Thread: BMT or NO BMT
View Single Post
  #2  
Old Tue Apr 9, 2013, 09:16 PM
Neil Cuadra Neil Cuadra is offline
Owner
 
Join Date: Jul 2006
Location: Los Angeles, California
Posts: 2,553
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.
Reply With Quote