Puget Stephanie, Blauwblomme Thomas, Grill Jacques
From the Necker Enfants Malades Hospital, Université Paris Descartes, Sorbonne Paris Cité, France; Gustave Roussy Cancer Institute, Universite Paris Sud, Villejuif, France.
Am Soc Clin Oncol Educ Book. 2012:629-33. doi: 10.14694/EdBook_AM.2012.32.59.
Diffuse intrinsic pontine gliomas (DIPGs), with a median survival of 9 months, represent the biggest therapeutic challenge in pediatric neuro-oncology. Despite many clinical trials, no major improvements in treatment have been made over the past 30 years. In most cases, biopsy is not needed for diagnosis because DIPG diagnosis is based on a typical clinical picture with radiologic evidence on magnetic resonance imaging. Therefore, little data on newly diagnosed DIPG have been published and are confounded by including autopsy (i.e., postradiation therapy) cases. In most cancers, advancing to cure has been linked to the discovery of relevant biomarkers, only found by access to tissue. Therefore, to further understand the biology of DIPG, fresh tissue samples must be obtained at diagnosis. However, most neurosurgical teams are reluctant to perform biopsy in pediatric patients, citing potential risks and lack of direct benefit. Yet, in reviewing 90 patients with and the published data on brainstem biopsy, these procedures have a diagnostic yield and morbidity and mortality rates similar to those reported for other brain locations. In addition, the quality and quantity of the material obtained confirm the diagnosis and inform an extended molecular screen, including biomarker study-information important to designing next-generation trials with targeted agents. Stereotactic biopsies can be considered a safe procedure in well-trained neurosurgical teams and could be incorporated in well-defined protocols for patients with DIPG.
弥漫性脑桥内生型胶质瘤（DIPG）的中位生存期为9个月，是小儿神经肿瘤学中最大的治疗挑战。尽管进行了许多临床试验，但在过去30年里治疗方面并未取得重大进展。在大多数情况下，诊断不需要活检，因为DIPG的诊断基于典型的临床表现以及磁共振成像的放射学证据。因此，关于新诊断DIPG的资料很少发表，而且由于纳入了尸检（即放疗后）病例而变得混淆不清。在大多数癌症中，实现治愈与发现相关生物标志物有关，而这些生物标志物只有通过获取组织才能发现。因此，为了进一步了解DIPG的生物学特性，必须在诊断时获取新鲜组织样本。然而，大多数神经外科团队因潜在风险和缺乏直接益处而不愿对小儿患者进行活检。然而，在回顾90例接受脑干活检的患者及已发表的数据时发现，这些手术的诊断率以及发病率和死亡率与其他脑区报告的情况相似。此外，所获取材料的质量和数量有助于确诊并为扩展的分子筛查提供信息，包括生物标志物研究——这对于设计使用靶向药物的下一代试验很重要。在训练有素的神经外科团队中，立体定向活检可被视为一种安全的手术，并且可以纳入针对DIPG患者的明确方案中。
