- Surveyed physicians in the TREAT-NMD network (Europe) and U.S. MDA clinics.
- Documented corticosteroid use in a clinical trial for ataluren, a DMD treatment.
Main Results:
- Significant variation in corticosteroid regimens was observed among 105 TREAT-NMD clinicians.
- Commonly used regimens included daily prednisone (0.75 mg/kg/day), alternate-day dosing, deflazacort (0.9 mg/kg/day), and weekend-only prednisone.
- Similar diversity in corticosteroid use was found in U.S. MDA clinics and the ataluren trial.
Conclusions:
- The wide range of corticosteroid regimens suggests ongoing uncertainty about the optimal balance of risks and benefits for DMD treatment.
- Further research is needed to establish evidence-based guidelines for corticosteroid use in Duchenne muscular dystrophy.
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