New Promacta (Eltrombopag) Clinical Trial at NIH

[Sally C]

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 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]

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)

[Sally C]

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]

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]

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]

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]

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]

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]

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]

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]

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]

Hi Sally,
You know they started this trial May 2009:
http://clinicaltrials.gov/ct2/show/N...pag+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]

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]

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/N...pag+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]

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]

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]

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. 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]

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]

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]

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]

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]

Sally,

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

[Sally C]

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]

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]

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/...C500089967.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