nih-gov/www.ncbi.nlm.nih.gov/omim/614320

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Entry
- #614320 - PANCREATIC CANCER, SUSCEPTIBILITY TO, 4; PNCA4
- OMIM
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<span class="h4">#614320</span>
<br />
<strong>Table of Contents</strong>
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<a href="#title"><strong>Title</strong></a>
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<a href="#phenotypeMap"><strong>Phenotype-Gene Relationships</strong></a>
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<a href="#text"><strong>Text</strong></a>
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<a href="#mapping">Mapping</a>
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<a href="#molecularGenetics">Molecular Genetics</a>
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<a href="#clinicalManagement">Clinical Management</a>
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<a href="#references"><strong>References</strong></a>
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<a href="#contributors"><strong>Contributors</strong></a>
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<li role="presentation">
<a href="#creationDate"><strong>Creation Date</strong></a>
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<div class="panel-body small mim-panel-body">
<div><a href="https://clinicaltrials.gov/search?cond=PANCREATIC CANCER" class="mim-tip-hint" title="A registry of federally and privately supported clinical trials conducted in the United States and around the world." target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'Clinical Trials', 'domain': 'clinicaltrials.gov'})">Clinical Trials</a></div>
<div><a href="https://www.orpha.net/consor/cgi-bin/ClinicalLabs_Search_Simple.php?lng=EN&LnkId=3708&Typ=Pat" class="mim-tip-hint" title="A list of European laboratories that offer genetic testing." target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'EuroGentest', 'domain': 'orpha.net'})">EuroGentest</a></div>
<div><a href="https://www.ncbi.nlm.nih.gov/books/NBK1247/" class="mim-tip-hint" title="Expert-authored, peer-reviewed descriptions of inherited disorders including the uses of genetic testing in diagnosis, management, and genetic counseling." target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'Gene Reviews', 'domain': 'ncbi.nlm.nih.gov'})">Gene Reviews</a></div>
<div><a href="https://www.diseaseinfosearch.org/x/9071" class="mim-tip-hint" title="Network of disease-specific advocacy organizations, universities, private companies, government agencies, and public policy organizations." target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'Genetic Alliance', 'domain': 'diseaseinfosearch.org'})">Genetic Alliance</a></div>
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<a href="#" class="mim-tip-icd" qtip_title="<strong>ICD+</strong>" qtip_text="
<strong>ORPHA:</strong> 1333<br />
">ICD+</a>
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<span class="mim-font mim-tip-hint" title="Phenotype description, molecular basis known">
<span class="text-danger"><strong>#</strong></span>
614320
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<h3>
<span class="mim-font">
PANCREATIC CANCER, SUSCEPTIBILITY TO, 4; PNCA4
</span>
</h3>
</div>
<div>
<br />
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<strong>Phenotype-Gene Relationships</strong>
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<th>
Location
</th>
<th>
Phenotype
</th>
<th>
Phenotype <br /> MIM number
</th>
<th>
Inheritance
</th>
<th>
Phenotype <br /> mapping key
</th>
<th>
Gene/Locus
</th>
<th>
Gene/Locus <br /> MIM number
</th>
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</thead>
<tbody>
<tr>
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<span class="mim-font">
