10,795
Views
6
CrossRef citations to date
0
Altmetric
Review

Immune thrombocytopenia

, , , &
Pages 1013-1025 | Received 12 Jul 2021, Accepted 15 Oct 2021, Published online: 18 Nov 2021

References

  • Rodeghiero F, Stasi R, Gernsheimer T, 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.
  • Cines DB, Blanchette VS. Immune thrombocytopenic purpura. N Engl J Med. 2002;346(13):995–1008.
  • Frederiksen H, Schmidt K. The incidence of idiopathic thrombocytopenic purpura in adults increases with age. Blood. 1999;94(3):909–913.
  • Neylon AJ, Saunders PWG, Howard MR, et al. Clinically significant newly presenting autoimmune thrombocytopenic purpura in adults: a prospective study of a population-based cohort of 245 patients. Br J Haematol. 2003;122(6):966–974.
  • Abrahamson PE, Hall SA, Feudjo-Tepie M, et al. The incidence of idiopathic thrombocytopenic purpura among adults: a population-based study and literature review. Eur J Haematol. 2009;83(2):83–89.
  • Kaushansky K. Thrombopoietin. N Engl J Med. 1998;339(11):746–754.
  • Gernsheimer T, Stratton J, Ballem PJ, et al. Mechanisms of response to treatment in autoimmune thrombocytopenic purpura. N Engl J Med. 1989;320(15):974–980.
  • Kuter DJ, and Gernsheimer TB. Thrombopoietin and platelet production in chronic immune thrombocytopenia. Hematol Oncol Clin North Am. 2009;23(6):1193–1211.
  • McMillan R, Wang L, Tomer A, et al. Suppression of in vitro megakaryocyte production by antiplatelet autoantibodies from adult patients with chronic ITP. Blood. 2004;103(4):1364–1369.
  • Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115(2):168–186.
  • Lacey JV, Penner JA. Management of idiopathic thrombocytopenic purpura in the adult. Semin Thromb Hemost. 1977;3(3):160–174.
  • Cortelazzo S, Finazzi G, Buelli M, et al. High risk of severe bleeding in aged patients with chronic idiopathic thrombocytopenic purpura. Blood. 1991;77(1):31–33.
  • Cohen YC, Djulbegovic B, Shamai-Lubovitz O, et al. The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts. Arch Intern Med. 2000;160(11):1630–1638.
  • Sarpatwari A, Bennett D, Logie JW, et al. Thromboembolic events among adult patients with primary immune thrombocytopenia in the United Kingdom general practice research database. Haematologica. 2010;95(7):1167–1175.
  • Warrier R, and Chauhan A. Management of immune thrombocytopenic purpura: an update. Ochsner J. 2012;12(3):221–227.
  • Hill QA, Newland AC. Fatigue in immune thrombocytopenia. Br J Haematol. 2015;170(2):141–149.
  • Newton JL, Reese JA, Watson SI, et al. Fatigue in adult patients with primary immune thrombocytopenia. Eur J Haematol. 2011;86(5):420–429.
  • Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) world impact survey (I-WISh): patient and physician perceptions of diagnosis, signs and symptoms, and treatment. Am J Hematol. 2021a;96:188–198.
  • Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) world impact Survey (I-WISh): impact of ITP on health-related quality of life. Am J Hematol. 2021b;96(2):199–207.
  • Bakchoul T, Irene Marini I. Drug-associated thrombocytopenia. Hematology Am Soc Hematol Educ Program. 2018;2018(1):576–583.
  • Almazni I, Stapley R, Morgan NV, et al. Inherited thrombocytopenia: update on genes and genetic variants which may be associated with bleeding. Front Cardiovasc Med. 2019;6:80.
  • Go RS. Idiopathic cyclic thrombocytopenia. Blood Rev. 2005;19(1):53–59.
  • Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3(22):3780–3817.
  • Arnold DM, Nazy I, Clare R, et al. Misdiagnosis of primary immune thrombocytopenia and frequency of bleeding: lessons from the McMaster ITP registry. Blood Adv. 2017;1(25):2414–2420.
