diff --git a/content/manual-references.json b/content/manual-references.json index 401ebd93..8eb2f5d7 100644 --- a/content/manual-references.json +++ b/content/manual-references.json @@ -3718,5 +3718,146 @@ "PMCID": "PMC3163565", "PMID": "21816040", "id": "doi:10.1186/1471-2105-12-323" + }, + { + "publisher": "American Association for Cancer Research (AACR)", + "issue": "18", + "abstract": "Abstract\n \n Purpose:\n PNOC003 is a multicenter precision medicine trial for children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG).\n \n \n Patients and Methods:\n 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).\n \n \n Results:\n 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.\n \n \n Conclusions:\n Upfront treatment-naïve biopsy provides insight into clinically relevant molecular alterations and prognostic biomarkers for H3K27-altered DIPGs.\n ", + "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" } ]