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.
Reference
Shatzel J, Taylor JA (2017) Syndromes of thrombotic microangiopathy. Med Clin North Am 101:395–415
Scully M, Cataland SR, Peyvandi F, Coppo P, Knobl PK, Hovinga JA, Metjian A, de la Rubia J, Pavenski K, Callewaert F, Biswas D, De Winter H, Zeldin RK (2019) Caplacizumab treatment for acquired thrombotic thrombocytopenic purpura. N Engl J Med 380:335–346
Davio K (2019) FDA Approves the First Drug Specifically Indicated for aTTP. AJMC Managed Markets Network. p 7 of 9. http://www.ajmc.com. Accessed 16 Nov 2019
Bendapudi P, Hurwitz S, Fry A, Marques MB, Waldo SW, Li A, Sun L, Upadhyay V, Hamdan A, Brunner AM, Gansner JM, Viswanathan S, Kaufman RM, Uhl L, Stowell CP, Dzik WH, Makar RS (2017) Derivation and external validation of the PLASMIC score for rapid assessment of adults with thrombotic microangiopathies: a cohort study. Lancet Hematol 4:e157–e164
Li A, Khalighi PR, Wu Q, Garcia DA (2018) External validation of the PLASMIC score: a clinical prediction tool for thrombotic thrombocytopenic purpura diagnosis and treatment. J Thromb Haemost. 16(1):164–169
Kim CH, Simmons SC, Williams LA III, Staley EM, Zheng XL, Pham HP (2017) ADAMST13 test and/or PLASMIC clinical score in management of acquired thrombotic thrombocytopenic purpura: a cost-effective analysis. Transfusion 57(11):2609–2618
Legendre C, Licht C, Muus P, Greenbaum LA, Babu S, Bedrosian C, Bingham C, Cohen DJ, Delmas Y, Douglas K, Eitner F, Feldkamp T (2013) Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med 368:2169–2181
Herper M (2010) The world’s most expensive drugs. Forbes. http://www.forbes.com. Accessed 8 Jan 2019
McKeage K (2019) Ravulizumab: first global approval. Drugs 79(3):347–352
Go RS, Winters JL, Leung N et al (2016) Thrombotic microangiopathy care pathway: a consensus statement for the mayo clinic complement alternative pathway-thrombotic microangiopathy (CAP-TMA) Disease-Oriented Group. Mayo Clin Proc 91(9):1189–1211
Smock KJ (2016) The Role of ADAMTS13 testing in the work up of suspected thrombotic thrombocytopenic purpura. AACC
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Murphree, C.R., Olson, S.R., DeLoughery, T.G. et al. When to consider targeted therapies in thrombotic microangiopathies in the modern era: walking the tightrope between cost, safety, and efficacy. J Thromb Thrombolysis 49, 602–605 (2020). https://doi.org/10.1007/s11239-020-02094-8
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DOI: https://doi.org/10.1007/s11239-020-02094-8