My Wife AA treatment

[PKumar]

My wife was diagnosed with AA In Feb 22. She is 76 years old and was treated with ATG , Promacta 75 mg and Tacrolimus 2 gm . Her white cell count came up to 2.5, neutrophils 1500 and hemoglobin to 9.6. She stopped blodd transfusion on May1,22 however she still needs platelets transfusion every 10 days as it drops to a level of 13,000.
In August 22 and Sept 22 doctor increased the dosage of Promacta to 125 mg and tacrolimus to 3 gm a day and also added steroid Danazol 400 mg per day to increase the platelets from low level of 13000 to a higher level of above 20,000 to stop platelet transfusion.

During the last week her hemoglobin has come down to 8.1 and white cell count to 2 after the increased dosage of promacta and addition of Danazol. Her platelets count has not increased much but it is only one week since the start of Danazol.
Has anybody gone thru this type of treatment and experienced this kind of drop in hemoglobin and white cell count. Could it be due to Danazol's initial reaction ? It has been almost 7 months since the original ATG treatment was given.
Can somebody help?

[Matthew42]

Your post fascinates me as I just found out recently that Danazol is used as a monotherapy drug to treat refractory aplastic anemia. There are many studies to show that it can have great results. Why some hematologists won't prescribe it is beyond me. Women with AA fare even better on Danazol than men with AA. That is what I read.

think it takes a few months or more to have any real response to Danazol for aplastic anemia, but I could be wrong. It might take 4-6 months. I just can't remember.

My mother is still transfusion-dependent after 15 months since ATG. They want to give her Campath, but we are not ready to for it just yet.

Let me tell you this: platelets are the most stubborn of the three lines in aplastic anemia. People can get off blood transfusions way before getting off platelet transfusions. They are also rare cases of someone getting off platelet transfusions, but still needing blood transfusions several months later. Every person with AA is different. Some people have all their levels rise and stay up all at one time. It is likely that your wife will eventually have a full response to ATG and the Danazol as her blood has already come up and stayed up since May of this year. That's a great sign.

Aplastic anemia is a very mysterious disease and very unpredictable until all levels stabilize.

I am wishing your wife the best of health and lots and lots of happiness.

May you both be well.

[triumphe64]

I used Danazol for my Pure Red Cell Aplasia. It took several months to start working.

[PKumar]

Thank you Matthew for your good wishes.
My wife has been on Danazol for 11 days at the same time doctor increased her dosage of Promacta to 125mg per day from 100 mg. This is to improve platelet count.
Last one week has been very hard for her, increased nausea, lack of appetite, light headed and headaches.
Her neutrophil dropped from 1500 to 800 and hemoglobin from 10 to 8.7 in one week.
No improvement in platelet count so after 10 days it is at 14000 and needed transfusion. Dr temporarily stopped Danazol.
Has anybody experienced this kind of drop from Danazol ? Could it be some kind of reaction in her body?
Any experience with this medicine will be a big help.
Thanks.

[Matthew42]

Danazol takes several months to work, so I am not sure why the doctor took your wife off of it so quickly. There is a high success rate with Danazol as a first-line monotherapy treatment for SAA. I believe the success rate is even higher in women with SAA. All that said, I am not your doctor, and don't know his or her reasons for doing so.

Could it be the Promacta with the Danazol?

I surely hope things get better for your wife. She's already off blood transfusions, which is amazing! Congratulations!

By the way, Danazol is lower dosages does not usually cause terrible side effects.

https://www.webmd.com/drugs/drugrevi...next_page=true

[Hopeful]

Hi PKumar,
I am sorry that your wife is struggling with this disease. It is encouraging that she had a response in some of her blood lines. Hopefully the platelet line will follow soon!

I would encourage you to use the search function on this forum to read others experiences with Danazol. It was used in a bunch of clinical trials for people with TERC/TERT mutations a few years back. This mutation causes short telomeres. You can follow links in the posts to view the clinical trials and results. The clinical trials also have posted side effects to the drug.

If you have any trouble finding the links, I?m happy to help sort through the information.

I hope this helps!

[triumphe64]

Originally Posted by Hopeful

Hi PKumar,
I am sorry that your wife is struggling with this disease. It is encouraging that she had a response in some of her blood lines. Hopefully the platelet line will follow soon!

I would encourage you to use the search function on this forum to read others experiences with Danazol. It was used in a bunch of clinical trials for people with TERC/TERT mutations a few years back. This mutation causes short telomeres. You can follow links in the posts to view the clinical trials and results. The clinical trials also have posted side effects to the drug.

If you have any trouble finding the links, I?m happy to help sort through the information.

I hope this helps!

I had forgotten about the TERC/TERT mutations.
And indeed, Danazol increases telomere length.

[Matthew42]

They are some in the benign hematology world who say that Danazol is underused in the treatment of severe aplastic anemia; in fact, women who have short telomeres with SAA are very much strong candidates for Danazol as a first-line treatment. It can be combined with an immunosuppressant, but not necessary for this subset of patients.

In one study, 70% of women enrolled achieved a complete response with Danazol for SAA after failing with treatment for ATG + cyclosporine. I was shocked by the number of "complete responses" (not partial responses). Men, however, did not respond nearly as well, although a few of them had complete responses. Partial response rates were not given, however.

If patients relapse a few years later, they can start taking Danazol again. It is true that the relapse rate is higher than those who respond well to only immunosuppressant therapy.

