Efficacy and safety of Privigen® in patients with chronic inflammatory demyelinating polyneuropathy: results of a prospective, single‐arm, open‐label Phase III study (the PRIMA study)
Identifieur interne : 000679 ( Main/Exploration ); précédent : 000678; suivant : 000680Efficacy and safety of Privigen® in patients with chronic inflammatory demyelinating polyneuropathy: results of a prospective, single‐arm, open‐label Phase III study (the PRIMA study)
Auteurs : Jean-Marc Léger [France] ; Jan L. De Bleecker [Belgique] ; Claudia Sommer [Allemagne] ; Wim Robberecht [Belgique] ; Mika Saarela [Finlande] ; Jerzy Kamienowski [Pologne] ; Zbigniew Stelmasiak [Pologne] ; Orell Mielke [Allemagne] ; Björn Tackenberg [Allemagne] ; Amgad Shebl [Allemagne] ; Artur Bauhofer [Allemagne] ; Othmar Zenker [Allemagne] ; Ingemar S. J. Merkies [Pays-Bas]Source :
- Journal of the Peripheral Nervous System [ 1085-9489 ] ; 2013-06.
Abstract
This prospective, multicenter, single‐arm, open‐label Phase III study aimed to evaluate the efficacy and safety of Privigen® (10% liquid human intravenous immunoglobulin [IVIG], stabilized with l‐proline) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Patients received one induction dose of Privigen (2 g/kg body weight [bw]) and up to seven maintenance doses (1 g/kg bw) at 3‐week intervals. The primary efficacy endpoint was the responder rate at completion, defined as improvement of ≥1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale. The preset success criterion was the responder rate being ≥35%. Of the 31 screened patients, 28 patients were enrolled including 13 (46.4%) IVIG‐pretreated patients. The overall responder rate at completion was 60.7% (95% confidence interval [CI]: 42.41%–76.43%). IVIG‐pretreated patients demonstrated a higher responder rate than IVIG‐naïve patients (76.9% vs. 46.7%). The median (25%–75% quantile) INCAT score improved from 3.5 (3.0–4.5) points at baseline to 2.5 (1.0–3.0) points at completion, as did the mean (standard deviation [SD]) maximum grip strength (66.7 [37.24] kPa vs. 80.9 [31.06] kPa) and the median Medical Research Council sum score (67.0 [61.5–72.0] points vs. 75.5 [71.5–79.5] points). Of 108 adverse events (AEs; 0.417 AEs per infusion), 95 AEs (88.0%) were mild or moderate in intensity and resolved by the end of study. Two serious AEs of hemolysis were reported that resolved after discontinuation of treatment. Thus, Privigen provided efficacious and well‐tolerated induction and maintenance treatment in patients with CIDP.
Url:
DOI: 10.1111/jns5.12017
Affiliations:
- Allemagne, Belgique, Finlande, France, Pays-Bas, Pologne
- Bavière, District de Basse-Franconie, District de Giessen, Hesse (Land)
- Marbourg, Paris, Wurtzbourg
Links toward previous steps (curation, corpus...)
