Moderate aplastic anemia - seeking information

[cologne74]

Hi, i am 46-year-old male and was recently diagnosed with moderate aplastic anemia. The official protocol for this is "watch & wait" but since I cannot deal very well with uncertainty, I want to find out more about my outlooks and options in the meantime. Therefore, if you know something about the one or other question I have I would really apreciate your input.

1. How likely is it that the MAA stays stable vs. a steadily declines/gets worse? Do you know of any studies analyzing MAA development over time and what are the results? I somehow have the feeling that blood values will worsen over time and how our AA is classified only depends on when our AA is discovered so that untreated we will all end in the vSAA cluster , but I have also seen some reports here about stable situations. Are they the rare exceptions ?
2. Are there any studies analyzing whether it is better to "watch" and treat later vs. fighting the roots and starting treatment early? What is the potential impact of early vs late treatment with regards to remission and relapse?
3. Is permanent remission after IST at all possible and or is it always only temporarily (since one is not healed) but it differs how long it lasts for someone? Do you know of people with remission >20 years?

Any other insights about moderate AA are highly appreciated. Thank you once again for your help.

[David M]

Hello Cologne74,

You have asked some very good questions -- many I have often asked myself! I have had "Moderate" AA for several years. But, I also feel like when it comes to AA, this stability is the exception and not the rule. You have come to a good place for information! This forum is a great resource that will help you as you move forward and deal with AA.

In addition to reading the many helpful articles in this forum, I invite you to go to the "My Story" section of this forum and read my entry: "Slow-moving AA / Pancytopenia, or What?" This thread describes my MAA journey which started almost 20 years ago (I still update it from time to time), and it also will introduce you to a few others who are on (or who have been on) this forum with similar symptoms.

If I can ever be of any help to you, feel free to contact me!

David M

[cologne74]

Hi David,
thank you for your reply and the nice words. Let me tell you that I have already read your story before I opened this thread and it lead me to some of the questions i have raised. it was a very interesting read!

[David M]

Cologne74,

As my hematologist said in my last visit, all treatment options have risks and side effects. You have to ask yourself if it is worth the risk for the potential improvement you might see? Or if you have reasonably high counts and are stable and have a good quality of life, is it better to continue on "as is" and avoid that risk until absolutely necessary?

For me, as we discussed the various treatment options available and their associated risks, it was obvious that to continue "watch and wait" is the best option in my case. I have been pretty stable for 10 years, and although my counts are low, and although my stamina has certainly been diminished, other than that things are pretty good. All available medications, transfusions, transplant options, etc. would introduce unnecessary risk. But this is my situation, not necessarily yours (or anyone else's).

In your case, I suppose it also depends on where your counts are -- and what kind of complications you are suffering from your pancytopenia. It all gets back to risk vs. reward, and if it is reasonable to take risks at this point in your case.

There are worse things than living in "watch and wait" mode!

[cologne74]

Originally Posted by David M

Cologne74,

As my hematologist said in my last visit, all treatment options have risks and side effects. You have to ask yourself if it is worth the risk for the potential improvement you might see? Or if you have reasonably high counts and are stable and have a good quality of life, is it better to continue on "as is" and avoid that risk until absolutely necessary?

For me, as we discussed the various treatment options available and their associated risks, it was obvious that to continue "watch and wait" is the best option in my case. I have been pretty stable for 10 years, and although my counts are low, and although my stamina has certainly been diminished, other than that things are pretty good. All available medications, transfusions, transplant options, etc. would introduce unnecessary risk. But this is my situation, not necessarily yours (or anyone else's).

In your case, I suppose it also depends on where your counts are -- and what kind of complications you are suffering from your pancytopenia. It all gets back to risk vs. reward, and if it is reasonable to take risks at this point in your case.

There are worse things than living in "watch and wait" mode!

