New Promacta (Eltrombopag) Clinical Trial at NIH [Archive]

Sally C

Thu May 12, 2011, 11:33 AM

My husband has hypocellular MDS with refractory platelets and red cells. He received Campath at NIH in April 2009. He has just started in a brand new clinical trial for Promacta (http://clinicaltrials.gov/ct2/show/NCT00961064) for low to Int. 2 risk MDS patients. They have had good success with AA patients. It is specifically for platelets but it has also helped with red cells as well. Don is their very first "guinea pig" and they have a second one coming in next Monday. They are very anxious to get additional MDS patients for the study. Here is the contact info for the clinical trial. NIH is absolutely wonderful!!! Extremely accessible!
God Bless,
Sally

Barbara Weinstein RN BSN CCRP
NHLBI/ Research Nurse Specialist
Building 10 CRC Room 5130
10 Center Drive
Bethesda, MD 20892
phone: 301-594-4180
fax: 301-594-1290

Neil Cuadra

Thu May 12, 2011, 12:51 PM

Sally,

Is this the trial?
A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS) (http://clinicaltrials.gov/ct2/show/NCT00961064)

Sally C

Thu May 12, 2011, 01:11 PM

Yes Neil, it is. Can't figure out how to put your link on my post though. Can you put it there?
Thanks!
Sally

Neil Cuadra

Thu May 12, 2011, 01:38 PM

I added the link to your post.

Thanks for participating in a trial, which has the potential to help your husband but also all future patients.

Sally C

Thu May 12, 2011, 01:44 PM

Very cool, Neil! Thanks for doing that.
And these clinical trials are such a blessing. I truly believe my husband wouldn't still be here were it not for the Campath clinical trial.
Thanks again!
Sally

Birgitta-A

Thu May 12, 2011, 02:05 PM

Hi Sally,
Very interesting for us all to follow your husband's result with Promacta! I have been looking at Promacta and the other drug for low platelet called Nplate since my dx 5 years ago and I do hope that your husband will have a positive result without adverse reactions. Neil is right when he thanks him for participating in the trial, so we all sooner or later perhaps will be able to get that drug.
Kind regards
Birgitta-A
72 yo. Platelets 65 at dx 2006 and slowly decreasing to 22 2010. Now about 100 after treatment with Thalidomide

Sally C

Thu May 12, 2011, 02:40 PM

Hi Birgitta-A,
What an honor to hear from you. I remain amazed at your knowledge of these diseases. I thought I was fairly well educated in MDS until I started reading Marrowforums. I realize I haven't even scratched the surface.
My husband's platelets never responded to Campath (4/09). The whites responded as well as the reds to some extent. He has done pretty good since 2009 even though his platelets have stayed in the teens except after transfusions. His reds have stayed in the 7 to 9 range even after transfusions. I think he has hit 10 maybe once. It's amazing how the body can adjust.
NIH really hasn't expected a response to Promacta until after he started taking the full dosage, which was yesterday (5/11/11) so we'll see. At least that has been the case with their AA patients.
Thank you so much for all your input to the forums. I am sure you have been instrumental in educating many of us.
God Bless and take care,
Sally

tytd

Thu May 12, 2011, 08:21 PM

Thanks very much Sally and to your husband for informing us about your experience with the new trial. I had inquired about this trial which , I think, is also available at Moffitt as well as NIH. Right now, my platelets are hanging steady but it is good to know that this trial is open in case they crash. When I read the inclusion criteria for the trial I was under the impression that one had to have failed other standard therapy like Vidaza but it is good to know that that may not be the case. I hope that your husband has a good response without side effects to the drug. Tytd

Sally C

Thu May 12, 2011, 10:00 PM

Hey tytd,
So glad this was helpful to you. Don's only treatments have been Campath at NIH in April, 2009 - Cyclosporine in July, 2010 thru March, 2011 (all that did was make his hair grow fast, curly and wavy - and as his hairdresser I was glad when he stopped taking it!). He received bi-weekly Aranesp shots for quite a while until he started the Promacta. I don't know that it ever helped much but don't know how he would have done had he not taken it.
So far he has tolerated the Promacta very well. Now that we're at max dosage he should start seeing some response if he is going to respond.
I wish you well. God Bless.
Sally

Birgitta-A

Fri May 13, 2011, 04:27 AM

Hi Sally,
Thank you for the kind words! I really don't understand why I can't find any preliminary reports from the trials with Promacta in MDS patients - they started long ago. At least 2 members of this forum (Kirby is one) have received Promacta off record with positive result.
Kind regards
Birgitta-A

Sally C

Fri May 13, 2011, 07:14 AM

Good morning Birgitta-A,
Our oncologist asked me to get some info from NIH on Promacta so she could learn about it. I was told from NIH that they couldn't release any information about the drug while it was still in trial. I don't know if that is an NIH rule or Smith Kline's but that is possibly why you can't find any preliminary studies at this point.
Have a great day!
Sally

Birgitta-A

Sat May 14, 2011, 05:37 AM

Hi Sally,
You know they started this trial May 2009:
http://clinicaltrials.gov/ct2/show/NCT00903422?term=eltrombopag+mds&rank=1
Often they show preliminary results when they have about 30 patients in the trial - for Nplate they showed preliminary results early.