- to stream Istex, to step Corpus: 001172
- to stream Istex, to step Curation: 001148
- to stream Istex, to step Checkpoint: 000336
- to stream Main, to step Merge: 000680
- to stream Main, to step Curation: 000679
Le document en format XML
<record><TEI wicri:istexFullTextTei="biblStruct"><teiHeader><fileDesc><titleStmt><title xml:lang="en">Efficacy and safety of Privigen® in patients with chronic inflammatory demyelinating polyneuropathy: results of a prospective, single‐arm, open‐label Phase III study (the PRIMA study)</title><author><name sortKey="Leger, Jean Arc" sort="Leger, Jean Arc" uniqKey="Leger J" first="Jean-Marc" last="Léger">Jean-Marc Léger</name></author><author><name sortKey="De Bleecker, Jan L" sort="De Bleecker, Jan L" uniqKey="De Bleecker J" first="Jan L." last="De Bleecker">Jan L. De Bleecker</name></author><author><name sortKey="Sommer, Claudia" sort="Sommer, Claudia" uniqKey="Sommer C" first="Claudia" last="Sommer">Claudia Sommer</name></author><author><name sortKey="Robberecht, Wim" sort="Robberecht, Wim" uniqKey="Robberecht W" first="Wim" last="Robberecht">Wim Robberecht</name></author><author><name sortKey="Saarela, Mika" sort="Saarela, Mika" uniqKey="Saarela M" first="Mika" last="Saarela">Mika Saarela</name></author><author><name sortKey="Kamienowski, Jerzy" sort="Kamienowski, Jerzy" uniqKey="Kamienowski J" first="Jerzy" last="Kamienowski">Jerzy Kamienowski</name></author><author><name sortKey="Stelmasiak, Zbigniew" sort="Stelmasiak, Zbigniew" uniqKey="Stelmasiak Z" first="Zbigniew" last="Stelmasiak">Zbigniew Stelmasiak</name></author><author><name sortKey="Mielke, Orell" sort="Mielke, Orell" uniqKey="Mielke O" first="Orell" last="Mielke">Orell Mielke</name></author><author><name sortKey="Tackenberg, Bjorn" sort="Tackenberg, Bjorn" uniqKey="Tackenberg B" first="Björn" last="Tackenberg">Björn Tackenberg</name></author><author><name sortKey="Shebl, Amgad" sort="Shebl, Amgad" uniqKey="Shebl A" first="Amgad" last="Shebl">Amgad Shebl</name></author><author><name sortKey="Bauhofer, Artur" sort="Bauhofer, Artur" uniqKey="Bauhofer A" first="Artur" last="Bauhofer">Artur Bauhofer</name></author><author><name sortKey="Zenker, Othmar" sort="Zenker, Othmar" uniqKey="Zenker O" first="Othmar" last="Zenker">Othmar Zenker</name></author><author><name sortKey="Merkies, Ingemar S J" sort="Merkies, Ingemar S J" uniqKey="Merkies I" first="Ingemar S. J." last="Merkies">Ingemar S. J. Merkies</name></author></titleStmt><publicationStmt><idno type="wicri:source">ISTEX</idno><idno type="RBID">ISTEX:4499980138C56F9612808B836593D865DE2D44CA</idno><date when="2013" year="2013">2013</date><idno type="doi">10.1111/jns5.12017</idno><idno type="url">https://api.istex.fr/document/4499980138C56F9612808B836593D865DE2D44CA/fulltext/pdf</idno><idno type="wicri:Area/Istex/Corpus">001172</idno><idno type="wicri:Area/Istex/Curation">001148</idno><idno type="wicri:Area/Istex/Checkpoint">000336</idno><idno type="wicri:doubleKey">1085-9489:2013:Leger J:efficacy:and:safety</idno><idno type="wicri:Area/Main/Merge">000680</idno><idno type="wicri:Area/Main/Curation">000679</idno><idno type="wicri:Area/Main/Exploration">000679</idno></publicationStmt><sourceDesc><biblStruct><analytic><title level="a" type="main" xml:lang="en">Efficacy and safety of Privigen® in patients with chronic inflammatory demyelinating polyneuropathy: results of a prospective, single‐arm, open‐label Phase III study (the PRIMA study)</title><author><name sortKey="Leger, Jean Arc" sort="Leger, Jean Arc" uniqKey="Leger J" first="Jean-Marc" last="Léger">Jean-Marc Léger</name><affiliation wicri:level="1"><country