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

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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.

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