Eltrombopag therapy in newly diagnosed steroid non-responsive ITP patients

Int J Hematol. 2014 Apr;99(4):413-7. doi: 10.1007/s12185-014-1533-y. Epub 2014 Feb 14.

Abstract

Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterised by isolated thrombocytopenia (peripheral blood platelet count <100 × 10(9)/L) in the absence of other causes or disorders that may be associated with thrombocytopenia. The upfront treatment in newly diagnosed ITP patients is steroids; however, about one-third patients do not respond, and require other treatment, including IVIg, anti-D, or splenectomy. Previous studies have shown decreased platelet production in some ITP patients, aside from the evidence of enhanced platelet destruction. Thrombopoietin receptor agonists (TPO-RA), such as eltrombopag have been shown to provide good response in steroid non-responsive chronic ITP patients. We have studied response to eltrombopag in 25 newly diagnosed steroid non-responsive ITP patients; 80 % patients showed response at the end of 1 month, and 76 % sustained response at the end of 3 months. The platelet count rose from a mean value of 17.5 ± 3.6-152.5 ± 107.9 × 10(9)/L at the end of 1 month. Our results suggest a possible role of eltrombopag in newly diagnosed steroid non-responsive ITP patients. However, our study is limited in that it is a single-centre study, with a small sample size, and lacks a long-term safety profile. Our findings highlight the potential value of a larger prospective study on the upfront use of TPO-RA in patients of ITP.

MeSH terms

  • Adolescent
  • Adult
  • Benzoates / therapeutic use*
  • Drug Resistance
  • Female
  • Humans
  • Hydrazines / therapeutic use*
  • Male
  • Middle Aged
  • Platelet Count
  • Prospective Studies
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Pyrazoles / therapeutic use*
  • Receptors, Thrombopoietin / agonists*
  • Steroids / therapeutic use
  • Time Factors
  • Treatment Outcome
  • Young Adult

Substances

  • Benzoates
  • Hydrazines
  • Pyrazoles
  • Receptors, Thrombopoietin
  • Steroids
  • eltrombopag