End of the Road - 8 weeks from Diagnosis to No Hope

[Alcof]

Well it has been a very short ride. Hopes were dashed today when our doctor said he didn't think my Dad would survive ATG treatment and there was nothing he could do for us. I asked how long he thought we had - that people with AA usually take a year to die without treatment, didn't they? He said it would be a lot sooner than that given his age and deterioration. It could be any time.

So now we have to decide how to die.
Or mortgage the farm to pay for some Eltrombopag. Can't get it any other way in New Zealand except to buy it ourselves. $8,000 USD a month. And who knows if that will work.

Has anyone else had experience with being turned down treatment with ATG? What path did you choose to take?

Sorry if this has been a dark post. It has been a bit of a dark day for us.

Appreciate any thoughts.

[Marlene]

Sorry to hear this. My first reaction is to get a second opinion. And IMO, if he's still on cyclo, I would consider stopping it. Doesn't appear it's helping. I would be looking into supporting him via nutrition and maybe some alternative approaches. Palliative care is needed in that his transfusions need to be managed well. Get his red cell up to decent level and determine a threshold for transfusions. It usually around 8 for HGB. For platelets, it can be as low as 5K as long as there is no prolonged bleeding. But most usually use 10K.

He may benefit from growth factors like procrit or neupogen. They can run a blood test to check his own EPO levels and if it's below 500, procrit may help boost the production of red blood cells. But it can take up to 14 weeks before you can actually see an increase in transfusion intervals.

What are his D, B12, folate, iron, copper, B6 and zinc levels? The D, B12 and folate need to be at an optimal level. Low-normal or high levels of B12/folate need to be explored. If he's taking supplements now, he would have to stop them 4 or 5 days before testing to a more accurate read.

I would also venture to guess that the antibiotics disrupted his digestive system's bacteria and he probably needs to restore it back to a good balance with probiotics.

Many time with this disease, it gets really dark before it gets better.

[Hopeful]

Well it has been a very short ride. Hopes were dashed today when our doctor said he didn't think my Dad would survive ATG treatment and there was nothing he could do for us. I asked how long he thought we had - that people with AA usually take a year to die without treatment, didn't they? He said it would be a lot sooner than that given his age and deterioration. It could be any time.

So now we have to decide how to die.
Or mortgage the farm to pay for some Eltrombopag. Can't get it any other way in New Zealand except to buy it ourselves. $8,000 USD a month. And who knows if that will work.

Has anyone else had experience with being turned down treatment with ATG? What path did you choose to take?

Sorry if this has been a dark post. It has been a bit of a dark day for us.

Appreciate any thoughts.

Alcof,

I am sorry to hear this news, but I would not give up hope! If I were in your situation, I would seek out another opinion. However, I would not stop the cyclosporine if his kidneys are handling it okay. It takes 2-3 months for cyclosporine to build up in a person's system. What is his current dosage and what is his weight in kilograms? I just want to confirm that he is on a standard dosage (5mg/kg/day).

Has he stopped the prednisone? Prednisone is not a treatment for AA and has terrible side effects including increasing the risk of infection.

The infections that he picked up are likely responsible for his increased transfusion requirements. Fevers will burn through platelets and RBCs at a rapid rate.

It takes ATG/Cyclosporine 3-9 months to work (typically). Cyclosporine alone is a weaker immune suppressant. So it seems logical to me to think that it may take longer than 8 weeks to see results, especially if he picked up some infections during that time. Are you seeing any signs of stabilization of his condition?

[Hopeful]

It is my understanding that the excitement around Eltrombopag is when it is used *in combination* with ATG. I haven't read or heard anything about its effectiveness when used alone. This is likely because the immune attack must still be stopped.

A good predictor of response to immune suppression from NIH is the level of ARC or ALC. Having ARC levels above 25,000 or ALC levels above 1,000 increases the probability of a response (60-80% vs 40% with low ARC/ANC).

I remember reading a paper about using a lower dose of ATG over a longer length of time for older patients. I posted it in this forum before but will have to dig it up.

Here is a great presentation out of Europe on treating older patients with AA that I found. It discusses using Cyclosporine alone, ATG success with older patients, and low dose ATG:

https://www.ebmt.org/Contents/Resources/Library/Slidebank/AACQEventNov2012/Documents/AA10_TreatmentElderly_TichelliFinal.pdf

Good luck!

