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Old Tue Nov 8, 2011, 12:02 AM
cfutia22 cfutia22 is offline
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5% cellularity and new diagnosis

Dear all, I'm new to the forums and I'm hoping some of you can give me some input on an important question I have.

Facts: My youngest son, 17 (adopted from Kolkata, India at 6 months) has just been diagnosed with Dyskeratosis Congenita, a rare genetic bone marrow failure disorder. About a year ago a routine CBC showed severe pancytopenia and a subsequent bone marrow biopsy showed <5% cellularity. His counts were very low across all 3 lines. A few months later, after losing weight and becoming very weak, he was diagnosed with severe diabetes. Once the diabetes was treated, some of his counts came up. He is now just below normal on white cells and ANC, just below normal on hemoglobin, and his platelets have been very low but stable at 25.

My son has no biological relatives and we will not put him through a bone marrow transplant with an unrelated donor (even if one could be found). In addition to everything else, he suffers from mental retardation and reactive bipolar disorder, moderately controlled with medication. Hence no androgens or other treatments that could disrupt whatever stability he has (and ruin whatever quality of life we've been able to achieve). We are comfortable with our decision for palliative/supportive care only under the circumstances. He will live as a "normal boy" until he isn't anymore. We'll minimize hospitals and procedures and continue maximizing happy everyday life.

So where am I going with this? This is my question, and no doctor seems to want or be able to address it. HOW LONG CAN SOMEONE LIVE WITH 5% CELLULARITY? In one way, it really doesn't matter, because my son will live until he can't anymore. But for the rest of the family, it's helpful to have a sense of timeframe. Should my older son continue living at home or move into his own place? If his brother only has a year or two left, he'd rather stay. So help me, folks. Can people live years and years with such a low cellularity? Is it possible that the 5% could continue working this effectively for a long time?

Christine
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Old Tue Nov 8, 2011, 08:31 AM
Marlene Marlene is offline
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Hi Christine,

Your son's situation is certainly complicated. Predicting anything with these diseases is really just a guess. Our doctor told us that what's important is peripheral blood counts. Like you already stated, somehow, the marrow can maintain almost normal counts even with low cellularity. And then there's the opposite, where some have pretty decent cellularity but their CBC is not.

When John went through treatment, we were told that most cannot survive beyond six months without a white count. At that time, John had no white cells and he was on multiple anti-biotic, anti-fungal and anti-virals. When his white cell started to produce again, they didn't come up to normal for very long time. He did fine with an ANC of .7 - 1 for a good year and a half. In fact, once his ANC got to .350, they started pulling the drugs.

With red cells, it's not as critical. John was dependent for two years on red cells. Of course you then have to deal with iron overload. But there are those who do fine for 10+ years getting red cell transfusions when only red cells are affected.

Platelets...you'd be surprised how well you can do with low platelets. Part depends on how well your platelets function. John was able to stop platelet transfusions once his platelets stabilized at 8K. He was transfusion dependent for 20 months and then it was slow climb.

There are so many variable, especially in your son's case.

Hope this helps.
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Marlene, wife to John DX w/SAA April 2002, Stable partial remission; Treated with High Dose Cytoxan, Johns Hopkins, June 2002. Final phlebotomy 11/2016. As of January 2017, FE is 233, HGB 11.7, WBC 5.1/ANC 4.0, Plts 146K.
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Old Wed Nov 9, 2011, 10:41 PM
Greg H Greg H is offline
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Quote:
Originally Posted by cfutia22 View Post
My son has no biological relatives and we will not put him through a bone marrow transplant with an unrelated donor (even if one could be found).
Hi Christine,

Marlene has given you excellent advice on the near impossibility of predicting what the marrow can and will do.

I respect your decisions about your son's health. But I detect in the sentence quoted above a perception that a transplant from a matched sibling donor may be superior to that from a matched unrelated donor. While this has certainly been the case historically, it is less and less so with each passing year. I recently heard an AA&MDS webinar on transplant for AA in which Dr. Joachim Deeg made this point.

This graph, though not specifically for transplants for DC (which is rare enough that there's probably not enough data to make such a chart), illustrates the point.

[IMG]
CIBMTR Survival Slides 2010-09-26 by hankins.greg, on Flickr[/IMG]

Again, I have complete respect for your decision as you have expressed it. I just wanted to share this information in case you hadn't run across it. My transplant doc is from India, and did by way of illustration, suggest to me at one point that it can be particularly hard to find unrelated matched donors for folks from that part of the world, because of the multiplicity of mixed ethnicities.

Good luck to you and your son.

Greg
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Greg, 59, dx MDS RCMD Int-1 03/10, 8+ & Dup1(q21q31). NIH Campath 11/2010. Non-responder. Tiny telomeres. TERT mutation. Danazol at NIH 12/11. TX independent 7/12. Pancreatitis 4/15. 15% blasts 4/16. DX RAEB-2. Beginning Vidaza to prep for MUD STC. Check out my blog at www.greghankins.com
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