<a href="/geneMap/17/610?start=-3&limit=10&highlight=610">
17q21.31
</a>
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<td>
<span class="mim-font">
{Pancreatic cancer, susceptibility to, 4}
</span>
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<td>
<span class="mim-font">
<a href="/entry/614320"> 614320 </a>
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<td>
<span class="mim-font">
</span>
</td>
<td>
<span class="mim-font">
<abbr class="mim-tip-hint" title="3 - The molecular basis of the disorder is known"> 3 </abbr>
</span>
</td>
<td>
<span class="mim-font">
BRCA1
</span>
</td>
<td>
<span class="mim-font">
<a href="/entry/113705"> 113705 </a>
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<strong>TEXT</strong>
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<p>A number sign (#) is used with this entry because this form of susceptibility to pancreatic cancer is caused by heterozygous mutation in the BRCA1 gene (<a href="/entry/113705">113705</a>).</p><p>For background, phenotypic description, and a discussion of genetic heterogeneity of pancreatic cancer, see <a href="/entry/260350">260350</a>.</p>
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<p>This form of susceptibility to pancreatic cancer is caused by mutations in the BRCA1 gene, which <a href="#3" class="mim-tip-reference" title="Miki, Y., Swensen, J., Shattuck-Eidens, D., Futreal, P. A., Harshman, K., Tavtigian, S., Liu, Q., Cochran, C., Bennett, L. M., Ding, W., Bell, R., Rosenthal, J., and 33 others. &lt;strong&gt;A strong candidate for the breast and ovarian cancer susceptibility gene BRCA1.&lt;/strong&gt; Science 266: 66-71, 1994.[PubMed: &lt;a href=&quot;https://pubmed.ncbi.nlm.nih.gov/7545954/&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;name&#x27;: &#x27;PubMed&#x27;, &#x27;domain&#x27;: &#x27;pubmed.ncbi.nlm.nih.gov&#x27;})&quot;&gt;7545954&lt;/a&gt;] [&lt;a href=&quot;https://doi.org/10.1126/science.7545954&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;destination&#x27;: &#x27;Publisher&#x27;})&quot;&gt;Full Text&lt;/a&gt;]" pmid="7545954">Miki et al. (1994)</a> mapped to chromosome 17q21. <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=7545954" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})"><span class="glyphicon glyphicon-plus-sign mim-tip-hint" title="Click this 'reference-plus' icon to see articles related to this paragraph in PubMed."></span></a></p>
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<strong>Molecular Genetics</strong>
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<p><a href="#1" class="mim-tip-reference" title="Al-Sukhni, W., Rothenmund, H., Eppel Borgida, A., Zogopoulos, G., O&#x27;Shea, A.-M., Pollett, A., Gallinger, S. &lt;strong&gt;Germline BRCA1 mutations predispose to pancreatic adenocarcinoma.&lt;/strong&gt; Hum. Genet. 124: 271-278, 2008.[PubMed: &lt;a href=&quot;https://pubmed.ncbi.nlm.nih.gov/18762988/&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;name&#x27;: &#x27;PubMed&#x27;, &#x27;domain&#x27;: &#x27;pubmed.ncbi.nlm.nih.gov&#x27;})&quot;&gt;18762988&lt;/a&gt;] [&lt;a href=&quot;https://doi.org/10.1007/s00439-008-0554-0&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;destination&#x27;: &#x27;Publisher&#x27;})&quot;&gt;Full Text&lt;/a&gt;]" pmid="18762988">Al-Sukhni et al. (2008)</a> found loss of heterozygosity at the BRCA1 locus in pancreatic tumor DNA from 5 (71%) of 7 patients with pancreatic cancer who carried a heterozygous germline BRCA1 mutation (see, e.g., <a href="/entry/113705#0003">113705.0003</a> and <a href="/entry/113705#0018">113705.0018</a>). Pancreatic tumor DNA was available for sequencing in 4 cases, and 3 demonstrated loss of the wildtype allele. In contrast, only 1 (11%) of 9 patients with sporadic pancreatic cancer and no germline BRCA1 mutations showed LOH at the BRCA1 locus. <a href="#1" class="mim-tip-reference" title="Al-Sukhni, W., Rothenmund, H., Eppel Borgida, A., Zogopoulos, G., O&#x27;Shea, A.