TNT003, an inhibitor of the serine protease C1s, prevents complement activation induced by cold agglutinins

Blood

Volumn 123, Issue 26, 2014, Pages 4015-4022

  Shi, Ju ^a   Rose, Eileen L ^a   Singh, Andrew ^a   Hussain, Sami ^a   Stagliano, Nancy E ^a   Parry, Graham C ^a   Panicker, Sandip ^a  

^a True North Therapeutics Inc ^*  (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANAPHYLATOXIN; COLD AGGLUTININ; COMPLEMENT COMPONENT C1; MONOCLONAL ANTIBODY; OPSONIN; SERINE PROTEINASE INHIBITOR; TNT 003; UNCLASSIFIED DRUG;

ADULT; AGED; ARTICLE; AUTOIMMUNE HEMOLYTIC ANEMIA; CLINICAL ARTICLE; COLD AGGLUTININ DISEASE; COMPLEMENT ACTIVATION; COMPLEMENT DEPOSITION; CONTROLLED STUDY; DRUG TARGETING; ENZYME ACTIVITY; ERYTHROCYTE; HEMOLYSIS; HUMAN; HUMAN CELL; LOW TEMPERATURE; MOUSE; NONHUMAN; PHAGOCYTOSIS; PRIORITY JOURNAL;

AGED; ANAPHYLATOXINS; ANEMIA, HEMOLYTIC, AUTOIMMUNE; ANIMALS; ANTIBODIES, MONOCLONAL, MURINE-DERIVED; CELL LINE, TUMOR; COMPLEMENT ACTIVATION; COMPLEMENT C1S; FEMALE; HEMOLYSIS; HUMANS; MALE; MICE; MIDDLE AGED; SERINE PROTEASES; SERINE PROTEINASE INHIBITORS;

EID: 84903640881     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2014-02-556027     Document Type: Article

References (40)

