Metz David C, Liu Eric, Joish Vijay N, Huynh Lynn, Totev Todor I, Duh Mei Sheng, Seth Kiernan, Giacalone Susan, Lapuerta Pablo, Morse Michael A
Neuroendocrine Tumor Program at Penn Medicine, Philadelphia, PA 19104, USA.
The Neuroendocrine Institute at Rocky Mountain Cancer Centers, Denver, CO 80218, USA.
Cancer Manag Res. 2020 Oct 7;12:9713-9719. doi: 10.2147/CMAR.S276519. eCollection 2020.
The TELEACE study showed reductions in tumor size in patients with neuroendocrine tumors, receiving telotristat ethyl in US clinical practice. Here, we report progression-free survival, time to tumor progression, changes in carcinoid syndrome symptoms, and indictors of overall health.
This was a retrospective, single arm, pre-post medical chart review of patients with locally advanced or metastatic neuroendocrine tumors and documented carcinoid syndrome receiving telotristat ethyl for at least 6 months. Patients with poorly differentiated tumors, mixed tumor types or conflicting clinical trial enrollment were excluded. Descriptive statistics, Kaplan-Meier and chi-square tests were used to evaluate PFS, tumor progression, changes in symptoms, body weight and ECOG performance status before and after telotristat ethyl initiation. Subgroup analyses were conducted in patients with the same pre- and post-telotristat ethyl background treatment.
Anonymized data for 200 patients were provided by 114 physicians; patients received telotristat ethyl for a median of 9 months. Median time to tumor progression was 39.8 months (IQR, 18.7-39.8); most had no tumor progression at 6 (92%) and 12 months (87%). Median progression-free survival was 23.7 months (17.8-39.8); most had progression-free survival at 6 (90%) and 12 months (80%). Results were consistent in the subgroup of 65 patients with the same pre/post background treatment. Nearly all patients had improved carcinoid syndrome symptoms, stable or improved weight and ECOG performance status.
Patients showed improvements in clinical outcomes and indicators of overall health following treatment with telotristat ethyl in this exploratory pilot study, consistent with previously observed reductions in tumor size.
TELEACE研究表明，在美国临床实践中，接受乙基替洛曲沙治疗的神经内分泌肿瘤患者的肿瘤大小有所减小。在此，我们报告无进展生存期、肿瘤进展时间、类癌综合征症状的变化以及总体健康指标。
这是一项回顾性、单臂、治疗前后病历审查研究，纳入局部晚期或转移性神经内分泌肿瘤且记录有类癌综合征并接受乙基替洛曲沙治疗至少6个月的患者。排除肿瘤分化差、肿瘤类型混合或临床试验入组情况存在冲突的患者。采用描述性统计、Kaplan-Meier法和卡方检验来评估乙基替洛曲沙开始治疗前后的无进展生存期、肿瘤进展、症状变化、体重和ECOG体能状态。对治疗前后背景治疗相同的患者进行亚组分析。
114名医生提供了200例患者的匿名数据；患者接受乙基替洛曲沙治疗的中位时间为9个月。肿瘤进展的中位时间为39.8个月（四分位间距，18.7 - 39.8）；大多数患者在6个月（92%）和12个月（87%）时无肿瘤进展。无进展生存期的中位时间为23.7个月（17.8 - 39.8）；大多数患者在6个月（90%）和12个月（80%）时无进展生存期。在65例治疗前后背景治疗相同的亚组患者中结果一致。几乎所有患者的类癌综合征症状都有所改善，体重稳定或增加，ECOG体能状态改善。
在这项探索性试点研究中，患者接受乙基替洛曲沙治疗后临床结局和总体健康指标有所改善，这与之前观察到的肿瘤大小减小一致。