We hope everything is alright with the trial.
Kind regards
Birgitta-A

Sally C

Sat May 14, 2011, 09:13 AM

Hi Birgitta-A,
I was aware that NIH had Promacta in trial before the one my husband is in. They also have been testing on AA patients. We were told that because of the success of Promacta on AA patients they are expanding the trial to low to Int. 2 risk MDS patients. Also during our visit last week we were told the risk for reticulin fibrosis is not as big a concern as previously thought. So my reading into what information we have received regarding ongoing trials along with the fact that they are further expanding the Promacta trials makes me believe they are having some success as well as less concern for the safety of the drug. At least I hope that's what it means. Don's MDS has some AA characteristics which is why they believe he will respond to the Promacta. As I mentioned before - he is the first MDS patient in this particular clinical trial. The second one is coming in next week. All I can guess is they don't want to release any information with new trials starting. When I was told that NIH couldn't release any Promacta trial information to our oncologist, I wasn't told whether that was due to NIH's rules or GlaxoSmithKline's. Don is at full dosage now and is tolerating the medication very well. Will let you know if and when we see a response. And if you do find any preliminary results I know you will share that with the forum.
Thanks as always for your interest and research.
Have a great day!
Sally
I have just read some past postings from you, Kirby and others regarding concerns about Promacta. I saw that Kirby's dosage, at least at that posting, was 75mg/day. Don's is now 150mg/day. Yikes! I also read Birgitta where you said you thought platelet transfusions were preferable to taking Promacta. One of the concerns regarding Don's platelet transfusions was that he continually had reactions to the platelets, although he hasn't reacted to the last few. Our oncologist said it was just a matter of time before he had a reaction that would result in anaphylactic shock. As you know, with this disease you have to pick your poison. Hopefully we have made the right decision. I do trust the doctors at NIH. They certainly don't want bad results or to do harm to their patients. But they don't have a crystal ball either. All we can do is just pray and hope for the best. I do appreciate everyone's candor regarding this drug and would appreciate any input - good or bad - that anyone has.

Birgitta-A

Sun May 15, 2011, 04:45 AM

Hi Sally,
You are right about the concern about the risk for fibrosis - they have now started the third trial for MDS patients and the second for AA patients.

I suppose Don is in this trial: http://clinicaltrials.gov/ct2/show/NCT01286038?term=eltrombopag+mds&rank=2

You know when the two new drugs for low platelets were approved I was hoping that they would soon be in trials for MDS because they were in trials for several other diseases with low platelets due to chemo like Hepatitis C. Then I was scared because of the reports about increased fibrosis and blasts after treatment with Nplate. At that time I wrote that platelet transfusions were safer though I know that most patients won't have a good effect after many platelet transfusions. Now I have changed my mind again - perhaps Promacta will be a good drug for MDS patients.

Hope Don will have a positive response!
Kind regards
Birgitta-A

Sally C

Sun May 15, 2011, 02:05 PM

Hi Birgitta-A,
Thank you for your post. It made me feel better as I trust your judgement and knowledge. The trial that Don is in is in Bethesda, Md.
"A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)"
http://clinicaltrials.gov/ct2/show/NCT00961064
Thanks as always for your input.
Take care,
Sally

Birgitta-A

Mon May 16, 2011, 07:52 AM

Hi Sally,
Then Don is participating in the second trial for MDS patients - as you can see this study was presented in 2009 and should have 30 patients. I think everything with Promacta seems to take very long time for MDS patients. Now we can only hope that the drug will have good effect - as you know bleedings is a very common death cause in MDS. Only infections are more dangerous.
Kind regards
Birgitta-A

Sally C

Mon May 16, 2011, 08:18 AM

Hey Birgitta-A,
You are correct that this is the second trial for MDS in addition to the trial/s for AA. Don is the first patient for low to Int. 2 MDS. I believe the other trial is for high risk MDS and maybe AML as well.
I have been told exactly what you said - that most MDS patients succumb to infections and bleeding as opposed to the MDS/progression to leukemia.
Don's whites are the only line that have normalized. But lately, the platelets and reds have slowed their drop but the whites have been going in and out of normal. His neutrophils were 1.5 last checked. Seems when he gets one line doing better another line goes down.:confused: He will get a CBC tomorrow (5/17) - his first since reaching max dosage (150 mg.) last Wed. May still be too early for a response but we'll see.
I'll keep you posted.
Thanks again and take care,
Sally

Sally C

Thu Aug 4, 2011, 08:16 AM

Hi all,
Just wanted to give you an update regarding my husband's participation in the Promacta clinical trial at NIH for platelets.
His platelets are holding in the 20,000's which is a record for him. Also he went 8 weeks without a platelet transfusion which is the longest he has gone since January, 2009. He recently received a platelet transfusion for a colonoscopy - not because they had droppped. They have also had some success with increasing red cells with their AA patients. He has tolerated the medication very well and has had no side effects that we know of. NIH in Bethesda is following him very closely. We were sending them weekly blood work but now that we're in extended study we'll send it once a month. They also will do bone marrow biopsies every 6 months since fibrosis is a concern. There was no increase in fibrosis with his first biopsy since starting the trial. It is our understanding that there are now 4 patients in trial and they are looking at a fifth. Barring any problems he will continue in the trial until the study is over which will be in Dec., 2012. At that point they will help us get into a program that will give financial aid to buy the drug ourselves. As you know, when in clinical trial all health-related expenses for any procedures done in Bethesda are paid for by NIH as well as the Promacta. For anyone interested here is the contact information:

