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Vol. 42. Issue S2.
Pages 95 (November 2020)
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Vol. 42. Issue S2.
Pages 95 (November 2020)
160
DOI: 10.1016/j.htct.2020.10.161
Open Access
OUTCOMES OF PATIENTS WITH WORSENING ACQUIRED THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP) DESPITE DAILY THERAPEUTIC PLASMA EXCHANGE IN THE PHASE 3 HERCULES TRIAL
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M. Scullya, J.L. Rubiab,c, F. Peyvandid,e, S. Catalandf, P. Coppog, J.A.K. Hovingah, P. Knoebli, K. Pavenskij, F. Callewaertk, J.M.M. Edoul, R.P. Sousam
a Cardiometabolic Programme, NIHR UCLH/UCL BRC, Department of Haematology, University College London Hospital, London, United Kingdom
b Hematology Department, Internal Medicine, School of Medicine and Dentistry, Catholic University of Valencia, Valencia, Spain
c Hospital Doctor Peset, Valencia, Spain
d Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’Granda Ospedale Maggiore Policlinico, Milan, Italy
e Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
f Division of Hematology, Department of Internal Medicine, The Ohio State University, Columbus, United States
g Department of Hematology, Reference Center for Thrombotic Microangiopathies (CNR-MAT), Saint-Antoine University Hospital, AP-HP, Paris, France
h Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland
i Division of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Wien, Austria
j Departments of Medicine and Laboratory Medicine, St. Michael's Hospital, University of Toronto, Toronto, Canada
k Sanofi, Diegem, Belgium
l Sanofi, Zwijnaarde, Belgium
m Sanofi, Lisbon, Portugal
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Aims: Describe outcomes of patients with suboptimal responses to therapeutic plasma exchange (TPE). Methods: In this post hoc analysis of the HERCULES (NCT02553317) intent-to-treat population (caplacizumab: n = 72; placebo: n = 73), we identified patients with a suboptimal response to daily TPE, defined by decreasing platelet counts and increasing lactate dehydrogenase (LDH) after initial but nonsustained partial improvement of platelet count and LDH during daily TPE. Baseline disease characteristics (previous TTP episodes, platelet count, cardiac troponin, LDH, serum creatinine, disease severity) and treatment outcomes (time to platelet count response, time to stop daily TPE, death, exacerbation) were descriptively summarized. Results: No patient in the caplacizumab group and 8 patients (11%) in the placebo group demonstrated a suboptimal response to TPE. Their median time to achieve platelet count response was 10.88 days and median time to stop daily TPE 13.5 days, compared with 2.88 and 7.00 days, respectively in the overall HERCULES placebo group. One patient had worsening TTP that led to coma and death. Subsequent exacerbations were reported in 5/8 patients (63%). Conclusions: These results show the unpredictability of suboptimal responses to TPE, which can be fatal, and which were not observed in the caplacizumab group. This highlights the importance of the fast and sustained protective response observed with caplacizumab treatment in patients with aTTP. Data first presented at ISTH 2020, 12th-14th July 2020. Study sponsored by Ablynx (a Sanofi company).

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Hematology, Transfusion and Cell Therapy

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