[Sally C]

Hi Hopeful,
I have to respectfully disagree with your statement about Eltrombopag. My husband has been transfusion free for 3 years now after taking Eltrombopag started in the spring of 2011. He came off of Eltrombopag once his platelets went over 100,000 - which he still maintains to this day - and has taken no more Eltrombopag. He never took ATG. He had over 125 platelet/blood transfusions before taking Eltrombopag.
He did have Campath in Spring of 2009 but it did not help his counts and nearly killed him with an infection a month after.
NIH has had wonderful success with Eltrombopag although I can't say what treatments the other patients had.
If anyone is interested in reading a more detailed account of my husband's journey, please look up my past posts on Promacta (Eltrombopag).
God Bless all,
Sally

[Hopeful]

Hi Sally,

I am going to respectfully counter-argue :)

Campath is a heavy-duty immune suppressant. Although your husband's marrow may not have been able to recover on its own after the treatment, I can still make the argument that it may have stopped the immune attack. If your husband didn't have the Campath, it is unclear whether he would have had the same result with eltrombopag alone. I am theorizing that his marrow needed the eltrombopag to kick start it into making blood cells again. Of course, I have no proof of this theory and am just basing it on what I am heard/read. If I am wrong, it wouldn't be the first time!

I remember some NIH presentations that talked about positive results from using eltrombopag for refractory AA that didn't respond to IST. They didn't talk about using it as a first line of treatment though.

[Hopeful]

Hi Sally,

I see that there are clinical trials out there right now that use eltrombopag alone for first line treatment of AA. I didn't realize this, as I had only heard the very positive preliminary results of eltrombopag used on refractory SAA patients. Hopefully, good news will come from these studies!

I am going to eat my humble pie now :)

[Alcof]

Hopeful, thank you so much for your continuing advice regarding AA. Its such a relief having an extra research head in this fight. Because we are back fighting today after yesterdays news. We are not giving up.

Sally C, thank you so much for your story! Ray of hope that Eltrombopag alone can work. I just need to get me some now...I will go find your story and read it all.

Hopeful, can you post links to the studies around Eltrombopag as first line treatment? I haven't found those myself yet. I too had only heard of Eltrombopag used for refractory AA or for moderate AA after ATG treatments. This is very promising.

In fact today I did a crazy, mad thing and wrote to Glaxosmithkline in NZ to see if they might be charitable and send some my way. Why not? Nothing to lose. Make them say no. If you don't ask, you don't get!

We considered a second opinion. But it is quite clear that the Doctor who has treated the most AA patients in this country, administers nearly all the ATG in this country is our Haematologist. Anyone else would probably just refer back to him. And he is very good. His concerns are around the fact that my Dad has diverticulitis in his gut (potential infection) and his kidneys flared up when he had pneumonia 6 weeks ago. That and the fact that my father can no longer walk and is not strong at the moment. 8 weeks ago he was fit as a fiddle, no underlying conditions. He is neutropenic now and that just makes him the ticking time bomb.

Hopeful, great advice about the fact that we're burning through blood products and that relating back to infections etc. That gave us a lift that it wasn't the disease necessarily getting worse. And today is day 9 since his last red transfusion. Only had platelets yesterday. This is the biggest gap we have seen. Too soon to call it an improvement. But I think what you say about cyclosporine taking a while to build up in the system makes sense. So we are going to try for at least another 4 weeks to trully make sure that avenue is exhausted. He is on 400mg per day for body weight around 95kg. (he is a tall man - good weight for his BMI).

Marlene, thanks so much for your very real advice on the palliative care side and managing the transfusions. Also the vitamin side is not something we had looked into fully. We are going next week to see a holistic healer of sorts to cover off all our chances. My Mum and Dad have been health freaks for years and will definitely look into levels. We are taking astaxanthin as a stab in a hopeful direction. We are going to contact hospice now also - just so that side is ready.

Thanks Marrowforum team. It really does feel like a team effort when I post on here. It gives me the confidence to say the things to the doctors, nurses etc that have to be said. (including expressing my frustration that they put him on prednisone in the first place. Glad we have kicked that one to the curb).

