Recombinant factor XIII and congenital factor XIII deficiency: an update from human and animal studies

References

• Soendergaard C, Kvist PH, Seidelin JB, Nielsen OH. Tissue- regenerating functions of coagulation factor XIII. J Thromb Haemost. 2013;11:806–816.
   Web of Science ®Google Scholar
• Richardson VP, Cordell P, Standeven KF, Carter AM. Substrates of factor XIII-A: roles in thrombosis and wound healing. Clin Sci. 2013;124:123–137.
   Web of Science ®Google Scholar
• Muszbek L, Yee VC, Hevessy Z. Blood coagulation factor XIII: structure and function. Thromb Res. 1999;94:271–305.
   PubMed Web of Science ®Google Scholar
• Ariens RAS, Lai TS, Weasel JW, Greenberg CS, Grant PJ. Role of FXIII in fibrin clot formation and effects of genetic polymorphisms. Blood. 2002;100:743–754.
   PubMed Web of Science ®Google Scholar
• Yee VC, Pedersen L, Le Trong I, Bishop PD, Stenkamp RE, Teller DC. Three dimensional structure of a transglutaminase: human blood coagulation factor XIII. Proc Natl Acad Sci U S A. 1994;91 : 7296–7300.
   Web of Science ®Google Scholar
• Mary A, Achyuthan KE, Greenberg CS. b-chains prevent the proteolytic inactivation of the a-chains of plasma factor XIII. Biochem Biophys Acta.1988;966:328–335.
   Google Scholar
• Bottenus RE, Ichinose A, Davie EW. Nucleotide sequence of the gene for the b subunit of human factor XIII. Biochemistry. 1990;29:11195–111209.
   PubMed Web of Science ®Google Scholar
• Board PG, Webb GC, McKee J, Ichinose A. Localization of the coagulation factor XIII A subunit gene (F13A) to chromosome bands 6p24–p25. Cytogenet Cell Genet. 1988;48:25–27.
   PubMedGoogle Scholar
• Ichinose A, Davie EW. Characterization of the gene for the a subunit of human factor XIII (plasma transglutaminase), a blood coagulation factor. Proc Natl Acad Sci U S A. 1988;85:5829–5833.
   PubMed Web of Science ®Google Scholar
• Webb GC, Coggan M, Ichinose A, Board PG. Localization of the coagulation factor XIII B subunit gene (F13B) to chromosome bands 1q31–32.1 and restriction fragment length polymorphism at the locus. Hum Genet. 1989;81:157–160.
   PubMed Web of Science ®Google Scholar
• Adany R, Kiss A, Muszbek L. Factor XIII: a marker of mono-, and megararyopoiesis. Br J Haematol. 1987;67:176–172.
   Web of Science ®Google Scholar
• Nurminskaya M, Kaartinen MT. Transglutaminases in mineralized tissues. Front Biosci. 2006;11:1591–1606.
   PubMed Web of Science ®Google Scholar
• Weisberg LJ, Shiu DT, Conkling PR, Shuman MA. Identification of normal human peripheral blood monocytes and liver as sites of synthesis of coagulation factor XIII a-chain. Blood. 1987;70: 579–582.
   PubMed Web of Science ®Google Scholar
• Poon MC, Russell JA, Lowe S, et al. Hemopoietic origin of factor XIII A subunits in platelets, monocytes, and plasma. Evidence from bone marrow transplantation studies. J Clin Invest. 1989;84:787–792.
   PubMed Web of Science ®Google Scholar
• Inbal A, Muszbek L, Lubetsky A, et al. Platelets but not monocytes contribute to the plasma levels of factor XIII subunit A in patients undergoing autologous peripheral blood stem cell transplantation. Blood Coagul Fibrinol 2004;15:249–253.
   Web of Science ®Google Scholar
• Souri M, Kaetsu H, Ichinose A. Sushi domains in the B subunit of factor XIII responsible for oligomer assembly. Biochemistry. 2008;47:8656–8664.
   PubMed Web of Science ®Google Scholar
• Karimi M, Bereczky Z, Cohan N, Muszbek L. Factor XIII deficiency. Semin ThrombHemost. 2009;35:426–438.
   PubMed Web of Science ®Google Scholar
• Lauer P, Metzner HJ, Zettimeissl G, et al. Targeted inactivation of the mouse locus encoding coagulation factor XIII-A: hemostatic abnormalities in mutant mice and characterization of the coagulation deficit. Thromb Haemost. 2002;88:967–974.
