Trametinib-induced Left Ventricular Dysfunction in a Child With Relapsed Neuroblastoma
Trametinib-induced Left Ventricular Dysfunction in a Child With Relapsed Neuroblastoma
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DOI：
10.1097/mph.0000000000000364
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发表时间：
2015-08-01
影响因子：
1.2
通讯作者：
Grana, Nanette
中科院分区：
文献类型：
--
作者：
Modak, Shakeel;Asante-Korang, Alfred;Grana, Nanette
MEK 抑制剂曲美替尼 (Trametinib) 在全球范围内获批用于治疗携带 BRAF 突变的转移性黑色素瘤。迄今为止还没有关于其在儿童中使用的报道。对一名患有进行性、多次复发的 4 期神经母细胞瘤的 11 岁男性复发肿瘤样本进行外显子组测序，结果显示 NRASQ61K 突变。在证明心脏功能正常后，他开始口服曲美替尼。治疗第 13 天，超声心动图显示中度左心室功能障碍。第 15 天停止曲美替尼并开始口服赖诺普利。停用曲美替尼后 37 天，左心室功能恢复至基线。然而，神经母细胞瘤显示出进一步进展。
The MEK inhibitor trametinib is globally approved for metastatic melanoma harboring BRAF mutations. There are no reports thus far on its use in children. Exome sequencing on a relapsed tumor sample from an 11-year-old male with progressive, multiply relapsed stage 4 neuroblastoma revealed NRASQ61K mutation. After demonstration of normal cardiac function, he was started on oral trametinib. On day 13 of treatment, echocardiogram showed moderate left ventricular dysfunction. Trametinib was discontinued on day 15 and oral lisinopril was started. Left ventricular function recovered to baseline 37 days after discontinuing trametinib. However, neuroblastoma showed further progression.