Patient Preferences For Health States Research Articles

PP1 - Patient Preferences for Health States Following Alternative Management Options for Ductal Carcinoma in Situ

PP1 - Patient Preferences for Health States Following Alternative Management Options for Ductal Carcinoma in Situ

Health State Utility Values for Ileostomies and Colostomies: a Systematic Review and Meta-Analysis

BackgroundIleostomies and colostomies may affect the quality of life of patients after colorectal surgery; however, the impact has been difficult to quantify using questionnaire-based measures. Utilities reflect patient preferences for health states and provide an alternate method of quality of life assessment. We aimed to systematically review the literature on utilities for ileostomy and colostomy health states. MethodsWe searched MEDLINE, EMBASE, and EBM Reviews (to August 16, 2017) to identify studies reporting utilities for colostomies or ileostomies using direct or indirect, preference-based elicitation tools. We categorized utilities based on elicitation group (patients with stoma, patients without stoma, healthcare providers, general population) and tool. We pooled utilities using random effects models to determine mean utilities for each elicitation group and tool. ResultsWe identified ten studies reporting colostomy utilities and three studies reporting ileostomy utilities. Utilities were most commonly obtained using direct elicitation measures administered to individuals with an understanding of the health state. Patients with stomas and providers gave high utility ratings for the colostomy state (range 0.88–0.92 and 0.86–0.92, respectively, using direct elicitation tools). Ileostomy utilities obtained from patients following surgery and from providers also demonstrated high values placed on the ileostomy health state (range 0.88–1.0). ConclusionsFollowing stoma surgery, values placed on quality of life are similar to those obtained from patients with conditions such as asthma and allergies or individuals of similar age without chronic conditions. This confirms the findings of questionnaire-based studies, which report minimal long-term decrements to overall quality of life among stomates.

TU‐C‐BRB‐04: Enhanced Modeling of Radiation Therapy for Head and Neck Cancers with Probabilistic Outcomes Using Mixed Predictors

Purpose: To develop a probabilistic model of outcomes of radiation therapy which includes both dosimetric and non‐dosimetric predictors, and includes a decision‐making component to quantify the balance between disease cure and radiation‐induced side‐effects. This model was implemented to assess IMRT treatment plans for individual patients for head and neck cancer. Materials and Methods: Physicians have available many resources that may not be easily reconcilable to predict patient outcomes. Dosimetric indicators, such as the EUD and NTCP are probabilistic in nature, without explicit representation of the underlying biology. Clinical trials focus on patient and disease characteristics, such as disease location, T‐stage, nodal involvement, Karnofsky performance status, and often include one treatment variable, such as DVH‐cutpoints or chemotherapy regimes. Newly recognized factors, such as HPV positivity, may affect outcome, however, without definitive clinical data, integrating such factors into clinical decision‐making is not straightforward. Finally, experience‐driven beliefs affect treatment choices and may vary between physicians. We combine all of the aforementioned resources using a Bayesian network in order to make an outcome prediction for each IMRT plan. Outcome predictions highlight the stark trade‐off between preventing recurrent disease that generally has a fatal prognosis and preventing radiation‐induced side effects that range from xerostemia to blindness to paralysis. We use a Markov Model to compute a quality‐adjusted life expectancy using patient preferences for health states. Results: Probabilities of local and distant control matched published values well, as did life expectancies. The trade‐offs between quality of life and quantity of life are explored. Sensitivity analysis highlighted physician beliefs that affected treatment choices. Conclusions: Modeling of radiation therapy has grown progressively more sophisticated. We present a method by which probabilities and expected values of clinically relevant outcomes, based on a range of variables, are calculated.

