Relapse SAA

[Kim2007]

I was diagnosed with SAA about six years ago and I did very well with ATG. For the past year or so my counts have been slowly creeping down. I had a bmb on 8/14 and I have relapsed. I was sent to see Dr. Venu at Rush in Chicago because my old specialist died. I am having the bone marrow slides sent to him and they are talking about putting me back on the cyclo for a bit to see if that makes a difference but I also have PNH so he wants to see if that has mutated before we make that decision because it can change treatment plan. Has anyone relapsed after ATG who also has PNH? I have a matching sibling if I need transplant but the new doc says transplant should be last option. My old doc leaned more towards just curing it. During this I am also going through a divorce after 16 years. Started having issues when I got sick. It was on hard both of us but I do get my insurance through my husbands job right now.

[NLJabbari]

Hi Kim, I am the parent of a 19 y-o son who also relapsed. He was dx'ed in 2004, 1 round of ATG good response and then a relapse. He was put back on Cyclosporine and responded, but once again relapsed (May 2012) when the weening was in process. He was also dx'ed with a PNH clone in 2012.

Recently, His Hematologist sent us to see a BMT Dr. at Stanford, but the BMT Dr. said that he believes there are other "options" to explore before BMT. He highly suggested we make an appointment to see Dr. Neal Young at the NIH. to see what he recommends.
I had called the NIH a couple of weeks ago to request an appointment. They asked me for specific info (a Summary of my sons Medical History) with specific reports etc...so that they could make an "evaluation" prior to deciding if Dr. Young would see him or not. It took me 2 weeks to gather all of the medical records requested etc...lots of calling back and forth with the medical secretary responsible for collecting all of this info. that dates back to 2004.

Earlier today, I decided to follow-up to see if they had received the faxed documents and I was told that the NIH had received all of the documents. They, (Olga Rios) said that Dr. Young had been out of the office all of last week and therefore had barely gotten them early in the day.
I was confused bc He had barely gotten back today and yet, the message was, that he would NOT be able to see my son and that my son should continue Cyclosporine! DUH!!!
I could have told you that myself...!!! No explanation as to "why" was given. Needless to say, I felt like I had been punched in the stomach and the door slammed in my face! I am very disappointed with this whole treatment. I feel that Dr. Young most likely didn't even look at my son's med info. I'd at least like to know WHY!?

Anyway, the BMT doctor believes that since my son's WBC and Platelets are within "Normal" range, but his Hgb is low, but transfusion free. He believes we should be treating for PNH. He also suggested my son do some Chelation (SQUID) Children's Oakland to maximize organ function? We'll be meeting with his hematologist soon to discuss "Other Options"

Please let me know what your specialist recommends for you.

Thanks and take care

[Sally C]

Hi NLJabbari,
I think what I'm telling you is correct and I hope it will make you feel better.
My husband has been a patient at NIH since the spring of 2009. He has participated in several clinical trials. In my opinion they saved his life.
NIH is where trials are performed for drugs not yet available to the public to see whether or not they do what they are supposed to do to ultimately get FDA approval for the general public.
Every clinical trial has a very firm protocol that NIH has to follow. The protocol is set by the pharmaceutical companies and I believe also a board at NIH. There are aspects of diseases that have to fit the requirements of the protocol as well as medical conditions that also can exclude a patient from qualifying. Sometimes things such as age can be a factor (probably not in your son's case). Co-morbidities and various other things such as previous treatments, etc. can exclude a patient from being accepted as well. NIH is not allowed to go outside the protocol without it being approved by a board. It is a drawn out process and I don't think it's an easy thing to get accomplished. Once a patient is accepted, all doctor visits, treatments and drugs are free to the patient because they are clinical trials.
My guess is that Dr. Young was able to look at your son's records and decide very quickly that your son didn't match any criteria for any clinical trials - or there wasn't a trial ongoing that your son qualified for. Olga Rios who I know personally, probably knew this before your records hit Dr. Young's desk. The protocols and the trials that are ongoing - what is accepted and what is not - are pretty easy to understand should you want to go to their web site and look them up.
To my knowledge Dr. Young does not take patients for consultation without the possibility of their qualifying for a clinical trial. Dr. Young's recommendation was for your son to stay on Cyclosporine. Dr. Young is considered the #1 hematologist in the world so his recommendation might be good advice for your son. I don't think Dr. Young would have just thrown out a recommendation without knowledge of your son's case.
Your post sounded like you took how it was handled personally and I'll admit they could have told you why he wasn't accepted at NIH. At the same time, I have very close up and personal knowledge of the people in the Hematology Dept. at NIH and they are some of the most caring and efficient people I have ever met in the medical field - and they are also very very busy.
I hope this helps. And if anyone reads this and I'm wrong about anything I said, please correct for the record. I don't want to give out any misinformation.
I wish you and your son well.
God Bless,
Sally

[NLJabbari]

Thank you Sally, I appreciate your reply and that's exactly why I was stunned! We too have been dealing with this since 2004 so obviously we're not new to the whole situation. I have done extensive research (Binders) and am very well aware of how the NIH does things. I have followed and read every paper put out by Dr. Young. I too agree that he is the utmost authority when it comes to these blood disorders and why I was stunned with his simple reply. I knew going into this process that there would be a chance that he wouldn't be seeing my son, but I was expecting to at least be told why and perhaps given a recommendation other than "Continue on CsA". My son is already on and has been taking CsA for years mind you. So, that really went without saying; I don't consider that a recommendation.

