Good practice statements (GPS) for the clinical care of patients with thrombotic thrombocytopenic purpura

X Long Zheng¹, Sara K Vesely², Spero R Cataland³, Paul Coppo⁴, Brian Geldziler⁵, Alfonso Iorio^{6

7}, Masanori Matsumoto⁸, Reem A Mustafa⁹, Menaka Pai⁷, Gail Rock¹⁰, Lene Russell¹¹, Rawan Tarawneh¹², Julie Valdes¹³, Flora Peyvandi^{14

15}

Affiliations

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, The 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.

PMID: 32914535
PMCID: PMC7880820
DOI: 10.1111/jth.15009

Good practice statements (GPS) for the clinical care of patients with thrombotic thrombocytopenic purpura

X Long Zheng et al. J Thromb Haemost. 2020 Oct.

. 2020 Oct;18(10):2503-2512.

doi: 10.1111/jth.15009. Epub 2020 Sep 11.

Authors

Affiliations

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, The 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.

PMID: 32914535
PMCID: PMC7880820
DOI: 10.1111/jth.15009

Abstract

Background: Despite advances in treatment options for thrombotic thrombocytopenic purpura (TTP), there are still limited high quality data to inform clinicians regarding its management.

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 both immune-mediated TTP (iTTP) and hereditary/congenital TTP (cTTP). The panel used the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach, including evidence-to-decision frameworks, to appraise evidence and formulate recommendations.

Results: The panel agreed on eleven recommendations based on evidence ranging from very low to moderate certainty. For first episode and relapses of acute iTTP, the panel made a strong recommendation for the addition of corticosteroids to therapeutic plasma exchange (TPE), and a conditional recommendation for addition of rituximab and caplacizumab. For asymptomatic iTTP with low ADAMTS13, the panel made a conditional recommendation for rituximab outside of pregnancy, and for prophylactic TPE during pregnancy. For asymptomatic cTTP, the panel made a strong recommendation for prophylactic plasma infusion during pregnancy, but 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 treatment effects of various approaches including suppressing inflammation, blocking platelet clumping, replacing the missing and/or inhibited ADAMTS13, and suppressing ADAMTS13 antibody production. There was insufficient evidence for further comparison of different treatment approaches, for which future high-quality studies in iTTP (e.g., rituximab, corticosteroids, recombinant ADAMTS13, and caplacizumab) and in cTTP (eg, recombinant ADAMTS13) are needed.

Keywords: TTP; diagnosis; good practice; guidelines; management.

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References

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Good practice statements (GPS) for the clinical care of patients with thrombotic thrombocytopenic purpura

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Good practice statements (GPS) for the clinical care of patients with thrombotic thrombocytopenic purpura

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