Recently completed High Dosage Cytoxan regimen at Johns Hopkins. Ask me anything.

[Technique21]

Hello Forum Members,

I'm a 29/m who was diagnosed with vSAA (anc - 200, wbc - 1,300, hgb 3.9) in May of this year. I researched the available treatment options and decided to move across the country to be treated by Dr. Brodsky at Johns Hopkins. My treatment regimen started on July 6th with 4 days of High Dosage Cytoxan. My first signs of white cell production came a month later when I produced 45 neutrophils. My white blood cell count bounced around for the rest of August. The 2nd day of this month, with the help of Neulasta, my neutrophil count rose to 3,774 and eventually topped out at 5,838 a few days later. My counts are currently: wbc - 2400, anc - 1500, hgb - 9.5, platelets - 22. I'm not currently on Neupogen/Neulasta. My platelets and hemoglobin are rising and I'm transfusion independent right now. I'd like to give back by answering any questions I can in regards to the treatment.

[pvinod]

Hi Technique21,

Congratulation first up!!!

it is really great that you have a good recovery. My daughter have a same problem and her current ANC is 300. We had a H-ATG treatment in Dec-2010, but still not response.

so I want to ask about treatment you take. Can you please give the brief idea about High Dosage Cytoxan. I never heard about it previous. I heard ATG and BMT option for AA. so is it the new one? How it is given? is there any risk? what are the chances of recovery? is it available in India?

Additionally, my daughter is 5 years old, so can we give this treatment to her?

is there any relapse chances in it?

Please guide me.

[Marlene]

Wow!!!! That's a quick response especially since you were vSAA. Dr. Brodsky treated my husband back in 2002 with HiCy. Glad you are doing so well.

[Marlene]

pvinod,

Here's the latest published data on the use of high dose cytoxan for aplastic anemia. Those who do high dose cytoxan as their first line of treatment have a better response than those who have tried other immunosuppressant treatments like ATG.

http://bloodjournal.hematologylibrar...2136.full.html

[Technique21]

Marlene,
Thank you! I'm remaining cautiously optimistic.

Pvinod,

You should definitely read the article Marlene submitted to understand the basics of the treatment. It is not new, but to my knowledge, Johns Hopkins Hospital is the only facility that uses it as a first line immunosuppressive treatment.

The idea is that, as opposed to using ATG, to specifically suppress the t-cell lymphocytes, which attack your bone marrow cells, high dosage cytoxan suppresses all white blood cells and reduces the number of white blood cells to zero (my total wbc was < 50 for about 35 days). The thinking is that the bone marrow cells are able to recover and create lymphocytes that no longer attack the bone marrow. Here is a youtube video illustrating the idea http://www.youtube.com/watch?v=w8-jx1dtg0U .

High Dosage Cytoxan is given via IV over a 4 days. The dosage is dependent upon height and body weight. Response rates are similar to that of ATG. It's hard to really compare the two, because a lot more people undergo ATG than Cytoxan. There are many risks involved. I evaluated all risks with treatment vs the risks involved with not being treated. Response typically takes longer than ATG. This means I spent more time without an immune system than I probably would've had I gone with ATG. The major risk was becoming infected while having no immune system. Relapses do occur and so does clonal evolution, but from everything I read, the chances are lower than that of ATG. I am not a Dr and I cannot say for sure. If you'd like the specifics, you should read his preciously linked study and reach out to Dr. Brodsky.

I can speak more intelligently as to the actual high dosage cytoxan treatment at Johns Hopkins. I can speak to the process, what to expect, how I was treated, etc... There is a special outpatient area of the hospital that saw and examined me everyday while I was immunosuppressed. I was assigned to a subset of Dr's and nurses that were familiar with my records, back ground, and diagnosis. I was transfused when my numbers dropped below a certain threshold and I was given prophylactics daily for both bacterial and fungal infections. I strongly believe that the supportive care at the hospital is what makes the difference. I chose the treatment that was right for me and moved across the country specifically for it.

