Avatrombopag for the Treatment of Adult Patients with Chronic Immune Thrombocytopenia (cITP): Focus on Patient Selection and Perspectives

References

• Cooper N, Ghanima W. Immune thrombocytopenia. N Engl J Med. 2019;381:945–955.
   PubMed Web of Science ®Google Scholar
• Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) World Impact Survey (iWISh): patient and physician perceptions of diagnosis, signs and symptoms, and treatment. Am J Hematol. 2021;96:188–198.
   PubMed Web of Science ®Google Scholar
• Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): impact of ITP on health-related quality of life. Am J Hematol. 2021;96:199–207.
   PubMed Web of Science ®Google Scholar
• Piel-Julian ML, Mahevas M, Germain J, et al. Risk factors for bleeding, including platelet count threshold, in newly diagnosed immune thrombocytopenia adults. J Thromb Haemost. 2018;16:1830–1842.
   PubMed Web of Science ®Google Scholar
• Zufferey A, Kapur R, Semple JW. Pathogenesis and therapeutic mechanisms in Immune Thrombocytopenia (ITP). J Clin Med. 2017;6:16.
   PubMed Web of Science ®Google Scholar
• Kosugi S, Kurata Y, Tomiyama Y, et al. Circulating thrombopoietin level in chronic immune thrombocytopenic purpura. Br J Haematol. 1996;93:704–706.
   PubMed Web of Science ®Google Scholar
• Makar RS, Zhukov OS, Sahud MA, Kuter DJ. Thrombopoietin levels in patients with disorders of platelet production: diagnostic potential and utility in predicting response to TPO receptor agonists. Am J Hematol. 2013;88:1041–1044.
   PubMed Web of Science ®Google Scholar
• Xu M, Li J, Neves MAD, et al. GPIbalpha is required for platelet-mediated hepatic thrombopoietin generation. Blood. 2018;132:622–634.
   PubMed Web of Science ®Google Scholar
• Schoonen WM, Kucera G, Coalson J, et al. Epidemiology of immune thrombocytopenic purpura in the general practice research database. Br J Haematol. 2009;145:235–244.
   PubMed Web of Science ®Google Scholar
• Frederiksen H, Schmidt K. The incidence of idiopathic thrombocytopenic purpura in adults increases with age. Blood. 1999;94(3):909–913.
   PubMed Web of Science ®Google Scholar
• Christiansen CF, Bahmanyar S, Ghanima W, et al. Chronic immune thrombocytopenia in Denmark, Sweden and Norway: the Nordic Country Patient Registry for Romiplostim. EClinicalMedicine. 2019;14:80–87.
   PubMedGoogle Scholar
• Adelborg K, Kristensen NR, Norgaard M, et al. Cardiovascular and bleeding outcomes in a population-based cohort of patients with chronic immune thrombocytopenia. J Thromb Haemost. 2019;17:912–924.
   PubMed Web of Science ®Google Scholar
• Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3:3829–3866.
   PubMed Web of Science ®Google Scholar
• Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3:3780–3817.
   PubMed Web of Science ®Google Scholar
• de Sauvage FJ, Hass PE, Spencer SD, et al. Stimulation of megakaryocytopoiesis and thrombopoiesis by the c-Mpl ligand. Nature. 1994;369:533–538.
   PubMed Web of Science ®Google Scholar
• Kaushansky K. The molecular mechanisms that control thrombopoiesis. J Clin Invest. 2005;115:3339–3347.
   PubMed Web of Science ®Google Scholar
• Li J, Yang C, Xia Y, et al. Thrombocytopenia caused by the development of antibodies to thrombopoietin. Blood. 2001;98:3241–3248.
   PubMed Web of Science ®Google Scholar
• Wang L, Gao Z, Chen XP, et al. Efficacy and safety of thrombopoietin receptor agonists in patients with primary immune thrombocytopenia: a systematic review and meta-analysis. Sci Rep. 2016;6:39003.
   PubMed Web of Science ®Google Scholar
• Gonzalez-Lopez TJ, Pascual C, Alvarez-Roman MT, et al. Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia. Am J Hematol. 2015;90:E40–E43.
   PubMed Web of Science ®Google Scholar
• Cervinek L, Mayer J, Doubek M. Sustained remission of chronic immune thrombocytopenia after discontinuation of treatment with thrombopoietin-receptor agonists in adults. Int J Hematol. 2015;102:7–11.
   PubMed Web of Science ®Google Scholar
• Mahevas M, Fain O, Ebbo 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;165:865–869.