[Mseth]

Hi Alcof,

In India, Glaxo markets eltrombopag under the brand name Revolade. I do not know the cost, but I am sure it is reasonably subsidised in India. You can try and see if there is a way to get it. You could search the internet for more information.

All the best!!

[Marlene]

Alcof,

Just make sure if you engage hospice that it's not the same as palliative care. Not all hospices do palliative care. There are distinctions and sometimes they get lost during these times.

And, if you choose to add in supplements, there are forms that are better to use than some of the ones doctors usually prescribe/use. You can always check back here when you find out his nutritional status. You may want to ask the doctor for copies of the blood test. They should have checked his B12, folate and iron early on and so it may be helpful to get those results now. And just know, the blood serum test for B12 and folate are not definitive.

My husband's journey was a long and difficult one. He had one foot in the grave more than once and when he was discharged from the hospital, he was getting red cells twice a week and platelets 2-3 times a week. And he was neutropenic.

It takes time to recover from the infections and toxicity of the antibiotics. Both take a toll on the bone marrow. Lots of baby steps.

[Sally C]

Dear Alcof,
I am so sorry for your Father's situation. But with this disease you never know when it could turn around. Don's situation was so dire at one point that he was on his oncologist's "death list".
I think contacting Glaxo was a great idea - I just wish you were in the US so you could possibly go to The National Institutes of Health (NIH). They truly saved Don's life and are on the cutting edge. The NIH had such great success with their AA patients taking Eltrombopag that they decided to try it on their MDS patients. Don was their first MDS guinea pig and he is now their star patient - the biggest response and the only one who has maintained his counts after stopping the drug.
I wish you and your Father the very best. Keep the faith - it could all turn around. It's such a strange disease and so unpredictable! I'm so glad you have found Marrowforums. The people who post here have personal experience for you to draw on - and they are very caring and so supportive. We're all in this together.
Hopeful, I was getting ready to eat my own humble pie as NIH had never mentioned a connection with immune suppression and Promacta so I really couldn't disagree with you. Thank you for investigating it further. As you know - knowledge is so key with these marrow diseases and treatments.
Warmest wishes to all - and God Bless,
Sally

[Marlene]

Wonder if Danazol would be an option. It's a synthetic androgen similar to testosterone and has been used in SAA. If you search on it on this forum you'll find a few people who participated in a clinical trial. I can't remember if it was for AA or MDS though.

http://www.ncbi.nlm.nih.gov/pubmed/21279356

[triumphe64]

Wonder if Danazol would be an option. It's a synthetic androgen similar to testosterone and has been used in SAA. If you search on it on this forum you'll find a few people who participated in a clinical trial. I can't remember if it was for AA or MDS though.

http://www.ncbi.nlm.nih.gov/pubmed/21279356

Danazol has definitely helped me. PRCA only affects red cells, but Danazol is now the only med I take.

"Greg H" on these boards has written many very good postings about it. There is also a poster with AA and a TERC mutation who has had success. I'm sorry, but I've forgotten her name. If you search the member list for Greg H you can see all his posts. He writes for a living, so they are among the best on this site.

[Chirley]

Alcof, you could also ask your fathers doctor to write a letter to Glaxo asking for compassionate use of the drug. It holds a little more power if the doctor does it. Surely, it's the least the doctor can do. As you say, if you don't ask you will never know.

[Hopeful]

Hi Alcof,

Currently, I see that Eltrombopag is only used in NIH clinical trials without IST and as a first line treatment for cases of moderate AA. Unfortunately, there are no results yet as to its effectiveness without IST. Ethically this makes sense to me because ATG/CyA is the gold standard for AA and has a 60-80% success rate. Moderate AA typically gets a watch-and-wait approach in the US. So, it seems like a less risky approach to try this experimental drug without IST on patients who aren't in crisis mode yet.

I still believe that if there is an immune component to a person's AA, that has to be addressed as well. However, there are probably some people who's AA was caused by something else like a toxin or virus that has since left their body but that has left their marrow too fragile to recover. So perhaps, Eltrombopag alone will help these people. It will be interesting to see the results from this trial! Who knows..perhaps Eltrombopag alone can alter the course of a person's immune response. It works for ITP, another auto immune disease.

If you do get a hold of Eltrombopag, you should write/call NIH on their suggested dosage/duration.

On a side note...
That link that I included previously shows that Danazol has a 47% success rate as a first line of treatment for AA.

