Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study
Identifieur interne : 000876 ( Main/Exploration ); précédent : 000875; suivant : 000877Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study
Auteurs : Aleksandar Sekulic [États-Unis] ; Michael R. Migden [États-Unis] ; Nicole Basset-Seguin [France] ; Claus Garbe [Allemagne] ; Anja Gesierich [Allemagne] ; Christopher D. Lao [États-Unis] ; Chris Miller [États-Unis] ; Laurent Mortier [France] ; Dedee F. Murrell [Australie] ; Omid Hamid [États-Unis] ; Jorge F. Quevedo [États-Unis] ; Jeannie Hou [États-Unis] ; Edward Mckenna [États-Unis] ; Natalie Dimier ; Sarah Williams ; Dirk Schadendorf [Allemagne] ; Axel Hauschild [Allemagne]Source :
- BMC Cancer [ 1471-2407 ] ; 2017.
Abstract
In the primary analysis of the ERIVANCE BCC trial, vismodegib, the first US Food and Drug Administration–approved Hedgehog pathway inhibitor, showed objective response rates (ORRs) by independent review facility (IRF) of 30% and 43% in metastatic basal cell carcinoma (mBCC) and locally advanced BCC (laBCC), respectively. ORRs by investigator review were 45% (mBCC) and 60% (laBCC). Herein, we present long-term safety and final investigator-assessed efficacy results in patients with mBCC or laBCC.
One hundred four patients with measurable advanced BCC received oral vismodegib 150 mg once daily until disease progression or intolerable toxicity. The primary end point was IRF-assessed ORR. Secondary end points included ORR, duration of response (DOR), progression-free survival, overall survival (OS), and safety.
At data cutoff (39 months after completion of accrual), 8 patients were receiving the study drug (69 patients in survival follow-up). Investigator-assessed ORR was 48.5% in the mBCC group (all partial responses) and 60.3% in the laBCC group (20 patients had complete response and 18 patients had partial response). ORRs were comparable across patient subgroups, including aggressive histologic subtypes (eg, infiltrative BCC). Median DOR was 14.8 months (mBCC) and 26.2 months (laBCC). Median OS was 33.4 months in the mBCC cohort and not estimable in the laBCC cohort. Adverse events remained consistent with clinical experience. Thirty-three deaths (31.7%) were reported; none were related to vismodegib.
This long-term update of the ERIVANCE BCC trial demonstrated durability of response, efficacy across patient subgroups, and manageable long-term safety of vismodegib in patients with advanced BCC.
This study was registered prospectively with
The online version of this article (doi:10.1186/s12885-017-3286-5) contains supplementary material, which is available to authorized users.
Url:
DOI: 10.1186/s12885-017-3286-5
PubMed: 28511673
PubMed Central: 5433030
Affiliations:
- Allemagne, Australie, France, États-Unis
- Arizona, Bade-Wurtemberg, Bavière, Californie, District de Basse-Franconie, District de Tübingen, Hauts-de-France, Michigan, Minnesota, Nord-Pas-de-Calais, Nouvelle-Galles du Sud, Pennsylvanie, Texas, Île-de-France
- Lille, Paris, Sydney, Tübingen, Wurtzbourg
Links toward previous steps (curation, corpus...)
- to stream Pmc, to step Corpus: 000A58
- to stream Pmc, to step Curation: 000A35
- to stream Pmc, to step Checkpoint: 000218
- to stream PubMed, to step Corpus: 000C80
- to stream PubMed, to step Curation: 000C77
- to stream PubMed, to step Checkpoint: 000C77
- to stream Ncbi, to step Merge: 004826
- to stream Ncbi, to step Curation: 004826
- to stream Ncbi, to step Checkpoint: 004826
- to stream Main, to step Merge: 000871
- to stream Main, to step Curation: 000876
Le document en format XML
<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study</title><author><name sortKey="Sekulic, Aleksandar" sort="Sekulic, Aleksandar" uniqKey="Sekulic A" first="Aleksandar" last="Sekulic">Aleksandar Sekulic</name><affiliation wicri:level="2"><nlm:aff id="Aff1"><institution-wrap><institution-id institution-id-type="ISNI">0000 0000 8875 6339</institution-id><institution-id institution-id-type="GRID">grid.417468.8</institution-id><institution></institution><institution>Mayo Clinic Scottsdale,</institution></institution-wrap>13400 East Shea Boulevard, Scottsdale, AZ 85259 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Arizona</region></placeName><wicri:cityArea>13400 East Shea Boulevard, Scottsdale</wicri:cityArea></affiliation></author><author><name sortKey="Migden, Michael R" sort="Migden, Michael R" uniqKey="Migden M" first="Michael R." last="Migden">Michael R. Migden</name><affiliation wicri:level="2"><nlm:aff id="Aff2"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 2291 4776</institution-id><institution-id institution-id-type="GRID">grid.240145.6</institution-id><institution>Departments of Dermatology and Head and Neck Surgery,</institution><institution>The University of Texas MD Anderson Cancer Center,</institution></institution-wrap>1400 Pressler