A cornerstone of paediatric neurological practice is to advise on prognosis and contribute to the risk versus benefit evaluation of interventions in a range of early onset, potentially treatable disorders. However, the decisionmaking paradigms used in vein of Galen malformation (VGM)—a previously lethal condition, now potentially curable with high-risk embolization—have been somewhat unusual. Historically, the Bicetre score, an indicator of systemic organ dysfunction, was used to triage patients. Its categories (emergency embolization, delayed embolization, no treatment) were pragmatic and intended to give some indication of survivability at a time when there were very few clinicians able to treat these children who sometimes needed to be transported long distances. However, as discussed by Taffin et al., this categorization is no longer current and has been superseded by more conventional approaches. Beyond survival, few groups have examined longer-term outcomes in treated VGM, nor its predictors or correlates. As in many conditions involving early brain injury, a key finding of Taffin et al. was that neurocognitive difficulties emerged with age that were not apparent earlier. Unfortunately, there were few predictors of outcome; although in this and other cohorts, severe cardiac failure and supra-systemic pulmonary artery pressure unsurprisingly predicted death. Imaging correlates of outcome were not examined in detail and it is important to note that a proportion of children with abnormal imaging will be unimpaired. This paper adds to the body of literature highlighting the importance of long-term evaluation and the concept of emergent deficits in children with static brain injury. The practical significance of this is that early focus is on survival, then on relatively gross, usually motor, milestones. By the time impairments in higher level functions emerge (e.g. in executive function), the child is often not engaged with medical services and could struggle to achieve their potential. While the Bicetre group present their institutional guidelines, their approach is not universally adopted in the interventional neuroradiology community. Currently, a multidisciplinary approach is advocated, incorporating brain imaging and aligned with decision-making paradigms in other paediatric conditions (e.g. congenital cardiac disease), and incorporating intensive care as well as cardiac, genetic, and paediatric care. The potential to manipulate endovascular treatment—other than its timing or management of hydrocephalus—is limited. There is, however, potential to manipulate physiological parameters especially in intensive care. Thus, a better understanding of factors that need to be controlled aggressively (e.g. blood pressure in the acute phase) could inform the overall management approach. Power is a major issue in study of rare diseases and there is potential for important biases in sample selection. One striking feature of these data are the relatively small proportion of the whole group who were recruited; presumably due to the requirement of having at least one Francophone parent. In other settings, the economics of accessing treatment will influence the demographics of the cohort. Finally, local antenatal care policies would influence the proportion of patients identified in utero versus having an early symptomatic presentation; certainly the proportion diagnosed antenatally reported here was much higher than that in the UK. While individual clinicians may be reluctant to alter their decision-making, a registry of all cases (including those where pregnancy was terminated or death occurred in utero) with a standardized and systematized analysis of imaging, particularly incorporating advanced imaging techniques, is likely the only way to make real progress in understanding this disease and the factors influencing outcome. Moreover, an agreement about a standardized and comprehensive neurocognitive assessment protocol would enable some meaningful conclusion to be made about outcome that would be invaluable in counselling families.

中文翻译：

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盖伦静脉畸形：超越生死

儿科神经病学实践的基石是就预后提供建议，并有助于对一系列早期发病、可能可治疗的疾病进行干预的风险与收益评估。然而，Galen 畸形 (VGM) 静脉使用的决策范式——以前是一种致命的疾病，现在可能通过高风险栓塞治疗——有些不寻常。历史上，Bicetre 评分是全身器官功能障碍的指标，用于对患者进行分类。它的类别（紧急栓塞、延迟栓塞、无治疗）是实用的，旨在在很少有临床医生能够治疗这些有时需要长途运输的儿童的情况下给出一些生存能力的指示。然而，正如 Taffin 等人所讨论的那样，这种分类不再流行，已被更传统的方法所取代。除了生存率之外，很少有小组研究了治疗 VGM 的长期结果，也没有研究其预测因素或相关性。与涉及早期脑损伤的许多情况一样，Taffin 等人的一个关键发现。是随着年龄的增长而出现的神经认知困难在早期并不明显。不幸的是，几乎没有预测结果的因素。尽管在这个队列和其他队列中，严重的心力衰竭和系统上肺动脉压力不出所料地预示着死亡。影像学与结果的相关性没有详细检查，重要的是要注意一部分影像学异常的儿童不会受损。这篇论文补充了大量文献，强调了长期评估的重要性和静态脑损伤儿童突发性缺陷的概念。这样做的实际意义在于，早期关注的是生存，然后是相对粗略的，通常是运动的里程碑。当更高层次的功能出现障碍（例如执行功能）时，孩子通常不会参与医疗服务，并且可能难以发挥他们的潜力。虽然 Bicetre 小组提出了他们的机构指南，但他们的方法并未在介入神经放射学界普遍采用。目前，提倡采用多学科方法，结合脑成像并与其他儿科疾病（例如先天性心脏病）的决策范例保持一致，并结合重症监护以及心脏、遗传和儿科护理。操纵血管内治疗的潜力——除了脑积水的时机或管理——是有限的。然而，有可能操纵生理参数，尤其是在重症监护中。因此，更好地了解需要积极控制的因素（例如急性期的血压）可以为整体管理方法提供信息。功效是罕见病研究中的一个主要问题，样本选择可能存在重要偏差。这些数据的一个显着特点是整个群体中被招募的比例相对较小；大概是因为要求至少有一个法语国家的父母。在其他设置中，获得治疗的经济学将影响该队列的人口统计数据。最后，当地的产前保健政策会影响在子宫内确诊的患者与早期症状表现的患者比例；当然，这里报告的产前诊断的比例远高于英国。虽然个别临床医生可能不愿意改变他们的决策，但对所有病例（包括终止妊娠或在子宫内发生死亡的病例）进行标准化和系统化的成像分析，特别是结合先进的成像技术的登记，可能是唯一的在了解这种疾病和影响结果的因素方面取得真正进展的方法。而且，