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

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

J Thromb Thrombolysis

Division of Hematology, Oregon Health & Science University, Knight Cancer Institute, 3181 SW Sam Jackson Park Rd, Portland, OR, 97239, USA.

Published: May 2020

AI Article Synopsis

  • Thrombotic Microangiopathy (TMA) includes conditions like Thrombotic Thrombocytopenic Purpura (TTP) and Hemolytic Uremic Syndrome (HUS), characterized by low platelet counts, anemia, and potential organ failure.
  • New treatments like caplacizumab for TTP and eculizumab for atypical HUS have been introduced, complicating traditional management that usually starts with plasma exchange.
  • The forum will explore current data and discuss management strategies for TMA patients, focusing on the challenges of making treatment decisions when a definitive diagnosis is pending.

Article 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.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9020175PMC
http://dx.doi.org/10.1007/s11239-020-02094-8DOI Listing

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