关键词: BRAF fusion MEK inhibitor children low-grade gliomas response trametinib

来  源:   DOI:10.3389/fonc.2024.1381354   PDF(Pubmed)

Abstract:
Dissemination in pediatric low-grade glioma may occur in about 4%-10% of patients according to retrospective cohort studies. Due to its low incidence, there is no consensus on treatment for these patients. According to the constitutional activation of the MAPK/ERK pathway in these tumors, MEK inhibitors such as trametinib have been used successfully in the relapsed setting. Skin toxicity is frequent in patients receiving trametinib, normally mild to moderate, but sometimes severe, needing to discontinue the drug, limiting the efficacy in the tumor. There is not much information in the literature regarding whether reducing the dose of trametinib is able to maintain efficacy while, at the same time, decreasing toxicity. Here, we present an adolescent, with severe skin toxicity, whose trametinib dose was reduced by 50% and efficacy on the tumor continued while skin toxicity significantly decreased.
摘要:
根据回顾性队列研究,儿科低度胶质瘤的传播可能发生在约4%-10%的患者中。由于发病率低,对于这些患者的治疗尚无共识.根据MAPK/ERK通路在这些肿瘤中的构成激活,MEK抑制剂如曲美替尼已成功用于复发情况。接受曲美替尼的患者皮肤毒性很常见,通常轻度至中度,但有时严重,需要停止药物,限制肿瘤的疗效。文献中关于减少曲美替尼的剂量是否能够维持疗效的信息不多,同时,降低毒性。这里,我们介绍一个青少年,有严重的皮肤毒性,曲美替尼剂量减少了50%,对肿瘤的疗效持续,而皮肤毒性显着降低。
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