[Alcof]

With thanks to marrow forum advice, I brought the possibility of Danazol as a treatment up with my Dad's doctor. And this evening he has just had his first dose. I did read in the research that there wasn't much success (if any) for Very Severe Aplastic Anemia but it is something. And something is better than nothing! So now Dad is continuing with Cyclosporine and Danazol.

We still haven't given up the idea of privately sourcing some Eltrombopag. I am writing to the Doctor to ask what suggestions he has and whether he will back up my letter to Glaxosmithkline. My brother is going to try and find it in various locations when he travels for his work at a hopefully cheaper rate. He is going to Europe and Middle East so fingers crossed he comes across a source.

Dad is in hospital at the moment fighting what was an unknown infection. My mother was the one who found it. She was helping him shower yesterday and found a black lump at the base of his spine. No wonder he was uncomfortable sitting! No one else had found it - and now it is being drained and HOPEFULLY that one will be taken care of and we can get infection free again. Each infection we find and beat is another day he is with us. And I had thought infection meant colds and flus. Oh what a steep learning curve this has been!

[Cheryl C]

Great news that you've taken a step forward (Danazol) with your dad's treatment! Sorry to hear of the infection - unfortunately all too common with marrow disease.

[marmab]

I did a trial of eltrombopag (Promacta) this past spring as a last-ditch effort, before going to transplant, to try to remedy my lack of platelet production. My marrow had no megakaryocytes and was thus making no platelets; basically all I had were the ones from transfusions twice a week. I have had two rounds of ATG/CsA, and various other drugs as well. Unfortunately, I had no response to eltrombopag. I had always dragged my feet about transplant, as I was/am extremely healthy and fit. This is an odd way to make a decision, but my doctors (and second opinion doctors) finally just wore me down -- after two years -- with their arguments in favor of transplant. I am one of the lucky ones. So far, so good -- no complications. I feel absolutely great, and am extremely fortunate and thankful that it has turned out this way.

[Marlene]

Great to hear you are doing well with the transplant. I know it was a difficult decision but looks like a good one.

[marmab]

Thanks so much, Marlene. It was indeed a difficult decision for me, but I am doing extremely well, and very happy and grateful :)

[Alcof]

It pays to be active in the care of the ones we love. Reading the forums and making sure EVERYTHING is explained to us is so important. We really are part of the team. As such the Doctors are really throwing the Kitchen Sink and everything else at this disease that we can think of.

Dad is tolerating the Danazol very well, continuing Cyclosporine, and now he is getting daily injections of G-CSF. In addition they are really pushing the boundaries of his transfusions. Trying not to give platelets just because we are <10. He is in hospital still and they are waiting to see if there are any bleeds and if he can stabilise at very low levels. And so far so good! He has the mouth blood blisters but they are not bleeding and are still clotting. And his energy is still quite good. Eating, sitting up and all that. Still not walking much though.

And we are STILL optimistic that we might have support from Glaxosmithkline to get some Eltrombopag. They responded to my letter and requested to speak to my Dad's doctor. The Dr filled in a heap of paper work and its gone off for consideration! Feels so good to have taken a chance and have had someone listen - even if it ends in a NO answer - I'll still feel like I tried. In the meantime we are enjoying our little spell of optimism....especially given the FDA have now green lighted Eltrombopag in the US...

[KMac]

Dear Alcaf,

Your father is very lucky to have you in his corner. Keep up the good fight! It really sounds like you are doing everything humanly possible to make sure he gets the best treatment available.

And yes, I agree that loved ones advocating for him likely spur his entire treatment team to work more vigorously on making him better.

It would be great if that Eltrombopag comes through**. This can be such a difficult and mysterious disease, but often with surprising positive outcomes, and as such it's very much worth it to try multiple treatments.

**My experience suggests that it probably will, in that your father seems to be close to the model patient for that mode of treatment, since ATG and BMT are not options for him, and his platelets are low. I was personally interested in the Eltrombopag, thinking it might finally boost all my counts into the normal range. But as you can see by my numbers below, I am very fortunate to have responded well to the ATG/CsA. NIH reviewed my case and basically felt we should leave well enough alone. My own hematologist strongly agreed, feeling that Eltrombopag could be dangerous for me since my platelets are already in the normal range. I still have some fatigue, but more or less I have my health and life back. So yes, this is a brutal disease in it's overwhelming symptoms and uncertain outcome, but there is reason to be optimistic!

