Once-daily, oral avatrombopag found efficacious in patients with thrombocytopenia prior to elective procedures

BOSTON — Avatrombopag “may be a promising new therapy” for the treatment of thrombocytopenia in patients with chronic liver disease, a researcher said during her presentation at The Liver Meeting.

Norah Terrault, MD, and colleagues conducted a phase 2, randomized, double blind, placebo-controlled study to examine the effectiveness of avatrombopag (AVA) in patients with chronic liver disease (CLD) and thrombocytopenia prior to elective surgical or diagnostic procedures. These patients are often ineligible for surgical and diagnostic procedures because of bleeding risk, she said.

Patients had a platelet count (PC) of ≥10 to ≤50 x 109/L [+15%]; an elective invasive procedure scheduled 1 to 4 days after the last dose of the study; and a MELD score of ≤24, according to Terrault.

There were two cohorts. Cohort A (n=67) received a 100-mg loading dose of AVA or placebo on day 1 and then 20 mg/d, 40 mg/d, or 80 mg/d or placebo for 6 days.

Terrault noted during her presentation that Cohort B (n=63) was added to evaluate a second generation AVA. Cohort B received an 80-mg loading dose or placebo on day 1 and then 10 mg/d or placebo for 6 days; then 20 mg/d and placebo for 3 days.

The study’s primary endpoint was percentage of responders — those with a PC increase ≥20 x 109/L from baseline and at least one PC >50 x 109/L during days 4 to 8. The secondary endpoint was the need for platelet transfusion, she said.

Of patients who received 7 days of AVA therapy, 31% to 77% achieved a PC increase from baseline of ≥20 x 109/L and a PC of ≥50 x 109/L a minimum of once a day for days 4 to 8 of treatment, according to Terrault. She also said adverse events were comparable overall.

Peak increase in PC occurred at days 10 to 13 for the combined AVA treatment groups and fewer platelet transfusions were given in the AVA groups, she said.

Terrault concluded saying these results suggest that AVA may potentially replace transfusion in this category of patients. She also noted that planning for a phase 3 study is under way.

Disclosure: Terrault reports that she is on the Advisory Committee/Review Panel of Eisai and has received grant/research support from Eisai.

For more information:

Terrault N. P122: Once-daily avatrombopag (E5501) prior to elective surgical or diagnostic procedures in patients with chronic liver disease and thrombocytopenia: results from a Phase 2, randomized, double-blind, placebo-controlled study (Study 202). Presented at: The Liver Meeting 2012; Nov. 9-13, Boston.