Maybe some people have different norms?

[edithr]

Anybody ever wonder if some people just have different norms? My sons BMB has him at 20% production, so they want to start ATG. But what if thats the way his body has always been? He has almost no symptoms - no fatigue, weight loss, bleeding... Are they jumping the gun in treating him? Their argument is it's easier to treat when they're younger and relatively healthy. But what if we wait and see what happens? He could be like this for, um, five years? Then treatment options I'm sure will be a whole nuther ballgame.

Does Western medicine promote too aggressive of treatment? Do other countries have a 'wait and see' approach'?

I don't want him to be the Guinea pig. It's just in the back of my mind. Any thoughts?

[Marlene]

More importantly, what are his counts? The odd thing is that some never recover to the "normal" range for their age BM cellularity after treatment, but achieve normal blood counts.

So it's important to look at the trend, if you have it, in his CBCs and get a second opinion or even third opinion. Have you gone to Cleveland Clinic? I understand Dr. Majewski (sp) has lots of experience with SAA.

I think treating younger is probably more important with transplants than ATG but could be wrong.

[edithr]

I'll have to get his counts later. We go to Rainbow, which is part of University Hospital. Do you think I should get a second opinion?

[Marlene]

I would get a second opinion. You may want to contact another fellow Clevelander.... Sherri directly. Her daughter was treated by Dr. Jaroslaw Maceijewski at the Cleveland Clinic. http://my.clevelandclinic.org/staff_...?doctorid=4362

Here's Sherri's profile info: http://forums.marrowforums.org/member.php?u=99

I think it is very important to get at least one more opinion by someone who is very experience with SAA. Anyone can treat, but I personally feel you have better outcomes with some who deals with this all the time.

Another good doc to check out is Dr. Margolis in Wisconsin. http://www.chw.org/display/ppf/docid...v/0/router.asp

[Hopeful]

As Marlene said, it is important to get a second opinion from a specialist in aplastic anemia. I wanted to emphasize doing this as quickly as possible.

I'm guessing that the doctor's wouldn't have done a BMB unless your son's blood counts were abnormal. Things progress rapidly with SAA (if that is what it is). Before transfusions, IST, and BMTs, people would die from untreated SAA within 6 months. I am not trying to scare you but just want to emphasize that although he may seem fine today, in another month he may be transfusion dependent with further damage to his stem cells.

So don't delay! Specialists understand the urgency of treating SAA patients and will fit you in quickly.

[edithr]

Hi Marlene,

Dr. Jaroslaw Maceijewsk only treats adults. Is CC that much better than UH?

[Marlene]

I would encourage you to contact his office. I know he's worked with children and he will either work with you directly or with a pediatric doc at the clinic. He worked with Dr. Young at NIH, who pretty much is considered one of the top docs when it comes to SAA.

UH has a good reputation as does CC. But I still feel you should seek another opinion especially since one of the top docs is in your backyard. The reason docs take on kids is because AA is so rare and there are so few doc with extensive experience with it. I know our doc at Hopkins worked with children indirectly. By that I mean, the parents set up a consult with him and then he continued to work on the case with the pediatric Hematologist to treat with his protocol.

Take some time to read through Sherri's caringbridge blog. I think they started at UH also. http://www.caringbridge.org/visit/an.../createdAt/asc

[Lisa V]

I agree with the others that Dr. Maciejewski is one of the top AA specialists in the country. If we were anywhere within a couple hundred miles of him, we'd go see him. You could at least ask your doctor to consult with him (or Dr. Margolis or Dr. Young) if you don't feel confident in his judgement.

Having said that, any decision to treat must be based on a combination of counts and cellularity, not symptoms. Many patients are essentially symptomless. Severe AA is defined as having a bone marrow cellularity of less than 25% and at least two of the following: a neutrophil count less than half a billion per liter (<500/mm³), a platelet count les than 20 billion per liter (20,000/mm³), a reticulocyte count less than 20 billion per liter (<20,000/mm³). VERY severe AA is defined by a neutrophil count of less than 0.2 billion per liter (<200/mm³).

If your son meets these criteria, then your doctor is right to want to move quickly to treatment, either with a marrow transplant if he has a suitable sibling donor match, or with ATG if he doesn't. People's norms may vary slightly, but not that much. It's also important to note how quickly his counts are dropping in order to gauge how much time you have to think about things. Watch and wait is appropriate for Moderate AA (higher counts and percentages than those listed, but still below normal), but once you get to where you are needing to be transfused, that in itself carries risks and complications which only increase with time. You may find one or two SAA patients that have spontaneously improved, but statistically, untreated SAA will result in death 80% of the time. Whatever your feelings about western medicine (and I don't disagree that many agressive procedures could probably be avoided), this is not a good playing field to test them on. Just my opinion.

[edithr]

Hi Lisa.

I know people keep recommending Dr. Maciejewski, but when I called to get an appt they said he only accepts adults. The doctor/hospital we are going to is literally right next door to the CC - University/Rainbow. I love his doc(s) - they explain things very well (with pictures!), answer every question (multiple times, because I can't seem to digest info too well), they explain everything to him and treat him as a person and not a kid. Do you think the next time I see or talk to my sons doc, I could ask her if she could talk to this other doc? Are doctors part of a small community that they would communicate with each other?

I know his bone marrow biopsy was 20%, I don't know that any of the other numbers qualified him to be severe, his wbc was 0.8, PLT 37, I don't know his retic count. The docs are the ones who told us it was severe, I didn't ask what the criteria was. I will now though - Thanks!

As for no symptoms, thats where I got to wondering. If not for a set of coincidences, it seems we'd never know he had this illness. Or condition. I'm still not sure which it's called. He seems perfectly healthy!

