AustralieFrV1, Main, Exploration, bibRecord, 009B67

The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria

Identifieur interne : 009B67 ( Main/Exploration ); précédent : 009B66; suivant : 009B68

The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria

Auteurs : Peter Hillmen [Royaume-Uni] ; Neal S. Young [États-Unis] ; Jörg Schubert [Allemagne] ; Robert A. Brodsky [États-Unis] ; Gerard Socie [France] ; Petra Muus [Pays-Bas] ; Alexander Roth [Allemagne] ; Jeffrey Szer [Australie] ; Modupe O. Elebute [Royaume-Uni] ; Ryotaro Nakamura [États-Unis] ; Paul Browne [Israël] ; Antonio M. Risitano [Italie] ; Anita Hill [Royaume-Uni] ; Hubert Schrezenmeier [Allemagne] ; Chieh-Lin Fu [États-Unis] ; Jaroslaw Maciejewski [États-Unis] ; Scott A. Rollins [États-Unis] ; Christopher F. Mojcik [États-Unis] ; Russell P. Rother [États-Unis] ; Lucio Luzzatto [Italie]

Source :

The New England journal of medicine [ 0028-4793 ] ; 2006.

RBID : Pascal:06-0449907

Descripteurs français

Pascal (Inist)
- Anémie hémolytique Marchiafava Micheli, Complément, Inhibiteur, Eculizumab, Médecine, Immunomodulateur.
Wicri :
- topic : Médecine.

English descriptors

KwdEn :
- Complement, Eculizumab, Immunomodulator, Inhibitor, Medicine, Nocturnal paroxystic anemia.

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<0.001). During the study, a median of 0 units of packed red cells was administered in the eculizumab group, as compared with 10 units in the placebo group (P<0.001). Eculizumab reduced intravascular hemolysis, as shown by the 85.8% lower median area under the curve for lactate dehydrogenase plotted against time (in days) in the eculizumab group, as compared with the placebo group (58,587 vs. 411,822 U per liter; P<0.001). Clinically significant improvements were also found in the quality of life, as measured by scores on the Functional Assessment of Chronic Illness Therapy-Fatigue instrument (P<0.001) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire. Of the 87 patients, 4 in the eculizumab group and 9 in the placebo group had serious adverse events, none of which were considered to be treatment-related; all these patients recovered without sequelae. CONCLUSIONS Eculizumab is an effective therapy for PNH.

Affiliations:

Allemagne, Australie, France, Israël, Italie, Pays-Bas, Royaume-Uni, États-Unis
Angleterre, Bade-Wurtemberg, District de Tübingen, Grand Londres, Gueldre, Maryland, Victoria (État), Île-de-France
Baltimore, Londres, Melbourne, Nimègue, Paris, Ulm

Links toward previous steps (curation, corpus...)

to stream PascalFrancis, to step Corpus: 004152
to stream PascalFrancis, to step Curation: 001F43
to stream PascalFrancis, to step Checkpoint: 003C21
to stream Main, to step Merge: 00A643
to stream Main, to step Curation: 009B67

