Promacta for Platelets

[knstone]

Hi fellow MDS colleagues,

Recently I applied and obtained Promacta (eltrombopag) in an effort to stimulate platelet production.

The Cleveland Clinic doctors suggested I try to obtain Promacta. It is only approved by the FDA for ITP patients. GlaxoSmithKline, the mfg, does offer it on a restricted distribution program. It can be applied for at Promactacares.com. Several application pages are filled in by the patient and his physician. I was doubtful that as an MDS patient I would receive the drug but it was processed quickly and I obtained the drug within a few days.
It is somewhat like a mini-drug trial. The doctor must check liver functions via CBCs and report the overall results back to Promactacares within 6 months.

Birgitta found a reference to Promacta: “2008 AACR Effect of eltrombopag on proliferation, differentiation and death of human leukemia and lymphoma cell lines…In conclusion, eltrombopag does not increase proliferation of the leukemia and lymphoma cell lines tested, and in fact, causes a dose dependent decrease in proliferation in many of the lines."
This seems to indicate that Promacta may be safe for MDS patients, althought there is a disclaimer on the drug info sheet warning that it is not approved for MDS.

The drug is a capsule so it is easy to take but it is fairly expensive, approx $3467/month for 50 mg/day before insurance. Those of us on Medicare Part D must work throught the "gap" before the monthly costs drop significantly.

I've been on Promacta for 2 months. It seems to have stabilized my platelets but thus far we can't really note a large increase. I'll try to keep the results updated.

[tytd]

Hello Kirby and fellow forum members,
I've been browsing this forum for approximately one year now ever since my dx with possible MDS. I appreciate all the useful info and support that the forum members provide. Kirby, I was particularly interested in your post about the Promacta as I have a very slowly dropping platelet count over many, many years with a mild macrocytic anemia. My white cells are fine. My hematologist suggested a trial of Prednisone to rule out an ITP type picture since my bone marrow only showed mild dysplasia without significant blasts or chromosomal abnormalities. It did not work. I have now been running platelet counts from 27,000 to 22,000 (most recent) over the last year with Hb in 9.5 - 10 range. I've had no bleeding justs bruises and petechiae. My options are limited. I wait and watch which is fine with me. My hematologists did not recommend Nplate due to safety concerns and trial results showing some pts developed leukemia. I guess the safety data is not yet in on Promacta. I hope that you will do well on it and the thalidomide. I would be interested to know what your actual platelet counts have been since starting the Promacta. Also how much did they drop when you were on Vidaza or Dacogen and on thalidomide? Thanks for sharing your inspiring personal story and best wishes. I would also like to thank Birgetta for all the useful information that she posts. TyTd

[Birgitta-A]

Hi tytd,
Thank you for the kind words !

There are many drugs in clinical trials for low platelets. Three of them can be promising.

LGD-4665 that now should be in trials for MDS though I can’t find any trail for MDS patients at the clin trials gov site:
http://ash.confex.com/ash/2008/webpr...Paper4547.html

AKR 501 but there has not been any news about results from trials for patients with thrombocytopenic purpura:
http://www.labmeeting.com/paper/2876...ceptor-agonist

R 788 but we have not seen any results recently – they try the drug for patients with arthritis but have not had much success:
http://ir.rigel.com/phoenix.zhtml?c=...637&highlight=
Kind regards
Birgitta-A

[knstone]

My MDS starting in early 2004 mainly affected the platelets and WBC, my Hgb was not affected as much initially.

Thalidomide raised all the counts after about 3 weeks starting in early 2004, the levels were normal. It worked well for 30 months.

Revlimid then raised the counts to normal or near normal levels for about 12 months. A slow decrease in Platelets and WBC then started. This is somewhat normal with Revlimid, the Hgb stays good but the platelets and WBC can start dropping. By Oct 2008 the platelets had fallen to the 20-30 range and we stopped Revlimid.

Vidaza was tried from Nov 2008 thru March 2009, 4 cycles at reduced dosages due to my low platelets. Nuemega was tried at low dose and seemed to help the platelets initially, raising the platelets to the 40's, this seemed to stop working after about 2 months. After the 4th cycle of Vidaza the platelets dropped to mid 20's.

Dacogen was tried on a 1 day/week cycle at 2/3 the normal 5 day dosage on 5/20/2009. The result was a sharp drop in Hgb from 7.8 to 5.0 in a 5 week period, the platelets dropped from 20 to as low as 8. No further Dacogen has been tried since the one day infusion.

Promacta was started on 6/5/2009 and the platelets increased to the mid teens with occasional readings in the high teens to low 20's. The dosage is 50 mg/day. I've had no bleeding.

Thalidomide was restarted on 6/22/2009 and the Hgb has improved slightly to 5.2 after being as low as 4.5. The platelets are holding in the mid teens.
We may consider increasing the Promacta to 75 mg/day since I've had no adverse effects to Promacta.

