Cancer Management and Research (Oct 2020)
Survival and Clinical Outcomes with Telotristat Ethyl in Patients with Carcinoid Syndrome
Abstract
David C Metz,1 Eric Liu,2 Vijay N Joish,3 Lynn Huynh,4 Todor I Totev,4 Mei Sheng Duh,4 Kiernan Seth,3 Susan Giacalone,3 Pablo Lapuerta,3 Michael A Morse5 1Neuroendocrine Tumor Program at Penn Medicine, Philadelphia, PA 19104, USA; 2The Neuroendocrine Institute at Rocky Mountain Cancer Centers, Denver, CO 80218, USA; 3Lexicon Pharmaceuticals, Inc, The Woodlands, TX 77831, USA; 4Analysis Group, Boston, MA 02199, USA; 5Duke Cancer Institute, School of Medicine, Duke University, Durham, NC 27710, USACorrespondence: David C MetzNeuroendocrine Tumor Program at Penn Medicine, Perelman Center for Advanced Medicine, South Pavilion, 4th Floor, 3400 Civic Center Boulevard, Philadelphia, PA 19104, USATel +1 800 789 7366Email [email protected]: 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.Patients and Methods: 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.Results: 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.Conclusion: 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.Keywords: neuroendocrine tumors, carcinoid syndrome, telotristat ethyl, survival
