AustralieFrV1, PascalFrancis, Curation, bibRecord, 005517

Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria

Identifieur interne : 005517 ( PascalFrancis/Curation ); précédent : 005516; suivant : 005518

Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria

Auteurs : Peter Hillmen [Royaume-Uni] ; Petra Muus [Pays-Bas] ; Alexander Röth [Allemagne] ; Modupe O. Elebute [Royaume-Uni] ; Antonio M. Risitano [Italie] ; Hubert Schrezenmeier [Allemagne] ; Jeffrey Szer [Australie] ; Paul Browne [Irlande (pays)] ; Jaroslaw P. Maciejewski [États-Unis] ; Jörg Schubert [Allemagne] ; Alvaro Urbano-Ispizua [Espagne] ; Carlos De Castro [États-Unis] ; Gérard Socie [France] ; Robert A. Brodsky [États-Unis]

Source :

British journal of haematology [ 0007-1048 ] ; 2013.

RBID : Pascal:13-0227704

Descripteurs français

Pascal (Inist)
- Eculizumab, Long terme, Efficacité traitement, Anémie hémolytique de Marchiafava-Micheli, Traitement, Homme, Hémolyse, Cancérologie, Hématologie.
Wicri :
- topic : Homme.

English descriptors

KwdEn :
- Cancerology, Eculizumab, Hematology, Hemolysis, Human, Long term, Nocturnal paroxystic anemia, Treatment, Treatment efficiency.

Abstract

Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by chronic, uncontrolled complement activation resulting in elevated intravascular haemolysis and morbidities, including fatigue, dyspnoea, abdominal pain, pulmonary hypertension, thrombotic events (TEs) and chronic kidney disease (CKD). The long-term safety and efficacy of eculizumab, a humanized monoclonal antibody that inhibits terminal complement activation, was investigated in 195 patients over 66 months. Four patient deaths were reported, all unrelated to treatment, resulting in a 3-year survival estimate of 97.6%. All patients showed a reduction in lactate dehydrogenase levels, which was sustained over the course of treatment (median reduction of 86.9% at 36 months), reflecting inhibition of chronic haemolysis. TEs decreased by 81.8%, with 96.4% of patients remaining free of TEs. Patients also showed a time-dependent improvement in renal function: 93.1% of patients exhibited improvement or stabilization in CKD score at 36 months. Transfusion independence increased by 90.0% from baseline, with the number of red blood cell units transfused decreasing by 54.7%. Eculizumab was well tolerated, with no evidence of cumulative toxicity and a decreasing occurrence of adverse events over time. Eculizumab has a substantial impact on the symptoms and complications of PNH and results a significant improvement in patient survival.

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A08	`01`	`1`	`ENG`	`@1 Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria`
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A11	`12`	`1`		`@1 DE CASTRO (Carlos)`
A11	`13`	`1`		`@1 SOCIE (Gérard)`
A11	`14`	`1`		`@1 BRODSKY (Robert A.)`
A14	`01`			`@1 St James's University Hospital @2 Leeds @3 GBR @Z 1 aut.`
A14	`02`			`@1 Radboud University Nijmegen Medical Centre @2 Nijmegen @3 NLD @Z 2 aut.`
A14	`03`			`@1 University of Duisburg-Essen @2 Essen @3 DEU @Z 3 aut.`
A14	`04`			`@1 Department of Haematological Medicine, King's College Hospital @2 London @3 GBR @Z 4 aut.`
A14	`05`			`@1 Federico II University @2 Naples @3 ITA @Z 5 aut.`
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A14	`12`			`@1 Duke University Medical Center @2 Durham, NC @3 USA @Z 12 aut.`
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C01	`01`		`ENG`	@0 Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by chronic, uncontrolled complement activation resulting in elevated intravascular haemolysis and morbidities, including fatigue, dyspnoea, abdominal pain, pulmonary hypertension, thrombotic events (TEs) and chronic kidney disease (CKD). The long-term safety and efficacy of eculizumab, a humanized monoclonal antibody that inhibits terminal complement activation, was investigated in 195 patients over 66 months. Four patient deaths were reported, all unrelated to treatment, resulting in a 3-year survival estimate of 97.6%. All patients showed a reduction in lactate dehydrogenase levels, which was sustained over the course of treatment (median reduction of 86.9% at 36 months), reflecting inhibition of chronic haemolysis. TEs decreased by 81.8%, with 96.4% of patients remaining free of TEs. Patients also showed a time-dependent improvement in renal function: 93.1% of patients exhibited improvement or stabilization in CKD score at 36 months. Transfusion independence increased by 90.0% from baseline, with the number of red blood cell units transfused decreasing by 54.7%. Eculizumab was well tolerated, with no evidence of cumulative toxicity and a decreasing occurrence of adverse events over time. Eculizumab has a substantial impact on the symptoms and complications of PNH and results a significant improvement in patient survival.
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C07	`02`	`X`	`FRE`	`@0 Anomalie de la membrane érythrocytaire @5 38`
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C07	`02`	`X`	`SPA`	`@0 Anomalía membrana hematía @5 38`
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N44	`01`			`@1 OTO`
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Le document en format XML

