Skip to main content

Advertisement

Log in

Romiplostim as early treatment for refractory primary immune thrombocytopenia

  • Original Article
  • Published:
International Journal of Hematology Aims and scope Submit manuscript

Abstract

Romiplostim is a thrombopoietin-receptor agonist approved to treat chronic immune thrombocytopenia (ITP). We treated eight patients with acute or persistent primary ITP, severe clinical bleeding, and resistance to corticosteroids and/or intravenous immunoglobulins (IVIg). Romiplostim, initially administered at 2 or 3 μg/kg/week, was subsequently increased to achieve and maintain platelet-count responses and control bleeding. Seven patients’ platelet counts rose above 30 G/L, representing ≥twofold increases, within a median of 14 days after 1–5 infusions. The weekly dose reached 9 μg/kg at week 5 for three patients; the other patients’ ITPs were controlled with ≤6 μg/kg/week. No thromboembolic events occurred. Five patients received rituximab concomitantly with romiplostim, four of whom could stop romiplostim within 2 months, thereby demonstrating rituximab efficacy. All three patients treated with romiplostim alone required maintenance therapy. Thus, romiplostim represents an alternative for patients with severe acute or persistent ITP refractory to conventional therapy.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Subscribe and save

Springer+ Basic
$34.99 /Month
  • Get 10 units per month
  • Download Article/Chapter or eBook
  • 1 Unit = 1 Article or 1 Chapter
  • Cancel anytime
Subscribe now

Buy Now

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Fig. 1
Fig. 2
Fig. 3

Similar content being viewed by others

References

  1. Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, 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  PubMed  CAS  Google Scholar 

  2. Cooper N, Bussel J. The pathogenesis of immune thrombocytopenic purpura. Br J Haematol. 2006;133:364–74.

    Article  PubMed  CAS  Google Scholar 

  3. Aledort LM, Hayward CP, Chen MG, Nichol JL, Bussel J, ITP Study Group. Prospective screening of 205 patients with ITP, including diagnosis, serological markers, and the relationship between platelet counts, endogenous thrombopoietin, and circulating antithrombopoietin antibodies. Am J Hematol. 2004;76:205–13.

    Article  PubMed  CAS  Google Scholar 

  4. McMillan R, Wang L, Tomer A, Nichol J, Pistillo J. Suppression of in vitro megakaryocyte production by antiplatelet autoantibodies from adult patients with chronic ITP. Blood. 2004;103:1364–9.

    Article  PubMed  CAS  Google Scholar 

  5. Provan D, Stasi R, Newland AC, Blanchette VS, Bolton-Maggs P, Bussel JB, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115:168–86.

    Article  PubMed  CAS  Google Scholar 

  6. Khellaf M, Michel M, Schaeffer A, Bierling P, Godeau B. Assessment of a therapeutic strategy for adults with severe autoimmune thrombocytopenic purpura based on a bleeding score rather than platelet count. Haematologica. 2005;90:829–32.

    PubMed  Google Scholar 

  7. Godeau B, Porcher R, Fain F, Lefrere F, Fenaux P, Cheze S, et al. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood. 2008;112:999–1004.

    Article  PubMed  CAS  Google Scholar 

  8. Tagariello G, Sartori R, Radossi P, Maschio N, Risato R, Stasi R. Romiplostim for the early management of severe immune thrombocytopenia unresponsive to conventional treatment. Br J Haematol. 2012;157:256–8.

    Article  PubMed  CAS  Google Scholar 

  9. Cantoni N, Heizmann M, Bargetzi M. Immune thrombocytopenia and anticoagulation: the role of romiplostim in the early treatment. Br J Haematol. 2012;157:639–41.

    Article  PubMed  CAS  Google Scholar 

  10. Mitsuhashi K, Ishiyama M, Imai Y, Shiseki M, Mori N, Teramura M, et al. Combined romiplostim and intravenous immunoglobulin therapy increased platelet count, facilitating splenectomy in a patient with refractory immune thrombocytopenic purpura unresponsive to monotherapy. Br J Haematol. 2012;158:798–800.

    Article  PubMed  Google Scholar 

  11. Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008;371:395–403.

    Article  PubMed  CAS  Google Scholar 

  12. Shirasugi Y, Ando K, Miyazaki K, Tomiyama Y, Okamoto S, Kurokawa M, et al. Romiplostim for the treatment of chronic immune thrombocytopenia in adult Japanese patients: a double-blind, randomized phase III clinical trial. Int J Hematol. 2011;94:71–80.

    Article  PubMed  CAS  Google Scholar 

  13. Andemariam B, Psaila B, Bussel JB. Novel thrombopoietic agents. Hematology Am Soc Hematol Educ Program. 2007:106–13.

  14. Kuter DJ. Thrombopoietin and thrombopoietin mimetics in the treatment of thrombocytopenia. Annu Rev Med. 2009;60:193–206.

    Article  PubMed  CAS  Google Scholar 

  15. Gardiner EE, Thom JY, Al-Tamani M, Hughes A, Berndt MC, Andrews RK, et al. Restored platelet function after romiplostim treatment in a patient with immune thrombocytopenic purpura. Br J Haematol. 2010;149:625–8.

    Article  PubMed  CAS  Google Scholar 

Download references

Conflict of interest

JFV has participated in advisory boards and Amgen and Glaxo SmithKline symposia (honoraria of less than 10,000 USD).

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Jean-François Viallard.

About this article

Cite this article

Contis, A., Lazaro, E., Greib, C. et al. Romiplostim as early treatment for refractory primary immune thrombocytopenia. Int J Hematol 98, 520–524 (2013). https://doi.org/10.1007/s12185-013-1439-0

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s12185-013-1439-0

Keywords