Hi Sally,
Don has really responded very well to Promacta
! You know several years ago they showed that Promacta could have anti-leukemia effect in MDS cell-lines. Perhaps the drug has had this kind of effect on Don's bone marrow?
I have only started to read all the abstracts about MDS so I don't know yet if there maybe is another report about Promacta.
Here is a short version of the abstract from Italy:
EFFICACY AND SAFETY OF PROMACTA FOR THE TREATMENT OF THROMBOCYTOPENIA OF LOW AND INT-1 RISK MDS: PRELIMINARY RESULTS OF A PROSPECTIVE, RANDOMIZED, SINGLE-BLIND PLACEBO-CONTROLLED TRIAL
Aims:
We present preliminary results of a multicenter, prospective, randomized, single blind placebo-controlled trial to evaluate the safety and efficacy of eltrombopag in low and intermediate-1 risk MDS with platelet count less than 30.
Methods:
Eltrombopag versus placebo (2:1 ratio) will be administered to 69 patients at a 50 mg daily initial dose with 50 mg increases every 2 weeks to a target platelet count of 100. Dose interruptions or reductions are required for platelets more than 200 or adverse events.
Results:
12/69 patients (
8 on active drug – Arm A) of median age 68 are enrolled. Baseline median platelet count was 16.
In Arm A, four cases obtained an response and one a complete response after 7 days and 3 of 6 evaluable patients had a complete response after 14 days at the initial 50 mg daily dose; an additional patient had a complete response at 6 weeks at a 250 mg dose.
In Arm A no bleeding events occurred; at week 8 there was an increase in median platelet count from 16 to 120, while no change was observed with placebo (arm B), in which 9 bleeding events occurred.
Adverse events occurred in 4 patients in Arm A: grades 2 (n=2), 3 (n=1) and 4 (n=1), all unrelated to eltrombopag. One patient experienced a second adverse event (asthenia, grade 1) with a possible association with eltrombopag. Neither peripheral nor BM blast counts increased in either arms at interim analysis.
Conclusions:
Preliminary data suggest that Eltrombopag is effective and safe in the prevention of bleeding and in raising platelet counts in lower risk MDS patients with severe thrombocytopenia.
Kind regards
Birgitta-A