Barbara Weinstein RN BSN CCRP
NHLBI/ Research Nurse Specialist
Building 10 CRC Room 4-5362 10 Center Drive
Bethesda, MD 20892
phone: 301- 594-4180
fax: 301-402-3088
Here is the link to the trial:
http://clinicaltrials.gov/ct2/show/NCT00961064

Please feel free to contact me with any questions you may have.
God Bless,
Sally

Birgitta-A

Fri Aug 5, 2011, 11:56 AM

Hi Sally,
Wonderful that your husband is responding to Promacta! Hope the response will continue and that he won't get fibrosis or other adverse reactions!
Kind regards
Birgitta-A

Sally C

Sat Aug 6, 2011, 07:20 AM

Hi Birgitta-A,
Thank you for your note. You are such a great support to everyone as well as an unending wealth of information.
I hope you're continuing to good as well. I'll keep you posted.
Best wishes,
Sally

Birgitta-A

Sat Aug 6, 2011, 12:06 PM

Hi sally,
Thank you for the kind words :)! I am still responding to Thalidomide - I don't need Exjade for iron overload now and have started to taper Neupogen for low WBCs.
Kind regards
Birgitta-A

Al's Wife

Wed Aug 10, 2011, 08:14 PM

Sally,

I sent you a private message regarding NIH. If you could respond when you have time. Thanks, Linda

Sally C

Sat Sep 17, 2011, 10:00 AM

Hello friends,
I just wanted to report how Don is doing on Promacta.
He started Promacta in March, 2011. On 9/13 his platelets were 30,000. His last platelet transfusion was 7/27 and that was to get a colonoscopy. The last needed platelets were 8 weeks before that. His HGB has perked up as well - as it did with NIH's AA patients. On 9/13 it was 9.7. He has been RBC/platelet transfusion dependent since 1/09.
His WBC is the highest it's been since all this started as well - 5.9. I did think it was strange that his neutrophils were almost as high - 5.1 - which made his Neutrophil % high at 84.8 - high normal is 74. Any input?
He did have a cardiac scare last week-end - ended up in ER/CCU overnight and was diagnosed with Atrial Flutter (racing heart) for which he now is on medication. Age (66) and hypertension are two of the risk factors. I haven't been able to find anything to link this to Promacta.
The one concern we have at this point is they found the beginnings of an unusual type of cataract in both eyes- subcapsular - which is at the back of his eyes rather than the usual front*. Cataracts are a known side effect. They will check again on 10/25. If there are changes we'll have to go off study. He will also have another bone marrow biopsy in Jan., 2012 to check for any negative marrow changes.
He is tolerating the Promacta (150mg./day) very well. No side effects that we are aware of.
Will keep you posted.
God Bless,
Sally
Wife of Don - MDS - RAMD - Int. 1 - diagnosed 1/09 - 46,XY,del (20) (q11.2q13.3) (18) / 46,XY (2) - Campath at NIH 4/09. Has been transfusion dependent since 1/09. That may be changing with Promacta.
p.s. As an aside - Don has gone from hypocellular to hypercellular but this showed up in a Nov. 2010 biopsy - pre-Promacta.
*It is at the back of the lens rather than the eye.

Al's Wife

Sat Sep 17, 2011, 01:41 PM

Sally,

Good to read how Don is doing on the Promacta. Hope things continue to improve. Sorry to hear about the heart scare; but, hopefully, it wasn't anything too serious. It's hard enough dealing with the MDS without all these other problems cropping up.
We're trying to keep a positive attitude on this Sapacitabine trial and should know something in a few weeks after Cycle 2, which begins next Wednesday.

Birgitta-A

Sat Sep 17, 2011, 01:52 PM

Hi Sally,
Too bad with the Atrial Flutter! Hope Don's heart will be OK with medication.

You know subcapsular cataract is cataract in the back of the lens not in the back of the eye. Here is a report about Promacta from the EMA in Europe corresponding to FDA - Promacta was approved later in Europe so this report is from 2009.

If you look at page 58 you will find adverse reactions regarding the eyes. They don't write what type of cataract the patients developed. The Promacta company has an ongoing study called LENS where they look for the risk for cataract in patients with Immune Thrombocytopenic Purpura (a bleeding disease due to a very low platelet count).
http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/001110/WC500089967.pdf

Very positive that all counts have increased :)! I am afraid that I don't know what a high Neutrophil % means.

Hope the cataract will be the same and not worse at the control 10/25!
Kind regards
Birgitta-A

Sally C

Sat Sep 17, 2011, 06:33 PM

Hi Linda,
Thank you for your kind note. Have been keeping up with your posts. I certainly wish you and Al the best. Please keep us posted.

Hi Birgitta,
Thank you for the clarification of a subcapsular cataract. This was new to me.
I pulled up your 70 page link. After skimming through it my eyes are now crossed. You and I need to sit over a case of wine ;) and let you explain all of that to me.
Thank you too for your kind words and your willingness to always share you vast knowledge and support.

Best wishes to you both and will keep you posted.
Sally

Birgitta-A

Sun Sep 18, 2011, 12:16 PM

Hi Sally,
It should have been very nice to meet you and discuss Promacta but I live in Sweden and only travel in my own car because I am so afraid of infections.

You know I have been waiting several years for reports on Promacta in MDS patients - many of us have low platelets and need treatment. Good that Don is participating!