Hi David,

Yes and no. Of course watch and wait is a good option if symptoms of pancytopenia are acceptable. But maybe I miss a good chance to get a remission or healing now that I will not have again if things get worse in 10 years. BMT for example is much more successfull in younger years and I can imagine that the side effects of IST are also more bearable when you are relatively fit. So if I know that things will get worse, it might be better to act sooner rather than later. Now I know nobody can tell me what the future will bring and there might also research advances in the next 10 years, but i am interested to get as much information as possible to make the best decision for myself.

[Hopeful]

Hi cologne74,

You ask some very relevant questions. I can offer some insights, based on my experience (10 years post IST )

I am a little short on time for the next couple of days, but wanted to let you know that I plan on responding.

What are your RBC, platelet, and ANC counts?

[GoodDay5150]

Hey Cologne74, I have never been to Europe but it is on my list. If you search my user name you will find many stories regarding my diagnosis, treatment, and recovery after a stem cell transplant for PNH. Obviously diff. than AA, but similar in many respects. A lot of patients develop PNH after having AA and some people have both at the same time.
The US health care system is diff than yours so I had dr's from my insurance company that work in conjunction w/ the specialty clinic that is affiliated w/ a hospital bcse neither that company, and my insurance company I have now does the actual transplant. Anyway, it was decided that bcse of my severe symptoms and my age that I was a good candidate for a transplant. That decision was based on the opinions of multiple dr's and the protocol used was partially developed by dr's at the specialty clinic that I go to. The drug used to treat PNH does not cure the disease, and I was never administered that drug b4 the actual procedure.
This is all scary stuff I know and different medical professionals have different opinions. I would ask a lot of questions and you can also get info from the AA/MDS organization. https://www.aamds.org/

we all wish you luck in your treatment



Mario

[Hopeful]

Hi cologne74,

Here are some thoughts on your questions. I'm not in the medical field, so take them with a grain of salt!

Originally Posted by cologne74

1. How likely is it that the MAA stays stable vs. a steadily declines/gets worse?

Sometimes the cause of MAA can be a virus, medication, or chemical. One my hematologists said that he will get 10+ patients a month referred to him for possible AA evaluation because of one of these causes.

If the cause of your suppressed bone marrow and counts is related to this, sometimes the marrow can heal itself when the offending agent is removed. It can take months for the healing to happen though. In case this relates to you, take a hard look at your diet and every medication that you are taking and try to eliminate any potential toxin. This includes seemingly benign things like tea, alcohol, and over-the-counter medications.

Assuming that your counts have been low or are lowering for 4+ months, then something else may be going. The question is whether it is immune mediated or genetic.

So MAA can get better, or stay stable, or get worse - it all depends on the originating cause, how quickly that cause is removed (if possible) and how strong enough your marrow is to recover.

Originally Posted by cologne74

Do you know of any studies analyzing MAA development over time and what are the results? I somehow have the feeling that blood values will worsen over time and how our AA is classified only depends on when our AA is discovered so that untreated we will all end in the vSAA cluster , but I have also seen some reports here about stable situations. Are they the rare exceptions ?

Some people are lucky and have symptoms that mimic MAA (like a bad virus or vitamin/mineral deficiency). Since AA is rare, yet my local hematologist sees so many potential AA patients a month, I would think that this is the most typical scenario. In other words, it may look like you are developing AA but you are not. This is one of the reasons for watch-and-wait.

The classification of AA depends on your bone marrow cellularity and counts. You can't have an AA diagnosis without a BMB.

MAA may not progress if the marrow is able to overcome the immune attack and heal.

Originally Posted by cologne74

2. Are there any studies analyzing whether it is better to "watch" and treat later vs. fighting the roots and starting treatment early? What is the potential impact of early vs late treatment with regards to remission and relapse?

I do remember reading a paper years ago that MAA patients can have a worse outcome then SAA patients if their disease progresses because success can be related to how quickly an immune attack is stopped. I was worried about this case for myself, as I was in watch-and-wait mode when first diagnosed because I didn't fit the complete diagnosis of SAA despite being transfusion dependent. I think that if you are categorized as MAA but become transfusion dependent, then things are obviously progressing in the wrong direction and treatment should be started quickly to prevent further damage to stem cells. This is just my opinion based on my own experience.

