Unfortunately, the care of people living with Duchenne muscular dystrophy differs from country to country, from state to state, and even from center to center. Email and stories that we get daily confirm that, despite the availability of Care Standards there is still significant practice variability, including centers and locations where clinicians have little or no knowledge of appropriate Duchenne care.
“There is no care for my youngest son in this country. He has Duchenne. After the death of my first son, I was devastated, so I sought help from the Internet and discovered prednisone, which right now I am giving him. 2 tabs every day. Is there someone somewhere who can help me to care for my son?”
“Please can you help me get to the UK or the US so that my son can get care? He suffers from Duchenne muscular dystrophy. No one in this country knows anything about Duchenne.”
“Can you please speak with our cardiologist? She has never heard of Duchenne and does not think she needs to see my son or that his heart needs to be checked until he is in his 20’s, when she feels he will develop problems. She will not order an echo or a cardiac MRI, and will not start him on any cardiac medications. Is care different in other states, or in other cities? Can you help me?”
Providers care for many people, with many diagnoses – far too many for any one health care provider to be an expert in them all. Any busy provider would find it difficult to have the time to look over a 2-volume document (like the Care Considerations [PDF]), or even a several-page document (like the Family Friendly Care Considerations [PDF]), during a busy clinic.
Most health care providers want to provide each of their patients with the best possible care, but having the time to investigate the needs of each individual patient is nearly impossible. Providers are in need of a quick, reliable resource that will give them a snapshot of what standardized comprehensive care for patients living with Duchenne should look like. Patients and parents are in need of a resource that quickly identifies the necessary components of comprehensive Duchenne care so they can be sure that they or their child is receiving the best possible care. PPMD and a small group of international experts (from UPPMD and TREAT-NMD) have created such a snapshot – Imperatives for DUCHENNE MD (PDF).
As science and discovery progress, our messaging must progress as well. You may notice that the anesthesia message on the “Imperatives” is slightly different than on the Duchenne Emergency Card. There are several known cases of Duchenne patient death following the use of the anesthetic agent, succinylcholine. For that reason, we are saying, “Avoid Succinylcholine.” In addition, there may be side effects in patients with Duchenne using some inhaled, as well as some intravenous (IV) anesthesia. These possible side effects may include breakdown of skeletal muscle tissue and the release of too much potassium into the bloodstream, which may cause in problems with heart rhythm and function. For this reason, we want all providers to, “Use caution with all anesthesia.” The messaging on the PPMD Duchenne Emergency Card, mobile app and website will be changed as well.
Imperatives for Duchenne MD (PDF) is meant to be a kind of “cheat sheet” for standardized, comprehensive Duchenne care. This information does not address the emergency needs of people living with Duchenne, and does not replace the Emergency Card. "Imperatives for Duchenne MD" is very focused and very brief, and includes a snapshot of the imperative components of comprehensive Duchenne care as described in the Care Considerations and included in the Certified Duchenne Care Center Program’s Duchenne Care Survey.
This information is meant for health care providers, but can also be used by patients and parent advocating for comprehensive Duchenne care for themselves or their children. Imperatives for Duchenne MD (PDF) can be uploaded on your mobile device or computer, can be printed, can be found on the PPMD website and can be found on the PPMD app. More detailed information for each area of care and the care required for each stage of Duchenne, as well as the complete publication of the Care Considerations and the Family Friendly version of the Care Considerations, are available on PPMD’s Care for Duchenne landing page.
We hope that you will find this a useful resource. It is our hope that Imperatives for Duchenne MD (PDF) will help improve the care of people living with Duchenne around the world.
Kathi Kinnett, PPMD
Katie Bushby, TREAT-NMD
Elizabeth Vroom, UPPMD
Annemeike Aartsma-Rus, TREAT-NMD
Sunil Rodger, TREAT-NMD/UPPMD/PPMD