Street, Houston, TX 77030 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Texas</region></placeName><wicri:cityArea>1400 Pressler Street, Houston</wicri:cityArea></affiliation></author><author><name sortKey="Basset Seguin, Nicole" sort="Basset Seguin, Nicole" uniqKey="Basset Seguin N" first="Nicole" last="Basset-Seguin">Nicole Basset-Seguin</name><affiliation wicri:level="1"><nlm:aff id="Aff3"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 2300 6614</institution-id><institution-id institution-id-type="GRID">grid.413328.f</institution-id><institution>Service de Dermatologie,</institution><institution>Hôpital Saint-Louis,</institution></institution-wrap>1 av claude Vellefaux, 75010 Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>1 av claude Vellefaux, 75010 Paris</wicri:regionArea><wicri:noRegion>75010 Paris</wicri:noRegion><placeName><settlement type="city">Paris</settlement><region type="région" nuts="2">Île-de-France</region></placeName></affiliation></author><author><name sortKey="Garbe, Claus" sort="Garbe, Claus" uniqKey="Garbe C" first="Claus" last="Garbe">Claus Garbe</name><affiliation wicri:level="3"><nlm:aff id="Aff4"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 0196 8249</institution-id><institution-id institution-id-type="GRID">grid.411544.1</institution-id><institution>Studienzentrum Dermatologische Onkologie,</institution><institution>Universitätsklinikum Tübingen,</institution></institution-wrap>Liebermeisterstr. 25, 72074 Tübingen, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Liebermeisterstr. 25, 72074 Tübingen</wicri:regionArea><placeName><region type="land" nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Tübingen</region><settlement type="city">Tübingen</settlement></placeName></affiliation></author><author><name sortKey="Gesierich, Anja" sort="Gesierich, Anja" uniqKey="Gesierich A" first="Anja" last="Gesierich">Anja Gesierich</name><affiliation wicri:level="3"><nlm:aff id="Aff5"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 1378 7891</institution-id><institution-id institution-id-type="GRID">grid.411760.5</institution-id><institution>Klinik für Dermatologie, Venerologie und Allergologie,</institution><institution>Universitätsklinikum Würzburg,</institution></institution-wrap>Josef-Schneider-Str. 2, 97080 Würzburg, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Josef-Schneider-Str. 2, 97080 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="Lao, Christopher D" sort="Lao, Christopher D" uniqKey="Lao C" first="Christopher D." last="Lao">Christopher D. Lao</name><affiliation wicri:level="2"><nlm:aff id="Aff6">University of Michigan, 1500 East Medical Center Drive, Ann Arbor, MI 48109 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Michigan</region></placeName><wicri:cityArea>University of Michigan, 1500 East Medical Center Drive, Ann Arbor</wicri:cityArea></affiliation></author><author><name sortKey="Miller, Chris" sort="Miller, Chris" uniqKey="Miller C" first="Chris" last="Miller">Chris Miller</name><affiliation wicri:level="2"><nlm:aff id="Aff7"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0435 1019</institution-id><institution-id institution-id-type="GRID">grid.412713.2</institution-id><institution>Department of Dermatology,</institution><institution>University of Pennsylvania Medical Center,</institution></institution-wrap>3400 Civic Center Boulevard, Philadelphia, PA 19104 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName><wicri:cityArea>3400 Civic Center Boulevard, Philadelphia</wicri:cityArea></affiliation></author><author><name sortKey="Mortier, Laurent" sort="Mortier, Laurent" uniqKey="Mortier L" first="Laurent" last="Mortier">Laurent Mortier</name><affiliation wicri:level="3"><nlm:aff id="Aff8"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0639 4004</institution-id><institution-id institution-id-type="GRID">grid.413875.c</institution-id><institution>Clinique de Dermatologie,</institution><institution>Hôpital Claude Huriez,</institution></institution-wrap>Inserm U1189, Lille, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Inserm U1189, Lille</wicri:regionArea><placeName><region type="region">Hauts-de-France</region><region type="old region">Nord-Pas-de-Calais</region><settlement type="city">Lille</settlement></placeName></affiliation></author><author><name sortKey="Murrell, Dedee F" sort="Murrell, Dedee F" uniqKey="Murrell D" first="Dedee F." last="Murrell">Dedee F. Murrell</name><affiliation wicri:level="3"><nlm:aff id="Aff9"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 4902 0432</institution-id><institution-id institution-id-type="GRID">grid.1005.4</institution-id><institution>Dermatology Department,</institution><institution>St George Clinical School, University of New South Wales,</institution></institution-wrap>Grey Street, Sydney, 2217 Australia</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>Grey