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摘要: 免疫性血小板减少症(immune thrombocytopenia,ITP)是一种常见的自身免疫性出血性疾病,表现为外周血血小板计数减低。针对ITP的经典初始治疗方案仍为糖皮质激素,但部分患者对糖皮质激素耐药或依赖,随着多种新药的出现及药物联合方案的应用,ITP患者的临床管理已由经典的免疫抑制治疗为主逐渐向个体化方案制定转变。但目前针对激素耐药或依赖的ITP患者的治疗方法尚无明确优先级别推荐。本文将对激素耐药或依赖的ITP患者的治疗进行整理概述,以期为临床医生治疗提供参考。Abstract: Immune thrombocytopenia(ITP) is a common autoimmune bleeding disorder characterized by decreased peripheral blood platelet count. The classical initial treatment is corticosteroids for newly diagnosed ITP in adults. However, some patients developed resistance to corticosteroids or become dependent on them. New therapeutic approaches and the strategic combination have altered the classical immunosuppressive therapies to individualized treatments. Until now, the priority of treatments for corticosteroid-resistant or dependent ITP patients is seldom articulated. Here we provide a comprehensive overview of the therapeutic strategies for corticosteroid-resistant or dependent ITP patients.
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Key words:
- corticosteroid-resistant /
- corticosteroid-dependent /
- immune thrombocytopenia /
- treatment
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[1] Frederiksen H, Ghanima W. Response of first line treatment with corticosteroids in a population-based cohort of adults with primary immune thrombocytopenia[J]. Eur J Intern Med, 2017, 37: e23-e25. doi: 10.1016/j.ejim.2016.09.001
[2] Yu J, Xu Z, Zhuo Y, et al. Development and validation of a nomogram for steroid-resistance prediction in immune thrombocytopenia patients[J]. Hematology, 2021, 26(1): 956-963. doi: 10.1080/16078454.2021.2003066
[3] Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia[J]. Blood Adv, 2019, 3(22): 3780-3817. doi: 10.1182/bloodadvances.2019000812
[4] Birocchi S, Podda GM, Manzoni M, et al. Thrombopoietin receptor agonists for the treatment of primary immune thrombocytopenia: a meta-analysis and systematic review[J]. Platelets, 2021, 32(2): 216-226. doi: 10.1080/09537104.2020.1745168
[5] Al-Samkari H, Kuter DJ. Thrombopoietin level predicts response to treatment with eltrombopag and romiplostim in immune thrombocytopenia[J]. Am J Hematol, 2018, 93(12): 1501-1508. doi: 10.1002/ajh.25275
[6] 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]. Br J Haematol, 2022, 197(3): 359-366. doi: 10.1111/bjh.18081
[7] Gonzalez-Porras JR, Godeau B, Carpenedo M. Switching thrombopoietin receptor agonist treatments in patients with primary immune thrombocytopenia[J]. Ther Adv Hematol, 2019, 10: 2040620719837906.
[8] Khellaf M, Viallard JF, Hamidou M, et al. A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia[J]. Haematologica, 2013, 98(6): 881-887. doi: 10.3324/haematol.2012.074633
[9] Wang S, Yang R, Zou P, et al. A multicenter randomized controlled trial of recombinant human thrombopoietin treatment in patients with primary immune thrombocytopenia[J]. Int J Hematol, 2012, 96(2): 222-228. doi: 10.1007/s12185-012-1124-8
[10] 蔡华聪, 王书杰, 富玲, 等. 重组人血小板生成素用于原发免疫性血小板减少症维持治疗的有效性和安全性——多中心临床研究[J]. 中华血液学杂志, 2017, 38(5): 379-383.
[11] Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study[J]. Blood, 2017, 130(23): 2527-2536. doi: 10.1182/blood-2017-04-748707
[12] Mei H, Liu X, Li Y, et al. A multicenter, randomized phase Ⅲ trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia[J]. J Hematol Oncol, 2021, 14(1): 37. doi: 10.1186/s13045-021-01047-9
[13] 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[J]. Br J Haematol, 2018, 183(3): 479-490. doi: 10.1111/bjh.15573
[14] Snell Taylor SJ, Nielson CM, Breskin A, et al. Effectiveness and Safety of Romiplostim Among Patients with Newly Diagnosed, Persistent and Chronic Immune Thrombocytopenia in European Clinical Practice[J]. Adv Ther, 2021, 38(5): 2673-2688. doi: 10.1007/s12325-021-01727-5
[15] Ghadaki B, Nazi I, Kelton JG, et al. Sustained remissions of immune thrombocytopenia associated with the use of thrombopoietin receptor agonists[J]. Transfusion, 2013, 53(11): 2807-2812. doi: 10.1111/trf.12139
[16] Zaja F, Carpenedo M, Barate C, et al. Tapering and discontinuation of thrombopoietin receptor agonists in immune thrombocytopenia: Real-world recommendations[J]. Blood Rev, 2020, 41: 100647. doi: 10.1016/j.blre.2019.100647
[17] Cooper N, Hill QA, Grainger J, et al. Tapering and Discontinuation of Thrombopoietin Receptor Agonist Therapy in Patients with Immune Thrombocytopenia: Results from a Modified Delphi Panel[J]. Acta Haematol, 2021, 144(4): 418-426. doi: 10.1159/000510676
[18] van Dijk WEM, Brandwijk ON, Heitink-Polle KMJ, et al. Hemostatic changes by thrombopoietin-receptor agonists in immune thrombocytopenia patients[J]. Blood Rev, 2021, 47: 100774. doi: 10.1016/j.blre.2020.100774
[19] Lucchini E, Zaja F, Bussel J. Rituximab in the treatment of immune thrombocytopenia: what is the role of this agent in 2019?[J]. Haematologica, 2019, 104(6): 1124-1135. doi: 10.3324/haematol.2019.218883
[20] Dong Y, Yue M, Hu M. The Efficacy and Safety of Different Dosages of Rituximab for Adults with Immune Thrombocytopenia: A Systematic Review and Meta-Analysis[J]. Biomed Res Int, 2021, 2021: 9992086.
