How know when to do transplant?
 

I'm sure this has been discussed many times on this forum, but I wasn't sure where / how to look for it. I have had reasonably stable pancytopenia / moderate AA for several years now. In fact, it was first diagnosed in 2000, and I have been monitoring it ever since with regular CBCs, occasional BMBs, etc.

My question is: What is the "trigger" for deciding to have a stem cell transplant? Is it best to do it when you are younger / somewhat healthy so that you will be stronger? Is it best to wait until your counts are such that you really have no choice (i.e. as a last resort) due to the risk? Has technology reduced the risk of a stem cell transplant to the point it is a more attractive option earlier in the process? Is a transplant worth the risk to gain an improvement in life-quality?

Ok, I guess that was more than one question...

My counts have been low, but stable for many years. As long as things continue as they are, I could continue to live this way indefinitely -- but if I could find a way to be rid of AA completely, that is also quite intriguing to me. Just looking at options... and I'd love to hear what you folks think.

David M

David,

The doctors give advice about all of these issues, because there are so many tradeoffs to consider. It's also specific to each patient's circumstances.

Paradoxically, patients can avoid or delay transplants if they are doing well, but if a patient is to go to translate they should be as healthy as possible, with counts as good as they can get, and being younger is an advantage.

It's both a blessing and a curse to have two different treatment approaches, because you have more options but you have to keeping weighing the choice.

It's also a blessing and a curse that the decision is ultimately up to you. You can put your trust in your physicians and listen to their recommendations, but you have the final say.

The "trigger," if there is one, is the point when the balance between transplant risks and the potential for a cure tips toward the transplant. It's less likely to be a doctor's recommendation when you're doing OK (your disease is "livable"), when you don't have a suitable donor lined up, or when you have other health issues that would make a transplant too risky.

Your quality of life and your personal opinion about living with the potential for AA to get worse are also factors to consider. Unless you speak up about these factors, doctors are likely to base their recommendations more on keeping you alive as long as possible than on quality of life or the emotional toll of living with a potentially deadly disease.

You mentioned improved technology. It's true that transplants are safer than in the past, and will presumably become safer still in the future. The most obvious improvements are that transplants can be done for patients at older ages, with less preconditioning, and for patients with less-matched donors.

Agree with these points and also it is important to know the DNA changes in your diseased bone marrow overtime, i.e., if they are more worrisome DNA changes on the bone marrow biopsy over time.

Transplant for AA is relatively safer than transplant for other diseases and can be performed even at the older age. This is because that chemotherapy used for transplant in AA is much milder.

Would recommend a consultation with a transplant physician so the donor situation and disease status can be adequately assessed.

Neil is right on as usual. I would only add this point. As a patient in watch and wait for nearly 3.5 years, I woke up one morning and knew it was time. All of the signs were pointing to it internally and sure enough, within 2 weeks the Dr. recommended that it was time as well. Not sure why this should be trusted, but I have heard from many patients that they just knew when it was time, and this made acceptance of the need to move forward an easy choice.

Wishing only the best,
Dan