Skip to main content
Top
Published in: Intensive Care Medicine 4/2015

01-04-2015 | Understanding the Disease

Understanding organ dysfunction in thrombotic thrombocytopenic purpura

Author: Pier Mannuccio Mannucci

Published in: Intensive Care Medicine | Issue 4/2015

Login to get access

Excerpt

Thrombotic thrombocytopenic purpura (TTP) is the epitome of thrombotic microangiopathies (TMA), i.e., rare multisystem diseases characterized by disseminated microvascular thrombosis leading to ischemic damage and dysfunction in multiple organs [1]. The main clinical syndromes presenting as TMA are, beside TTP, the hemolytic uremic syndrome, disseminated intravascular coagulation, pre-eclampsia, the HELLP syndrome, the catastrophic antiphospholipid antibody syndrome, disseminated malignancy, and heparin-induced thrombocytopenia. …
Literature
1.
2.
go back to reference Furlan M, Robles R, Lammle B (1996) Partial purification and characterization of a protease from human plasma cleaving von Willebrand factor to fragments produced by in vivo proteolysis. Blood 87:4223–4234PubMed Furlan M, Robles R, Lammle B (1996) Partial purification and characterization of a protease from human plasma cleaving von Willebrand factor to fragments produced by in vivo proteolysis. Blood 87:4223–4234PubMed
3.
go back to reference Tsai HM (1996) Physiologic cleavage of von Willebrand factor by a plasma protease is dependent on its conformation and requires calcium ion. Blood 87:4235–4244PubMed Tsai HM (1996) Physiologic cleavage of von Willebrand factor by a plasma protease is dependent on its conformation and requires calcium ion. Blood 87:4235–4244PubMed
4.
go back to reference Furlan M, Robles R, Solenthaler M, Wassmer M, Sandoz P, Lammle B (1997) Deficient activity of von Willebrand factor-cleaving protease in chronic relapsing thrombotic thrombocytopenic purpura. Blood 89:3097–3103PubMed Furlan M, Robles R, Solenthaler M, Wassmer M, Sandoz P, Lammle B (1997) Deficient activity of von Willebrand factor-cleaving protease in chronic relapsing thrombotic thrombocytopenic purpura. Blood 89:3097–3103PubMed
5.
go back to reference Zheng X, Chung D, Takayama TK, Majerus EM, Sadler JE, Fujikawa K (2001) Structure of von Willebrand factor-cleaving protease (ADAMTS13), a metalloprotease involved in thrombotic thrombocytopenic purpura. J Biol Chem 276:41059–41063CrossRefPubMed Zheng X, Chung D, Takayama TK, Majerus EM, Sadler JE, Fujikawa K (2001) Structure of von Willebrand factor-cleaving protease (ADAMTS13), a metalloprotease involved in thrombotic thrombocytopenic purpura. J Biol Chem 276:41059–41063CrossRefPubMed
6.
go back to reference Moake JL, Rudy CK, Troll JH, Weinstein MJ, Colannino NM, Azocar J, Seder RH, Hong SL, Deykin D (1982) Unusually large plasma factor VIII:von Willebrand factor multimers in chronic relapsing thrombotic thrombocytopenic purpura. N Engl J Med 307:1432–1435CrossRefPubMed Moake JL, Rudy CK, Troll JH, Weinstein MJ, Colannino NM, Azocar J, Seder RH, Hong SL, Deykin D (1982) Unusually large plasma factor VIII:von Willebrand factor multimers in chronic relapsing thrombotic thrombocytopenic purpura. N Engl J Med 307:1432–1435CrossRefPubMed
7.
