About 4 months out from my mom's aplastic anemia diagnosis...

[Matthew42]

Dear mola-tecta,

I am so happy that your mother's blood has held. That is fabulous!

I wasn't ignoring you, by the way. I am very sorry if you thought otherwise.

Every blood draw for my mother has turned into a life-or-death feeling for me. Even though her neutrophils are staying up, I just don't like her living transfusion-dependent this long. If her neutrophils have come up in the past few months with occasional rises in blood, I can't see how the ATG is not working.

@ Marlene: That is amazing that John is still doing well. You don't need high levels of anything to live a normal life. There is a woman from India whose hemoglobin has always been between 8-9 (not aplastic anemia, but some iron deficiency issue), and she lives a normal life, although she won't be running a marathon. Interestingly, she can work and do housework.


I wish you all the best for health and happiness.

[Sally C]

Mola-Tecta and Matthew,
The body is an amazingly adaptable machine. Our hematologist said she has patients who live with zero platelets. Obviously with care but they are able to live with it.
All the best to you both and your loved ones.
God Bless,
Sally

[Matthew42]

God bless you, too, Sally!

I'm just grateful that my mother's neutrophils are between 800-1200.

Yes, the body adapts well to very low platelets and lower hemoglobin. My older mother can do a lot with a hemoglobin of 6.5. It's really amazing.

At the beginning, my mother got terrible bruises, blood blisters in mouth, etc. with very low platelets. Now, she only gets a tiny bit of blood out of her nose (only if she blows it hard enough). Amazing!

Please take good care!

[mola-tecta]

Thank you. As of the second weekly blood test, both her hemoglobin and platelets have stayed steady. Platelets at 27, hemoglobin at 8.2. I'm amazed. It's been 5 weeks since the last blood transfusion and now two weeks without a platelet transfusion.

Cautious feelings of hope. I have to remember that things can still fluctuate as they would even in a person without blood disease. If more transfusions are needed, they do not necessarily mean failure. Things like pentamidine could make it fluctuate. She is scheduled for a new bone marrow biopsy in a week and a half. I suppose I will never know if it's possible the ATG has finally taken over, or if lowering the medication actually helped.

I'm sorry for being overly emotional in my last posts. I know I need to contain myself better. Things are too much.

Take care all.

[Matthew42]

That's really good news, mola-tecta. I think your mom is on the road to recovery.

ATG can take well over a year to work. I've read of people who were on transfusions for two years before ATG finally kicked in. Yes, it's true. They're glad that didn't resort to another treatment in the meantime.

My mother is not doing that well. Her platelets are back up (1 platelet transfusion a week, as opposed to two)), and blood is about the same (2-4 rbc transfusions a month). Her neutrophils are down to 700 from 800-1000, but I am not worried about that yet, as she went down to 600 and back up to 1000 in a week. I don't know what's going on. Every so many weeks, she goes into a different "trend". It's all really weird. I read that different trends all the time could signify the body evolving from the atg. Who knows?

It's all very difficult, but you just have to forge ahead. I cry just about every day. It's all very exhausting.

Update: My mother's hematologist said that Dr. Young has recommended a new course of treatment. I don't think it's rabbit ATG, but something like Rapamycin, or something like that. I think he told my mother's doctor that he wants to "boost" the horse-ATG. I'm not sure. My mother's doctor said she is going to do exactly what Dr. Young tells her to do. He will be calling the shots, and will consult my mother by phone or internet, if necessary.

I wish your mother all the best.

Take good care of yourself.

[Matthew42]

I think Dr. Young is going to prescribe Rapamycin to bolster my mother's horse-atg treatment (according to phone conversation my father had with hematologist). She wouldn't give that much info over the phone. I don't think there was any talk about rabbit-serum being used.

It is also true that my mother's aplastic anemia is very much immune-mediated from the PNH clone (confirmed again by the doctors). So, I ask: can aplastic anemia not be immune-mediated? I am very confused.

Take good care, everyone!

[Marlene]

Glad that you are making progress on getting some guidance from NIH.
John has never had any PNH clone. My guess would be that SAA is not 100% immune-mediated. It can be caused by high levels of radiation exposure, drugs and toxic chemicals like benzene. So I guess not all would be immune-mediated. I haven't kept up with all the research like I used to. Hope to never have to go down that road again.

[Matthew42]

Thank you so much, dear Marlene.

We're seeing my mother's hematologist next week regarding the new treatment. I believe it's Rapaymycin/Rapamune added to the cyclosporine. Dr. Young's team has done clinical trials with this drug where it's been added to horse-atg/cyclosporine treatment if a person doesn't get off transfusions by month 9 or so. I don't think Dr. Young wants my mother to do the rabbit-alg now. All that said above, it might not be Rapamycin/Rapamune, but from what I gathered from research, I bet it is.

