Father AA - ATG treatment 4 months

[LJones]

Hi everybody, I'm seeking a little help and knowledge sharing in regards to my father who had Horse ATG for AA 4 months ago.

He's currently having 2 platelets transfusions a week and mostly 1 red blood a week. His platelet count is anything between 9-20 and his HG is around 70-80. If he's any lower than 10 platelet and 70 blood it really affects him.

My question is we do not really know what to look for in his blood results after the ATG treatment. We're not seeing any decrease in transfusions and the numbers are staying pretty much the same. My father is disappointed nothing has happened after 4 months and he's convinced ATG has not worked.

Is there anything else we can look at in the blood results other than platelet and HG number that may give us an indication something is happening Or the ATG is slowly helping repair the bone marrow etc.

Any knowledge would help, I feel like my father has given up already.

[Ruth Cuadra]

Hello and welcome to Marrowforums. I hope you will find useful information for helping your father as well as hope from the journeys of others that he will have a successful outcome.

You undoubtedly know that every patient is different, which here means that general information about response times for ATG given for AA may not reflect your father's particular experience. That said, we know that ATG improves blood counts for AA patients about half the time. When used with cyclosporine or Eltrombopag, ATG will improve counts for about 7 out of 10 patients. If it works, ATG usually stops the need for blood transfusions after about 3 months but a full response can take up to 9 months. The need for platelet transfusions may continue longer than the need for whole blood.

Some things to consider about your father's situation:
-- did he have a strong reaction (e.g., serum sickness) to ATG?
-- is he taking cyclosporine or Eltrombopag?
-- does he have other health issues that could be compromising his recovery?
-- how old is he? (We know younger patients tend to recover faster.)

I hope this brief response helps you and your father. Please post additional questions or more information about your father so our members can help.

Regards,
Ruth Cuadra

[LJones]

Thanks Ruth appreciate the reply.

He only experienced mild fever the first night of ATG treatment after that no side effects or serum sickness at all.

My father is 67 years old and on cyclosporine 125 day/night.

He does not have any other complications that we are aware off but he is constantly ?not feeling good? (but I think that may be mental rather than physically). He also seems to have regular heart palpitations in the evening but the dr?s can?t not see any issues so we are putting this down to low bloods or the cyclosporine.

I do see a few comments on the forums relating to ?neutrophils? and other things that can be detected in the blood results. Will there be any indication something might be working in any of the them numbers ?

Thanks again and to anybody else that may respond it really is appreciated

[Matthew42]

High LJones,

I am a bit late to your question.

AA affects three lines:

red blood cells (hemoglobin)
platelets
neutrophils (a type of white blood cell that fights bacterial and fungal infections)

What are you father's current numbers? That way I can get a better idea of his situation.

ATG-horse/cyclosporine/Promacta (triple therapy) is usually the first-line treatment. It can take a long time for the therapy to work. Every person responds differently. How long your doctor will wait until trying a second-line treatment also depends on several factors: overall health of patient, age, how severe neutropenia/platelets are/other risk factors in waiting. There are cases where it's taken over 2 years for horse-ATG to work. YES -- I am serious!! But it's not wise to wait that long, especially if the patient is transfused (blood and platelets) with very neutrophil levels. Remember: what makes AA most serious is not the need for blood transfusions, but rather the low neutrophils and low platelets that can trigger a medical emergency at any time (infection or serious brain bleed). For the record, no one lets their hemoglobin to get down to dangerous levels once a person is known to have a blood problem. Of course, an AA patient can get iron overload if they are transfused with blood for a few years or more, but that's another story.

Note: If a person has heart issues, low hemoglobin becomes more of an issue, and thus transfusion threshold protocols change.

Here are some second-line treatments for refractory AA (refractory = first treatment didn't work). It's not that the same things as relapsed AA (where first treatment did work for a period of time).

For AA refractory settings, these are your options that I am currently aware of:

1 Rabbit-ATG (instead of horse) + cyclosporine/tacrolimus/sirolimus
2 Campath (Alemtuzumab)
3 High-dose N-Plate (sister drug to Promacta)
4 Danazol/other androgens
5 Drug Trial enrollment
6 Bone Marrow Transplant (your father's age makes this option much less likely, but some doctors will do them under 70, or even 75 (pushing it).

