Identifying symptom and quality of life improvements in patients with carcinoid syndrome treated with telotristat ethyl: Qualitative patient exit interviews from the TELESTAR Trial

Abstract

Background: Carcinoid syndrome (CS) is a rare condition in patients (pts) with neuroendocrine tumors (NETs), characterized by diarrhea, flushing, and abdominal pain that impact health‐related quality of life (HRQoL).We assessed symptoms and HRQoL in pts with inadequately‐controlled CS enrolled in TELESTAR (NCT01677910). Methods: English or German‐speaking pts randomized 1:1:1 to 12 wks of doubleblinded (DB) treatment with telotristat ethyl (TE) 250mg, 500mg, placebo were invited to a blinded, qualitative, semi‐structured exit interview after the DB period to assess symptoms, HRQoL concepts (improved/not changed/worsened), and TE treatment effects. Concepts were freely reported by pts and not solicited. EORTC QLQ‐C30 and GINET21 questionnaires assessed HRQoL, daily diaries assessed baseline (BL) bowel movement (BM) frequency. Analyses compared pts with durable response (DRs; predefined as BM frequency reduction of ≥30% from BL for ≥50% of DB period) and without durable response. Results: TELESTAR enrolled 135 pts, 45 per group; 34 pts (9, 16, 9 in TE 250mg, 500mg, placebo, respectively), including 10 DRs consented to interviews. BL age, gender, race, BMs/wk of interviewed pts (IPs) were similar to non‐interviewed pts. Most qualitative concepts were captured, to an extent, by the QLQ‐C30; most common HRQoL qualitative concepts identified were daily life activities, physical, and psychological, with most TE‐treated pts improving in these over the DB period. The most common symptoms (improvement/no change) reported in all IPs were diarrhea consistency (n=17/n=10), frequency (n=17/n=9), urgency (n=11/n=7); flushing (n=11/n=7), and fatigue (n=9/n=4). A higher proportion of TE‐treated pts and DRs reported symptom and HRQoL concept improvements. Durable response (p<0.001) and treatment satisfaction (p=0.0137) correlated with diarrhea QLQ‐C30, but no other QLQ‐C30/GINET21 scores. Conclusions: The QLQ‐C30 covered most qualitative concepts, but due to the lack of emphasis on key symptoms, may not adequately reflect pt perspectives. Interviews suggested improvements in symptoms and HRQoL with TE treatment and in DRs.