Steroid treatment – a step by step guide

Steroids are used in many other medical conditions and there is a lot of experience in their use worldwide. There is no doubt they can benefit many boys with DMD but this benefit needs to be balanced with proactive management of possible side effects. Use of steroids is very important in DMD and should be discussed with all families early.

The basics

  • Steroids (also called glucocorticoids or corticosteroids) are the only drugs known to slow the decline in muscle strength and motor function in DMD. The goal of steroid use is to help the child walk independently for longer to allow enhanced participation and to later minimise breathing, heart and orthopaedic problems. They can also reduce the risk of scoliosis (curvature of the spine).
  • Prevention and management of steroid side effects needs to be proactive and anticipatory. Interventions should be put in place EARLY in an effort to prevent problems and to make sure they do not become severe. Side effects associated with steroid use vary and are listed here.

Starting and stopping steroids

  • The optimal time for starting steroid treatment is when motor function is in a “plateau phase” – when the boy’s motor skills have stopped improving, but have not yet started to get worse. This is normally sometime between the age of 4-6 years. It is not recommended to start steroids in children who are still gaining motor skills, especially if they are under 2 years of age.
  • The recommended national vaccination schedule should be complete before steroid treatment is started, and varicella (chicken pox) immunity should be established.
  • Starting steroid treatment in boys/young men who are no longer walking independently is a matter for individual decision and needs to be discussed with the doctor, taking into consideration the effect of pre-existing risk factors. In boys who used steroids when they were walking, many experts recommend continuation of medication after loss of ambulation. The goal in the non-ambulatory person is to preserve upper limb strength, slow the progression of scoliosis, and delay the decline of respiratory and cardiac function.

Important facts to remember

  1. Steroids are the only medicines known to help slow down muscle weakness.
  2. Always tell doctors and other healthcare providers that your son is taking steroids. It is especially important if he is having surgery or has an infection or injury because steroids can suppress the immune system.
  3. Your son should never stop taking steroids suddenly.
  4. Your son should have regular visits with a doctor who is skilled in managing steroids. The doctor will explain possible side effects and tell you if your son is at risk of developing them.

Information based on consensus statement (published in January 2010)