What is the role of oral thrombopoietin receptor agonists (TPO-RA) in the treatment of thrombocytopenia among patients with chronic liver disease?

Updated: Oct 15, 2020
  • Author: David C Wolf, MD, FACP, FACG, AGAF, FAASLD; Chief Editor: BS Anand, MD  more...
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The oral thrombopoietin receptor agonist (TPO-RA) avatrombopag (Doptelet) was approved by the FDA in May 2018 for adults with thrombocytopenia secondary to chronic liver disease who are scheduled to undergo a procedure. [49]

Approval was based on the ADAPT-1 and ADAPT-2 clinical trials (N=435). The trials investigated the use of avatrombopag at two doses, 40 and 60 mg. The doses were selected on the basis of baseline platelet count and were administered orally over 5 consecutive days. Patients underwent their procedure 5 to 8 days after receiving the last dose. At both doses tested, a higher proportion of patients who received avatrombopag demonstrated an increase in platelet count compared with patients who received placebo. The primary endpoint was the proportion of patients not requiring platelet transfusions or rescue procedures for bleeding up to 7 days following the procedure. Patients who received avatrombopag met the primary endpoint 65.6%-68.6% relative to 22.9%-33.3% who received placebo. Patients who received avatrombopag 40 mg met the primary endpoint 87.9%-88.1% compared with 38.2%-33.3% who received placebo. [50]

A second TPO-RA, lusutrombopag (Mulpleta) was approved in July 2018 for treatment of thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure. [51]  Approval was based on an international, randomized trial (N=215). The study met its primary efficacy endpoint: 64.8% of patients in the lusutrombopag group required no platelet transfusion and no rescue therapy for bleeding, compared with 29% in placebo group (P< 0.0001). [52]

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