Glucocorticoid corticosteroids for Duchenne muscular dystrophy

Abstract

There is evidence from randomised controlled studies that glucocorticoid corticosteroid therapy in Duchenne muscular dystrophy improves muscle strength and function in the short-term (six months to two years). The most effective prednisolone regime appears to be 0.75 mg/kg/day, given daily. In the short term, adverse effects were significantly more common but not clinically severe. Long-term benefits and hazards of glucocorticoid treatment cannot be evaluated from the currently published randomised studies. Non-randomised studies support the conclusions of functional benefits but also identify clinically significant adverse effects of long-term treatment. These benefits and adverse effects have implications for future research studies and clinical practice.

The l-arginine/NO pathway and homoarginine are altered in Duchenne muscular dystrophy and improved by glucocorticoids

“…The effects of steroids on the progression of DMD have been investigated by many groups. Administered glucocorticoids seem to improve muscle strength and to delay the development of cardiac and respiratory complications in DMD (Manzur et al 2008;Annexstad et al 2014;Leung et al 2011). DMD patients on steroid medication were found to excrete less nitrate than DMD patients who have not been medicated with steroids.…”

“…Stage 0 describes a patient with DMD without any clinical symptoms, while a patient at stage 11 is bedfast and not even able to sit (Thompson and Vignos 1959;Mortier 1994). DMD cannot be cured; however, a therapy with glucocorticoids can decelerate the progression of muscle weakness and even enhance muscle strength (Manzur et al 2008;Annexstad et al 2014). Also, administration of creatine to DMD patients has been reported to improve muscle strength in short and medium term in muscular dystrophies (Kley et al 2013;Banerjee et al 2010;Tarnopolsky et al 2004).…”

Parental attitudes toward newborn screening for Duchenne/Becker muscular dystrophy and spinal muscular atrophy

“…Side effects of early steroid treatment include excessive weight gain and behavioral abnormalities (Merlini, 2003;Manzur, 2008). It is the current recommendation that glucocorticoids should be initiated once the gain in motor milestones has plateaued, but before motor function begins to decline (Bushby, 2010).…”

Low bone density and bone metabolism alterations in Duchenne muscular dystrophy: response to calcium and vitamin D treatment

“…Clinically, they improve muscle strength and function and prolong autonomous walking, slowing down the progression of the disease. However, according to a recent Cochrane review, there are no randomized studies on large samples to evaluate the long-term side effects of GCs, and only studies on very few patients have been published [2].…”