Multicenter, prospective study to evaluate the efficacy of biweekly romiplostim administration in patients with immune thrombocytopenia
详细信息    查看全文
  • 作者:Silvia Park ; Sung Soo Yoon ; Jung Hee Lee…
  • 关键词:Immune thrombocytopenia ; Romiplostim ; Biweekly schedule
  • 刊名:International Journal of Hematology
  • 出版年:2016
  • 出版时间:January 2016
  • 年:2016
  • 卷:103
  • 期:1
  • 页码:44-52
  • 全文大小:703 KB
  • 参考文献:1.Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med. 2002;346:995–1008.PubMed CrossRef
    2.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.PubMed CrossRef
    3.Frederiksen H, Schmidt K. The incidence of idiopathic thrombocytopenic purpura in adults increases with age. Blood. 1999;94:909–13.PubMed
    4.Neylon AJ, Saunders PW, Howard MR, Proctor SJ, Taylor PR. Clinically significant newly presenting autoimmune thrombocytopenic purpura in adults: a prospective study of a population-based cohort of 245 patients. Br J Haematol. 2003;122:966–74.PubMed CrossRef
    5.Segal JB, Powe NR. Prevalence of immune thrombocytopenia: analyses of administrative data. J Thromb Haemost. 2006;4:2377–83.PubMed CrossRef
    6.McMillan R. The pathogenesis of chronic immune thrombocytopenic purpura. Semin Hematol. 2007;44:S3–11.PubMed CrossRef
    7.Cines DB, McMillan R. Pathogenesis of chronic immune thrombocytopenic purpura. Curr Opin Hematol. 2007;14:511–4.PubMed CrossRef
    8.McMillan R. Autoantibodies and autoantigens in chronic immune thrombocytopenic purpura. Semin Hematol. 2000;37:239–48.PubMed CrossRef
    9.Cines DB, Bussel JB, Liebman HA. Luning Prak ET. The ITP syndrome: pathogenic and clinical diversity. Blood. 2009;113:6511–21.PubMed PubMedCentral CrossRef
    10.Lakshmanan S, Cuker A. Contemporary management of primary immune thrombocytopenia in adults. J Thromb Haemost. 2012;10:1988–98.PubMed CrossRef
    11.Louwes H. Zeinali Lathori OA, Vellenga E, de Wolf JT. Platelet kinetic studies in patients with idiopathic thrombocytopenic purpura. Am J Med. 1999;106:430–4.PubMed CrossRef
    12.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 PubMedCentral CrossRef
    13.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.PubMed CrossRef
    14.Houwerzijl EJ, Blom NR, van der Want JJ, Esselink MT, Koornstra JJ, Smit JW, 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.PubMed CrossRef
    15.Dameshek W, Miller EB. The megakaryocytes in idiopathic thrombocytopenic purpura, a form of hypersplenism. Blood. 1946;1:27–50.PubMed
    16.Nugent D, McMillan R, Nichol JL, Slichter SJ. Pathogenesis of chronic immune thrombocytopenia: increased platelet destruction and/or decreased platelet production. Br J Haematol. 2009;146:585–96.PubMed CrossRef
    17.Pisciotta AV, Stefanini M, Dameshek W. Studies on platelets. X. Morphologic characteristics of megakaryocytes by phase contrast microscopy in normals and in patients with idiopathic thrombocytopenic purpura. Blood. 1953;8:703–23.PubMed
    18.Branehog I, Kutti J, Ridell B, Swolin B, Weinfeld A. The relation of thrombokinetics to bone marrow megakaryocytes in idiopathic thrombocytopenic purpura (ITP). Blood. 1975;45:551–62.PubMed
    19.Stoll D, Cines DB, Aster RH, Murphy S. Platelet kinetics in patients with idiopathic thrombocytopenic purpura and moderate thrombocytopenia. Blood. 1985;65:584–8.PubMed
    20.Nomura S, Dan K, Hotta T, Fujimura K, Ikeda Y. Effects of pegylated recombinant human megakaryocyte growth and development factor in patients with idiopathic thrombocytopenic purpura. Blood. 2002;100:728–30.PubMed CrossRef
    21.Perreault S, Burzynski J. Romiplostim: a novel thrombopoiesis-stimulating agent. Am J Health Syst Pharm. 2009;66:817–24.PubMed CrossRef
    22.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.PubMed CrossRef
    23.Broudy VC, Lin NL. AMG531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine. 2004;25:52–60.PubMed CrossRef
    24.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;355:1672–81.PubMed CrossRef
    25.Amgen Europe B.V. Summary of product characteristics 2009. Breda, The Netherlands: Amgen Europe B.V.; 2009.
    26.Wang B, Nichol JL, Sullivan JT. Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor ligand. Clin Pharmacol Ther. 2004;76:628–38.PubMed CrossRef
    27.Kumagai Y, Fujita T, Ozaki M, Sahashi K, Ohkura M, Ohtsu T, et al. Pharmacodynamics and pharmacokinetics of AMG 531, a thrombopoiesis-stimulating peptibody, in healthy Japanese subjects: a randomized, placebo-controlled study. J Clin Pharmacol. 2007;47:1489–97.PubMed CrossRef
    28.Neunert C, Lim W, Crowther M, Cohen A, Solberg L Jr, Crowther MA. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011;117:4190–207.PubMed CrossRef
    29.Wang YM, Krzyzanski W, Doshi S, Xiao JJ, Perez-Ruixo JJ, Chow AT. Pharmacodynamics-mediated drug disposition (PDMDD) and precursor pool lifespan model for single dose of romiplostim in healthy subjects. AAPS J. 2010;12:729–40.PubMed PubMedCentral CrossRef
    30.Cao Y, Jusko WJ. Incorporating target-mediated drug disposition in a minimal physiologically-based pharmacokinetic model for monoclonal antibodies. J Pharmacokinet Pharmacodyn. 2014;41:375–87.PubMed PubMedCentral CrossRef
    31.Li J, Xia Y, Kuter DJ. Interaction of thrombopoietin with the platelet c-mpl receptor in plasma: binding, internalization, stability and pharmacokinetics. Br J Haematol. 1999;106:345–56.PubMed CrossRef
    32.Broudy VC, Lin NL, Sabath DF, Papayannopoulou T, Kaushansky K. Human platelets display high-affinity receptors for thrombopoietin. Blood. 1997;89:1896–904.PubMed
    33.Mager DE, Jusko WJ. General pharmacokinetic model for drugs exhibiting target-mediated drug disposition. J Pharmacokinet Pharmacodyn. 2001;28:507–32.PubMed CrossRef
    34.Cronkite EP, Bond VP, Fliedner TM. Studies on the origin, production and destruction of platelets. In: Johnson SA, W. MR, Rebuck JW, Horn RC editors. Blood Platelets. Boston: Little, Brown; 1961.
    35.Jackson CW, Arnold JT, Pestina TI, Stenberg PE. Megakaryocyte biology. In: Kuter D, Hunt P, Sheridan WP, Zucker-Franklin D, editors. Thrombopoiesis and thrombopoietins. Totowa: Humana; 1997.
    36.Harker LA, Roskos LK, Marzec UM, Carter RA, Cherry JK, Sundell B, et al. Effects of megakaryocyte growth and development factor on platelet production, platelet life span, and platelet function in healthy human volunteers. Blood. 2000;95:2514–22.PubMed
    37.Mahevas 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;165:865–9.PubMed CrossRef
    38.Bussel JB, Wang X, Eisen M. Case studies of remission in adults with immune thrombocytopenia (ITP) following cessation of treatment with the TPO receptor agonist romiplostim. 55th ASH Annual Meeting and Exposition. New Orleans, LA; 2013:Abstract 328. 2013.
    39.Go RS. Idiopathic cyclic thrombocytopenia. Blood Rev. 2005;19:53–9.PubMed CrossRef
  • 作者单位:Silvia Park (1)
    Sung Soo Yoon (2)
    Jung Hee Lee (3)
    Joon Seong Park (4)
    Jun Ho Jang (1)
    Jong Wook Lee (5)

