Need Help

[Missy D.]

I'm new to the forum and so far have found it very helpful but really need some help with my dad. Quick history: dx with AA in September. Started on cyclosporine right away. Has been getting blood and platelet transfusions once a week since June. Then magnesium once he started cyc. Has had problems handling cyclosporine. Extremely tired(only awake a few hours a day), confusion and not eating. Starting weight in June-132. Yesterday-112. Kidney levels also have gone up. Numbers as of yesterday- WBC-1.1, RBC-2.71, hgb-8, hct-24, ANC-.50 and platelets 24( transfused 4days prior).

History on my dad: my dad is 66. At 35 he had a heart attack and massive stroke. At 48 he had quadruple bypass surgery. Has a seizure disorder and used to be on dilantin and is now on Keppra.

Here's my problem. Yesterday my dad saw his doc and the doc believes he should stop the cyclosporine. He said at a high dose that seems to help(saw a slight increase in his numbers the last few weeks) my dad doesn't tolerate it. At a lower dose the doc feels it's not doing any good. He's been on it for 6 weeks.
He doesn't want to do atg as he believes the side effects will be too hard on him. He also doesn't want to do promacta either for the same reason. His suggestion is to stop treatment and do supportive care only. He would then release him to his old hematologist. The doc he's seeing now was who is other hematologist referred him to when he couldn't diagnose him. We feel like he's giving up on my dad. His doc now is supposed to be the best around my parents( Levine cancer center, Charlotte, NC.)
So here's my questions:

I understand the reason for not doing atg but not sure why not promacta? Are the side effects as bad as atg?

If my dad isnt getting any treatment then what's his prognosis? How quickly does AA progress?

He had a BMB in July do u think it would be good to do another?

Do we need to give the cyclosporine longer time at the lower dose that my dad can tolerate? Are we stopping too soon?

They want to put a pic line in as it's getting very hard for them to find a vein. Thoughts on a pic line over a port?

I would like to get another opinion by my mom is hesitant to do it. I live very close to Duke and would like to have him seen there. What do u think? Is it worth it? I'm 3 hours from my parents so I'm not sure how easy this would be for my them.

I'm really at a loss and just don't know what to do. As of now my dad is staying on cyclosporine at 400mg/day. They meet with the doc again on the 25th, which at that point if my dad's numbers haven't improved then he wants to take him off cyc and put in pic line. Any help would be GREATLY appreciated and just can't say how thankful I am for this forum.

[Amz904]

Hi Missy,

Sorry to hear about your father! Im going to try to answer as much as I can from my personal experience. A quick note about me: i'm 22 and was diagnosed with VSAA Dec 2013 (ANC: 0.02, WBC: "less than 0.1") i had ATG Jan 2014 and was transfusion dependent until June 2014. Currently, my white count and hemoglobin are normal, platelets are still a little low hovering around 60) I also have been on Promacta for about 7 months.

Heres my experience with cyclosporine (for reference I'm around 120 pounds), I initially was on 150mg/day (which made my cyclosporine trough level around 150) but my creatinine began to slowly rise and after about three months my creatinine was 1.85. At that point my doctor reduced my cyclosporine to 100mg/day and my creatinine dropped to around 1.4 after about two weeks. My blood counts didn't show any real decline or anything, so he then dropped it down again to 50mg/day and I've been on that dose since May, and my creatinine has been back to normal and stays around 0.7. Decreasing my cyclosporine was pretty scary, since most experts say a high dose is necessary and that its better to let your kidneys suffer a bit. But my doctor seemed very confident in his decision and I was getting my blood checked twice a week, so i was being very closely monitored. His view on it was that in transplant situations its absolutely necessary to stay on high doses of cyclosporine and you can't lower it, however with AA, his patients have responded well with a lot lower doses. So i've been on 50mg/day since May and it's been working great for me (and for my kidneys!) Also at that time, I was possibly considering getting a bone marrow transplant, which also contributed to my doctor lowering my cyclosporine levels since having kidney failure would compromise the chance of a bone marrow transplant.

