BMT or NO BMT
 

My 17 yr old son has classic PNH 55% and is on soliris. He had meningococcal sepsis in Nov 2012 even with the vacc. He is also now taking a daily penicillin along with a multivitamin and folic acid. He has no transfusions and is doing very well on the soliris LDH stays around 160.
He has a 100% HLA match sibling,16 yr old sister. We took him to the NIH for a second opinion from DR Neal Young, and they said do the BMT because he is young and healthy enough and has a matched sibling. His reg Hemog said she was shocked at this and said no talk to out transplat team, the transplant Dr. from our regular hospital said they would not want to do it right now because he is doing good on the soliris.
We are going to see one more transplant team at U of M in Ann Arbor MI to see what they say.
I am having a hard time not just going with what Dr Young told as, seeing as we drove 15 hrs to see him. My son is young and I dont want to see him have to go every two weeks for the rest of his life to get his meds, but I dont want to see his life cut short because of a BMT went bad.:confused:

Any help would be great, as a mother I can't think of a harder choice to help him make.

Thank You

Shelby,

I've seen your earlier posts so I know how your dilemma came about. Although nobody can make this decision for you and your family, I can offer my perspectives as a caregiver. My wife and I once faced the transplant decision ourselves.

You are clearly aware of the big tradeoff: a chance for a cure and a way to put this all behind you versus the transplant risks. Conversely, transfusion independence with a treatment that's been working versus the disadvantages of a potential lifetime obligation to undergo tests and treatments.

Many PNH patients might envy your son for having a matched sibling, and that's fortunate indeed, but you still have to do what's best for your son.

Without knowing the future there's no way to be certain about the right choice. Nobody wants to "play the odds" when it's a matter of survival, but we have to rely on the information we have at hand. With such a rare disease, statistics are woefully lacking. The doctors must rely on cases that are similar, not exactly the same, to make predictions. It's important for all concerned to know the history of your son's symptoms, test results, and treatment responses, especially as they have changed over time.

Your son is old enough to contribute to this decision. He doesn't have an adult's long-term perspective, but this is his decision as much as yours.

Your daughter may have qualms about being a donor, and her concerns should be addressed, but I think you should handle that directly with her and not consider it to be a deciding factor in the transplant decision. Her risks would be very minimal and any discomfort very temporary. It might sound scary but if a transplant for your son makes sense, it's a necessary sacrifice that she'll learn to be very proud of.

I think that the devlopment of Soliris was a huge step forward for PNH patients, but that it hasn't been around long enough to establish a long-term track record. Although treatments improve over time and other drugs might be developed, I'd be very concerned about assuming that Soliris could control PNH or put it in permanent remission for someone with a whole lifetime ahead of them. For middle aged or senior patients the transplant choice would be riskier, but sibling transplants are so routinely successful for 17-year-olds that I would lean toward that choice as long as your son is judged to be in the necessary good health. I admit that I'm probably biased because of my wife's successful transplant, but that's my layperson's opinion.

Getting the third expert opinion is a great idea. I'm glad to hear that you are doing that. I hope you will share what you learn.

Since your son is still 17, you might consider consulting Dr. Margolis.
He is an expert in pediatric transplants.

http://www.mcw.edu/PhysicianDirector... r%2526gt%253b

Thank you for the responce.
Dr. Margolis is the Dr they recommended at the NIH. It's close enough to Michigan that the trips should not be that bad (when the ferry is open lol).
My oldest son and my daughter were more than happy to be tested for a match and when my daughter came up at a perfect match she was like lets do this. She is more than happy to do what ever her big brother needs.
But it is up to him to make the final decision, we just want him armed with as much info a possible not just with stats but from people who have been through it.

There really aren't any stats out there for transplants that a) don't involve cancer and b) with only younger patients, so those general stats that get distributed really aren't very applicable to patients like myself or your son, just that his odds are going to be much, much better than those!

If you want a good case study reason on why he should go for the transplant, read my story on these forums. I'm running my second marathon next month.

Hi Shelby!
 

My 14 yr old son, Mac, has PNH, is on Soliris and is a patient of Dr. Margolis. We live in a suburb of Milwaukee and are fortunate to have Dr M so close. Mac was dx in 2009. Altho neither of his sisters are a match, he has several excellent non related donor matches available. As a txp specialist, Dr M has recommended Soliris over txp at this point due to high risk and long term side effects of txp. We also took our son to see Dr Maciejewske in Cleveland who stated that with txp, a patient often trades one disease for another (GVHD). Mac's soliris infusion are done at home. Couldn't be more convenient!
I would love to talk with you abt our boys and their treatment choices. Pls let me know if you would like to exchange phone # or e-mails. Thnx, Amy