Myelodysplastic syndromes (MDS) are a major and very personal target of this website. Because of this, I have found that I have been delaying the creation of this page as I have so much information I would like to include, all of it specific to an individual's FISH (cytogenetic, chromosomal) picture.
It also occurs to me that many patients that may stop by this site are missing information that could save their lives. Therefore I would like to "cut to the chase" if you will and direct you to where you should go in the meantime.
MDS is characterized by an excess of blasts in the Bone Marrow 5-20%. While you may avoid the thought of leukemiaterm when reading information on the internet, the ONLY difference between MDS and leukemia is that the term "leukemia" is only used when the number of blasts reaches 20% or more.
This is not all negative. A person with a 5q- deletion may take Revlimid and live quite a long time with no transfusions, but make no mistake about it, if you do not visit the doctors listed below you are risking being treated by a physician with little to no experience with this specialized field who will prescribe you chemotherapyterm which may be inappropriate. This site was created as an impartial advocate for the patient. I am not a physician and can not treat you. I, as the rest of the contributors here, am a research scientist. We are here to educate the interested patient on research that is being translated to the clinic, and where that is being done. If you have this disease you have both the time and ability to learn about it through sites such as these, so you should not leave your care entirely up to your physician or sue them when you do not get positive results.
I am so emphatic about this because a physician would lose his job should he refer you to another center. At the same time, if he refers away every one of these difficult cases he will never gain the experience needed to treat the diseases himself. We do not have this conflict of interest.
A typical example is this. A patient contacts me re their MDS, at which point they tell me of the cancer center they are going to. They are 66 years old and do not know their cytogenetic (gene) profile. I suggest that the center they are going to, although "famous", does not offer the full breadth of techniques available for MDS and they should consider going to one of those listed below. Because of the existing appointment, they go to this center. Upon returning I ask "How did it go?." "They seemed very good! They suggested Vidaza." Ok, Vidaza is a typical approach for MDS, it is a drug which attempts to reawaken dormant genes that might still function. "What did they say about stem cell transplants?" I say. "They said he is too old."
A 66 year old patient is not too old and this age group is transplanted every day at better centers. What they said, read between the lines is, "WE do not transplant patients of that age." The only cure for MDS or the leukemias at this time (besides CML/Gleevec) is a stem cell transplant. That is the only way a person will be cured of these diseases. Chemotherapy drugs like Vidaza can put a person into a remission (16%) for a period of time, in fact it may be used to reach a remission for stem cell transplant, but without a transplant the person will eventually relapse and they will definitely be weaker. Vidaza could also kill a "Good" MDS (5q-) and leave room for a "Bad" MDS (7-) to grow in its place. If you are not working with the intent to transplant at a moment's notice, then you are not prepared.
Elderly patients are perfect targets for non-myeloablative transplants. The theory behind a non-myeloablative transplant is that the radiation used for the past 50 years might not be necessary and it is in fact the donor's immune system which is destroying the MDS or leukemia upon transplantation (GVL - Graft Vs. Leukemia). Studies are going on today at the best centers in the country using this technique. Almost no patients die from the transplant, but they may die in the months following if the transplant doesn't hold or from infections, like any other chemo or transplant. If it does not work, all of the typical chemos are still available for that patient, but the transplant may not be available if the patient has taken other chemos previously. This is why it is important to pick the right one from the start, and stick with it.
My choice as the most personable and cutting edge at a major center:
-- Dr. Karen Ballen at Massachusetts General Hospital --
Dr. Azra Raza world reknowned MDS researcher and physician.
John Pagel, MD, PhD 206-667-1868 Fred Hutchinson Center Seattle. Amazing nursing and clean patient contact protocols, what you need when severely immunocompromised.
Research I admire but have not personally visited:
Dr. Rizzieri at Duke Non-Myeloablative Transplant
Steven Goldstein, M.D. 215-615-3138 Pennsylvania Non-Myeloablative Transplant
The University of Minnesota and Moffit Center in Tampa are also at the cutting edge.
If you cannot go to these centers, find a haemotologist who trained there.
The last point I would like to leave you with is that patients expire for the same reasons under chemotherapy as they do from a stem cell transplant, and that is infection. Do not be so afraid of the therapy that you disregard its availability. The stigma of a bone marrow transplant is very strong, bringing to mind images of bones being slit open. For this reason the term "Stem Cell Therapy" is becoming more widely used. The truth is that your marrow is rarely used anymore. The haematopoetic (blood) stem cells are mobilized to your blood stream using Granulocyte Colony Stimulating Factor and they are spun out, replacing your own blood as it goes.
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