Romiplostim therapy in children with unresponsive chronic immune thrombocytopenia

References

• Ballem PJ, Segal GM, Stratton JR, Gernsheimer T, Adamson JW, Slichter SJ. Mechanisms of thrombocytopenia in chronic autoimmune thrombocytopenic purpura: Evidence of both impaired platelet production and increased platelet clearance. J Clin Invest 1987; 80: 33–40
   PubMed Web of Science ®Google Scholar
• Bussel JB, Kuter DJ, George JN, McMillan R, Aledort LM, Conklin GT, Lichtin AE, Lyons RM, Nieva J, Wasser JS, Wiznitzer I, Kelly R, Chen CF, Nichol JL. AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP. N Engl J Med. 2006;355(16):1672–1681. Erratum in: N Engl J Med. 2006 Nov 9;355(19):2054.
   Google Scholar
• 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: 1364–1369
   PubMed Web of Science ®Google Scholar
• Li J, Yang C, Xia Y, Bertino A, Glaspy J, Roberts M, Kuter DJ. Thrombocytopenia caused by the development of antibodies to thrombopoietin. Blood 2001; 98: 3241–3248
   PubMed Web of Science ®Google Scholar
• Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med 2002; 346: 995–1008
   PubMed Web of Science ®Google Scholar
• Carcao MD, Zipursky A, Butchart S, Leaker M, Blanchette VS. Short-course oral prednisone therapy in children presenting with acute immune thrombocytopenic purpura (ITP). Acta Paediatr 1998; 424: 71–74
   Google Scholar
• Buchanan GR. Overview of ITP treatment modalities in children. Blut 1989; 59(1)96–104
   PubMedGoogle Scholar
• Kaushansky K. The molecular mechanisms that control thrombopoiesis. J Clin Invest 2005; 115: 3339–3347
   PubMed Web of Science ®Google Scholar
• Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med 2007; 357: 2237–2247
   PubMed Web of Science ®Google Scholar
• Cheng G. Eltrombopag for the treatment of immune thrombocytopenia. Expert Rev Hematol 2011; 4(3)261–269
   PubMed Web of Science ®Google Scholar
• Matsuki E, Miyakawa Y, Yamane A, Okamoto S. Humanized VB22B minibody for human Mpl stimulates human megakaryopoiesis but does not enhance platelet aggregation. Exp Hematol 2011; 39(8)829–836
   PubMed Web of Science ®Google Scholar
• Meletis J, Katsandris A, Raptis SD, Mantzourani M. Successful treatment of Immune Thrombocytopenic Purpura (ITP) with the thrombopoietin-mimeticromiplostim. Med Sci Monit 2010; 16(8)CS100–CS102
   PubMed Web of Science ®Google Scholar
• Cuker A, Cines DB. Immune thrombocytopenia. Hematology Am Soc Hematol Educ Program 2010; 2010: 377–384
   PubMed Web of Science ®Google Scholar
• Elalfy M, Abdelmaksoud AA, Eltonbary KY, Romiplostim in children with chronic refractory ITP: Randomized placebo controlled study. Ann Hematol 2011;Online First
   Google Scholar
• Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, 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)2386–2393
   PubMed Web of Science ®Google Scholar
• Amgen. Draft EU summary of product characteristics. Amgen Europe B.V, ZK BredaThe Netherlands 2008
   Google Scholar
• Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: A double-blind randomised controlled trial. Lancet 2008; 371: 395–403
   PubMed Web of Science ®Google Scholar
• Frederiksen H, Schmidt K. The incidence of idiopathic thrombocytopenic purpura in adults increases with age. Blood 1999; 94: 909–913
   PubMed Web of Science ®Google Scholar
• Hubulashvili D, Marzella N. Romiplostim (Nplate), a Treatment Option for Immune (Idiopathic) Thrombocytopenic Purpura. P T. 2009; 34(9): 482–485.
   Google Scholar
• Kuter DJ, Bain B, Mufti G, Bagg A, Hasserjian RP. Bone marrow fibrosis: Pathophysiology and clinical significance of increased bone marrow stromal fibres. Br J Haematol 2007; 139(3)351–362
   PubMed Web of Science ®Google Scholar
• Kuter DJ. New drugs for familiar therapeutic targets: thrombopoietin receptor agonists and immune thrombocytopenic purpura. Eur J Haematol 2008;80(suppl. s69):9–18.
   Google Scholar
• Stasi R, Evangelista ML, Stipa E, Stipa E, Buccisano F, Venditti A, Amadori S. Idiopathic thrombocytopenic purpura: Current concepts in pathophysiology and management. Thromb Haemost 2008; 99: 4–13
   PubMed Web of Science ®Google Scholar
• Kuter DJ, Bussel JB, Senecal FM, Kuter DJ, Bussel JB, Senecal FM, Aledort LM, Kessler CM, Sanz MA, Liebman HA, et al. Evaluation of AMG 531 in nonsplenectomized patients with chronic immune thrombocytopenic purpura in a randomized placebo-controlled phase 3 study. Blood 2007; 110: 173a
   Web of Science ®Google Scholar
• Pohlen M, Sargin B, Zicholl S, Bisping G, Müller-Tidow C, Berdel WE, Mesters R, Koschmieder S. Combination of romiplostim and rituximab: Effective therapy of severe immune thrombocytopenia. Eur J Haematol 2010; 84: 362–364
   PubMed Web of Science ®Google Scholar
• Lyons RM, George JN, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Kelly R, Guo M, Nichol J. Evaluation of AMG 531 safety in Splenectomized (S) and Nonsplenectomized (NS) patients with chronic Immune Thrombocytopenic Purpura (ITP) in two randomized placebo−controlled phase 3 studies. Blood (ASH Annual Meeting Abstracts) 2007; 110: Abstract 1300
   Web of Science ®Google Scholar