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

Keywords:

• Asian continental ancestry
• Chinese patients
• chronic immune thrombocytopenia (ITP)
• eltrombopag
• platelet count

Acknowledgements

The authors thank Emily Teng of Novartis Business Services and Yehua Zhu of Novartis Oncology, China, for providing medical editorial assistance with this manuscript. This study (NCT01762761) was sponsored by GlaxoSmithKline; however, as of March 2, 2015, eltrombopag became an asset of Novartis AG.

Authorship Contributions

All authors declare that they have no competing interests. R.Y. contributed to the design and conceptualization of the research, data analyses, interpretation of data, collection of data, coordination of research as the lead investigator, and the revision of the manuscript. X.L., J.L., J.J., M. Huang, Z.Y., X.X., and X.Z. contributed to the collection of data, interpretation of data, and revision of the manuscript. M. Hou contributed to the design and conceptualization of the research, collection of data, interpretation of data, and revision of the manuscript. All authors reviewed the manuscript and approved the final version for submission.

Disclosure of Interest

The authors have no competing interests.

Supplementary Material

Supplemental data for this article can be accessed on the publisher’s website.

Additional information

Funding

This work was supported by the Novartis AG.