Street, Sydney</wicri:regionArea><placeName><settlement type="city">Sydney</settlement><region type="état">Nouvelle-Galles du Sud</region></placeName></affiliation></author><author><name sortKey="Hamid, Omid" sort="Hamid, Omid" uniqKey="Hamid O" first="Omid" last="Hamid">Omid Hamid</name><affiliation wicri:level="2"><nlm:aff id="Aff10">The Angeles Clinic and Research Institute, 1818 Wilshire Boulevard, Los Angeles, California USA</nlm:aff><country>États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>The Angeles Clinic and Research Institute, 1818 Wilshire Boulevard, Los Angeles</wicri:cityArea></affiliation></author><author><name sortKey="Quevedo, Jorge F" sort="Quevedo, Jorge F" uniqKey="Quevedo J" first="Jorge F." last="Quevedo">Jorge F. Quevedo</name><affiliation wicri:level="2"><nlm:aff id="Aff11"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0459 167X</institution-id><institution-id institution-id-type="GRID">grid.66875.3a</institution-id><institution></institution><institution>Mayo Clinic,</institution></institution-wrap>200 First Street SW, Rochester, MN 55905 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Minnesota</region></placeName><wicri:cityArea>200 First Street SW, Rochester</wicri:cityArea></affiliation></author><author><name sortKey="Hou, Jeannie" sort="Hou, Jeannie" uniqKey="Hou J" first="Jeannie" last="Hou">Jeannie Hou</name><affiliation wicri:level="2"><nlm:aff id="Aff12"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0534 4718</institution-id><institution-id institution-id-type="GRID">grid.418158.1</institution-id><institution></institution><institution>Genentech, Inc.,</institution></institution-wrap>1 DNA Way, South San Francisco, California 94080 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>1 DNA Way, South San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Mckenna, Edward" sort="Mckenna, Edward" uniqKey="Mckenna E" first="Edward" last="Mckenna">Edward Mckenna</name><affiliation wicri:level="2"><nlm:aff id="Aff12"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0534 4718</institution-id><institution-id institution-id-type="GRID">grid.418158.1</institution-id><institution></institution><institution>Genentech, Inc.,</institution></institution-wrap>1 DNA Way, South San Francisco, California 94080 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>1 DNA Way, South San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Dimier, Natalie" sort="Dimier, Natalie" uniqKey="Dimier N" first="Natalie" last="Dimier">Natalie Dimier</name><affiliation><nlm:aff id="Aff13">Roche Products Limited, Hexagon Place, 6 Falcon Way, Shire Park, Welwyn Garden City, Hertfordshire Al7 1TW UK</nlm:aff><wicri:noCountry code="subfield">Hertfordshire Al7 1TW UK</wicri:noCountry></affiliation></author><author><name sortKey="Williams, Sarah" sort="Williams, Sarah" uniqKey="Williams S" first="Sarah" last="Williams">Sarah Williams</name><affiliation><nlm:aff id="Aff13">Roche Products Limited, Hexagon Place, 6 Falcon Way, Shire Park, Welwyn Garden City, Hertfordshire Al7 1TW UK</nlm:aff><wicri:noCountry code="subfield">Hertfordshire Al7 1TW UK</wicri:noCountry></affiliation></author><author><name sortKey="Schadendorf, Dirk" sort="Schadendorf, Dirk" uniqKey="Schadendorf D" first="Dirk" last="Schadendorf">Dirk Schadendorf</name><affiliation wicri:level="1"><nlm:aff id="Aff14"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 0262 7331</institution-id><institution-id institution-id-type="GRID">grid.410718.b</institution-id><institution>Klinikum für Dermatologie, Venerologie und Allergologie,</institution><institution>Universitätsklinikum Essen,</institution></institution-wrap>Hufelandstrabe 55, 45147 Essen, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Hufelandstrabe 55, 45147 Essen</wicri:regionArea><wicri:noRegion>45147 Essen</wicri:noRegion><wicri:noRegion>45147 Essen</wicri:noRegion><wicri:noRegion>45147 Essen</wicri:noRegion></affiliation></author><author><name sortKey="Hauschild, Axel" sort="Hauschild, Axel" uniqKey="Hauschild A" first="Axel" last="Hauschild">Axel Hauschild</name><affiliation wicri:level="1"><nlm:aff id="Aff15"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0646 2097</institution-id><institution-id institution-id-type="GRID">grid.412468.d</institution-id><institution></institution><institution>Universitätsklinikum Schleswig-Holstein,</institution></institution-wrap>Schittenhelmstr, 7, D-24 105 Kiel, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Schittenhelmstr, 7, D-24 105 Kiel</wicri:regionArea><wicri:noRegion>D-24 105 Kiel</wicri:noRegion><wicri:noRegion>D-24 105 Kiel</wicri:noRegion><wicri:noRegion>D-24 105 Kiel</wicri:noRegion></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">28511673</idno><idno type="pmc">5433030</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5433030</idno><idno type="RBID">PMC:5433030</idno><idno type="doi">10.1186/s12885-017-3286-5</idno><date when="2017">2017</date><idno type="wicri:Area/Pmc/Corpus">000A58</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">000A58</idno><idno type="wicri:Area/Pmc/Curation">000A35</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Curation">000A35</idno><idno type="wicri:Area/Pmc/Checkpoint">000218</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Checkpoint">000218</idno><idno type="wicri:source">PubMed</idno><idno type="wicri:Area/PubMed/Corpus">000C80</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">000C80</idno><idno type="wicri:Area/PubMed/Curation">000C77</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Curation">000C77</idno><idno type="wicri:Area/PubMed/Checkpoint">000C77</idno><idno type="wicri:explorRef" wicri:stream="Checkpoint" wicri:step="PubMed">000C77</idno><idno type="wicri:Area/Ncbi/Merge">004826</idno><idno type="wicri:Area/Ncbi/Curation">004826</idno><idno type="wicri:Area/Ncbi/Checkpoint">004826</idno><idno type="wicri:Area/Main/Merge">000871</idno><idno type="wicri:Area/Main/Curation">000876</idno><idno type="wicri:Area/Main/Exploration">000876</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study</title><author><name sortKey="Sekulic, Aleksandar" sort="Sekulic, Aleksandar" uniqKey="Sekulic A" first="Aleksandar" last="Sekulic">Aleksandar Sekulic</name><affiliation wicri:level="2"><nlm:aff id="Aff1"><institution-wrap><institution-id institution-id-type="ISNI">0000 0000 8875 6339</institution-id><institution-id institution-id-type="GRID">grid.417468.8</institution-id><institution></institution><institution>Mayo Clinic Scottsdale,</institution></institution-wrap>13400 East Shea Boulevard, Scottsdale, AZ 85259 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Arizona</region></placeName><wicri:cityArea>13400 East Shea Boulevard, Scottsdale</wicri:cityArea></affiliation></author><author><name sortKey="Migden, Michael R" sort="Migden, Michael R" uniqKey="Migden M" first="Michael R." last="Migden">Michael R. Migden</name><affiliation wicri:level="2"><nlm:aff id="Aff2"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 2291 4776</institution-id><institution-id institution-id-type="GRID">grid.240145.6</institution-id><institution>Departments of Dermatology and Head and Neck Surgery,</institution><institution>The University of Texas MD Anderson Cancer Center,</institution></institution-wrap>1400 Pressler Street, Houston, TX 77030 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Texas</region></placeName><wicri:cityArea>1400 Pressler Street, Houston</wicri:cityArea></affiliation></author><author><name sortKey="Basset Seguin, Nicole" sort="Basset Seguin, Nicole" uniqKey="Basset Seguin N" first="Nicole" last="Basset-Seguin">Nicole Basset-Seguin</name><affiliation wicri:level="3"><nlm:aff id="Aff3"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 2300 6614</institution-id><institution-id institution-id-type="GRID">grid.413328.f</institution-id><institution>Service de Dermatologie,</institution><institution>Hôpital Saint-Louis,</institution></institution-wrap>1 av claude Vellefaux, 75010 Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>1 av claude Vellefaux, 75010 Paris</wicri:regionArea><placeName><region type="region" nuts="2">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Garbe, Claus" sort="Garbe, Claus" uniqKey="Garbe C" first="Claus" last="Garbe">Claus Garbe</name><affiliation wicri:level="3"><nlm:aff id="Aff4"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 0196 8249</institution-id><institution-id institution-id-type="GRID">grid.411544.1</institution-id><institution>Studienzentrum Dermatologische Onkologie,</institution><institution>Universitätsklinikum Tübingen,</institution></institution-wrap>Liebermeisterstr. 25, 72074 Tübingen, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Liebermeisterstr. 25, 72074 Tübingen</wicri:regionArea><placeName><region type="land" nuts="1">Bade-Wurtemberg</region><region type="district" nuts="2">District de Tübingen</region><settlement type="city">Tübingen</settlement></placeName></affiliation></author><author><name sortKey="Gesierich, Anja" sort="Gesierich, Anja" uniqKey="Gesierich A" first="Anja" last="Gesierich">Anja Gesierich</name><affiliation wicri:level="3"><nlm:aff id="Aff5"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 1378 7891</institution-id><institution-id institution-id-type="GRID">grid.411760.5</institution-id><institution>Klinik für Dermatologie, Venerologie und Allergologie,</institution><institution>Universitätsklinikum Würzburg,</institution></institution-wrap>Josef-Schneider-Str. 2, 97080 Würzburg, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Josef-Schneider-Str. 2, 97080 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="Lao, Christopher D" sort="Lao, Christopher D" uniqKey="Lao C" first="Christopher D." last="Lao">Christopher D. Lao</name><affiliation wicri:level="2"><nlm:aff id="Aff6">University of Michigan, 1500 East Medical Center Drive, Ann Arbor, MI 48109 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Michigan</region></placeName><wicri:cityArea>University of Michigan, 1500 East Medical Center Drive, Ann Arbor</wicri:cityArea></affiliation></author><author><name sortKey="Miller, Chris" sort="Miller, Chris" uniqKey="Miller C" first="Chris" last="Miller">Chris Miller</name><affiliation wicri:level="2"><nlm:aff id="Aff7"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0435 1019</institution-id><institution-id institution-id-type="GRID">grid.412713.2</institution-id><institution>Department of Dermatology,</institution><institution>University of Pennsylvania Medical Center,</institution></institution-wrap>3400 Civic Center Boulevard, Philadelphia, PA 19104 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName><wicri:cityArea>3400 Civic Center Boulevard, Philadelphia</wicri:cityArea></affiliation></author><author><name sortKey="Mortier, Laurent" sort="Mortier, Laurent" uniqKey="Mortier L" first="Laurent" last="Mortier">Laurent Mortier</name><affiliation wicri:level="3"><nlm:aff id="Aff8"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0639 4004</institution-id><institution-id institution-id-type="GRID">grid.413875.c</institution-id><institution>Clinique de Dermatologie,</institution><institution>Hôpital Claude Huriez,</institution></institution-wrap>Inserm U1189, Lille, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Inserm U1189, Lille</wicri:regionArea><placeName><region type="region">Hauts-de-France</region><region type="old region">Nord-Pas-de-Calais</region><settlement type="city">Lille</settlement></placeName></affiliation></author><author><name sortKey="Murrell, Dedee F" sort="Murrell, Dedee F" uniqKey="Murrell D" first="Dedee F." last="Murrell">Dedee F. Murrell</name><affiliation wicri:level="3"><nlm:aff id="Aff9"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 4902 0432</institution-id><institution-id institution-id-type="GRID">grid.1005.4</institution-id><institution>Dermatology Department,</institution><institution>St George Clinical School, University of New South Wales,</institution></institution-wrap>Grey Street, Sydney, 2217 Australia</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>Grey Street, Sydney</wicri:regionArea><placeName><settlement type="city">Sydney</settlement><region type="état">Nouvelle-Galles du Sud</region></placeName></affiliation></author><author><name sortKey="Hamid, Omid" sort="Hamid, Omid" uniqKey="Hamid O" first="Omid" last="Hamid">Omid Hamid</name><affiliation wicri:level="2"><nlm:aff id="Aff10">The Angeles Clinic and Research Institute, 1818 Wilshire Boulevard, Los Angeles, California USA</nlm:aff><country>États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>The Angeles Clinic and Research Institute, 1818 Wilshire Boulevard, Los Angeles</wicri:cityArea></affiliation></author><author><name sortKey="Quevedo, Jorge F" sort="Quevedo, Jorge F" uniqKey="Quevedo J" first="Jorge F." last="Quevedo">Jorge F. Quevedo</name><affiliation wicri:level="2"><nlm:aff id="Aff11"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0459 167X</institution-id><institution-id institution-id-type="GRID">grid.66875.3a</institution-id><institution></institution><institution>Mayo Clinic,</institution></institution-wrap>200 First Street SW, Rochester, MN 55905 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Minnesota</region></placeName><wicri:cityArea>200 First Street SW, Rochester</wicri:cityArea></affiliation></author><author><name sortKey="Hou, Jeannie" sort="Hou, Jeannie" uniqKey="Hou J" first="Jeannie" last="Hou">Jeannie Hou</name><affiliation wicri:level="2"><nlm:aff id="Aff12"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0534 4718</institution-id><institution-id institution-id-type="GRID">grid.418158.1</institution-id><institution></institution><institution>Genentech, Inc.,</institution></institution-wrap>1 DNA Way, South San Francisco, California 94080 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>1 DNA Way, South San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Mckenna, Edward" sort="Mckenna, Edward" uniqKey="Mckenna E" first="Edward" last="Mckenna">Edward Mckenna</name><affiliation wicri:level="2"><nlm:aff id="Aff12"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0534 4718</institution-id><institution-id institution-id-type="GRID">grid.418158.1</institution-id><institution></institution><institution>Genentech, Inc.,</institution></institution-wrap>1 DNA Way, South San Francisco, California 94080 USA</nlm:aff><country xml:lang="fr">États-Unis</country><placeName><region type="state">Californie</region></placeName><wicri:cityArea>1 DNA Way, South San Francisco</wicri:cityArea></affiliation></author><author><name sortKey="Dimier, Natalie" sort="Dimier, Natalie" uniqKey="Dimier N" first="Natalie" last="Dimier">Natalie Dimier</name><affiliation><nlm:aff id="Aff13">Roche Products Limited, Hexagon Place, 6 Falcon Way, Shire Park, Welwyn Garden City, Hertfordshire Al7 1TW UK</nlm:aff><wicri:noCountry code="subfield">Hertfordshire Al7 1TW UK</wicri:noCountry></affiliation></author><author><name sortKey="Williams, Sarah" sort="Williams, Sarah" uniqKey="Williams S" first="Sarah" last="Williams">Sarah Williams</name><affiliation><nlm:aff id="Aff13">Roche Products Limited, Hexagon Place, 6 Falcon Way, Shire Park, Welwyn Garden City, Hertfordshire Al7 1TW UK</nlm:aff><wicri:noCountry code="subfield">Hertfordshire Al7 1TW UK</wicri:noCountry></affiliation></author><author><name sortKey="Schadendorf, Dirk" sort="Schadendorf, Dirk" uniqKey="Schadendorf D" first="Dirk" last="Schadendorf">Dirk Schadendorf</name><affiliation wicri:level="1"><nlm:aff id="Aff14"><institution-wrap><institution-id institution-id-type="ISNI">0000 0001 0262 7331</institution-id><institution-id institution-id-type="GRID">grid.410718.b</institution-id><institution>Klinikum für Dermatologie, Venerologie und Allergologie,</institution><institution>Universitätsklinikum Essen,</institution></institution-wrap>Hufelandstrabe 55, 45147 Essen, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Hufelandstrabe 55, 45147 Essen</wicri:regionArea><wicri:noRegion>45147 Essen</wicri:noRegion><wicri:noRegion>45147 Essen</wicri:noRegion><wicri:noRegion>45147 Essen</wicri:noRegion></affiliation></author><author><name sortKey="Hauschild, Axel" sort="Hauschild, Axel" uniqKey="Hauschild A" first="Axel" last="Hauschild">Axel Hauschild</name><affiliation wicri:level="1"><nlm:aff id="Aff15"><institution-wrap><institution-id institution-id-type="ISNI">0000 0004 0646 2097</institution-id><institution-id institution-id-type="GRID">grid.412468.d</institution-id><institution></institution><institution>Universitätsklinikum Schleswig-Holstein,</institution></institution-wrap>Schittenhelmstr, 7, D-24 105 Kiel, Germany</nlm:aff><country xml:lang="fr">Allemagne</country><wicri:regionArea>Schittenhelmstr, 7, D-24 105 Kiel</wicri:regionArea><wicri:noRegion>D-24 105 Kiel</wicri:noRegion><wicri:noRegion>D-24 105 Kiel</wicri:noRegion><wicri:noRegion>D-24 105 Kiel</wicri:noRegion></affiliation></author></analytic><series><title level="j">BMC Cancer</title><idno type="eISSN">1471-2407</idno><imprint><date when="2017">2017</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><sec><title>Background</title><p>In the primary analysis of the ERIVANCE BCC trial, vismodegib, the first US Food and Drug Administration–approved Hedgehog pathway inhibitor, showed objective response rates (ORRs) by independent review facility (IRF) of 30% and 43% in metastatic basal cell carcinoma (mBCC) and locally advanced BCC (laBCC), respectively. ORRs by investigator review were 45% (mBCC) and 60% (laBCC). Herein, we present long-term safety and final investigator-assessed efficacy results in patients with mBCC or laBCC.</p></sec><sec><title>Methods</title><p>One hundred four patients with measurable advanced BCC received oral vismodegib 150 mg once daily until disease progression or intolerable toxicity. The primary end point was IRF-assessed ORR. Secondary end points included ORR, duration of response (DOR), progression-free survival, overall survival (OS), and safety.</p></sec><sec><title>Results</title><p>At data cutoff (39 months after completion of accrual), 8 patients were receiving the study drug (69 patients in survival follow-up). Investigator-assessed ORR was 48.5% in the mBCC group (all partial responses) and 60.3% in the laBCC group (20 patients had complete response and 18 patients had partial response). ORRs were comparable across patient subgroups, including aggressive histologic subtypes (eg, infiltrative BCC). Median DOR was 14.8 months (mBCC) and 26.2 months (laBCC). Median OS was 33.4 months in the mBCC cohort and not estimable in the laBCC cohort. Adverse events remained consistent with clinical experience. Thirty-three deaths (31.7%) were reported; none were related to vismodegib.</p></sec><sec><title>Conclusions</title><p>This long-term update of the ERIVANCE BCC trial demonstrated durability of response, efficacy across patient subgroups, and manageable long-term safety of vismodegib in patients with advanced BCC.</p></sec><sec><title>Trial registration</title><p>This study was registered prospectively with <ext-link ext-link-type="uri" xlink:href="http://clinicaltrials.gov">Clinicaltrials.gov</ext-link>, number <ext-link ext-link-type="uri" xlink:href="https://clinicaltrials.gov/ct2/show/NCT00833417?term=NCT00833417&rank=1">NCT00833417</ext-link> on January 30, 2009.