[21] Pasa S, Altintas A, Cil T, et al. The efficacy of rituximab in patients with splenectomized refractory chronic idiopathic thrombocythopenic purpura[J]. J Thromb Thrombolysis, 2009, 27(3): 329-333. doi: 10.1007/s11239-008-0208-z
[22] Arnold DM, Heddle NM, Carruthers J, et al. A pilot randomized trial of adjuvant rituximab or placebo for nonsplenectomized patients with immune thrombocytopenia[J]. Blood, 2012, 119(6): 1356-1362. doi: 10.1182/blood-2011-08-374777
[23] Miyakawa Y, Katsutani S, Yano T, et al. Efficacy and safety of rituximab in Japanese patients with relapsed chronic immune thrombocytopenia refractory to conventional therapy[J]. Int J Hematol, 2015, 102(6): 654-661. doi: 10.1007/s12185-015-1887-9
[24] Marangon M, Vianelli N, Palandri F, et al. Rituximab in immune thrombocytopenia: gender, age, and response as predictors of long-term response[J]. Eur J Haematol, 2017, 98(4): 371-377. doi: 10.1111/ejh.12839
[25] Serris A, Amoura Z, Canoui-Poitrine F, et al. Efficacy and safety of rituximab for systemic lupus erythematosus-associated immune cytopenias: A multicenter retrospective cohort study of 71 adults[J]. Am J Hematol, 2018, 93(3): 424-429. doi: 10.1002/ajh.24999
[26] Bussel J, Arnold DM, Grossbard E, et al. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: Results of two phase 3, randomized, placebo-controlled trials[J]. Am J Hematol, 2018, 93(7): 921-930. doi: 10.1002/ajh.25125
[27] Bussel JB, Arnold DM, Boxer MA, et al. Long-term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program[J]. Am J Hematol, 2019, 94(5): 546-553. doi: 10.1002/ajh.25444
[28] Vianelli N, Palandri F, Polverelli N, et al. Splenectomy as a curative treatment for immune thrombocytopenia: a retrospective analysis of 233 patients with a minimum follow up of 10 years[J]. Haematologica, 2013, 98(6): 875-880. doi: 10.3324/haematol.2012.075648
[29] Tada K, Ohta M, Saga K, et al. Long-term outcomes of laparoscopic versus open splenectomy for immune thrombocytopenia[J]. Surg Today, 2018, 48(2): 180-185. doi: 10.1007/s00595-017-1570-2
[30] Chater C, Terriou L, Duhamel A, et al. Reemergence of Splenectomy for ITP Second-line Treatment?[J]. Ann Surg, 2016, 264(5): 772-777. doi: 10.1097/SLA.0000000000001912
[31] Boyle S, White RH, Brunson A, et al. Splenectomy and the incidence of venous thromboembolism and sepsis in patients with immune thrombocytopenia[J]. Blood, 2013, 121(23): 4782-4290. doi: 10.1182/blood-2012-12-467068
[32] Liu XG, Hou Y, Hou M. How we treat primary immune thrombocytopenia in adults[J]. J Hematol Oncol, 2023, 16(1): 4. doi: 10.1186/s13045-023-01401-z
[33] Robak T, Kazmierczak M, Jarque I, et al. Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia[J]. Blood Adv, 2020, 4(17): 4136-4146. doi: 10.1182/bloodadvances.2020002003
[34] Newland AC, Sanchez-Gonzalez B, Rejto L, et al. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia[J]. Am J Hematol, 2020, 95(2): 178-187. doi: 10.1002/ajh.25680
[35] Kuter DJ, Efraim M, Mayer J, et al. Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia[J]. N Engl J Med, 2022, 386(15): 1421-1431. doi: 10.1056/NEJMoa2110297
[36] Broome CM, Roth A, Kuter DJ, et al. Safety and efficacy of classical complement pathway inhibition with sutimlimab in chronic immune thrombocytopenia[J]. Blood Adv, 2023, 7(6): 987-996. doi: 10.1182/bloodadvances.2021006864
[37] Zhou H, Qin P, Liu Q, et al. A prospective, multicenter study of low dose decitabine in adult patients with refractory immune thrombocytopenia[J]. Am J Hematol, 2019, 94(12): 1374-1381. doi: 10.1002/ajh.25646
[38] Zhou H, Xu M, Qin P, et al. A multicenter randomized open-label study of rituximab plus rhTPO vs rituximab in corticosteroid-resistant or relapsed ITP[J]. Blood, 2015, 125(10): 1541-1547. doi: 10.1182/blood-2014-06-581868
[39] Wu YJ, Liu H, Zeng QZ, et al. All-trans retinoic acid plus low-dose rituximab vs low-dose rituximab in corticosteroid-resistant or relapsed ITP[J]. Blood, 2022, 139(3): 333-342. doi: 10.1182/blood.2021013393
[40] Feng FE, Feng R, Wang M, et al. Oral all-trans retinoic acid plus danazol versus danazol as second-line treatment in adults with primary immune thrombocytopenia: a multicentre, randomised, open-label, phase 2 trial[J]. Lancet Haematol, 2017, 4(10): e487-e496. doi: 10.1016/S2352-3026(17)30170-9
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