Crohn’s Disease Patients’ Risk-Benefit Preferences: Serious Adverse Event Risks Versus Treatment Efficacy

Johnson, F. Reed; Özdemir, Semra; Mansfield, Carol; Hass, Steven; Miller, David W.; Siegel, Corey A.; Sands, Bruce E.

doi:10.1053/j.gastro.2007.04.075

Cited by 171 publications

(139 citation statements)

References 22 publications

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“…Because some individuals may have very strong preferences over a specific treatment attribute, there was no plan to exclude those who dominated from the multivariate analysis. [22] However, individuals who showed no variation in their responses – that is, they always chose treatment A or treatment B – were excluded.…”

Section: Methodsmentioning

confidence: 99%

Patients’ Willingness to Accept the Risks and Benefits of New Treatments for Chronic Hepatitis C Virus Infection

Kauf

Mohamed

Hauber

et al. 2012

Patient-Patient-Centered-Outcome-Res

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BackgroundSome patients with chronic hepatitis C virus (HCV) infection forego treatment due to concerns about treatment efficacy, treatment duration, and side effects.ObjectiveThe purpose of this study was to quantify patient preferences among possible outcomes associated with new, direct-acting antiviral agents (DAAs) for the treatment of HCV infection and determine which treatment features are most important to patients in making treatment decisions.MethodsAdult participants with a self-reported physician diagnosis of HCV infection in five countries completed a web-enabled, choice-format conjoint analysis survey. The survey presented participants with 20 treatment-choice questions. Each treatment-choice question included a pair of hypothetical treatment profiles with varying levels of six attributes: treatment duration; chance of getting rid of the virus completely (i.e. likelihood of a sustained virologic response [SVR]); weeks on an additional, third medicine (i.e. a DAA); risk of a severe rash; risk of severe anemia; and number of times a day the third medicine is taken. Treatment-choice questions were based on a predetermined experimental design with known statistical properties. Random-parameters logit was used to estimate preference weights for all attribute levels and the mean relative importance of each attribute.Results284 participants completed the survey. Likelihood of an SVR was the most important outcome to participants, followed by severe anemia risk, severe rash risk, therapy type (a combination of total weeks of treatment and weeks on the third medicine), and dosing of the third medicine. Controlling for other factors, preferences were similar across all therapy types examined.ConclusionPatients with HCV infection indicate a willingness to accept an increased risk of side effects for sufficient improvement in the likelihood of treatment response.Electronic Supplementary MaterialSupplementary material is available for this article at 10.1007/BF03262498 and is accessible for authorized users.

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Section: Methodsmentioning

confidence: 99%

Patients’ Willingness to Accept the Risks and Benefits of New Treatments for Chronic Hepatitis C Virus Infection

Kauf

Mohamed

Hauber

et al. 2012

Patient-Patient-Centered-Outcome-Res

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“…Eligible patients underwent stem cell mobilisation using cyclophosphamide 2gm/m 2 x two days and non-glycosylated Granulocyte Colony Stimulating Factor (G-CSF, filgrastim) 10 μg/kg/day. 14 Patients underwent leukapheresis when the CD34+ count exceeded 20x10 4 /ml to a target of 3-8 x10 6 CD34+ cells/kg body weight and if successful underwent HSCT immediately or after a delay of one year. An intermediate intensity conditioning regimen was used which comprised intravenous cyclophosphamide 50 mg/kg/day x four days and, from day three, rabbit antithymocyte globulin (Genzyme) 2.5 mg/kg/day and methylprednisolone 1 mg/kg/day x three days, with infusion of unselected autologous stem cells (minimum 3x10 6 CD34+ cells/kg) on day seven.…”

Section: Mobilisation and Conditioning Regimenmentioning

confidence: 99%

“…1,5 Patients with treatment refractory CD are prepared to undergo a significant treatment related risk to achieve remission. 6 There has been considerable interest in case reports that autologous haematopoietic stem cell transplantation (HSCT) can induce sustained disease remission in such patients. [7][8][9][10][11][12][13] The ASTIC trial compared outcome at one year in 23 patients with refractory CD after autologous HSCT to 22 undergoing stem cell mobilisation alone.…”

Section: Introductionmentioning

confidence: 99%

Autologous stem-cell transplantation in treatment-refractory Crohn's disease: an analysis of pooled data from the ASTIC trial

