Yazarlar : Bussel JB, Kuter DJ, Aledort LM

Yayın : Blood.

Yayın Yılı : 2014

Pubmed Linki : http://www.ncbi.nlm.nih.gov/pubmed/24802775

Konu : Diğer

Literatür İçeriği :  Stimulation of platelet production by thrombopoietin-receptor agonists (TPO-RAs) is an effective second-line treatment strategy in immune thrombocytopenia (ITP). Avatrombopag (E5501) is an investigational non-peptide, TPO-RA active in humans. This 28-day Phase II study assigned subjects with ITP of ≥3 months to once-daily oral avatrombopag (2.5, 5, 10, 20 mg) or placebo; subjects completing randomized treatment could enroll in a 24-week extension study. Of 64 randomized subjects, 13% (avatrombopag 2.5 mg), 53% (5 mg), 50% (10 mg), and 80% (20 mg), versus 0% for placebo, achieved a platelet count (PC) response of ≥50×10⁹/L with ≥20×10⁹/L increase above baseline at Day 28. Fifty-three (83%) subjects entered the extension study and received long-term avatrombopag treatment: 52% had a durable response (PC response at ≥75% of their platelet assessments over the last 14 weeks) and 76% achieved an overall PC response (stable response or response at any ≥2 consecutive visits). All subjects experienced ≥1 adverse event (AE) (most commonly fatigue, headache, epistaxis) with 19% (n=12) reporting ≥1 serious AE. Across studies, 10 (16%) subjects withdrew due to an AE; five of these were due to increased PC. Avatrombopag was active and generally well-tolerated with PC response rates and AE incidence comparable with other TPO-RAs. Studies were registered at http://clinicaltrials.gov/, identifiers: NCT00441090 and NCT00625443.

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