Neuro Oncol：MEK-1/2抑制剂selumetinib明显改善复发性/进展性儿童低级别胶质瘤预后

2021-02-26 Nebula MedSci原创

儿童低级别胶质瘤(pLGGs)是儿童最常见的脑肿瘤

儿童低级别胶质瘤(pLGGs)是儿童最常见的脑肿瘤。无进展生存率(PFS)远低于总体生存率，强调了替代治疗的必要性。散发性(无1型神经纤维瘤病)视路和下丘脑胶质瘤(OPHGs)通常是多重复发的，导致明显的视力障碍。

近期，研究人员对儿童低级别胶质瘤患儿的功能性预后进行了优先排序。

本文报道了对复发/进展性OPHGs儿童进行PBTC 2期试验的结果，该试验评估了MEK-1/2抑制剂司美替尼(selumetinib，AZD6244，ARRY-142886)用于这类患者的疗效。PBTC-029的第4层纳入散发性复发/进展性OPHGs患者，以推荐的2期剂量(25 mg/m2，2/日)治疗，最多26个疗程。

共有25位复合条件的可评估的患者被纳入分析，既往疗程中位数为4个(1-11)。在这25位中，有6位(24%)获得部分缓解，14位(56%)的病情稳定，5位(20%)的病情发生进展。中位疗程为26个(2-26)，其中14位患者完成了全部26个疗程。

2年PFS为78±8.5%。25位患者中19位的视力可评估，其中4位(21%)患者的视力有所提高，13位(68%)的视力稳定，2位(11%)患者的视力进一步下降。19位患者中有5位(26%)的视野有所改善，14位(74%)的视野稳定。

最常见的不良反应是1/2级CPK升高、贫血、腹泻、头痛、恶心/呕吐、乏力、AST和ALT升高、低蛋白血症和皮疹。

综上，司美替尼对复发/进展性OPHGs患儿的耐受性良好，还可改善影像学缓解、PFS和视力预后，并延长疾病稳定期。

原始出处：

Fangusaro Jason,Onar-Thomas Arzu,Poussaint Tina Young et al. A Phase 2 Trial of Selumetinib in Children with Recurrent Optic Pathway and Hypothalamic Low-Grade Glioma without NF1: A Pediatric Brain Tumor Consortium Study. Neuro Oncol, 2021, https://doi.org/10.1093/neuonc/noab047

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2021-12-07 12498ebem28暂无昵称

#低级别胶质瘤#

74 0
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2021-11-13 hb2008ye

#复发性#

73 0
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2021-10-27 minlingfeng

#Oncol#

56 0
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2021-12-15 naiwu77

#MEK#

76 0
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2021-12-18 liuquan

#2抑制剂#

67 0
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2021-09-01 仁心济世

#MET#

69 0
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2021-11-21 jklm09

#抑制剂#

68 0
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2021-02-27 anti-cancer

谢谢梅斯分享这么多精彩信息

75 0

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Neuro Oncol：MEK-1/2抑制剂selumetinib明显改善复发性/进展性儿童低级别胶质瘤预后

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