ATG or BMT

[Fana]

Hi everyone, I am new to this forum my son was diagnosed with Aplastic anemia on the 25th of May 2014.from then to now it has been a shocking journey...he had to do two bone marrow aspirations..and his platelet counts drop to low 9 ..he has to get a transfusion every 10 days as his counts get very low..the doctors did a HLA test on his sister and she is thank god a good match.after the results came we we're send to a bmt consultant who told us even thought the sister is a match we still have the medicine option as well and in his condition meds might work..so where considering ATG but went for a second opinion to a another BMT consultant who told us BMT is the only option as ATG won't cure SAA..now we are confused as ever in making a decision

My main question is can a child answer well to ATG? By the way my son is 8 years old.Do I need to go for the BMT?

[KMac]

Hi Fana,

I am sorry to hear you have to go through this, but welcome to the forum. You have found a wonderful resource.

This is a hard decision for you. I believe either option, ATG or BMT, holds great possibility for an excellent outcome. However, both of course also have very serious risks. I believe BMT is the riskiest on the short term (rejection/GvHD), with the risk of ATG being longer term - relapse or clonal evolution to MDS/AML or PNH (though that can also happen after BMT, but less frequently).

As for myself, I am a 44 year old male, and the first thing my hospital (CBCI/PSL in Denver) did was check if my two siblings were a match. Neither was a good match, but if one of them was, I would have went ahead with the transplant then and there, even at my age. This is because hemtologists are getting so much better at BMT, and I understand the younger you are, the better chances of a good BMT outcome (but I think the same can be said of ATG). And BMT can be a true and complete cure, forever. I have a friend whose grandson received BMT from his sibling's cord blood over 20 years ago, at about your son's age, and he is doing great now, completely normal.

I am doing quite well 2+ years after ATG, but I recovered very slowly, and my counts still are not completely normal (I'm still neutropenic, but my platelets and hemoglobin are low-normal), and I still work through some fatigue. That is the hard thing about ATG, though it is less risky on the short-term, it is not a complete cure - few people achieve completely normal counts after it. But many do go on to feel completely normal post-ATG, and stay that way for decades or perhaps forever. So it can be an excellent treatment.

Not an easy choice for you. I wish you and your family the best as you make your decision. As hard as the choice is between ATG and BMT, I find solace and good fortune in considering how we have at least two good options to choose from, whereas in previous decades those with SAA had few if any treatment options.

[Fana]

Thank you for the reply kmac glad to hear that you are doing good..

Your friend's grandson's story is very encouraging for me as it showed me there might not be lots of long term side effects in bmt that was one of my worries about bit.i know in ATG there is a chance of relapse is the same risk involved in bmt also?
You have also mentioned about clonal evolution to MDS/AML or PNH can you explain it little bit more to me

[Hopeful]

Hi Fana,

I am sorry to read that you have to face this with your son.

The outcome for children with AA is much better than adults. The current standard of thinking is to go directly to BMT for children with matched sibling donors. If no match sibling exists, then you would go with ATG. The BMT survival rate for children under 20 with matched sibling donors is very high and offers the chance for a cure. ATG is not a cure.

Of course, with children it is imperative to check that the AA is not an inherited form of the disease (like Fanconi's etc). Be sure that your son is being seen by a pediatric hematologist with expertise in this rare condition and that they are ruling everything else out that it could be. Where are you from?

I would encourage you to check out the excellent AA videos at the AAA&MDSIF website for information from experts across the US:

https://live.blueskybroadcast.com/bs...T=944&CAT=1031

Also, here is an excellent article by Dr. Neal Young about how AA is treated:
http://bloodjournal.hematologylibrar...ent/120/6/1185

I wish you the best!

[tserdogan]

Hi Fana,

Five years ago,i thought like you,what was the best for me.

First of all, the doctor tried Atg,it did not work.

i waited four months.And my doctor said to me,it was the time for BMT.He advised me another doctor for BMT.

And the doctor who is my BMT doctor,always says me you should not get ATG, if you have donor.

Because if ATG did not cure the patient,she need BMT.This means second ATG for the BMT.

You know the risks of BMT,but pediatric patients outcome better than adults.

I wish good luck for your choise...