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<author><name sortKey="Hillmen, Peter" sort="Hillmen, Peter" uniqKey="Hillmen P" first="Peter" last="Hillmen">Peter Hillmen</name>
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<author><name sortKey="Elebute, Modupe O" sort="Elebute, Modupe O" uniqKey="Elebute M" first="Modupe O." last="Elebute">Modupe O. Elebute</name>
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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="Schrezenmeier, Hubert" sort="Schrezenmeier, Hubert" uniqKey="Schrezenmeier H" first="Hubert" last="Schrezenmeier">Hubert Schrezenmeier</name>
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<author><name sortKey="Szer, Jeffrey" sort="Szer, Jeffrey" uniqKey="Szer J" first="Jeffrey" last="Szer">Jeffrey Szer</name>
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<author><name sortKey="Browne, Paul" sort="Browne, Paul" uniqKey="Browne P" first="Paul" last="Browne">Paul Browne</name>
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<author><name sortKey="Maciejewski, Jaroslaw P" sort="Maciejewski, Jaroslaw P" uniqKey="Maciejewski J" first="Jaroslaw P." last="Maciejewski">Jaroslaw P. Maciejewski</name>
<affiliation wicri:level="1"><inist:fA14 i1="09"><s1>Taussig Cancer Center, Cleveland Clinic</s1>
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<author><name sortKey="Schubert, Jorg" sort="Schubert, Jorg" uniqKey="Schubert J" first="Jörg" last="Schubert">Jörg Schubert</name>
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<author><name sortKey="Urbano Ispizua, Alvaro" sort="Urbano Ispizua, Alvaro" uniqKey="Urbano Ispizua A" first="Alvaro" last="Urbano-Ispizua">Alvaro Urbano-Ispizua</name>
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<author><name sortKey="De Castro, Carlos" sort="De Castro, Carlos" uniqKey="De Castro C" first="Carlos" last="De Castro">Carlos De Castro</name>
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<author><name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socie">Gérard Socie</name>
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<s2>Paris</s2>
<s3>FRA</s3>
<sZ>13 aut.</sZ>
</inist:fA14>
<country>France</country>
</affiliation>
</author>
<author><name sortKey="Brodsky, Robert A" sort="Brodsky, Robert A" uniqKey="Brodsky R" first="Robert A." last="Brodsky">Robert A. Brodsky</name>
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<s2>Baltimore, MD</s2>
<s3>USA</s3>
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<country>États-Unis</country>
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<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria</title>
<author><name sortKey="Hillmen, Peter" sort="Hillmen, Peter" uniqKey="Hillmen P" first="Peter" last="Hillmen">Peter Hillmen</name>
<affiliation wicri:level="1"><inist:fA14 i1="01"><s1>St James's University Hospital</s1>
<s2>Leeds</s2>
<s3>GBR</s3>
<sZ>1 aut.</sZ>
</inist:fA14>
<country>Royaume-Uni</country>
</affiliation>
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<author><name sortKey="Muus, Petra" sort="Muus, Petra" uniqKey="Muus P" first="Petra" last="Muus">Petra Muus</name>
<affiliation wicri:level="1"><inist:fA14 i1="02"><s1>Radboud University Nijmegen Medical Centre</s1>
<s2>Nijmegen</s2>
<s3>NLD</s3>
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</inist:fA14>
<country>Pays-Bas</country>
</affiliation>
</author>
<author><name sortKey="Roth, Alexander" sort="Roth, Alexander" uniqKey="Roth A" first="Alexander" last="Röth">Alexander Röth</name>
<affiliation wicri:level="1"><inist:fA14 i1="03"><s1>University of Duisburg-Essen</s1>
<s2>Essen</s2>
<s3>DEU</s3>
<sZ>3 aut.</sZ>
</inist:fA14>
<country>Allemagne</country>
</affiliation>
</author>
<author><name sortKey="Elebute, Modupe O" sort="Elebute, Modupe O" uniqKey="Elebute M" first="Modupe O." last="Elebute">Modupe O. Elebute</name>
<affiliation wicri:level="1"><inist:fA14 i1="04"><s1>Department of Haematological Medicine, King's College Hospital</s1>
<s2>London</s2>
<s3>GBR</s3>
<sZ>4 aut.</sZ>
</inist:fA14>
<country>Royaume-Uni</country>
</affiliation>
</author>
<author><name sortKey="Risitano, Antonio M" sort="Risitano, Antonio M" uniqKey="Risitano A" first="Antonio M." last="Risitano">Antonio M. Risitano</name>
<affiliation wicri:level="1"><inist:fA14 i1="05"><s1>Federico II University</s1>
<s2>Naples</s2>
<s3>ITA</s3>
<sZ>5 aut.</sZ>
</inist:fA14>
<country>Italie</country>
</affiliation>
</author>
<author><name sortKey="Schrezenmeier, Hubert" sort="Schrezenmeier, Hubert" uniqKey="Schrezenmeier H" first="Hubert" last="Schrezenmeier">Hubert Schrezenmeier</name>
