H3K27M突变儿童弥漫性胶质瘤个性化治疗数据表1

数据集获取（Figshare）

• 源站标题：Table_1_Personalized Treatment of H3K27M-Mutant Pediatric Diffuse Gliomas Provides Improved Therapeutic Opportunities.XLSX
• DOI：10.3389/fonc.2019.01436.s001
• 发布日期：2020-01-10
• 文件数：1 个
• 文件总体积：13.61 KB
• 源站页面：https://frontiersin.figshare.com/articles/dataset/Table_1_Personalized_Treatment_of_H3K27M-Mutant_Pediatric_Diffuse_Gliomas_Provides_Improved_Therapeutic_Opportunities_XLSX/11564712

命令行下载

curl -L -o "Table_1_Personalized Treatment of H3K27M-Mutant Pediatric Diffuse Gliomas Provides Improved Therapeutic Opportunities.XLSX" "https://ndownloader.figshare.com/files/20794770"

文件清单

• Table_1_Personalized Treatment of H3K27M-Mutant Pediatric Diffuse Gliomas Provides Improved Therapeutic Opportunities.XLSX（13.61 KB）

文件总体积 13.61 KB（源站 API 实测）。

数据说明

Diffuse gliomas with K27M histone mutations (H3K27M glioma) are generally characterized by a fatal prognosis, particularly affecting the pediatric population. Based on the molecular heterogeneity observed in this tumor type, personalized treatment is considered to substantially improve therapeutic options. Therefore, clinical evidence for therapy, guided by comprehensive molecular profiling, is urgently required. In this study, we analyzed feasibility and clinical outcomes in a cohort of 12 H3K27M glioma cases treated at two centers. Patients were subjected to personalized treatment either at primary diagnosis or disease progression and received backbone therapy including focal irradiation. Molecular analyses included whole-exome sequencing of tumor and germline DNA, RNA-sequencing, and transcriptomic profiling. Patients were monitored with regular clinical as well as radiological follow-up. In one case, liquid biopsy of cerebrospinal fluid (CSF) was used. Analyses could be completed in 83% (10/12) and subsequent personalized treatment for one or more additional pharmacological therapies could be recommended in 90% (9/10). Personalized treatment included inhibition of the PI3K/AKT/mTOR pathway (3/9), MAPK signaling (2/9), immunotherapy (2/9), receptor tyrosine kinase inhibition (2/9), and retinoic receptor agonist (1/9). The overall response rate within the cohort was 78% (7/9) including one complete remission, three partial responses, and three stable diseases. Sustained responses lasting for 28 to 150 weeks were observed for cases with PIK3CA mutations treated with either miltefosine or everolimus and additional treatment with trametinib/dabrafenib in a case with BRAFV600E mutation. Immune checkpoint inhibitor treatment of a case with increased tumor mutational burden (TMB) resulted in complete remission lasting 40 weeks. Median time to progression was 29 weeks. Median overall survival (OS) in the personalized treatment cohort was 16.5 months. Last, we compared OS to a control cohort (n = 9) showing a median OS of 17.5 months. No significant difference between the cohorts could be detected, but long-term survivors (>2 years) were only present in the personaliz

许可

CC BY 4.0

引用

学术使用请引用 DOI 10.3389/fonc.2019.01436.s001（source: https://frontiersin.figshare.com/articles/dataset/Table_1_Personalized_Treatment_of_H3K27M-Mutant_Pediatric_Diffuse_Gliomas_Provides_Improved_Therapeutic_Opportunities_XLSX/11564712）。