针对新确诊脉络丛癌儿童的骨髓消融巩固化疗和分子靶向治疗：一项进行中的工作

IF 3.7 Q1 CLINICAL NEUROLOGY

Neuro-oncology advances Pub Date : 2024-07-05 DOI:10.1093/noajnl/vdae109

Maxim Yankelevich, W. Zaky, L. Lafay-Cousin, Diana Osorio, Jenny K Adamski, U. Kordes, Jonathan L Finlay, Michael Prados, Sabine Mueller

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引用次数: 0

摘要

脉络丛癌（CPC）是以 TP53 功能缺失和生存率低为特征的儿童早期癌症。我们正在分析接受化疗和骨髓消融化疗（HDCx）巩固治疗的最大一组 CPC 患者的 TP53 状态、生存率和二次癌症数据。此外，我们还讨论了对 CPC 患者进行靶向治疗的理由。目前，13 名患有李-弗劳米尼综合征（LFS）相关 CPC 的患者中，有 8 人接受了治疗并继续保持无 CPC 状态，这表明 HDCx 提高了 TP53 突变 CPC 幼儿的无 CPC 生存率。这些数据证明了将 HDCx 纳入计划中的 TP53 突变 CPC 患儿前瞻性国际试验是正确的。

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Marrow-ablative Consolidation Chemotherapy and Molecular Targeted Therapy Delivered in a Risk-adapted Manner for Newly Diagnosed Children with Choroid Plexus Carcinoma: A Work in Progress

Choroid plexus carcinomas (CPC) are early childhood cancers characterized by loss of TP53 function and poor survival. We are analyzing data on TP53 status, survival and second cancers from the largest cohort of CPC receiving chemotherapy followed by consolidation with marrow-ablative chemotherapy (HDCx). Additionally, we discuss the rationale for targeted therapies for CPC patients. Currently, eight of 13 with Li-Fraumeni Syndrome (LFS)-associated CPC were treated and continued CPC-free, indicating that HDCx improves CPC-free survival in young children with TP53-mutated CPC. These data justify the inclusion of HDCx in the planned prospective international trial for children with TP53-mutated CPC.

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来源期刊

Neuro-oncology advances

CiteScore

6.20

自引率

0.00%

发文量

审稿时长

12 weeks