At any rate, platelets are holding a tiny bit better than the past month, and she went almost 2 weeks on one unit of blood. That's really good for her. Normally she needs transfused by day 8 or 9. This time, she stayed at 8.5.-8.7 for well over a week. Unfortunately, all of sudden, her blood dropped down in the low 7's from 8.5. It's just bizarre how her body works. Very strangely, she also gets spontaneous rises where she will go from 7.5 to 10.3 (not transfused-induced rises) and then drop back to 7.0.

Her neutrophils are still holding around 1000, which is really good. ATG did something to make her neutrophils stay up for the past 3 months or so.

I wish everyone happiness and the best of health.

[Matthew42]

My mother's blood is holding better (still gets blood about 3 times a month) but her platelets are still very low. She doesn't have platelet refractory, however. Her neutrophils jumped up to 1200 and then back to 600, then back up to 1000? It's all really bizarre. He neutrophil average is about 1000 for the past two months.

We're going to discuss Dr. Young's treatment for her on Thursday with her current hematologist (Dr. Young is going through my mother's current hematologist to treat her). I am afraid I was wrong about Rapamune for her treatment (after some research): I believe it's a new drug for severe aplastic anemia called N-plate.

Dr. Young gives way better information than my mother's hematologist. Her doctor never made much ado about her neutrophils going up to 1200 and averaging around 1000 for the past few months. He said that is indeed proof that my mother has responded to ATG/cyclosporine (immuno-suppressant therapy). But she needs something "to boost her over" without going so far as to give her the rabbit-alg. That, I believe, is N-plate (used mainly for people with ITP disease). It has a high success rate for helping aplastic anemics get off platelet and blood transfusions. There is + 80% chance for the person to become transfusion-independent with platelets or blood, and 45% for both. That's pretty good. Of course, there are no guarantees.

^ Disregard what I said about N-plate. That is not the treatment that Dr. Young recommends for my mother. It is the same drug she was currently taking, Promacta, but given a different name (N-plate).

[Sally C]

Matthew,
If I'm not mistaken, N-Plate is in the same family with Promacta (Eltrombopag) which is what finally brought my husband into remission. His platelets also lagged behind the reds and whites. If you remember he has been in remission since 2012 after 125+ blood/platelet transfusions. It was NIH under Dr. Young who gave him Promacta. Keep the Faith - it takes a while.
Blessings,
Sally

[Matthew42]

Originally Posted by Sally C

Matthew,
If I'm not mistaken, N-Plate is in the same family with Promacta (Eltrombopag) which is what finally brought my husband into remission. His platelets also lagged behind the reds and whites. If you remember he has been in remission since 2012 after 125+ blood/platelet transfusions. It was NIH under Dr. Young who gave him Promacta. Keep the Faith - it takes a while.
Blessings,
Sally

Hi Sally,

I hope you and your husband are doing well.

My mother didn't respond to Promacta, although she had a good response in the neutrophil line to horse-Atg + cyclosporine. But she is still transfusion-dependent on blood and platelets 10 months after ATG.

I am not sure the drug is N-plate, which is similar to Promacta, but still very different (that's what they say online). We were told that Dr. Young would be giving her a drug that he's been using in his latest clinical trial. And, after doing some research, I'm pretty sure it's N-plate.

Wishing your husband the very best of health.

Love and peace,
Matthew

[Matthew42]

Well, an update: We went to see my mother's hematologist.

The medicine that Dr. Young proposes is Campath (not N-plate), but he wants to wait 5 more months before doing it. He believes that my mother might need close to 1.5-2 years for a good response to horse-atg and cyclosporine because of her advanced age.

She can stop Promacta as it has no real value for her. He wants her on 100 mg of cyclosporine a day for a while yet. She cannot stop it as it anchors the ATG. It must be a slow taper.

My mother's blood has held at 7.7 for two weeks after one unit of blood. This has never happened before. She's had some real rises in blood in the past but she always dropped down into transfusion-level territory by 10 days. But, this time, things are different: her blood is holding steady for 2 weeks (14-15 days). This is a new thing. But, you never know, things can go back to the way they were at any moment.

