References
- Joly BS, Coppo P, Veyradier A. Thrombotic thrombocytopenic purpura. Blood. 2017 25;129(21):2836–2846.
- Mariotte E, Azoulay E, Galicier L, et al. Epidemiology and pathophysiology of adulthood-onset thrombotic microangiopathy with severe ADAMTS13 deficiency (thrombotic thrombocytopenic purpura): a cross-sectional analysis of the French national registry for thrombotic microangiopathy. Lancet Haematol. 2016 Mai;3(5):e237–45.
- Peyvandi F, Scully M, Kremer Hovinga JA, et al. Caplacizumab for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2016 févr 11;374(6):511–522.
- Coppo P, Veyradier A. Thrombotic microangiopathies: towards a pathophysiology-based classification. Cardiovasc Hematol Disord Drug Targets. 2009 mars;9(1):36–50.
- Sadler JE. Pathophysiology of thrombotic thrombocytopenic purpura. Blood. 2017 07;130(10):1181–1188.
- Sarig G. ADAMTS-13 in the diagnosis and management of thrombotic microangiopathies. Rambam Maimonides Med J. 2014 oct;5(4):e0026.
- Nichols L, Berg A, Rollins-Raval MA, et al. Cardiac injury is a common postmortem finding in thrombotic thrombocytopenic purpura patients: is empiric cardiac monitoring and protection needed? Ther Apher Dial. 2015 févr;19(1):87–92.
- Benhamou Y, Boelle P-Y, Baudin B, et al. Cardiac troponin-I on diagnosis predicts early death and refractoriness in acquired thrombotic thrombocytopenic purpura. Experience of the French thrombotic microangiopathies reference center. J Thromb Haemost. 2015 févr;13(2):293–302.
- Falter T, Schmitt V, Herold S, et al. Depression and cognitive deficits as long-term consequences of thrombotic thrombocytopenic purpura. Transfusion. 2017 mai;57(5):1152–1162.
- Soucemarianadin M, Benhamou Y, Delmas Y, et al. Twice-daily therapeutical plasma exchange-based salvage therapy in severe autoimmune thrombotic thrombocytopenic purpura: the French TMA Reference Center experience. Eur J Haematol. 2016 août;97(2):183–191.
- Coppo P, Cuker A, George JN. Thrombotic thrombocytopenic purpura: toward targeted therapy and precision medicine. Res Pract Thromb Haemost. 2019 janv;3(1):26–37.
- George JN, Al-Nouri ZL. Diagnostic and therapeutic challenges in the thrombotic thrombocytopenic purpura and hemolytic uremic syndromes. Hematology Am Soc Hematol Educ Program. 2012;2012:6049.
- Coppo P, Veyradier A. Current management and therapeutical perspectives in thrombotic thrombocytopenic purpura. Presse Med. 2012 mars;41(3 Pt 2):e163–176.
- Peyvandi F, Scully M, Kremer Hovinga JA, et al. Caplacizumab reduces the frequency of major thromboembolic events, exacerbations and death in patients with acquired thrombotic thrombocytopenic purpura. J Thromb Haemost. 2017;15(7):1448–1452.
- Kremer Hovinga JA, Vesely SK, Terrell DR, et al. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2010 févr 25;115(8):1500–11;quiz 1662.
- Jestin M, Benhamou Y, Schelpe A-S, et al. Preemptive rituximab prevents long-term relapses in immune-mediated thrombotic thrombocytopenic purpura. Blood. 2018 nov 15;132(20):2143–2153.
- Scully M, McDonald V, Cavenagh J, et al. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011 août 18;118(7):1746–1753.
- Westwood J-P, Webster H, McGuckin S, et al. Rituximab for thrombotic thrombocytopenic purpura: benefit of early administration during acute episodes and use of prophylaxis to prevent relapse. J Thromb Haemost. 2013 mars;11(3):481–490.
- Scully M, Cataland SR, Peyvandi F, et al. Caplacizumab treatment for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2019 24;380(4):335–346.
- Falter T, Herold S, Weyer-Elberich V, et al. Relapse rate in survivors of acute autoimmune thrombotic thrombocytopenic purpura treated with or without rituximab. Thromb Haemost. 2018 oct;118(10):1743‑51.
- George JN. How I treat patients with thrombotic thrombocytopenic purpura: 2010. Blood. 2010 nov 18;116(20):4060–4069.
- Coppo P. French reference center for thrombotic microangiopathies. Treatment of autoimmune thrombotic thrombocytopenic purpura in the more severe forms. Transfus Apher Sci. 2017 févr;56(1):52–56.
- Rock GA, Shumak KH, Buskard NA, et al. Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. Canadian apheresis study group. N Engl J Med. 1991 Aug;325(6):393–397.
- Froissart A, Buffet M, Veyradier A, et al. Efficacy and safety of first-line rituximab in severe, acquired thrombotic thrombocytopenic purpura with a suboptimal response to plasma exchange of the French thrombotic microangiopathies reference center. Crit Care Med. 2012 janv;40(1):104–111.
