Survival and Clinical Outcomes with Telotristat Ethyl in Patients with Carcinoid Syndrome.

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.

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© 2020 Metz et al.

Déclaration de conflit d'intérêts

DCM, EL and MAM served as consultants for the study design and conduct and were compensated for their time and effort from Lexicon. DCM served as the chair emeritus for NANETS without compensation; reports personal fees from Curium, and Crinetics. He also reports grants from Wren. LH, TIT and MSD are employees of Analysis Group which received funding for data collection and analysis from Lexicon. LH also reports grants from Novartis, Pfizer, Taiho, Epizyme, and Takeda, outside the submitted work. VNJ, KS, SG and PL were employees of Lexicon Pharmaceuticals, Inc., the study sponsor. The authors report no other conflicts of interest in this work.