<affiliation wicri:level="1"><inist:fA14 i1="06"><s1>Institute of Clinical Transfusion Medicine and Immunogenetics, German Red Cross Blood Transfusion Service and Institute of Transfusion Medicine, University of Ulm</s1>
<s2>Ulm</s2>
<s3>DEU</s3>
<sZ>6 aut.</sZ>
</inist:fA14>
<country>Allemagne</country>
</affiliation>
</author>
<author><name sortKey="Szer, Jeffrey" sort="Szer, Jeffrey" uniqKey="Szer J" first="Jeffrey" last="Szer">Jeffrey Szer</name>
<affiliation wicri:level="1"><inist:fA14 i1="07"><s1>Royal Melbourne Hospital</s1>
<s2>Melbourne</s2>
<s3>AUS</s3>
<sZ>7 aut.</sZ>
</inist:fA14>
<country>Australie</country>
</affiliation>
</author>
<author><name sortKey="Browne, Paul" sort="Browne, Paul" uniqKey="Browne P" first="Paul" last="Browne">Paul Browne</name>
<affiliation wicri:level="1"><inist:fA14 i1="08"><s1>St. James's Hospital, Trinity College Dublin</s1>
<s2>Dublin</s2>
<s3>IRL</s3>
<sZ>8 aut.</sZ>
</inist:fA14>
<country>Irlande (pays)</country>
</affiliation>
</author>
<author><name sortKey="Maciejewski, Jaroslaw P" sort="Maciejewski, Jaroslaw P" uniqKey="Maciejewski J" first="Jaroslaw P." last="Maciejewski">Jaroslaw P. Maciejewski</name>
<affiliation wicri:level="1"><inist:fA14 i1="09"><s1>Taussig Cancer Center, Cleveland Clinic</s1>
<s2>Cleveland, OH</s2>
<s3>USA</s3>
<sZ>9 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
</affiliation>
</author>
<author><name sortKey="Schubert, Jorg" sort="Schubert, Jorg" uniqKey="Schubert J" first="Jörg" last="Schubert">Jörg Schubert</name>
<affiliation wicri:level="1"><inist:fA14 i1="10"><s1>Saarland University Medical School</s1>
<s2>Homburg-Saarland</s2>
<s3>DEU</s3>
<sZ>10 aut.</sZ>
</inist:fA14>
<country>Allemagne</country>
</affiliation>
</author>
<author><name sortKey="Urbano Ispizua, Alvaro" sort="Urbano Ispizua, Alvaro" uniqKey="Urbano Ispizua A" first="Alvaro" last="Urbano-Ispizua">Alvaro Urbano-Ispizua</name>
<affiliation wicri:level="1"><inist:fA14 i1="11"><s1>Grupo de Trabajo de HPN de la Sociedad Española de Hematologia y Hemoterapia</s1>
<s2>Barcelona</s2>
<s3>ESP</s3>
<sZ>11 aut.</sZ>
</inist:fA14>
<country>Espagne</country>
</affiliation>
</author>
<author><name sortKey="De Castro, Carlos" sort="De Castro, Carlos" uniqKey="De Castro C" first="Carlos" last="De Castro">Carlos De Castro</name>
<affiliation wicri:level="1"><inist:fA14 i1="12"><s1>Duke University Medical Center</s1>
<s2>Durham, NC</s2>
<s3>USA</s3>
<sZ>12 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
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<author><name sortKey="Socie, Gerard" sort="Socie, Gerard" uniqKey="Socie G" first="Gérard" last="Socie">Gérard Socie</name>
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<author><name sortKey="Brodsky, Robert A" sort="Brodsky, Robert A" uniqKey="Brodsky R" first="Robert A." last="Brodsky">Robert A. Brodsky</name>
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<series><title level="j" type="main">British journal of haematology</title>
<title level="j" type="abbreviated">Br. j. haematol.</title>
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<front><div type="abstract" xml:lang="en">Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by chronic, uncontrolled complement activation resulting in elevated intravascular haemolysis and morbidities, including fatigue, dyspnoea, abdominal pain, pulmonary hypertension, thrombotic events (TEs) and chronic kidney disease (CKD). The long-term safety and efficacy of eculizumab, a humanized monoclonal antibody that inhibits terminal complement activation, was investigated in 195 patients over 66 months. Four patient deaths were reported, all unrelated to treatment, resulting in a 3-year survival estimate of 97.6%. All patients showed a reduction in lactate dehydrogenase levels, which was sustained over the course of treatment (median reduction of 86.9% at 36 months), reflecting inhibition of chronic haemolysis. TEs decreased by 81.8%, with 96.4% of patients remaining free of TEs. Patients also showed a time-dependent improvement in renal function: 93.1% of patients exhibited improvement or stabilization in CKD score at 36 months. Transfusion independence increased by 90.0% from baseline, with the number of red blood cell units transfused decreasing by 54.7%. Eculizumab was well tolerated, with no evidence of cumulative toxicity and a decreasing occurrence of adverse events over time. Eculizumab has a substantial impact on the symptoms and complications of PNH and results a significant improvement in patient survival.</div>
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Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria

Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria

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