Since none of the treatments for AA are benign and people can die from them, doctors don't want to initiate them early ("first do no harm"). Besides, with IST, having a partial response is considered a great thing, even though your counts may be shockingly low to the normal person. So, if you are already in a "stable" state, even with below normal counts, it doesn't seem like you would want to rush treatment and risk death or other long term consequences. You can live a great life despite having below normal counts, assuming things are stable.

Originally Posted by cologne74

3. Is permanent remission after IST at all possible and or is it always only temporarily (since one is not healed) but it differs how long it lasts for someone? Do you know of people with remission >20 years?

Any other insights about moderate AA are highly appreciated. Thank you once again for your help.

I have always heard that IST is not a cure. That being said, I know of people that have been stable for 15 years. I have watched presentations in the past where some of the leading hematologists have said some of their patients are stable for 25 years after IST. If your AA is because of an immune attack, and IST stops that immune attack, and your marrow is strong enough to heal, you could very well be a long term surviver.

Wishing you the best of luck!

[MelbourneAA]

Hi from Australia,
I am 43 years old and have NSAA. I was diagnosed in March 2019. I have not needed transfusions and other than feeling a bit tired from time to time, I can cope with all that standard working and family life. I swim 1.5-2km once to twice a week. The first 3 months were the hardest, chasing information and worrying so much. I am writing this to provide background for you and any other patients who have NSAA and are wondering how others have managed this.

Question 1:
I have only found 1 study of moderate/non-severe AA in Adults from Korea. I can email it to you if you like.
AA is very rare. There are very few studies on this. Most of the studies are on SAA or VSAA. No one knows whether your bloods will decline quickly, decline slowly, plateau, or improve. Most haematologists will use the rule of thumb that a third will stay stable, a third will improve and a third will progress to SAA. Which is another way for saying they don't know what will happen with your individual case.


Question 2:
Are there any studies analyzing whether it is better to "watch" and treat later vs. fighting the roots and starting treatment early? What is the potential impact of early vs late treatment with regards to remission and relapse?

I could not find any studies analysing treatment options for NSAA.

The treatments are (1) supportive (blood transfusion, antibiotics, etc) and (2) BMT, and (3) immunosuppressive therapy which can be intensive with ATG/Cyclosporin, or non-intensive with Cyclosporin only.
Even low dose cyclosporin can have serious side effects, eg fatigue, hypertension, real impairment, etc. Steroids can cause Diabetes, avascular necrosis of hip needing hip replacement etc.
The side effects can be worse than the disease, if you are in the NSAA category.
BMT is really a treatment of last resort, as once you go down this path, you cannot go back. This is why it is reserved for SAA and VSAA.


Question 3:
Is permanent remission after IST at all possible and or is it always only temporarily (since one is not healed) but it differs how long it lasts for someone? Do you know of people with remission >20 years?

Permanent remission after IST is possible. Sometimes the remission is partial. Sometimes a second or third course of IST is required.

I have fortunately not required IST and also in watch and wait category. My counts have been stable since March and my haematologist thinks that once I have stable counts for a year, then it is likely that they will stay this way long term. I have my bloods taken every 8 weeks. Hb 104-114, WCC 3.5-3.7, Plt 55-60. All these numbers are lower than the normal range, but still ok for normal exercise, fighting off infections and not bleeding/bruising. These numbers are what people with VSAA or SAA hope for when they are treated with IST.

My haematologist says he has another patient like me whom he has been observing for 12 years.

At this stage do you need transfusions? To answer the question if your blood cells are going up or down needs regular blood tests to observe the pattern.

No one knows what the future holds. After a few months following your diagnosis, you should limit your time to researching this illness to only an hour or so every day, otherwise you can get bogged down in obsessing over this and that, what if, what then . . . Do your reading in the morning or early part of the day. If you research this late in the day, then you will obsess over it and think more and more.

Best wishes from Melbourne