In the report from Europe you can see that 7% of the patients got some kind of eye disorders - 2% was cararact, 1% dry eye, 1% blurred vision, 1% lenticular opacities and 1% other diseases.

2% is not much considering that the study patients had the autoimmune bleeding disease ITP. Many of these patients had been treated with Prednisone or similar drugs that can give cataract. The problem is that rats got cataract when they got large doses of Promacta and that is why they suspect that cataract could be an adverse effect.

Did they control Don's eyes before he got Promacta?
Kind regards
Birgitta-A

Sally C

Sun Sep 18, 2011, 01:34 PM

Hi Birgitta,
They did do an extensive exam of Don's eyes before starting Promacta. The doctor made note of something she saw in his eyes but didn't take any pictures so we don't know if it was the beginnings of cataracts - which is what they saw with his 2nd exam. They took over 100 pictures at his 2nd exam so they should have what they need for comparison. You mentioned Predinose - Don is on a 2 month slow taper of Prednisone for a colon inflammation. Started at 20mg. per day for 2 weeks and has been dropping 5mg. every 2 weeks until he finishes the last 2 weeks at 5mg. per day. I don't know if Predinose is the only steroid that can cause cataracts. If other types affect it, that could have worked against him as well as he always gets an IV dose of Solu-Medrol - which is also a steroid - with every transfusion to help prevent reactions.
Thank you for answering as always and will keep you posted.
Take care,
Sally
Well Birgitta, just out of curiosity I looked up Solu-Medrol and one of the side effects they specifically noted was posterior subcapsular cataracts. You are truly a storehouse of knowledge. I'll swear with these drugs you're damned if you do - damned if you don't. You just have to pick your poison. Thanks again for the information.

Birgitta-A

Mon Sep 19, 2011, 07:06 AM

Hi Sally,
Yes most drugs are dubble edged swords. You know I am so afraid of adverse effects of drugs that I only accepted supportive therapy the first 4 years after dx. Now I take a very low dose of Thalidomide and 5 mg Prednisone with good effect on my counts. I can only hope that I won't get any adverse effects.
Kind regards
Birgitta-A

Sally C

Fri Jan 27, 2012, 01:08 PM

Hello all,
Just wanted to update you on how Don's doing in the Promacta clinical trial he started in March, 2011 at the National Institutes of Health in Bethesda, Md.
First of all he has tolerated Promacta without any problems whatsoever.
He has been transfusion independent since spring of 2011 except for one transfusion of red cells in Oct. He had been transfusion dependent since 1/09 with approx. 60 transfusions in '09 and the same in '10 - for both red cells and especially platelets.
He has gone from RBC's in the 7 and 8's to 11.2 this week. (1/25/12). His platelets had been totally refractory since '09 - low point being 4,000 but for the most part staying in the teens or lower. They were 61,000 this week as well.
They did a BMB this month and I will paste the summary from our doctor we received yesterday. I might add that he received Campath in April, 2009 with a minor partial response and received Cyclosporine from late fall, 2010 until starting the Promacta in March, 2011 with no response.

"Everything looked good. Overall, his bone marrow was 40-50% cellular which is a little more than last July. There was no increase in blasts and no change in overall fibrosis (still 2 out of 4). His chromosome analysis still shows the deletion 20q in 90% of the cells analyzed. There were no other chromosomal abnormalities.
...His bone marrow has more cells than it did in July which makes sense since his blood counts have improved. Overall, it isn't much different than the July marrow. I think it was 30-40% cellular at that time if I'm not mistaken. Someone Mr. Calvert's age should have about 35-40% cellularity, but with MDS that can vary.
The main thing is that the cells we saw looked good as opposed to there being blast cells (which is what we do not want to see!)."

They only have 4 people in the Promacta/MDS trial now so I'm sure they are more than open for new patients.
I would appreciate any input from the Marrowforums experts re: the increase in cellularity and the fact that the deletion 20 was in 90% of the cells analyzed. Do either have any meaning in the overall picture?
If anyone has any questions please feel free to ask.
Thanks so much and God Bless.
Sally

Birgitta-A

Fri Jan 27, 2012, 02:05 PM

Hi Sally,
Congratulations to Don and you :)! Wonderful results with very good counts and no increase in fibrosis or blasts in the bone marrow!

As you know del20q is a "good" chromosome aberration. Promacta is no chemo so I don't think you could expect any decrease in the % of cells with the chromosome aberration.

As far as I understand increased cellularity should be a good sign.
Kind regards
Birgitta-A

Sally C

Fri Jan 27, 2012, 02:18 PM

Thank you Birgitta,
You are not only a great source of knowledge - you are everyone's cheerleader.
Thank you for your kind words of support.
God Bless,
Sally

Greg H

Fri Jan 27, 2012, 09:23 PM

Hey Sally!

That is truly great news! Glad to hear it.None of those BMB results sound bad at all, as Birgitta has said.

Congratulations!

Greg

Sally C

Sun Jan 29, 2012, 01:35 PM

Greg,
Thank you so much for your kind words as well. You too are a storehouse of knowledge and a great support for all. Your wit and way with words not only make your posts enjoyable - you make difficult information easy to understand.
While I'm here I want to again post the MDS/Promacta clinical trial contact information that has helped Don's platelets so much.
Thanks again Greg!
Sally

Barbara Weinstein RN BSN CCRP
NHLBI/ Research Nurse Specialist
Building 10 CRC Room 5130
10 Center Drive
Bethesda, MD 20892
phone: 301-594-4180
fax: 301-594-1290

Birgitta-A

Sun Jan 29, 2012, 02:29 PM

Hi Sally,
Thank you for giving info about Promacta trials. You know when I got my dx 2006 with platelets at 65 I understood that I should need treatment for low platelets. I have been following Promacta and the other drug for low platelets called Nplate during 6 years now.