Cost-Utility of Screening for Barrettʼs Esophagus with Esophageal Capsule Endoscopy Versus Conventional Upper Endoscopy

Purpose: Esophageal adenocarcinoma is rising in incidence, and screening with conventional upper endoscopy to decrease cancer mortality is recommended. Esophageal capsule endoscopy (ECE) has recently been shown to be accurate in detecting Barrett's esophagus, the accepted precursor of this malignancy. We aimed to compare the cost-effectiveness of screening by ECE with screening by conventional upper endoscopy for esophageal adenocarcinoma. Methods: A Markov model of hypothetical 50 year-old Caucasian men with symptoms of gastroesophageal reflux was constructed to calculate outcomes associated with Barrett's esophagus and esophageal cancer. The model incorporated direct medical costs, costs of lost productivity, and patient preferences for health states (utilities), and followed the patients until age 80 or death. The primary outcome was the incremental cost-effectiveness ratio, and was analyzed from the societal perspective. Other outcomes included were life expectancy, quality-adjusted life expectancy, and proportion of cancer deaths averted. Results: Screening by conventional upper endoscopy prevented 60.4% of cancer deaths, at a cost of $11,254 per quality-adjusted life-year gained compared to no screening. ECE prevented 59.0% of cancer deaths, and provided 2 fewer quality-adjusted days and at greater cost than conventional upper endoscopy. The only scenario in which ECE would be the preferred strategy is if the patient and his driver earned more than $153,423 annually, resulting in substantial lost productivity to society compared to the incremental gain in patient outcomes with EGD. Conclusions: Screening for esophageal adenocarcinoma with either conventional endoscopy or ECE result in similar outcomes, but conventional endoscopy is the preferred strategy. Both strategies appear cost-effective and the model does not take into account patient or health care provider preferences for screening modality or adherence.

Preference elicitation using the time trade-off method in patients with 1 to 3 newly diagnosed brain metastases in a prospective randomized clinical trial comparing radiosurgery with and without whole brain radiation

Preference elicitation is used to address the value of healthcare outcomes. Patient preferences for health states were compared in brain metastasis patients randomized to be treated with radiosurgery (SRS) with or without whole brain radiation (WBRT). The objectives of the preference elicitation study were to 1) evaluate differences in utilities between treatment groups and 2) to evaluate changes in utilities over time.

Decision analysis and economic modelling: a primer.

In this primer, the reader is introduced to the concepts governing decision analysis and cost-effectiveness analysis. The construction of decision trees and Markov models is presented to provide the necessary background to critique research articles in published literature. Specific sub-topics related to cost-effectiveness analysis are discussed including quality adjustment and utilities (patient preferences for health states), discounting, and sensitivity analysis including Monte Carlo simulation. Evidence based methods to critique decision and cost-effectiveness analysis are provided, and limitations to these analytic methods are examined. In summary, the major functions of decision analysis and cost-effectiveness analysis are to provide: (1) a quantitative summary of existing data, and (2) hypothesis generation for further research.

Health state preference assessment in diabetic peripheral neuropathy.

To assess patient preferences for health states associated with diabetic peripheral neuropathy (DPN). Seven health states describing varying stages of disease severity in DPN were developed: mild neuropathy, painful neuropathy, severe neuropathy, minor ulcer, severe ulcer, minor amputation and major amputation. Using a computer interview, both rating scale (RS) and standard gamble (SG) preference scores were elicited from 52 patients with diabetes mellitus. A US university medical centre and a Veterans Affairs clinic. Adult patients with type 1 and type 2 diabetes mellitus between the ages of 18 and 80 years. Patients were excluded if they had been diagnosed with DPN. Preference scores decreased in accordance with greater symptomatic and functional impairment. Median RS scores ranged from 0.89 to 0.23 for the seven health states. Median SG scores ranged from 0.96 to 0.65. Patient preferences for health states decrease as a function of increasing disease severity in DPN. The result is robust across the RS and SG methods of preference measurement. These results may be useful in informing policy analyses for resource allocation in patients with diabetes mellitus.

