Muscular Dystrophy and Kids on Corticosteroid

[MotoSook]

Ok guys. I know there are some doctors on this board and there are folks here who may also have some first hand experience with MD.

I have a nephew who has the gene defect. I’ve been researching this and there is no cure, nor any adequate treatments. My nephew has Duchenne's muscular dystrophy, and apparently the only treatment is anti-inflammatory corticosteroid medication (prednisone) which may help improve muscle strength and delay the progression of the MD.

There are side effects of corticosteroid that my sister and her husband are not comfortable with.

Anyone have experience with corticosteroid as administered to children under 12 yrs old?

Here is what the Mayo Clinic has to say about it:

Muscular dystrophy

Treatment

There's currently no cure for any form of muscular dystrophy. Research into gene therapy may eventually provide treatment to stop the progression of some types of muscular dystrophy. Current treatment is designed to help prevent or reduce deformities in the joints and the spine and to allow people with MD to remain mobile as long as possible. Treatments may include various types of physical therapy, medications, assistive devices and surgery.
Physical therapy
As muscular dystrophy progresses and muscles weaken, fixations (contractures) can develop in joints. Tendons can shorten, restricting the flexibility and mobility of joints. Contractures are uncomfortable and may affect the joints of your hands, feet, elbows, knees and hips.
One goal of physical therapy is to provide regular range-of-motion exercises to keep your joints as flexible as possible, delaying the progression of contractures, and reducing or delaying curvature of your spine. Using hot baths (hydrotherapy) also can help maintain range of motion in joints.
Medications

Doctors prescribe medications to treat some forms of muscular dystrophy:

•Myotonic dystrophy. Medications that may be used to help manage the muscle spasms, stiffness and weakness associated with this condition include mexiletine (Mexitil), phenytoin (Dilantin, Phenytek), baclofen, dantrolene (Dantrium) and carbamazepine (Tegretol, Carbatrol).

•Duchenne's muscular dystrophy. The anti-inflammatory corticosteroid medication prednisone may help improve muscle strength and delay the progression of Duchenne's MD.

Assistive devices
Braces can both provide support for weakened muscles of your hands and lower legs and help keep muscles and tendons stretched and flexible, slowing the progression of contractures. Other devices such as canes, walkers and wheelchairs can help maintain mobility and independence. If respiratory muscles become weakened, using a ventilator may become necessary.

Surgery
To release the contractures that may develop and that can position joints in painful ways, doctors can perform a tendon release surgery. This may be done to relieve tendons of your hip and knee and on the Achilles tendon at the back of your foot. Surgery may also be needed to correct curvature of the spine.

Other treatments
Because respiratory infections may become a problem in later stages of muscular dystrophy, it's important to be vaccinated for pneumonia and to keep up-to-date with influenza shots.

[Tobra]

Prednisone can do some pretty nasty things to you. Those other meds on your list are no joke either. You have to wonder about your risk/benefit ratio.

I would be more inclined to do a Murphy procedure(looking at it from the side, you move the attachment of the Achilles from the back of the heel bone to the top of it, changing the lever arm) rather than a tenotomy/release. This is something you think about after your conservative stuff is exhausted.

The kid and his parents need to get up close and personal with physical therapist, and will have an active home program. They will have to be as relentless as the disease process is.

[Sarc]

let's keep this on the first page...

[artplumber]

Originally Posted by Tobra

Prednisone can do some pretty nasty things to you. Those other meds on your list are no joke either. You have to wonder about your risk/benefit ratio.

I would be more inclined to do a Murphy procedure(looking at it from the side, you move the attachment of the Achilles from the back of the heel bone to the top of it, changing the lever arm) rather than a tenotomy/release. This is something you think about after your conservative stuff is exhausted.

OK no Duchenne's expert, but while conservative measures may help with contractures, and your suggested op helps with lower limb mobility, those aren't the only muscles of the body. Steriods are systemic, and can cause increased glucose, thin bones etc, but offer therapy of many more muscular groups which PT can't always address/or have time to work eg/diaphragm or all extremities.

