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. 2019 Oct 26;3(1):64.
doi: 10.1186/s41687-019-0153-y.

Evaluation of meaningful change in bowel movement frequency for patients with carcinoid syndrome

Stacie Hudgens  1 , John Ramage  2 , Matthew Kulke  3 , Emily Bergsland  4 , Lowell Anthony  5 , Martyn Caplin  6 , Kjell Öberg  7 , Marianne Pavel  8 , Jonathon Gable  9 , Phillip Banks  10 , Qi Melissa Yang  10 , Pablo Lapuerta  10
Affiliations

Evaluation of meaningful change in bowel movement frequency for patients with carcinoid syndrome

Stacie Hudgens et al. J Patient Rep Outcomes. .

Abstract

Background: Carcinoid syndrome is associated with a reduced quality of life that can be attributed to symptoms such as diarrhea and fatigue as well as social and financial issues. This study was conducted to psychometrically assess meaningful change in bowel movement frequency among carcinoid syndrome patients using data from the TELESTAR clinical study.

Methods: An anchor-based approach for deriving meaningful change thresholds consisted of mapping change from baseline bowel movement frequency to other patient-reported assessments of change. These included the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core Questionnaire (QLQ-C30) Diarrhea Symptom responders, the EORTC Gastrointestinal NET questionnaire (GI.NET21) GI Symptom responders, and reported adequate relief at Week 12 (≥ 10-point score decrease from Day 1 to Week 12). Parameters included within-group mean change from baseline to Week 12, t-tests of the change (Wilcoxon rank sum for adequate relief), and effect size.

Results: There were 135 carcinoid syndrome patients with a mean baseline frequency of 5.7 bowel movements a day. A distribution-based method yielded meaningful change estimates of 0.62 bowel movements a day for overall frequency and 0.83 bowel movements a day at Week 12. Anchor-based analysis indicated a large effect size among patients who reported adequate relief at Week 12 (- 1.58; n = 18; P = 0.014), the QLQ-C30 Diarrhea domain responders (- 1.24; n = 40; P < 0.001), and the GI.NET21 GI Symptoms Domain responders (- 1.49; n = 25; P = 0.005). Exit interview data for meaningful change yielded effect size estimates of - 1.57 for overall change during the Double-blind Treatment Period and - 1.97 for change between Baseline and Week 12.

Conclusions: Meaningful change derivation is critical to interpret patient outcomes for evaluating treatment efficacy. In this study, carcinoid syndrome patients experienced clinically meaningful reductions in bowel movement frequency of ≥30% over 12 weeks with telotristat ethyl treatment.

Trial registration: NCT01677910 .

Keywords: Bowel movement frequency; Carcinoid syndrome; Meaningful change.

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Conflict of interest statement

S. Hudgens and J. Gable are employed by Clinical Outcomes Solutions, the organization contracted by Lexicon to analyze the data and develop this manuscript. These authors have no additional financial relationships or otherwise to declare. M. Kulke has received consulting fees from Lexicon, Novartis, and Ipsen outside the submitted work. E. Bergsland is an uncompensated adviser for Lexicon Pharmaceuticals, Inc., and Ipsen. L. Anthony has received grants from Lexicon Pharmaceuticals Inc. M. Caplin has received compensation from Lexicon Pharmaceuticals Inc for participation on an advisory board. K. Öberg has received honoraria and speakers’ bureau fees from Novartis and Ipsen. M. Pavel has received consulting fees from Lexicon Pharmaceuticals Inc, Novartis, and Ipsen. P. Banks, Q.M. Yang, and P. Lapuerta are all currently employees of Lexicon Pharmaceuticals Inc and own stock. The authors have declared that they have no other competing interests regarding the content of this article.

Figures

Fig. 1
Fig. 1
Effect Sizes of Change in Overall BM Frequency from Baseline (ISP population)
Fig. 2
Fig. 2
Effect Sizes of Change in BM Frequency at Week 12 from Baseline (ISP population)
Fig. 3
Fig. 3
CDF of Overall Change from Baseline in Average BM Frequency
Fig. 4
Fig. 4
CDF of Change in Average BM Frequency from Baseline at Week 12
See this image and copyright information in PMC

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