[Chirley]

Alcof, I'm so pleased to hear everything is coming together and that the Doctor is filling out the forms. I'm just a bit disappointed that you had to start the ball rolling rather than the Doctor initiating this himself. Thank goodness you are a very good advocate for your Dad. But what about other patients of this Doctor who don't have advocates such as yourself? Do you ever wonder why sometimes they just don't mention these options themselves and you have to do the work? I was in that position a lot for myself and now for my mother and father...it's frustrating isn't it?

[Cheryl C]

Well here's a good news story about doctors for once. Just started with a new GP today, because we've moved and I have been fighting an URTI for the last couple of weeks (silly me). Had asked the medical centre to request my records from my previous centre a couple of weeks ago.

When I went in to see the doctor, he immediately said that he'd received my fat file and had read through it! Amazing. He knew what MDS was and that I had low immunity and was really interested in my version of my history. That was a first for me and made me feel incredibly positive.

[Chirley]

I'm impressed.

My GP recently phoned me to say that he had been banned from practicing as a doctor in Australia and he was doing me the courtesy of letting me know that he wasn't just disappearing off the face of the earth and leaving me in the lurch. That makes two GPs now. The first one had an inappropriate sexual relationship with a vulnerable mentally ill patient. I don't know what my last one is supposed to have done but I hear he has moved to Qatar. I'm disappointed because he was one of the best GPs I've ever had.

Now comes the search for another GP...geez, I hate that.

I hope I find one who is willing to read my notes....that would be a bonus!

[Neil Cuadra]

Chirley,

It might be worth checking up on any doctor you consider, using the Medical Board of Australia (http://www.medicalboard.gov.au) website, to help you avoid doctors that are likely to skip town!

Here in California we can refer to the Medical Board of California (http://www.mbc.ca.gov) website to check a doctor's education, certifications, and disciplinary history.

[Chirley]

I did that Neil. We also have a site called AHPRA which tells us where they Registered, how long they have been Registered and any conditions they have had on their right to Practice. This last Doctor was a Specialist in General Practice..he actually did extra exams in General Practice, he had been doing it for 16 years, had never had a complaint. He had been head of a large NHS Practice in England before he emigrated to Australia. I grilled him thoroughly on my first visit that he had all the qualities that I wanted because I needed someone to be my advocate. He was so good my parents changed GPs to be with him. He went the extra mile. I'm completely confused.

He was a devout Muslim who even donated his time to provide free medical care to small villages in Yemen where he his life was in danger on many occasions. I will miss him. He was a pacifist who abhorred any type of violence.

[Alcof]

Glaxosmithkline have approved my Dad the supply of Eltrombopag. They have responded to a rather emotive letter I wrote - and working with my Dad's doctor, have given approval to supply my Dad with this little bit of hope in a tablet. Something we could never have afforded ourselves and is currently not subsidised in NZ.

Hurrah!

Now don't worry - we are totally realistic of where our hopes must be with regards to a) staying infection free with Zero Neutrophils long enough for the drug to have a chance to work and b) the effectiveness of the drug % wise.
But for those who have been following my posts will know that two weeks ago we had no further options. Now we have possibilities.

I am now going to put all my Dad's info and the eltrombopag dosage that he is on in an email to NIH to see if they can approve the dosage and how compatible it is with Danazol and Cyclosporine which it is intended he will continue to take.

I have learnt on this road, that it pays to be knowledgeable and the more heads working on a problem the better. My Dad's doctors are good. I am happy with what they are doing. Even if I have had to push and ask all the questions and make all the suggestions. Just because we put our Dad in a hospital, doesn't mean he stops being ours.

So keep up the good work, marrowforums. Thanks so much for being there to help me advocate for my Dad. Today we are in a much more hopeful place.

[Alcof]

Alcof, I'm so pleased to hear everything is coming together and that the Doctor is filling out the forms. I'm just a bit disappointed that you had to start the ball rolling rather than the Doctor initiating this himself. Thank goodness you are a very good advocate for your Dad. But what about other patients of this Doctor who don't have advocates such as yourself? Do you ever wonder why sometimes they just don't mention these options themselves and you have to do the work? I was in that position a lot for myself and now for my mother and father...it's frustrating isn't it?