It is precisely because of a protocol that Dr. Townsely had mentioned (dealing with Telemerse length) at the San Francisco AAMDS conference that we decided to initially contact NIH. Actually, there are two Clinical Trials that I thought my son "might have qualified for", one in particular caught my attention. But regardless, we're still trying to decide between a couple of treatments and BMT. Like I said before, my son's Hematologist and BMT Dr. are not in agreement and therefore are recommending different approaches. I was hoping Dr. Young could have put in his expert advice and shed some light on this. As for us, we're still stuck in the middle...I think it's going to have to come to "Us" making the final decision as to what our next step will be.

I am happy for you and that your husband is getting the care he needs. I was told recently that a patient on "Clinical Trials" is probably getting the best care possible as every minute detail has to be reported. So, that's a good thing.

We'll just keep trying and hopefully will come out with the best plan of action. I know that it sounds like I took it personally, but I actually did We've been fighting for so long and I'm not about to give up now.

Thanks again and God bless

[Whizbang]

Kim, forgive me if I'm being too forward, and my decisions shouldn't be yours (I don't fully know your situation, IPSS, etc.)... I am very conservative by nature, but I also like to analyze odds... And given our pre-existing conditions, Health insurance is a life or death sentence (given the cost of treatment / BMT)...

The fact that you may lose coverage (sooner rather than later), and the fact that you have a matching sibling, (not knowing your age) but I assume you are young enough to be a candidate for BMT...

I'm wondering why your not considering a BMT as a cure more seriously?

Again, please forgive me if I'm out of line...

Originally Posted by Kim2007

...I have a matching sibling if I need transplant but the new doc says transplant should be last option....During this I am also going through a divorce after 16 years. Started having issues when I got sick. It was on hard both of us but I do get my insurance through my husbands job right now.

[Sally C]

Hey again NLJabbari,
After reading your post I totally understand why you felt the way you did regarding NIH. Your are obviously a wonderful Dad who is going to find a way to help his son.
We do have a man on the forums who is in that clinical trial for telomeres - Greg H. I am going to send you an e-mail with a post of his. He is going to a different doctor at NIH - that could be why you didn't get the response you expected. I would have thought your case would have been passed to the right place but then again - they are extremely busy - but that doesn't help your son does it? Maybe if you try to contact Greg H or Greg's doctor he might be able to help.
I wish you well and will send you a separate e-mail.
God Bless,
Sally

[NLJabbari]

Thank you very much Sally, I did indeed receive the email and found it to be very interesting and informative. I'm regrouping and thinking of how I should approach this now. I just have to catch my breath!

BTW, I'm a SHE...a mama bear

Thanks again

[Sally C]

You are so welcome, Mama Bear (sorry about that - hard to tell with your name). Please keep me informed and let me know if there is anything I can do to help.
God Bless,
Sally

[TonyBegg]

Kim - I saw the bit about divorce and worries about insurance. When Karin and I separated we stayed married for a while so she could stay on my insurance, but she thought it unfair on her boyfriend so we divorced. She has had PNH for 23 years but on July 5th she needed RBC and platelet transfusions so looking like associated AA (neutrophils 0.75 x 10^9 / Liter so moderate AA). We considered re-marrying so she could be back on my insurance, but there is a pre-existing condition exclusion period of at least 6 months, and we discovered that the Affordable Care Act (unless the politicians delay it) will allow pre-existing conditions with no exclusion period starting Jan 1st 2014 so we were saved from another marriage :-). They will have Bronze to Platimum health care plans if you earn less than 400% of the Federal Poverty Level ($16000 for a single person), and Federal Assistance if between 100 and 400%. Luckily the local hospital (with help from the hematologists promoting her case) put her on a charity program that paid her recent blood transfusion costs and lasts for a year. Anyway, I wish you luck.

[BobbyJD]

Kim, my sister too has PNH clone with AA and has relapsed recently. She just started CsA last week and in her most recent blood test platelets dropped a bit again. I hope the medication will begin to kick in soon and stop the downward trend.

Important things to note are, make sure the Dr is definite on whether you are primarily PNH or AA because the treatment is very different. Seems like standard treatment after relapse of AA is back on CsA without ATG. Looks like most people have a response to this. **Out of curiosity anyone out there take CsA after relapse, how long did it take for counts to begin trending upward??

As for BMT, our Dr always says that should be the last resort and he is a specialist in AA. However if your young and have a sibling match, it is worth researching more. But usually if your transfusion free and medication is helping keep counts stable, your in good shape!