  • Neunert C, Terrell DR, Arnold DM, et al. American society of hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23): 3829–3866.
  • Care A, Pavord S, Knight M, et al. Severe primary autoimmune thrombocytopenia in pregnancy: a national cohort study. BJOG. 2018;125(5):604–612.
  • Kalaycı H, Durdağ GD, Baran SY, et al. Pregnancy of patients with idiopathic thrombocytopenic purpura: maternal and neonatal outcomes. J Turk Ger Gynecol Assoc. 2020;21(2):97–101.
  • Neunert C, Lim W, Crowther M, et al. The American society of hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011;117(16):4190–4207.
  • Cines DB, Bussel JB. How I treat idiopathic thrombocytopenic purpura (ITP). Blood. 2005;106(7):2244–2251.
  • Khellaf M, Viallard JF, Hamidou M, et al. A retrospective pilot evaluation of switching thrombopoietic receptor-agonists in immune thrombocytopenia. Haematologica. 2013;98:881–887.
  • Kuter DJ, Macahilig C, Grotzinger KM, et al. Treatment patterns and clinical outcomes in patients with chronic immune thrombocytopenia (ITP) switched to eltrombopag or romiplostim. Int J Hematol. 2015;101(3):255–263.
  • Lakhwani S, Perera M, Fernández-Fuertes F, et al. Thrombopoietin receptor agonist switch in adult primary immune thrombocytopenia patients: a retrospective collaborative survey involving 4 Spanish centres. Eur J Haematol. 2017;99(4):372–377.
  • Palandri F, Rossi E, Bartoletti D, et al. Real-world use of thrombopoietin receptor agonists in elderly patients with primary immune thrombocytopenia. Blood. 2021 Apr 22:blood.2021010735. DOI:https://doi.org/10.1182/blood.2021010735.
  • Wei Y, Ji XB, Wang YW, et al. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016;127(3):296–302.
  • Mithoowani S, Gregory-Miller K, Goy J, et al. High-dose dexamethasone compared with prednisone for previously untreated primary immune thrombocytopenia: a systematic review and meta-analysis. Lancet Haematol. 2016;3(10):e489–e496.
  • Bussel JB, Kuter DJ, George JN, et al. AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP. N Engl J Med. 2006;355(16):1672–1681.
  • Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008;371(9610):395–403.
  • Bussel JB, Cheng G, Saleh MN, et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007;357(22):2237–2247.
  • Bussel JB, Kuter DJ, Aledort LM, et al. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014;123(25):3887–3894.
  • Cheloff AZ, Al-Samkari H. Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease. J Blood Med. 2019;10:313–321.
  • Cheng G, Saleh MN, Marcher C, et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011;377(9763):393–402.
  • Newland A, Godeau B, Priego V, et al. Remission and platelet responses with romiplostim in primary immune thrombocytopenia: final results from a phase 2 study. Br J Haematol. 2016;172(2):262–273.
  • Lucchini E, Palandri F, and Volpetti S, et al. Eltrombopag as second line therapy in adult patients with primary immune thrombocytopenia (ITP) in attempt to treatment-free remission. Updated Results of a Phase II Prospective Study by GIMEMA Group. Presented at the 24th European Hematology Association (EHA) Congress; 2019; Amsterdam, The Netherlands; PF695. Updated Results of a Phase II Prospective Study by GIMEMA Group https://library.ehaweb.org/eha/2019/24th/266494/elisa.lucchini.eltrombopag.as.second.line.therapy.in.adult.patients.with.html?f=listing%3D3%2Abrowseby%3D8%2Asortby%3D1%2Amedia%3D1 Accessed 22 October 2021
  • Saleh MN, Bussel JB, Cheng G, 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.
  • Wong RSM, Mansoor N, Saleh MN, 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.
  • Khellaf M, Michel M, Quittet P, et al. Romiplostim safety and efficacy for immune thrombocytopenia in clinical practice: 2-year results of 72 adults in a romiplostim compassionate-use program. Blood. 2011;118(16):4338–4345.