-M., Pollett, A., Gallinger, S. &lt;strong&gt;Germline BRCA1 mutations predispose to pancreatic adenocarcinoma.&lt;/strong&gt; Hum. Genet. 124: 271-278, 2008.[PubMed: &lt;a href=&quot;https://pubmed.ncbi.nlm.nih.gov/18762988/&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;name&#x27;: &#x27;PubMed&#x27;, &#x27;domain&#x27;: &#x27;pubmed.ncbi.nlm.nih.gov&#x27;})&quot;&gt;18762988&lt;/a&gt;] [&lt;a href=&quot;https://doi.org/10.1007/s00439-008-0554-0&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;destination&#x27;: &#x27;Publisher&#x27;})&quot;&gt;Full Text&lt;/a&gt;]" pmid="18762988">Al-Sukhni et al. (2008)</a> concluded that BRCA1 germline mutations likely predispose to the development of pancreatic cancer and suggested that individuals with these mutations be considered for pancreatic cancer-screening programs. <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=18762988" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})"><span class="glyphicon glyphicon-plus-sign mim-tip-hint" title="Click this 'reference-plus' icon to see articles related to this paragraph in PubMed."></span></a></p><p><a href="#4" class="mim-tip-reference" title="Zhen, D. B., Rabe, K. G., Gallinger, S., Syngal, S., Schwartz, A. G., Goggins, M. G., Hruban, R. H., Cote, M. L., McWilliams, R. R., Roberts, N. J., Cannon-Albright, L. A., Li, D., Moyes, K., Wenstrup, R. J., Hartman, A.-R., Seminara, D., Klein, A. P., Petersen, G. M. &lt;strong&gt;BRCA1, BRCA2, PALB2, and CDKN2A mutations in familial pancreatic cancer: a PACGENE study.&lt;/strong&gt; Genet. Med. 17: 569-577, 2015.[PubMed: &lt;a href=&quot;https://pubmed.ncbi.nlm.nih.gov/25356972/&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;name&#x27;: &#x27;PubMed&#x27;, &#x27;domain&#x27;: &#x27;pubmed.ncbi.nlm.nih.gov&#x27;})&quot;&gt;25356972&lt;/a&gt;] [&lt;a href=&quot;https://doi.org/10.1038/gim.2014.153&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;destination&#x27;: &#x27;Publisher&#x27;})&quot;&gt;Full Text&lt;/a&gt;]" pmid="25356972">Zhen et al. (2015)</a> tested germline DNA from 727 unrelated probands with pancreatic cancer and a positive family history for mutations in BRCA1 and BRCA2 (<a href="/entry/600185">600185</a>) (including deletions and rearrangements), PALB2 (<a href="/entry/610355">610355</a>), and CDKN2A (<a href="/entry/600160">600160</a>). Among these probands, 521 met criteria for familial pancreatic cancer (FPC; at least 2 affected first-degree relatives). The prevalence of deleterious mutations, excluding variants of unknown significance, among FPC probands was BRCA1, 1.2%; BRCA2, 3.7%; PALB2, 0.6%; and CDKN2A, 2.5%. Four novel deleterious mutations were detected. FPC probands carried more mutations in the 4 genes (8.0%) than nonfamilial pancreatic cancer probands (3.5%; OR = 2.40, 95% CI 1.06-5.44, p = 0.03). The probability of testing positive for deleterious mutations in any of the 4 genes ranged up to 10.4%, depending on family history of cancers. <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=25356972" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})"><span class="glyphicon glyphicon-plus-sign mim-tip-hint" title="Click this 'reference-plus' icon to see articles related to this paragraph in PubMed."></span></a></p>
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<strong>Clinical Management</strong>
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<p><a href="#2" class="mim-tip-reference" title="Golan, T., Hammel, P., Reni, M., Van Cutsem, E., Macarulla, T., Hall, M. J., Park, J.-O., Hochhauser, D., Arnold, D., Oh, D.-Y., Reinacher-Schick, A., Tortora, G., Algul, H., O&#x27;Reilly, E. M., McGuinness, D., Cui, K. Y., Schlienger, K., Locker, G. Y., Kindler, H. L. &lt;strong&gt;Maintenance olaparib for germline BRCA-mutated metastatic pancreatic cancer.&lt;/strong&gt; New Eng. J. Med. 381: 317-327, 2019.[PubMed: &lt;a href=&quot;https://pubmed.ncbi.nlm.nih.gov/31157963/&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;name&#x27;: &#x27;PubMed&#x27;, &#x27;domain&#x27;: &#x27;pubmed.ncbi.nlm.nih.gov&#x27;})&quot;&gt;31157963&lt;/a&gt;] [&lt;a href=&quot;https://doi.org/10.1056/NEJMoa1903387&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;destination&#x27;: &#x27;Publisher&#x27;})&quot;&gt;Full Text&lt;/a&gt;]" pmid="31157963">Golan et al. (2019)</a> conducted a randomized, double-blind, placebo-controlled, phase 3 trial to evaluate the efficacy of olaparib, a PARP (see <a href="/entry/173870">173870</a>) inhibitor, as maintenance therapy in patients who had a germline BRCA1 or BRCA2 mutation and metastatic pancreatic cancer and disease that had not progressed during first-line platinum-based chemotherapy. The primary end point was progression-free survival, which was assessed by blinded independent central review. Of the 3,315 patients who underwent screening, 154 underwent randomization and were assigned to a trial intervention (92 received olaparib and 62 received placebo). The median progression-free survival was significantly longer in the olaparib group than in the placebo group (7.4 months vs 3.8 months; hazard ratio for disease progression or death, 0.53; 95% confidence interval (CI), 0.35 to 0.82; p = 0.004). An interim analysis of overall survival, at a data maturity of 46%, showed no difference between the olaparib and placebo groups. There was no significant between-group difference in health-related quality of life. The incidence of grade 3 or higher adverse events was 40% in the olaparib group and 23% in the placebo group. <a href="#2" class="mim-tip-reference" title="Golan, T., Hammel, P., Reni, M., Van Cutsem, E., Macarulla, T., Hall, M. J., Park, J.-O., Hochhauser, D., Arnold, D., Oh, D.-Y., Reinacher-Schick, A., Tortora, G., Algul, H., O&#x27;Reilly, E. M., McGuinness, D., Cui, K. Y., Schlienger, K., Locker, G. Y., Kindler, H. L. &lt;strong&gt;Maintenance olaparib for germline BRCA-mutated metastatic pancreatic cancer.&lt;/strong&gt; New Eng. J. Med. 381: 317-327, 2019.[PubMed: &lt;a href=&quot;https://pubmed.ncbi.nlm.nih.gov/31157963/&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;name&#x27;: &#x27;PubMed&#x27;, &#x27;domain&#x27;: &#x27;pubmed.ncbi.nlm.nih.gov&#x27;})&quot;&gt;31157963&lt;/a&gt;] [&lt;a href=&quot;https://doi.org/10.1056/NEJMoa1903387&quot; target=&quot;_blank&quot; onclick=&quot;gtag(&#x27;event&#x27;, &#x27;mim_outbound&#x27;, {&#x27;destination&#x27;: &#x27;Publisher&#x27;})&quot;&gt;Full Text&lt;/a&gt;]" pmid="31157963">Golan et al. (2019)</a> concluded that among patients with germline BRCA mutation and metastatic pancreatic cancer, progression-free survival was longer with maintenance olaparib than with placebo. <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=31157963" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})"><span class="glyphicon glyphicon-plus-sign mim-tip-hint" title="Click this 'reference-plus' icon to see articles related to this paragraph in PubMed."></span></a></p>
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<strong>REFERENCES</strong>
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<p />
</div>
<div id="mimReferencesFold" class="collapse in mimTextToggleFold">
<ol>
<li>
<a id="1" class="mim-anchor"></a>
<a id="Al-Sukhni2008" class="mim-anchor"></a>
<div class="">
<p class="mim-text-font">
Al-Sukhni, W., Rothenmund, H., Eppel Borgida, A., Zogopoulos, G., O'Shea, A.-M., Pollett, A., Gallinger, S.
<strong>Germline BRCA1 mutations predispose to pancreatic adenocarcinoma.</strong>
Hum. Genet. 124: 271-278, 2008.
[PubMed: <a href="https://pubmed.ncbi.nlm.nih.gov/18762988/" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">18762988</a>, <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=18762988" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">related citations</a>]
[<a href="https://doi.org/10.1007/s00439-008-0554-0" target="_blank">Full Text</a>]
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<a id="2" class="mim-anchor"></a>
<a id="Golan2019" class="mim-anchor"></a>
<div class="">
<p class="mim-text-font">
Golan, T., Hammel, P., Reni, M., Van Cutsem, E., Macarulla, T., Hall, M. J., Park, J.-O., Hochhauser, D., Arnold, D., Oh, D.-Y., Reinacher-Schick, A., Tortora, G., Algul, H., O'Reilly, E. M., McGuinness, D., Cui, K. Y., Schlienger, K., Locker, G. Y., Kindler, H. L.
<strong>Maintenance olaparib for germline BRCA-mutated metastatic pancreatic cancer.</strong>
New Eng. J. Med. 381: 317-327, 2019.
[PubMed: <a href="https://pubmed.ncbi.nlm.nih.gov/31157963/" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">31157963</a>, <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=31157963" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">related citations</a>]
[<a href="https://doi.org/10.1056/NEJMoa1903387" target="_blank">Full Text</a>]
</p>
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<a id="3" class="mim-anchor"></a>
<a id="Miki1994" class="mim-anchor"></a>
<div class="">
<p class="mim-text-font">
Miki, Y., Swensen, J., Shattuck-Eidens, D., Futreal, P. A., Harshman, K., Tavtigian, S., Liu, Q., Cochran, C., Bennett, L. M., Ding, W., Bell, R., Rosenthal, J., and 33 others.
<strong>A strong candidate for the breast and ovarian cancer susceptibility gene BRCA1.</strong>
Science 266: 66-71, 1994.
[PubMed: <a href="https://pubmed.ncbi.nlm.nih.gov/7545954/" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">7545954</a>, <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=7545954" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">related citations</a>]
[<a href="https://doi.org/10.1126/science.7545954" target="_blank">Full Text</a>]
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<a id="Zhen2015" class="mim-anchor"></a>
<div class="">
<p class="mim-text-font">
Zhen, D. B., Rabe, K. G., Gallinger, S., Syngal, S., Schwartz, A. G., Goggins, M. G., Hruban, R. H., Cote, M. L., McWilliams, R. R., Roberts, N. J., Cannon-Albright, L. A., Li, D., Moyes, K., Wenstrup, R. J., Hartman, A.-R., Seminara, D., Klein, A. P., Petersen, G. M.
<strong>BRCA1, BRCA2, PALB2, and CDKN2A mutations in familial pancreatic cancer: a PACGENE study.</strong>
Genet. Med. 17: 569-577, 2015.
[PubMed: <a href="https://pubmed.ncbi.nlm.nih.gov/25356972/" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">25356972</a>, <a href="https://pubmed.ncbi.nlm.nih.gov/?cmd=link&linkname=pubmed_pubmed&from_uid=25356972" target="_blank" onclick="gtag('event', 'mim_outbound', {'name': 'PubMed Related', 'domain': 'pubmed.ncbi.nlm.nih.gov'})">related citations</a>]
[<a href="https://doi.org/10.1038/gim.2014.153" target="_blank">Full Text</a>]
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Ada Hamosh - updated : 08/12/2019
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Ada Hamosh - updated : 10/19/2015
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Matthew B. Gross : 10/31/2011
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alopez : 10/19/2015<br>alopez : 10/19/2015<br>mgross : 10/31/2011
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<strong>#</strong> 614320
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PANCREATIC CANCER, SUSCEPTIBILITY TO, 4; PNCA4
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<strong>ORPHA:</strong> 1333; &nbsp;
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<strong>Phenotype-Gene Relationships</strong>
</span>
</h4>
<div>
<table class="table table-bordered table-condensed small mim-table-padding">
<thead>
<tr class="active">
<th>
Location
</th>
<th>
Phenotype
</th>
<th>
Phenotype <br /> MIM number
</th>
<th>
Inheritance
</th>
<th>
Phenotype <br /> mapping key
</th>
<th>
Gene/Locus
</th>
<th>
Gene/Locus <br /> MIM number
</th>
</tr>
</thead>
<tbody>
<tr>
<td>
<span class="mim-font">
17q21.31
</span>
</td>
<td>
<span class="mim-font">
{Pancreatic cancer, susceptibility to, 4}
</span>
</td>
<td>
<span class="mim-font">
614320
</span>
</td>
<td>
<span class="mim-font">
</span>
</td>
<td>
<span class="mim-font">