   Web of Science ®Google Scholar
• Nahrendorf M, Hu K, Frantz S, et al. Factor XIII deficiency causes cardiac rupture, impairs wound healing, and aggravates cardiac remodeling in mice with myocardial infarction. Circulation. 2006;113:1196–1202.
   PubMed Web of Science ®Google Scholar
• Souri M, Koseki-Kuno S, Takeda N, et al. Male-specific cardiac pathologies in mice lacking either the A or B subunit of factor XIII. Thromb Haemost. 2008;99:401–408.
   Web of Science ®Google Scholar
• Inbal A, Lubetsky A, Krapp T, et al. Impaired wound healing in factor XIII deficient mice. Thromb Haemost. 2005;94:432^37.
   Web of Science ®Google Scholar
• Kasahara K, Souri M, Kaneda M, Miki T, Yamamoto N, Ichinose A. Impaired clot retraction in factor XIII A subunit-deficient mice. Blood. 2010;115:1277–1279.
   Web of Science ®Google Scholar
• D’Argenio G, Grossman A, Cosenza V, Valle ND, Mazzacca G, Bishop PD. Recombinant factor XIII improves established experimental colitis in rats. Dig Dis Sci. 2000;45:987–997.
   PubMed Web of Science ®Google Scholar
• Hirahara K, Shinbo K, Takahashi M, Matsuishi T. Suppressive effect of human blood coagulation factor XIII on the vascular permeability induced by anti-guinea pig endothelial cell antiserum in guinea pigs. Thromb Res. 1993;71:139–148.
   Web of Science ®Google Scholar
• Dardik R, Solomon A, Loscalzo J, et al. Novel proangiogenic effect of factor XIII associated with suppression of thrombospondin 1 expression. Arterioscler Thromb Vasc Biol. 2003;23:1472–1477.
   PubMed Web of Science ®Google Scholar
• Dardik R, Leor J, Skutelsky E, et al. Evaluation of the pro-angiogenic effect of factor XIII in heterotopic mouse heart allografts and FXIII- deficient mice. Thromb Haemost. 2006;95:546–550.
   Web of Science ®Google Scholar
• Duckert F, Jung E, Shmerling DH. A hitherto undescribed congenital haemorrhagic diathesis probably due to fibrin stabilizing factor deficiency. Thromb Diath Haemorrh. 1960;5:179–186.
   PubMedGoogle Scholar
• Lak M, Peyvandi F, Ali Sharifian A, Karimi K, Mannucci PM. Pattern of symptoms in 93 Iranian patients with severe factor XIII deficiency. J Thromb Haemost. 2003;8:1852–1853.
   Google Scholar
• Koseki-Kuno S, Yamakawa M, Dickneite G, Ichinose A. Factor XIII-A subunit deficient mice developed severe uterine bleeding events and subsequent spontaneous miscarriages. Blood. 2003;102:4410–4412.
   Web of Science ®Google Scholar
• Inbal A, Muszbek L. Inherited coagulation factor deficiencies and pregnancy loss. Semin Thromb Hemost. 2003;29:171–174.
   PubMed Web of Science ®Google Scholar
• Ivaskevicius V, Seitz R, Kohler HP, et al; Study Group. International registry on factor XIII deficiency: a basis formed mostly on European data. Thromb Haemost. 2007;97:914–921.
   PubMed Web of Science ®Google Scholar
• Dardik R, Loscalzo J, Inbal A. Factor XIII and angiogenesis. J Thromb Haemost. 2006;4:19–25.
   PubMed Web of Science ®Google Scholar
• Muszbek L, Adany R, Mikkola H. Novel aspects of blood coagulation factor XIII. I. Structure, distribution, activation and function. Crit Rev Clin Lab Sci. 1996;33:357–421.
   PubMed Web of Science ®Google Scholar
• Grothaus-Pinke B, Gunzelmann S, Fauser AA, Kiehl MG. Factor XIII replacement in stem cell transplant (SCT) recipients with severe graft-versus-host disease of the bowel: report of an initial experience. Transplantation. 2001;72:1456–1458.
   PubMed Web of Science ®Google Scholar
• Dardik R, Krapp T, Rosenthal E, Loscalzo J, Inbal A. Effect of factor XIII (FXIII) on migration, proliferation and apoptosis of monocytes, smooth muscle cells and fibroblasts. Cell Physiol Biochem. 2007;19:113–120.
   Google Scholar
• Sarvary A, Szucs S, Balogh I, et al. Possible role of factor XIII subunit A in Fc gamma and complement receptor-mediated phagocytosis. Cell I mmunol. 2004;228:81–90.
   Web of Science ®Google Scholar
• Dardik R, Loscalzo J, Eskaraev R, Inbal A. Molecular mechanisms underlying the proangiogenic effect of factor XIII. Arterioscler Thromb Vasc Biol. 2005;25:526–532.
