Steroids: A Certain Can of Worms (A Complicated State of Affairs)

Steroids. What regimen, what dose, for what individual? When to start? When to stop? A complicated question to be sure.

Steroids are a complicated story and have clearly changed the natural history of Duchenne. We know they work for some, for most in fact, but not all. We know they increase strength and prolong ambulation, and statistics back this up. Predicting ambulation is prolonged on average for 2+ years. But this is not the end of the story. Genetic modifiers have been identified that are thought to dramatically push out this timeline. Individuals with specific genetic modifiers continue walking into their mid- to late teens, often called outliers.

It is clear that steroids have changed the natural history and we cannot forget about the impact of night splints and aggressive stretching. We can mention supplements, though there is scant data which is largely anecdotal. And hopefully there has been a general change in attitude, less therapeutic nihilism today than 10 years back – a therapeutic dose of hope. Physicians are less likely, today, to say ‘no hope and no help’ and send us on our way.

The Right Regimen
Twenty-eight years of steroids, multiple publications, Cochrane Report, and significant unpublished data, and we still have no definitive answers about the RIGHT regimen and I’m guessing we never will. Some years back we surveyed families, inquiring about their son’s regimen and interestingly we learned about 20 or so regimens in our very informal survey.

They included:

  • 10 days on and 10 days off
  • Daily Prednisone 0.35mg/kg
  • Daily Prednisone 0.75mg/kg
  • Daily Deflazacort 0.9 mg/kg
  • Alternate Day 0.75mg/kg
  • High Dose Weekend 0.75mg/kg X 14, divided into 2 doses
  • Low Dose Weekend 0.75mg/kg X 7, divided into 2 doses

And variations on the theme:

  • 10 days on and 5 days off
  • 10 days on and 2 days off
  • Daily with changing dosage
  • Random – modifications done along the way based on appetite (PM rather than AM dosing), behavior (every other day during the week, 2 consecutive days of the weekend)

There will never be the perfect regimen that is best for all of the boys. Is there such a thing as the average boy with Duchenne? With genetic diagnosis and hopefully soon, expanded knowledge about genetic modifiers, we may have a more informed ways to discuss steroids. At the end of the day, it is up to the family, the individual, and the clinical team to discuss what makes most sense. Keep in mind, medicine is personalized. And one day, we are all hopeful to have a replacement... something that improves strength with fewer side effects.

Spectrum of Responses
Over the last 20 years, I have met more than 1,000 boys with Duchenne, most of them on steroids. For me, they fall into specific subsets.

  1. No response to steroids – this is the minority, but there are clearly non-responders.
  2. Increase in strength accompanied by severe changes in behavior, which do not lessen over time.
  3. Increase in strength but dramatic increase in weight (insulin resistance, glycemic diet currently recommended by some clinics)
  4. Increase in strength accompanied by changes in behavior (easier to push their buttons)
  5. Dramatic increase in strength, nearly a Becker like phenotype.

Of course, it is important to keep in mind, this is a spectrum, with the lines blurring on each end. But, if you speak with parents you might hear these common themes.

When to Start? What Dose?
And there is the ‘when to start steroids’ dilemma. There are some physicians who suggest that starting early (say 3 or 4 years old) minimizes behavior side effects. This may be true, but the side effects after 5-10 years old are pretty obvious and for some boys and their families, really hard to deal with.

Other physicians suggest starting steroids when the boys plateau and some, with signs of decline. The argument from the other side, is that the best medicine is preventative, initiating therapy to prevent decline.

Still others suggest that the boys start steroids when the parents are ready, having fully weighed risk and benefit.

And there is the dose issue. Some physicians initiate therapy with 0.75mg/kg (or 0.9mg/kg Deflazacort) and never adjust the dose as the individual changes in height and weight. Others initiate therapy and adjust (increase) the dose over time as height and weight changes and consider 30-35mg/steroid day as the maximum permissible dose.

Continued Discussion
Whatever the decision, the discussion continues – each visit, each year – evaluating benefit and risk and potentially modifying the plan.