3
</span>
</td>
<td>
<span class="mim-font">
BRCA1
</span>
</td>
<td>
<span class="mim-font">
113705
</span>
</td>
</tr>
</tbody>
</table>
</div>
</div>
<div>
<br />
</div>
<div>
<h4>
<span class="mim-font">
<strong>TEXT</strong>
</span>
</h4>
<span class="mim-text-font">
<p>A number sign (#) is used with this entry because this form of susceptibility to pancreatic cancer is caused by heterozygous mutation in the BRCA1 gene (113705).</p><p>For background, phenotypic description, and a discussion of genetic heterogeneity of pancreatic cancer, see 260350.</p>
</span>
<div>
<br />
</div>
<div>
<h4>
<span class="mim-font">
<strong>Mapping</strong>
</span>
</h4>
</div>
<span class="mim-text-font">
<p>This form of susceptibility to pancreatic cancer is caused by mutations in the BRCA1 gene, which Miki et al. (1994) mapped to chromosome 17q21. </p>
</span>
<div>
<br />
</div>
<div>
<h4>
<span class="mim-font">
<strong>Molecular Genetics</strong>
</span>
</h4>
</div>
<span class="mim-text-font">
<p>Al-Sukhni et al. (2008) found loss of heterozygosity at the BRCA1 locus in pancreatic tumor DNA from 5 (71%) of 7 patients with pancreatic cancer who carried a heterozygous germline BRCA1 mutation (see, e.g., 113705.0003 and 113705.0018). Pancreatic tumor DNA was available for sequencing in 4 cases, and 3 demonstrated loss of the wildtype allele. In contrast, only 1 (11%) of 9 patients with sporadic pancreatic cancer and no germline BRCA1 mutations showed LOH at the BRCA1 locus. Al-Sukhni et al. (2008) concluded that BRCA1 germline mutations likely predispose to the development of pancreatic cancer and suggested that individuals with these mutations be considered for pancreatic cancer-screening programs. </p><p>Zhen et al. (2015) tested germline DNA from 727 unrelated probands with pancreatic cancer and a positive family history for mutations in BRCA1 and BRCA2 (600185) (including deletions and rearrangements), PALB2 (610355), and CDKN2A (600160). Among these probands, 521 met criteria for familial pancreatic cancer (FPC; at least 2 affected first-degree relatives). The prevalence of deleterious mutations, excluding variants of unknown significance, among FPC probands was BRCA1, 1.2%; BRCA2, 3.7%; PALB2, 0.6%; and CDKN2A, 2.5%. Four novel deleterious mutations were detected. FPC probands carried more mutations in the 4 genes (8.0%) than nonfamilial pancreatic cancer probands (3.5%; OR = 2.40, 95% CI 1.06-5.44, p = 0.03). The probability of testing positive for deleterious mutations in any of the 4 genes ranged up to 10.4%, depending on family history of cancers. </p>
</span>
<div>
<br />
</div>
<div>
<h4>
<span class="mim-font">
<strong>Clinical Management</strong>
</span>
</h4>
</div>
<span class="mim-text-font">
<p>Golan et al. (2019) conducted a randomized, double-blind, placebo-controlled, phase 3 trial to evaluate the efficacy of olaparib, a PARP (see 173870) inhibitor, as maintenance therapy in patients who had a germline BRCA1 or BRCA2 mutation and metastatic pancreatic cancer and disease that had not progressed during first-line platinum-based chemotherapy. The primary end point was progression-free survival, which was assessed by blinded independent central review. Of the 3,315 patients who underwent screening, 154 underwent randomization and were assigned to a trial intervention (92 received olaparib and 62 received placebo). The median progression-free survival was significantly longer in the olaparib group than in the placebo group (7.4 months vs 3.8 months; hazard ratio for disease progression or death, 0.53; 95% confidence interval (CI), 0.35 to 0.82; p = 0.004). An interim analysis of overall survival, at a data maturity of 46%, showed no difference between the olaparib and placebo groups. There was no significant between-group difference in health-related quality of life. The incidence of grade 3 or higher adverse events was 40% in the olaparib group and 23% in the placebo group. Golan et al. (2019) concluded that among patients with germline BRCA mutation and metastatic pancreatic cancer, progression-free survival was longer with maintenance olaparib than with placebo. </p>
</span>
<div>
<br />
</div>
</div>
<div>
<h4>
<span class="mim-font">
<strong>REFERENCES</strong>
</span>
</h4>
<div>
<p />
</div>
<div>
<ol>
<li>
<p class="mim-text-font">
Al-Sukhni, W., Rothenmund, H., Eppel Borgida, A., Zogopoulos, G., O'Shea, A.-M., Pollett, A., Gallinger, S.