Assessing the Preferences of Patients With Psoriasis

Patient preferences for health outcomes can be explicitly assessed and expressed in quantitative terms known as utilities. Three standard methods for utility assessment have been used to quantify patient preferences, but these methods have not previously been applied to skin disease. Eighty-seven patients with psoriasis from a tertiary medical center were interviewed, using an interactive, computer-based utility assessment questionnaire, U-Titer. Utilities for three categories of psoriasis severity and potential adverse outcomes of methotrexate therapy were assessed by the vertical rating scale, time trade-off, and standard gamble. Patients assigned a broad range of utilities for each of the health states. Utilities obtained by the vertical rating scale did not correlate well with utilities obtained by standard gamble or time trade-off methods. However, utilities assessed by standard gamble and time trade-off were not significantly different. Patient characteristics such as age, gender, and education were not correlated with utility and did not explain the variation. Indicators of the patients' disease severity were not predictive of utilities for the assessed health states. The relatively high utility for liver biopsy suggests that there is less patient aversion to the procedure than suspected. Utilities, or quantitative measures of patient preferences for health states, are measurable and vary widely for mild, moderate, and severe psoriasis and possible adverse outcomes of methotrexate treatment. The process of elucidating individual patient utilities for various health outcomes can be used to incorporate patient preferences into the process of clinical decision making. Guidelines that are based solely on severity of symptoms, without input from patients on how they value such symptoms, must be questioned.

The implications of using patient preferences (utilities) vs. health status on decision making and policy recommendations for patients with ulcerative colitis

Q THE IMPLICATIONS OF USING PATIENT PREFERENCES (UTILITIES1 VS. HEALTH STATUS ON DECISION MAKING AND POLICY RECOMMENDATIONS FOR PATIE~S WITH ULCERATIVE COLITIS. D. Provenzale, M. Shearin, W. Tillinger, B. Phillips-Bute, F.S. Sherman R. Bollmger, M. Koruda, D. Drossman, J.B. Wong, Di~sion of Gastro'enterotogy: Epidemimogy, ~ecision Analysis, Outcomes t~esearch Grotto, Duke Umv~sltL¢, Durham., N=C., Universit3/of N.C., Chapel Hill N.C. Tufts-New tmgtand Meaical ~Str. ~oston, MA. ,~ , ' ' f o r patients wire pancotitis or more man 7-1u years auration, most authorines recommend surveillance colonoscopy with biopsies, but others advocate prophylactic colectomy after 10 years of disease to minimize cancer risk. Health status and quality of life arter colectomy are important outcomes, in decisions for surveillance and colectomy. Health status measures evaluate physical, social .and emotional function, while quality of life measures examine patient preferences for health states. Both measures provide valuable information about the patient-specific outcomes of surveillance programs. In an earlier report, we measured quality of life values ofpost-cotectomy patients using tlae time trade-off tecfinique (TTO) and, in a decision analysis, examined their effect on management decisions for patients with ulcerative colitis. In this study, we measured the health status of 21 patients who had undergone proctocolectomy with ileal pouchanal anastomosis. Health status was measured with the SF-36, a general measure of health status and well being, which has been validated m postcolectomy patients. We incorporated these results into the decision model to determine the effect of post-operative physical functioning on decisions for surveillance and colectomy. We compared these results with the results obtained using the measured quality o f life values. The decision model compares strategies of prophylactic proctocolectomy and ileal pouch-anal anastomosis with surveillance every 1-5 years and surveillance at variable intervals based on the duration of the disease, with no surveillance. The details of the model are published in an earlier report. TrO SF-36 Quality adjustment for colectomy 0.97 (perfect health=l.0) 0.81 (perfect health=l.0) Preferred strategy Prophylactic colectomy Surveillance very 5 years Using the TTO results, prophylactic colectomy would increase qualityadjusted life expectancy by 7months compared to no surveillance and b y up to 6 months compared to surveillance strategies. Using the results ot~tained with the SF-36, surveillance every 5 years would increase quality-adjusted life expectancy by up to 5.3 years compared to prophylactac colectomy, and by 4.6 years compared to surveillance with co[ectomy for low grade dysplasia. The optimalmanagement strategy depends upon the perspective of the decision maker. The individual patient and physician who might consider the quality of life post-colectomy as most important, would adopt the optimal management strategy based on the TTO results. Individuals Who consider physical functioning most important, and those who make health policy, might base their recommendations on management strategies that incorporate the physical functioning score. Quality of life and health status are unportant outcomes for post-colectomypatients. Their measurement should be incorporated into all individualdecisions and policy recommendations for patients with ulcerative colitis. ETHICALLY JUSTIFIED, CLINICALLY COMPREHENSIVE GUIDELINES FOR PERCUTANEOUS ENDOSCOPIC GASTROSTOMY (PEG) TUBE PLACEMENT. L. Rabaneck. L. B. McCullough, N.P. Wray. VA Medical Center HSRD persistent vegetative state; uncomplicated dysphagia; and complicated dysphagia. We then developed an algorithm to guide physicians in deciding when to offer and recommend PEG tube placement for patients in each category. In the past, the decision to place a PEG tube has mainly centered on the patient's inability to take food by mouth. We contend that PEG tube placement is a medical intervention no different from any other. In deciding whether to offer PEG tube placement, beneficence-based clinical judgment must be complete, and take into account the patient's overall quality of life. The patient's autonomy must also be respected. By placing the decision in the context of this ethical framework, we have developed clinically sensible and ethically sound guidelines for the procedure.