The only other news we got is that mother still has iron overload (ferritin = 3000). Her level 6 months ago was 3500. So, there was a little improvement., which was shocking considering she got 30 blood transfusions since her last iron test (3500). It is shocking that her levels were not +5000. Her doctor would like to see it around 1500 or less in the next 12 months (rather safe level). Some natural things do work to lower ferritin for some people. She says that Exjade is not worth it at this point (too many side effects). Also, the iron overload hasn't festered in her body long enough, nor is it excessively high. She can't let her have iron levels over 3000 for more than a few years, even though it can take much longer than that to cause damage to the liver and heart.

[Matthew42]

I also wanted to say that I very much value Dr. Young's expertise on my mother's aplastic anemia. Even though we never spoke to him in person, he and his team time took time to assess my mother's condition, and came back with a future treatment plan, should she need it.

I forgot to say: Dr. Young said "no" to rabbit-alg for now. He believes that Campath would be a much better option (more likely to work), as it works differently than atg. Rabbit is just a stronger version of horse-atg, working, more or less, in the same fashion as horse-atg. He is also concerned that rabbit-atg could cause negative changes in her bone marrow, which would not be the case with Campath. But, he didn't rule out the rabbit, but said it would be a last resort.

He said, basically, that it's still a waiting game to see if the horse-atg works its mojo in the next 5 months or so. She seems to be a very, very, very slow responder but indeed a responder because of her improved neutrophil line and increased cellularity in bone marrow.

Now, we know where things stand. If things don't improve in the next 5 months or so, we're not going to wait the full 2 years because of iron overload, etc. We will do the Campath by late summer/early fall.

[Matthew42]

Originally Posted by Sally C

Matthew,
If I'm not mistaken, N-Plate is in the same family with Promacta (Eltrombopag) which is what finally brought my husband into remission. His platelets also lagged behind the reds and whites. If you remember he has been in remission since 2012 after 125+ blood/platelet transfusions. It was NIH under Dr. Young who gave him Promacta. Keep the Faith - it takes a while.
Blessings,
Sally

You are correct, dear Sally: N-Plate is the same as Promacta. I got very confused.

Anyways, Dr. Young recommends Campath for my mother if she doesn't respond in the next 5 months or so.

Promacta is no longer worth taking for her, as it has done nothing to bring her platelets up. In fact, it could be keeping her levels down a bit, and not letting the ATG + cyclosporine work as it should. In other words, in some people, it can do more harm than good. When it doesn't help in 6-9 months, it's time to stop it.

Take good care.

[mola-tecta]

I have been going insane waiting for the results of the bone marrow biopsy that my mother had on 2/28. Today she got some of the results back - all of it looks good so far. Cellularity has increased with no signs of clonal transformations. (I don't have the exact numbers yet as the report has not been uploaded) Still waiting on the full genetic tests, but I am so relieved there is nothing else sinister lurking.

My mother's counts have been slowly improving still. Her Hgb finally fell to 8.0 so she was given 1 unit of blood on 2/21, and has stayed at an average of 9.4 since then. Platelets had fallen to exactly 20 on the day of the biopsy so she was given 2 bags. As of today they were still at 30, two weeks later.

Since stopping the prednisone she has been having more side effects from other medications, but seeing an overall improvement in the bloodwork numbers. Creatinine is still a bit of an issue, fluctuating between 1.5-1.8. So far no extra treatment has been needed except for extra IV fluids.

Her ferritin has been much improved, even with her being not very compliant on the Exjade - last test was around 1300, quite a decrease from 2400 a few months ago! That number was even after the last bag of blood, so I assume it was even lower than that before.

I fear getting ahead of myself and having too much hope. But my mother's hematologist seemed to think it was good, and he and my mother agreed that for right now we shall stay the course as-is. Continued improvement would be fantastic. Although she still needed some transfusion, she had gone 6 weeks without needing blood and almost a month before needing more platelets.

Matthew42, my mother's heme considered recommending N-Plate before now. I think we will stay with the Promacta a bit longer. He had also suggested possibly switching to tacrolimus if necessary since it's supposed to be less toxic on the liver and kidneys.
It seems you have gotten some good information from Dr. Young's team. I'm glad they were able to give you some workable paths to take in the future. Having multiple plans is really useful.

Best wishes to everyone.

[Matthew42]

I am so happy for your dear mother, mola-tecta. I am jumping with joy for her.

Well, we sort of got pretty good news today from my mother's nurse at her hematology clinic. The nurse/doctor said that her blood is indeed holding for the first time in 10 months post-ATG. She will have gone almost 20 days without a blood transfusion. This is unprecedented on one unit of blood. She only dropped 2-3 tenths of a point in hemoglobin in 15 days. This is the first time my mother's hematologist was ever excited for her. While it's no guarantee that things will stay this way, it is a very good sign. The doctor said "holding" is key, and the most reliable for showing a real response to treatment (much better than rises with very steep falls that follow).

