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Clinical Trials Considering or participating in research studies

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  #1  
Old Fri Aug 12, 2011, 02:30 PM
Al's Wife Al's Wife is offline
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Need help - Sapacitabine or Estybon or help

Our news this week was not the result we were hoping for. After fighting with our medicare advantage insurance company for two months and trying to seek other treatment options (finally filing a complaint with Medicare), we were approved for treatment at M. D. Anderson Cancer Center. Al had his bone marrow on Monday and it showed 10% blasts, platelets 22,000, and a progression from RCMD-1 to high risk. They are now talking to us about stem cell transplant not too far down the road. To say we are devastated is putting it mildly. We had hoped that with him not requiring any transfusions so far and his numbers being stable, we were in a good place. But now things seem to be moving rapidly, and in the wrong direction.
We were given three options at M. D. Anderson for now. Because we would have to stay in Houston pretty much the whole time if we choose the Estybon (it is IV), the doctors recommended he go on Sapacitabine, which is in pill form and we could come back home after the first month, and just go back every few weeks after that, I think.
But I want him to have what is BEST, even if it means moving to Houston. Does anyone have any knowledge of how successful either of these trials have been? I've tried reading online and can't seem to grasp the findings so far.
As I said above, his platelets are the main problem right now. He has complex cytogenetics, however. The doctors have told us that his other blood lines will go down - the wbc and rbc. However right now they are holding close to normal.
If anyone has ANY knowledge or opinion about the best drug for him, I would really appreciate your input.
We considered (and were approved) to go to NIH for the Promacta trial but when his blasts increased it disqualified him for that trial.
Thanks for your help.
Linda
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Linda, Al's wife, 75; dx MDS 5/2010; Vidaza 6/2010; ARRY614 & Sapacitabine clinical trials at Emory, no results, stopped 12/2011. Had BMB at NIH on 6/5/12, blasts 10-15% so he's not eligible for trial there. :eek Promacta trial, Tampa, blasts 25-30% 8/17/12 AML, trying Dacogen now and praying.
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  #2  
Old Fri Aug 12, 2011, 06:22 PM
DanL DanL is offline
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Linda,

Are Vidaza and Dacogen off the table? I didn't see that your husband had been treated with any hypomethylating agents as yet. They both have pretty well-tested results for higher risk mds patients and are considered the treatments of choice for most patients. Vidaza in particular is recommended under the NCCN guidelines for MDS patients with low platelet counts.

Recent studies on Estybon seem to indicate that it can prolong life for people who have already failed hypomethylating agents, and does not have significant side effects such as bone marrow suppression. Early observed response rates are as high as 50% (includes all response from complete response down to hematological improvement), but the complete response and marrow complete response is up to 36%, which seems pretty good. It is currently entering phase II/III testing where they are randomizing patients to see if it is better than best supportive care. See this article in the MDS Beacon:

http://www.mdsbeacon.com/news/2011/0...ogen-eha-2011/

Sapacitabine is in phase II trials I believe. It appears that the best response rate is about 35%, which includes complete response, marrow complete response, partial response, and hematological improvement. This compares to rates over 50% for Vidaza and Dacogen. See the report in the link below:

http://www.news-medical.net/news/201...ng.aspx?page=2

I believe that MD Anderson has been using oral Vidaza in trial with pretty good results as well. It sounds like there may be several options available at this time.

I do hope this helps a little. Good luck to you and your husband!
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MDS RCMD w/grade 2-3 fibrosis. Allo-MUD Feb 26, 2014. Relapsed August 2014. Free and clear of MDS since November 2014 after treatment with Vidaza and Rituxan. Experiencing autoimmune attack on CNS thought to be GVHD, some gut, skin and ocular cGVHD. Neuropathy over 80% of body.