No, I won't second guess his doctor; they know so much more than I do.

Thanks for your help,
edith

Originally Posted by Lisa V

I agree with the others that Dr. Maciejewski is one of the top AA specialists in the country. If we were anywhere within a couple hundred miles of him, we'd go see him. You could at least ask your doctor to consult with him (or Dr. Margolis or Dr. Young) if you don't feel confident in his judgement.

Having said that, any decision to treat must be based on a combination of counts and cellularity, not symptoms. Many patients are essentially symptomless. Severe AA is defined as having a bone marrow cellularity of less than 25% and at least two of the following: a neutrophil count less than half a billion per liter (<500/mm³), a platelet count les than 20 billion per liter (20,000/mm³), a reticulocyte count less than 20 billion per liter (<20,000/mm³). VERY severe AA is defined by a neutrophil count of less than 0.2 billion per liter (<200/mm³).

If your son meets these criteria, then your doctor is right to want to move quickly to treatment, either with a marrow transplant if he has a suitable sibling donor match, or with ATG if he doesn't. People's norms may vary slightly, but not that much. It's also important to note how quickly his counts are dropping in order to gauge how much time you have to think about things. Watch and wait is appropriate for Moderate AA (higher counts and percentages than those listed, but still below normal), but once you get to where you are needing to be transfused, that in itself carries risks and complications which only increase with time. You may find one or two SAA patients that have spontaneously improved, but statistically, untreated SAA will result in death 80% of the time. Whatever your feelings about western medicine (and I don't disagree that many agressive procedures could probably be avoided), this is not a good playing field to test them on. Just my opinion.

[Lisa V]

Originally Posted by edithr

Do you think the next time I see or talk to my sons doc, I could ask her if she could talk to this other doc? Are doctors part of a small community that they would communicate with each other?

Yes, I think that's an excellent idea, Edith.

[Hopeful]

Hi Edithr,

You haven't said the name of your doctor, but you can just google him and see where his area of expertise lie. Perhaps he is an AA expert, but if he isn't, it is worthwhile to have an AA expert on the side to consult with you and your doctor.

If your son has a sibling, and your doctor has not mentioned testing to see if he is an HLA match, that would be a red flag to me. If he has no sibling, then I'd be less concerned. I am assuming that your son is young.

[edithr]

My son's doctor is Dr. Connie Piccone (http://www.uhhospitals.org/Physician...4/Default.aspx), a pediatric hematology/oncologist, specializing in hematology and sickle cell disease. Rainbow is a teaching hospital with Case Western Reserve Medical School, so we see lots of docs. But she said she is the attending.

They tested his sister, she was not a match. I took that hard, but then realized we just have a different road to travel.

While waiting for the results of the match, I prayed and prayed for it to be so. But then I realized I should instead just pray for strength - strength for me to be helpful to him and especially strength for him to deal with this.

Fortunately teenage boys are not known for reading, and mine is definitely typical! Instead he just seems to take it one day at a time, which is probably a better way to do it.

Thanks for all your help, I know I'll have more questions.

[Lisa V]

Your doctor sounds well qualified to make a diagnosis and treatment decisions, Edith. You could still ask her to consult with Dr. Maciejewski, but if tests show that your son is SAA rather than MAA, and if his sister is not a match, I'm sure he would recommend ATG too. That is by the book.

Sometimes it's actually better not to have too much time to think about it. When my husband was diagnosed, his counts were so low by the time he started showing symptoms (retinal hemmorhaging) that they admitted him directly to the hospital. This was on a Sunday, he got his diagnosis on Monday, and started ATG on Wednesday, which was as soon as they could get the serum. We didn't know what hit us and had virtually no time to process it or weigh the options. We didn't even have a computer, so I couldn't go online and look it up, but by the time I was able to get more information, it was clear they had done the right thing. No regrets here.

I shudder to think what might have happened had his blurred vision not sent us to the ER. He had petichiae and bruising too, but hadn't paid much attention to it as he often gets bruised on the job. The ER doctor actually turned pale when she saw his blood test results. His platelets were at 8k and his hemoglobin at 6.4. She was shocked he was still walking around, but he felt fine!

[Hopeful]

I agree with Lisa. It sounds like you are in very good hands. Check out this AA paper by your doctor presented at the 2009 ASH:

http://ash.confex.com/ash/2009/webpr...aper23682.html

Studies show a more favorable outcome when there is a short time between diagnosis and treatment. So don't delay!

Everyone handles ATG differently. For me, the whole treatment was no worse than a bad flu, and I had headaches only while actually getting the ATG. I was back to easy biking and then running 2-3 weeks later. Hopefully, your son will have an easy time with it as well, and more importantly, a strong and durable response!

[edithr]

Thanks Hopeful for that link. Most of it is confusing to me at this point. Will I understand it much better in six months? My daughter is 2nd year biology major, maybe I'll ask her to translate.

I'm so glad to hear you were back to feeling great a few weeks after ATG.

Oh - docs called today they want him (and us) to participate in a research study affiliated with Childrens Hospital of Philly, then I see thats where Dr. Piccone was before she came to Rainbow.

[peter v. c.]

I replied to your e-mail a few days ago. I included my phone numbers both cell and home. I live in Amherst and yes I survived over 17 years with aplastic anemia. I wish Dr. Shahidi (sp) was still practicing. He specialized in pediatric conditions involving AA and other blood disorders. I seen him a few times myself when first diagnosed, even though, I was a patient of Clev. Clinic.f I don't hear from you hope to see you at AA/MDS in conference in Cleveland in June.

[pvinod]

Could you please tell me what is the lymphocytes counts before ATG and after 1 month of ATG?