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RISK-AVERSION PROFILE OF CROHN’S PATIENTS IN CALGARY: A FEASIBILITY STUDY FOR STEM CELL TRANSPLANTATION
S Asfaha, M Geddes, Y Leung, J Storek, S Devlin, J Jones, G Kaplan, PL Beck, R Panaccione
University of Calgary, Department of Medicine
The optimal therapy for patients with severe Crohn’s disease (CD) refractory to standard immunosuppressive and biologic therapies is unclear. Case reports and an ongoing prospective clinical trial report promising long term Crohn’s remission with high dose immunosuppressive therapy followed by infusion of autologous stem cells to “reset” the immune system; possible cure of Crohn’s is reported in patients given allogeneic stem cell transplant (SCT) for hematologic diseases. We designed a questionnaire to assess the willingness of Crohn’s patients in Calgary to accept risk of adverse effects to assess the feasibility of future SCT trials for treatment of CD.
METHODS: All CD patients >18 yrs of age assessed in the Inflammatory Bowel Disease Clinic at the University of Calgary were eligible to complete the questionnaire. Based on an autologous SCT scenario with an estimated 80%, or alternatively, 60% chance of a disease-free interval for 18 months and 10% long-term morbidity risk, patients were asked if they were willing to undergo an experimental treatment given a 0%, 2.5%, 5% or 10% risk of death. Based on an allogeneic SCT scenario, with an estimated 80% chance of being cured or symptom-free for 8 years and a 20% long-term morbidity risk, patients were asked if they were willing to undergo treatment with a 10%, 15%, 20% or 30% chance of death.
RESULTS: To date 50 CD patients (26 females; 24 males) have completed the questionnaire. Given a 0%, 2.5%, 5% or 10% risk of treatment-related mortality, 81%, 48%, 26% and 17% of patients were willing to undergo the autologous scenario with an 80% chance of an 18 month disease-free interval, and 70%, 33%, 19%, and 11% for a 60% chance of remission, respectively. Given a 10%, 15%, 20% or 30% chance of death in association with allogeneic SCT, only 36%, 17%, 10 and 9% of patients were willing to proceed with therapy.
CONCLUSIONS: Approximately half of CD patients are willing to accept an experimental therapy with morbidity (10%) and mortality (2.5%) typical for autologous SCT for hematologic disease. This supports the feasibility of trials of autologous SCT for severe refractory Crohn’s disease. Acceptance of trials of allogeneic SCT and thus their feasibility will likely be lower than for autologous SCT.