Home         Forums  

Go Back   Marrowforums > Treatments > Clinical Trials
Register FAQ Search Today's Posts Mark Forums Read

Clinical Trials Considering or participating in research studies

Reply
 
Thread Tools Search this Thread
  #1  
Old Wed May 15, 2013, 05:04 AM
Birgitta-A Birgitta-A is offline
Member
 
Join Date: Oct 2007
Location: Stockholm, Sweden
Posts: 1,918
Promacta for low platelets

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
Reply With Quote
Reply


Thread Tools Search this Thread
Search this Thread:

Advanced Search

Posting Rules
You may not post new threads
You may not post replies
You may not post attachments
You may not edit your posts

vB code is On
Smilies are On
[IMG] code is On
HTML code is Off
Forum Jump

Similar Threads
Thread Thread Starter Forum Replies Last Post
Promacta an side effects to liver Missy D. AA 1 Tue Apr 21, 2015 10:34 PM
Low platelets S001 MDS 27 Thu Aug 23, 2012 12:07 PM
LOW platelets with reaction & Vidaza cheri MDS 6 Thu Nov 18, 2010 07:49 AM
Promacta for Platelets knstone Drugs and Drug Treatments 13 Wed Dec 9, 2009 09:08 PM
Promacta for low platelets approved by FDA Birgitta-A Drugs and Drug Treatments 1 Fri Nov 21, 2008 07:53 PM


All times are GMT -4. The time now is 12:23 AM.


Powered by vBulletin® Version 3.6.7
Copyright ©2000 - 2024, Jelsoft Enterprises Ltd.
Forum sites may contain non-authoritative and unverified information.
Medical decisions should be made in consultation with qualified medical professionals.
Site contents exclusive of member posts Copyright © 2006-2020 Marrowforums.org