xml:lang="fr">France</country><wicri:regionArea>Reference Center for Rare Neuromuscular Diseases, Hôpital Pitié‐Salpêtrière and University Paris VI, Paris</wicri:regionArea><placeName><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="De Bleecker, Jan L" sort="De Bleecker, Jan L" uniqKey="De Bleecker J" first="Jan L." last="De Bleecker">Jan L. De Bleecker</name><affiliation wicri:level="1"><country xml:lang="fr">Belgique</country><wicri:regionArea>AZ St‐Lucas, Gent</wicri:regionArea><wicri:noRegion>Gent</wicri:noRegion></affiliation></author><author><name sortKey="Sommer, Claudia" sort="Sommer, Claudia" uniqKey="Sommer C" first="Claudia" last="Sommer">Claudia Sommer</name><affiliation wicri:level="3"><country xml:lang="fr">Allemagne</country><wicri:regionArea>Department of Neurology, Universitätsklinikum Würzburg, Würzburg</wicri:regionArea><placeName><region type="land" nuts="1">Bavière</region><region type="district" nuts="2">District de Basse-Franconie</region><settlement type="city">Wurtzbourg</settlement></placeName></affiliation></author><author><name sortKey="Robberecht, Wim" sort="Robberecht, Wim" uniqKey="Robberecht W" first="Wim" last="Robberecht">Wim Robberecht</name><affiliation wicri:level="1"><country xml:lang="fr">Belgique</country><wicri:regionArea>UZ Leuven, Leuven</wicri:regionArea><wicri:noRegion>Leuven</wicri:noRegion></affiliation></author><author><name sortKey="Saarela, Mika" sort="Saarela, Mika" uniqKey="Saarela M" first="Mika" last="Saarela">Mika Saarela</name><affiliation wicri:level="1"><country xml:lang="fr">Finlande</country><wicri:regionArea>Department of Neurology, Helsinki University Central Hospital, Helsinki</wicri:regionArea><wicri:noRegion>Helsinki</wicri:noRegion></affiliation></author><author><name sortKey="Kamienowski, Jerzy" sort="Kamienowski, Jerzy" uniqKey="Kamienowski J" first="Jerzy" last="Kamienowski">Jerzy Kamienowski</name><affiliation wicri:level="1"><country xml:lang="fr">Pologne</country><wicri:regionArea>Dolnośląski Szpital Specjalistyczny, Wrocław</wicri:regionArea><wicri:noRegion>Wrocław</wicri:noRegion></affiliation></author><author><name sortKey="Stelmasiak, Zbigniew" sort="Stelmasiak, Zbigniew" uniqKey="Stelmasiak Z" first="Zbigniew" last="Stelmasiak">Zbigniew Stelmasiak</name><affiliation wicri:level="1"><country xml:lang="fr">Pologne</country><wicri:regionArea>Samodzielny Publiczny Szpital Kliniczny, Lublin</wicri:regionArea><wicri:noRegion>Lublin</wicri:noRegion></affiliation></author><author><name sortKey="Mielke, Orell" sort="Mielke, Orell" uniqKey="Mielke O" first="Orell" last="Mielke">Orell Mielke</name><affiliation wicri:level="3"><country xml:lang="fr">Allemagne</country><wicri:regionArea>CSL Behring GmbH, Marburg</wicri:regionArea><placeName><region type="land" nuts="1">Hesse (Land)</region><region type="district" nuts="2">District de Giessen</region><settlement type="city">Marbourg</settlement></placeName></affiliation></author><author><name sortKey="Tackenberg, Bjorn" sort="Tackenberg, Bjorn" uniqKey="Tackenberg B" first="Björn" last="Tackenberg">Björn Tackenberg</name><affiliation wicri:level="3"><country xml:lang="fr">Allemagne</country><wicri:regionArea>Department of Neurology, Philipps University, Marburg</wicri:regionArea><placeName><region type="land" nuts="1">Hesse (Land)</region><region type="district" nuts="2">District de Giessen</region><settlement type="city">Marbourg</settlement></placeName></affiliation></author><author><name sortKey="Shebl, Amgad" sort="Shebl, Amgad" uniqKey="Shebl A" first="Amgad" last="Shebl">Amgad Shebl</name><affiliation wicri:level="3"><country xml:lang="fr">Allemagne</country><wicri:regionArea>CSL