Oops, you're using an old version of your browser so some of the features on this page may not be displaying properly.

MINIMAL Requirements: Google Chrome 24+Mozilla Firefox 20+Internet Explorer 11Opera 15–18Apple Safari 7SeaMonkey 2.15-2.23

Poster display session

5394 - Long-term Survival of Patients With Carcinoid Syndrome in Clinical Trials of Telotristat Ethyl


10 Sep 2017


Poster display session


Pablo Lapuerta


Annals of Oncology (2017) 28 (suppl_5): v142-v157. 10.1093/annonc/mdx368


P. Lapuerta1, M.H. Kulke2, M. Caplin3, E. Bergsland4, L. Anthony5, K. Öberg6, M. Pavel7, D. Hörsch8, T.M. O'Dorisio9, J.S. Dillon9, K. Kassler-Taub10, W. Jiang10

Author affiliations

  • 1 Lexicon Pharmaceuticals, Lexicon Pharmaceuticals, Inc., 77381 - The Woodlands/US
  • 2 Gastrointestinal Cancer Treatment Center, Dana-Farber Cancer Institute, Boston/US
  • 3 Neuroendocrine Tumour Unit, Royal Free Hospital, London/GB
  • 4 Hereditary Gi Cancer Prevention Program, UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco/US
  • 5 Division Of Medical Oncology, University of Kentucky Chandler Medical Center, Lexington/US
  • 6 Endocrine Oncology, University Hospital, Uppsala University, Uppsala/SE
  • 7 Gastroenterology And Hepatology, Endocrinology & Metabolic Diseases, Charité–Universitätsmedizin, Berlin/DE
  • 8 Department Of Internal Medicine, Zentralklinik Bad Berka, Bad Berka/DE
  • 9 Department Of Internal Medicine – Endocrinology And Metabolism, University of Iowa, Iowa City/US
  • 10 Lexicon Pharmaceuticals, Lexicon Pharmaceuticals, Inc., The Woodlands/US


Abstract 5394


Patients with carcinoid syndrome (CS) in the setting of metastatic neuroendocrine tumors (NETs) experience considerable morbidity and mortality. From the time of diagnosis of metastatic NETs, median survival has been estimated to be approximately 31–75 months. CS is associated with tumoral secretion of serotonin and subsequent debilitating diarrhea, which poses a significant health risk. In previous studies, telotristat ethyl (TE), a tryptophan hydroxylase inhibitor, was effective and well tolerated in treating CS diarrhea. At enrollment, patients in these studies had already survived an average of 6–8 years with metastatic NETs since their initial diagnoses.


Adverse events reported during treatment with TE were pooled from 2 Phase 2 and 3 Phase 3 clinical trials of TE in patients with CS. The long-term safety of TE was examined, causes of hospitalization and death were reviewed, and an estimate of overall survival was obtained.


A total of 239 patients with CS received treatment with TE in Phase 2 and 3 clinical trials. For these patients, as of the end of 2016, the mean duration of exposure was 1.3 years, and maximum 5.7 years. The leading causes of hospitalization were gastrointestinal disorders and surgical and medical procedures, mostly attributable to the underlying tumor and related treatment. Survival estimates at 1, 2, and 3 years were 93%, 88%, and 77%, respectively. Nearly all deaths were due to progression or complication of the underlying disease, and none were attributable to TE. There was 1 death in Year 4 and no deaths in Years 5 and 6 of patient follow-up in this data set. The median survival with TE was not reached at the end of the 6-year Follow-up period.


Our review of the long-term safety data for TE indicates that patients with CS treated with TE in Phase 2 and 3 studies experienced encouraging survival rates.

Clinical trial identification

NCT02026063, NCT02063659, NCT01677910, NCT01104415, NCT00853047

Legal entity responsible for the study

Lexicon Pharmaceuticals, Inc.


Lexicon Pharmaceuticals, Inc.


P. Lapuerta, K. Kassler-Taub, W. Jiang: Employee of Lexicon Pharmaceuticals, Inc. M.H. Kulke: Relationships with Lexicon Pharmaceuticals, Inc., Ipsen Bioscience, and Novartis Pharmaceuticals. M. Caplin: Advisory board/speaker honoraria, research funding from Lexicon Pharmaceuticals, Inc., Novartis Pharmaceuticals and Ipsen Biopharmaceuticals. E. Bergsland: Advisory boards for Ipsen Bioscience, Lexicon Pharmaceuticals, Inc., and Novartis Pharmaceuticals; research support from Lexicon Pharmaceuticals, Inc., and Novartis Pharmaceuticals. L. Anthony: Grants from Lexicon Pharmaceuticals, Inc. M. Pavel: Grants and personal fees from Ipsen Bioscience and Novartis Pharmaceuticals; personal fees from Lexicon Pharmaceuticals, Inc., and Pfizer, inc. D. Hörsch: Grants and personal fees from Lexicon Pharmaceuticals, Inc., Ipsen Bioscience, Novartis Pharmaceuticals and Pfizer, Inc. J.S. Dillon: Received grants from Lexicon Pharmaceuticals, Inc. All other authors have declared no conflicts of interest.

This site uses cookies. Some of these cookies are essential, while others help us improve your experience by providing insights into how the site is being used.

For more detailed information on the cookies we use, please check our Privacy Policy.

Customise settings
  • Necessary cookies enable core functionality. The website cannot function properly without these cookies, and you can only disable them by changing your browser preferences.