The key references for management of patients with DMD.

Advances in medical management of DMD have made a significant difference to the natural history of the condition. The key interventions relate to the use of corticosteroids to improve muscle strength and function, surgical management of scoliosis, and surveillance for and timely management of respiratory and cardiac complications. The predictable nature of the complications of DMD lends itself to the implementation of a planned programme of surveillance and management which makes a real difference to survival and quality of life. But this needs to be taken on board by a committed multidisciplinary team- lack of awareness of the recent advances in this field can be a significant threat to the health and wellbeing of someone with DMD.

These three references review the evidence for the use of glucocorticosteroids in DMD and conclude that their use is the gold standard of treatment in DMD, provided they are administered with due care to the possible side effects:

• AY Manzur, T Kuntzer, M Pike, A Swan. Glucocorticoid corticosteroids for Duchenne muscular dystrophy The Cochrane Database of Systematic Reviews 2005 Issue 1
• Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report on the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids. 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord. 2004 Sep;14(8-9):526-34.
• Moxley RT 3rd, Ashwal S, Pandya S, Connolly A, Florence J, Mathews K, Baumbach L, McDonald C, Sussman M, Wade C; Quality Standards Subcommittee of the American Academy of Neurology; Practice Committee of the Child Neurology Society Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology. 2005 Jan 11;64(1):13-20.

There are few good evidence based studies on the use of exercise and physiotherapy in DMD. The ones that do exist are reviewed in:

• Eagle M. Report on the Muscular Dystrophy Campaign workshop: Exercise in neuromuscular diseases Newcastle. Neuromuscular Disorders, January 2002 Volume 12, Issue 10, 975-983

Spinal surgery in specialised centres is a safe and effective treatment option for the scoliosis that occurs in children with DMD as they get older:

• Cervellati S, Bettini N, Moscato M, Gusella A, Dema E, Maresi R. Surgical treatment of spinal deformities in Duchenne muscular dystrophy: a long term follow-up study. Eur Spine J. 1004 (Aug, 13(5):441-8. Epub 2004 Apr 24

Proactive management of problems with the breathing muscles, especially the prevention and treatment of chest infections and the timely provision of home nocturnal ventilation has made a huge difference to life expectancy in DMD:

• Finder JD, Birnkrant D, Carl J, Farber HJ, Gozal D, Iannaccone ST, Kovesi T, Kravitz RM, Panitch H, Schramm C, Schroth M, Sharma G, Sievers L, Silvestri JM, Sterni L; American Thoracic Society. Respiratory care of the patient with Duchenne muscular dystrophy: ATS consensus statement. Am J Respir Crit Care Med. 2004 Aug 15;170(4):456-65.
• Eagle M, Baudouin SV, Chandler C, Giddings DR, Bullock R and Bushby K. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscular Disorders, Volume 12, Issue 10, 926-929
• Simonds AK, Muntoni F, Heather S, Fielding S. Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy. Thorax 1998; 53:949-953

Heart muscle is involved to some extent in people with DMD and also in some carriers. Regular surveillance and early treatment is of benefit in preventing problems:

• Bushby KMD, Muntoni F, Bourke JP. The management of cardiac complications in muscular dystrophy and myotonic dystrophy. Proceedings of 107th ENMC Workshop. Neuromuscular Disorders 2003, 13:166-172.
  
  10. Duboc et al. Effect of perindropril on the onset and progression of LV dysfunction in DMD. Journal of the American Cardiology Association 2005, 45: 855-7

Low bone density is increasingly recognised as a problem in people with DMD and this can be made worse with steroids. There have been two workshops recently to discuss this and generate some recommendations:

• Quinlivan R, Roper H, Davie M, Shaw NJ, McDonagh J and Bushby K. Report of a Muscular Dystrophy Campaign funded workshop Birmingham, UK, January 16th 2004. Osteoporosis in Duchenne muscular dystrophy; its prevalence, treatment and prevention. Neuromuscular Disorders 2005, 15: 72-79
• Biggar, L.K. Bachrach, R.C. Henderson, H. Kalkwarf, H. Plotkin and B.L. Wong. Bone health in Duchenne muscular dystrophy: a workshop report from the meeting in Cincinnati, Ohio, July 8, Neuromuscular Disorders 2005, 15: 1 80-85
• Laura Bachrach K. Taking steps towards reducing osteoporosis in Duchenne muscular dystrophy Neuomuscular Disorders 2005, 15: 86-87

When someone with DMD has an anaesthetic it is very important that the anaesthetist is aware of this and takes precautions. This is described in:

• Klingler W, Lehmann-Horn F and Jurkat-Rott K. Complications of anaesthesia in neuromuscular disorders Neuromuscular Disorders. 2005 15; 3: 195-206

Advances in medical management have made a huge impact on life expectancy in DMD, which needs to be taken into account in planning for educational and social opportunities:

• Rahbeck J, Werge B, Madsen A, Marquardt J, Steffensen BF, Jeppesen J. Adult life with Duchenne muscular dystrophy: Observations among an emerging and unforeseen patient population. Pediatric Rehabilitation. 2005; 8 (1): 1-12