Her platelets are still really low, with no improvement at all.

Your mother's iron is fine. Please don't worry about it!!!

My mother's hematologist said that my mother needs to get her ferritin down to about 1500, and then under 1000. But she said that 3000 is not that big of deal at this point.


All is well, getter better!

[mola-tecta]

Matthew42, I was never really worried about my mother's iron counts anyway, it's been the hematologist that has been harping on it It's still good to see it go down because Exjade is a real pain to deal with. (You know it comes in massive tablets you have to crush and drink with water?? Yuck)

I have been looking over the full genomic report that was posted to the chart and for the most part it was fine except for one thing:

DETECTED GENOMIC ALTERATIONS: Mutations were identified in the following
gene(s): ASXL1 (p.Gly646Trpfs*12). Variants of unclear significance, if
identified, are listed in the scanned report.

After a long bout of research I found a few articles that were very helpful in understanding what this means.

Clonal hematopoiesis in acquired aplastic anemia

Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia

A brief, but comprehensive, guide to clonal evolution in aplastic anemia

Secondary myelodysplastic syndrome and leukemia in acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria

Basically, from what I understand, the mutation is not diagnostic of anything, but it confers a higher likelihood of progression to MDS one day.

I don't know how to feel about this. On one hand, I know it's possible that the mutation could shrink or disappear, or that new ones will show up. There's still a good chance it does nothing. A lot of people, especially older ones, will show up with MDS-related mutations post-treatment from AA.

On the other hand, I am frightened and stressed out about this. My mother being older, and that we are 8 months out and only just now achieving partial response makes me worried as well.

I know there is nothing to be done about it regardless, except to continue to monitor blood counts going forward and likely more bone marrow biopsies or genetic testing.

It's hard to consider all of this. It's a lot of information I don't fully understand, and it seems the research is also a little fuzzy simply due to lack of data. Some days it's also harder to consider that both of my parents developed blood disorders.

For now, I suppose I'll have to be satisfied that the biopsy currently shows no evidence of dysplasia, and a good increase in bone marrow cellularity. My mother's blood counts are improving. My focus in on wanting my mother to feel better, regain strength and improve functioning. And I'll keep donating platelets when I can, because it's at least something tangible and physical I can manage to do.

[Matthew42]

@ mola-tecta: Yes, I know it was the hematologist who was harping on your mother's ferritin levels. My mother's doctor is the opposite: while she mentions it, she isn't concerned it about now, as I already said. Hematologists have different views on iron overload (what's dangerous, when to treat, etc.). My mother's ferritin levels actually dropped 500 points when she was tested 30 RBC transfusions ago. The doctor was happy to see that there was a drop.

That mutation in your mother's bone marrow could quickly change. I've seen people on here whose cytogenetics change a lot from one biopsy to the next. So, it could be nothing at all.

I forget to tell you that at our last appointment, we were told that my mother is not likely at all to get PNH, even though she has a tiny PNH clone. The doctor told me to not to worry about it, even though she can't rule it out completely. She also said that my mother has about a 15% of getting some subset of MDS. That's another reason to stay away from the rabbit-alg, as it can bring about chromosomal changes to the bone marrow. Campath doesn't affect the bone marrow.

My mother's last bone marrow report was fine except for a small amount of dysplastic red blood cells. The doctor said not to worry about it, as it is normal to have tiny amounts of dysplasia in bone marrow biopsies, even with people without bone marrow disease. A lot of people have very small amounts of dysplastic white blood cells, too (there's a normal range for this). Dr. Young never considered it as anything when he looked over her bone marrow biopsy. She has classic aplastic anemia that is highly immune-mediated. That's what we were told.

I have a hard time understanding chromosomal changes in bone marrow. It takes a lot of research, and you also need someone with background in research hematology to assist when you get figure it out. Where do you get that but Dr. Young's team at the NIH? LOL.

Your mother is doing so well. She is getting better, even though it's hard to have hope when it takes so long to get better.

Finally, my mother's hematologist left the room last week, saying: "It's a very bizarre and unpredictable disease...no two aplastic anemics are alike."

Take good care of yourself.

[mola-tecta]

I am rather stunned - for the first time I have ever seen, my mother's platelets rose on their own. Twice this week! From 30 on 3/17, to 33 on 3/21, and now 45 on 3/24! Hemoglobin has fluctuated several times both up and down but is still holding above 9.

Really hoping this is a good trend forward. Also hoping the hematologist will actually acknowledge it. This entire process has been awful and I want something to start happening. And I desperately want the hematologist to just acknowledge something is happening?

