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
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.
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.
Stasi R, Provan D. Management of immune thrombocytopenia in adults. Mayo Clin Proc. 2004;79:504–22.
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.
McMillan R. The pathogenesis of chronic immune thrombocytopenic purpura. Semin Hematol. 2007;44(4 Suppl 5):S3–11.
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.
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.
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.
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.
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.
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.
Kuter DJ, Rummel M, Boccia R, et al. Romiplostim or standard of care in patients with immune thrombocytopenia. NEJM. 2010;363(20):1889–961.
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.
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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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