Arseneau Kristen O, Sultan Shahnaz, Provenzale Dawn T, Onken Jane, Bickston Stephen J, Foley Eugene, Connors Alfred F, Cominelli Fabio
Division of Gastroenterology & Hepatology, University of Virginia Health System, Charlottesville, Virginia, USA.
Clin Gastroenterol Hepatol. 2006 Sep;4(9):1135-42. doi: 10.1016/j.cgh.2006.05.003. Epub 2006 Jul 10.
BACKGROUND & AIMS: Patients with steroid-refractory ulcerative colitis face a difficult treatment decision between colectomy and therapy with infliximab or cyclosporine. The aim of this study was to understand how individual patient preferences for the various treatment outcomes influence the optimal treatment decision for a given patient.
A Markov model was used to simulate treatment with total colectomy with an ileo pouch-anal anastomosis (TC/IPAA), cyclosporine (CSA), infliximab (INFLX), and infliximab followed by cyclosporine for treatment failures (INFLX-->CSA). Utility weights for treatment outcomes were elicited from 48 patients using both time trade-off and visual rating scale methods. Preference sets were applied to the model to identify the therapy that maximized quality-adjusted life years (QALYs) for each patient. Sensitivity analyses were performed to assess model robustness.
Optimal treatment was highly variable among patients (INFLX-->CSA = 42%, 20/48; TC/IPAA = 37%, 18/48; CSA = 21%, 10/48; INFLX = 0%, 0/48). However, when average preference weights from our sample were applied to the model, medical treatments were superior to TC (CSA = .26 QALYs gained vs TC/IPAA; INFLX-->CSA = .25 QALYs gained vs TC/IPAA).
Patient preferences have a clear impact on the optimal treatment for steroid-refractory ulcerative colitis. Although averaged preferences support the use of medical interventions, a third of individual patients may benefit most from proceeding directly to colectomy. Failure to fully assess individual preferences may result in suboptimal treatment for these patients.
激素难治性溃疡性结肠炎患者在结肠切除术与英夫利昔单抗或环孢素治疗之间面临艰难的治疗决策。本研究的目的是了解患者对各种治疗结果的个体偏好如何影响特定患者的最佳治疗决策。
使用马尔可夫模型模拟全结肠切除回肠储袋肛管吻合术(TC/IPAA)、环孢素(CSA)、英夫利昔单抗(INFLX)以及英夫利昔单抗治疗失败后改用环孢素(INFLX→CSA)的治疗情况。采用时间权衡法和视觉评定量表法从48例患者中获取治疗结果的效用权重。将偏好集应用于模型,以确定使每位患者的质量调整生命年(QALY)最大化的治疗方法。进行敏感性分析以评估模型的稳健性。
患者的最佳治疗方案差异很大(INFLX→CSA = 42%,20/48;TC/IPAA = 37%,18/48;CSA = 21%,10/48;INFLX = 0%,0/48)。然而,当将我们样本中的平均偏好权重应用于模型时,药物治疗优于全结肠切除术(CSA与TC/IPAA相比,获得的QALY为0.26;INFLX→CSA与TC/IPAA相比,获得的QALY为0.25)。
患者偏好对激素难治性溃疡性结肠炎的最佳治疗有明显影响。虽然平均偏好支持采用药物干预,但三分之一的个体患者可能直接进行结肠切除术获益最大。未能充分评估个体偏好可能导致这些患者接受次优治疗。