Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia
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Open Access
- 7 September 2018
- journal article
- research article
- Published by Wiley in British Journal of Haematology
- Vol. 183 (3), 479-490
- https://doi.org/10.1111/bjh.15573
Abstract
Avatrombopag, an oral thrombopoietin receptor agonist, was compared with placebo in a 6‐month, multicentre, randomised, double‐blind, parallel‐group Phase 3 study, with an open‐label extension phase, to assess the efficacy and safety of avatrombopag (20 mg/day) in adults with chronic immune thrombocytopenia (ITP) and a platelet count 9/l (ClinicalTrials.gov identifier NCT01438840). The primary endpoint was the cumulative number of weeks of platelet response (platelet count ≥50 × 109/l) without rescue therapy for bleeding; secondary endpoints included platelet response rate at day 8 and reductions in the use of concomitant medications. Amongst the 49 patients randomised, avatrombopag (N = 32) was superior to placebo (N = 17) in the median cumulative number of weeks of platelet response (12·4 vs. 0·0 weeks, respectively; P < 0·0001). At day 8, a greater platelet response rate was also observed for patients treated with avatrombopag compared with placebo (65·63% vs. 0·0%; P < 0·0001), and use of concomitant ITP medications was also reduced amongst patients receiving avatrombopag. The safety profile of avatrombopag was consistent with Phase 2 studies; the most common adverse events were headache and contusion. Overall, avatrombopag was well tolerated and efficacious for the treatment of chronic ITP.Keywords
Funding Information
- Eisai
- AbbVie
- Celgene
- Gilead Sciences
- TG Therapeutics
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