Yes, Chirley I agree. In an ideal world we could just relax and just provide the moral support. It is so exhausting, constantly being on top of what is happening - and even knowing what to ask for.

I guess the way I've been rationalising it is that in NZ with rare diseases there is so little experience and knowledge - and also nearly all medical treatment is government funded. Public health in New Zealand is by and large very good. But it is a largely a thankless task that I do not think I would have the patience nor inclination to do.

But yes I do agree. it would be lovely if everyone that got diagnosed with a scary disease had a loving advocate with the time and energy to put into researching everything for them and standing by their side the whole time. I used to be an advocate carer for intellectually disabled people and that is what we were taught. Be an advocate for the person. I guess it is forgotten sometimes in the medical world that sick people need advocates too. That they sometimes don't have the strength, inclination or will power it takes to fight their own corner and ask WHY all the time.

[Chirley]

I was in a position a couple of years ago when I was too sick to voice my wishes and no one advocated for me and I almost ended up in an aged care facility at the age of 55. Luckily I recovered enough function to state my wish for rehab even though I was told it was futile.

I,deep down, can never forgive my friends and family for not advocating for me. It is a hurt that won't ever go away. That is why I am such a vocal advocate for the rights of patients now. For the need for Doctors to be aware that patients have rights and feelings and don't have to agree with everything the Doctor says. I advocate for my elderly parents to the point where I am considered a thorn in the side of the health services but I don't care as long as they get the best treatment available.....I'm damned if I will let their care be compromised because I know how absolutely vulnerable you are when you can't speak for yourself.

[Marlene]

It may be worthwhile to have a consult set up with your doctor and NIH. I can't think of a good reason why your doc would oppose one unless his ego won't let him.

I agree that it's a good idea to make sure the combination you have him on is appropriate as well as the dosages.

Good luck.

[Alcof]

Today is not a good day. My Dad has really taken a turn for the worse. He has burning fever, his head is covered in a red large lump rash, he has large ulcers through his mouth and his face is swollen. His tongue and gums are so swollen that he can hardly talk and is struggling to eat at all. He is really struggling.

The doctors are quite at a loss. They are looking for infection. They are growing cultures off everything we can find. They have done MRI, ECG and all sorts and cannot find the source of infection. It will take a few days for the cultures to grow anything. In the meantime Dad seems to be declining. He's trying so hard and we can't do anything.

Goes to show that with Neutrophils of Zero its just so verydifficult to stay infection free.

I'm now going through the process of considering all his medications and what might be causing such a reaction in case its not an infection...

[Marlene]

So sorry to hear this. Is he still on cyclosporine? May be worth considering stopping it for now since it suppresses the immune system. And are they treating the infection? If he's on antibiotics, then the cultures may not show much. I assume they did a blood culture also. Good that you are also looking into possible drug allergy/interactions.

[Hopeful]

I am also sorry to hear this. My thoughts are with you and your dad.

[Cheryl C]

I'm also very sorry to hear this news about your dad and hope a solution can be found quickly.

In the mean time, I don't know if you're aware of the wonderful NZ product Medihoney. I find it very soothing to swish a spoonful around in my mouth if I have ulcers.

See http://www.express.co.uk/life-style/health/32316/Healing-honey-helps-patients-survive-chemo

[aussie danielle]

Just a little hello from across the Tasman,i was sad to read your post .I dont know what to say to help, other than to say i am thinking of you,and I hope things improve for you and yours .

Life can deal us some hard knocks at times ..cant it??my prayers are with you all.
I recently had a bone marrow done to see if i would qualify to take Revlimid in Australia,which could have, hopefully, seen my red cell count improve..and make me transfusion independant. BUT the test came back negative...i was very dissapointed ... simply an another curved ball to handle....

Keep your chin up and know that this is a very helpfull forum and there are lots of information and very kind people who may be able to help you....aussie danielle:)

[Alcof]

Thank you so much for all the support. Its so easy to get discouraged when I see my Dad trying to be brave when he is in so much discomfort. We are not out of trouble yet, but Dad is definitely improving.

