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Sustained remission of chronic immune thrombocytopenia after discontinuation of treatment with thrombopoietin-receptor agonists in adults

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Abstract

Thrombopoietin-receptor agonists (TPO-RAs) are highly effective in immune thrombocytopenia (ITP). Recently, reports of sustained remission after TPO-RA discontinuation in adult ITP have been reported. We aimed to describe the subset of patients in whom TPO-RA therapy may induce a durable response. We retrospectively analyzed all of our adult ITP patients treated with TPO-RAs, and focused on patients with discontinued TPO-RA therapy due to treatment response. Forty-six relapsed or refractory ITP patients were treated with TPO-RAs. In 11 of these cases, TPO-RA therapy (seven romiplostim; four eltrombopag) was stopped after achieving treatment response. No side effects of TPO-RAs were observed. These patients were pretreated with 1–3 therapy lines plus splenectomy in six patients prior to TPO-RA administration. None of these patients experienced disease relapse after therapy discontinuation over a median follow-up of 33 months (16–54). Substantial proportion of ITP patients receiving TPO-RAs can maintain a durable response after treatment discontinuation. Sustained ITP remission probably does not depend on previous treatment, splenectomy, or disease duration.

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References

  1. Donald MA, Kelton JG. Current options for the treatment of idiopathic trombocytopenic purpura. Semin Hematol. 2007;44(Suppl 5):S12–23.

    Google Scholar 

  2. British Committee for Standard in Haematology General Haematology Task Force. Guidelines for the investigation and management of idiopathic thrombocytopenic purpura (ITP) in adults, children and in pregnancy. Br J Haematol. 2003;120:574–96.

    Article  Google Scholar 

  3. 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  CAS  PubMed  Google Scholar 

  4. Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, 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. 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  CAS  PubMed  Google Scholar 

  6. Bussel J, Kuter D. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2013;113:2161–71.

    Article  Google Scholar 

  7. Rodeghiero F, Stasi R, Giagounidis A, Viallard JF, Godeau B, Pabinger I, et al. Long-term safety and tolerability of romiplostim in patients with primary immune thrombocytopenia (ITP): a pooled analysis of 13 clinical trials. Eur J Haematol. 2013;91:423–36.

    Article  CAS  PubMed  Google Scholar 

  8. Bussel J, Saleh M. Repeated short-term use of eltrombopag in patients with chronic immune thrombocytopenia. Br J Haematol. 2013;160:538–46.

    Article  CAS  PubMed  Google Scholar 

  9. Ghadaki B, Nazi I, Kelton G, Donald MA. Sustained remissions of immune thrombocytopenia associated with the use of thrombopoetin receptor agonist. Transfusion. 2013;53:2807–12.

    Article  CAS  PubMed Central  PubMed  Google Scholar 

  10. Mahévas M, Fain O, Ebbo M, Roudot-Thoraval F, Limal N, Khellaf M, et al. The temporary use of thrombopoietin-receptor agonists may induce a prolonged remission in adult chronic immune thrombocytopenia. Results of a French observational study. Br J Haematol. 2014;165(6):865–9.

    Article  PubMed  Google Scholar 

  11. 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(11):2386–93.

    Article  CAS  PubMed  Google Scholar 

  12. Newland A, Cervinek L, Eggermann J, Lefrere F, Kreuzbauer G. Sustained hemostatic platelet counts in adult patients with primary immune thrombocytopenia (ITP) following cessation of romiplostim—four European case studies. Haematologica. 2011;96:98.

    Google Scholar 

  13. González-López TJ, González-Porras JR, Areji M, De Cabo E, Sanchez Gonzalez B, Alonso A, et al. Sustained response after short-medium-term treatment with eltrombopag in patients with ITP. Blood. 2013;122:2323.

    Google Scholar 

  14. Bussel J, Wang X, Eisen M. Case studies of remission in adults with immune thrombocytopenia (ITP) following cessation of treatment with the TPO receptor agonist romiplostim. Blood. 2013;122:328.

    Article  Google Scholar 

  15. Stasi R, Newland A, Godeau B, Priego V, Viallard J-F, Polez Fernandez MF, et al. An interim analysis of a phase 2, single-arm study of platelet responses and remission rates in patients with immune thrombocytopenia (ITP) receiving romiplostim. Blood. 2013;122:1074.

    Google Scholar 

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Acknowledgments

Supported in part by Research Grant MSMT CR CZ.1.05/1.1.00/02.0068 (CEITEC), and by the Czech Leukemia Study Group—for Life (CELL).

Conflict of interest

The authors have had costs of participating in certain scientific meetings reimbursed by the pharmaceutical industry.

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Correspondence to Michael Doubek.

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Červinek, L., Mayer, J. & Doubek, M. Sustained remission of chronic immune thrombocytopenia after discontinuation of treatment with thrombopoietin-receptor agonists in adults. Int J Hematol 102, 7–11 (2015). https://doi.org/10.1007/s12185-015-1793-1

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  • DOI: https://doi.org/10.1007/s12185-015-1793-1

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