Real-world use of thrombopoietin receptor agonists for the management of immune thrombocytopenia in adult patients in the United Kingdom: Results from the TRAIT study.
eltrombopag
immune thrombocytopenia (ITP)
real-world
romiplostim
thrombopoietin receptor agonist (TPO-RA)
Journal
British journal of haematology
ISSN: 1365-2141
Titre abrégé: Br J Haematol
Pays: England
ID NLM: 0372544
Informations de publication
Date de publication:
01 Mar 2024
01 Mar 2024
Historique:
revised:
01
02
2024
received:
29
09
2022
accepted:
06
02
2024
medline:
2
3
2024
pubmed:
2
3
2024
entrez:
2
3
2024
Statut:
aheadofprint
Résumé
Few studies have reported the real-world use of both romiplostim and eltrombopag in immune thrombocytopenia (ITP). TRAIT was a retrospective observational study aimed to evaluate the platelet responses and adverse effects associated with the use of these thrombopoietin receptor agonists (TPO-RAs) in adult patients with ITP in the United Kingdom. Of 267 patients (median age at diagnosis, 48 years) with ITP (primary ITP [n = 218], secondary ITP [n = 49]) included in the study, 112 (42%) received eltrombopag and 155 (58%) received romiplostim as the first prescribed TPO-RA. A platelet count ≥30 × 10
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Novartis Pharmaceuticals UK Limited
Informations de copyright
© 2024 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.
Références
Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23):3829-3866.
Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3(22):3780-3817.
Al-Samkari H, Kuter DJ. Immune thrombocytopenia in adults: modern approaches to diagnosis and treatment. Semin Thromb Hemost. 2020;46(3):275-288.
Zaja F, Carpenedo M, Baratè C, Borchiellini A, Chiurazzi F, Finazzi G, et al. Tapering and discontinuation of thrombopoietin receptor agonists in immune thrombocytopenia: real-world recommendations. Blood Rev. 2020;41:100647.
Wong RSM, Saleh MN, Khelif A, Salama A, Portella MSO, Burgess P, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017;130(23):2527-2536.
Cines DB, Gernsheimer T, Wasser J, Godeau B, Provan D, Lyons R, et al. Integrated analysis of long-term safety in patients with chronic immune thrombocytopaenia (ITP) treated with the thrombopoietin (TPO) receptor agonist romiplostim. Int J Hematol. 2015;102(3):259-270.
Kuter DJ, Bussel JB, Newland A, Baker RI, Lyons RM, Wasser J, et al. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013;161(3):411-423.
Revolade summary of product characteristics [Internet]. European Medicines Agency, Novartis Europharm Limited [cited 2022 Apr]. Available from: https://www.ema.europa.eu/en/documents/product-information/revolade-epar-product-information_en.pdf
Nplate summary of product characteristics [Internet]. European Medicines Agency, Amgen Europe B.V. [cited 2022 Oct]. Available from: https://www.ema.europa.eu/en/documents/product-information/nplate-epar-product-information_en.pdf
National Institute for Health and Care Excellence (NICE). Eltrombopag for treating chronic immune thrombocytopenia [Internet]. Technology appraisal guidance [TA293]. 2013 [cited 2018 Oct 26]. Available from: https://www.nice.org.uk/guidance/ta293
National Institute for Health and Care Excellence (NICE). Romiplostim for the treatment of chronic immune thrombocytopenia [Internet]. Technology appraisal guidance [TA221]. 2011 [cited 2018 Oct 26]. Available from: www.nice.org.uk/guidance/ta221
Doobaree IU, Newland A, McDonald V, Nandigam R, Mensah L, Leroy S, et al. Primary immune thrombocytopenia (ITP) treated with romiplostim in routine clinical practice: retrospective study from the United Kingdom ITP Registry. Eur J Haematol. 2019;102(5):416-423.
Palandri F, Rossi E, Bartoletti D, Ferretti A, Ruggeri M, Lucchini E, et al. Real-world use of thrombopoietin receptor agonists in older patients with primary immune thrombocytopenia. Blood. 2021;138(7):571-583.
Hamed EM, Ibrahim ARN, Meabed MH, Khalaf AM, Demerdash DME, Elgendy MO, et al. The outcomes and adverse drug patterns of immunomodulators and thrombopoietin receptor agonists in primary immune thrombocytopenia Egyptian patients with hemorrhage comorbidity. Pharmaceuticals (Basel). 2023;16(6):868.
Rampotas A, Watson E, Burton K, Hill QA, Pavord S. A real-world study of immune thrombocytopenia management during the COVID-19 pandemic in the UK. Br J Haematol. 2022;196(2):351-355.
Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007;357(22):2237-2247.
Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009;373(9664):641-648.
Cheng G, Saleh MN, Marcher C, Vasey S, Mayer B, Aivado M, et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011;377(9763):393-402.
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(9610):395-403.
Saleh MN, Bussel JB, Cheng G, Meyer O, Bailey CK, Arning M, et al. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013;121(3):537-545.
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(11):2386-2393.
Guitton Z, Terriou L, Lega JC, Nove-Josserand R, Hie M, Amoura Z, et al. Risk of thrombosis with anti-phospholipid syndrome in systemic lupus erythematosus treated with thrombopoietin-receptor agonists. Rheumatology (Oxford). 2018;57(8):1432-1438.
Gonzalez-Lopez TJ, Alvarez-Roman MT, Pascual C, Sanchez-Gonzalez B, Fernandez-Fuentes F, Perez-Rus G, et al. Use of eltrombopag for secondary immune thrombocytopenia in clinical practice. Br J Haematol. 2017;178(6):959-970.
González-López TJ, Alvarez-Román MT, Pascual C, Sánchez-González B, Fernández-Fuentes F, Jarque I, et al. Eltrombopag safety and efficacy for primary chronic immune thrombocytopenia in clinical practice. Eur J Haematol. 2016;97(3):297-302.
Moulis G, Germain J, Rueter M, Lafaurie M, Aroichane M, Comont T, et al. Eltrombopag in adult patients with immune thrombocytopenia in the real-world in France, including off-label use before 6 months of disease duration: the multicenter, prospective ELEXTRA study. Am J Hematol. 2022;97(2):E40-E44.
Gonzalez-Porras JR, Godeau B, Carpenedo M. Switching thrombopoietin receptor agonist treatments in patients with primary immune thrombocytopenia. Ther Adv Hematol. 2019;10:2040620719837906.
Cantoni S, Carpenedo M, Mazzucconi MG, De Stefano V, Carrai V, Ruggeri M, et al. Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: a retrospective collaborative survey from Italian hematology centers. Am J Hematol. 2018;93(1):58-64.
Khellaf M, Viallard JF, Hamidou M, Cheze S, Roudot-Thoraval F, Lefrere F, et al. A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia. Haematologica. 2013;98(6):881-887.
Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, et al. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017;102(8):1342-1351.
Cuker A, Buckley B, Mousseau M, Barve AA, Haenig J, Bussel JB. Early initiation of second-line therapy in primary immune thrombocytopenia: insights from real-world evidence. Ann Hematol. 2023;102(8):2051-2058.