[Birgitta-A]

Hi All,
Here is a study that shows positive results for Promacta (eltrombopag) in patients with low platelet count.

Berlin 2013 P-310
Eltrombopag for the treatment of thrombocytopenia of low and intermediate-1 IPSS risk myelodysplastic syndromes: Results of a prospective, randomized trial
E.N. Oliva1, V. Santini2, G. Zini3 et al R. Latagliata17, F. Nobile1. 1Hematology Unit, Azienda Ospedaliera Bianchi-Melacrino-Morelli, Reggio Calabria, Italy

Background: About 10% of low and Int-1 IPSS risk MDS patients experience severe thrombocytopenia. Hemorrhage and the scarce efficacy of PLT transfusions drive research in new therapies. Eltrombopag is an oral agonist of the thrombopoetin-receptor (TPO-R) indicated for treating chronic immune thrombocytopenic purpura.

Introduction: Eltrombopag’s potential in increasing PLT counts in lower risk MDS has not been evaluated. We present interim results of a Phase II, multicentre, prospective, placebo-controlled, single-blind study (EQoL-MDS).

Purpose: Primary endpoints are safety and efficacy of eltrombopag in low and intermediate-1 risk. Secondary endpoints include changes in quality of life (QoL), PLT transfusion requirement, incidence and severity of bleeding, and survival.

Materials and Methods: Patients (N=69) are included if: adult; PLT<30 Gi/L; ECOG performance status < 4; ineligible for, relapsed or refractory to other treatments; and naive to TPO-R agonists. Eltrombopag/ placebo (2:1) will be administered at a 50 mg daily starting dose with 50 mg increases every 2 weeks tomaximum 300 mg to target PLT 100 Gi/L. Dose interruptions or reductions are required for PLT >200 Gi/L or adverse events.

PLT response is defined as Response if: 1) baseline PLT>20 Gi/L: absence of bleeding and PLT≥50Gi/L; 2) baseline PLT<20 Gi/L: PLT>20 Gi/L and increase by at least 100%, not due to PLT transfusions; and Complete Response if PLT≥100 Gi/L and absence of bleeding. Centralized morphology and cytogenetics are performed throughout the study. QoL scores are evaluated by EORTC QLQ-C30 and QOL-E.

Results: Twenty-six patients (17 on eltrombopag – ArmA), mean age 65 (SD 11) years, have been randomized and 8 are in screening at the time of this report.

Baselinemean PLT count was 16 (SD 8) Gi/L. Three cases in Arm A and 1 in Arm B had significant bleeding requiring PLT transfusions. Fourteen patients reached a 16-week follow-up: 8 of 9 cases in Arm A obtained PLT responses at median 75 mg dosing associated with disappearance of bleeding and PLT transfusion independence.
There were no responses in Arm B: bleeding events and PLT transfusions were required.

At 16 weeks, PLT count increased by mean 82 (SD 69) Gi/L (p=0.008) in Arm A versus no change in Arm B. QoL improved from baseline. Grade III-IV unrelated adverse events occurred in 2 patients in Arm A. Bone marrow blasts reduced in 2 cases during treatment in Arm A, versus 1 progression in Arm B.

Conclusions: Preliminary results suggest safety and efficacy of eltrombopag in low and Intermediate-1 risk IPSS MDS patients with thrombocytopenia.

Kind regards
Birgitta-A