Caplacizumab reduces the frequency of major thromboembolic events, exacerbations, and death in patients with acquired thrombotic thrombocytopenic purpura.

Peyvandi, F; Scully, M; Kremer Hovinga, Johanna Anna; Knöbl, P; Cataland, S; De Beuf, K; Callewaert, F; De Winter, H; Zeldin, R K (2017). Caplacizumab reduces the frequency of major thromboembolic events, exacerbations, and death in patients with acquired thrombotic thrombocytopenic purpura. Journal of thrombosis and haemostasis, 15(7), pp. 1448-1452. Wiley-Blackwell 10.1111/jth.13716

[img]
Preview
Text
JKH_Peyvandi_et_al-2017-Journal_of_Thrombosis_and_Haemostasis.pdf - Accepted Version
Available under License Creative Commons: Attribution-Noncommercial-No Derivative Works (CC-BY-NC-ND).

Download (695kB) | Preview
[img]
Preview
Text
Peyvandi_et_al-2017-Journal_of_Thrombosis_and_Haemostasis.pdf - Published Version
Available under License Creative Commons: Attribution-Noncommercial-No Derivative Works (CC-BY-NC-ND).

Download (179kB) | Preview

BACKGROUND Acquired thrombotic thrombocytopenic purpura (aTTP) is a life-threatening autoimmune thrombotic microangiopathy. In spite of treatment with plasma exchange and immunosuppression, patients remain at risk for thrombotic complications, exacerbations and death. In the Phase II TITAN study, treatment with caplacizumab, an anti-vWF Nanobody(®) , was shown to reduce the time to confirmed platelet count normalization and exacerbations during treatment. OBJECTIVE The clinical benefit of caplacizumab was further investigated in a post-hoc analysis of the incidence of major thromboembolic events and exacerbations during the study drug treatment period and TTP-related death during the study. METHODS The Standardized MedDRA Query (SMQ) for 'embolic and thrombotic events' was run to investigate the occurrence of major thromboembolic events and exacerbations in the safety population of the TITAN study, which consisted of 72 patients of whom 35 received caplacizumab and 37 received placebo. RESULTS Four events (1 pulmonary embolism and 3 aTTP exacerbations) were reported in 4 patients in the caplacizumab group, while 20 such events were reported in 14 patients in the placebo group (2 acute myocardial infarctions, 1 ischemic and 1 hemorrhagic stroke, 1 pulmonary embolism, 1 deep vein thrombosis, 1 venous thrombosis and 13 aTTP exacerbations). Two of the placebo-treated patients died from aTTP during the study. CONCLUSION In total, 11.4% of caplacizumab-treated patients versus 43.2% of placebo-treated patients experienced one or more major thromboembolic event, an exacerbation or died. This analysis shows the potential for caplacizumab to reduce the risk of major thromboembolic morbidities and mortality associated with aTTP. This article is protected by copyright. All rights reserved.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Haematology and Central Haematological Laboratory
04 Faculty of Medicine > Pre-clinic Human Medicine > BioMedical Research (DBMR) > Unit Childrens Hospital > Forschungsgruppe Hämatologie (Erwachsene)

UniBE Contributor:

Kremer Hovinga, Johanna Anna

Subjects:

600 Technology > 610 Medicine & health

ISSN:

1538-7836

Publisher:

Wiley-Blackwell

Language:

English

Submitter:

Katrin Kölliker-Schütz

Date Deposited:

10 Oct 2017 09:32

Last Modified:

16 Jul 2018 14:11

Publisher DOI:

10.1111/jth.13716

PubMed ID:

28445600

Uncontrolled Keywords:

Purpura Thrombotic Thrombocytopenic caplacizumab morbidity mortality von Willebrand Factor

BORIS DOI:

10.7892/boris.100071

URI:

https://boris.unibe.ch/id/eprint/100071

Actions (login required)

Edit item Edit item
Provide Feedback