[Matthew42]

Originally Posted by mola-tecta

I am rather stunned - for the first time I have ever seen, my mother's platelets rose on their own. Twice this week! From 30 on 3/17, to 33 on 3/21, and now 45 on 3/24! Hemoglobin has fluctuated several times both up and down but is still holding above 9.

Really hoping this is a good trend forward. Also hoping the hematologist will actually acknowledge it. This entire process has been awful and I want something to start happening. And I desperately want the hematologist to just acknowledge something is happening?

That's EXCELLENT news. If her platelets are rising on their own, then recovery should be around the corner. So happy for your mother (and for you, too!).

They did a peripheral blood bone marrow biopsy on my mother (not as detailed as a tissue sample), and there were no cytogenetic changes, no dysplastic cells, etc.

My mother's blood held steady for almost 3 weeks. She dropped, but very, very slowly (for her). She ended up getting transfused just before the 3-week mark, but it was a good thing. It was the first time that my mother's hematologist said she was excited for her. Unfortunately, a week later after the blood transfusion, she needs blood again (tomorrow). She went down fourth-tenths of point in hemoglobin in three days, but her red blood cell count didn't drop that much like usual. I still think things may be improving somehow (you need to pay attention the RBC count and how it drops, as same levels of RBC's can yield different hemoglobin levels). At any rate, she may get away with only needing 2 units for the whole month. That is unprecedented for her (usually at least 3 units a month).

No improvement in the platelet line. And neutrophils are a bit under 1000 for the past week, but I am not worried about it. She is still averaging well above 500.

Lymphocytes and Neutrophil percentages are close to equal at times. I read that was a sign the autoimmune condition was quieting down. Not sure, though.

Best of health to everyone!

[mola-tecta]

I was rather stunned again that when my mother had her routine bloodwork on Monday her counts were even better! Hemoglobin went all the way up to 10.2 on it's own, and platelets were at 54! She has not gotten blood in 5+ weeks and no platelets in 4 weeks.

The hematologist didn't seem to be that impressed though? I can only assume he is being cautious and not wanting to jump the gun on it. But man, I just want some feeling of positivity from him sometime. I recognize that her counts may drop again and fluctuate because that happens even in normal blood. But I have never seen my mother's platelets actually rise on their own like this.

White cell counts, including neutrophils, were actually normal this test too. ANC 2.3, ALC 1.4, WBC 4.3.

Matthew42, I am glad that your mother is also showing signs of improvement. Needing less blood is a great thing! The longer the times between transfusions, the better. I don't know much about how hemoglobin and RBC are correlated, but it's interesting to see how they change. Hoping that your mom is feeling decent as well, my mom is still having side effects from being on prednisone so long as well as cyclosporine just being a real pain of a drug.

[Marlene]

Mola-tecta,

I think you can take a chance at celebrating her progress. All three lines are looking good. It's good news and should be acknowledged.

Marelene

[Hopeful]

Congratulations to you and your mom mola-teca!

Wishing her a durable response!

[Matthew42]

I cannot figure out why my mother's hemoglobin stayed more or less the same for three weeks (held steady), and now it's back to the way it was before the 3-week stabilization period. The blood is still not dropping as dramatically as in the past, but...

Her neutrophils, while still not in danger zone, dropped considerably, too. I just don't understand. Her neutrophils went from 1200 to 600 and then swing back up to 1000. It's just really bizarre.

Her hematologist said she was excited about the 3-week hold and now her threshold for getting blood was lowered. I suppose that's a good thing. I just don't understand the wild swings and sudden changes.

All the best!

[Matthew42]

My mother's blood jumped from 7.1 to 12.8 after a transfusion last week. The doctor's nurse said it might be a mistake. I really don't know. She said 11.8 could be possible, but she was shocked, nonetheless. She wouldn't say flat-out it was a mistake.

The platelets are no better, and her neutrophils, which have averaged around 1000, have gone down to 400. That really worries me. I have to put her back on antibiotics.

The internet says that it's rare for hemoglobin tests to be wrong (more than 4/10's of a point). If the RBC count and hemoglobin correspond, then you can believe it was a very accurate test (my mother's)

What do you all think?

Update: it's very unlikely to have been a mistake. The doctor said that she had a big natural rise in blood + lingering effects of last week's blood transfusion. That's how it happened, but she's not sure. It was a very good thing. She said hemoglobin tests are just not off like that, when RBC count correlates. My mother's hematologist doesn't know what to believe now: Blood is 12.8; neutrophils are 400; platelets are 14. LOL.