Supportive Treatment Options for DMD

Amy D. Harper, MD: Supportive options come from the multidisciplinary team. As I mentioned, standard-of-care treatment is with the use of steroids, meaning there is an increased risk of infection ,and so it is important that that child have a yearly flu shot and also stay up-to-date on their pneumococcal vaccine. Then, thinking about the heart, the cardiologist’s role is very important because patients will need ongoing surveillance for cardiomyopathy, echocardiograms, and medications which help to preserve the heart longer. As that child becomes older and is having worsening functioning of the heart, we’ll involve our heart function doctors who are typically adult cardiologists.

From a pulmonary standpoint, there is a lot that we can do. Each child is given a CoughAssist with instructions of what is the respiratory plan going to be when they are sick. We also teach them with our respiratory therapists how to do different techniques such as breath stacking as well as monitor them closely and work closely with our sleep medicine colleagues to provide them sleep studies. If patients are under breathing or there is hypoventilation on the study then we can prescribe and titrate a BiPAP so they can continue to get a restful sleep. These are the primary players.

From an orthopedic standpoint, there can be progression of scoliosis as the child becomes weaker over time, it’s important to treat that scoliosis so you can maximize the pulmonary status and comfort receding. There are a lot of benefits from these orthopedic interventions. Your physical therapist is going to start really early in trying to have that child be placed on a good routine as far as their stretching and the use of night splints, which if you can start those routines early it’s going to be easier to prevent contractures at the ankles long-term, which will result in prolonged ambulation as well. Later, we are looking at different adaptive devices and appropriate wheelchair for that child. It’s an ongoing progression with the help of the therapist and the physiatrist to make sure that patient is best supported.

As a result of all these best practices, there are a number of standards-of-care guidelines that you can follow, Lancet has those published. We are definitely seeing a change in longevity and lifespan for these boys, or, maybe I should say, men now.