They have isolated an infection in his mouth and a targeted treatment is starting to work. He still has difficulty eating and swallowing - which has caused him to start putting his kidneys under stress - which is aggravated further by the necessary nature of cyclosporine and the other medicines. But the doctors kicked it all up a gear, got a team of renal specialist in and put Dad on IV fluids and now they're happy with him, an Oral Specialist has joined in now and is working on getting his mouth right and a whole team of Gastro doctors looked in his gullet today and the treatment is working. The large nasty rash and swelling on his head has completely gone. His tongue is starting to shed the sores, and I believe I actually saw my Dad's teeth today!LOL!

We aren't giving up. And he isn't either.

[Cheryl C]

Good news at last! Thanks for sharing it with us all. So pleased that there is improvement in your dad's condition. He's very fortunate to have such faithful, loving support in you guys too.

[Alcof]

Well what a difference. Dad is getting over this infection in leaps and bounds. He almost looks normal again! Mouth is going to take a while to get completely over. He still has sores and pain and is unable to eat anything solid. But the swelling is right down and each day it improves. Temperature is stable. He is even getting mobile again, thank God.

I have presented information to the doctors with regards to the G-CSF and they have agreed to stop this now after 3 weeks with no response. Took a while for them to get on the same page, but they listened and now today they have stopped the injections.

Also today they have stopped cyclosporine which I have mixed feelings about. There was no taper. Straight from 300mg per day to Zero. Any thoughts about this? I assume as there is no response to cyclosporine there is no real reason to taper gently.

Also have not found any information that suggests that either Danazol or Eltrombopag may be more or less effective with cyclosporine. Any ideas here?

In any case, Dad is delighted to have kicked the cyclosporine as it should help his gums recover even more rapidly - we hope! Thanks to those who have posted in this forum in this regards!! And he always had difficulty taking them. Horrid smelly things!

Tomorrow is Dad's birthday. He's dreaming of eating Ciabatta bread rolls with salad and ham! Shame he can't have that until that mouth is better. Its just protein formula until he can swallow properly. But great he has an appetite!

So a good news day. We are quietly hopeful that we may even get to bring him home soon - maybe in another week. Which will be a great relief to my 74 year old mother who stays every night in the hospital and is actively nursing him due to the over worked and slim staffing they have in our public hospitals. And 5 weeks of night shifts is hard going for anyone.

So fingers crossed now. No more infections. And we continue to pray that he has a response in his bloods.

[Hopeful]

Alcof,

I am glad that he is doing so much better! To my knowledge, there is no risk with stopping cyclosporine abruptly. It isn't like prednisone, for example.

I must warn you though that it can cause intense arthritic-like pain in the joints for some people when the dosage decreases (even slightly). So, look out for this and know that it will pass within a few days/weeks.

[Alcof]

Thanks Hopeful! Its always hard double guessing what some new symptom is. Nice to be ahead of the game for once...hope he just eases off it without too many issues...just happy gums...!

[Sally C]

Hi Alcof,
I have been keeping up with your posts. So thankful your Dad is doing better. You are a great advocate for him and obviously love him very much.
My husband had great success with Eltrombopag without being on Cyclosporin. I do have to add that he had no response with the Cyclosporin but don't know if that makes a difference.
If you would like, please read my posts about his journey. He came from being on our Oncologist's "death list" and over 125 blood/platelet transfusions to being transfusion independent for over 3 years post Eltrombopag. He stopped the med in 2012 due to platelets going over 100,000 and has yet to lose his reponse.
While I know every case is different, I hope this helps. By the way, he has MDS.
Best wishes and God Bless,
Sally

[Alcof]

Well at last Dad is infection free. And home from the hospital after 6 weeks of constant battling to get clean of so many infections including 5 hospital bugs. He is so well its amazing. Unfortunately this is due to a magical balance of anti-virals, anti-biotics, and anti-fungals and not because his blood has improved at all.

However it won't be until Monday next week that he goes on to the full 150mg Eltrombopag dose. Then we will really start to be on tenterhooks. Waiting and hoping.

We just need to get Dad's strength back now and muscle mass. A long slow, taxing and emotionally draining process for him. And pray and hope we don't get more infections.

Sally your husbands story is so inspiring and uplifting. Its what keeps us all going - knowing that people have lived through it and come out with SUCCESS! Only for Marrowforum and I would never have heard of Eltrombopag, would never read your story and never found the courage to hope and pursue the stuff.