There are several studies in MDS patients about Nplate and we know since several years that Nplate can give increased fibrosis and blast in MDS patients.

There are still no reports about Promacta in MDS patients but in cell lines from MDS patients the drug has had a positive effect.
Kind regards
Birgitta-A

Sally C

Sun Jan 29, 2012, 02:38 PM

Hi Birgitta,
Do you know how long it took for Nplate to cause problems with fibrosis and blasts?
Will keep you posted on Don. So far so good. Will be a year in March since he started.
Thanks as always!
Sally

Birgitta-A

Sun Jan 29, 2012, 03:15 PM

Hi Sally,
That is a difficult question because the researcher report that increased blasts and fibrosis in MDS patients can depend on the disease. That is true of cause. The latest reports from ASH 2011 tell us that Nplate has a positive effect in MDS.

The same year FDA wrote "On 18 February 2011, the independent Data Monitoring Committee (DMC) for a randomised clinical study of Nplate in MDS patients with thrombocytopenia recommended to discontinue Nplate in all patients due to the increased risk of progression to AML. In this study, there were numerically more subjects in the Nplate arm with disease progression to AML (placebo 2, Nplate 9) and with increases in circulating blasts >10% (placebo 3, Nplate 25). The randomisation was 2 to1, Nplate to placebo. Of the cases of MDS disease progression to AML that were observed, patients with RAEB-1 classification of MDS at baseline were more likely to have disease progression to AML compared to lower risk.
"http://www.imb.ie/images/uploaded/documents/DHPC%20on%20the%20risk%20of%20disease%20progression%20to%20AML%20with%20romiplostim%20(Nplate)%20use %20in%20patients%20with%20MDS%20%2022.09.2011.pdf

I still hope Promacta will function better for us.
Kind regards
Birgitta-A

Nadia T

Mon Jan 30, 2012, 02:09 AM

Thanks so much for this information. I'm leaving for NIH this weekend to start the Campath trial. Though all my numbers are low, my platelets are the biggest concern, so I'll be sure to ask about this.

By the way, did the Campath help?

Sally C

Mon Jan 30, 2012, 09:08 AM

Hi Nadia,
Don had Campath in the spring of '09. He had a partial response with only his white cells. But I do believe it also saved his life.
I don't know if you will have the same protocol. He received Campath for 10 days in the hospital. I think I read somewhere that they are giving Campath as out-patient now and possibly a lower dose.
I think you have to wait a while before you can take Promacta after Campath. They do wait approx. 6 months to see if you respond although sometimes it can take longer. But you may get a great response and your platelets won't be a problem.
NIH is wonderful!!! And the doctors are so accessible and helpful. It's a first class operation - a real healing, positive, upbeat place. You couldn't be in better hands.
Keep us posted. Good luck and God Bless!
Sally

Greg H

Mon Jan 30, 2012, 12:03 PM

Nadia,

Much good luck with the Campath. The first couple of doses are kind of scary, with the rigors and all. But it's pretty boring by the end of the ten day cycle.

I sure hope it works for you. Keep us posted!

Take care!

Greg

Lisa Z

Fri Feb 3, 2012, 08:40 PM

Good luck with the Campath trial. I went through it in March 2009. While all my counts have not gone to normal, my platelets went from 40 to 140 and my whites and reds, and hemaglobin, while still low, I am living a normal life.
I haven't been sick since the trial, other than the usual winter upper respiratory stuff I always got anyway. Never a fever and my hemaglobin is trying hard to get to normal range. I do also have PNH though, and that is likely the cause of it not climbing any higher.
I feel I could live a normal, long life with my current counts. Haven't had a transfusion since June of '09.
NIH is great! My 2 primary docs are no longer there, but I'm sure the care is still the best!
I haven't been on anti-virals for a LONG time and really am only taking thyroid meds, since the Campath screwed up my thyroid.
So, basically, I do not have to be treated for my MDS at this point; I have been in remission since June '09.
I hope you have a great success story to share soon!

Sally C

Tue Apr 17, 2012, 06:14 PM

Hello all,
Greetings from Bethesda, Md.
Just wanted to give you an update on Don's success with Promacta which he started taking 3/11. Today his platelets were 91,000 and his reds were
11.5.
Just to put in perspective - he was transfusion dependent from 1/09 until spring of 2011. He needed about 120 transfusions in 2009 and 2010. His platelets at one time got down to 4,000 so you can see the unbelievable improvement.
They also checked his eyes again for a worsening of subcapsular cataracts. The eye surgeon we saw today said she wasn't sure she would even call them cataracts as the eyes change as we age. But the main thing is there has been no worsening of whatever they want to call it.
The NIH doctors are even talking about cutting back on the Promacta if his platelets hit 100,000 out of concern for blood clots. They have this concern because clots appeared on lab rats so they are just being careful. He has an atrial fib/flutter and they actually want us to ask his cardiologist about a blood thinner. We'll see about that.
He has had absolutely no side effects from the Promacta that we can tell.
Also NIH is still recruiting for the trial for AA as well as MDS. If anyone has any questions about this please feel free to contact me. shcalvert3@aol.com.
God Bless to all,
Sally

Lisa Z

Tue Apr 17, 2012, 06:59 PM

Just for future reference, do you have a link to this protocol? Thank you, and continued good luck!

