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A New Care Guide to Give to Your Provider – Imperatives for Duchenne Care

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

 

Review Team:

  • James Dowling, Sick Kids, Toronto, Canada
  • Norbert Weidner, Cincinnati Children's Hosptial Medical Center, Cincinnati, OH Brenda Wong, Cincinnati Children's Hosptial Medical Center, Cincinnati, OH
  • Linda Cripe, Nationwide Children’s Hospital, Columbus, OH
  • Doug Biggar, Holland Bloorview Kids Rehab, Toronto, Canada
  • Garey Noritz, Nationwide Children’s Hospital, Columbus, OH
  • Jonathan Finder, Children’s Hospital of Pittsburgh, Pittsburgh, PA 

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Comment by Fred Coates on June 13, 2016 at 9:25am

This trail ended on April 30 2016, I would think it will take awhile to report, but does anyone have any insight into the results?


Staff
Comment by Kathi Kinnett on April 16, 2014 at 4:57pm

Dr. Anne Connolly (Washington University, St. Louis) is conducting a study of boys younger than 2 1/2 yo and prednisone.  This study will evaluate the risks and benefits of early weekend prednisone treatment in very young patients living with Duchenne.  Further information can be found at: 

http://quest.mda.org/news/mda-funds-study-prednisone-very-young-boy...

Comment by Peter on April 15, 2014 at 5:12pm

May you please, shed more light on the Anne Connolley's study.

Comment by Simone & Elias on April 15, 2014 at 12:31pm

Thanks a lot for explaining. The best is I will discuss this issue again with our doctors at our next appointment and showing them your care guide.


Staff
Comment by Kathi Kinnett on April 15, 2014 at 12:01pm

Anne's study is just starting, so we are a ways from results. In the mean time, you are right - there are many side effects to using steroids. However the benefits are worth the side effects for most people.  Using steroids is a very personal decision, and when to start them is a personal decision as well.  As I said, there is no hard and fast rule for when to start or for how to take them.  Many parents and clinicians are opting to start weekend dosing for younger patients who are growing, then switching to daily dose after the plateau phase.  However, as I said, we have no evidence to say that this, or any other, regiment is best. We do know that daily steroids started at least by the plateau phase prolongs ambulation, and deflazacort seems to extend ambulation further than prednisone (by about 2 years). Other than that, we are all waiting for the For DMD and Anne's study results.

Comment by Simone & Elias on April 15, 2014 at 11:32am

Thanks Kathi for your immediate answer. When will the data of Annes study be available? It is so difficult to decide whether to start so early or not. We are afraid of the adverse effects - doctors told us they will multiply the longer a boy gets treated and that would be the reason not to start so young.... Can you confirm this?

Thanks again :-) Simone


Staff
Comment by Kathi Kinnett on April 15, 2014 at 11:23am
Simone - many countries wait for deterioration to start steroids. When we said to start the conversation early, it is with the hope that steroids will be started before deterioration, usually before or during the plateau phase. Unfortunately we do not yet have evidence that tells us exactly when to start steroids. Anne Connolley's study with weekend dosing of very young patients may tell us what we need to know. Until then, we hope that all providers will begin steroid treatment before or during plateau.
Comment by Simone & Elias on April 15, 2014 at 11:09am

My son (nearly 3) gets "treatment" in Germany. Doctors here say to start corticosteroids only at the time he does no longer develop (so called plateau phase), not earlier. You say START ASAP. Is there any statistically data saying the earlier the better?


Staff
Comment by Kathi Kinnett on April 15, 2014 at 10:05am
Oops! Thanks Janine!
Comment by Janine on April 15, 2014 at 10:03am

It is Pittsburgh, with an h at the end

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