Pediatric Brainstem Gliomas: New Understanding Leads to Potential New Treatments for Two Very Different Tumors
Pediatric Brainstem Gliomas: New Understanding Leads to Potential New Treatments for Two Very Different Tumors
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DOI：
10.1007/s11912-014-0436-7
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发表时间：
2015-03-01
影响因子：
4.7
通讯作者：
Kieran, Mark W.
中科院分区：
文献类型：
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作者：
Green, Adam L.;Kieran, Mark W.
儿童脑干胶质瘤包括低级别局灶性脑干胶质瘤（FBSG）和高级别弥漫性内源性脑桥胶质瘤（DIPG）。这些肿瘤的位置都位于大脑的一个重要且重要的区域，这给治疗带来了共同的挑战。但除此之外，这两种疾病在表现、生物学、治疗和预后方面都非常不同。 FBSG 通常出现症状超过 3 个月，而 DIPG 通常在症状出现后 3 个月内被诊断出来。手术仍然是 FBSG 的首选初始治疗方法，化疗用于治疗持续性、复发性或无法手术的疾病；相反，放射治疗是 DIPG 唯一已知有效的治疗方法。对这两种肿瘤的生物学理解的最新发展带来了新的治疗可能性。在 FBSG 中，与 BRAF 相关的两种遗传变化是大多数肿瘤的特征，其生物学效应的关键差异为靶向化疗的使用策略提供了信息。在 DIPG 中，广泛的组蛋白 H3 和 ACVR1 突变为有效的靶向治疗带来了新的希望。 FBSG 具有良好的预后，而 DIPG 的长期生存率可悲地仍然接近于零。在这篇综述中，我们涵盖了 FBSG 和 DIPG 的流行病学、生物学、表现、影像学特征、多模式治疗和预后，重点关注最新的生物学发现。
Pediatric brainstem gliomas include low-grade focal brainstemgliomas (FBSG) and high-grade diffuse intrinsic pontine gliomas (DIPG). These tumors share a crucial and eloquent area of the brain as their location, which carries common challenges for treatment. Otherwise, though, these two diseases are very different in terms of presentation, biology, treatment, and prognosis. FBSG usually present with greater than 3 months of symptoms, while DIPG are usually diagnosed within 3 months of symptom onset. Surgery remains the preferred initial treatment for FBSG, with chemotherapy used for persistent, recurrent, or inoperable disease; conversely, radiation is the only known effective treatment for DIPG. Recent developments in biological understanding of both tumors have led to new treatment possibilities. In FBSG, two genetic changes related to BRAF characterize the majority of tumors, and key differences in their biological effects are informing strategies for targeted chemotherapy use. In DIPG, widespread histone H3 and ACVR1 mutations have led to new hope for effective targeted treatments. FBSG has an excellent prognosis, while the long-term survival rate of DIPG tragically remains near zero. In this review, we cover the epidemiology, biology, presentation, imaging characteristics, multimodality treatment, and prognosis of FBSG and DIPG, with a focus on recent biological discoveries.