Low risk MDS with declining numbers

[PaulS]

Hi - I've never posted before but have found the forums enormously helpful. I am 55 and was diagnosed with low risk MDS slightly over two years ago. Have been watching and waiting since, but in the last several months, after a bout of diverticulitis - my HG has declined to as low as 6 and I've needed several transfusions. We are planning to start lower intensity Vidaza treatment (5 days instead of 7) - and I am very nervous and not especially optimistic. If that fails they are thinking transplant. What should I expect from Vidaza - am I missing something or does it only work in a small percentage of patients? Anyone else go this route? Anything other thoughts/ideas? Thanks.

[bailie]

Welcome ! I think it is always good to start thinking transplant early on. Vidaza will work for about 40-50 percent of patients. I was on Vidaza for eight months and it most likely saved my life. What have been the results of your bone marrow biopsies? Blast count?

I wish you the best.

[Birgitta-A]

Hi Paul!
You know most studies show that Vidaza is working in about 50% of the high risk patients.

In the low risk patients the results are very different - from more than 60% responding patients to a negative result. Here is a study from ASH from Korea https://ash.confex.com/ash/2014/webp...aper72135.html. Eventually didn't the Vidaza patients get the best possible supportive therapy.

In EU Vidaza is not approved for low risk MDS.

Many low risk patients manage well with supportive therapy during many years while some patients have a more serious disease and should get a SCT early.
Kind regards
Birgitta-A
75 yo, dx MDS Interm-1 2006, tx dependent from dx with HGB 7.0 and very serious bone marrow fibrosis. Supportive therapy with txs, Neupogen for low WBC and iron chelators until 2010. Responded 3 years on Thalidomide and one year on Revlimid. Now tx dependent.

[PaulS]

Thanks for the responses - I think at this point supportive care would involve frequent transfusions - which doesn't seem like a good option. The hope is Vidaza would eliminate the need for transfusions - but not sure how realistic that is. My marrow is relatively OK - no blasts or mutations - and we had been hoping to watch and wait for longer...

[maggiemag]

Hi Paul, welcome to the forum. Have you had a BMB/A since your numbers have changed? My physician always insists on performing one whenever there has been a change. I understand that risk can sometimes change quickly. Also, growth factors like Procrit are considered supportive therapy as well as tx. Good luck with your treatment plan.

[DanL]

Paul,

Whenever something changes, a bmb/bma does seem to be in order to confirm that nothing has changed, or that it is time to pursue other options. Vidaza can take 4-6 cycles to begin showing results, so getting started earlier and having supportive care early may be helpful. I was in the watch and wait mode for almost 4 years with no supportive care, then one day in November of last year it all changed, HGB dropped quickly and I started Vidaza for a couple of months, then went to transplant in February. I relapsed in August and have been on Vidaza for 4 months with pretty good results - I am on a 5 day regimen, but it is the 7 day dosage compressed into 5 days.

I have seen and heard of people staying on vidaza for as long as 5 years, but that is pretty rare. There are also several clinical trials that are moving along nicely and may give additional options.

I wish you the best in choosing the next steps.

[Hopeful]

Paul,

What type of MDS do you have? Have you been tested for PNH?

[PaulS]

Hi and thanks again for the responses - I had a BMB in October after my numbers dropped and it was essentially the same as the first one. I'm not sure if I was tested for PNH - I did just have a number of tests to see if my cells were breaking up (hemolysis) - which were all negative. My diagnosis is refractory anemia with multi lineal dysplasia. The doctor thinks the drop might have something to do with the intestinal infection I had - that coincided with the first time I needed a transfusion. I also have recurring skin nodules - they look like hives but are more painful than itchy - which makes us think there is an autoimmune component to my MDS... Thanks again for the help.