Last edited by DanL : Fri Aug 12, 2011 at 06:22 PM. Reason: incomplete original entry
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  #3  
Old Fri Aug 12, 2011, 07:07 PM
Al's Wife Al's Wife is offline
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DanL,

Thanks for your reply. He was first tried on Vidaza when diagnosed in May 2010 and showed no improvements after six months. He also did a clinical trial of ARRY614 with no results.
At M. D. Anderson they said (because of logistics, I think - us living in Georgia and commuting back and forth) they would first try him on sapacitabine and if no results then they would try Estybon. I'm not sure, but maybe we should try the Estybon first even though it will mean us staying in Houston for longer periods.
I'm just the caregiver, but he leaves it all up to me, and I'm so afraid I'm not going to make the right decision. It's terrifying.
Thanks again for your input.
Linda
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Linda, Al's wife, 75; dx MDS 5/2010; Vidaza 6/2010; ARRY614 & Sapacitabine clinical trials at Emory, no results, stopped 12/2011. Had BMB at NIH on 6/5/12, blasts 10-15% so he's not eligible for trial there. :eek Promacta trial, Tampa, blasts 25-30% 8/17/12 AML, trying Dacogen now and praying.
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  #4  
Old Fri Aug 12, 2011, 07:35 PM
Greg H Greg H is offline
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Hey Linda!

I think it's really important to understand that, with this disease, there is no right decision. And there is no wrong decision.

The only right decision is to keep fighting. And the only wrong decision is to quit.

You're fighting; that's the right decision.

I'm reading the same numbers as Dan; the Estybon response rates are higher. But MDS is such a strange disease; it could be that the variety Al happens to have would respond better to Sapacitabine. There is just no way to know. You just have to go with one, and, if it doesn't work, try the other. That's probably why the Houston docs are suggesting trying the more convenient one first.

It might be worth asking them about the expected side effects of the two drugs. The early results from Estybon say there were no hematological side effects -- that is, no reduced counts. But the Sapacitabine results do mention some neutropenia and thrombocytopenia. There's no data in the abstract about how common or severe these were. The docs in Houston should be able to tell you what they have seen in real life. With Al's platelets at 22,000, you don't want to see them dropping further if you can avoid it.

I know you've already had an heck of a fight with your insurer to authorize Anderson, so you might not be interested in other locations, but the Phase III trial of Estybon that they are doing in Houston is also being done at several locations in Florida, as well as in some other states (not in Georgia, however). You can find the list here on clinicaltrials.gov. I'm pretty sure it's the right trial.

Believe me, there's no wrong decision here, just tough decisions.

Take care and good luck!

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

Last edited by Greg H : Fri Aug 12, 2011 at 07:35 PM. Reason: Forgot to subscribe
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  #5  
Old Sat Aug 13, 2011, 01:42 PM
Al's Wife Al's Wife is offline
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Greg,

As always, thank you so much for your input. I looked through the trials in Florida but don't think our insurance will cover. However, I did find that they are doing the same clinical trial here at Emory that they want to put him on in Houston. He was on Arry614 clinical trial at Emory until April of this year. I'm thinking that the reason they did not recommend Sapacitabine when he had no response on the Arry614 is that he was still at low risk Int-1 at that point. But now that he has 10% blasts they may say that the Sapacitabine is an option. I have put a call in to our doctor at Emory to call us first thing Monday morning. Why is it that you always need an answer on the weekend? It would be a sure answer to pray if we could be treated locally. I just wish that the Estybon were being offered locally.
But like you said, if we try the Sapacitabine and it doesn't show improvement, then we may be able to then try the Estybon.
They are wanting us to go ahead and get everything in order for a stem cell transplant in the future. While we were set up to talk to a doctor at M D Anderson about SCT, if we are able to do this trial here at Emory, I'd want to talk to the doctor here instead. I would much rather be here near home for transplant than Texas. Of course I want him to get the best care. We'll just have to wait and see.
I'm sorry, I know I'm talking all over the charts. Just anxious to get something working in our favor.
Thanks again for your help and encouragement.
Linda
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Linda, Al's wife, 75; dx MDS 5/2010; Vidaza 6/2010; ARRY614 & Sapacitabine clinical trials at Emory, no results, stopped 12/2011. Had BMB at NIH on 6/5/12, blasts 10-15% so he's not eligible for trial there. :eek Promacta trial, Tampa, blasts 25-30% 8/17/12 AML, trying Dacogen now and praying.
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  #6  
Old Sat Aug 13, 2011, 02:08 PM
Greg H Greg H is offline
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Linda,

It's great to hear that the trial is offered locally!

I know from what I've read about SCT that it is a long haul, and often involves an outpatient time when you are living near the transplant center but going in most every day. It sure would be nice to be closer to home during that period. It also might mean, if you have relatives nearby, that you, as caregiver, would have a greater opportunity for a little respite now and again.

Given that Al is over 70, it might also be a good idea to check the track record of Anderson and Emory with older transplant patients. I would guess they'd be doing reduced intensity conditioning, so you'll want to make sure the center you choose is good at that.