Sally C

Tue Apr 17, 2012, 07:18 PM

Hi Lisa Z,
I'm more than glad to provide the link - and thank you for your best wishes.
http://clinicaltrials.gov/ct2/show/NCT00961064

Here is the contact info...
Barbara Weinstein RN BSN CCRP
NHLBI/ Research Nurse Specialist
Building 10 CRC Room 5130
10 Center Drive
Bethesda, MD 20892
phone: 301-594-4180
fax: 301-594-1290

Best wishes to you too!
Sally

Sally C

Tue Apr 24, 2012, 05:16 PM

Birgitta-A

Wed Apr 25, 2012, 04:56 AM

Wonderful Sally!
Kind regards
Birgitta-A

tytd

Wed Apr 25, 2012, 12:29 PM

Hi Sally,
That is so great that Don has responded to the Promacta. 116k wow!. I wondered if you knew how the other patients on the trial were doing? I think that you said in the beginning that Don had a hypoplastic marrow so I wondered if people like me with hyperplastic marrows might not tend to respond so well or be more prone to blasts developing. Also I think you mentioned that Don had Atrial fib. Did that develop before or after starting the Promacta? Here's hoping for continued success and thanks for posting the news. tytd

Sally C

Wed Apr 25, 2012, 04:12 PM

Birgitta,
Thank you for the kind words.
Tytd,
Don was hypocellular to begin with but that changed to hypercellular before the Promacta so that shouldn't be an issue.
He started Promacta in March, 2011 and had the heart problem in Sept., 2011. According to what the NIH doctors have told us there have been no issues with the heart in previous Promacta trials. (AA trials started before MDS trials.)
This is only a guess but his heart issues could have been a result of all the steroids he received as part of the pre-meds before his many transfusions. He was prone to reactions - especially with his platelet transfusions.
We haven't been told of the results of the other MDS patients in the trial - there are only 3 others. They have had good success with their AA patients with the Promacta not only helping with platelets but with red cells as well. Promacta helped Don with both lines also - in fact one thing I forgot to mention was that last week was the first time since 12/08 that Don's Hgb was in the normal range.
Please let me know if you want any further information and thank you for your kind words as well!
Best wishes,
Sally

Neel

Thu Apr 26, 2012, 03:01 AM

My father started revolade 50 mg on 02 april 2012. He tolerated it well for first week, but no improvement in counts. However in the second week he developed fever and is hospitalised since 14 april. His urine culture now shows infection , so the resaon looks like uti . It sems that he developed uti due to revolade. There is stil no improvement in ciunts, on the contrary the drop in his platelet count is unprecendented. He require platelet every alternate day now. His fever is also not under control and only subsized for 4 hrs after taking paracetamol. We have now stopped revolade since ysday. Any inputs

Regards neel

Sally C

Thu Apr 26, 2012, 04:45 PM

Neel,
I am very sorry about your Dad no matter what the cause. I'm sure you are very concerned. One of the many things that is so wicked about these diseases is what may help one may hurt another.
I wish you and your Dad the best.

And to correct one of my previous statements...
As of yesterday his platelet count was 116,000. They are now going to start cutting back on the Promacta - 25mg. every 2 weeks - until he gets below 100,000. If I'm not mistaken this is so there won't be the danger of causing blasts.

I asked NIH why they don't want his platelets over 100,000. They do this out of concern for clots. The MDS patients have the same protocol as ITP patients - some whose platelets went over 200,000 - so they set the limit at 100,000.
Sorry for the misinformation.
Sally

Sally C

Tue Jul 3, 2012, 04:21 PM

Hi everyone,
I am pleased to report that, as of this Thurs., 7/5, my husband will come off Promacta. Once his platelets went over 100,000 they started a slow taper from 150mg., dropping the dosage 25mg. every 2 weeks until his last dosage, which was 25mg. - trying to get those stubborn platelets below 100,000.;)
His platelets today were 121,000. Also during the time he was on Promacta - which he started 3/11 - his reds reached the normal range for the first time since 11/08. You can read his history in this clinical trial at the start of my other thread under "Clinical Trials" named "New Promacta (Eltrombopag) clinical trial at NIH" as well as a little history in this thread. He virtually had no side effects that we know of - he tolerated the Promacta very well.
They will do CBC's every 2 weeks to monitor the situation and we go to NIH on 7/17 for a scheduled BMB but we don't expect any surprises.
He was the first MDS guinea pig for Promacta at NIH and I think he will be Promacta's star MDS patient.
If anyone has any questions regarding this treatment or NIH please feel free to contact me.
God Bless,
Sally

Greg H

Tue Jul 3, 2012, 04:52 PM

Hey Sally!

That's really great news. When they do the Promacta paper at ASH, they should put Don's picture on the cover page.

What a great success story!

Take Care!

