Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria.

Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Research Abstract Details 

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Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Abstract Text:

Robert A Brodsky,Neal S Young,Elisabetta Antonioli,Antonio M Risitano,Hubert Schrezenmeier,Jörg Schubert,Anna Gaya,Luke Coyle,Carlos de Castro,Chieh-Lin Fu,Jaroslaw P Maciejewski,Monica Bessler,Henk-André Kroon,Russell P Rother,Peter Hillmen,Robert A Brodsky,Neal S Young,Elisabetta Antonioli,Antonio M Risitano,Hubert Schrezenmeier, Schubert,Anna Gaya,Luke Coyle,Carlos de Castro,Chieh-Lin Fu,Jaroslaw P Maciejewski,Monica Bessler, Kroon,Russell P Rother,Peter Hillmen,

The terminal complement inhibitor eculizumab was recently shown to be effective and well tolerated in patients with paroxysmal nocturnal hemoglobinuria (PNH). Here, we extended these observations with results from an open-label, non-placebo-controlled, 52-week, phase 3 clinical safety and efficacy study evaluating eculizumab in a broader PNH patient population. Eculizumab was administered by intravenous infusion at 600 mg every 7 +/- 2 days for 4 weeks; 900 mg 7 +/- 2 days later; followed by 900 mg every 14 +/- 2 days for a total treatment period of 52 weeks. Ninety-seven patients at 33 international sites were enrolled. Patients treated with eculizumab responded with an 87% reduction in hemolysis, as measured by lactate dehydrogenase levels (P < .001). Baseline fatigue scores in the FACIT-Fatigue instrument improved by 12.2 +/- 1.1 points (P < .001). Eculizumab treatment led to an improvement in anemia. The increase in hemoglobin level occurred despite a reduction in transfusion requirements from a median of 8.0 units of packed red cells per patient before treatment to 0.0 units per patient during the study (P < .001). Overall, transfusions were reduced 52% from a mean of 12.3 to 5.9 units of packed red cells per patient. Forty-nine patients (51%) achieved transfusion independence for the entire 52-week period. Improvements in hemolysis, fatigue, and transfusion requirements with eculizumab were independent of baseline levels of hemolysis and degree of thrombocytopenia. Quality of life measures were also broadly improved with eculizumab treatment. This study demonstrates that the beneficial effects of eculizumab treatment in patients with PNH are applicable to a broader population of PNH patients than previously studied. This trial is registered at http://clinicaltrials.gov as NCT00130000.

Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Publishing Authors By Initials

RA Brodsky,NS Young,E Antonioli,AM Risitano,H Schrezenmeier,J Schubert,A Gaya,L Coyle,C de Castro,CL Fu,JP Maciejewski,M Bessler,HA Kroon,RP Rother,P Hillmen,RA Brodsky,NS Young,E Antonioli,AM Risitano,H Schrezenmeier,J Schubert,A Gaya,L Coyle,C de Castro,CL Fu,JP Maciejewski,M Bessler,HA Kroon,RP Rother,P Hillmen,

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Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Journal Published:

PUBLICATION TYPE: Journal Article

Journal: Blood

VOLUME: 111

Page Numbers: 1840-7

Journal Abbreviation: Blood

ISSN: 0006-4971

DAY: 30

MONTH: 11

YEAR: 2007

Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Information

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LANGUAGE: eng

NlmUniqueID: 7603509

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