High dose cyclophosphamide at Hopkins?

[destiny09]

We were advised to stay away from this treatment from our doctors, due to the fact that the national instutue of health shut down their study due to a high death rate.... We to have a 12year old that is diagnosed from severe aplastic anemia. She didn't respond to ATG she is still 95% production lost at her cellural level. She is recieving one red cell and two platetlet tranfusions a week. She has had over 112 transfusions since may. The childrens hospital wants to do an emergency bone marrow transplant on her, we are currently waiting for the bone marrow center to find a match as two donars haven't panned out.

[flyguy]

Originally Posted by destiny09

We were advised to stay away from this treatment from our doctors, due to the fact that the national instutue of health shut down their study due to a high death rate.... We to have a 12year old that is diagnosed from severe aplastic anemia. She didn't respond to ATG she is still 95% production lost at her cellural level. She is recieving one red cell and two platetlet tranfusions a week. She has had over 112 transfusions since may. The childrens hospital wants to do an emergency bone marrow transplant on her, we are currently waiting for the bone marrow center to find a match as two donars haven't panned out.

Hi Destiny,
I'm so sorry about your daughter. I certainly hope she starts producing more cells via some treatment. She certainly needs some relief to those transfusions every week. When I went to Johns Hopkins I was not transfusion dependent and was diagnosed with mAA. I went off cyclosporin and within 3 months was not producing many cells and went toward sAA when my platelets were 4,000 or so. I went thru ATG locally and spent 12 days in the hospital and still have side effects from the treatment. I now require 2 units of platelets weekly but about 3-4 weeks between packed red blood cells. I won't know if the ATG helped for 4 or 6 months, I'm told. I am 68 years old so my response is a lot different from your daughter's, I would think. Please let us know what is happening. I hope a donor will work out. I know you life and hers is in tatters with the waiting, but I really think something will turn around for you.
Much love and support,
Flyguy

[Marlene]

Destiny,

The NIH HiCy study used a different protocol and did not have the anti-fungal meds that are available today. The advice from your doctors is out of date and misleading. If you are interested, then you need to contact Hopkins directly to get the data on outcomes for all populations. There are different response rates depending on whether or not you've had ATG before doing HiCy. Having HiCy as the first line of treatment has a better response rate than if you had ATG first. BTW, NIH funded the High Dose Cytoxan clinical trial at Hopkins so they still thought it had merit even though their trail failed.

At the time when John went through HiCy in 2002, no one with SAA had died from the treatment.

I hope they find a donor for your daughter quickly.

Take care,
Marlene

[bchenaille]

Hey Destiny:

I continue to pray for your daughter's health. I can't imagine how you must feel having her endure this prolonged neutropenic state.

My daughter Kathryn went through the HyCy treatments. The one thing that I would really encourage is to reconsider your options. Everyone agrees that the most dangerous situation is prolonged neutropenia. The longer the wait to find a MUD donor, the more dangerous her situation.

As for the recommendation NOT to look into the Hopkins protocol using cyclophosphamide, I am a bit dismayed that the medical community still sticks to the merits of the NIH study which wrongly added cyclosporin. This clearly caused toxicity in the patients and the results were skewed. Dr. Brodsky just published the results from the Hopkins clinical trial and the figures are very exciting. You can read about it at: http://bloodjournal.hematologylibrar...09-06-225375v1

What were the dangers for Kathryn? Yep, catching a virus, fungus, or other germ during her recovery phase. Did she make it? Yep, just like everyone else I know who has gone through this treatment. She is back to living a normal life once again. Praise God!

Unless I am missing something very important, here's the weighted scales:
1. Cytoxan @ Hopkins: 4 days of hi-dose chemo, antibiotics & GCSF. After a slightly longer neutropenic state, the body begins to regrow its own hematopoietic stem cells. No radiation, no GVHD, no steroids, no other drugs.
2. MUD BMT: 4 days of hi-dose chemo, full body radiation, foreign marrow, GVHD, steroids, et all.

With all the possible side effects of the MUD BMT, it may be worth exploring this route that could be started now and get her body recovering sooner rather than later. Nothing is foolproof with this dreaded autoimmune disease. But having gone through it, I am so incredibly thankful that others who had blazed the Cytoxan trail had encouraged me and our family.

Baltimore is also home to the Ravens. They come to the local Ronald McDonald House to give out signed footballs to the kids. If for no other reason, maybe you would choose Hopkins to get her a football? :-) (I know . . . dumb joke, sorry!)

bchenaille@comcast.net