go back to reference Levy GG, Nichols WC, Lian EC, Foroud T, McClintick JN, McGee BM, Yang AY, Siemieniak DR, Stark KR, Gruppo R, Sarode R, Shurin SB, Chandrasekaran V, Stabler SP, Sabio H, Bouhassira EE, Upshaw JD Jr, Ginsburg D, Tsai HM (2001) Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature 413:488–494CrossRefPubMed Levy GG, Nichols WC, Lian EC, Foroud T, McClintick JN, McGee BM, Yang AY, Siemieniak DR, Stark KR, Gruppo R, Sarode R, Shurin SB, Chandrasekaran V, Stabler SP, Sabio H, Bouhassira EE, Upshaw JD Jr, Ginsburg D, Tsai HM (2001) Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature 413:488–494CrossRefPubMed
8.
go back to reference Tsai HM, Lian EC (1998) Antibodies to von Willebrand factor-cleaving protease in acute thrombotic thrombocytopenic purpura. N Engl J Med 339:1585–1594CrossRefPubMedCentralPubMed Tsai HM, Lian EC (1998) Antibodies to von Willebrand factor-cleaving protease in acute thrombotic thrombocytopenic purpura. N Engl J Med 339:1585–1594CrossRefPubMedCentralPubMed
9.
go back to reference Moatti-Cohen M, Garrec C, Wolf M, Boisseau P, Galicier L, Azoulay E, Stepanian A, Delmas Y, Rondeau E, Bezieau S, Coppo P, Veyradier A (2012) Unexpected frequency of Upshaw-Schulman syndrome in pregnancy-onset thrombotic thrombocytopenic purpura. Blood 119:5888–5897CrossRefPubMed Moatti-Cohen M, Garrec C, Wolf M, Boisseau P, Galicier L, Azoulay E, Stepanian A, Delmas Y, Rondeau E, Bezieau S, Coppo P, Veyradier A (2012) Unexpected frequency of Upshaw-Schulman syndrome in pregnancy-onset thrombotic thrombocytopenic purpura. Blood 119:5888–5897CrossRefPubMed
10.
go back to reference Scully M, Thomas M, Underwood M, Watson H, Langley K, Camilleri RS, Clark A, Creagh D, Rayment R, Mcdonald V, Roy A, Evans G, McGuckin S, Ni AF, Maclean R, Lester W, Nash M, Scott R, Brien O (2014) Thrombotic thrombocytopenic purpura and pregnancy: presentation, management, and subsequent pregnancy outcomes. Blood 124:211–219CrossRefPubMed Scully M, Thomas M, Underwood M, Watson H, Langley K, Camilleri RS, Clark A, Creagh D, Rayment R, Mcdonald V, Roy A, Evans G, McGuckin S, Ni AF, Maclean R, Lester W, Nash M, Scott R, Brien O (2014) Thrombotic thrombocytopenic purpura and pregnancy: presentation, management, and subsequent pregnancy outcomes. Blood 124:211–219CrossRefPubMed
11.
go back to reference Lotta LA, Wu HM, Mackie IJ, Noris M, Veyradier A, Scully MA, Remuzzi G, Coppo P, Liesner R, Donadelli R, Loirat C, Gibbs RA, Horne A, Yang S, Garagiola I, Musallam KM, Peyvandi F (2012) Residual plasmatic activity of ADAMTS13 is correlated with phenotype severity in congenital thrombotic thrombocytopenic purpura. Blood 120:440–448CrossRefPubMedCentralPubMed Lotta LA, Wu HM, Mackie IJ, Noris M, Veyradier A, Scully MA, Remuzzi G, Coppo P, Liesner R, Donadelli R, Loirat C, Gibbs RA, Horne A, Yang S, Garagiola I, Musallam KM, Peyvandi F (2012) Residual plasmatic activity of ADAMTS13 is correlated with phenotype severity in congenital thrombotic thrombocytopenic purpura. Blood 120:440–448CrossRefPubMedCentralPubMed
12.
go back to reference Rieger M, Mannucci PM, Kremer Hovinga JA, Herzog A, Gerstenbauer G, Konetschny C, Zimmermann K, Scharrer I, Peyvandi F, Galbusera M, Remuzzi G, Bohm M, Plaimauer B, Lammle B, Scheiflinger F (2005) ADAMTS13 autoantibodies in patients with thrombotic microangiopathies and other immunomediated diseases. Blood 106:1262–1267CrossRefPubMed Rieger M, Mannucci PM, Kremer Hovinga JA, Herzog A, Gerstenbauer G, Konetschny C, Zimmermann K, Scharrer I, Peyvandi F, Galbusera M, Remuzzi G, Bohm M, Plaimauer B, Lammle B, Scheiflinger F (2005) ADAMTS13 autoantibodies in patients with thrombotic microangiopathies and other immunomediated diseases. Blood 106:1262–1267CrossRefPubMed
