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Article Dans Une Revue Blood Année : 2021

Redefining outcomes in immune TTP: an international working group consensus report

Adam Cuker
  • Fonction : Auteur
Spero Cataland
  • Fonction : Auteur
Javier de La Rubia
  • Fonction : Auteur
Kenneth Friedman
  • Fonction : Auteur
James George
  • Fonction : Auteur
Paul Knoebl
  • Fonction : Auteur
Johanna Kremer Hovinga
  • Fonction : Auteur
Bernhard Lӓmmle
  • Fonction : Auteur
Masanori Matsumoto
  • Fonction : Auteur
Katerina Pavenski
  • Fonction : Auteur
Flora Peyvandi
  • Fonction : Auteur
Kazuya Sakai
  • Fonction : Auteur
Ravi Sarode
  • Fonction : Auteur
Mari Thomas
  • Fonction : Auteur
Yoshiaki Tomiyama
  • Fonction : Auteur
John-Paul Westwood
  • Fonction : Auteur
Marie Scully
  • Fonction : Auteur

Résumé

Abstract Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a potentially fatal thrombotic microangiopathy caused by autoantibody-mediated severe deficiency of ADAMTS13. Standardized definitions of response, exacerbation, remission, and relapse were initially proposed in 2003 and modified by the International Working Group for TTP in 2017. These definitions, which have been widely used in clinical practice and research, are based primarily on the platelet count and are benchmarked against the timing of discontinuation of therapeutic plasma exchange (TPE). They do not incorporate ADAMTS13 activity or the temporizing effects on the platelet count of caplacizumab, a novel anti–von Willebrand factor (VWF) nanobody. In light of these limitations, the IWG aimed to develop revised consensus outcome definitions that incorporate ADAMTS13 activity and the effects of anti-VWF therapy, by using an estimate-talk-estimate approach. The updated definitions distinguish clinical remission and clinical relapse (defined primarily by platelet count) from ADAMTS13 remission and ADAMTS13 relapse (defined by ADAMTS13 activity). The revised definitions of exacerbation and remission are benchmarked against not only the timing of discontinuation of TPE but also that of anti-VWF therapy. Retrospective validation of the revised definitions is described, although they have yet to be prospectively validated. Clinical implications of the updated outcome definitions are also discussed and an example of their application to clinical practice is provided to highlight their clinical relevance.

Dates et versions

hal-03244695 , version 1 (01-06-2021)

Identifiants

Citer

Adam Cuker, Spero Cataland, Paul Coppo, Javier de La Rubia, Kenneth Friedman, et al.. Redefining outcomes in immune TTP: an international working group consensus report. Blood, 2021, 137 (14), pp.1855-1861. ⟨10.1182/blood.2020009150⟩. ⟨hal-03244695⟩

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