PDF(Pubmed)
Abstract：
Neuroendocrine carcinoma (NEC) of the gallbladder origin is particularly rare, accounting for only 0.38% of primary malignancies of the gallbladder, and standard therapies are limited. The MET gene encodes the tyrosine kinase receptor, c-Met. Pathogenic variants of MET, such as MET exon 14 skipping and MET amplification, result in excessive downstream signaling that promotes tumor progression. A MET inhibitor, capmatinib, blocks signaling of c-Met and has been approved by the Food and Drug Administration for non-small cell lung cancer with MET exon 14 skipping. The effectiveness of capmatinib has been reported in other cancers with MET amplification, but NEC with MET variants has not been reported. Here, we present a case of a 72-year-old woman with NEC of the gallbladder with multiple liver and lymph node metastases, who was resistant to conventional chemotherapy including carboplatin plus etoposide as first-line treatment and irinotecan as second-line treatment, but she responded to capmatinib. After 6 weeks of treatment, CT scan showed a partial response (80% reduction in size), but after 13 weeks, regrowth of liver metastasis was observed. Herein, we report a meaningful efficacy of capmatinib to the patient of NEC of the gallbladder origin with MET amplification.
摘要：
胆囊起源的神经内分泌癌（NEC）特别罕见，仅占原发性胆囊恶性肿瘤的0.38%，标准疗法是有限的。MET基因编码酪氨酸激酶受体，C-Met.MET的致病变异，如MET外显子14跳跃和MET扩增，导致过度的下游信号，促进肿瘤进展。一种MET抑制剂，卡马替尼,阻断c-Met的信号传导，并已被食品和药物管理局批准用于具有MET外显子14跳跃的非小细胞肺癌。据报道，卡马替尼在其他具有MET扩增的癌症中的有效性，但尚未报道具有MET变体的NEC。这里,我们介绍了一个72岁的女性胆囊的NEC与多个肝脏和淋巴结转移，对常规化疗耐药，包括卡铂加依托泊苷作为一线治疗和伊立替康作为二线治疗，但她对卡马替尼有反应.治疗6周后,CT扫描显示部分反应（尺寸减少80％），但13周后,观察到肝转移的再生长。在这里，我们报告了卡马替尼对MET扩增的胆囊源性NEC患者的有意义疗效.
