Steroids in Duchenne: What You Need to Know

A Guide for HCPs, Caregivers, and Individuals Living With Duchenne

Steroids and Their Effects

Steroids (also called glucocorticoids or corticosteroids) and important medications with proven benefits1-9

Steroids Ambulation

Ambulation

Preserve ability to walk longer

Steroids Breathing

Breathing

Preserve lung function longer and delay need for nocturnal ventilation

Steroids Heart

Heart

Preserve heart function longer

Steroids Spine

Spine

Preserve heart function longer

Steroids Motor Function

Motor Function

Improve muscle strength

Steroids Survival

Survival

Improve health outcomes and allow boys to live longer


Two steroids are used in Duchenne10

  • Prednisone (also called prednisolone)
  • Deflazacort*

Steroids can undoubtedly benefit many boys with Duchenne. This benefit needs to be balanced with proactive management of possible side effects11

*Deflazacort is the only FDA-approved steroid for use in Duchenne.

Note: Sudden discontinuation or missed doses of steroids (>24 hours) may lead to serious consequences resulting from acute adrenal crisis. During surgical or medical stress situations, the administration of supplemental (stress) doses may be recommended. Symptoms of acute adrenal crisis can include: abdominal pain, hypovolemic shock, confusion or coma, dehydration, dizziness or light-headedness, fatigue, flank pain, headache, high fever, and appetite loss. Consult HCP for appropriate withdrawal protocols.13

What to Consider When12

Steroids Early Ambulatory

Early ambulatory

Time to get informed. Find out about steroid options. Recent evidence suggests initiation of steroids before motor skills start to level out or “plateau.” (Typically steroid therapy is initiated between ages 3 and 5.) Ensure vaccinations are complete. Be proactive and anticipate any risk factors for the steroid side effects. For example, starting nutritional plans for weight management may be considered here.

Steroids Late Ambulatory

Late ambulatory

Time to tailor steroid therapy. Individualize the type of steroid and dosing for each child. Twice yearly assessments to monitor muscle strength/function and concerns about side effects are important. Manage side effects by adjusting steroid dose and/or adding medications to alleviate.

Steroids Early Non Ambulatory

Early non-ambulatory

Maintain steroid therapy for as long as possible. Steroid therapy is an important part of this phase whether started previously and continued or initiated at this phase. Continue to manage side effects by adjusting steroid dose and/or adding medications to alleviate.

Steroids Late Non Ambulatory

Late non-ambulatory

Review and discuss with care team. Continue steroid use but reduce dose as needed to manage side effects.

CureDuchenne would like to thank Emma Ciafaloni, MD, University of Rochester, for content development.

Steroid Decision Path—Starting and Monitoring Steroid Therapy12

Managing Side Effects of Steroids

Steroids Weight Gain

Weight gain*

  • Nutrition goals: avoid excessive sodium, aim for lower carbohydrate intake, reduce saturated fats, increase water intake
  • Healthy lifestyle/exercise
  • Metformin
  • Dietitian consultation
  • Endocrinologist consultation
Steroids Growth Delay

Growth delay / Pubertal delay

  • Endocrinologist consultation
  • Human growth hormone (hGH)
  • Testosterone
Steroids Cataracts

Cataracts**

  • Ophthalmologist consulation
Steroids Bone Demineralization

Bone demineralization

  • Bisphosphonates
  • Vitamin D and calcium
  • Weight-bearing activities
Steroids Behavioral Changes

Behavioral changes

  • Ophthalmologist consulation
  • Change timing of administration
  • Psychologist/therapist consultation • Behavioral therapy
Steroids Cushingoid

Cushingoid appearance

  • Diet/reduction in sugar and salt

References

  1. Biggar WD, Harris VA, Eliasoph L, Alman B. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade. Neuromuscul Disord. 2006;16:249–255.
  2. Lebel DE, Corston JA, McAdam LC, et al. Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term follow-up. J Bone Joint Surg Am. 2013;95:1057–1061.
  3. Schram G, Fournier A, Leduc H, et al. All-cause mortality and cardiovascular outcomes with prophylactic steroid therapy in Duchenne muscular dystrophy. J Am Coll Cardiol. 2013;61:948–954.
  4. Narayanan S, Jin F, Souza M, et al. Disease burden and treatment landscape in Duchenne muscular dystrophy (DMD) in the United States. Poster presented at: ISPOR 22nd Annual Conference; May 23, 2017; Boston, MA.
  5. Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016;87(20):2123–2131.
  6. Gloss D, Moxley RT 3rd, Ashwal S, Oskoui M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. 2016;86:465–472.
  7. McDonald M, Henricson EK, Abresch RT, et al. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet. 2018;391:451–461.
  1. Barber BJ, Andrews JG, Lu Z, et al. Oral corticosteroids and onset of cardiomyopathy in Duchenne muscular dystrophy. J Pediatr. 2013;163:1080–1084.
  2. Lebel DE, Corston JA, McAdam, et al. Glucocorticoid treatment for the prevention of scoliosis in children with Duchenne muscular dystrophy: long-term followup. J Bone Joint Surg Am 2013;95:1057-1061.
  3. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17:251–267.
  4. Bello L, Gordish-Dressman H, Morgenroth LP, et al. Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. Neurology. 2015;85:1048–1055.
  5. The Diagnosis and Management of Duchenne Muscular Dystrophy. A guide for families. Available at: http:// www.treat-nmd.eu/diagnosis-andmanagement-of-DMD/. Assessed November 14, 2018.
  6. Kinnett K, Noritz G. The PJ Nicholoff Steroid Protocol for Duchenne and Becker Muscular Dystrophy and Adrenal Suppression. PLoS Curr. 2017; June 27: 9.
  7. Bonifati MD, Ruzza G, Bonometto P, et al. A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. Muscle Nerve. 2000;23:1344–1347

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