Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a potentially fatal blood disorder resulting from acquired deficiency of plasma ADAMTS13 activity. Despite recent advances in early diagnosis and novel therapeutics, the mortality rate of acute iTTP remains as high as 10% to 20%. Moreover, a reliable clinical and laboratory parameter that predicts disease severity and outcomes is lacking. We show in the present study that plasma levels of syndecan-1 (Sdc-1) and soluble thrombomodulin (sTM) on admission were dramatically increased in patients with acute iTTP and remained substantially elevated in a subset of patients compared with healthy controls. The elevated admission plasma levels of Sdc-1 and sTM were associated with abnormal Glasgow coma scale scores, low estimated glomerular filtration rates, the need for intensive care, and in-hospital mortality rates. Moreover, a further simultaneous increase in plasma Sdc-1 and sTM levels at the time of clinical response/remission (eg, when normalization of platelet counts and substantial reduction of serum lactate dehydrogenase activity were achieved) was highly predictive of iTTP recurrence. These results demonstrate that endothelial injury, resulting from disseminated microvascular thromboses, is severe and persistent in patients with acute iTTP. Plasma levels of Sdc-1 and sTM on admission and in remission are predictive of in-hospital mortality and recurrence of acute iTTP, respectively. Thus, an incorporation of such novel plasma biomarkers into the risk assessment in acute iTTP may help implement a more vigorous and intensive therapeutic strategy for these patients.
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http://dx.doi.org/10.1182/bloodadvances.2020003065 | DOI Listing |
J Manag Care Spec Pharm
December 2024
Health Economics and Value Assessment, Sanofi, Boston, MA.
Background: Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy. Caplacizumab is the only treatment approved by the European Medicines Agency and the US Food and Drug Administration for iTTP, to be given in combination with plasma exchange therapy (PEX) and immunosuppression (IS). The National Institute for Health and Care Excellence's independent appraisal committee assessed the cost-effectiveness of caplacizumab and concluded that the addition of caplacizumab to PEX+IS is cost-effective under a patient access scheme in the United Kingdom.
View Article and Find Full Text PDFJ Thromb Haemost
November 2024
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy; Department of Pathophysiology and Transplantation, Fondazione Luigi Villa, Università degli Studi di Milano, Milan, Italy. Electronic address:
Background: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) patients are not responsive to standard rituximab in approximately 10% to 15% of cases, and oral immunosuppressants showed controversial results with significant toxicity. Targeting plasma cells with bortezomib appears promising, but the available evidence is scarce and stems only from isolated reports in the precaplacizumab era.
Objectives: To evaluate the safety and efficacy of bortezomib in rituximab-refractory iTTP patients.
Kidney Int Rep
November 2024
Atrium Health Levine Children's Hospital, Charlotte, North Carolina, USA.
J Clin Med
October 2024
Service of Haemorrhagic and Thrombotic Diseases, Fondazione Policlinico Universitario Agostino Gemelli-IRCCS, 00168 Rome, Italy.
: Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by the formation of anti-ADAMTS13 antibodies. Caplacizumab is approved for the treatment of acute episodes of iTTP in conjunction with plasma exchange (PEX) and immunosuppression. Real-world data for the use of caplacizumab in Italy have been recently published by a limited number of centers located in the northern and middle regions of the country only.
View Article and Find Full Text PDFClin Adv Hematol Oncol
October 2024
Division of Hematology and Medical Oncology, NewYork-Presbyterian Hospital and Weill Cornell Medicine, New York, New York.
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