Efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia: stage 2 results from a multicenter phase III study.

Abstract

This phase III, randomized, placebo-controlled study conducted in three stages (6-week, randomized, placebo-controlled stage 1; 24-week, open-label stage 2; and continuous extension stage 3) assessed the long-term efficacy and safety of eltrombopag use in Chinese patients with chronic immune thrombocytopenia (ITP). This article presents the results from stage 2. Overall, 150 patients (placebo-eltrombopag [P-E], 50; eltrombopag-eltrombopag [E-E], 100) received open-label eltrombopag. The median platelet count was maintained between 41 × 109/L and 80 × 109/L. Most patients in both groups (P-E, 90.0%; E-E, 81.8%) achieved platelet counts ≥30 × 109/L and ≥2 times the baseline platelet count at least once with eltrombopag treatment. Overall, 32% of patients achieved platelet counts ≥50 × 109/L in ≥75% of platelet count assessments. Both groups showed a decreased tendency to infrequent bleeding and clinically significant bleeding events during stage 2 compared with baseline. Among patients who received ≥1 ITP medication at baseline, 70.4% in the P-E group and 40.8% in the E-E group reduced or permanently stopped ≥1 of their ITP medications. The stage 2 results further demonstrated a sustainable long-term efficacy and good tolerability of eltrombopag with a favorable benefit-risk ratio in Chinese chronic ITP patients.Trial registration: Clinicaltrials.gov NCT01762761. Registered 8 January 2013, https://clinicaltrials.gov/ct2/show/NCT01762761.

• Authors: Xiaofan Liu, Ming Hou, Junmin Li, Jie Jin, Meijuan Huang, Ziqiang Yu, Xiaojun Xu, Xiaohui Zhang, Renchi Yang
• Journal: Platelets (Platelets) Vol. 33 Issue 1 Pg. 82-88 (Jan 02 2022) ISSN: 1369-1635 [Electronic] England
• PMID: 33251910 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study, Randomized Controlled Trial)

Chemical References

• Benzoates
• Hydrazines
• Pyrazoles
• eltrombopag

Topics

• Asian People
• Benzoates (pharmacology, therapeutic use)
• Chronic Disease
• Humans
• Hydrazines (pharmacology, therapeutic use)
• Neoplasm Staging
• Purpura, Thrombocytopenic, Idiopathic (drug therapy)
• Pyrazoles (pharmacology, therapeutic use)
• Treatment Outcome