[ill]

can you write at the first cbc(2012) and last cbc? rbc,hgb,mcv,plt,wbc

[PaulS]

I'm not sure about all the counts - we mostly talk about Hgb and Plt - the first Hgb was around 11.5 initially and gradually declined to about 9 in two years - then in the last few months it dropped to 7.5 when i was a sick and then to as low as 6 after I went 6 weeks without a transfusion. I've had four transfusions since October. Plt's started around 140 and have gradually declined - they were 88 at my last CBC - up slightly from what they'd been a couple weeks before. My WBC have always been in the normal range and have been pretty steady around 5,5 to 6.5 at my last CBC. The RBC and HCT have paralleled the Hgb, but I don't remember exactly what they've been. Thanks.

[Hopeful]

Hi PaulS,

Do you have significant dysplasia in multiple lines (over 10% in each blood line)? What is your bone marrow cellularity? Since you are thinking there may be an immune component to your condition, I would seek a second opinion before beginning Vidaza. With your young-for-MDS age and a MDS-RA subtype, immune suppression may be a more appropriate treatment to consider.

Also, drops in RBCs and Platelets while sick are expected. Look to see that they come back up when you are recovered!

[PaulS]

Thanks again for the advice and concern - I'm hyper cellular - if that is the question. Not sure the degree of dysplasia's and I can't find the biopsy report - I'll get it Monday. We did discuss cyclosporine - but I think she thinks the MDS is primary to the autoimmune and the Vidaza will interfere with the MDS process which will lead to the autoimmune symptoms going away along with the MDS symptoms. I'm supposed to start Vidaza on Monday - but I'll raise the question again.

[bailie]

What follows the Vidaza treatment? I started the Vidaza treatment with the stem cell transplant being the objective following Vidaza. Luckily a donor was found for me, but it took almost the entire Vidaza treatment time (eight months) to find the donor. The question I would ask would be "what follows Vidaza"?

[PaulS]

Good question - we're hoping the Vidaza works for a considerable time and then not sure - probably BMT if new drug doesn't come along. With low risk we don't want the BMT to kill me but on the other hand quality of life is also a concern if I'm getting very frequent transfusions. Really don't like any of the options. Baile - how are you doing now? All the best.

[bailie]

The Vidaza got my counts into the normal range. I knew (and the doctors knew) that I would not get better without the transplant. So we developed the plan from initial diagnosis while knowing that there could (and probably would) be variations of the "plan". Generally Vidaza has an indeterminable length of effectiveness.

Naturally everyone is different with these diseases/transplants. I am on Day +116 following transplant. I feel as good as before I had MDS. I fast walked four miles last week. Of course I am fully aware that this easily can change at any time. I have only had one transfusion and that was a day before the transplant (just after the chemo) when my platelets got to 8. I have zero regrets about the transplant because I probably wouldn't be alive today without it. I went into the transplant in good physical condition and I feel (and many studies confirm) that that is/was very important. In all of this it is important to be aware of optimum time for each procedure.

The thing that I soon realized is that there is no absolute course of action for so much of this. Education about this is so important so a person can work with the doctors in every phase.

[Birgitta-A]

Hi Paul!
If you look at the algorithm for MDS treatment you will see that a low risk patient with low HGB as the major problem often are treated with EPO drugs like Aranesp + eventually drugs like Neupogen if their own EPO initially is less than 500.
http://www.bloodjournal.org/content/124/18/2793

If that treatment fails they can try Vidaza.

Then we don't know if you have "no good" chromosome aberrations like problems with chromosome 7 or somatic mutations like TP53. If so it is important with a SCT even if you have low risk MDS.
Kind regards
Birgitta-A

[PaulS]

Thanks again for your responses - Bailie - I happy you are doing so well after the SCT - definitely inspiring. We did try Aranesp but without much luck. So Vidaza it is for now. I suspect the endgame will be a SCT but we're hoping to get as much time as possible feeling reasonably good before then - but at this point power walking four miles after an SCT sounds pretty good. Had first shot of Vidaza this morning - so far feel OK. Hemoglobin was 7.5 for we're on for another transfusion tomorrow as well....