[Tobra]

I would not presume to speak too much out of my area of expertise. My board certification is in foot surgery, but the premises are the same. If you change the lever arm rather than lengthen the tendon, you are left with a more funtional limb because of the way the muscle works. The insertion of the Achilles is probably one of few places where you could really do this with any degree of facility, now that I think about it, but it has an impact on three pretty major joints so you get a lot of bang for your buck, so to speak.

As I said, prednisone is no joke, trust me I am familiar with what it can do. I know several cases of avascular necrosis of the head of the femur happening from even short courses, which is not what we are talking about here. Bo Jackson had to have some great big guy snatch his hip out of the socket to do this, and you can get the same effect from a tiny little pill.

Every patient is different, so you have to tailor your treatment. You get their muscles working better with the prednisone, and turn them into a diabetic or bag their hip. I am not a surgery first kind of guy, but I have a lot of patients I do some type of procedure on them the day we meet. I am no expert on Duchenne's either, but you could think of it as a battle you can't win, not yet anyway, only fight delaying actions.

It ain't just PT workin' with the kid, it is the whole family or a poor outcome is much more likely. You can do some amazing stuff with kids and physical therapy, some of I would never think of, like that Ride to Walk deal with the horses.

[artplumber]

Originally Posted by Tobra

As I said, prednisone is no joke, trust me I am familiar with what it can do. I know several cases of avascular necrosis of the head of the femur happening from even short courses, which is not what we are talking about here. Bo Jackson had to have some great big guy snatch his hip out of the socket to do this, and you can get the same effect from a tiny little pill.

You're falling back on anecdotes. AVN is not very common considering the number of patients that are on systemic steriods for one reason or the other. I know that because I'm upstream (ie seeing patients started on steriods for lung disease, collagen vascular disease, transplants etc.)

Originally Posted by Tobra

Every patient is different, so you have to tailor your treatment. You get their muscles working better with the prednisone, and turn them into a diabetic or bag their hip. I am not a surgery first kind of guy, but I have a lot of patients I do some type of procedure on them the day we meet. I am no expert on Duchenne.....

The point is that steriods can be part of a tailored therapy, and decrying them to someone (who already appears to have been affected by opinionated pieces which may have little basis in fact) is a disservice. Much like the disinformation spread on numerous websites and bulletin boards about pharmocotherapy for cholesterol reduction. Let the experts talk to the problem, and don't push individuals into doing just what you're familiar with.

Finally, Souk Chi town is a big city. Plenty of MD's and Institutes of various kinds. I'm sure you can find a true expert locally that would know what is best. Tell that person your concerns (or your relatives tell that person). Get the info. Then make a decision.

[livi]

This is completely impossible to have an opinion on over the Internet. The clinical diversity and variance in symptoms and severity is enormous. There are definitely situations and periods with this condition where steroids are beneficial, but that needs a thorough knowledge of a specific patients age, duration of symptomatic disease, severity, specific symptoms etc etc. There is no general rule for or against.

[gchappel]

Please, Please, Please
Find a physician that you can trust for the treatment of this- or any other disease. Then trust them, and let them go forward with treatment. Questions and dialog certainly are appropriate- with this doctor. Searching for treatment options and opinions on line is, imho, not in anyones best interest. This is not like rebuilding a transmission. Each patient and each disease is different. Treatment plans need to be individualized. All medications and treatments have side effects, it takes a professional with experience to balance the risks and benefits. If you don't trust your doctor find another, but if you do trust them let them do their job. Or go to Walmart's, ask the cashier what they think and do that.
Gary

[MotoSook]

Originally Posted by Sarc

let's keep this on the first page...

Thanks man.


