Real-World Clinical and Patient-Reported Outcomes from the Longitudinal Telotristat Ethyl Treatment Registry of Patients with Neuroendocrine Tumors.

Abstract

BackgroundTelotristat ethyl (TE) is an oral tryptophan hydroxylase inhibitor approved for the treatment of carcinoid syndrome diarrhea (CSD) in combination with somatostatin analogs (SSAs).MethodsThis prospective, observational, single-arm study evaluated long-term patient-reported outcomes for adults initiating TE in US clinical practice from 2017 through January 2022. The primary objective was satisfaction with overall CS symptom control 6 months after initiating TE. Secondary objectives evaluated satisfaction with control of CSD, flushing, and CS symptoms, as well as work productivity/activity impairment, SSA use, and weight. All analyses were descriptive in nature.ResultsA total of 223 patients completed the baseline survey; 56% also completed the 6-month follow-up survey. Mean age was 61 years and 61% were women. After 6 months of TE treatment, the majority of patients (76%, n=95/125) reported being satisfied with control of their CS symptoms which was markedly improved from baseline (41%, n=91). Similarly, the majority of patients (78%, n=97/125) were satisfied with control of their CSD after 6 months of TE, markedly improved from baseline (36%).ConclusionThis longitudinal observational study showed improvements in real-world clinical and humanistic outcomes for patients with CS and at least 6 months of TE treatment.