[Fana]

Hopeful thank you very much for sharing the links with me..I have already watched the video..the article was very helpful. I am getting treatment for my son in Singapore.he was tested for falconi anemia which turned out negative.i was reading the dr Neal's article and saw that avoiding Heavy transfusions will help the BMT to be more successful do you know how many transfusions are they talking about..my son already had 5

Tserdogan I hope you are doing well after the transplant I have a few questions for you..after the ATG did you have to get transfusions? Before the transplant how many days of chemo was give to you? I read that after a transplant your blood group will change to the Donor's group how does that work? After a successful transplant how long will the body take to produce new cells..

[Hopeful]

Hi Fana,

There are a lot of excellent videos on that website, if you poke around. Here is one on pediatric transplants for AA:

https://live.blueskybroadcast.com/bs...T=944&CAT=1090

5 platelet transfusions is not a lot. They do like to move quickly to transplant though, once you have made the decision. Blood products should be irradiated, leukocyte depleted, and CMV negative (if your son is CMV negative). All of these are important for a future transplant and you should check that your son's blood products are each time.

There were some pediatric people on this forum who relapsed after 5 to 15 years of stability. For an adult patient, 15 years would be awesome! For a pediatric patient though like your son, he will likely have to deal with this again and the timing will be less convenient (like when he is in high school or college or ?). There have been some grown pediatric patients on the forum that don't want to deal with being sick again after a relapse or going through the treatment. It is hard living with the uncertainty of the illness.

I would recommend talking to a transplant team, if you haven't already. Their perspective is different from the regular hematologist. I remember seeing survival rates of 80-90% in the pediatric population with a matched sibling donor.

[tserdogan]

Hi Fana,

İt is ok but side effects of drugs unfortunately. You learn how to live with this issue.
More than 50 blood,20 PLT transfusion i got.
I dont remember completely,6 is possible and two kind of chemo i got
I waited 10 months,may be more, to change My blood group.
After transplant more than 60 blood,10 PLT transfusion,i got.

[KMac]

Hi Fana,

My understanding is that while relapse is a possibility after both ATG and BMT, the chances of relapse is much higher with ATG. I believe about 70% of patients have a good response to ATG, but of those, about 30% eventually relapse and about 10% have clonal evolution to PNH, MDS or AML. Also with BMT, clonal evolution is a possibility, but a much smaller probability than with ATG (not sure of the exact percent).

The clonal evolution is usually found via a bone marrow biopsy, where they look at the chromosomes and other features of the cells in your marrow to see if there are any indicators of these other diseases. I believe hematologists are not certain whether this clonal evolution is due to the ATG/cyclosporine treatment itself, or is part of the natural progression of aplastic anemia in certain patients. But either way, since a successful BMT should fully replace your diseased cells with new healthy ones, the probability of any clonal evolution in that case is much lower.

With both ATG and BMT, the longer a patient remains healthy with good counts after a successful response to treatment, the less of a probability there will be a relapse or evolution to another blood disease.

[Fana]

Hi everyone thanks for the replies. We had go for a transfusion last week and my son had some reaction he got a rash and a stomachache is this normal has this happened to anybody.his dr said its a very rear thing but possible..

After lots of research we have decided on bmt. I spoke to the his dr and she wants me to start his dental as the first step....looks like a very long journey a head..

[Neil Cuadra]

A rash, hives, or itching after a transfusion is an allergic reaction. It can be treated with an antihistamine such as Benadryl before the transfusion. I don't know why a transfusion would produce a stomach ache, but let the doctor know about your son's reaction so an allergic reaction can be prevented with any future transfusions.

[wsmeds]

dear Fana:
I hope everything worked out well for your son. Our journeys are very similar. My 10 year old son was also diagnosed with very severe aplastic anemia. Thankfully, my daughter is his donor match and he had a bone marrow transplant on July 1. He stayed in the hospital for 35 days after the transplant. His last transfusion so far was August 4 (the day he left the hospital).

We are now on strict isolation at home with weekly clinic visits to check blood counts.
I hope all went well with you!
Smeds

[Fana]

Hi Smeds
Thanks for the encouraging words my son will have the transplant on the 1st of September....right now he is getting the conditioning for the BMT..tomorrow he will get cyclophosphamide for the first time I am bit worried how he will react to it was your son give cyclophosphamide too?
How is he doing now? How long do you have to stay in isolation? Praying for a speedy recover for your son