Not to be nosy, but I am curious, if you don't mind my asking. Is the problem obtaining coverage because you are enrolled in a Medicare Advantage program instead of regular Medicare? In other words, if you had regular Medicare, would crossing state lines be as big a problem? It has always seemed ridiculous to me that our health insurance in the US usually only works effectively in one state.

Take care and good luck.

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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Old Sat Aug 13, 2011, 02:36 PM
Al's Wife Al's Wife is offline
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Yes, Greg, we have a Medicare Advantage plan and I have had to fight with them every step of the way, even for treatment here in Georgia. When they refused his treatment at M D Anderson, I filed a Complaint against them for delay, and miraculously two days later, they agreed to let us go to M D Anderson. However, M D Anderson (because they have had so many problems with getting paid from this particular company) made them sign a letter of agreement. And I didn't know until we got to M D Anderson that they only agreed to pay 75%. The business office said all the other Medicare Advantage insurance companies pay 90%. You can rest assured that come new enrollment for 2012, we will go with straight Medicare and get a supplement. I never want to be in a position again where an insurance company tells me I can not get the care my husband needs. The disease is heartbreaking enough, but when you have to fight the insurance company too, it becomes overwhelming.
And as you know, the pharmaceutical company pays for the actual drug, but not the labs, bone marrows, etc. which are very expensive.
I'll keep you posted.
Linda
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Linda, Al's wife, 75; dx MDS 5/2010; Vidaza 6/2010; ARRY614 & Sapacitabine clinical trials at Emory, no results, stopped 12/2011. Had BMB at NIH on 6/5/12, blasts 10-15% so he's not eligible for trial there. :eek Promacta trial, Tampa, blasts 25-30% 8/17/12 AML, trying Dacogen now and praying.
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  #8  
Old Sat Aug 13, 2011, 05:03 PM
Greg H Greg H is offline
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Linda,

That just stinks. I know we're a big country, but we really need a health insurance and health care system that doesn't tie you to a specific state. With a disease like MDS, it may be that the type you have really does ned to be treated in a center o the other side of the country.

Since my clinical trial was at NIH, I didn't have to deal with that problem, since they pay for everything that happens on their campus. I did have to cover the cost of the local CBCs and other blood work, though, since that was done in my home state, my health insurance picked up most of the tab. But, with those labs, bone marrows, and transfusions, I will definitely hit my out of pocket max before the end of the year.

Thanks for the explanation.

Good luck, and do let me know if I can help dig any more info out of the internet as you make your decision.

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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  #9  
Old Sat Aug 13, 2011, 08:26 PM
Al's Wife Al's Wife is offline
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Greg,

You might have read that we were approved for Promacta at NIH, but received the approval AFTER we got our appointment at M. D. Anderson. Then when the blast numbers came back at 10%, that disqualified him. I just hope, since the Medicare Advantage plan paid earlier for his care at Emory this year, that they will not balk at him going back to Emory, if we are able to get in the clinical there. Monday can't get here soon enough for me.
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Linda, Al's wife, 75; dx MDS 5/2010; Vidaza 6/2010; ARRY614 & Sapacitabine clinical trials at Emory, no results, stopped 12/2011. Had BMB at NIH on 6/5/12, blasts 10-15% so he's not eligible for trial there. :eek Promacta trial, Tampa, blasts 25-30% 8/17/12 AML, trying Dacogen now and praying.
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  #10  
Old Sun Aug 14, 2011, 04:55 AM
Birgitta-A Birgitta-A is offline
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Sapacitabine or Estybon

Hi Linda,
You have already got very good info from Dan and Greg I will only show very short results from the studies:

Sapacitabine
Patients: High risk MDS treated with Vidaza or Dacogen
Response: 35% when the patients received 300mg b i d x 7 days
Adverse effects: low counts in all three cell lines

Estybon
Patients: High risk MDS treated with Vidaza or Dacogen
Response: 54%
Adverse effects: No suppression of the bone marrow

Then we know that because MDS isn't one disease but many we don't know who will respond (yet) as Greg wrote.
Kind regards
Birgitta-A
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  #11  
Old Sun Aug 14, 2011, 05:34 PM
Sally C Sally C is offline
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Hey Linda,
I will say a prayer that Emory will offer the same clinical trial and will accept Al. That would be wonderful.
God Bless,
Sally
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