    1. Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, 50 Irwon-dong, Gangnam-gu, Seoul, 135-710, Korea
    2. Seoul National University Hospital, Seoul, Korea
    3. Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
    4. Ajou University Hospital, Suwon, Korea
    5. Division of Hematology, Department of Internal Medicine, Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, 222 Banpo-daero, Seocho-gu, Seoul, 137-701, Korea
  • 刊物主题:Hematology; Oncology;
  • 出版者:Springer Japan
  • ISSN:1865-3774
文摘
Multicenter, prospective study was conducted to evaluate the efficacy of biweekly romiplostim in maintaining platelet ≥30 × 109/L for at least 4 weeks. Treatment was started with a weekly injection (1 mcg/kg), and the dose was escalated until a titrated dose was achieved that maintained a platelet 50–200 × 109/L for four consecutive weeks. Patients were scheduled to a biweekly schedule, and returned to a weekly schedule if platelets fell to <30 × 109/L. Eighteen patients were enrolled (median platelet, 14 × 109/L). After the first weekly schedule, ten of eighteen (55.6 %) attained a median titrated dose of 3 mcg/kg and proceeded to the first biweekly schedule. However, all failed to maintain a platelet ≥30 × 109/L for at least 4 weeks, and returned to a second weekly schedule, where eight of the ten achieved a titrated dose (median, 5 mcg/kg) and moved to a second schedule of biweekly romiplostim. Three of the eight (37.5 %) showed platelet ≥30 × 109/L for 4, 8, and 10 weeks, but all eight patients eventually experienced a drop in platelets. Lengthening the dose interval of romiplostim to greater than a week is not feasible to maintain stable platelet count.

© 2004-2018 中国地质图书馆版权所有 京ICP备05064691号 京公网安备11010802017129号

地址:北京市海淀区学院路29号 邮编:100083

电话:办公室:(+86 10)66554848;文献借阅、咨询服务、科技查新:66554700