Department of Neurological Surgery, NewYork-Presbyterian Weill Cornell Medicine, New York, New York, USA.
Department of Neurological Surgery, NewYork-Presbyterian Weill Cornell Medicine, New York, New York, USA.
World Neurosurg. 2024 Oct;190:582-585. doi: 10.1016/j.wneu.2024.06.146.
The field of neuro-oncology has seen significant advances that have allowed the expansion of the therapeutic armamentarium. Nevertheless, overall outcomes have not improved significantly particularly for high-grade tumors. The relative rarity of these pathologies in the pediatric population limits the capacity to design large-scale, multicenter, randomized clinical trials. The emergence of precision medicine as a direct result of better, more widespread genetic and molecular testing affords clinicians the possibility of envisioning new clinical trial paradigms. Each patient becomes their own singular trial receiving the most tailored treatment at every stage of their disease while serving as their own controls. Although limitations still exist for the widespread adoption of these technologies and incorporation into standard clinical care, the prospect of being able to offer directed therapies and monitor disease progression based on single-patient testing represents a much-needed paradigm shift in neuro-oncology.
神经肿瘤学领域取得了重大进展，使得治疗手段得到了扩展。然而，整体治疗效果并没有显著改善，尤其是对于高级别肿瘤。这些儿科罕见病限制了大规模、多中心、随机临床试验的设计能力。精准医学的出现是更好、更广泛的遗传和分子检测的直接结果，为临床医生提供了设想新临床试验模式的可能性。每个患者都成为自己的独特试验，在疾病的每个阶段接受最适合的治疗，同时作为自己的对照。尽管这些技术的广泛应用和纳入标准临床护理仍存在局限性，但能够根据单患者检测提供靶向治疗并监测疾病进展的前景代表了神经肿瘤学中急需的范式转变。