I read a few other studies concerning Danazol. It only works well for a certain subset of SAA patients. Again, it's mainly used with adults with SAA with short telomeres or "suboptimal" chromosomes. Without PNH-clone increase is preferable, but not necessary.

I never knew any of this until recently.

[PKumar]

Thanks Matthew42 and Triumphe64 for your kind words and suggestions regarding Danazol.
We might request doctor to try again after some time.
My wife hemoglobin was down to 8.3 few weeks back. Doctor decided to give her blood transfusion on 10/7/22 as she was feeling weak. On 10/18/22 her hemoglobin came up to 10.4 which was a good improvement even though transfused blood stays in the body for 4 weeks. her white cell count has improved to 3.1 on 10/18/22 blood test however her platelet is still staying around 13,000- ten days after transfusion so she needs platelets every 10 days. She is on higher dosage of Promacta ( 125mg) and tacrolimus ( 3 mg)
which may be helping her.

Are there any food supplements you know of which may help platelet count.
I read some articles on Reishi Mushroom extract which is supposed to help but afraid to try due to possibility of interaction with her regular medicines.
Also she has lack of appetite and nausea . She takes Zofran and Ativan alternatively to relieve nausea, any other suggestion you may have to combat it.

[Marlene]

You can search for papaya leaf on this site. There were some past discussion on it for platelets. Also look into sesame oil to increase platelets.

If she is holding at 13K without any bleeding, you may want to discuss stopping platelet transfusions with her and her doctor. John was able to stop transfusions at 8K. His clotting times were good. If her clotting times are off, then Vitamin K may help that. Vitamin K is found in leafy greens and vegetables.

[Matthew42]

Originally Posted by Marlene

You can search for papaya leaf on this site. There were some past discussion on it for platelets. Also look into sesame oil to increase platelets.

If she is holding at 13K without any bleeding, you may want to discuss stopping platelet transfusions with her and her doctor. John was able to stop transfusions at 8K. His clotting times were good. If her clotting times are off, then Vitamin K may help that. Vitamin K is found in leafy greens and vegetables.

Yes, I agree. My mother's current hematologist will not agree to stop platelets transfusions under 20,000. She is super strict on this. There are some doctors who let you go down to as low as 8 before transfusing It's complicated.

There are patients with ITP disease whose never receive platelets, even when they are basically zero, as platelet transfusions are a waste of time (back to where they were in a few hours or so). There are a subset of ITP patients for whom no treatment works. They have to learn to live with extremely low platelets all the time.

@Pkumar: all the best . Things are still looking up for your dear wife. Keep us informed and please take good care.


PS: Please reconsider Danazol for your wife at some point. Ask the doctor!

[PKumar]

Thanks Matthew for your suggestions.
We had the blood test done on 10-28-22. we were pleased with the progress
The white cell count increased to 4.1 and neutrophil to 2.5 which are both in the normal range. Her hemo was 9.9 which is not normal but in the safe zone.
Her platelets improved to 15K which were higher than 13K in the last test.
She is on 125 Mg of promacta, 3mg of Tacro and 10mg of prednisone
Prednisone was added 3 weeks back.
We dont know exactly what is helping her the most increased dose of Promacta and Tacro or addition of Prednisone or HTAG treatment.
The issue of nausea and lack of appetite still persists and we are using Zofran and Ativan to control it. We try to supplement her food with different protein shakes and vegetable juices so she can get her daily nutrition of minimum 40 gms of protein.

We have used following diet as a supplement to improve the white cell count
smoothie made from equal amount of Kiwi and frozen dragon fruit and protein powder.
For Platelets and red cells
Green leafy juice, Pomegranate seed extract, papaya, beets, oranges etc
We want to thank all well wishers and hope her platelets improve above 20K so they can stop transfusion. It has been a tough journey for last 8 months from the time she got her HATG treatment in Feb 2022 and hope that our experience helps others.

[triumphe64]

Sometimes prednisone works as a catalyst to make the others work better.

[Hopeful]

Hi PKumar and Matthew42,

I listened to a very interesting presentation by Dr Taha Bat entitled "Aplastic Anemia and Non-Transplant Therapies".

There is a Q&A session at the end where the doctor passionately talks about the importance of trying different strategies if transfusion dependent and not just waiting on the sidelines. For example, he mentions considering the use of N-Plate for refectory AA that is not responding to the traditional IST approach.

Also, he encourages patients to email him if they need direction in their treatment strategy based on his experience and research.

You can watch the presentation here:
https://www.aamds.org/webinar/aplast...lant-therapies

PKumar - It is encouraging that your wife's blood counts are sticking! I hope the changes made continue to have a positive impact.

Prednisone can cause a false rise in WBC, so just be wary for this blood line.

[Matthew42]

My mother's doctor said that platelets are often the most stubborn of the three lines. She's had patients where red blood cells and neutrophils went up and stayed up, but very low platelet levels persisted many months after getting off blood transfusions. At any rate, if 2 lines come up and stay up, the remaining third line should do so at some point, but it could be a half a year or more. Hard to believe.


Please know that steroid therapy is underused in aplastic anemia, according to some experts in AA. Interestingly, some patients respond beautifully to it. Every AA patient has different body chemistry. There is no one-size-fits-all approach.

[triumphe64]

Hopeful,
I saw that also. He is excellent. He is at UT Southwestern here in Dallas, Texas.