[Lisa V]

Stephanie, the first thing you learn when you or a family member has been diagnosed with a rare disease is that medicine is not an exact science. This goes against our expectations that it will be something like auto mechanics: find the malfunctioning system and repair it and voila! No more problem. In reality, there can be as many different interpretations of symptoms and philosophies of treatment as there are doctors and personalities. That's why it's so important that either the patient of someone close to them educate themselves as much as possible on the condition in question. If you can have reasonably informed discussions with Dylan's doctor, you can have more influence on his course of treatment than you might have imagined, and it will help you overcome your feelings of powerlessness and frustration if you at least understand why they are making the decisions they are. Fortunately the web has made research and interaction with others in the same boat so much easier these days! Just be aware that there is a lot of misinformation out there too, so try not to jump to too many conclusions based on a single piece of evidence.

I have to say I don't quite understand their timing here either. Why do a 4th BMB if it's not going to affect the course of treatment? I agree with Hopeful that if it appears to be autoimmune-related, ATG is his best option, and sooner rather than later. It's true the treatment is not benign, but neither is waiting, so what it comes down to is often a choice between what's in the box or what's behind curtain #2, i.e. a gamble either way. Which option any given doctor will choose will depend on weighing all the factors and a lot depends on their interpretation. As Hopeful has pointed out, there can be varying degrees of what might be considered dysplasia. Most AAers (and perhaps even the rest of us for all I know) have found notes on their CBCs about various oddly-shaped cells, but apparently most of them are not enough to qualify as the kind of "frank dysplasia" that would be a clear indication of MDS.

The issue of determining if a condition is truly immune-mediated can be a tough call too. Most doctors seem to arrive at that conclusion AFTER it responds to immunosuppressive treatment, rather than the other way around. There could be other factors such as shortened telomeres, which may indicate a hereditary condition rather than an acquired autoimmune one, but this is still new research and testing for it is not often done. I suspect the cost and time involved are a big issue, and it also creates a dilemma if there are no other good options available. Better to at least try the thing that has the best track record than to do nothing, at least that's the thinking.

As for what ATG will do to your son, hopefully he'll sail right through it like my husband did, but some people do develop serum sickness from the horse serum. This is why they prescribe prednisone, to counteract that, but prednisone, cyclosporine and ATG are all powerful medications with side effects. Also, any time you have to take something to knock out a portion of your immune system, you're making yourself more vulnerable to infections and other secondary diseases, but the body is remarkable in its ability to compensate and adjust, particularly children and young adults. You'll have to follow certain precautions, but it's only temporary. If he responds well and his counts rebound, he should be off all medications in about 6 months.

I hope none of this scared or discouraged you, I just wanted to shine a light on some of the issues involved. It's a scary time, but you'll get through it. It's what we do.

All the best,