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Twenty-eight years of steroids, multiple publications, Cochrane Report, and significant unpublished data, and we still have no definitive answers about the RIGHT regimen and I’m guessing we never will.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.
And variations on the theme:
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.
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.
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
Read more PPMD Blogs