Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use

Overview

Journal Patient Prefer Adherence

Date 2020 Aug 18

PMID 32801654

Citations 4

Authors

Megan M Gilbert

Amanda B Grimes

Taylor Olmsted Kim

Jenny M Despotovic

Affiliations

Soon will be listed here.

Abstract

Immune thrombocytopenia (ITP) is an immune-mediated disorder resulting in platelet destruction and subsequent thrombocytopenia. Bleeding symptoms range from mild cutaneous bleeding to life-threatening hemorrhage. Romiplostim, a peptide-antibody fusion product, is a thrombopoietin receptor agonist (TPO-RA) indicated for use in patients with ITP. Romiplostim is US Food and Drug Administration (FDA) approved in children ≥1 year of age with ITP of >6 months' duration who have had an inadequate response to first-line therapies or splenectomy. FDA approval in adults with chronic ITP was expanded in October 2019 to include adults with newly diagnosed (<3 months' duration) and persistent (3-12 months' duration) ITP who demonstrated an inadequate response to first-line therapies, including corticosteroids and immunoglobulins, or splenectomy. The newly published 2019 American Society of Hematology ITP Guidelines place TPO-RAs, including romiplostim, as second-line therapies in both children and adults. Here, we review the use of romiplostim as second-line therapy with a spotlight on health-related quality of life, ease of use, and patient preference.

Citing Articles

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Rovo A, Cantoni N, Samii K, Rufer A, Koenen G, Ivic S PLoS One. 2022; 17(4):e0267342.

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References

Neunert C, Terrell D, Arnold D, Buchanan G, Cines D, Cooper N . American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019; 3(23):3829-3866. PMC: 6963252. DOI: 10.1182/bloodadvances.2019000966. View

Lee J, Lee S, Jung C, Park S, Keta H, Park S . Romiplostim in patients with refractory aplastic anaemia previously treated with immunosuppressive therapy: a dose-finding and long-term treatment phase 2 trial. Lancet Haematol. 2019; 6(11):e562-e572. DOI: 10.1016/S2352-3026(19)30153-X. View

Grace R, Shimano K, Bhat R, Neunert C, Bussel J, Klaassen R . Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019; 94(7):741-750. PMC: 6527349. DOI: 10.1002/ajh.25479. View

Olsson B, Andersson P, Jernas M, Jacobsson S, Carlsson B, Carlsson L . T-cell-mediated cytotoxicity toward platelets in chronic idiopathic thrombocytopenic purpura. Nat Med. 2003; 9(9):1123-4. DOI: 10.1038/nm921. View

Kong Z, Qin P, Xiao S, Zhou H, Li H, Yang R . A novel recombinant human thrombopoietin therapy for the management of immune thrombocytopenia in pregnancy. Blood. 2017; 130(9):1097-1103. DOI: 10.1182/blood-2017-01-761262. View

Wang Y, Krzyzanski W, Doshi S, Xiao J, Perez-Ruixo J, Chow A . Pharmacodynamics-mediated drug disposition (PDMDD) and precursor pool lifespan model for single dose of romiplostim in healthy subjects. AAPS J. 2010; 12(4):729-40. PMC: 2976980. DOI: 10.1208/s12248-010-9234-9. View

Wang S, Yang R, Zou P, Hou M, Wu D, Shen Z . A multicenter randomized controlled trial of recombinant human thrombopoietin treatment in patients with primary immune thrombocytopenia. Int J Hematol. 2012; 96(2):222-8. DOI: 10.1007/s12185-012-1124-8. View

Grozovsky R, Giannini S, Falet H, Hoffmeister K . Novel mechanisms of platelet clearance and thrombopoietin regulation. Curr Opin Hematol. 2015; 22(5):445-51. PMC: 5326579. DOI: 10.1097/MOH.0000000000000170. View

McIntosh B, Kaushansky K . Transcriptional regulation of bone marrow thrombopoietin by platelet proteins. Exp Hematol. 2008; 36(7):799-806. PMC: 2517608. DOI: 10.1016/j.exphem.2008.02.012. View

10.

Neunert C, Noroozi N, Norman G, Buchanan G, Goy J, Nazi I . Severe bleeding events in adults and children with primary immune thrombocytopenia: a systematic review. J Thromb Haemost. 2014; 13(3):457-64. PMC: 4991942. DOI: 10.1111/jth.12813. View

11.

Foster D, Sprecher C, Grant F, Kramer J, Kuijper J, Holly R . Human thrombopoietin: gene structure, cDNA sequence, expression, and chromosomal localization. Proc Natl Acad Sci U S A. 1994; 91(26):13023-7. PMC: 45573. DOI: 10.1073/pnas.91.26.13023. View

12.

Ahmed R, Devasia A, Viswabandya A, Lakshmi K, Abraham A, Karl S . Long-term outcome following splenectomy for chronic and persistent immune thrombocytopenia (ITP) in adults and children : Splenectomy in ITP. Ann Hematol. 2016; 95(9):1429-34. DOI: 10.1007/s00277-016-2738-3. View

13.

Gonzalez K, Zuluaga S, DaRos C, Rodriguez P, Marti A . Sequential treatment with thrombopoietin-receptor agonists (TPO-RAs) in immune thrombocytopenia (ITP): experience in our center. Ann Hematol. 2016; 96(3):507-508. DOI: 10.1007/s00277-016-2896-3. View

14.

ROBINETTE C, Fraumeni Jr J . Splenectomy and subsequent mortality in veterans of the 1939-45 war. Lancet. 1977; 2(8029):127-9. DOI: 10.1016/s0140-6736(77)90132-5. View

15.

Lok S, Kaushansky K, Holly R, Kuijper J, Oort P, Grant F . Cloning and expression of murine thrombopoietin cDNA and stimulation of platelet production in vivo. Nature. 1994; 369(6481):565-8. DOI: 10.1038/369565a0. View

16.

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(1):7-11. DOI: 10.1007/s12185-015-1793-1. View

17.

Mahevas M, Fain O, Ebbo M, Roudot-Thoraval F, Limal N, Khellaf M . 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. DOI: 10.1111/bjh.12888. View

18.

Neunert C, Lim W, Crowther M, Cohen A, Solberg Jr L, Crowther M . The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011; 117(16):4190-207. DOI: 10.1182/blood-2010-08-302984. View

19.

Broudy V, Lin N . AMG531 stimulates megakaryopoiesis in vitro by binding to Mpl. Cytokine. 2003; 25(2):52-60. DOI: 10.1016/j.cyto.2003.05.001. View

20.

Mathias S, Li X, Eisen M, Carpenter N, Crosby R, Blanchette V . A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Effect of Romiplostim on Health-Related Quality of Life in Children with Primary Immune Thrombocytopenia and Associated Burden in Their Parents. Pediatr Blood Cancer. 2016; 63(7):1232-7. PMC: 5071741. DOI: 10.1002/pbc.25984. View