[pvinod]

Thank you Marlene, Technique21 ,

I read the post. unfortunately we already did with H-ATG.

But still I am not sure, if this treatment have a good results than why still it is non famous compare to ATG and BMT. we can see the good results in VSAA in very short period, so why not all doctors use it?

Anyway, we are from India and already with ATG, so it is lower chance for us for response from High Dosage Cytoxan.

Though I will ask our hematologist for this option.

Thanks again...

[Technique21]

You're welcome. I asked my Dr, who was only familiar with ATG, about the treatment and he pretty much disregarded the idea. Every Dr doesn't do this because there was a clinical trial performed by the NIH. The NIH failed to replicate the results, in part, because they did not have the facilities to treat the immunosuppressed patients that Johns Hopkins Hospital does (my opinion). Either way, people who've undergone ATG treatment do undergo the High dosage cytoxan treatment. I was asked to speak with a person who'd undergone hATG successfully and relapsed 3 times. The Dr said that they have the best results with patients who respond to ATG. The patients that didn't respond to ATG don't fare as well. I hope this helps.

[pvinod]

Marlene,

Your husband treated with High Dosage Cytoxan in 2002. So how he is now? A long time pass out for this treatment, so is there any other complication he face after high dose cytoxan?

IN your signature he have a Stable partial remission, so what him count now? Sorry to become bother, but just worried for my daughter and thinking should we have to take high dose cytoxan or not, so asking?

Also you write FE is down to 444, so what does FE means?

[Marlene]

To echo Techique21's comment, NIH did do a High Dose Cytoxan clinical trial that ended early. Their trial included the use of cyclosprorine after the administration of the 4 days of cytoxan. This lead to one or two fatalities from fungal infections. Johns Hopkins trial was different but the stigma from the NIH trial stuck with many. The NIH then turned their focus on the use of ATG.

Now, there are better antibiotics and anit-fungals to help people get through the period where they have no white count. This is true for BMT's also.

John had a difficult time and did not respond like most. He went 84 days without a white blood cell count and it took him 22 months to get red cell transfusion free and 20 months to be platelet transfusion free. Well outside the norm. The anti-fungals, at that time, were very hard on him. He was also on a new antibiotic that caused some issues for him. But like I said, they have newer anti-fungals that are not as hard on the body now.

After 9 years, his WBC/ANC is now normal, his HGB is stable around 12 and his platelets are at 114.

[Neil Cuadra]

Originally Posted by pvinod

Also you write FE is down to 444, so what does FE means?

FE stands for ferritin, the way iron is stored in the body.

Normal adults have a ferritin level less then about 250 (men) or 160 (women). Children the age of your daughter normally have less than 140. These numbers are approximate.

Patients receiving transfusions often end up with higher-than-normal ferritin, and if it gets too high (over 1000 for example) it can be dangerous and may require "iron overload" treatment. There are discussions about it in the Transfusions and Iron Overload forum.

Since your daughter is transfusion dependent her iron level should be monitored.

[pvinod]

Thanks Marlene,

It means Mr John was not took any other treatment except High Dose Cytoxan. He got the late response but treatment is only High Dose Cytoxan. He also has not any other diagnosis like PNH, MDC etc tiil date after (due to) High Dose Cytoxan therapy. Does he continue CSA till 24 month?

Thanks Neil Cuadra for FE explanation.

[Marlene]

John's only treatment for SAA was the High Dose Cytoxan. And you are correct, he had a late response to the treatment.

He has not progressed to any other diseases like PNH, MDS or any other cancers. He never had CSA. CSA is not part of the High Dose Cytoxan treatment.

[pvinod]

Thanks Marlene.

[Andrya D]

I am trying to decide where to transfer my care. We are being stationed around Fort Meade so we have our choice of Johns Hopkins and University of Maryland. They are both equidistant from Fort Meade. How are the doctor's at Johns Hopkins? I am currently getting care at the University of Kansas, and I am less than satisfied with my current care. They treat you like you are on a bone marrow transplant assembly line. They haven't taken the time to get to know me or understand my unique situation. Knowing what the doctor's are like, will weigh heavily on my decision of where to get treatment.

