Safety and efficacy of hetrombopag in patients with chronic immune thrombocytopenia: a single-arm, open-label, multi-center phase 1 study.

ABSTRACT:

Background

Thrombopoietin receptor agonists (TPO-RAs) are promising therapeutic strategy for patients with immune thrombocytopenia (ITP). We conducted this phase 1 trial (NCT02614846) to evaluate the preliminary efficacy and safety of hetrombopag (a TPO-RA) in patients with ITP.

Methods

Patients with ITP who had an insufficient response or had progressed on at least one standard treatment for ITP were given hetrombopag orally at an initial dose of 5 mg once daily for up to 6 weeks. The primary endpoint was the proportion of patients who achieved platelet counts of ≥50×10⁹/L at week 6.

Results

A total of 37 eligible patients received hetrombopag treatment. This study met its primary endpoint, 22 (59.5%, 95% CI: 42.1-75.3) patients responded to hetrombopag, a

SUBMITTER: Mei H 

PROVIDER: S-EPMC8848441 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature