Lambrechts C, Chhajlani S, Islam O, Verbruggen L, De Weerdt C, Mariën L, Simoens M, Ulenaers M, Galdermans D, Demey W, Van der Massen I, Lybaert W, Vandamme T
Department of Gastro-Enterology, Heilig Hart Ziekenhuis, Mol, Belgium.
Department of Medical Oncology, Antwerp University Hospital ? NETwerk, Antwerp, Belgium.
Acta Gastroenterol Belg. 2025 Apr-Jun;88(2):119-127. doi: 10.51821/88.1.13722.
Neuroendocrine neoplasms (NEN) are rare tumors originating from neuroendocrine cells, commonly found in the gastrointestinal tract and the pulmonary tract. Metastatic welldifferentiated neuroendocrine tumors (NET) grade 3 present unique challenges, as they are positioned between the more indolent NET grade 1-2 and the aggressive neuroendocrine carcinomas (NEC). Due to the scarcity of data regarding the optimal systemic treatment for metastatic NET grade 3 and aggressive NET grade 2 subtypes, guidelines remain inconclusive. This retrospective study analyzed data from the NETwerk database, encompassing patients treated with the capecitabinetemozolomide (CAPTEM) regimen between June 2016 and January 2024. The cohort included patients with NET grades 1-3 and NEC. The study focused on assessing the efficacy and safety of CAPTEM. In total, data from 36 patients was analyzed. The median progression-free survival (mPFS) was 13 months, and median overall survival (mOS) was 17 months. Overall response rate (ORR) was 25.8%, and the disease control rate (DCR) was 67.7%. NET grade 2 patients had the highest mPFS, while NET grade 3 exhibited the most favorable mOS. Subgroup analysis showed that panNEN had superior mPFS and mOS compared to other primary tumor sites, with significant differences in mOS based on NEN type. Safety analysis in 20 patients indicated good tolerance and safety . CAPTEM is an efficient and safe regimen for metastatic NEN, with promising outcomes in NET grade 2-3 patients. The promising findings pave the way for further exploration into various aspects of CAPTEM, to better define its position in the therapeutic landscape of NEN.
神经内分泌肿瘤（NEN）是起源于神经内分泌细胞的罕见肿瘤，常见于胃肠道和呼吸道。转移性3级高分化神经内分泌肿瘤（NET）带来了独特的挑战，因为它们介于惰性更强的1-2级NET和侵袭性神经内分泌癌（NEC）之间。由于关于转移性3级NET和侵袭性2级NET亚型的最佳全身治疗的数据稀缺，指南仍无定论。这项回顾性研究分析了NETwerk数据库中的数据，涵盖了2016年6月至2024年1月期间接受卡培他滨-替莫唑胺（CAPTEM）方案治疗的患者。该队列包括1-3级NET和NEC患者。该研究重点评估CAPTEM的疗效和安全性。总共分析了36例患者的数据。中位无进展生存期（mPFS）为13个月，中位总生存期（mOS）为17个月。总缓解率（ORR）为25.8%，疾病控制率（DCR）为67.7%。2级NET患者的mPFS最高，而3级NET的mOS最有利。亚组分析显示，与其他原发肿瘤部位相比，泛神经内分泌肿瘤（panNEN）的mPFS和mOS更高，基于NEN类型的mOS存在显著差异。对20例患者的安全性分析表明耐受性和安全性良好。CAPTEM是一种治疗转移性NEN的有效且安全的方案，在2-3级NET患者中取得了有前景的结果。这些有前景的发现为进一步探索CAPTEM的各个方面铺平了道路，以更好地确定其在NEN治疗格局中的地位。