Behring GmbH, Marburg</wicri:regionArea><placeName><region type="land" nuts="1">Hesse (Land)</region><region type="district" nuts="2">District de Giessen</region><settlement type="city">Marbourg</settlement></placeName></affiliation></author><author><name sortKey="Bauhofer, Artur" sort="Bauhofer, Artur" uniqKey="Bauhofer A" first="Artur" last="Bauhofer">Artur Bauhofer</name><affiliation wicri:level="3"><country xml:lang="fr">Allemagne</country><wicri:regionArea>CSL Behring GmbH, Marburg</wicri:regionArea><placeName><region type="land" nuts="1">Hesse (Land)</region><region type="district" nuts="2">District de Giessen</region><settlement type="city">Marbourg</settlement></placeName></affiliation></author><author><name sortKey="Zenker, Othmar" sort="Zenker, Othmar" uniqKey="Zenker O" first="Othmar" last="Zenker">Othmar Zenker</name><affiliation wicri:level="3"><country xml:lang="fr">Allemagne</country><wicri:regionArea>CSL Behring GmbH, Marburg</wicri:regionArea><placeName><region type="land" nuts="1">Hesse (Land)</region><region type="district" nuts="2">District de Giessen</region><settlement type="city">Marbourg</settlement></placeName></affiliation></author><author><name sortKey="Merkies, Ingemar S J" sort="Merkies, Ingemar S J" uniqKey="Merkies I" first="Ingemar S. J." last="Merkies">Ingemar S. J. Merkies</name><affiliation wicri:level="1"><country xml:lang="fr">Pays-Bas</country><wicri:regionArea>Spaarne Hospital, Hoofddorp and Maastricht University Medical Centre, Maastricht</wicri:regionArea><wicri:noRegion>Maastricht</wicri:noRegion></affiliation></author></analytic><monogr></monogr><series><title level="j">Journal of the Peripheral Nervous System</title><title level="j" type="abbrev">J Peripher Nerv Syst</title><idno type="ISSN">1085-9489</idno><idno type="eISSN">1529-8027</idno><imprint><publisher>Wiley Periodicals, Inc.</publisher><pubPlace>Malden, USA</pubPlace><date type="published" when="2013-06">2013-06</date><biblScope unit="volume">18</biblScope><biblScope unit="issue">2</biblScope><biblScope unit="page" from="130">130</biblScope><biblScope unit="page" to="140">140</biblScope></imprint><idno type="ISSN">1085-9489</idno></series><idno type="istex">4499980138C56F9612808B836593D865DE2D44CA</idno><idno type="DOI">10.1111/jns5.12017</idno><idno type="ArticleID">JNS512017</idno></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">1085-9489</idno></seriesStmt></fileDesc><profileDesc><textClass></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="abstract">This prospective, multicenter, single‐arm, open‐label Phase III study aimed to evaluate the efficacy and safety of Privigen® (10% liquid human intravenous immunoglobulin [IVIG], stabilized with l‐proline) in patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Patients received one induction dose of Privigen (2 g/kg body weight [bw]) and up to seven maintenance doses (1 g/kg bw) at 3‐week intervals. The primary efficacy endpoint was the responder rate at completion, defined as improvement of ≥1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale. The preset success criterion was the responder rate being ≥35%. Of the 31 screened patients, 28 patients were enrolled including 13 (46.4%) IVIG‐pretreated patients. The overall responder rate at completion was 60.7% (95% confidence interval [CI]: 42.41%–76.43%). IVIG‐pretreated patients demonstrated a higher responder rate than IVIG‐naïve patients (76.9% vs. 46.7%). The median (25%–75% quantile) INCAT score improved from 3.5 (3.0–4.5) points at baseline to 2.5 (1.0–3.0) points at completion, as did the mean (standard deviation [SD]) maximum grip strength (66.7 [37.24] kPa vs. 80.9 [31.06] kPa) and the median Medical Research Council sum score (67.0 [61.5–72.0] points vs. 75.5 [71.5–79.5] points). Of 108 adverse events (AEs; 0.417 AEs per infusion), 95 AEs (88.0%) were mild or moderate in intensity and resolved by the end of study. Two serious AEs of hemolysis were reported that resolved after discontinuation of treatment. Thus, Privigen provided efficacious and well‐tolerated induction and maintenance treatment in patients with CIDP.