Sibling was not tested for pediatric AA patient
Has anyone ever heard of a case that went right to immunosuppressive treatment without checking the sibling for a marrow match?
Hello, my son was diagnosed with severe AA in February 2009. His BM biopsy showed cellularity less than 10%, and platelets less than 4,000 when we checked into Childrens Hospital for unexplained bruising. His ANC was 880. We moved fairly quickly into immunosuppressive treatment with ATG and then cyclosporine. He became transfusion independent within four months and is being treated with cyclosporine currently. His counts have been slow to respond and his platelets hover at 35,000. Since diagnosis, he hasn't gotten sick (infection, fever, even sniffles) and other than his port and having to get regular bloodwork and take pills daily, he lives a normal 12 year old life and has been asymptomatic (though restricted on his beloved contact sports).
While I feel like we are in good hands with our hematologists, I'm confused. Every parent I've talked with, everything I've read, every Margolis/Young webcast I've listened to, and even the "Best Doctors" 2nd opinion I got, indicate that the first course of treatment for a child is a BMT if there is an HLA match. He has a younger brother. Our doctors tell us the risk of a BMT is too great since he is asymptomatic, but we also learned at the last check-up that this could be as good as his counts get. Reading about some of the relapses is scary, and I can't help but feel it's a matter of time until counts start to slip, or worse.
Don't get me wrong, I'm thrilled with his lifestyle and progress at this time. But knowledge is power, and knowing our options is important to ensure we're doing what's in his best interest as time goes on. I also have some concern when I hear BMTs are most successful in children (ages 18 and under). If there is a limited window, and my other son is a match, I need to know.
Insurance will not pay for a bone marrow match test if a BMT is not the course of action the doctors have recommended. Does anyone have knowledge of a child with a similar case whose sibling was not tested? Any suggestions? Not having this piece of data (is my other son a match??) is frustrating.
I'm as puzzled as you that your younger son wasn't tested as a potential transplant donor for his brother. Transplants have risks but a successful transplant is curative and the long-term outlook is especially important for children since they have practically their whole lives ahead of them. Both ATG and transplants are statistically successful AA treatments for children so you want to make sure that these choices are weighed.
I wouldn't call platelets of 35,000 "asymptomatic." It's good to appreciate that your son can live a mostly normal life, and your family could probably manage with temporarily curtailing some of his physical activity, but until his counts are normal or very close to normal I doubt you'll feel you've reached your long-term goal.
It's hard for us as patients and caregivers, no matter how well-meaning, to second-guess doctors who know the details of each case. There are really two questions here: why did he get ATG in the first place without checking for a sibling match, and what is the best course of action now? The answer to the first question probably needs to come from the doctors who made the decision. I'd ask them straight-out: what factors determined their choice between transplant and ATG? Perhaps they had good reasons; it's part of their job to explain them. If you can't have that kind of conversation with your sons doctors, he may not have the right medical team.
The second question is more important, because it determines where you go from here, and it should be answered by a doctor you trust. You didn't mention what city you are in (there are a few Childrens Hospitals) but I think you should get a second opinion from doctors at a treatment center known for expertise with pediatric aplastic anemia. This will provide you with a fresh look at the treatment choices and give you confidence with your decisions as well.
Becca, I don't have an answer either. It sounds like your understanding of the protocol is much the same as mine, and I agree with Neil that you should ask his medical team directly. That is the only way you're going to find out.
My husband also went immediately to ATG without any sibling testing, but that's because he's over 50. It's different for kids, and the only reason I bring this up at all is because we also decided we wanted to know if he had a match, and we had to really push for HLA testing. If your insurance won't pay for it, you may have to pay for it yourself. It's not cheap, but with only one sibling to test, it could be worse. It might be worth it just for your own peace of mind. If you find out his brother is not a match, it will be much easier to accept what has already been done and where he is now.
If you find out he IS a match, then that opens the door for the next conversation. If the HLA testing is done by a transplant center, their team will likely review his case and make their recommendations one way or the other. My tendency at this point would be to wait until he does start to relapse, but I can certainly understand why you would want to put this behind you while he's still relatively young. You really need to get the opinion of someone trained in the issues of pediatric AA.
Our experience was that the treating doctors rushed the sibling HLA testing when our son was diagnosed with VSAA in 2006. As soon as it was determined his younger brother was not a match, ATG began. I would have the preliminary HLA testing done now for peace of mind and to know what your potential options are in the event circumstances change in the future.
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