Thrombopoietin receptor agonists: Ten Years Later.

Thrombopoietin receptor agonists: Ten Years Later. Haematologica. 2019 May 09;: Authors: Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB Abstract The two thrombopoietin receptor agonists (TPO-RAs), Eltrombopag and Romiplostim, were licensed in the United States for treatment of ITP in 2008 and progressively around the world in more than 100 countries. The six largest randomized controlled trials conducted in ITP have used one of these two agents. All studies have demonstrated a platelet response rate between 50-90%, depending upon the criteria used, with good safety and tolerability. As well, TPO-RAs were shown to be effective in reducing bleeding and need for concomitant and rescue medication. Many other explorations of mechanism of effect, prospective and retrospective trials, and studies focusing on toxicity have been performed providing extensive knowledge of these two agents. Initial concerns such as myelofibrosis have not materialized. Only a small minority of patients develops moderate-severe reticulin fibrosis and/or collagen fibrosis which usually reverses after discontinuation of TPO-RAs. Studies indicates, however, that TPO-RAs may increase the risk of venous thromboembolism. Both TPO-RA are currently approved in patients with chronic ITP 1 year of age and older who are refractory to at least one other treatment. Eltrombopag, has acquired two additional indications: severe aplastic anemia refractory to first line tr...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research