Immune thrombocytopenia - null regimens

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The purpose of this page is to provide references to "null therapy" such as placebo and observation. These references provide further insight into the historical development of the treatment landscape. See the main ITP page for regimens that include active anticancer treatment.
=Relapsed or refractory=
==Placebo== 
===Regimen===
Study 	Dates of enrollment 	Evidence 	Comparator 	Comparative Efficacy
Bussel et al. 2007 (TRA100773 phase 2)	2005-02 to 2005-11 	Randomized Phase 2 (C)	Eltrombopag 	Lower RR
Kuter et al. 2008 (Amgen 20030105) 	2005-03-01 to 2006-12-31	Phase 3 (C) 	Romiplostim 	Lower durable platelet response
Kuter et al. 2008 (Amgen 20030212) 	2005-03-01 to 2006-12-31	Phase 3 (C) 	Romiplostim 	Lower durable platelet response
Bussel et al. 2009 (TRA100773) 	2006-02-06 to 2006-04-17	Phase 3 (C) 	Eltrombopag 	Lower RR
Ghanima et al. 2015 (RITP) 	2006-08-17 to 2011-06-30	Phase 3 (C) 	Rituximab 	Did not meet primary endpoint of rate of treatment failure within 78 weeks
Cheng et al. 2010 (RAISE-ITP) 	2006-11-22 to 2007-07-31	Phase 3 (C) 	Eltrombopag 	Lower RR
Tarantino et al. 2016 (Amgen 20080279)	2012-01-24 to 2014-09-03	Phase 3 (C) 	Romiplostim 	Lower durable platelet response
Jurczak et al. 2018 (E5501-G000-302) 	2012-02-06 to 2015-04-09	Phase 3 (C) 	Avatrombopag	Lower cumulative weeks of platelet response without rescue therapy for bleeding (primary endpoint)
Grainger et al. 2015 (PETIT2) 	2012-03-15 to 2014-01-02	Phase 3 (C) 	Eltrombopag 	Lower durable platelet response
Broome et al. 2023 (ADVANCE IV) 	2019-12-09 to 2022-02-03	Phase 3 (C) 	Efgartigimod	Seems to have lower sustained platelet count response (primary endpoint)
Hu et al. 2024 (ESLIM-01) 	2021-09-29 to 2022-12-31	Phase 3 (C) 	Sovleplenib 	Lower durable platelet response
No active treatment; used as a comparator arm and here for reference purposes only. Note that RAISE should not be confused with the trial by the same name in colorectal cancer.
===References===
# TRA100773 phase 2: Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, Arning M, Provan D, Jenkins JM. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007 Nov 29;357(22):2237-47. link to original article EuroPMC NCT00102739
# Amgen 20030105: Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article EuroPMC NCT00102323
# Amgen 20030212: Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article EuroPMC NCT00102336
# TRA100773: Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. link to original article EuroPMC NCT00102739
# RAISE-ITP: 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. Epub 2010 Aug 23. Erratum in: Lancet. 2011 Jan 29;377(9763):382. link to original article EuroPMC NCT00370331
# RITP: Ghanima W, Khelif A, Waage A, Michel M, Tjønnfjord GE, Romdhan NB, Kahrs J, Darne B, Holme PA; RITP study group. Rituximab as second-line treatment for adult immune thrombocytopenia (the RITP trial): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2015 Apr 25;385(9978):1653-61. Epub 2015 Feb 4. link to original article EuroPMC NCT00344149
# PETIT2: 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. Epub 2015 Jul 28. link to original article EuroPMC NCT01520909
# Amgen 20080279: Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Epub 2016 Apr 18. link to original article EuroPMC NCT01444417
# 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. link to original article EuroPMC
# E5501-G000-302: Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. Epub 2018 Sep 7. link to original article link to PMC article EuroPMC NCT01438840
#ADVANCE IV: Broome CM, McDonald V, Miyakawa Y, Carpenedo M, Kuter DJ, Al-Samkari H, Bussel JB, Godar M, Ayguasanosa J, De Beuf K, Rodeghiero F, Michel M, Newland A; ADVANCE Investigator Study Group. Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2023 Nov 4;402(10413):1648-1659. Epub 2023 Sep 28. link to original article EuroPMC NCT04188379
# ESLIM-01: Hu Y, Liu X, Zhou H, Wang S, Huang R, Wang Y, Du X, Sun J, Zhou Z, Yan Z, Chen W, Wang W, Liu Q, Zeng Q, Gong Y, Yin J, Shen X, Ye B, Chen Y, Xu Y, Sun H, Cheng Y, Liu Z, Wang C, Yuan G, Zhang X, Li X, Cheng P, Guo X, Jiang Z, Yang F, Yang L, Luo C, Xiao T, Fu S, Yin H, Guo X, Xu Q, Fan S, Shi MM, Su W, Mei H, Yang R. Efficacy and safety of sovleplenib (HMPL-523) in adult patients with chronic primary immune thrombocytopenia in China (ESLIM-01): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Haematol. 2024 Aug;11(8):e567-e579. Epub 2024 Jun 14. link to original article EuroPMC NCT05029635
Category:Immune thrombocytopenia regimens
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Category:Autoimmune hematologic conditions
Category:Bleeding disorders
Category:Cytopenias
Category:Null regimens