[Marlene]

Couple of things to keep in mind. It's very difficult for any on the blood lines to improve when fighting infections. Fevers chew through platelets and red cells. And any white cells will have been called to the areas of infection/inflammation with very little left in the blood. All of this, plus the meds, are taxing on the bone marrow.

John went 84 days without a white count. He too was on all the "anti" meds. As soon as his ANC hit .300, they would pull one med at a time. If he did not spike a fever they kept going. If he got a fever, they would resume the med.

At this point, he was on G-CSF (neupogen).

Keep an eye out for GI problems, specifically, very smelly stools. Could indicate a C-Diff infection which can happen being on the other meds.

Lastly, good sources of protein will help build the body back up and start off with mild exercise. If he's on Danazol, that may help too.

[curlygirl]

I know a lot of people with autoimmune disorders eschew dairy, but milk was great when my son got a mouth full of sores along with fevers along for two weeks after going of his prednisone following ATG (It could have been serum sickness, or a virus, it was hard to tell). The sores made it hurt too much to eat, the coolness of the milk helped his sores, and clearer liquids made him feel like would throw up. It's also high in calories, protein, Vitamin D, and vitamin B. Just a suggestion. If you dad has any kind of congestion it can make it worse so you'll have to see if it agrees with him.

[Sally C]

Hi Alcof,
Your post warmed my heart. So thankful that Don's story has helped you and your Father on his journey. Please keep us posted.
Best wishes and God Bless,
Sally

[Alcof]

Its really tough staying optimistic. No movement in bloods across the board. And we are fighting infections, in and out of hospital. Near death so many times that we have to ask how often can he come back? Its the repeated flare ups of the ESBL and their friends that do the damage. And we are in week 8 of Eltrombopag and praying for a miracle to happen. Its a battle between the blood recovery clock and the antibiotic resistant bugs.

The neutrophil count is 0.05....so zero. Consistently. Never changing. Only the Hb and platelets change as they fall between transfusions.

I struggle to avoid the question what if. What if eltrombopag doesn't work? What then?

Such a cruel condition as Dad fully comes back in between bouts and he seems next to normal for a day or two. And then the doctors remind us that he has little or no hope. To say they aren't optimistic is an understatement.

Anyhow, we keep praying and hoping and making the trips to the hospital and not sleeping. Its far more unpleasant for Dad.

Keep the fingers and toes crossed for us...

[Chirley]

I don't know if it's any comfort but my Mum has had ESBL and VRE for two and a half years and still doing okay.

[Alcof]

My father passed away peacefully at home on Wednesday 29th. It was 18 weeks from diagnosis to the true end of the road.

Thanks to the Marrowforum community for all your great stories and support and thoughts and prayers over the last 4 months.

[Neil Cuadra]

Alcof,

I'm very sorry you've lost your father. From your posts I've had the impression that both you and your dad were positive people and I wish he could have won this fight. I'm saddened at the short time he had after his VSAA diagnosis.

You told us that you had been an advocate carer for other people, and it's clear you were there when your father needed you. He must have drawn strength and comfort from having you as a medical advocate. I'm glad to hear that the end was peaceful and at home, but I know this is anything but peaceful for you. You have my best wishes at this sad time.

[Chirley]

I'm so sorry to hear this and this won't be any comfort at this time but in the future you can take some comfort from knowing you both did everything you could to win the battle.

[Caregive]

Thank you for posting your father's journey. Prayers to you and your family.

[curlygirl]

Alcof, I'm very sorry to hear that. Thank you for taking such good care of your Dad on this sad journey.

[Cheryl C]

Deepest sympathy to you Alcof and all your family. No one could have done more to help your father than you did. If you have a bible, Revelation 21:1-4 may be of comfort to you. There is a better day coming. God bless.

[Marlene]

Alcof,

I'm so sorry to hear that your Father passed away. He was very lucky to have you as his advocate. I'm sure he appreciated all that you did for him.

[Whizbang]

Alcof,

So sorry for your loss, may eternal light shine upon him...

God Bless,
David

[Hopeful]

So sorry to hear this!

Peace to you both -

[Sue H.]

Dear Alcof,
I have been following your story and send you my deepest condolences at this sad time. I wish you much strength. The way you looked after your father and researched every possibility for him was so very inspiring. May your dear father rest in peace.
Thinking of you,
Sue H.