TPO-RAs Combining Anti-CD 20 Monoclonal Antibody Versus TPO-RAs in the Management of Pediatric Primary Immune Thrombocytopenia (ITP)

Summary

This multicenter randomized, open-label study aim to compare the efficacy and safety of TPO-RAs combining anti-CD 20 monoclonal antibody with TPO-RAs in China pediatric ITP patients .This study will be conducted in persistent or chronic pediatric ITP patients who had not responded to or had relapsed after previous glucocorticoid treatment.

Timeline

Start

2023-02-23

Primary completion

2025-05-14

Completion

2025-11-14

Publications

• Results Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866. doi: 10.1182/bloodadvances.2019000966.
• Results Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.
• Results Working Group of Chinese Guideline for the Diagnosis and Treatment of Childhood Primary Immune Thrombocytopenia; Subspecialty Group of Hematologic Diseases, the Society of Pediatrics, Chinese Medical Association; Editorial Board, Chinese Journal of Pediatrics. [Adapted guideline for the diagnosis and treatment of primary immune thrombocytopenia for Chinese children (2021)]. Zhonghua Er Ke Za Zhi. 2021 Oct 2;59(10):810-819. doi: 10.3760/cma.j.cn112140-20210509-00397. Chinese.
• Results Cheng G, Saleh MN, Marcher C, Vasey S, Mayer B, Aivado M, Arning M, Stone NL, Bussel JB. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011 Jan 29;377(9763):393-402. doi: 10.1016/S0140-6736(10)60959-2. Epub 2010 Aug 23.
• Results Wong RSM, Saleh MN, Khelif A, Salama A, Portella MSO, Burgess P, Bussel JB. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 Dec 7;130(23):2527-2536. doi: 10.1182/blood-2017-04-748707. Epub 2017 Oct 17.
• Results Yang R, Li J, Jin J, Huang M, Yu Z, Xu X, Zhang X, Hou M. Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia. Br J Haematol. 2017 Jan;176(1):101-110. doi: 10.1111/bjh.14380. Epub 2016 Oct 13.
• Results Cheng X, Fu L, Ma J, Gu H, Chen Z, Zhao L, Wang X, Wu R. Spotlight on eltrombopag in pediatric ITP in China: a long-term observational study in real-world practice. Blood Adv. 2021 Oct 12;5(19):3799-3806. doi: 10.1182/bloodadvances.2020004110.
• Results Qu M, Zhou J, Yang SJ, Zhou ZP. Efficacy and safety of rituximab for minors with immune thrombocytopenia: a systematic review and meta-analysis. J Int Med Res. 2020 Oct;48(10):300060520962348. doi: 10.1177/0300060520962348.
• Results Ayad N, Grace RF, Al-Samkari H. Thrombopoietin receptor agonists and rituximab for treatment of pediatric immune thrombocytopenia: A systematic review and meta-analysis of prospective clinical trials. Pediatr Blood Cancer. 2022 Mar;69(3):e29447. doi: 10.1002/pbc.29447. Epub 2021 Dec 28.
• Results Sui T, Zhang L, Zhou ZP, Xue F, Ge J, Yang RC. [Efficacy and safety of two different low-dose rituximab regimens for Chinese adult patients with immune thrombocytopenia]. Zhonghua Xue Ye Xue Za Zhi. 2011 Sep;32(9):583-6. Chinese.
• Results Liu X, Huang Y, Chen Y, Liu W, Xue F, Zhang L, Yang R. [Clinical analysis of lower doses rituximab for children primary immune thrombocytopenia]. Zhonghua Xue Ye Xue Za Zhi. 2014 Dec;35(12):1079-82. doi: 10.3760/cma.j.issn.0253-2727.2014.12.007. Chinese.
• Results Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, Ghanima W, Godeau B, Gonzalez-Lopez TJ, Grainger J, Hou M, Kruse C, McDonald V, Michel M, Newland AC, Pavord S, Rodeghiero F, Scully M, Tomiyama Y, Wong RS, Zaja F, Kuter DJ. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019 Nov 26;3(22):3780-3817. doi: 10.1182/bloodadvances.2019000812.
• Results Bussel JB, de Miguel PG, Despotovic JM, Grainger JD, Sevilla J, Blanchette VS, Krishnamurti L, Connor P, David M, Boayue KB, Matthews DC, Lambert MP, Marcello LM, Iyengar M, Chan GW, Chagin KD, Theodore D, Bailey CK, Bakshi KK. Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicentre, placebo-controlled study. Lancet Haematol. 2015 Aug;2(8):e315-25. doi: 10.1016/S2352-3026(15)00114-3. Epub 2015 Jul 28.
• Results Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, Pongtanakul B, Komvilaisak P, Sosothikul D, Drelichman G, Sirachainan N, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Bussel JB, Bakshi KK, Iyengar M, Chan GW, Chagin KD, Theodore D, Marcello LM, Bailey CK. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015 Oct 24;386(10004):1649-58. doi: 10.1016/S0140-6736(15)61107-2. Epub 2015 Jul 28.

Drugs

Indications