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Romiplostim (AMG531, Nplate) for secondary failure of platelet recovery after allo-SCT

Bone Marrow Transplantation volume 46, pages 1587–1589 (2011)Cite this article

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Romiplostim (Rm) (AMG531, Nplate), a protein that binds to and stimulates the TPO receptor, has been shown to increase and maintain platelet counts in splenectomized and non-splenectomized adult patients with chronic immune thrombocytopenic purpura (ITP), and few adverse events have been reported.1, 2, 3, 4, 5 An overall response, defined as platelet count ⩾50 000/μL for ⩾4 weeks, was observed in 83% of patients, and 49% of these had a durable response.3 Interestingly, no antibodies against Rm or endogenous TPO were detected.2, 3 The study by Kuter et al.3 suggested that Rm could increase BM reticulin in some patients, though there was improvement upon withdrawal of the drug. The clinical significance of this finding remains unclear and warrants further study.6 In addition, discontinuation of Rm can cause a rapid decrease in platelet count in many patients. However, the safety and efficacy of long-term Rm treatment has been established in patients for up to 156 weeks of treatment.4 Altogether, these data have led to the approval of Rm for adult chronic ITP therapy in North America, Australia and Europe.

The role of Rm has also been evaluated in thrombocytopenic patients with myelodysplastic syndromes (MDS) that are treated with either supportive care7 or azacitidine.8 These studies show that Rm was well tolerated, effective and that no neutralizing antibodies to either Rm or endogenous TPO were produced. However, these data remain preliminary and further studies are warranted to define the role and long-term safety profile of Rm in patients with MDS.

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References

  1. Bussel JB, Kuter DJ, George JN, McMillan R, Aledort LM, Conklin GT et al. AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP. N Engl J Med 2006; 355: 1672–1681.

    Article  CAS  PubMed  Google Scholar 

  2. Newland A, Caulier MT, Kappers-Klunne M, Schipperus MR, Lefrere F, Zwaginga JJ et al. An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. Br J Haematol 2006; 135: 547–553.

    Article  PubMed  Google Scholar 

  3. 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 

  4. Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL . Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood 2009; 113: 2161–2171.

    Article  CAS  PubMed  Google Scholar 

  5. Kuter DJ, Rummel M, Boccia R, Macik BG, Pabinger I, Selleslag D et al. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med 2010; 363: 1889–1899.

    Article  CAS  PubMed  Google Scholar 

  6. Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M . Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood 2009; 114: 3748–3756.

    Article  CAS  PubMed  Google Scholar 

  7. Kantarjian H, Fenaux P, Sekeres MA, Becker PS, Boruchov A, Bowen D et al. Safety and efficacy of romiplostim in patients with lower-risk myelodysplastic syndrome and Thrombocytopenia. J Clin Oncol 2010; 28: 437–444.

    Article  CAS  PubMed  Google Scholar 

  8. Kantarjian HM, Giles FJ, Greenberg PL, Paquette RL, Wang ES, Gabrilove JL et al. Phase 2 study of romiplostim in patients with low- or intermediate-risk myelodysplastic syndrome receiving azacitidine therapy. Blood 2010; 116: 3163–3170.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  9. Bruno B, Gooley T, Sullivan KM, Davis C, Bensinger WI, Storb R et al. Secondary failure of platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2001; 7: 154–162.

    Article  CAS  PubMed  Google Scholar 

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Acknowledgements

We are indebted to Erwan Floch for his editorial assistance.

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Authors and Affiliations

  1. Service des Maladies du Sang, CHU Haut-Lévêque, Bordeaux, France

    C Calmettes, S Vigouroux, R Tabrizi & N Milpied

  2. Université Bordeaux 2 Segalen, Bordeaux, France

    N Milpied

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  1. C Calmettes
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  2. S Vigouroux
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Correspondence to S Vigouroux.

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Calmettes, C., Vigouroux, S., Tabrizi, R. et al. Romiplostim (AMG531, Nplate) for secondary failure of platelet recovery after allo-SCT. Bone Marrow Transplant 46, 1587–1589 (2011). https://doi.org/10.1038/bmt.2011.179

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