[Birgitta-A]

Hi Paul!
Now we hope for an early and good response to Vidaza without adverse effects !
Kind regards
Birgitta-A

[bailie]

Paul, Vidaza is usually tolerated very well. I had no adverse effects except the expected reddening around the injection sites. I often was golfing the same day as the shots. I did notice that I felt a little sluggish by about the third or fourth day of shots. The only problem I noticed was from possibility of constipation from the anti-nausea pills which I took with each injection. Pay close attention to that because it can be very serious.

[PaulS]

Thanks again for the informative responses and kind thoughts. Completed two rounds of Vidaza without any ill effects so far, except minor pain around injection sites. I didn't take the preventative anti-nausea drug yesterday out of fear of constipation - and that seemed to work out - no nausea or constipation. Don't think I'm cured yet - but I'll keep plugging away. Thanks again.

[mdhihwjv]

Originally Posted by PaulS

Hi - I've never posted before but have found the forums enormously helpful. I am 55 and was diagnosed with low risk MDS slightly over two years ago. Have been watching and waiting since, but in the last several months, after a bout of diverticulitis - my HG has declined to as low as 6 and I've needed several transfusions. We are planning to start lower intensity Vidaza treatment (5 days instead of 7) - and I am very nervous and not especially optimistic. If that fails they are thinking transplant. What should I expect from Vidaza - am I missing something or does it only work in a small percentage of patients? Anyone else go this route? Anything other thoughts/ideas? Thanks.

I noticed on your post that you had/have recurring skin nodules. My late husband also had those for a while. Before his death in 2013, he was diagnosed with thrombocytopenia/pancytopenia at age 58 in 2006. In 2012, this changed to MDS which quickly changed to acute myeloid leukemia. He died in 2013 from complications of the chemo he was receiving in an attempt to prepare him for a bone marrow transplant. You may be aware that MDS, if no family history, is usually caused by environmental toxins. My husband was exposed to Agent Orange 1968-1969. Are you a Vietnam veteran, or do you have an occupational history which could have exposed you to toxins (like benzene and dioxin)? MDS is currently not on the presumptive list for AO, but many in the medical profession feel it should be. My husband also had diabetes and heart disease (both of which are on the list for AO).

[PaulS]

Hi - sorry to hear about the loss of your husband. I did not serve in Vietnam and to the best of my knowledge haven't been especially exposed to toxins other than what one normally encounters in life - I've never been in an occupation that handles or otherwise deals extensively with chemicals. Hope you are doing well.

Best wishes
Paul

[bailie]

Hello Paul,

What is your situation now with regards to Vidaza? How have your blood counts reacted? I just finished my second cycle of Vidaza and Sprycel. My counts have been heading in a positive direction except for RBCs (2.70). WBCs and neutrophils are back in normal range.

I wish you comfort and stamina.

[mdhihwjv]

Originally Posted by PaulS

Hi - sorry to hear about the loss of your husband. I did not serve in Vietnam and to the best of my knowledge haven't been especially exposed to toxins other than what one normally encounters in life - I've never been in an occupation that handles or otherwise deals extensively with chemicals. Hope you are doing well.

Best wishes
Paul

If you do not have a family history of this disease or have never had chemotherapy, something in your environment probably caused it. I know from working on my husband's VA claim that smoking, working in or around gasoline/fuel, furniture factories, medical labs, agriculture, and paper mills all put people at risk for MDS. The rate of this disease among Vietnam veterans is much higher than the general population. I wish you good luck.

[PaulS]

Hi - glad to hear your counts are heading in the right direction - I hope you continue to do well.

Thanks for asking - Unfortunately, the Vidaza didn't seem to do to much - and my bone marrow biopsy showed a genetic abnormality for the first time - and patchy but elevated blasts - as a consequence they want to transplant me while I'm still relatively healthy - almost weekly transfusions are getting depressing.

I'm talking to a couple of transplant doctors with slightly different techniques - and will make a decision with my oncologist as to the what she/we feel is the best approach. Good news is my brother is a match - I'll probably start the transplant process - vidaza for five days, chemo and transplant - in several weeks - definitely dreading it - but hopeful that I may be cured and restored to health. My goal is to be skiing by March.

So keep getting better - your perseverance is inspiring!

All the best
paul