When to consider targeted therapies in thrombotic microangiopathies in the modern era: walking the tightrope between cost, safety, and efficacy

Research output: Contribution to journalArticlepeer-review

3 Scopus citations

Abstract

Thrombotic Microangiopathy (TMA) is a heterogeneous collection of syndromes that encompasses TTP, HUS, and other processes characterized by thrombocytopenia, microangiopathic hemolytic anemia, and, if untreated, organ failure and death. Novel therapies have recently been approved for the management of certain thrombotic microangiopathies, including caplacizumab for immune-mediated TTP, and eculizumab for atypical HUS. These options have complicated the standard workflow, which includes initiation of plasma exchange until ADAMTS13 testing can be resulted. Given such results may take several days, there is indecision regarding the appropriate initial management of TMA. Decisions regarding caplacizumab and eculizumab are complex, and include considerations over costs, side effects, and efficacy. In the following forum, we discuss the current data and pose possible management strategies in patients with TMA before final diagnosis can be obtained.

Original languageEnglish (US)
Pages (from-to)602-605
Number of pages4
JournalJournal of Thrombosis and Thrombolysis
Volume49
Issue number4
DOIs
StatePublished - May 1 2020

Keywords

  • Caplacizumab
  • Eculizumab
  • Microangiopathy
  • Novel drugs
  • TMA
  • TTP

ASJC Scopus subject areas

  • Hematology
  • Cardiology and Cardiovascular Medicine

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