Platelet drug (eltrombopag/Promacta) for MDS patients

[Birgitta-A]

Hi all,
Here is a report (the first?) about positive results from a study where MDS patients with low platelets were treated with Promacta :
http://bloodjournal.hematologylibrar...ct/114/18/3899

Unfortunately there is a report in the same number of Blood about both Nplate and Promacta causing increased bone marrow fibrosis in some patients with Immune Thrombocytopenic Purpura (a bleeding disorder).
Kind regards
Birgitta-A

[lfeinsmith]

Originally Posted by Birgitta-A

Hi all,
Here is a report (the first?) about positive results from a study where MDS patients with low platelets were treated with Promacta :
http://bloodjournal.hematologylibrar...ct/114/18/3899

Unfortunately there is a report in the same number of Blood about both Nplate and Promacta causing increased bone marrow fibrosis in some patients with Immune Thrombocytopenic Purpura (a bleeding disorder).
Kind regards
Birgitta-A

Is there any new info for Patients with predominantly thrombocytopenia 36000: IPSS inter 1 : Hgb 12.8, Abs neutr
1,100 and quite stable; less than 5% blasts: cytogenetics normal

[Birgitta-A]

Hi leslei,
As you perhaps have seen the member Kirby Stone is participating in one of the trials with Promacta for patients with MDS and low platelets. If the trials will show positive results the drug Promacta - that is approved for Immune Thrombocytopenic Purpura - probably will be approved for MDS patients but that can take years.

As far as I understand there are two trials with Promacta - one for patients with > 20% blasts in bone marrow and one for patients with Low to Interm-2 risk MDS:
http://clinicaltrials.gov/ct2/result...ds+eltrombopag

The last trial is for 30 patients with less than 30 platelets. The location is NIH Clinical Center Rockville Pike, Bethesda, Maryland.
Kind regards
Birgitta-A

[lfeinsmith]

thanks for info

[knstone]

HI Leslie,

I obtained Promacta from GlaxoSmithKline, it was obtained on a restricted distribution program. It can be applied for at Promactacares.com.
If you go to the site you can obtain information.
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.

I don't think the way I obtained it is like a conventional clinical trial, it seemed more like restricted distribution program. One does have to pay for it as a drug and it is fairly expensive. The list price for 75 mg (25mg and 50mg tabs) is $5201. On Medicare part D you have to pay thru the gap to get it at 5% of that cost.