</p></sec><sec><title>Electronic supplementary material</title><p>The online version of this article (doi:10.1186/s12885-017-3286-5) contains supplementary material, which is available to authorized users.</p></sec></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Macha, Ma" uniqKey="Macha M">MA Macha</name></author><author><name sortKey="Batra, Sk" uniqKey="Batra S">SK Batra</name></author><author><name sortKey="Ganti, Ak" uniqKey="Ganti A">AK Ganti</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Wu, S" uniqKey="Wu S">S Wu</name></author><author><name sortKey="Han, J" uniqKey="Han J">J Han</name></author><author><name sortKey="Li, W Q" uniqKey="Li W">W-Q Li</name></author><author><name sortKey="Li, T" uniqKey="Li T">T Li</name></author><author><name sortKey="Qureshi, Aa" uniqKey="Qureshi A">AA Qureshi</name></author></analytic></biblStruct><biblStruct></biblStruct><biblStruct><analytic><author><name sortKey="Sekulic, A" uniqKey="Sekulic A">A Sekulic</name></author><author><name sortKey="Mangold, Ar" uniqKey="Mangold A">AR Mangold</name></author><author><name sortKey="Northfelt, Dw" uniqKey="Northfelt D">DW Northfelt</name></author><author><name sortKey="Lorusso, Pm" uniqKey="Lorusso P">PM LoRusso</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Varga, E" uniqKey="Varga E">E Varga</name></author><author><name sortKey="Korom, I" uniqKey="Korom I">I Korom</name></author><author><name sortKey="Rask, Z" uniqKey="Rask Z">Z Raskó</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Wadhera, A" uniqKey="Wadhera A">A Wadhera</name></author><author><name sortKey="Fazio, M" uniqKey="Fazio M">M Fazio</name></author><author><name sortKey="Bricca, G" uniqKey="Bricca G">G Bricca</name></author><author><name sortKey="Stanton, O" uniqKey="Stanton O">O Stanton</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Lo, Js" uniqKey="Lo J">JS Lo</name></author><author><name sortKey="Snow, Sn" uniqKey="Snow S">SN Snow</name></author><author><name sortKey="Reizner, Gt" uniqKey="Reizner G">GT Reizner</name></author><author><name sortKey="Mohs, Fe" uniqKey="Mohs F">FE Mohs</name></author><author><name sortKey="Larson, Po" uniqKey="Larson P">PO Larson</name></author><author><name sortKey="Hruza, Gj" uniqKey="Hruza G">GJ Hruza</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Snow, Sn" uniqKey="Snow S">SN Snow</name></author><author><name sortKey="Sahl, W" uniqKey="Sahl W">W Sahl</name></author><author><name sortKey="Lo, Js" uniqKey="Lo J">JS Lo</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Reifenberger, J" uniqKey="Reifenberger J">J Reifenberger</name></author><author><name sortKey="Wolter, M" uniqKey="Wolter M">M Wolter</name></author><author><name sortKey="Weber, Rg" uniqKey="Weber R">RG Weber</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Xie, J" uniqKey="Xie J">J Xie</name></author><author><name sortKey="Murone, M" uniqKey="Murone M">M Murone</name></author><author><name sortKey="Luoh, S M" uniqKey="Luoh S">S-M Luoh</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Goppner, D" uniqKey="Goppner D">D Göppner</name></author><author><name sortKey="Leverkus, M" uniqKey="Leverkus M">M Leverkus</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Daya Grosjean, L" uniqKey="Daya Grosjean L">L Daya-Grosjean</name></author><author><name sortKey="Couve Privat, S" uniqKey="Couve Privat S">S Couvé-Privat</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Lorusso, Pm" uniqKey="Lorusso P">PM LoRusso</name></author><author><name sortKey="Rudin, Cm" uniqKey="Rudin C">CM Rudin</name></author><author><name sortKey="Reddy, Jc" uniqKey="Reddy J">JC Reddy</name></author></analytic></biblStruct><biblStruct></biblStruct><biblStruct></biblStruct><biblStruct><analytic><author><name sortKey="Migden, Mr" uniqKey="Migden M">MR Migden</name></author><author><name sortKey="Guminski, A" uniqKey="Guminski A">A Guminski</name></author><author><name sortKey="Gutzmer, R" uniqKey="Gutzmer R">R Gutzmer</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Sekulic, A" uniqKey="Sekulic A">A Sekulic</name></author><author><name sortKey="Migden, Mr" uniqKey="Migden M">MR Migden</name></author><author><name sortKey="Oro, Ae" uniqKey="Oro A">AE Oro</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Therasse, P" uniqKey="Therasse P">P Therasse</name></author><author><name sortKey="Arbuck, Sg" uniqKey="Arbuck S">SG Arbuck</name></author><author><name sortKey="Eisenhauer, Ea" uniqKey="Eisenhauer E">EA Eisenhauer</name></author></analytic></biblStruct><biblStruct></biblStruct><biblStruct><analytic><author><name sortKey="Sekulic, A" uniqKey="Sekulic A">A Sekulic</name></author><author><name sortKey="Migden, Mr" uniqKey="Migden M">MR Migden</name></author><author><name sortKey="Lewis, K" uniqKey="Lewis K">K Lewis</name></author></analytic></biblStruct><biblStruct></biblStruct><biblStruct><analytic><author><name sortKey="Mccusker, M" uniqKey="Mccusker M">M McCusker</name></author><author><name