The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria

Peter Hillmen, Neal S. Young, Jörg Schubert, Robert A Brodsky, Gerard Socié, Petra Muus, Alexander Röth, Jeffrey Szer, Modupe O. Elebute, Ryotaro Nakamura, Paul Browne, Antonio M. Risitano, Anita Hill, Hubert Schrezenmeier, Chieh Lin Fu, Jaroslaw Maciejewski, Scott A. Rollins, Christopher F. Mojcik, Russell P. Rother, Lucio Luzzatto

Research output: Contribution to journalArticle

Abstract

BACKGROUND: We tested the safety and efficacy of eculizumab, a humanized monoclonal antibody against terminal complement protein C5 that inhibits terminal complement activation, in patients with paroxysmal nocturnal hemoglobinuria (PNH). METHODS: We conducted a double-blind, randomized, placebo-controlled, multicenter, phase 3 trial. Patients received either placebo or eculizumab intravenously; eculizumab was given at a dose of 600 mg weekly for 4 weeks, followed 1 week later by a 900-mg dose and then 900 mg every other week through week 26. The two primary end points were the stabilization of hemoglobin levels and the number of units of packed red cells transfused. Biochemical indicators of intravascular hemolysis and the patients' quality of life were also assessed. RESULTS: Eighty-seven patients underwent randomization. Stabilization of hemoglobin levels in the absence of transfusions was achieved in 49% (21 of 43) of the patients assigned to eculizumab and none (0 of 44) of those assigned to placebo (P

Original languageEnglish (US)
Pages (from-to)1233-1243
Number of pages11
JournalNew England Journal of Medicine
Volume355
Issue number12
DOIs
StatePublished - Sep 21 2006

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Complement Inactivating Agents
Paroxysmal Hemoglobinuria
Placebos
Hemoglobins
Complement C5
Antibodies, Monoclonal, Humanized
Complement Activation
Random Allocation
Hemolysis
Complement System Proteins
Quality of Life
eculizumab
Safety

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Hillmen, P., Young, N. S., Schubert, J., Brodsky, R. A., Socié, G., Muus, P., ... Luzzatto, L. (2006). The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. New England Journal of Medicine, 355(12), 1233-1243. https://doi.org/10.1056/NEJMoa061648

The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. / Hillmen, Peter; Young, Neal S.; Schubert, Jörg; Brodsky, Robert A; Socié, Gerard; Muus, Petra; Röth, Alexander; Szer, Jeffrey; Elebute, Modupe O.; Nakamura, Ryotaro; Browne, Paul; Risitano, Antonio M.; Hill, Anita; Schrezenmeier, Hubert; Fu, Chieh Lin; Maciejewski, Jaroslaw; Rollins, Scott A.; Mojcik, Christopher F.; Rother, Russell P.; Luzzatto, Lucio.

In: New England Journal of Medicine, Vol. 355, No. 12, 21.09.2006, p. 1233-1243.

Research output: Contribution to journalArticle

Hillmen, P, Young, NS, Schubert, J, Brodsky, RA, Socié, G, Muus, P, Röth, A, Szer, J, Elebute, MO, Nakamura, R, Browne, P, Risitano, AM, Hill, A, Schrezenmeier, H, Fu, CL, Maciejewski, J, Rollins, SA, Mojcik, CF, Rother, RP & Luzzatto, L 2006, 'The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria', New England Journal of Medicine, vol. 355, no. 12, pp. 1233-1243. https://doi.org/10.1056/NEJMoa061648
Hillmen, Peter ; Young, Neal S. ; Schubert, Jörg ; Brodsky, Robert A ; Socié, Gerard ; Muus, Petra ; Röth, Alexander ; Szer, Jeffrey ; Elebute, Modupe O. ; Nakamura, Ryotaro ; Browne, Paul ; Risitano, Antonio M. ; Hill, Anita ; Schrezenmeier, Hubert ; Fu, Chieh Lin ; Maciejewski, Jaroslaw ; Rollins, Scott A. ; Mojcik, Christopher F. ; Rother, Russell P. ; Luzzatto, Lucio. / The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. In: New England Journal of Medicine. 2006 ; Vol. 355, No. 12. pp. 1233-1243.
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