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Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria

Open access
Hillmen, Peter, Muus, Petra, Dührsen, Ulrich, Risitano, Antonio M., Schubert, Jörg, Luzzatto, Lucio, Schrezenmeier, Hubert, Szer, Jeffrey, Brodsky, Robert A., Hill, Anita, Socié, Gerard, Bessler, Monica, Rollins, Scott A., Bell, Leonard, Rother, Russell P., Young, Neal S.·2007·Blood

Abstract

Hemolysis and hemoglobinemia contribute to serious clinical sequelae in hemolytic disorders. In paroxysmal nocturnal hemoglobinuria (PNH) patients, hemolysis can contribute to thromboembolism (TE), the most feared complication in PNH, and the leading cause of disease-related deaths. We evaluated whether long-term treatment with the complement inhibitor eculizumab reduces the rate of TE in patients with PNH. Clinical trial participants included all patients in the 3 eculizumab PNH clinical studies, which recruited patients between 2002 and 2005 (n = 195); patients from these studies continued treatment in the current multinational open-label extension study. Thromboembolism rate with eculizumab treatment was compared with the pretreatment rate in the same patients. The TE event rate with eculizumab treatment was 1.07 events/100 patient-years compared with 7.37 events/100 patient-years (P < .001) prior to eculizumab treatment (relative reduction, 85%; absolute reduction, 6.3 TE events/100 patient-years). With equalization of the duration of exposure before and during treatment for each patient, TE events were reduced from 39 events before eculizumab to 3 events during eculizumab (P < .001). The TE event rate in antithrombotic-treated patients (n = 103) was reduced from 10.61 to 0.62 events/100 patient-years with eculizumab treatment (P < .001). These results show that eculizumab treatment reduces the risk of clinical thromboembolism in patients with PNH. This study is registered at http://clinicaltrials.gov (study ID no. NCT00122317).

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Metadata

Authors
Hillmen, P., Muus, P., Dührsen, U., Risitano, A. M., Schubert, J., Luzzatto, L., Schrezenmeier, H., Szer, J., Brodsky, R. A., Hill, A., Socié, G., Bessler, M., Rollins, S. A., Bell, L., Rother, R. P., & Young, N. S.
Venue
Blood
Published
2007-12-01
Volume
110
Issue
12
Pages
4123-4128
ISSN
  • 0006-4971 (ISSN-L)
  • 1528-0020
OpenAlex
W2058082996
Cited by
579
References
32
FWCI
10.53
Type
journal article
Language
en
Open access
bronze

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CoChat. (2026). Citation check: Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. https://app.cochat.ai/papers/10.1182/blood-2007-06-095646
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Hillmen, P., Muus, P., Dührsen, U., Risitano, A. M., Schubert, J., Luzzatto, L., Schrezenmeier, H., Szer, J., Brodsky, R. A., Hill, A., Socié, G., Bessler, M., Rollins, S. A., Bell, L., Rother, R. P., & Young, N. S. (2007). Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. https://doi.org/10.1182/blood-2007-06-095646

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