13.
go back to reference Rock GA, Shumak KH, Buskard NA, Blanchette VS, Kelton JG, Nair RC, Spasoff RA (1991) Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. Canadian Apheresis Study Group. N Engl J Med 325:393–397CrossRefPubMed Rock GA, Shumak KH, Buskard NA, Blanchette VS, Kelton JG, Nair RC, Spasoff RA (1991) Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. Canadian Apheresis Study Group. N Engl J Med 325:393–397CrossRefPubMed
14.
go back to reference Cataland SR, Wu HM (2014) How I treat: the clinical differentiation and initial treatment of adult patients with atypical hemolytic uremic syndrome. Blood 123:2478–2484CrossRefPubMed Cataland SR, Wu HM (2014) How I treat: the clinical differentiation and initial treatment of adult patients with atypical hemolytic uremic syndrome. Blood 123:2478–2484CrossRefPubMed
15.
go back to reference Scully M, Gattens M, Khair K, Liesner R (2006) The use of intermediate purity factor VIII concentrate BPL 8Y as prophylaxis and treatment in congenital thrombotic thrombocytopenic purpura. Br J Haematol 135:101–104CrossRefPubMed Scully M, Gattens M, Khair K, Liesner R (2006) The use of intermediate purity factor VIII concentrate BPL 8Y as prophylaxis and treatment in congenital thrombotic thrombocytopenic purpura. Br J Haematol 135:101–104CrossRefPubMed
16.
go back to reference Hie M, Gay J, Galicier L, Provot F, Presne C, Poullin P, Bonmarchand G, Wynckel A, Benhamou Y, Vanhille P, Servais A, Bordessoule D, Coindre JP, Hamidou M, Vernant JP, Veyradier A, Coppo P (2014) Preemptive rituximab infusions after remission efficiently prevent relapses in acquired thrombotic thrombocytopenic purpura. Blood 124:204–210CrossRefPubMed Hie M, Gay J, Galicier L, Provot F, Presne C, Poullin P, Bonmarchand G, Wynckel A, Benhamou Y, Vanhille P, Servais A, Bordessoule D, Coindre JP, Hamidou M, Vernant JP, Veyradier A, Coppo P (2014) Preemptive rituximab infusions after remission efficiently prevent relapses in acquired thrombotic thrombocytopenic purpura. Blood 124:204–210CrossRefPubMed
17.
go back to reference Peyvandi F for the TITAN Investigators, Duby C (2014) Caplacizumab, anti-VWF nanobody potentially changing the treatment paradigm in thrombotic thrombocytopenic purpura: results of the TITAN trial. In: Abstracts of the 56th ASH annual meeting and exposition, San Francisco, 6–9 December 2014, abstract no. 229 Peyvandi F for the TITAN Investigators, Duby C (2014) Caplacizumab, anti-VWF nanobody potentially changing the treatment paradigm in thrombotic thrombocytopenic purpura: results of the TITAN trial. In: Abstracts of the 56th ASH annual meeting and exposition, San Francisco, 6–9 December 2014, abstract no. 229
Metadata
Title
Understanding organ dysfunction in thrombotic thrombocytopenic purpura
Author
Pier Mannuccio Mannucci
Publication date
01-04-2015
Publisher
Springer Berlin Heidelberg
Published in
Intensive Care Medicine / Issue 4/2015
Print ISSN: 0342-4642
Electronic ISSN: 1432-1238
DOI
https://doi.org/10.1007/s00134-014-3630-z

Other articles of this Issue 4/2015

Intensive Care Medicine 4/2015 Go to the issue