  • Bussel JB, Hsieh L, Buchanan GR, et al. Long-term use of the thrombopoietin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP). Pediatr Blood Cancer. 2015;62(2):208–213.
  • Zaja F, Barcellini W, Cantoni S, et al. Thrombopoietin receptor agonists for preparing adult patients with immune thrombocytopenia to splenectomy: results of a retrospective, observational GIMEMA study. Am J Hematol. 2016;91(5):E293–E295.
  • Allen R, Bryden P, Grotzinger KM, et al. Cost-effectiveness of eltrombopag versus romiplostim for the treatment of chronic immune thrombocytopenia in England and Wales. Value Health. 2016;19(5):614–622.
  • Fust K, Parthan A, Li X, et al. Cost per response analysis of strategies for chronic immune thrombocytopenia. Am J Manag Care. 2018;24((8 Spec No.):SP294–SP302.
  • Tremblay G, Dolph M, Roy AN, et al. The cost-effectiveness of eltrombopag for the treatment of immune thrombocytopenia in the United States. Clin Ther. 2020;42(5):860–872. e8.
  • Michel M, Ruggeri M, Gonzalez-Lopez TJ, et al. Use of thrombopoietin receptor agonists for immune thrombocytopenia in pregnancy: results from a multicenter study. Blood. 2020 Aug 19:blood.2020007594. DOI:https://doi.org/10.1182/blood.2020007594.
  • Arnold DM, Dentali F, Crowther MA, et al. Systematic review: efficacy and safety of rituximab for adults with idiopathic thrombocytopenic purpura. Ann Intern Med. 2007;146(1):25–33.
  • Patel VL, Mahévas M, Lee SY, et al. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012;119(25):5989–5995.
  • Zaja F, Volpetti S, Chiozzotto M, et al. Long-term follow-up analysis after rituximab salvage therapy in adult patients with immune thrombocytopenia. Am J Hematol. 2012;87(9):886–889.
  • Khellaf M, Anaïs Charles-Nelson A, Fain O, et al. Safety and efficacy of rituximab in adult immune thrombocytopenia: results from a prospective registry including 248 patients. Blood. 2014;124(22):3228–3236.
  • Cooper N, Stasi R, Cunningham-Rundles S, et al. The efficacy and safety of B-cell depletion with anti-CD20 monoclonal antibody in adults with chronic immune thrombocytopenic purpura. Br J Haematol. 2004;125(2):232–239.
  • Braendstrup P, Bjerrum OW, Nielsen OJ, et al. Rituximab chimeric anti-CD20 monoclonal antibody treatment for adult refractory idiopathic thrombocytopenic purpura. Am J Hematol. 2005;78(4):275–280.
  • Peñalver FJ, Jiménez-Yuste V, Almagro M, et al. Rituximab in the management of chronic immune thrombocytopenic purpura: an effective and safe therapeutic alternative in refractory patients. Ann Hematol. 2006;85(8):400–406.
  • Zaja F, Vianelli N, Battista M, et al. Earlier administration of rituximab allows higher rate of long-lasting response in adult patients with autoimmune thrombocytopenia. Exp Hematol. 2006;34(5):571–572.
  • Garcia-Chavez J, Majluf-Cruz A, Montiel-Cervanteset L, et al. Rituximab therapy for chronic and refractory immune thrombocytopenic purpura: a long-term follow-up analysis. Ann Hematol. 2007;86(12):871–877.
  • Godeau B, Porcher R, Fain O, et al. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood. 2008;112(4):999–1004.
  • Medeot M, Zaja F, Vianelli N, et al. Rituximab therapy in adult patients with relapsed or refractory immune thrombocytopenic purpura: long-term follow-up results. Eur J Haematol. 2008;81(3):165–169.
  • Zaja F, Vianelli N, Volpetti S, et al. Low-dose rituximab in adult patients with primary immune thrombocytopenia. Eur J Haematol. 2010;85(4):329–334.