While there are suggested regimens and regimens favored by certain clinics, sometimes the side effects dictate that a change is necessary. This is where clinical judgment comes into play. Families must discuss their concerns with their physician, weighing benefit and risk, altering regimens and timing of the dose (from AM to PM).

Steroids will hopefully be replaced in the near future with something better, more predictable, less toxic. But for today, there is no easy or simple solution as all boys need and deserve personalized medicine.

Steroids are complicated. And it is important to remember, you know your son best. Consult with physicians, experts, other families in the community, and then make the decision as a family.



Pat Furlong, Founding President, CEO
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Comment by Pat Furlong on April 10, 2012 at 1:54pm

Thanks very much for your comment Berch and Katie. I understand the point of the study is to 'level the playing field' in a way, to try to collect data relevant to what approach (10/10 vs daily deflazacort or prednisone) may be optimal for most boys.  I understand this is particularly relevant for countries outside the US (Eastern Europe, France, etc) where steroids are not typically recommended.   In the US, thankfully, most clinics do recommend steroids a steroid regimen. 

Kevin Flanigan and Beth McNally have submitted a paper to NEJM (currenty in review) identifying a snip that seems to enhance the steroid effect in some boys.   I am hopeful you and your colleagues will include this as part of the screening process.   We are all anxious to see this paper in print as we learn as much as possible about Duchenne and each of our sons and make informed decisions about their care and treatment..


Comment by Robert Griggs on April 9, 2012 at 3:14pm

We share Pat Furlong’s conviction that corticosteroids benefit boys with Duchenne.  We also agree that the right regimen has not been established.  However, we are committed to finding the best regimen as well as defining standards of care that prevent or minimize corticosteroid side effects.  We are the principal investigators of an NIH-funded grant --- FOR DMD ---“Finding the Optimum Regimen for DMD” that is a collaboration of 40 centers in 5 countries.  This study was developed in partnership with patient groups including PPMD.  This study begins recruiting boys later in 2012 and will define standards of care and provide the data we need to advise boys and their parents concerning the best treatment for them.  This study is of great importance since corticosteroids may be needed by boys for their entire life.  The study will also provide data on the genetic factors that determine the severity of Duchenne and of steroid side effects.


Kate Bushby, M.D., Newcastle, UK

Robert C. Griggs, M.D., Rochester, NY

Comment by PPMD on March 28, 2012 at 4:42pm

Just wanted to update you all and let you know that Pat's travelling back from a meeting right now, so there's been a bit of a delay in response time. We've marked this discussion for followup and you can expect a response when she returns!

Thanks for your patience and we appreciate your comments! 

Comment by Ivy Scherbarth on March 22, 2012 at 11:38am

I understand that we are waiting on a soon-to-be-published article by Dr. Kevin Flanigan which will discuss his findings about these genetic modifiers. Webinar, perhaps?

Just another example of the amazing hard work that researchers are putting in every day, to the enormous benefit of our DBMD community, present and future. I am so grateful to the people who devote their professional lives to the Herculean task of understanding and eradicating Duchenne. Thank you all!

Comment by Deanna Wayne on March 22, 2012 at 10:34am

Thank you for writing this informative post. My son just started daily Prednisolone so I found it interesting that many different regimens are used. His DMD is caused by a frameshift mutation which I understand causes a more severe form of the disease. I would really like to see this information and more details comparing DMD boys with frameshift, deletion and duplication mutations. Do you know of any such studies or reports? Thanks.

Comment by lisa burke on March 22, 2012 at 6:24am

Yes, I'm interested to know what these genetic modifiers are too. Do we know if there are there specific mutations that do/don't respond as well as others? My little boy is probably going to start steroids this year so this is ,of course, very much on my mind at the moment....

Comment by Ivy Scherbarth on March 21, 2012 at 6:55pm

I'd like to know more about those "specific genetic modifiers" that you mention in your second paragraph. Where are we on the research for identifying these modifiers? How can we tell what modifiers we might have? Are there modifiers that indicate a likely non-response as well as modifiers that suggest a more dramatic benefit to steroids? Very interesting. Thanks!

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