Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.

<h4>Introduction</h4>Thrombotic thrombocytopenic purpura (TTP) is a diagnostic and therapeutic emergency. Therapeutic plasma exchange (TPE) combined with immunosuppression has been the cornerstone of the initial management. To produce optimal benefits, emerging treatments must be used ag...

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Autores principales:	Andry Van de Louw, Eric Mariotte, Michael Darmon, Austin Cohrs, Douglas Leslie, Elie Azoulay
Formato:	article
Lenguaje:	EN
Publicado:	Public Library of Science (PLoS) 2021
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/df514f54a2344415b52ce37799b62a31
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spelling	oai:doaj.org-article:df514f54a2344415b52ce37799b62a312021-12-02T20:18:16ZOutcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.1932-620310.1371/journal.pone.0256024https://doaj.org/article/df514f54a2344415b52ce37799b62a312021-01-01T00:00:00Zhttps://doi.org/10.1371/journal.pone.0256024https://doaj.org/toc/1932-6203<h4>Introduction</h4>Thrombotic thrombocytopenic purpura (TTP) is a diagnostic and therapeutic emergency. Therapeutic plasma exchange (TPE) combined with immunosuppression has been the cornerstone of the initial management. To produce optimal benefits, emerging treatments must be used against a background of best standard of care. Clarifying current uncertainties is therefore crucial.<h4>Methods</h4>The objective of this study was to analyze a large high-quality database (Marketscan) of TTP patients managed between 2005 and 2014, in the pre-caplacizumab era, in order to assess the impact of time to first TPE and use of first-line rituximab on mortality, and whether mortality declines over time.<h4>Results</h4>Among the 1096 included patients (median age 46 [IQR 35-55], 70% female), 28.8% received TPE before day 2 in the ICU. Hospital mortality was 7.6% (83 deaths). Mortality was independently associated with older age (hazard ratio [HR], 1.024/year; 95% confidence interval [95%CI], [1.009-1.040]), diagnosis of sepsis (HR, 2.360; 95%CI [1.552-3.588]), and the need for mechanical ventilation (HR, 4.103; 95%CI, [2.749-6.126]). Factors independently associated with lower mortality were TPE at ICU admission (HR, 0.284; 95%CI, [0.112-0.717]), TPE within one day after ICU admission (HR, 0.449; 95%CI, [0.275-0.907]), and early rituximab therapy (HR, 0.229; 95% CI, [0.111-0.471]). Delayed TPE was associated with significantly higher costs.<h4>Conclusions</h4>Immediate TPE and early rituximab are associated with improved survival in TTP patients. Improved treatments have led to a decline in mortality over time, and alternate outcome variables such as the use of hospital resources or longer term outcomes therefore need to be considered.Andry Van de LouwEric MariotteMichael DarmonAustin CohrsDouglas LeslieElie AzoulayPublic Library of Science (PLoS)articleMedicineRScienceQENPLoS ONE, Vol 16, Iss 8, p e0256024 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Andry Van de Louw Eric Mariotte Michael Darmon Austin Cohrs Douglas Leslie Elie Azoulay Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.
description	<h4>Introduction</h4>Thrombotic thrombocytopenic purpura (TTP) is a diagnostic and therapeutic emergency. Therapeutic plasma exchange (TPE) combined with immunosuppression has been the cornerstone of the initial management. To produce optimal benefits, emerging treatments must be used against a background of best standard of care. Clarifying current uncertainties is therefore crucial.<h4>Methods</h4>The objective of this study was to analyze a large high-quality database (Marketscan) of TTP patients managed between 2005 and 2014, in the pre-caplacizumab era, in order to assess the impact of time to first TPE and use of first-line rituximab on mortality, and whether mortality declines over time.<h4>Results</h4>Among the 1096 included patients (median age 46 [IQR 35-55], 70% female), 28.8% received TPE before day 2 in the ICU. Hospital mortality was 7.6% (83 deaths). Mortality was independently associated with older age (hazard ratio [HR], 1.024/year; 95% confidence interval [95%CI], [1.009-1.040]), diagnosis of sepsis (HR, 2.360; 95%CI [1.552-3.588]), and the need for mechanical ventilation (HR, 4.103; 95%CI, [2.749-6.126]). Factors independently associated with lower mortality were TPE at ICU admission (HR, 0.284; 95%CI, [0.112-0.717]), TPE within one day after ICU admission (HR, 0.449; 95%CI, [0.275-0.907]), and early rituximab therapy (HR, 0.229; 95% CI, [0.111-0.471]). Delayed TPE was associated with significantly higher costs.<h4>Conclusions</h4>Immediate TPE and early rituximab are associated with improved survival in TTP patients. Improved treatments have led to a decline in mortality over time, and alternate outcome variables such as the use of hospital resources or longer term outcomes therefore need to be considered.
format	article
author	Andry Van de Louw Eric Mariotte Michael Darmon Austin Cohrs Douglas Leslie Elie Azoulay
author_facet	Andry Van de Louw Eric Mariotte Michael Darmon Austin Cohrs Douglas Leslie Elie Azoulay
author_sort	Andry Van de Louw
title	Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.
title_short	Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.
title_full	Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.
title_fullStr	Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.
title_full_unstemmed	Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.
title_sort	outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the caplacizumab era.
publisher	Public Library of Science (PLoS)
publishDate	2021
url	https://doaj.org/article/df514f54a2344415b52ce37799b62a31
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Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.

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