In regards to picc line vs port, I had a picc line in my chest. My first picc line was put in for my ATG treatment in January, but was removed after about a week due a blood infection. Its unsure whether it was due to an infected picc line or not, but protocol was to remove it. A month later I got another picc line to help with blood draws/transfusions and i had it for about 4 months until I stopped needing transfusions. It required weekly dressing changes and was SUPER difficult to shower in, and just a hassle in general. I honestly only chose a picc line over a port, because i was worried about the scaring a port would leave behind (i know, that should have been the least of my worries haha) but because of that i opted to deal with annoyance of a picc line. I also lived with my family, so it was easy to make my sister change my picc line dressing and inspect it whenever I needed so that aspect was easy. Although it was annoying to have, it made getting my blood drawn SO much easier, which is why i after my first picc line got removed, i still got another one put in. The month in between picc lines i remember dreading getting my blood drawn since they would poke me a million times, so a picc line or port definitely makes life easier when your transfusion dependent!!

One more thing to add about picc vs port, I noticed i began feeling a lot more active once my picc line was removed, obviously since my blood counts were doing better, but also because without it i could move around easier. i could actually try working out a little without the fear that my picc line dressing would come loose or move around too much. So since you mentioned your father is already so fatigued, maybe a port would be better so on days when he does muster up some energy he would be more likely to try walking around more if he had a port rather than a picc in my opinion.

In regards to promacta/atg side effects, I didn't have any real side effects from either. I had my atg transfused super slow, which may have helped. (I think most people get it over 4 days for 7-10 hours a day, i got mine over 4 days but for 20 hours each day) so it was way slower which may have helped limit the side effects. Im not sure which side effects in particular your doctors referring to with promacta, but like with any medicine its about weighing the pros and cons.

With the whole BMB thing, I think the experts recommend getting one done every 3 or 6 months for the first two years or something. However I only had one done back in December 2013 when I was diagnosed, in my doctors opinion he said my blood counts are a pretty good indicator of whats happening and because of my low platelets he didn't think it was completely necessary to have to go through one since I'm being monitored so closely with all my blood counts. I guess it all comes down to personal opinion, i was okay with not getting another one (after hearing some horror stories of biopsies going wrong haha) so I didn't push for it, but if it'll make your dad and you more comfortable to get another one then don't hesitate to ask your doctor!

Sorry this was so long haha, but i hope it helped

[Missy D.]

Thank you Amz940 for responding. You were very helpful! I've been researching everything I can on AA but it's so nice to hear from people about their own experiences. It sounds like a lower dose of cyc might be helpful. I just can't imagine doing no treatment with his numbers that continue to fall. He's back at the doc today to get his bloodwork done and I'll be curious to see what they are.

On a personal note, my dad retired last year and it's so disappointing that this is how he's spending it. With all of my dad's medical issues his whole life he has worked very hard to provide for his family and I was really looking forward to seeing him enjoy his time to relax not spend so much time at the docs and now sleeping all the time. I just have to chalk it up to that life just isn't always fair.

[dfantle]

Missy, i'm so sorry for what your dad's going through and how it affects you and your family.

I do think exploring staying on a lower dose of cyclosporine might be worth while but it, like many drugs, is very hard on the kidneys. For anyone who has pre-existing kidney issues, especially so.

If you haven't already, I might suggest you have a real heart to heart with your Dad and ask what his wishes are & then proceed by fully following his wishes. After what I've been through, it was so reassuring to me that my family allowed me to make the choices with regards to my health care & treatments. Although it's difficult sometimes to do this, the patient is the one who I think should make those choices especially when things get tough...

Best wishes

[clairekim]

Missy,

I've been meaning to ask this. Was the AA diagnosed after your father started taking Keppra? or did he change from Dilantin after this diagnosis?

I've heard stories of people who rely on supportive care for many, many years. Recently, I came across a man who's been on the supportive care for more than 10 years before going through BMT with his daughter's donation after she reached the proper age. I guess it all depends on how severe the problem is. If my wife (very severe, waiting for ATG response in Oct) would only need a transfusion once every several months, I'd seriously consider keeping her in supportive care than to go through the treatments. Along with treatments comes other problems.... but that's just me whose wife has gone through 2 rounds of ATG + Cyclo + Promecta (plus Keppra for her seizures). We are just sick of these medications......

