[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.