  • Bussel JB, Lee CS, Seery C, et al. Rituximab and three dexamethasone cycles provide responses similar to splenectomy in women and those with immune thrombocytopenia of less than two years duration. Haematologica. 2014;99(7):1264–1271.
  • Marangon M, Vianelli N, and Palandri F, et al. Rituximab in immune thrombocytopenia: gender, age, and response as predictors of long-term response. Eur J Haematol. 2017;98(4):371–377 Accessed 22 October 2021.
  • Gracie C, Zaidi A, and Doobaree U, et al. Comparison of standard- and low-dose rituximab in primary immune thrombocytopenia (ITP): data from the UK ITP registry. Presented at European Hematology Association (EHA) Stockholm 2018. Available from: https://library.ehaweb.org/eha/2018/stockholm/214599/caitlin.gracie.comparison.of.standard-.and.low-dose.rituximab.in.primary.html?f=menu=6*ce_id=1346*ot_id=19068*media=3*marker=170 Accessed 22 October 2021.
  • Newland A, Lee EJ, McDonald V, et al. Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy. 2018;10(1):9–25.
  • Cooper N, Ghanima W. Immune thrombocytopenia. N Engl J Med. 2019;381(10):945–955.
  • Newland A, McDonald V. Fostamatinib: a review of its clinical efficacy and safety in the management of chronic adult immune thrombocytopenia. Immunotherapy. 2020;12(18):1325–1340.
  • 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. Am J Hematol. 2018;93(7): 921–930.
  • Bussel J, Arnold DM, Duliege AM, et al. Long‐term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program. Am J Hematol. 2019;94(5):546–553.
  • Boccia R, Cooper N, Ghanima W, et al. Fostamatinib is an effective second-line therapy in patients with immune thrombocytopenia. Br J Haematol. 2020;190(6):933–938.
  • Cooper N, Altomare I, Thomas MR, et al. Assessment of thrombotic risk during long-term treatment of immune thrombocytopenia with fostamatinib. Ther Adv Hematol. 2021;12:20406207211010875.
  • Gernsheimer TB, George JN, Aledort LM, et al. Evaluation of bleeding and thrombotic events during long-term use of romiplostim in patients with chronic immune thrombocytopenia (ITP). J Thromb Haemost. 2010;8(6):1372–1382.
  • Gernsheimer TB, Kuter DJ, Cines DB, et al. Analysis of mortality rates during romiplostim clinical studies of patients (Pts) with immune thrombocytopenia (ITP). Blood. 2010;116(21):3701.
  • Janssens A, Rodeghiero F, Anderson D, et al. Changes in bone marrow morphology in adults receiving romiplostim for the treatment of thrombocytopenia associated with primary immune thrombocytopenia. Ann Hematol. 2016;95(7):1077–1087.
  • 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. Br J Haematol. 2018;183(3):479–490.
  • Kuter DJ, Bussel JB, Newland A, et al. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013;161(3):411–423.
  • Altomere I, Markovtsov VV, Todd L, et al. Potential anti-thrombotic effect without accompanying hemorrhage with fostamatinib use in patients with immune thrombocytopenia. Blood. 2019;134(Suppl_1):4889.
  • 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. Haematologica. 2013;98(6):875–880.
  • Boyle S, White RH, Brunson A, et al. Splenectomy and the incidence of venous thromboembolism and sepsis in patients with immune thrombocytopenia. Blood. 2013;121(23):4782–4790.
  • Provan D, Newland AC. Current management of primary immune thrombocytopenia. Adv Ther. 2015;32(10):875–887.
  • Todd S, Sardo Infirri S, Miah H, et al. Splenectomy in immune thrombocytopenia: do changing treatment patterns for ITP affect outcome? data from the UK ITP registry. Br J Haematol. 2020;189(S1):11–12.
  • Sarpatwari A, Provan D, Erqou S, et al. Autologous 111 in-labelled platelet sequestration studies in patients with primary immune thrombocytopenia (ITP) prior to splenectomy: a report from the United Kingdom ITP registry. Br J Haematol. 2010;151(5):477–487.
