Telotristat in the management of Carcinoid diarrhoea – A real world experience of patients from ENETs centre of excellence in Neuroendocrine tumours

Background and Aims

Diarrhoea is a common and debilitating symptom of Carcinoid syndrome. Effective control of symptoms is achieved with somatostatin analogues (SSAs) and additional loperamide and/or codeine phosphate. Symptom control is lost with time and disease progression. There is, therefore, a strong need for additional and more effective therapies. Telotristat-ethyl is a peripheral tryptophan-hydroxylase inhibitor approved for treatment of diarrhoea. Here, we present our experience of Telotristat for treating carcinoid diarrhoea in a large cohort of patients outside of clinical trials. The primary outcome was reduction in stool frequency of >30%, as defined in most studies.

Methods

Patients were identified from a prospective database and information collected retrospectively from the hospital's electronic patient records. We included 31 patients (25 males, 6 females; Median age 69 years) on Telotristat and 10 patients on fortnightly high-dose SSA (6 males, 4 females; Median age 67 years).

Results

The mean (range) duration of treatment in telotristat and 2 weekly SSA groups was 258 (15-479) and 689 (219-1446) days respectively. Primary endpoint was achieved in 82% (23/28) patients on Telotristat with median percentage reduction in stool frequency of 60% (IQR 50-69), compared to 28% (2/7) (22 (−30 to 55) %) in the control group. Odds ratio for reduction in stool frequency >30% was −11, (95% CI 1.71-77.1).

Conclusion

This real world experience supports the effectiveness and safety of Telotristat to treat carcinoid diarrhoea not adequately controlled by standard treatment with SSA.