TY - JOUR
T1 - When to consider targeted therapies in thrombotic microangiopathies in the modern era
T2 - walking the tightrope between cost, safety, and efficacy
AU - Murphree, Catherine R.
AU - Olson, Sven R.
AU - DeLoughery, Thomas G.
AU - Shatzel, Joseph J.
N1 - Publisher Copyright:
© 2020, Springer Science+Business Media, LLC, part of Springer Nature.
PY - 2020/5/1
Y1 - 2020/5/1
N2 - 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.
AB - 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.
KW - Caplacizumab
KW - Eculizumab
KW - Microangiopathy
KW - Novel drugs
KW - TMA
KW - TTP
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U2 - 10.1007/s11239-020-02094-8
DO - 10.1007/s11239-020-02094-8
M3 - Article
C2 - 32219721
AN - SCOPUS:85082959497
SN - 0929-5305
VL - 49
SP - 602
EP - 605
JO - Journal of Thrombosis and Thrombolysis
JF - Journal of Thrombosis and Thrombolysis
IS - 4
ER -