Meenakshi Bewtra1, Shelby D Reed2, F Reed Johnson2, Frank I Scott3, Erin Gilroy4, Robert S Sandler5, Wenli Chen5, James D Lewis6. 1. Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, Pennsylvania; Division of Gastroenterology, University of Pennsylvania, Philadelphia, Pennsylvania; Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, Pennsylvania. Electronic address: mbewtra@pennmedicine.upenn.edu. 2. Duke Clinical Research Institute, Duke University, Durham, North Carolina. 3. Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, Pennsylvania; Division of Gastroenterology, University of Colorado Anschutz Medical Campus, Aurora, Colorado. 4. Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, Pennsylvania. 5. Department of Epidemiology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina. 6. Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania, Philadelphia, Pennsylvania; Division of Gastroenterology, University of Pennsylvania, Philadelphia, Pennsylvania; Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, Pennsylvania.
Abstract
BACKGROUND & AIMS: Patients with Crohn's disease (CD) must make decisions about their treatment. We aimed to quantify patients' preferences for different treatment outcomes and adverse events. We also evaluated the effects of latent class heterogeneity on these preferences. METHODS: An online stated-preference survey was completed by 812 individuals with CD in the Crohn's and Colitis Foundation Partners cohort (IBD Partners). Patients were given information on symptoms and severity of active disease; duration of therapy with corticosteroids; and risks of serious infection, cancer and surgery. Patients were asked to assume that their treatment was not working and to choose an alternative therapy. The primary outcome was remission-time equivalents (RTE) of a given duration of symptom severity or treatment-related risk. Latent class choice models identified groups of patients with dominant treatment-outcome preferences and associated patient characteristics with these groups. RESULTS: Latent class analysis demonstrated 3 distinct groups of survey responders whose choices were strongly influenced by avoidance of active symptoms (61%), avoidance of corticosteroid use (25%), or avoidance of risks of cancer, infection or surgery (14%) when choosing a therapy. Class membership was correlated with age, sex, mean short CD activity index score and corticosteroid avoidance. RTEs in each latent class differed significantly from the mean RTEs for the overall sample, although the symptom-avoidant class most closely approximated the overall sample. CONCLUSIONS: In an online survey of patients with CD, we found substantial heterogeneity in preference for medication efficacy and risk of harm. Physicians and regulators should therefore not assume that all patients have mean-value preferences-this could result in significant differences in health-technology assessment models.
BACKGROUND & AIMS:Patients with Crohn's disease (CD) must make decisions about their treatment. We aimed to quantify patients' preferences for different treatment outcomes and adverse events. We also evaluated the effects of latent class heterogeneity on these preferences. METHODS: An online stated-preference survey was completed by 812 individuals with CD in the Crohn's and Colitis Foundation Partners cohort (IBD Partners). Patients were given information on symptoms and severity of active disease; duration of therapy with corticosteroids; and risks of serious infection, cancer and surgery. Patients were asked to assume that their treatment was not working and to choose an alternative therapy. The primary outcome was remission-time equivalents (RTE) of a given duration of symptom severity or treatment-related risk. Latent class choice models identified groups of patients with dominant treatment-outcome preferences and associated patient characteristics with these groups. RESULTS: Latent class analysis demonstrated 3 distinct groups of survey responders whose choices were strongly influenced by avoidance of active symptoms (61%), avoidance of corticosteroid use (25%), or avoidance of risks of cancer, infection or surgery (14%) when choosing a therapy. Class membership was correlated with age, sex, mean short CD activity index score and corticosteroid avoidance. RTEs in each latent class differed significantly from the mean RTEs for the overall sample, although the symptom-avoidant class most closely approximated the overall sample. CONCLUSIONS: In an online survey of patients with CD, we found substantial heterogeneity in preference for medication efficacy and risk of harm. Physicians and regulators should therefore not assume that all patients have mean-value preferences-this could result in significant differences in health-technology assessment models.
Authors: Anouk M Wijnands; Maarten Te Groen; Yonne Peters; Ad A Kaptein; Bas Oldenburg; Frank Hoentjen; Maurice W M D Lutgens Journal: Inflamm Bowel Dis Date: 2022-07-01 Impact factor: 7.290
Authors: Siddharth Singh; Deborah Proctor; Frank I Scott; Yngve Falck-Ytter; Joseph D Feuerstein Journal: Gastroenterology Date: 2021-06 Impact factor: 33.883
Authors: Glen S Hazlewood; Gyanendra Pokharel; Robert Deardon; Deborah A Marshall; Claire Bombardier; George Tomlinson; Christopher Ma; Cynthia H Seow; Remo Panaccione; Gilaad G Kaplan Journal: PLoS One Date: 2020-01-16 Impact factor: 3.240