X Long Zheng1, Sara K Vesely2, Spero R Cataland3, Paul Coppo4, Brian Geldziler5, Alfonso Iorio6,7, Masanori Matsumoto8, Reem A Mustafa9, Menaka Pai7, Gail Rock10, Lene Russell11, Rawan Tarawneh12, Julie Valdes13, Flora Peyvandi14,15. 1. Department of Pathology and Laboratory Medicine, University of Kansas Medical Center, Kansas City, KS, USA. 2. Hudson College of Public Health, University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA. 3. Department of Medicine, Ohio State University, Columbus, OH, USA. 4. Centre de Référence des Microangiopathies Thrombotiques, Service d'Hématologie, Hôpital Saint-Antoine, Sorbonne Université, Assistance Publique, Hôpitaux de Paris, Paris, France. 5. Somerset, NJ, USA. 6. Department of Health Research Methods, Research, and Impact, McMaster University, Hamilton, ON, Canada. 7. Department of Medicine, McMaster University, Hamilton, ON, Canada. 8. Department of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan. 9. Department of Medicine, University of Kansas Mediccal Center, Kansas City, KS, USA. 10. University of Ottawa, Ottawa, ON, Canada. 11. Department of Intensive Care, Copenhagen University Hospital, Copenhagen, Denmark. 12. Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA. 13. Morristown, NJ, USA. 14. Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. 15. Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.
Abstract
BACKGROUND: Despite advances in treatment options for thrombotic thrombocytopenic purpura (TTP), there are still limited high quality data to inform clinicians regarding its appropriate treatment. METHODS: In June 2018, the ISTH formed a multidisciplinary guideline panel to issue recommendations about treatment of TTP. The panel discussed 12 treatment questions related to immune-mediated TTP (iTTP) and hereditary or congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach, including evidence-to-decision frameworks, to appraise evidence and formulate recommendations. RESULTS: The panel agreed on 11 recommendations based on evidence ranging from very low to moderate certainty. For first acute episode and relapses of iTTP, the panel made a strong recommendation for adding corticosteroids to therapeutic plasma exchange (TPE) and a conditional recommendation for adding rituximab and caplacizumab. For asymptomatic iTTP with low plasma ADAMTS13 activity, the panel made a conditional recommendation for the use of rituximab outside of pregnancy, but prophylactic TPE during pregnancy. For asymptomatic cTTP, the panel made a strong recommendation for prophylactic plasma infusion during pregnancy, and a conditional recommendation for plasma infusion or a wait and watch approach outside of pregnancy. CONCLUSIONS: The panel's recommendations are based on all the available evidence for the effects of an individual component of various treatment approaches, including suppressing inflammation, blocking platelet clumping, replacing the missing and/or inhibited ADAMTS13, and suppressing the formation of ADAMTS13 autoantibody. There was insufficient evidence for further comparing different treatment approaches (eg, TPE, corticosteroids, rituximab, and caplacizumab, etc.), for which high quality studies are needed.
BACKGROUND: Despite advances in treatment options for thrombotic thrombocytopenic purpura (TTP), there are still limited high quality data to inform clinicians regarding its appropriate treatment. METHODS: In June 2018, the ISTH formed a multidisciplinary guideline panel to issue recommendations about treatment of TTP. The panel discussed 12 treatment questions related to immune-mediated TTP (iTTP) and hereditary or congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development, and Evaluation approach, including evidence-to-decision frameworks, to appraise evidence and formulate recommendations. RESULTS: The panel agreed on 11 recommendations based on evidence ranging from very low to moderate certainty. For first acute episode and relapses of iTTP, the panel made a strong recommendation for adding corticosteroids to therapeutic plasma exchange (TPE) and a conditional recommendation for adding rituximab and caplacizumab. For asymptomatic iTTP with low plasma ADAMTS13 activity, the panel made a conditional recommendation for the use of rituximab outside of pregnancy, but prophylactic TPE during pregnancy. For asymptomatic cTTP, the panel made a strong recommendation for prophylactic plasma infusion during pregnancy, and a conditional recommendation for plasma infusion or a wait and watch approach outside of pregnancy. CONCLUSIONS: The panel's recommendations are based on all the available evidence for the effects of an individual component of various treatment approaches, including suppressing inflammation, blocking platelet clumping, replacing the missing and/or inhibited ADAMTS13, and suppressing the formation of ADAMTS13 autoantibody. There was insufficient evidence for further comparing different treatment approaches (eg, TPE, corticosteroids, rituximab, and caplacizumab, etc.), for which high quality studies are needed.
Authors: Marie Scully; Spero R Cataland; Flora Peyvandi; Paul Coppo; Paul Knöbl; Johanna A Kremer Hovinga; Ara Metjian; Javier de la Rubia; Katerina Pavenski; Filip Callewaert; Debjit Biswas; Hilde De Winter; Robert K Zeldin Journal: N Engl J Med Date: 2019-01-09 Impact factor: 91.245
Authors: Spero R Cataland; Peter J Kourlas; Shangbin Yang; Susan Geyer; Leslie Witkoff; Haiwa Wu; Camila Masias; James N George; Haifeng M Wu Journal: Blood Adv Date: 2017-10-23
Authors: J Barbot; E Costa; M Guerra; M S Barreirinho; P Isvarlal; R Robles; H E Gerritsen; B Lämmle; M Furlan Journal: Br J Haematol Date: 2001-06 Impact factor: 6.998
Authors: X Long Zheng; Sara K Vesely; Spero R Cataland; Paul Coppo; Brian Geldziler; Alfonso Iorio; Masanori Matsumoto; Reem A Mustafa; Menaka Pai; Gail Rock; Lene Russell; Rawan Tarawneh; Julie Valdes; Flora Peyvandi Journal: J Thromb Haemost Date: 2020-09-11 Impact factor: 5.824
Authors: April S Kennedy; Qurana F Lewis; James G Scott; Johanna A Kremer Hovinga; Bernhard Lämmle; Deirdra R Terrell; Sara K Vesely; James N George Journal: Transfusion Date: 2009-02-13 Impact factor: 3.157
Authors: Xinglong Zheng; Arnel M Pallera; Lawrence T Goodnough; J Evan Sadler; Morey A Blinder Journal: Ann Intern Med Date: 2003-01-21 Impact factor: 25.391