Any information would be extremely helpful!

[mausmish]

I have experience with both facilities. I elected to have my BMT at Johns Hopkins because they had more experience as well as a clinical trial I wanted to participate in. However, I was told at my initial consult at Hopkins that if I had a transplant elsewhere I could not get follow-up care at Johns Hopkins because they were too busy with their own transplant patients.

I was under the care of Dr. Ivana Gojo at University of Maryland for nine months prior to my transplant and highly recommend her and her nurse practitioner, Michael Tidwell. I also was tended to as an inpatient by Dr. Maria Baer, head of the hematologic malignancies department. Although I saw her only once, I was pleased with the encounter. (I was only in the hospital for two days.)

Please feel free to contact me privately if I can be of assistance.

Karen

[KellyDIL]

Happy to hear of your good results! My mother-in-law is at GWU and finished her ATG treatment 2 weeks ago. Since then she has been very out of it mentally here and there, she contracted a very bad infection in her arm, and now has pneumonia. I'm not sure what is next, how long to expect her to live, etc. She is 65. Anyhow, I discovered the doc at Hopkins after she decided to do the ATG, although I wasn't really a part of any of the discussions anyway, I still like to share my opinion if I can and have good info. So would she be able to do the Cyotxan if she already did the ATG? I still am not clear on that. Or is it a case-by-case basis. Thank you!!

[Marlene]

You can if the ATG doesn't work but high dose cytoxan is best when done as the first line treatment. It has a better success rate. So you need to wait to see how she responds to the the ATG first. If she does not respond, then you need to explore your options of a repeat of ATG, HiCY, or BMT. If she does not respond, I would suggest meeting with Dr. Brodsky for an opinion. Responses vary, you can have a complete remission, partial and transfusion free, or partial but still needing transfusions.

[amandana]

I also received high dose cy in Feb, after being diagnosed with SAA, I was seen by Dr.Brodsky, and choose the treatment following his recommendations on my diagnosis. He didn't treat me, I came back to Canada for my treatment to be near family for support. I am canadian, I moved to Brooklyn in 2010. I was able to receive mine at PMH in toronto, Dr Brodsky referred me. I started cell production about 5 weeks after treatment, however I had some serious complications due to the hidh dose chemo received, I accuired a severe punemonia infection which I was in an induced coma for about 4 months in total leading to life support, as punemonia turned into Acute respitory distress syndrome which I was diagnosed with two months after being admitted for treatment. It took a while for my body to start making cells, but through my complications remarkably cell production was improving at an astonishing rate. I was on transfussions and blood filters for oxygenation throughout my coma, I am currently down to only having needed to go in twice for transfussions, I was also diagnosed with diabetes in july my doctor says it is a complication from the high dose chemo and everything I went through, my pancrease damaged, I have type 1 and insulin dependant which has been affecting my cell production, having difficulty with maintaining my blood sugars which I learned it also impedes my counts. I am also happy to answer any questions or advice needed.

[Susan]

Were your kidneys checked during treatment? How were they affected? If kidney function was reduced, have your numbers gone up at all over time?
Thank you

[amandana]

Yes they checked my kidneys throughout and as well as all my vital organs, I was on life support. My numbers steadily went up but didnt start seeing any real progress until about a month after my treatment. The kidneys are affected by lack of erythropoietin which helps to stimulate your bone marrow to create red blood cells, but since their is no bone marrow it will not produce red blood cells so kidney failure can also occur with shortage of erythropoietin, which chemotheraphy can reduce, and any other enviromental toxins and such

[viperphil11]

What treatment would everyone perfer now that you have been through it? Im 20 years old and had AA and have no previous treaments. I would like to know which is the better treatment, Cytoxan or ATG?