Gary -

Aren't you being a little harsh Gary? Asking for opinion from doctors or people who have first hand experience with corticosteroid doesn't mean that the family physician hasn't been consulted. As I stated, there is no cure for MD and treatments have mixed results and for the most part only provide marginal benefit with side effects that can be worst than the desease. This is not a convenient desease and no one involved is taking it lightly nor out shopping for medical advice at Walmart. If you don't think the family (our family) hasn't considered all options or are doing everything we can, you are mistaken. The likelihood that this child will pass on without living a full adult life is very real to our family, and I am not going to limit my search for knowledge and understanding of the medical and personal affects of the desease.

Because there are few options in treatment of this desease, I think it is appropriate to explore all options, including asking for opinion on this board. My hope was that a doctor or parent with a child struggling with MD would share first hand experience about the affects of corticosteroid. Surely corticosteroid affects individuals differently, but having some knowledge of the range of effects and how it affects patients and their family is the goal of this thread. So please, if you don't have anything positive to add, move along.

[gchappel]

Sorry if I came across a little harsh, but I deal with this almost every day with oncology patients. And yes, I am trying to be helpful.
I see families spend enormous amounts of time and effort searching the web endlessly for therapy ideas. The vast majority- in fact every single one that I am aware of- would be better off spending that time and energy with their loved one. First, I am making the assumption that you have a doctor that you trust. If you trust them, let them make the decisions on the direction of the care. If you have questions, ask your physician. Questions such as why, other alternatives, side effects, etc are all appropriate but should be directed to him/her. If you don't have that level of trust- find another doctor. I find this board to be unbelievably helpful and wise, but even it cannot answer this type of question. A question like- "Does anyone know of a physician that specializes in MD that could see nephew for another opinion?"- can be useful. A question, which I get routinely, like is adriamycin appropriate for my treatment? is not answerable without a compete evaluation. So, can this board- or any other board for that matter- answer a question such as are steroids appropriate treatment for my nephew- no it cannot. Even if 100 people chime in that steroids are inappropriate here, without specific knowledge of this case we are all guessing, so ignore us.
Please, explore all your options. Find a physician that specializes in MD, one that has studied this disease for years. He will know more then we.
Gary

[Nathans_Dad]

Yes steroids have side effects. Increased glucose, early osteoporosis, Cushingoid appearance (mainly central obesity also the "moon face"), early coronary disease, thinning of the skin, hypertension, just to name a few.

The bad news is that steroids are still the mainstay of therapy for several disorders and tend to work the best many times. The idea is to use the lowest amount possible to get the desired effect. Whether steroid therapy is best for your nephew is really a decision that should be left to his doctor. Unfortunately muscular dystrophy is a devastating disease and there are no easy answers. Good luck.

[Tobra]

Peter, the steroids are a part of most treatment plans for this, my point was to proceed with caution. The catastrophic side effects are indeed very rare, say one in a million, that is fine for about 999,999 people in the mix, but for that one guy...

Clearly this is something best addressed by the treating doctor, but here is a flash for you, lots of doctors communicate poorly and the patient leaves with more questions than they walked in with, or fail to mention or make clear some pretty significant things.

If I thought it were a disservice to disseminate factual information I would not do so. There are a lot of scary treatment regimens out there, talk to anyone with RA or being treated for cancer. This kid will be taking steroids at some point, but that does not make them any less scary. The eye can only see what the mind knows. If you are in the dark about what could happen, you will never realize if it does.

I would encourage someone with a complex problem or chronic disease such as the ones mentioned keep a notebook. Write down all your questions you think of at home and what the doc is telling you during visits. Keeps things focused during visits and improves your understanding of what is happening.

Gary makes a good point. Your doctor spent years to learn what they know. It is ridiculous to think you will gain much insight when you google Duchenne, Charcot or any other dead French neurologist's names compared to asking a guy who has dedicated his life to understanding and treating a disease process.

[Nathans_Dad]

Tobra I'm curious, are you a podiatrist or an orthopod?