It merits saying that Campath is now preferred to Rabbit-ATG (actually, it's Rabbit-ALG, but they say "ATG", as they do for horse). Why? It's about 10% or more likely to work than rabbit, plus there is no other treatment used alongside it; that means it's a monotherapeutic treatment (no cyclosporine, etc.).

If you have any more questions, please let me know.

May your father go into remission.

Take good care.

[LJones]

Thanks for the reply Matthew that?s very helpful
My fathers current numbers are

Red blood cells between 70-85 (transfusions weekly)
Platelets between 10-20 (transfusions twice weekly)
Neutrophils remaining constant at 0.1

We are now exactly 5 months after Horse ATG and absolutely zero improvement on any of them figures.

That?s good to know about other treatments. My father?s haematology dr has suggested a week in hospital with daily transfusions to get my dads numbers up to a ?normal person? number which will hopefully make him feel a bit better but obviously he will still need weekly transitions to keep his levels up that high. He seems to be shaking every evening now between 5-6pm for no reason.

Just hoping something positive happens soon.

[Matthew42]

Originally Posted by LJones

Thanks for the reply Matthew that?s very helpful
My fathers current numbers are

Red blood cells between 70-85 (transfusions weekly)
Platelets between 10-20 (transfusions twice weekly)
Neutrophils remaining constant at 0.1

We are now exactly 5 months after Horse ATG and absolutely zero improvement on any of them figures.

That?s good to know about other treatments. My father?s haematology dr has suggested a week in hospital with daily transfusions to get my dads numbers up to a ?normal person? number which will hopefully make him feel a bit better but obviously he will still need weekly transitions to keep his levels up that high. He seems to be shaking every evening now between 5-6pm for no reason.

Just hoping something positive happens soon.

Because of your father's blood count numbers and his older age, it might wise to seek out a second treatment now. Please contact Judith Marsh's team in London. She is an expert in aplastic anemia. They should be able to advise you as to what to do.

Your father's neutrophils are very, very low, putting at him at a very high risk of infection. Is he on antibiotics to prevent infections? If not, he very much should be. I am not trying to scare or upset you, but this number bothers me the most. I'd like to see his neutrophils at least rise to .5, so he's not profoundly neutropenic. A .5 neutrophil count is on the border between severe-moderate neutropenia.

Also, the need for blood is my least concern, as it usually is the case with AA, even though the patient gets tired, and definitely needs blood when they need blood. However, I don't like platelets under 20 because of the risk for bleeding. While people can have very low platelets (even zero), and live for years with no adverse bleeding event, the risk remains high. Also, older people have leakier blood vessels than younger people, increasing their risk for an internal bleeding event.

Look at the neutropenia chart below:

mild neutropenia = 1.0 - 1.5
moderate neutropenia = .5 - 1.0
severe neutropenia = .2 -.5
profound neutropenia = below .2 or .1

Please ask about Campath (Alemtuzumab) or Rabbit-ATG. Also, he can try high-dose N-Plate injections for a few weeks to see if that helps.

Please don't give up. Many AA patients recover well and go into long-term remissions, despite being in a very bad place medically before a treatment works.

By the way, I don't know why your father is shaking. Does his blood sugar get too low? He may need to eat more.

One more thing: the only sign that an AA patient is getting better is improved blood counts. I've known people whose bone marrow biopsies showed improvement, but there was no improvement in actual blood counts (peripheral blood).

If you have any more questions, please let me know. I am here for you.

[LJones]

Hi all,

Thanks for your replies. My father?s professor at Bart?s hospital London has now suggested that it?s been 6 months with zero response to HATG and now unlikely to work.

He is now suggestion BMT, which is a bit scary to hear. He?s also asking does my father have children - I?m made 40 years old in general
Good health. Would I be considered to see if I?m a match and what happens if I am ?

Very down that the HATG did not work.