Lindsay

Allez

Clark

et al. 2017

The Lancet Gastroenterology & Hepatology

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BACKGROUND The randomised controlled ASTIC trial showed no benefit of mobilisation and autologous haematopoietic stem-cell transplantation (HSCT) compared with mobilisation followed by conventional therapy using a stringent primary endpoint (steroid-free clinical remission for 3 months with no endoscopic or radiological evidence of intestinal inflammation) in patients with treatment-refractory Crohn's disease. We now assess HSCT in patients enrolled in the ASTIC trial using endpoints that are traditional for clinical trials in Crohn's disease, and identify factors that predict benefit or harm. METHODS Patients who underwent mobilisation and were randomly assigned to conventional therapy in the ASTIC trial were offered HSCT at 1 year and underwent complete assessment for a further year. We report analyses of the combined cohort of patients who underwent HSCT at any time during the ASTIC trial programme. The primary outcome for this analysis was 3-month steroid-free clinical remission at 1 year after HSCT (Crohn's Disease Activity Index [CDAI] <150). We also examined the degree of endoscopic healing at 1 year. Multivariate analysis was performed to identify factors associated with achieving the primary endpoint by using logistic regression, and factors associated with experiencing a serious adverse event using Poisson regression. Participants were not masked to treatment, but the adjudication panel that reviewed radiology and endoscopy was masked to allocation and visits. All patients who underwent HSCT and had data available at baseline and 1-year follow-up were included in the primary and safety analysis. This trial is registered with ClinicalTrials.gov, number NCT00297193. FINDINGS Between June 28, 2007, and Sept 1, 2011, 45 patients were enrolled in the ASTIC trial from 11 European transplant units. 23 patients were randomly assigned to immediate HSCT, and 22 patients were assigned to mobilisation followed by conventional care. After completion of the ASTIC trial, 17 patients from the conventional care group received HSCT. In the combined cohort, data were available for 40 patients at baseline and 38 patients at 1 year after HSCT (one patient died, one withdrew). At 1 year after HSCT, 3-month steroid-free clinical remission was seen in 13 (38%, 95% CI 22-55) of 34 patients with available data for the whole year. Complete endoscopic healting was noted in 19 (50%, 34-66) of 38 patients. On multivariate analyses, factors associated with the primary outcome were short disease duration (odds ratio [OR] 0·64, 95% CI 0·41-0·997 per year; p=0·048) and low baseline CDAI (0·82, 0·74-0·98 per 10 units; p=0·031). 76 serious adverse events occurred in 23 of 40 patients with available data. The most common serious adverse event was infection, most of which were treatment related. Smoking and perianal disease at baseline were independent factors associated with the number of serious adverse events (OR 3·07 [95% CI 1·75-5·38; p=0·0001] for smoking and 3·97 [2·17-7·25; p<0·0001] for perianal disease) on multivariate...

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Section: What Is Already Known About This Subjectmentioning

confidence: 99%

“…12,13 A previous study of adult patients with Crohn's disease found that for an improvement from severe symptoms to remission, patients were willing to accept an annual MAR of 0.70% for PML (95% confidence interval [CI] = 0.60%-0.80%), 0.73% for serious infection (95% CI = 0.66%-0.81%), and 0.82% for lymphoma (95% CI = 0.72%-0.92%). 12 For an improvement from moderate symptoms to remission, patients were willing to accept an annual MAR of 0.39% for PML (95% CI = 0.27%-0.52%), 0.55% for serious infection (95% CI = 0.48%-0.61%), and 0.55% for lymphoma (95% CI = 0.48%-0.62%). 12 For a 10-year treatment exposure, the mean tolerance for PML risks ranged between 6.3% (95% CI = 4.1%-8.5%) for reduction in severity of symptoms from severe to remission and 0.9% (95% CI = 0.4%-1.6%) for reduction in severity of symptoms from mild to remission (unpublished analysis of reference 12 data).…”

Section: What Is Already Known About This Subjectmentioning

confidence: 99%

Are Gastroenterologists Less Tolerant of Treatment Risks than Patients? Benefit-Risk Preferences in Crohn's Disease Management

Hauber¹,

Özdemir²,

Siegel³

et al. 2010

JMCP

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BACKGROUND: Crohn's disease is a serious and debilitating gastrointestinal disorder with a high, unmet need for new treatments. Biologic agents have the potential to alter the natural course of Crohn's disease but present known risks of potential serious adverse events (SAEs). Previous studies have shown that patients are willing to accept elevated SAE risks in exchange for clinical efficacy. Gastroenterologists and patients may have similar goals of maximizing treatment benefit while minimizing risk; however, gastroenterologists may assess the relative importance of risk differently than patients.

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Crohn’s Disease Patients’ Risk-Benefit Preferences: Serious Adverse Event Risks Versus Treatment Efficacy

Cited by 171 publications

References 22 publications

Patients’ Willingness to Accept the Risks and Benefits of New Treatments for Chronic Hepatitis C Virus Infection

Patients’ Willingness to Accept the Risks and Benefits of New Treatments for Chronic Hepatitis C Virus Infection

Autologous stem-cell transplantation in treatment-refractory Crohn's disease: an analysis of pooled data from the ASTIC trial

Are Gastroenterologists Less Tolerant of Treatment Risks than Patients? Benefit-Risk Preferences in Crohn's Disease Management

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