</div></front></TEI><affiliations><list><country><li>Allemagne</li><li>Belgique</li><li>Finlande</li><li>France</li><li>Pays-Bas</li><li>Pologne</li></country><region><li>Bavière</li><li>District de Basse-Franconie</li><li>District de Giessen</li><li>Hesse (Land)</li></region><settlement><li>Marbourg</li><li>Paris</li><li>Wurtzbourg</li></settlement></list><tree><country name="France"><noRegion><name sortKey="Leger, Jean Arc" sort="Leger, Jean Arc" uniqKey="Leger J" first="Jean-Marc" last="Léger">Jean-Marc Léger</name></noRegion></country><country name="Belgique"><noRegion><name sortKey="De Bleecker, Jan L" sort="De Bleecker, Jan L" uniqKey="De Bleecker J" first="Jan L." last="De Bleecker">Jan L. De Bleecker</name></noRegion><name sortKey="Robberecht, Wim" sort="Robberecht, Wim" uniqKey="Robberecht W" first="Wim" last="Robberecht">Wim Robberecht</name></country><country name="Allemagne"><region name="Bavière"><name sortKey="Sommer, Claudia" sort="Sommer, Claudia" uniqKey="Sommer C" first="Claudia" last="Sommer">Claudia Sommer</name></region><name sortKey="Bauhofer, Artur" sort="Bauhofer, Artur" uniqKey="Bauhofer A" first="Artur" last="Bauhofer">Artur Bauhofer</name><name sortKey="Mielke, Orell" sort="Mielke, Orell" uniqKey="Mielke O" first="Orell" last="Mielke">Orell Mielke</name><name sortKey="Shebl, Amgad" sort="Shebl, Amgad" uniqKey="Shebl A" first="Amgad" last="Shebl">Amgad Shebl</name><name sortKey="Tackenberg, Bjorn" sort="Tackenberg, Bjorn" uniqKey="Tackenberg B" first="Björn" last="Tackenberg">Björn Tackenberg</name><name sortKey="Zenker, Othmar" sort="Zenker, Othmar" uniqKey="Zenker O" first="Othmar" last="Zenker">Othmar Zenker</name></country><country name="Finlande"><noRegion><name sortKey="Saarela, Mika" sort="Saarela, Mika" uniqKey="Saarela M" first="Mika" last="Saarela">Mika Saarela</name></noRegion></country><country name="Pologne"><noRegion><name sortKey="Kamienowski, Jerzy" sort="Kamienowski, Jerzy" uniqKey="Kamienowski J" first="Jerzy" last="Kamienowski">Jerzy Kamienowski</name></noRegion><name sortKey="Stelmasiak, Zbigniew" sort="Stelmasiak, Zbigniew" uniqKey="Stelmasiak Z" first="Zbigniew" last="Stelmasiak">Zbigniew Stelmasiak</name></country><country name="Pays-Bas"><noRegion><name sortKey="Merkies, Ingemar S J" sort="Merkies, Ingemar S J" uniqKey="Merkies I" first="Ingemar S. J." last="Merkies">Ingemar S. J. Merkies</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
EXPLOR_STEP=$WICRI_ROOT/Wicri/Belgique/explor/OpenAccessBelV2/Data/Main/Exploration
HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 000679 | SxmlIndent | more
Ou
HfdSelect -h $EXPLOR_AREA/Data/Main/Exploration/biblio.hfd -nk 000679 | SxmlIndent | more
Pour mettre un lien sur cette page dans le réseau Wicri
{{Explor lien
|wiki= Wicri/Belgique
|area= OpenAccessBelV2
|flux= Main
|étape= Exploration
|type= RBID
|clé= ISTEX:4499980138C56F9612808B836593D865DE2D44CA
|texte= Efficacy and safety of Privigen® in patients with chronic inflammatory demyelinating polyneuropathy: results of a prospective, single‐arm, open‐label Phase III study (the PRIMA study)
}}
| This area was generated with Dilib version V0.6.25. |  |