How targeted therapy disrupts the treatment paradigm for acquired TTP: the risks, benefits, and unknowns

被引:37
作者
Mazepa, Marshall A. [1 ]
Masias, Camila [2 ]
Chaturvedi, Shruti [3 ]
机构
[1] Univ Minnesota, Dept Med, Box 736 UMHC, Minneapolis, MN 55455 USA
[2] Ohio State Univ, Dept Med, Div Hematol, Columbus, OH 43210 USA
[3] Johns Hopkins Univ, Sch Med, Dept Med, Div Hematol, 720 Rutland Ave,Ross Res Bldg,Room 1025, Baltimore, MD 21205 USA
关键词
THROMBOTIC THROMBOCYTOPENIC PURPURA; PLASMA-EXCHANGE; ANTIBODY DEPLETION; ADAMTS13; ACTIVITY; RITUXIMAB; MICROANGIOPATHIES; BORTEZOMIB; EXPERIENCE; REMISSION; SAFETY;
D O I
10.1182/blood.2019000954
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Insights into immune-mediated thrombotic thrombocytopenic purpura (iTTP) pathophysiology have led to novel targeted therapies. Immunomodulatory strategies target anti-ADAMTS13 antibodies: rituximab is effective in inducing responses in refractory/relapsed TTP and increasing relapse-free survival; caplacizumab targets the von Willebrand factor-platelet interaction to hasten platelet count recovery and reduce mortality and TTP-related ischemic events. Bortezomib and recombinant ADAMTS13 are under investigation. This review examines how targeted therapies are disrupting current treatment paradigms to improve outcomes of iTTP.
引用
收藏
页码:415 / 420
页数:6
相关论文
共 40 条
[1]   IMPROVED SURVIVAL IN THROMBOTIC THROMBOCYTOPENIC PURPURA HEMOLYTIC UREMIC SYNDROME - CLINICAL-EXPERIENCE IN 108 PATIENTS [J].
BELL, WR ;
BRAINE, HG ;
NESS, PM ;
KICKLER, TS .
NEW ENGLAND JOURNAL OF MEDICINE, 1991, 325 (06) :398-403
[2]   Derivation and external validation of the PLASMIC score for rapid assessment of adults with thrombotic microangiopathies: a cohort study [J].
Bendapudi, Pavan K. ;
Hurwitz, Shelley ;
Fry, Ashley ;
Marques, Marisa B. ;
Waldo, Stephen W. ;
Li, Ang ;
Sun, Lova ;
Upadhyay, Vivek ;
Hamdan, Ayad ;
Brunner, Andrew M. ;
Gansner, John M. ;
Viswanathan, Srinivas ;
Kaufman, Richard M. ;
Uhl, Lynne ;
Stowell, Christopher P. ;
Dzik, Walter H. ;
Makar, Robert S. .
LANCET HAEMATOLOGY, 2017, 4 (04) :E157-E164
[3]   Cardiac troponin-I on diagnosis predicts early death and refractoriness in acquired thrombotic thrombocytopenic purpura. Experience of the French Thrombotic Microangiopathies Reference Center [J].
Benhamou, Y. ;
Boelle, P-Y ;
Baudin, B. ;
Ederhy, S. ;
Gras, J. ;
Galicier, L. ;
Azoulay, E. ;
Provot, F. ;
Maury, E. ;
Pene, F. ;
Mira, J. -P. ;
Wynckel, A. ;
Presne, C. ;
Poullin, P. ;
Halimi, J. -M. ;
Delmas, Y. ;
Kanouni, T. ;
Seguin, A. ;
Mousson, C. ;
Servais, A. ;
Bordessoule, D. ;
Perez, P. ;
Hamidou, M. ;
Cohen, A. ;
Veyradier, A. ;
Coppo, P. .
JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2015, 13 (02) :293-302
[4]   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 [J].