My bone marrow cellularity is 20% so there is not a lot to work with.
Blasts were 16-17% on 5/6/2009, we are not sure if the Dacogen or Thalidomide has reduced the blasts or not. At this point we are just watching the blood counts each week and hoping that I havn't transformed to AML.

[tytd]

Birgitta, Sorry I mispelled your name. Thanks for the up-to-date info on clinical trials for platelets - will be listening out for more results. I hope you are doing well and I enjoyed reading your profile too. Also your post on Vidaza access in UK was surprising. It makes me wonder about changing some of the health care system in USA.

Kirby, Thanks so much for taking the time again to list your responses to Vidaza, Dacogen and Promacta. I know that your energy must be very low with your severe anemia. I was wondering if you had had your iron or ferritin level in blood checked (or iron stain in last bone marrow) recently to make sure it wasn't low since you had the sharp drop in Hb and platelets. Low iron would be unlikely but possible. My first bone marrow showed no iron stores.

[Helen Robinson]

Hi Kirby

I saw your post and am glad you are still managing this disease. David is in a stable place at present. It seems the revlimid has helped spread the Txs a little. He had some cardiac investigations and there was know sign of iron affecting his heart which is good news. I hope you continue in good health.
Regards

Helen

[knstone]

My response to Promacta seems to be holding, my platelets have improved to the 20's. I've been on Thalidomide for 10 weeks and the last 8 weeks have resulted in an increase in my Hgb from a low of 4.5 eight weeks ago to 6.4 on 9/2/09. The trend in the Hgb and platelets is encouraging.

We are increasing the Promacta to 75 mg/day. So far I've had no noticible side effects from the Promacta, I take it after midnight during a bathroom run.
Blood tests show normal renal functions.

I hope all of you are doing good in your fight against MDS.

[tytd]

Good to hear from you Kirby and it's great that your counts are steadily climbing. Keep up the good fight.

[Birgitta-A]

Hi Kirby,
Very good that your platelets and HGB are increasing and that you don't have any adverse effects from Promacta. I wouldn't like that my HGB was 4.5 since I have a feeling (I may be wrong) that my bone marrow doesn't work so well when the HGB is so low.
Kind regards
Birgitta-A

[knstone]

Hi All,
After 12 weeks on Thalidomide at 75mg/day and 14 weeks on Promacta my Hgb has increased to 7.0 (from a minumum of 4.5) and the platelets to 31(from a minimum of 9-10).

We think the Thalidomide may be the main contributor to the good response but the Promacta may be helping the platelets. I've been on the Promacta for 14 weeks and there was some increase in the platelets prior to the Thalidomide.

Whatever the reason we are thankful that my counts are improving steadily and hope the good response continues.

[Birgitta-A]

Hi Roddy,
Since I don't live in the US I can't answer your question about if Medicare will cover Promacta or Nplate (Romiplostim). I can only give you info about the latest results from a study about Promacta and MDS:
http://bloodjournal.hematologylibrar...ct/114/18/3899

Then there is a trial for MDS patients. If you look at clin trials gov you will find info about Promacta in patients with advanced MDS and a platelet count less than 30. Only the clinic in San Antonio, Texas, is recruiting patients but many other clinics (in California, Massachusetts, Kansas City, Philadelphia, Houston, New Braunfels) will recruit patients but we don't know when.

You know the FDA has a special warning about Nplate for MDS patients because patients with low risk MDs have transformed to AML. I think Promacta is the best drug for us today.
Kind regards
Birgitta-A
Last platelet count 37

[Neil Cuadra]

Roddy,

I suggest that you read How Medicare Drug Plans Use Pharmacies, Formularies, and Common Coverage Rules (PDF file, 456K) to get an overview of how Medicare plans handle prescription drugs.

Then use the Medicare Prescription Drug Plan Finder. Fill in your state and county under "Learn More About Plans in Your Area" and it will show you a table of prescription drug plans that will be available to you on Medicare. You can click the name of any plan and then click "View plan formulary" to find out coverage details under that plan for promacta, romiplostim, or any other drugs you might be taking.

[knstone]

Hi All,

I've had an interesting Nov-Dec. I was hospitalized with pneumonia on 11/8 for 5 days of antibiotics.

I've continued to use Promacta and thalidomide since June 09, We added low dose Dacogen (one infusion per week) in mid Oct 09.

My platelets have increased to 65 and the other counts are increasing also, especially since the pneumonia was treated.

We are not sure what causing the increased counts, I'm on 3 drugs at the same time: Promacta, thalidomide, and low dose Dacogen. Since things are looking good we are not changing anything at the moment.

[tytd]

Hello Kirby,
It's great to hear that your counts are all responding to your 3 drug regimen but sorry to hear you had pneumonia. I suppose you have not had another bone marrow done to see if your blast count is down? As long as you are doing OK I guess there is no need for that. Thanks for keeping us informed and giving us some hopes that these drugs may really help. tytd