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

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

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)2503-2512
JournalJournal of Thrombosis and Haemostasis
Issue number10
Publication statusPublished - 2020


  • diagnosis
  • good practice
  • guidelines
  • management
  • TTP

ASJC Scopus subject areas

  • Hematology

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