Efficacy and Safety of Eltrombopag for Aplastic Anemia: A Systematic Review and Meta-analysis

Clin Drug Investig. 2019 Feb;39(2):141-156. doi: 10.1007/s40261-018-0725-2.


Background and objectives: Eltrombopag seems to be effective in treating patients with aplastic anemia in several clinical trials. This paper aims to perform the first meta-analysis analyzing the efficacy and safety of eltrombopag for aplastic anemia.

Methods: Literatures were retrieved from PubMed, EMBASE, OVID, Web of Science, Cochrane, Wanfang, http://clinicaltrials.gov and World Health Organization International Clinical Trials Registry Platform search portal from establishment to July 2018. Using Stata statistical software version 12.0, subgroup analyses and sensitivity analyses were conducted.

Results: The overall hematologic response rate is 88% (95% CI 83-94%) for patients treated with eltrombopag plus immunosuppressive therapy, and 47% (95% CI 38-56%) for patients with refractory aplastic anemia using eltrombopag alone. Karyotype abnormality rates include an overall rate of 10% (95% CI 7-14%), a subtotal rate of 8% (95% CI 3-13%) for patients who are treated with eltrombopag plus immunosuppressive therapy without using antithymocyte globulin before, and a subtotal rate of 17% (95% CI 10-24%) for patients with refractory aplastic anemia treated with eltrombopag alone.

Conclusions: With different treatments and in different conditions eltrombopag showed a distinctive effect for aplastic anemia. However, clone evolution and adverse events were associated with treatment.

Publication types

  • Meta-Analysis
  • Systematic Review

MeSH terms

  • Anemia, Aplastic / drug therapy*
  • Benzoates / administration & dosage
  • Benzoates / adverse effects
  • Benzoates / therapeutic use*
  • Humans
  • Hydrazines / administration & dosage
  • Hydrazines / adverse effects
  • Hydrazines / therapeutic use*
  • Immunosuppressive Agents / administration & dosage
  • Pyrazoles / administration & dosage
  • Pyrazoles / adverse effects
  • Pyrazoles / therapeutic use*


  • Benzoates
  • Hydrazines
  • Immunosuppressive Agents
  • Pyrazoles
  • eltrombopag