   PubMed Web of Science ®Google Scholar
• Al-Samkari H. Avatrombopag maleate for the treatment of periprocedural thrombocytopenia in patients with chronic liver disease. Drugs Today. 2018;54:647–655.
   PubMed Web of Science ®Google Scholar
• Kuter DJ, Allen LF. Avatrombopag, an oral thrombopoietin receptor agonist: results of two double-blind, dose-rising, placebo-controlled Phase 1 studies. Br J Haematol. 2018;183:466–478.
   PubMed Web of Science ®Google Scholar
• Nomoto M, Pastino G, Rege B, Aluri J, Ferry J, Han D. Pharmacokinetics, pharmacodynamics, pharmacogenomics, safety, and tolerability of avatrombopag in healthy Japanese and white subjects. Clin Pharmacol Drug Dev. 2018;7:188–195.
   PubMed Web of Science ®Google Scholar
• Nomoto M, Zamora CA, Schuck E, et al. Pharmacokinetic/pharmacodynamic drug-drug interactions of avatrombopag when coadministered with dual or selective CYP2C9 and CYP3A interacting drugs. Br J Clin Pharmacol. 2018;84:952–960.
   PubMed Web of Science ®Google Scholar
• Fukushima-Shintani M, Suzuki K, Iwatsuki Y, et al. AKR-501 (YM477) in combination with thrombopoietin enhances human megakaryocytopoiesis. Exp Hematol. 2008;36:1337–1342.
   PubMed Web of Science ®Google Scholar
• Fukushima-Shintani M, Suzuki K, Iwatsuki Y, et al. AKR-501 (YM477) a novel orally-active thrombopoietin receptor agonist. Eur J Haematol. 2009;82:247–254.
   PubMed Web of Science ®Google Scholar
• Michelson AD, Smolensky Koganov E, Forde EE, Carmichael SL, Frelinger AL 3rd. Avatrombopag increases platelet count but not platelet activation in patients with thrombocytopenia resulting from liver disease. J Thromb Haemost. 2018;16:2515–2519.
   PubMed Web of Science ®Google Scholar
• Kuter DJ. The structure, function, and clinical use of the thrombopoietin receptor agonist avatrombopag. Blood Rev. 2021:100909. doi:10.1016/j.blre.2021.100909
   PubMed Web of Science ®Google Scholar
• Bussel JB, Kuter DJ, Aledort LM, et al. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014;123:3887–3894.
   PubMed Web of Science ®Google Scholar
• Jurczak W, Chojnowski K, Mayer J, et al. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018;183:479–490.
   PubMed Web of Science ®Google Scholar
• Ghanima W, Cooper N, Rodeghiero F, Godeau B, Bussel JB. Thrombopoietin receptor agonists: ten years later. Haematologica. 2019;104:1112–1123.
   PubMed Web of Science ®Google Scholar
• Steurer M, Quittet P, Papadaki HA, et al. A large observational study of patients with primary immune thrombocytopenia receiving romiplostim in European clinical practice. Eur J Haematol. 2016;98:112–120.
   PubMed Web of Science ®Google Scholar
• Terrault NA, Hassanein T, Howell CD, et al. Phase II study of avatrombopag in thrombocytopenic patients with cirrhosis undergoing an elective procedure. J Hepatol. 2014;61:1253–1259.
   PubMed Web of Science ®Google Scholar
• Terrault N, Chen YC, Izumi N, et al. Avatrombopag before procedures reduces need for platelet transfusion in patients with chronic liver disease and thrombocytopenia. Gastroenterology. 2018;155:705–718.
   PubMed Web of Science ®Google Scholar
• Gonzalez-Porras JR, Godeau B, Carpenedo M. Switching thrombopoietin receptor agonist treatments in patients with primary immune thrombocytopenia. Ther Adv Hematol. 2019;10:2040620719837906.
   Web of Science ®Google Scholar
• Al-Samkari H, Jiang D, Gernsheimer T, et al. Adults with immune thrombocytopenia who switched to avatrombopag following prior treatment with eltrombopag or romiplostim: A multicentre US study.  J Haematol. 2022. doi:10.1111/bjh.18081
   Google Scholar
• Ghanima W, Gernsheimer T, Kuter DJ. How I treat primary ITP in adult patients who are unresponsive to or dependent on corticosteroid treatment. Blood. 2021;137:2736–2744.
   PubMed Web of Science ®Google Scholar
• Miltiadous O, Hou M, Bussel JB. Identifying and treating refractory ITP: difficulty in diagnosis and role of combination treatment. Blood. 2020;135:472–490.
   PubMed Web of Science ®Google Scholar