sortKey="Basset Seguin, N" uniqKey="Basset Seguin N">N Basset-Seguin</name></author><author><name sortKey="Dummer, R" uniqKey="Dummer R">R Dummer</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Von Domarus, H" uniqKey="Von Domarus H">H von Domarus</name></author><author><name sortKey="Stevens, Pj" uniqKey="Stevens P">PJ Stevens</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Lear, Jt" uniqKey="Lear J">JT Lear</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Basset Seguin, N" uniqKey="Basset Seguin N">N Basset-Seguin</name></author><author><name sortKey="Hauschild, A" uniqKey="Hauschild A">A Hauschild</name></author><author><name sortKey="Grob, J J" uniqKey="Grob J">J-J Grob</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Lacouture, Me" uniqKey="Lacouture M">ME Lacouture</name></author><author><name sortKey="Dreno, B" uniqKey="Dreno B">B Dréno</name></author><author><name sortKey="Ascierto, Pa" uniqKey="Ascierto P">PA Ascierto</name></author></analytic></biblStruct></listBibl></div1></back></TEI><affiliations><list><country><li>Allemagne</li><li>Australie</li><li>France</li><li>États-Unis</li></country><region><li>Arizona</li><li>Bade-Wurtemberg</li><li>Bavière</li><li>Californie</li><li>District de Basse-Franconie</li><li>District de Tübingen</li><li>Hauts-de-France</li><li>Michigan</li><li>Minnesota</li><li>Nord-Pas-de-Calais</li><li>Nouvelle-Galles du Sud</li><li>Pennsylvanie</li><li>Texas</li><li>Île-de-France</li></region><settlement><li>Lille</li><li>Paris</li><li>Sydney</li><li>Tübingen</li><li>Wurtzbourg</li></settlement></list><tree><noCountry><name sortKey="Dimier, Natalie" sort="Dimier, Natalie" uniqKey="Dimier N" first="Natalie" last="Dimier">Natalie Dimier</name><name sortKey="Williams, Sarah" sort="Williams, Sarah" uniqKey="Williams S" first="Sarah" last="Williams">Sarah Williams</name></noCountry><country name="États-Unis"><region name="Arizona"><name sortKey="Sekulic, Aleksandar" sort="Sekulic, Aleksandar" uniqKey="Sekulic A" first="Aleksandar" last="Sekulic">Aleksandar Sekulic</name></region><name sortKey="Hamid, Omid" sort="Hamid, Omid" uniqKey="Hamid O" first="Omid" last="Hamid">Omid Hamid</name><name sortKey="Hou, Jeannie" sort="Hou, Jeannie" uniqKey="Hou J" first="Jeannie" last="Hou">Jeannie Hou</name><name sortKey="Lao, Christopher D" sort="Lao, Christopher D" uniqKey="Lao C" first="Christopher D." last="Lao">Christopher D. Lao</name><name sortKey="Mckenna, Edward" sort="Mckenna, Edward" uniqKey="Mckenna E" first="Edward" last="Mckenna">Edward Mckenna</name><name sortKey="Migden, Michael R" sort="Migden, Michael R" uniqKey="Migden M" first="Michael R." last="Migden">Michael R. Migden</name><name sortKey="Miller, Chris" sort="Miller, Chris" uniqKey="Miller C" first="Chris" last="Miller">Chris Miller</name><name sortKey="Quevedo, Jorge F" sort="Quevedo, Jorge F" uniqKey="Quevedo J" first="Jorge F." last="Quevedo">Jorge F. Quevedo</name></country><country name="France"><region name="Île-de-France"><name sortKey="Basset Seguin, Nicole" sort="Basset Seguin, Nicole" uniqKey="Basset Seguin N" first="Nicole" last="Basset-Seguin">Nicole Basset-Seguin</name></region><name sortKey="Mortier, Laurent" sort="Mortier, Laurent" uniqKey="Mortier L" first="Laurent" last="Mortier">Laurent Mortier</name></country><country name="Allemagne"><region name="Bade-Wurtemberg"><name sortKey="Garbe, Claus" sort="Garbe, Claus" uniqKey="Garbe C" first="Claus" last="Garbe">Claus Garbe</name></region><name sortKey="Gesierich, Anja" sort="Gesierich, Anja" uniqKey="Gesierich A" first="Anja" last="Gesierich">Anja Gesierich</name><name sortKey="Hauschild, Axel" sort="Hauschild, Axel" uniqKey="Hauschild A" first="Axel" last="Hauschild">Axel Hauschild</name><name sortKey="Schadendorf, Dirk" sort="Schadendorf, Dirk" uniqKey="Schadendorf D" first="Dirk" last="Schadendorf">Dirk Schadendorf</name></country><country name="Australie"><region name="Nouvelle-Galles du Sud"><name sortKey="Murrell, Dedee F" sort="Murrell, Dedee F" uniqKey="Murrell D" first="Dedee F." last="Murrell">Dedee F. Murrell</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
EXPLOR_STEP=$WICRI_ROOT/Wicri/Asie/explor/AustralieFrV1/Data/Main/Exploration
HfdSelect -h $EXPLOR_STEP/biblio.hfd -nk 000876 | SxmlIndent | more
Ou
HfdSelect -h $EXPLOR_AREA/Data/Main/Exploration/biblio.hfd -nk 000876 | SxmlIndent | more
Pour mettre un lien sur cette page dans le réseau Wicri
{{Explor lien
|wiki= Wicri/Asie
|area= AustralieFrV1
|flux= Main
|étape= Exploration
|type= RBID
|clé= PMC:5433030
|texte= Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study
}}
Pour générer des pages wiki
HfdIndexSelect -h $EXPLOR_AREA/Data/Main/Exploration/RBID.i -Sk "pubmed:28511673" \
| HfdSelect -Kh $EXPLOR_AREA/Data/Main/Exploration/biblio.hfd \
| NlmPubMed2Wicri -a AustralieFrV1
| This area was generated with Dilib version V0.6.33. |  |