Greg

Al's Wife

Tue Jul 3, 2012, 07:00 PM

Wow, Sally, that is such great news. I know y'all are thrilled. I can remember when we were thankful for platelets of 40,000, so it's kind of scary now with them hovering around 16,000. But we're more concerned with the blasts now as they seem to be going up rapidly. Since we weren't able to get into Moffitt before next week, Al is going to have to have another BMB before they will start him on anything else, I'm afraid, since his last BMB done on June 5th at NIH is only good for 30 days. We did everything in our power to try and get him in sooner, but to no avail.
I am just glad that Don has been helped and NIH worked out so well for y'all. God bless you both. And I continue to thank you for your help and support.

tytd

Tue Jul 3, 2012, 11:17 PM

Hello Sally, Greg, Linda,
So glad to hear that Don is still responding to Promacta and with few side effects, that is great. Greg, glad to see you are back (we've missed your informative posts lately) and hope you are doing well on the Danazol. Linda, Moffitt is a very good center and hope they will find a trial for Al. Maybe they can use the BMB from NIH. I am sorry he did not qualify for the Promacta trial but wondered why NIH did not provide you with any other options??
Today, there is a study from NIH published in the New England Journal of Medicine about a phase 2 study of Promacta in refractory AA patients. I have not read the whole article but the abstract says that 44% of patients responded , some even with responses in red cells and WBCs as well as platelets. Hopefully this will prove useful in MDS patients as well without causing increased blasts but only time will tell. I cannot provide a link to this article but if anyone wants to read a copy of it just send me an email and I can forward it to you.

Sally C

Fri Jul 6, 2012, 08:09 AM

Greg,
I think that's a great idea about the picture.;) And thank you for you kind words. It is good to see you posting again. You are always so informative and humorous at the same time.
Linda,
Thank you for your kind words as well. You always manage to provide support in spite of what you and Al are going through. I wish you both well at Moffitt. Please keep us posted and let me know if there is anything I can do - even if just a shoulder to lean on.
Tytd,
Thank you for your support as well. I would appreciate getting a copy of the clinical trial for AA/Promacta. shcalvert3@aol.com.
God Bless all!
Sally

cathybee1

Sat Jul 7, 2012, 06:49 PM

Awesome news, Sally. Congratulations to both of you on getting to this milestone!

I also wanted to let you know how much I appreciate your updates about clinical trials/findings, etc.

Sally C

Sun Jul 8, 2012, 07:08 AM

Thank you so much Catherine! I wish you and Bruce the best.
God Bless,
Sally

debbienfl

Thu Jul 26, 2012, 09:11 PM

My husband Don started on Promacta in March, 2011 with platelets that had stayed in the teens for 2 years - once getting as low as 4,000. (You can read about it in more detail under "Clinical Trials".)
As of yesterday his platelet count was 116,000. They are now going to start cutting back on the Promacta - 25mg. every 2 weeks - until he gets below 100,000. If I'm not mistaken this is so there won't be the danger of causing blasts.
They are still wanting new patients for this trial. Don has tolerated the Promacta without any problems whatsoever.
Best wishes,
Sally

Hi Sally. I am glad to hear that your husband is doing well. Whom do we contact about the trial evaluating Promacta for use in treating MDS? My father is 83 with advanced MDS and is not a candidate for Vidaza. We are looking for alternatives. Can you advise me?

debbienfl

Thu Jul 26, 2012, 09:25 PM

Hi,
My father is 83 with advanced MDS and is not a candidate for Vidaza. We are looking for alternatives like Promacta. Can you please send me a copy of the study. My email address is: Joslind@bellsouth.net
Thanks, Debbie

Hello Sally, Greg, Linda,
So glad to hear that Don is still responding to Promacta and with few side effects, that is great. Greg, glad to see you are back (we've missed your informative posts lately) and hope you are doing well on the Danazol. Linda, Moffitt is a very good center and hope they will find a trial for Al. Maybe they can use the BMB from NIH. I am sorry he did not qualify for the Promacta trial but wondered why NIH did not provide you with any other options??
Today, there is a study from NIH published in the New England Journal of Medicine about a phase 2 study of Promacta in refractory AA patients. I have not read the whole article but the abstract says that 44% of patients responded , some even with responses in red cells and WBCs as well as platelets. Hopefully this will prove useful in MDS patients as well without causing increased blasts but only time will tell. I cannot provide a link to this article but if anyone wants to read a copy of it just send me an email and I can forward it to you.

Sally C

Fri Jul 27, 2012, 08:13 AM

Hi Debbie,
I sent the link to you in an e-mail but want to post the link on the forums in case someone else wants to read it.
If anyone has any questions about Promacta please feel free to contact me - shcalvert3@aol.com.
God Bless,
Sally

Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia
http://www.nejm.org/doi/full/10.1056/NEJMoa1200931

Contact information at NIH:
Barbara Weinstein RN BSN CCRP
NHLBI/ Research Nurse Specialist
Building 10 CRC Room 5130
10 Center Drive
Bethesda, MD 20892
phone: 301-594-4180
fax: 301-594-1290

