Department of Medicine and Bioregulatory Science, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
Neuroendocrine Tumor Centre, Fukuoka Sanno Hospital, Fukuoka, Japan.
Jpn J Clin Oncol. 2024 Jun 1;54(6):647-657. doi: 10.1093/jjco/hyae026.
Streptozocin has been used to treat neuroendocrine tumors in Europe and the USA; however, its actual status in Japan has not been fully clarified owing to the rarity of this disease and the relatively recent approval of streptozocin in Japan.
We retrospectively analyzed 53 patients with gastroenteropancreatic neuroendocrine tumors who were treated with streptozocin-based chemotherapy at two Japanese hospitals between January 2004 and June 2023.
The overall response and disease control rates were 27.7 and 74.5%, respectively, and the median progression-free survival and overall survival were 7.1 and 20.3 months, respectively. Performance status ≥1 showed a significant negative correlation with progression-free survival, and performance status ≥1 and liver tumor burden ≥25% showed a significant negative correlation with overall survival. No significant differences were observed in the treatment response between pancreatic and gastrointestinal neuroendocrine tumors. No treatment-related serious adverse events were observed; however, 87.7% of patients expressed a decrease in the estimated glomerular filtration rate, which negatively correlated with the duration of streptozocin treatment (r = 0.43, P = 0.0020). In the streptozocin re-administration group (n = 5), no differences were found in efficacy between the initial and second streptozocin treatments.
Although streptozocin is a safe, streptozocin-induced renal dysfunction is a dilemma in streptozocin responders. Streptozocin may benefit patients with gastroenteropancreatic neuroendocrine tumors, especially those with a good performance status; however, in some cases, planned streptozocin withdrawal or switching to other drugs should be considered.
链脲佐菌素已在欧美用于治疗神经内分泌肿瘤，但由于该病在日本较为罕见，且链脲佐菌素在日本的批准时间相对较晚，其在日本的实际情况尚未完全明确。
我们回顾性分析了 2004 年 1 月至 2023 年 6 月期间，在日本的两家医院接受基于链脲佐菌素的化疗的 53 例胃肠胰神经内分泌肿瘤患者的资料。
总体缓解率和疾病控制率分别为 27.7%和 74.5%，中位无进展生存期和总生存期分别为 7.1 个月和 20.3 个月。体力状态≥1 与无进展生存期显著负相关，体力状态≥1 和肝肿瘤负担≥25%与总生存期显著负相关。胰腺和胃肠道神经内分泌肿瘤的治疗反应无显著差异。未观察到与治疗相关的严重不良事件；然而，87.7%的患者肾小球滤过率估计值下降，且与链脲佐菌素治疗持续时间呈负相关（r=0.43，P=0.0020）。在链脲佐菌素再治疗组（n=5）中，初始和第二次链脲佐菌素治疗的疗效无差异。
尽管链脲佐菌素是一种安全有效的药物，但链脲佐菌素引起的肾功能障碍是链脲佐菌素应答者面临的困境。链脲佐菌素可能使胃肠胰神经内分泌肿瘤患者受益，尤其是体力状态良好的患者；然而，在某些情况下，应考虑计划停止链脲佐菌素治疗或改用其他药物。
