Yamamura Shogo, Kanai Masashi, Takeuchi Yasuhide, Okita Natsuko, Kondo Tomohiro, Yoshioka Masahiro, Matsubara Junichi, Matsumoto Shigemi, Muto Manabu
Department of Clinical Oncology, Kyoto University Hospital, Kyoto, Japan.
Department of Diagnostic Pathology, Kyoto University Hospital, Kyoto, Japan.
Int Cancer Conf J. 2024 Jan 2;13(2):83-87. doi: 10.1007/s13691-023-00643-5. eCollection 2024 Apr.
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 gene encodes the tyrosine kinase receptor, c-Met. Pathogenic variants of , such as exon 14 skipping and 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 exon 14 skipping. The effectiveness of capmatinib has been reported in other cancers with amplification, but NEC with 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 amplification.
胆囊来源的神经内分泌癌(NEC)极为罕见,仅占胆囊原发性恶性肿瘤的0.38%,且标准治疗方法有限。该基因编码酪氨酸激酶受体c-Met。其致病性变异,如14号外显子跳跃和扩增,会导致过度的下游信号传导,从而促进肿瘤进展。一种MET抑制剂卡马替尼可阻断c-Met信号传导,已被美国食品药品监督管理局批准用于治疗存在14号外显子跳跃的非小细胞肺癌。卡马替尼在其他存在扩增的癌症中的有效性已有报道,但存在变异的NEC尚未见报道。在此,我们报告一例72岁患有胆囊NEC并伴有多处肝转移和淋巴结转移的女性病例,该患者对包括卡铂加依托泊苷作为一线治疗以及伊立替康作为二线治疗的传统化疗均耐药,但对卡马替尼有反应。治疗6周后,CT扫描显示部分缓解(肿瘤大小缩小80%),但13周后,观察到肝转移灶复发。在此,我们报告卡马替尼对具有扩增的胆囊来源NEC患者具有显著疗效。