Sally C

Sat Jul 28, 2012, 09:31 AM

Hello my friends,
I just wanted to update you on Don's progress since stopping Promacta on July 5. He stopped after a slow taper didn't bring his platelets below 100,000 (what a
problem:)) out of concern for clots.
Two weeks (7/17) after stopping Promacta his platelets dropped from 121,000 to 88,000; HGB dropped from 12.6 to12.4; WBC dropped from 6.8 to 3.61; neutrophils dropped from 6.0 to 2.49.
On his 7/27 CBC his platelets were still a respectable 83,000; his HGB was 12.9; his WBC was 6.2; and neutrophils were 5.1. He will stay off Promacta as long as his platelets stay above 50,000.
He started Promacta in May of 2011 but didn't see much movement until the fall. While on Promacta his reds reached the normal range for the first time since 11/08. He received Campath in 4/09 and Cyclosporine in 2010 but remained very transfusion dependent until Promacta - especially for platelets.
We also received a short note regarding his bone marrow biopsy - 'the cells seen are essentially unchanged and most importantly there is no increase in blasts, signifying no progression'.
I hope this gives hope to all who may be discouraged. There was a time that Don was on our doctor's "death list". We were told this once she took him off. There was a time when his neutrophils reached 0.0.
There are so many treatments out there for MDS and they are coming up with new ones all the time. Everyone's MDS is different and responses vary. Just keep the faith that the right one is out there and you'll eventually find one that will work for you.
If anyone would like to contact me regarding Promacta please feel free to do so - shcalvert3@aol.com.
God Bless,
Sally

Birgitta-A

Sat Jul 28, 2012, 12:23 PM

Hi Sally,
Don's story is a wonderful reading for all of us :)!

I had a mail from Kirby Stone. Nplate is better for him than Promacta - it is really true that "everyone's MDS is different and responses vary".
Kind regards
Birgitta-A

Sally C

Sat Jul 28, 2012, 04:50 PM

Hi Birgitta,
Thank you so much for your kind words and support. My greatest wish with telling Don's story is that people will read it and see that, even though things can seem so dire and futile, where there is life there is always hope. Don's story certainly is a testament to that!
I hope you are doing well. So glad you are posting again - I missed your wisdom and kindness.
God Bless,
Sally

Greg H

Sat Jul 28, 2012, 09:40 PM

Sally,

I think the other great thing about your sharing Don's story is that it may encourage folks to have a positive attitude about participating in clinical trials.

With a rare disease like MDS, where there are relatively few options that have been FDA approved, a clinical trial can sometimes be the only want to get the drug or treatment that fits your particular type of MDS.

It doesn't always work, of course but it has certainly worked for you all -- and, by participating in a trial, you've helped countless MDS patients who come behind you.

Take care!

Greg

Birgitta-A

Sun Jul 29, 2012, 04:47 AM

Hi Sally,
You know I have been staying at my summerhouse (without pc) and only visited Stockholm once a week without time to post. I still feel fine after a little more than 2 years on Thalidomide + Prednisone though my HGB and platelets are now very slowly decreasing. The WBCs are holding.

Here is an abstract about Promacta and how that drug can inhibit the proliferation of leukemia cells:
http://bloodjournal.hematologylibrary.org/content/120/2/386.abstract
Kind regards
Birgitta-A

Relentless Against SAA

Sat Feb 8, 2014, 09:54 AM

Hi Sally,
I sent you a private message. Looking forward to hearing from you.:)

Sally C

Sat Feb 8, 2014, 09:58 AM

I just checked and there wasn't a private message there. Please e-mail me at shcalvert3@aol.com

AAteen

Mon Dec 1, 2014, 12:46 AM

Hi Sally,

Thank you for sharing your husband's experience in taking Promacta. Very encouraging. I have just sent you a private message and hope to hear from you.

Best wishes

Leung

Sally C

Mon Dec 1, 2014, 08:31 AM

Hi Leung,
I have answered your private message. Please let me know if I can be of further help.
Blessings,
Sally

Dishavirk

Sat Dec 29, 2018, 02:34 AM

Hi Sally

I read your husband’s story.
Very encouraging for someone like me
I m 35 years old,a mother of 5 years old daughter
I was diagnosed with severe aplastic anemia on
30 September 2017. My treatment was started fast
I got atg,immuno supression n Eltrombopag (150) by dec 2017
I was platelets transfusions free by February 2018
N blood transfusions free by August 2018
As of 25 December 2018 my counts r hb 10.8
Tlc 2800 n platelets 78000
My doctor are going to start tapering medicine by 9 jan
Initially doing 150/125 on alternate days n then after 2 weeks 125
Then montly decrease it by 25
I was wondering wat are your husbands count now?
N wat to except while tapering

Sally C

Sat Dec 29, 2018, 08:58 AM

Hi Dishavirk,
It is wonderful that Promacta has helped you - and so quickly.
I went back and read some of the posts I made along with others who posted since I started this thread in 2011. So much I don't remember!!! I can give you a brief update but you can get a more accurate account by the posts I made during that time - which you may already have done.
He finished his Promacta taper in July, 2012 and has been in remission ever since - no transfusions since 2011 after over 125 blood/platelet transfusions. With his last blood draw (12/18) his counts did start slipping - platelets were 74,000. We are hoping that the slippage is due to some fairly serious health issues in Oct., 2018 - unrelated to his MDS. He will be checked again on Jan. 8.
I don't recall his having any reaction to taking Promacta or tapering from it.
As I stated in my previous posts, they waited to taper him until his platelets reached 100,000. Should they drop to 50,000, he would start Promacta again with our local oncologist. While it's not approved for MDS outside a clinical trial, he does have some overlapping AA that would allow him to be given the Promacta.
Please let me know if I can give you any further information and please keep us posted.
All the best,
Sally

Dishavirk

Sun Dec 30, 2018, 01:16 AM

Dishavirk

Sun Dec 30, 2018, 01:45 AM