<strong>Germline BRCA1 mutations predispose to pancreatic adenocarcinoma.</strong>
Hum. Genet. 124: 271-278, 2008.
[PubMed: 18762988]
[Full Text: https://doi.org/10.1007/s00439-008-0554-0]
</p>
</li>
<li>
<p class="mim-text-font">
Golan, T., Hammel, P., Reni, M., Van Cutsem, E., Macarulla, T., Hall, M. J., Park, J.-O., Hochhauser, D., Arnold, D., Oh, D.-Y., Reinacher-Schick, A., Tortora, G., Algul, H., O'Reilly, E. M., McGuinness, D., Cui, K. Y., Schlienger, K., Locker, G. Y., Kindler, H. L.
<strong>Maintenance olaparib for germline BRCA-mutated metastatic pancreatic cancer.</strong>
New Eng. J. Med. 381: 317-327, 2019.
[PubMed: 31157963]
[Full Text: https://doi.org/10.1056/NEJMoa1903387]
</p>
</li>
<li>
<p class="mim-text-font">
Miki, Y., Swensen, J., Shattuck-Eidens, D., Futreal, P. A., Harshman, K., Tavtigian, S., Liu, Q., Cochran, C., Bennett, L. M., Ding, W., Bell, R., Rosenthal, J., and 33 others.
<strong>A strong candidate for the breast and ovarian cancer susceptibility gene BRCA1.</strong>
Science 266: 66-71, 1994.
[PubMed: 7545954]
[Full Text: https://doi.org/10.1126/science.7545954]
</p>
</li>
<li>
<p class="mim-text-font">
Zhen, D. B., Rabe, K. G., Gallinger, S., Syngal, S., Schwartz, A. G., Goggins, M. G., Hruban, R. H., Cote, M. L., McWilliams, R. R., Roberts, N. J., Cannon-Albright, L. A., Li, D., Moyes, K., Wenstrup, R. J., Hartman, A.-R., Seminara, D., Klein, A. P., Petersen, G. M.
<strong>BRCA1, BRCA2, PALB2, and CDKN2A mutations in familial pancreatic cancer: a PACGENE study.</strong>
Genet. Med. 17: 569-577, 2015.
[PubMed: 25356972]
[Full Text: https://doi.org/10.1038/gim.2014.153]
</p>
</li>
</ol>
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Contributors:
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<span class="mim-text-font">
Ada Hamosh - updated : 08/12/2019<br>Ada Hamosh - updated : 10/19/2015
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Creation Date:
</span>
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<div class="col-lg-6 col-md-6 col-sm-6 col-xs-6">
<span class="mim-text-font">
Matthew B. Gross : 10/31/2011
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Edit History:
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<span class="mim-text-font">
alopez : 08/12/2019<br>alopez : 10/19/2015<br>alopez : 10/19/2015<br>mgross : 10/31/2011
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