首页> 美国卫生研究院文献>Neuro-Oncology >QOL-28. CUTANEOUS REACTIONS TO TARGETED THERAPIES IN CHILDREN WITH CNS TUMORS: A CROSS-SECTIONAL STUDY
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QOL-28. CUTANEOUS REACTIONS TO TARGETED THERAPIES IN CHILDREN WITH CNS TUMORS: A CROSS-SECTIONAL STUDY

机译:QOL-28。具有中枢神经系统肿瘤的儿童对靶向治疗的同步反应:横断面研究

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摘要

The MAPK and mTOR pathways play important roles in cell proliferation. BRAF, MEK, and mTOR inhibitors are novel therapies that inhibit these pathways and are increasingly utilized in the pediatric population with CNS tumors. While associated cutaneous reactions are common, data in children are sparse. The objective of this cross-sectional study was to investigate the frequency and type of cutaneous reactions in children with brain tumors treated with BRAF, MEK, and mTOR inhibitor monotherapy. Patients less than 21 years of age with CNS tumors on BRAF V600E (dabrafenib), MEK (trametinib), or mTOR inhibitors (everolimus) were enrolled over a one-year period. Twenty-two patients were enrolled, including 11 on MEK inhibitors, 6 on BRAF V600E inhibitors, and 5 on mTOR inhibitors. Median age at study visit was 12 years (3-19 yrs). Ninety-six percent (21/22) of patients had at least one skin finding. In patients on the MEK inhibitors (11), common reactions were follicular eruptions (9), xerosis (8), paronychia (4), and alopecia (2). In patients on the BRAF inhibitors (6), common reactions included follicular eruptions (6), hand-foot syndrome (5), and xerosis (3). In patients on the mTOR inhibitors (5), reactions included xerosis (4), paronychia (1), and follicular eruption (1). Two patients on the MEK inhibitors and one patient on the BRAF inhibitor required therapy cessation due to severe cutaneous reactions. Treatment was recommended for study participants as indicated. The study demonstrates that cutaneous reactions were extremely common but generally well-tolerated, though for a small subset, their reactions were severe and required therapy cessation.
机译:MAPK和mTOR途径在细胞增殖中起重要作用。 BRAF,MEK和mTOR抑制剂是抑制这些途径的新型疗法,并越来越多地用于患有CNS肿瘤的儿科人群。尽管相关的皮肤反应很普遍,但儿童的数据却很少。这项横断面研究的目的是研究接受BRAF,MEK和mTOR抑制剂单一疗法治疗的脑瘤患儿皮肤反应的频率和类型。入选了在21年以下使用BRAF V600E(dabrafenib),MEK(曲美替尼)或mTOR抑制剂(依维莫司)治疗CNS肿瘤的患者,为期一年。招募了22位患者,其中11位使用MEK抑制剂,6位使用BRAF V600E抑制剂,5位使用mTOR抑制剂。研究访问的中位年龄为12岁(3-19岁)。 96%(21/22)的患者患有至少一种皮肤。使用MEK抑制剂的患者(11)的常见反应是卵泡爆发(9),干燥症(8),甲沟炎(4)和脱发(2)。使用BRAF抑制剂的患者(6),常见反应包括卵泡爆发(6),手足综合征(5)和干燥症(3)。使用mTOR抑制剂的患者(5)的反应包括干燥症(4),甲状旁腺(1)和卵泡疹(1)。由于严重的皮肤反应,有两名MEK抑制剂患者和一名BRAF抑制剂患者需要停止治疗。如所建议的,建议对研究参与者进行治疗。该研究表明,皮肤反应极为普遍,但一般耐受性良好,尽管对于一小部分患者,皮肤反应严重,需要停止治疗。

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