European Hematology Association

[Birgitta-A]

Hi All,
Now the abstracts from EHA are available. I saw for example the preliminary results from a study of Eltrombopag (Promacta/Revolade) in MDS patients. I have been waiting several years for a report on this drug for us.
Kind regards
Birgitta-A

[Birgitta-A]

Sorry. I forgot the link: https://www.eventure-online.com/even...ngress=6_12EHA
Birgitta-A

[Sally C]

Hi Birgitta,
Thank you for this information. However it is a bit complicated for me to understand. Since my husband has been on Promacta for over a year I am interested in what the assessments are. Would you mind giving me a short summary of what you gleaned from these studies? I would greatly appreciate it.
NIH is in the process of lowering Don's dosage to get his platelets under 100,000 out of concern for blood clots. They have already cut him from 150mg./day to 100mg./day. If his platelets are still above 100,000 this week, they will cut him down to 75mg./day. His reds are normal for the first time since 2009. I think it's feasible that he could at some point come off Promacta and still maintain a decent platelet count. We'll see. But at this point you wouldn't know he even has MDS. It truly has been a miracle drug.
NIH will do another BMB in July to check his marrow and the effects if any of Promacta. Will let you know.
Thanks so much!
Sally

[Birgitta-A]

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

[Sally C]

Thank you so much Birgitta,
You not only are a genius - you have a caring heart that is always willling to inform and support us all.
I wish you the best!
Sally

[Birgitta-A]

Hi Sally,
Thank you very much for the kind words !
Kind regards
Birgitta-A