Le document en format XML

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<affiliation wicri:level="1"><inist:fA14 i1="17"><s1>Istituto To scano Tumori</s1>
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<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria</title>
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<author><name sortKey="Risitano, Antonio M" sort="Risitano, Antonio M" uniqKey="Risitano A" first="Antonio M." last="Risitano">Antonio M. Risitano</name>
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<author><name sortKey="Hill, Anita" sort="Hill, Anita" uniqKey="Hill A" first="Anita" last="Hill">Anita Hill</name>
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</affiliation>
</author>
<author><name sortKey="Schrezenmeier, Hubert" sort="Schrezenmeier, Hubert" uniqKey="Schrezenmeier H" first="Hubert" last="Schrezenmeier">Hubert Schrezenmeier</name>
<affiliation wicri:level="3"><inist:fA14 i1="13"><s1>Institute ofTransfusion Med icine, University Hospital</s1>
<s2>Ulm</s2>
<s3>DEU</s3>
<sZ>14 aut.</sZ>
</inist:fA14>
<country>Allemagne</country>
<placeName><region type="land" nuts="1">Bade-Wurtemberg</region>
<region type="district" nuts="2">District de Tübingen</region>
<settlement type="city">Ulm</settlement>
</placeName>
</affiliation>
</author>
<author><name sortKey="Fu, Chieh Lin" sort="Fu, Chieh Lin" uniqKey="Fu C" first="Chieh-Lin" last="Fu">Chieh-Lin Fu</name>
<affiliation wicri:level="1"><inist:fA14 i1="14"><s1>Cleveland Clinic Florida</s1>
<s2>Weston, FL</s2>
<s3>USA</s3>
<sZ>15 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<wicri:noRegion>Cleveland Clinic Florida</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Maciejewski, Jaroslaw" sort="Maciejewski, Jaroslaw" uniqKey="Maciejewski J" first="Jaroslaw" last="Maciejewski">Jaroslaw Maciejewski</name>
<affiliation wicri:level="1"><inist:fA14 i1="15"><s1>Taussig Cancer Center, land Clinic Foundation</s1>
<s2>Cleveland</s2>
<s3>USA</s3>
<sZ>16 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<wicri:noRegion>Cleveland</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Rollins, Scott A" sort="Rollins, Scott A" uniqKey="Rollins S" first="Scott A." last="Rollins">Scott A. Rollins</name>
<affiliation wicri:level="1"><inist:fA14 i1="16"><s1>Alexion Pharmaceuticals</s1>
<s2>Cheshire, CT</s2>
<s3>USA</s3>
<sZ>17 aut.</sZ>
<sZ>18 aut.</sZ>
<sZ>19 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<wicri:noRegion>Alexion Pharmaceuticals</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Mojcik, Christopher F" sort="Mojcik, Christopher F" uniqKey="Mojcik C" first="Christopher F." last="Mojcik">Christopher F. Mojcik</name>
<affiliation wicri:level="1"><inist:fA14 i1="16"><s1>Alexion Pharmaceuticals</s1>
<s2>Cheshire, CT</s2>
<s3>USA</s3>
<sZ>17 aut.</sZ>
<sZ>18 aut.</sZ>
<sZ>19 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<wicri:noRegion>Alexion Pharmaceuticals</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Rother, Russell P" sort="Rother, Russell P" uniqKey="Rother R" first="Russell P." last="Rother">Russell P. Rother</name>
<affiliation wicri:level="1"><inist:fA14 i1="16"><s1>Alexion Pharmaceuticals</s1>
<s2>Cheshire, CT</s2>
<s3>USA</s3>
<sZ>17 aut.</sZ>
<sZ>18 aut.</sZ>
<sZ>19 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<wicri:noRegion>Alexion Pharmaceuticals</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Luzzatto, Lucio" sort="Luzzatto, Lucio" uniqKey="Luzzatto L" first="Lucio" last="Luzzatto">Lucio Luzzatto</name>
<affiliation wicri:level="1"><inist:fA14 i1="17"><s1>Istituto To scano Tumori</s1>
<s2>Florence</s2>
<s3>ITA</s3>
<sZ>20 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<wicri:noRegion>Istituto To scano Tumori</wicri:noRegion>
</affiliation>
</author>
</analytic>
<series><title level="j" type="main">The New England journal of medicine</title>
<title level="j" type="abbreviated">N. Engl. j. med.</title>
<idno type="ISSN">0028-4793</idno>
<imprint><date when="2006">2006</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
<seriesStmt><title level="j" type="main">The New England journal of medicine</title>
<title level="j" type="abbreviated">N. Engl. j. med.</title>
<idno type="ISSN">0028-4793</idno>
</seriesStmt>
</fileDesc>
<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Complement</term>
<term>Eculizumab</term>
<term>Immunomodulator</term>
<term>Inhibitor</term>
<term>Medicine</term>
<term>Nocturnal paroxystic anemia</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Anémie hémolytique Marchiafava Micheli</term>
<term>Complément</term>
<term>Inhibiteur</term>
<term>Eculizumab</term>
<term>Médecine</term>
<term>Immunomodulateur</term>
</keywords>
<keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Médecine</term>
</keywords>
</textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en">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<0.001). During the study, a median of 0 units of packed red cells was administered in the eculizumab group, as compared with 10 units in the placebo group (P<0.001). Eculizumab reduced intravascular hemolysis, as shown by the 85.8% lower median area under the curve for lactate dehydrogenase plotted against time (in days) in the eculizumab group, as compared with the placebo group (58,587 vs. 411,822 U per liter; P<0.001). Clinically significant improvements were also found in the quality of life, as measured by scores on the Functional Assessment of Chronic Illness Therapy-Fatigue instrument (P<0.001) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire. Of the 87 patients, 4 in the eculizumab group and 9 in the placebo group had serious adverse events, none of which were considered to be treatment-related; all these patients recovered without sequelae. CONCLUSIONS Eculizumab is an effective therapy for PNH.</div>
</front>
</TEI>
<affiliations><list><country><li>Allemagne</li>
<li>Australie</li>
<li>France</li>
<li>Israël</li>
<li>Italie</li>
<li>Pays-Bas</li>
<li>Royaume-Uni</li>
<li>États-Unis</li>
</country>
<region><li>Angleterre</li>
<li>Bade-Wurtemberg</li>
<li>District de Tübingen</li>
<li>Grand Londres</li>
<li>Gueldre</li>
<li>Maryland</li>
<li>Victoria (État)</li>
<li>Île-de-France</li>
</region>
<settlement><li>Baltimore</li>
<li>Londres</li>
<li>Melbourne</li>
<li>Nimègue</li>
<li>Paris</li>
<li>Ulm</li>
</settlement>
</list>
<tree><country name="Royaume-Uni"><noRegion><name sortKey="Hillmen, Peter" sort="Hillmen, Peter" uniqKey="Hillmen P" first="Peter" last="Hillmen">Peter Hillmen</name>
</noRegion>
<name sortKey="Elebute, Modupe O" sort="Elebute, Modupe O" uniqKey="Elebute M" first="Modupe O." last="Elebute">Modupe O. Elebute</name>
<name sortKey="Hill, Anita" sort="Hill, Anita" uniqKey="Hill A" first="Anita" last="Hill">Anita Hill</name>
</country>
<country name="États-Unis"><noRegion><name sortKey="Young, Neal S" sort="Young, Neal S" uniqKey="Young N" first="Neal S." last="Young">Neal S. Young</name>
</noRegion>
<name sortKey="Brodsky, Robert A" sort="Brodsky, Robert A" uniqKey="Brodsky R" first="Robert A." last="Brodsky">Robert A. Brodsky</name>
<name sortKey="Fu, Chieh Lin" sort="Fu, Chieh Lin" uniqKey="Fu C" first="Chieh-Lin" last="Fu">Chieh-Lin Fu</name>
<name sortKey="Maciejewski, Jaroslaw" sort="Maciejewski, Jaroslaw" uniqKey="Maciejewski J" first="Jaroslaw" last="Maciejewski">Jaroslaw Maciejewski</name>
<name sortKey="Mojcik, Christopher F" sort="Mojcik, Christopher F" uniqKey="Mojcik C" first="Christopher F." last="Mojcik">Christopher F. Mojcik</name>
<name sortKey="Nakamura, Ryotaro" sort="Nakamura, Ryotaro" uniqKey="Nakamura R" first="Ryotaro" last="Nakamura">Ryotaro Nakamura</name>
<name sortKey="Rollins, Scott A" sort="Rollins, Scott A" uniqKey="Rollins S" first="Scott A." last="Rollins">Scott A. Rollins</name>
<name sortKey="Rother, Russell P" sort="Rother, Russell P" uniqKey="Rother R" first="Russell P." last="Rother">Russell P. Rother</name>
</country>
<country name="Allemagne"><noRegion><name sortKey="Schubert, Jorg" sort="Schubert, Jorg" uniqKey="Schubert J" first="Jörg" last="Schubert">Jörg Schubert</name>
</noRegion>
<name sortKey="Roth, Alexander" sort="Roth, Alexander" uniqKey="Roth A" first="Alexander" last="Roth">Alexander Roth</name>
<name sortKey="Schrezenmeier, Hubert" sort="Schrezenmeier, Hubert" uniqKey="Schrezenmeier H" first="Hubert" last="Schrezenmeier">Hubert Schrezenmeier</name>
</country>
<country name="France"><region name="Île-de-France"><name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gerard" last="Socie">Gerard Socie</name>
</region>
</country>
<country name="Pays-Bas"><region name="Gueldre"><name sortKey="Muus, Petra" sort="Muus, Petra" uniqKey="Muus P" first="Petra" last="Muus">Petra Muus</name>
</region>
</country>
<country name="Australie"><region name="Victoria (État)"><name sortKey="Szer, Jeffrey" sort="Szer, Jeffrey" uniqKey="Szer J" first="Jeffrey" last="Szer">Jeffrey Szer</name>
</region>
</country>
<country name="Israël"><noRegion><name sortKey="Browne, Paul" sort="Browne, Paul" uniqKey="Browne P" first="Paul" last="Browne">Paul Browne</name>
</noRegion>
</country>
<country name="Italie"><noRegion><name sortKey="Risitano, Antonio M" sort="Risitano, Antonio M" uniqKey="Risitano A" first="Antonio M." last="Risitano">Antonio M. Risitano</name>
</noRegion>
<name sortKey="Luzzatto, Lucio" sort="Luzzatto, Lucio" uniqKey="Luzzatto L" first="Lucio" last="Luzzatto">Lucio Luzzatto</name>
</country>
</tree>
</affiliations>
</record>

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The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria

The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria

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