1. Ricklin D, Lambris JD. Complement in immune and inflammatory disorders: pathophysiological mechanisms. J Immunol. 2013;190(8):3831-3838.
2. Chen M, Daha MR, Kallenberg CG. The complement system in systemic autoimmune disease. J Autoimmun. 2010;34(3):J276-J286.
3. Markiewski MM, Lambris JD. The role of complement in inflammatory diseases from behind the scenes into the spotlight. Am J Pathol. 2007;171(3):715-727. (Pubitemid 47440971)
4. Ricklin D, Lambris JD. Complement in immune and inflammatory disorders: therapeutic interventions. J Immunol. 2013;190(8):3839-3847.
5. Berentsen S, Ulvestad E, Langholm R, et al. Primary chronic cold agglutinin disease: a population based clinical study of 86 patients. Haematologica. 2006;91(4):460-466.
6. Berentsen S. How I manage cold agglutinin disease. Br J Haematol. 2011;153(3):309-317.
7. Swiecicki PL, Hegerova LT, Gertz MA. Cold agglutinin disease. Blood. 2013;122(7):1114-1121.
8. Ulvestad E, Berentsen S, Bø K, Shammas FV. Clinical immunology of chronic cold agglutinin disease. Eur J Haematol. 1999;63(4):259-266. (Pubitemid 29470281)
9. Jaffe CJ, Atkinson JP, Frank MM. The role of complement in the clearance of cold agglutinin-sensitized erythrocytes in man. J Clin Invest. 1976;58(4):942-949.
10. Frank MM, Atkinson JP, Gadek J. Cold agglutinins and cold-agglutinin disease. Annu Rev Med. 1977;28:291-298.
11. Rosse WF, Adams JP. The variability of hemolysis in the cold agglutinin syndrome. Blood. 1980;56(3):409-416. (Pubitemid 11251020)
12. Kirschfink M, Fritze H, Roelcke D. Complement activation by cold agglutinins. Vox Sang. 1992;63(3):220-226.
13. Atkinson JP, Frank MM. Studies on the in vivo effects of antibody. Interaction of IgM antibody and complement in the immune clearance and destruction of erythrocytes in man. J Clin Invest. 1974;54(2):339-348.
14. Kirschfink M, Knoblauch K, Roelcke D. Activation of complement by cold agglutinins. Infusionsther Transfusionsmed. 1994;21(6):405-409.
15. Röth A, Hüttmann A, Rother RP, Dührsen U, Philipp T. Long-term efficacy of the complement inhibitor eculizumab in cold agglutinin disease. Blood. 2009;113(16):3885-3886.
16. Ulvestad E, Berentsen S, Mollnes TE. Acute phase haemolysis in chronic cold agglutinin disease. Scand J Immunol. 2001;54(1-2):239-242. (Pubitemid 32592351)
17. Berentsen S, Beiske K, Tjønnfjord GE. Primary chronic cold agglutinin disease: an update on pathogenesis, clinical features and therapy. Hematology. 2007;12(5):361-370. (Pubitemid 47487842)
18. Salama A, Göttsche B, Vaidya V, Santoso S, Mueller-Eckhardt C. Complement-independent lysis of human red blood cells by cold hemagglutinins. Vox Sang. 1988;55(1):21-25.
19. Michel M. Classification and therapeutic approaches in autoimmune hemolytic anemia: an update. Expert Rev Hematol. 2011;4(6):607-618.
20. Stone MJ. Heating up cold agglutinins. Blood. 2010;116(17):3119-3120.
21. Stone MJ, Merlini G, Pascual V. Autoantibody activity in Waldenstrom's macroglobulinemia. Clin Lymphoma. 2005;5(4):225-229.
22. Kumar V, Ali SR, Konrad S, et al. Cell-derived anaphylatoxins as key mediators of antibodydependent type II autoimmunity in mice. J Clin Invest. 2006;116(2):512-520. (Pubitemid 43228369)
23. Mollnes TE, Brekke OL, Fung M, et al. Essential role of the C5a receptor in E coli-induced oxidative burst and phagocytosis revealed by a novel lepirudin-based human whole blood model of inflammation. Blood. 2002;100(5):1869-1877. (Pubitemid 34925168)
24. Brekke OL, Christiansen D, Fure H, Fung M, Mollnes TE. The role of complement C3 opsonization, C5a receptor, and CD14 in E. coli-induced up-regulation of granulocyte and monocyte CD11b/CD18 (CR3), phagocytosis, and oxidative burst in human whole blood. J Leukoc Biol. 2007;81(6):1404-1413. (Pubitemid 46827963)
25. Lindorfer MA, Pawluczkowycz AW, Peek EM, Hickman K, Taylor RP, Parker CJ. A novel approach to preventing the hemolysis of paroxysmal nocturnal hemoglobinuria: both complement-mediated cytolysis and C3 deposition are blocked by a monoclonal antibody specific for the alternative pathway of complement. Blood. 2010;115(11):2283-2291.
26. Berentsen S, Ulvestad E, Gjertsen BT, et al. Rituximab for primary chronic cold agglutinin disease: a prospective study of 37 courses of therapy in 27 patients. Blood. 2004;103(8):2925-2928. (Pubitemid 38451661)
27. Engelhardt M, Jakob A, Ruter B, Trepel M, Hirsch F, Lübbert M. Severe cold hemagglutinin disease (CHD) successfully treated with rituximab. Blood. 2002;100(5):1922-1923. (Pubitemid 34925177)
28. Schöllkopf C, Kjeldsen L, Bjerrum OW, et al. Rituximab in chronic cold agglutinin disease: a prospective study of 20 patients. Leuk Lymphoma. 2006;47(2):253-260. (Pubitemid 43084563)
29. Berentsen S, Randen U, Vågan AM, et al. High response rate and durable remissions following fludarabine and rituximab combination therapy for chronic cold agglutinin disease. Blood. 2010;116(17):3180-3184.
30. Berentsen S, Tjønnfjord GE. Diagnosis and treatment of cold agglutinin mediated autoimmune hemolytic anemia. Blood Rev. 2012;26(3):107-115.
31. Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111(4):1840-1847. (Pubitemid 351451491)
32. Dmytrijuk A, Robie-Suh K, Cohen MH, Rieves D, Weiss K, Pazdur R. FDA report: eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Oncologist. 2008;13(9):993-1000.
33. Keating GM. Eculizumab: a review of its use in atypical haemolytic uraemic syndrome. Drugs. 2013;73(18):2053-2066.
34. Legendre CM, Licht C, Muus P, et al. Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med. 2013;368(23):2169-2181.
35. Farkas H, Varga L. Human Plasma-Derived, Nanofiltered, C1-Inhibitor Concentrate (Cinryze®), a Novel Therapeutic Alternative for the Management of Hereditary Angioedema Resulting from C1-Inhibitor Deficiency. Biol Ther. 2012;2:2.
36. Craig TJ, Bewtra AK, Bahna SL, et al. C1 esterase inhibitor concentrate in 1085 Hereditary Angioedema attacks - final results of the I.M.P.A. C.T.2 study. Allergy. 2011;66(12):1604-1611.
37. Frank MM. Recombinant and plasma-purified human c1 inhibitor for the treatment of hereditary angioedema. World Allergy Organ J. 2010; 3(9 Suppl):S29-S33.
38. Wouters D, Stephan F, Strengers P, et al. C1-esterase inhibitor concentrate rescues erythrocytes from complement-mediated destruction in autoimmune hemolytic anemia. Blood. 2013;121(7):1242-1244.
39. Risitano AM, Notaro R, Marando L, et al. Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009;113(17):4094-4100.
40. Risitano AM, Perna F, Selleri C. Achievements and limitations of complement inhibition by eculizumab in paroxysmal nocturnal hemoglobinuria: the role of complement component 3. Mini Rev Med Chem. 2011;11(6):528-535.