  • Cuker A, and Cines DB. Evidence-based mini-review: is indium-labeled autologous platelet scanning predictive of response to splenectomy in patients with chronic immune thrombocytopenia? Hematology Am Soc Hematol Educ Program. 2010;2010:385–386.
  • Kazi S, Mckiddie F, Anderson J, et al. Autologous 111 in-labelled platelet scan as a predictor of splenectomy outcome in ITP. Br J Haematol. 2019;184(6):1043–1045.
  • Kuter D, Efraim M, Mayer J, et al. Phase I/II, open-label, ongoing study of PRN1008 (Rilzabrutinib), an oral Bruton tyrosine kinase inhibitor, in patients with heavily pretreated immune thrombocytopenia (ITP). Res Pract Thromb Haemost. 2020;4(Suppl 1):PB1318. Available from: https://abstracts.isth.org/abstract/phase-i-ii-open-label-ongoing-study-of-prn1008-rilzabrutinib-an-oral-bruton-tyrosine-kinase-inhibitor-in-patients-with-heavily-pretreated-immune-thrombocytopenia-itp/. Accessed August 21, 2020.
  • Robak T, Kaźmierczak M, Jarque I, et al. Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia. Blood Adv. 2020;4(17):4136–4146.
  • Newland AC, Sánchez-González B, Rejtő L, et al. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Am J Hematol. 2020;95(2):178–187.
  • Shao L, Wu Y, Zhou H, et al. Successful treatment with oseltamivir phosphate in a patient with chronic immune thrombocytopenia positive for anti-GPIb/IX autoantibody. Platelets. 2015;26(5):495–497.
  • Sun L, Liu X, Zhou H, et al. High-dose dexamethasone (HD-DXM) plus oseltamivir versus high-dose dexamethasone for treatment of adult primary immune thrombocytopenia: interim analysis of a prospective, multicenter, randomized, controlled trial. Blood. 2019;134(Suppl_1):900.
  • Nikitin PA, Rose EL, Byun TS, et al. C1s inhibition by BIVV009 (Sutimlimab) prevents complement-enhanced activation of autoimmune Human B Cells in vitro. J Immunol. 2019;202(4):1200–1209.
  • Broome CM, Röth A, Kuter DJ, et al. Long-term safety and efficacy of sutimlimab in patients with chronic immune thrombocytopenia. Blood. 2020;136(Suppl_1):14–15.
  • Beckman JD, Rollins-Raval MA, Raval JS, et al. Bortezomib for refractory immune-mediated thrombocytopenia purpura (ITP). Am J Ther. 2018;25(2):e270–e272.
  • Migdady Y, Ediriwickrema A, Jackson RP, et al. Successful treatment of thrombocytopenia with daratumumab after allogeneic transplant: a case report and literature review. Blood Adv. 2020;4(5):815–818.
  • Mahévas M, Azzaoui I, Crickx E, et al. Efficacy, safety and immunological profile of combining rituximab with belimumab for adults with persistent or chronic immune thrombocytopenia: results from a prospective phase 2b trial. Haematologica. 2020. DOI:https://doi.org/10.3324/haematol.2020.259481.
  • Zhu X, Wang Y, Jiang Q, et al. All- trans retinoic acid protects mesenchymal stem cells from immune thrombocytopenia by regulating the complement-interleukin-1β loop. Haematologica. 2019;104(8):1661–1675.
  • He Y, Xu -L-L, Feng F-E, et al. Mesenchymal stem cell deficiency influences megakaryocytopoiesis through the TNFAIP3/NF-κB/SMAD pathway in patients with immune thrombocytopenia. Br J Haematol. 2018;180(3):395–411.
  • Feng F-E, 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. Lancet Haematol. 2017;4(10):e487–e496.
  • Huang Q-S, Liu Y, Wang J-B, et al. Combination of ATRA and high-dose dexamethasone as first-line treatment in adult immune thrombocytopenia: a randomized, phase 2, open-label trial. Blood. 2019;134(Suppl_1):899.