   Web of Science ®Google Scholar
• Peyvandi F, Duga S, Akhavan S, Mannucci PM. Rare coagulation deficiencies. Haemophilia. 2002;8:308–321.
   PubMed Web of Science ®Google Scholar
• Biswas A, Ivaskevicius V, Seitz R, Thomas A, Oldenburg J. An update of the mutation profile of factor 13 A and B genes. Blood Rev. 2011;25:193–204.
   PubMed Web of Science ®Google Scholar
• Saito M, Asakura H, Yoshida T, Ito K, Okafuji K, Matsuda T. A familial factor XIII subunit B deficiency. Br J Haematol. 1990;74:290–294.
   PubMed Web of Science ®Google Scholar
• Fickenscher K, Aab A, Stuber W. A Photometric assay for blood coagulation factor XIII. Thromb Haemost. 1991;65:535–540.
   PubMed Web of Science ®Google Scholar
• Ajzner E, Muszbek L. Kinetic spectrophotometric factor XIII activity assays: the subtraction of plasma blank is not omissible [corrected]. J Thromb Haemost. 2004;2:2075–2077.
   Google Scholar
• Katona E, Haramura G, Karpati L, Fachet J, Muszbek L. A simple, quick one-step ELISA assay for the determination of complex plasma factor XIII (A2B2). Thromb Haemost. 2000;83:268–273.
   PubMed Web of Science ®Google Scholar
• Gootenberg JE. Factor concentrates for the treatment of factor XIII deficiency. Curr Opin Hematol. 1998;5:372–375.
   Google Scholar
• Anwar R, Miloszewski KJ. Factor XIII deficiency. Br J Haematol. 1999;107:468–484.
   Web of Science ®Google Scholar
• Lusher J, Pipe SW, Alexander S, Nugent D. Prophylactic therapy with Fibrogammin P is associated with a decreased incidence of bleeding episodes: a retrospective study. Haemophilia. 2010;16:316–321.
   PubMed Web of Science ®Google Scholar
• Yoshida S, Fukue H, Sugimura D, et al. Efficiency of intermittent prophylactic factor XIII substitution in congenital factor XIIIA subunit deficiency. Journal of the Japan Society of Blood Transfusions. 1996;42:173–177.
   Google Scholar
• Dreyfus M, Barrois D, Borg JY, et al. Groupe d’Etudes Francophone du FXIII. Successful long-term replacement therapy with FXIII concentrate (Fibrogammin® P) for severe congenital factor XIII deficiency: a prospective multicentre study. J Thromb Haemost. 2011 ;9: 1264–1266.
   Google Scholar
• Lahav J, Tvito A, Bagoly Z, Dardik R, Inbal A. Factor XIII improves platelet adhesion to fibrinogen by protein disulfide isomerase-mediated activity. Thromb Res. 2013;131:338–341.
   Web of Science ®Google Scholar
• Reynolds TC, Butine MD, Visich JE, et al. Safety, pharmacokinetics, and immunogenicity of single dose rFXIII administration to healthy volunteers. J Thromb Haemost. 2005;3:922–928.
   PubMed Web of Science ®Google Scholar
• Visich JE, Zuckerman LA, Butine MD, et al. Safety and pharmacokinetics of recombinant factor XIII in healthy volunteers: a randomized, placebo-controlled, double-blind, multi-dose study. Thromb Haemost. 2005;94:802–807.
   PubMed Web of Science ®Google Scholar
• Levy JH, Gill R, Nussmeier NA, et al. Repletion of factor XIII following cardiopulmonary bypass using a recombinant A-subunit homodimer. A preliminary report. Thromb Haemost. 2009;102:765–771.
   Web of Science ®Google Scholar
• Lovejoy AE, Reynolds TC, Visich JE, et al. Safety and pharmacokinetics of recombinant factor XIII-A2 administration in patients with congenital factor XIII deficiency. Blood. 2006;108:57–62.
   PubMed Web of Science ®Google Scholar
• Inbal A, Oldenburg J, Carcao M, Rosholm A, Tehranchi R, Nugent D. Recombinant factor XIII: a safe and novel treatment for congenital factor XIII deficiency. Blood. 2012;119:5111–5117.
   PubMed Web of Science ®Google Scholar
• Brackmann HH, Egbring R, Ferster A, et al. Pharmacokinetics and tolerability of factor XIII concentrates prepared from human placenta or plasma: a crossover randomized study. Thromb Haemost. 1995;74:622–625.
   Web of Science ®Google Scholar