Outcomes in 1096 patients with severe thrombotic thrombocytopenic purpura before the Caplacizumab era.
Journal
PloS one
ISSN: 1932-6203
Titre abrégé: PLoS One
Pays: United States
ID NLM: 101285081
Informations de publication
Date de publication:
2021
2021
Historique:
received:
25
01
2021
accepted:
28
07
2021
entrez:
12
8
2021
pubmed:
13
8
2021
medline:
15
12
2021
Statut:
epublish
Résumé
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. 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. 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. 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.
Identifiants
pubmed: 34383822
doi: 10.1371/journal.pone.0256024
pii: PONE-D-21-02729
pmc: PMC8360509
doi:
Substances chimiques
Antineoplastic Agents, Immunological
0
Single-Domain Antibodies
0
caplacizumab
2R27AB6766
Rituximab
4F4X42SYQ6
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e0256024Déclaration de conflit d'intérêts
“I have read the journal`s policy and the authors of this manuscript have the following competing interests: EA has received fees for lectures from Gilead, Pfizer, Baxter, and Alexion. His research group has been supported by Ablynx, Fisher & Payckle, Jazz Pharma, and MSD. MD has received fees for lectures from Gilead, Astelas, and MSD. His institution has received a research grant from MSD. All other authors have no conflict of interest to disclose. This does not alter our adherence to PLOS ONE policies on sharing data and materials
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