Table 3.

Efficacy of Avatrombopag in ITP in Phase II and III Trials

Study/Phase	Duration	Location	Number of Patients (n) Enrolled	Main Inclusion Criteria	Main Results
Bussel et al30 2014 Phase II double-blind, randomized dose-ranging, placebo-controlled, parallel group study	Randomized study 28 days	US	Total (n= 64) - Placebo (n= 5) - 2.5 mg (n=15) - 5 mg (n=15) -10 mg (n=14) - 20 mg (n= 15)	● Age ≥18 years ● ITP ≥3 month ● Platelet counts <30X109/L, or < 50X109/L if receiving steroids ● Failure to respond ≥1 prior line of ITP therapy.	Platelet response ratea: 0% placebo 13% at 2.5 mg avatrombopag 53% at 5 mg avatrombopag 50% at 10 mg avatrombopag 80% at 20 mg avatrombopag
	Extension part 24 weeks		Total (n=53) - Responders (n=25) - non-responders (n=28)	● completed treatment in the randomized part	Overall response: 76% Durable response: 53% 13 out of 24 reduced steroid doses by ≥50% 8 out of 24 discontinued steroids
Jurczak et al31, 2018 Phase III multicenter, randomized, double- blinded, parallel- group study	Core study 6 months	Asia, Australia, and New Zealand, Africa, Europe	Total (n =49) - placebo (n=17) - avatrombopag (n=32)	● age≥18 years ● ITP≥12 months ● platelet counts<30X109/L ● ≥1 prior line of therapy. ● Initial response to ≥1prior line of therapy or bone marrow examination to rule out other causes of thrombocytopenia	Duration of a platelet count ≥50X109/L(avatrombopag/placebo) Median: 12.4 vs 0.0 weeks Mean: 12.0 vs 0.0 weeks Durable response rate: 34.4% vs 0.0%
	Extension phase 90 weeks		Total (n=39)	● Completed maintenance in a core study or discontinued early due to lack of response ● No safety or tolerability issues

Note: ^aPlatelet response: platelet count ≥50x10⁹/L at a given assessment time point.

Abbreviation: ITP, immune thrombocytopenia.