We will keep your father in our prayer. He will be fine.

[Missy D.]

My dad was on Keppra once he was dx but it took several months and 2 doctors to get the dx. The doctors do believe the dilantin is what caused the AA.

Thank you so much for your kind words and prayers for my dad and we are sending prayers your way also! Honestly the thought of supportive care just scares me. I've seen such a decline in my dad since the beginning of all of this in June that I guess I was just hoping for more.

[clairekim]

By the way, if it so happens that your father need to stay in supportive care, I recommend that someone in the family with same blood type to HLA typed for platelet match. What will likely happen is that his days between transfusion will get shorter and shorter due to build up of antibodies. But if he can find someone who is platelet HLA match, transfusions will last longer. This is what I am trying to do for my wife.

Anyone can donate platelet once every 10 days.

Good luck,
David Kim

[Missy D.]

Since I last posted my dad has seen his doctor again, along with my mom and my sister who is a ICU nurse. Got a lot of answers and hopefully a new plan. He also had a pic line put in. The doc did say my dad is SAA not AA, which is what I thought but never heard it from the doc. He still thinks we shouldn't do the atg, which we all agree but would like to try promacta. He's still on cyc at a dose of 300mg/day which he is tolerating a lot better. The doc wants to finish out the cyc, about 2 weeks left and then start promacta. My dad's latest CBC was: ANC .70, WBC 1.3, hgb 8.3, RBC 2.87 and platelets 12(transfused 4 days prior). So I have a few more questions.

Since my dad has started cyc he has been able to skip a week twice without getting blood. His hgb is dropping but at a slower rate. Does this mean cyc is starting to work and we need to give it more time before starting promacta.

With the promacta, my dad's insurance won't cover all the cost. They will have to pay $2400 a month. We are looking at trying to get some assistance since my parents couldn't do that long term. Anyone have any suggestions on what I should look up? Any trials?

Again thank you very much for any advise!

[Hopeful]

I think you should give cyclosporine more time. It takes a couple of months for it to build up in your system. Cyclosporine isn't as strong as ATG, yet people usually have to wait 3-9 months for a response to ATG. So, it seems like you should give Cyclosporine at least that amount of time to work. The fact that the length of time between transfusions increased is a very positive sign, in my opinion. I wouldn't want to lose that by stopping the drug prematurely, if he is tolerating it well. I am not a medical expert. These are just my thoughts.

Something also to consider is ATG given at a lower/slower dosage over 10 days. I remember reading a paper about this in the past as an option for older patients. I will try to dig it up....

[Missy D.]

Thank u so much for the response Hopeful! I feel the same and think we should stay on the cyclosporine longer. The doc doesn't think it's working. He goes today to get his CBC and transfusions. It's been a week since his last so I'm curious on what it will show. Fingers crossed. The doc doesn't think my dad could handle atg even at a lower dose.

[Missy D.]

We'll my dad got his latest CBC on Tuesday and again he was able to skip a week with getting blood and only needed to get platelets. Third time this happened since starting cyc. Although his kidney levels were up again which is why the doc doesn't want to continue with it. He wants him to start promacta at the first of the year. The doc doesn't have a lot of experience using promacta for SAA. So I have a couple of questions.

What dose do they typically start with and how long does it take to get to max dose? What is the max dose? Side effects?

Good news is with the help of the doc and pharmacist they are able to get the cost down and make it very affordable for my parents!

[Marlene]

http://www.onclive.com/web-exclusive...plastic-Anemia

[Missy D.]

Thank You Marlene!!!!!

[Hopeful]

Hi Missy,

Are you sure that your dad is being treated by an expert in AA? I would encourage you to have his doctor contact Neal Young at NIH to discuss his treatment options (continued Cyclosporine vs trying Eltrombopag outside of a clinical trial). I think someone else on this forum was able to get dosing information for Eltrombopag from the experts at NIH.

Make sure your dad is drinking LOTS of water while taking high dosages of cyclosporine.

Here are some numbers for the contact nurses at NIH:
Barbara Weinstein = 301-594-4180
Olga Riof = 301-496-4462