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Add clin cancer short name
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jashapiro committed Apr 26, 2023
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"PMCID": "PMC3163565",
"PMID": "21816040",
"id": "doi:10.1186/1471-2105-12-323"
},
{
"publisher": "American Association for Cancer Research (AACR)",
"issue": "18",
"abstract": "<jats:title>Abstract</jats:title>\n <jats:sec>\n <jats:title>Purpose:</jats:title>\n <jats:p>PNOC003 is a multicenter precision medicine trial for children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG).</jats:p>\n </jats:sec>\n <jats:sec>\n <jats:title>Patients and Methods:</jats:title>\n <jats:p>Patients (3–25 years) were enrolled on the basis of imaging consistent with DIPG. Biopsy tissue was collected for whole-exome and mRNA sequencing. After radiotherapy (RT), patients were assigned up to four FDA-approved drugs based on molecular tumor board recommendations. H3K27M-mutant circulating tumor DNA (ctDNA) was longitudinally measured. Tumor tissue and matched primary cell lines were characterized using whole-genome sequencing and DNA methylation profiling. When applicable, results were verified in an independent cohort from the Children's Brain Tumor Network (CBTN).</jats:p>\n </jats:sec>\n <jats:sec>\n <jats:title>Results:</jats:title>\n <jats:p>Of 38 patients enrolled, 28 patients (median 6 years, 10 females) were reviewed by the molecular tumor board. Of those, 19 followed treatment recommendations. Median overall survival (OS) was 13.1 months [95% confidence interval (CI), 11.2–18.4] with no difference between patients who followed recommendations and those who did not. H3K27M-mutant ctDNA was detected at baseline in 60% of cases tested and associated with response to RT and survival. Eleven cell lines were established, showing 100% fidelity of key somatic driver gene alterations in the primary tumor. In H3K27-altered DIPGs, TP53 mutations were associated with worse OS (TP53mut 11.1 mo; 95% CI, 8.7–14; TP53wt 13.3 mo; 95% CI, 11.8–NA; P = 3.4e−2), genome instability (P = 3.1e−3), and RT resistance (P = 6.4e−4). The CBTN cohort confirmed an association between TP53 mutation status, genome instability, and clinical outcome.</jats:p>\n </jats:sec>\n <jats:sec>\n <jats:title>Conclusions:</jats:title>\n <jats:p>Upfront treatment-naïve biopsy provides insight into clinically relevant molecular alterations and prognostic biomarkers for H3K27-altered DIPGs.</jats:p>\n </jats:sec>",
"DOI": "10.1158/1078-0432.ccr-22-0803",
"type": "article-journal",
"page": "3965-3978",
"source": "Crossref",
"title": "Upfront Biology-Guided Therapy in Diffuse Intrinsic Pontine Glioma: Therapeutic, Molecular, and Biomarker Outcomes from PNOC003",
"volume": "28",
"author": [
{
"given": "Cassie",
"family": "Kline"
},
{
"given": "Payal",
"family": "Jain"
},
{
"given": "Lindsay",
"family": "Kilburn"
},
{
"given": "Erin R.",
"family": "Bonner"
},
{
"given": "Nalin",
"family": "Gupta"
},
{
"given": "John R.",
"family": "Crawford"
},
{
"given": "Anu",
"family": "Banerjee"
},
{
"given": "Roger J.",
"family": "Packer"
},
{
"given": "Javier",
"family": "Villanueva-Meyer"
},
{
"given": "Tracy",
"family": "Luks"
},
{
"given": "Yalan",
"family": "Zhang"
},
{
"given": "Madhuri",
"family": "Kambhampati"
},
{
"given": "Jie",
"family": "Zhang"
},
{
"given": "Sridevi",
"family": "Yadavilli"
},
{
"given": "Bo",
"family": "Zhang"
},
{
"given": "Krutika S.",
"family": "Gaonkar"
},
{
"given": "Jo Lynne",
"family": "Rokita"
},
{
"given": "Adam",
"family": "Kraya"
},
{
"given": "John",
"family": "Kuhn"
},
{
"given": "Winnie",
"family": "Liang"
},
{
"given": "Sara",
"family": "Byron"
},
{
"given": "Michael",
"family": "Berens"
},
{
"given": "Annette",
"family": "Molinaro"
},
{
"given": "Michael",
"family": "Prados"
},
{
"given": "Adam",
"family": "Resnick"
},
{
"given": "Sebastian M.",
"family": "Waszak"
},
{
"given": "Javad",
"family": "Nazarian"
},
{
"given": "Sabine",
"family": "Mueller"
}
],
"container-title": "Clinical Cancer Research",
"container-title-short": "Clin Cancer Res",
"language": "en",
"issued": {
"date-parts": [
[
2022,
7,
19
]
]
},
"URL": "https://doi.org/gqh9gb",
"PMCID": "PMC9475246",
"PMID": "35852795",
"id": "doi:10.1158/1078-0432.ccr-22-0803"
}
]

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