Benhamou, Ygal ;
Assie, Cyrielle ;
Boelle, Pierre-Yves ;
Buffet, Marc ;
Grillberger, Rana ;
Malot, Sandrine ;
Wynckel, Alain ;
Presne, Claire ;
Choukroun, Gabriel ;
Poullin, Pascale ;
Provot, Francois ;
Gruson, Didier ;
Hamidou, Mohamed ;
Bordessoule, Dominique ;
Pourrat, Jacques ;
Mira, Jean-Paul ;
Le Guern, Veronique ;
Pouteil-Noble, Claire ;
Daubin, Cedric ;
Vanhille, Philippe ;
Rondeau, Eric ;
Palcoux, Jean-Bernard ;
Mousson, Christiane ;
Vigneau, Cecile ;
Bonmarchand, Guy ;
Guidet, Bertrand ;
Galicier, Lionel ;
Azoulay, Elie ;
Rottensteiner, Hanspeter ;
Veyradier, Agnes ;
Coppo, Paul .
HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2012, 97 (08) :1181-1186
[5]   Cyclosporine or steroids as an adjunct to plasma exchange in the treatment of immune-mediated thrombotic thrombocytopenic purpura [J].
Cataland, Spero R. ;
Kourlas, Peter J. ;
Yang, Shangbin ;
Geyer, Susan ;
Witkoff, Leslie ;
Wu, Haiwa ;
Masias, Camila ;
George, James N. ;
Wu, Haifeng M. .
BLOOD ADVANCES, 2017, 1 (23) :2075-2082
[6]   Successful treatment of severe thrombotic thrombocytopenic purpura with the monoclonal antibody rituximab [J].
Chemnitz, J ;
Draube, A ;
Scheid, C ;
Staib, P ;
Schulz, A ;
Diehl, V ;
Söhngen, D .
AMERICAN JOURNAL OF HEMATOLOGY, 2002, 71 (02) :105-108
[7]   A phase-II sequential case-series study of all patients presenting to four plasma exchange centres with presumed relapsed/refractory thrombotic thrombocytopenic purpura treated with rituximab [J].
Clark, William F. ;
Rock, Gail ;
Barth, David ;
Arnold, Donald M. ;
Webert, Kathyrn E. ;
Yenson, Paul R. ;
Kelton, John G. ;
Li, Lihua ;
Foley, Steven R. .
BRITISH JOURNAL OF HAEMATOLOGY, 2015, 170 (02) :208-217
[8]   Bortezomib therapy in patients with relapsed/refractory acquired thrombotic thrombocytopenic purpura [J].
Eskazan, Ahmet Emre .
ANNALS OF HEMATOLOGY, 2016, 95 (11) :1751-1756
[9]   Rituximab in autoimmune thrombotic thrombocytopenic purpura: A success story [J].
Froissart, Antoine ;
Veyradier, Agnes ;
Hie, Miguel ;
Benhamou, Ygal ;
Coppo, Paul .
EUROPEAN JOURNAL OF INTERNAL MEDICINE, 2015, 26 (09) :659-665
[10]   Efficacy and safety of first-line rituximab in severe, acquired thrombotic thrombocytopenic purpura with a suboptimal response to plasma exchange. Experience of the French Thrombotic Microangiopathies Reference Center [J].
Froissart, Antoine ;
Buffet, Marc ;
Veyradier, Agnes ;
Poullin, Pascale ;
Provot, Francois ;
Malot, Sandrine ;
Schwarzinger, Michael ;
Galicier, Lionel ;
Vanhille, Philippe ;
Vernant, Jean-Paul ;
Bordessoule, Dominique ;
Guidet, Bertrand ;
Azoulay, Elie ;
Mariotte, Eric ;
Rondeau, Eric ;
Mira, Jean-Paul ;
Wynckel, Alain ;
Clabault, Karine ;
Choukroun, Gabriel ;
Presne, Claire ;
Pourrat, Jacques ;
Hamidou, Mohamed ;
Coppo, Paul .
CRITICAL CARE MEDICINE, 2012, 40 (01) :104-111