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Eltrombopag therapy in newly diagnosed steroid non-responsive ITP patients

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Abstract

Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterised by isolated thrombocytopenia (peripheral blood platelet count <100 × 109/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 × 109/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.

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References

  1. Rodeghiero F, Roberto S, Terry GM, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009;113:2386–93.

    Article  CAS  PubMed  Google Scholar 

  2. Houwerzijl EJ, Blom NR, Van der Want JJL, et al. Ultrastructural study shows morphologic features of apoptosis and para-apoptosis in megakaryocytes from patients with idiopathic thrombocytopenic purpura. Blood. 2004;103:500–6.

    Article  CAS  PubMed  Google Scholar 

  3. Stasi R, Provan D. Management of immune thrombocytopenia in adults. Mayo Clin Proc. 2004;79:504–22.

    Article  PubMed  Google Scholar 

  4. Bussel JB, Cheng G, Saleh MN, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007;357:2237–47.

    Article  CAS  PubMed  Google Scholar 

  5. McMillan R. The pathogenesis of chronic immune thrombocytopenic purpura. Semin Hematol. 2007;44(4 Suppl 5):S3–11.

    Article  CAS  PubMed  Google Scholar 

  6. Olsson B, Anderson PO, Jemas M, et al. T cell mediated cytotoxicity toward platelets in chronic idiopathic thrombocytopenic purpura. Nat Med. 2003;9(9):1123–4.

    Article  CAS  PubMed  Google Scholar 

  7. Chow L, Aslam R, Speck ER, et al. A murine model of severe thrombocytopenia is induced by antibody and CD 8+ T cell mediated responses that are differentially sensitive to therapy. Blood. 2010;115(6):1247–53.

    Article  CAS  PubMed  Google Scholar 

  8. Sakakura M, Wada H, Tawara I, et al. Reduced CD4+ CD25− T cells in patients with idiopathic thrombocytopenic purpura. Thromb Res. 2007;120(2):187–93.

    Article  CAS  PubMed  Google Scholar 

  9. Ballem PJ, Segal GM, Stratton JR, et al. Mechanism of thrombocytopenia in chronic autoimmune thrombocytopenic purpura. Evidence of both impaired platelet production and increased platelet clearance. J Clin Invest. 1987;80(1):33–40.

    Article  CAS  PubMed Central  PubMed  Google Scholar 

  10. Cheng G, Saleh MN, Marcher C, et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomized, phase 3 study. Lancet. 2011;377(9763):393–402.

    Article  CAS  PubMed  Google Scholar 

  11. Saleh MN, Bussel JB, Cheng G, et al. EXTEND Study Group. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013;121(3):537–45.

    Article  CAS  PubMed  Google Scholar 

  12. Kuter DJ, Rummel M, Boccia R, et al. Romiplostim or standard of care in patients with immune thrombocytopenia. NEJM. 2010;363(20):1889–961.

    Article  CAS  PubMed  Google Scholar 

  13. Erickson-Miller CL, Delorme E, Tian SS, et al. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009;27(2):424–30.

    Article  CAS  PubMed Central  PubMed  Google Scholar 

  14. Tripathi A, Mishra S, Yadav D et al. Megakaryocyte morphology and its impact in predicting response to steroid in immune thrombocytopenia. Platelets 2013 (in press).

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Correspondence to Ayush Shukla.

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Tripathi, A.K., Shukla, A., Mishra, S. et al. Eltrombopag therapy in newly diagnosed steroid non-responsive ITP patients. Int J Hematol 99, 413–417 (2014). https://doi.org/10.1007/s12185-014-1533-y

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  • DOI: https://doi.org/10.1007/s12185-014-1533-y

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