- Froissart A, Veyradier A, Hié M, et al. French reference center for thrombotic microangiopathies. Rituximab in autoimmune thrombotic thrombocytopenic purpura: A success story. Eur J Intern Med. 2015 nov;26(9):659–665.
- Benhamou Y, Paintaud G, Azoulay E, et al. Efficacy of a rituximab regimen based on B cell depletion in thrombotic thrombocytopenic purpura with suboptimal response to standard treatment: results of a phase II, multicenter noncomparative study. Am J Hematol. 2016;91(12):1246–1251.
- Beloncle F, Buffet M, Coindre J-P, et al. Splenectomy and/or cyclophosphamide as salvage therapies in thrombotic thrombocytopenic purpura: the French TMA reference center experience. Transfusion. 2012 nov;52(11):2436–2444.
- Coppo P. French reference center for thrombotic microangiopathies. Management of thrombotic thrombocytopenic purpura. Transfus Clin Biol. 2017 sept;24(3):148–153.
- Cataland SR, Kourlas PJ, Yang S, et al. Cyclosporine or steroids as an adjunct to plasma exchange in the treatment of immune-mediated thrombotic thrombocytopenic purpura. Blood Adv. 2017 oct 24;1(23):2075–2082.
- Tersteeg C, Roodt J, van Rensburg WJ, et al. N-acetylcysteine in preclinical mouse and baboon models of thrombotic thrombocytopenic purpura. Blood. 2017 23;129(8):1030–1038.
- Pandey MR, Vachhani P, Ontiveros EP. Remission of severe, relapsed, and refractory TTP after multiple cycles of bortezomib. Case Rep Hematol. 2017;2017:9681832.
- Eskazan AE. Bortezomib therapy in patients with relapsed/refractory acquired thrombotic thrombocytopenic purpura. Ann Hematol. 2016 oct;95(11):1751–1756.
- Scully M, Knöbl P, Kentouche K, et al. Recombinant ADAMTS-13: first-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura. Blood. 2017 09;130(19):2055–2063.
- Plaimauer B, Schiviz A, Kaufmann S, et al. Neutralization of inhibitory antibodies and restoration of therapeutic ADAMTS13 activity levels in inhibitor-treated rats through defined doses of recombinant ADAMTS13. J Thromb Haemost. 2015 sept 4;13(11):2053–2062.
- Tersteeg C, Schiviz A, de Meyer SF, et al. Potential for recombinant ADAMTS13 as an effective therapy for acquired thrombotic thrombocytopenic purpura. Arterioscler Thromb Vasc Biol. 2015 sept 3;35(11):2336–2342.
- Duggan S. Caplacizumab: first global approval. Drugs. 2018 oct;78(15):1639–1642.
- Callewaert F, Roodt J, Ulrichts H, et al. Evaluation of efficacy and safety of the anti-VWF Nanobody ALX-0681 in a preclinical baboon model of acquired thrombotic thrombocytopenic purpura. Blood. 2012 oct 25;120(17):3603–3610.
- Masias C, Cataland SR. Novel therapies in thrombotic thrombocytopenic purpura. Res Pract Thromb Haemost. 2018 janv;2(1):19–26.
- Cablivi. European medicines agency commission. 2018. [cited 2019 Aug 1]. Available from: https://www.ema.europa.eu
- Kremer Hovinga J Safety of caplacizumab for the treatment of patients with acquired thrombotic thrombocytopenic purpura - results normalized to time of exposure in a double-blind, placebo-controlled, phase 3 hercules study. [cited 2019 Aug 1]. Available from: https://ash.confex.com/ash/2018/webprogram/Paper112177.html
- Benhamou Y, Assié C, Boelle P-Y, et al. Development and validation of a predictive model for death in acquired severe ADAMTS13 deficiency-associated idiopathic thrombotic thrombocytopenic purpura: the French TMA reference center experience. Haematologica. 2012 août;97(8):1181‑6.
- Coppo P, Schwarzinger M, Buffet M, et al. Predictive features of severe acquired ADAMTS13 deficiency in idiopathic thrombotic microangiopathies: the French TMA reference center experience. PLoS One. 2010 avril;5(4):e10208.
- Bendapudi PK, Hurwitz S, Fry A, et al. Derivation and external validation of the PLASMIC score for rapid assessment of adults with thrombotic microangiopathies: a cohort study. Lancet Haematol. 2017 avril;4(4):e157–e164.
- Olson SR, Samuelson-Bannow BT. Caplacizumab for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2019 mai;380(18):e32.
- Aymé G, Adam F, Legendre P, et al. A novel single-domain antibody against von willebrand factor A1 domain resolves leukocyte recruitment and vascular leakage during inflammation-brief report. Arterioscler Thromb Vasc Biol. 2017 sept;37(9):1736–1740.