Patient preferences for stroke outcomes.

In clinical trials stroke is reported as a major morbid outcome, but the impact of stroke on patients is not directly assessed. This study examines patient preferences for different outcomes of stroke, including death. We presented patients with written case scenarios of stroke outcomes. The scenarios represented four categories of stroke severity (mild, moderate, severe, and fatal), and for nonfatal strokes the scenarios described motor, language, and cognitive deficits. Patients reported values for each of the 10 stroke scenarios using a rank-and-scale method over a 100-point range, with 100 representing perfect health and 0 corresponding to the worst possible health state. One hundred seventeen of 209 consecutive patients at risk for stroke participated in this study. Severe strokes were uniformly rated as having low preference weights (mean +/- SD [median]: 3 +/- 4 [1] for disabling hemiplegia, 8 +/- 9 [5] for confusion, and 15 +/- 14 [10] for global aphasia), and severe motor impairment (a disabling hemiplegia) was rated as significantly worse than death. Even mild deficits resulted in substantial loss to patients (54 +/- 21 [55] for dysarthria and 53 +/- 21 [50] for mild anomia). Strokes may result in a wide variety of post-stroke consequences for patients. Severe strokes may be viewed by patients as tantamount to or worse than death. Even mild strokes may cause significant declines in patient preferences for health states. These data are useful in interpreting studies that report stroke and death, in designing new studies that measure stroke in at-risk populations, and in helping patients reach treatment decisions about therapies designed to prevent strokes.

Management and prevention of thromboembolic events in patients with cancer-related hypercoagulable states: a risky business.

To determine the optimal strategy for managing and preventing thromboembolic events in malignancy-associated hypercoagulable states. A Markov-based decision and cost-effectiveness analysis was performed. The authors explicitly considered consequences of embolic and bleeding events, filter complications, and cancer-related excess mortality. Data were drawn from the current literature. The main outcome measure for each strategy was the quality-adjusted life expectancy and the total average variable costs. Patients with advanced malignancies prone to develop thromboembolic events, patients with acute proximal deep venous thrombosis (DVT), and patients who have survived a first episode of pulmonary embolism (PE). The authors considered three different interventions: 1) OBSERVATION, in which neither anticoagulant therapy nor filter placement is pursued, 2) ANTICOAGULATION, in which long-term anticoagulant therapy is started immediately, and 3) VENA CAVAL FILTER. Vena caval filter was the preferred strategy for every malignancy studied, yielding an 11% gain in quality-adjusted life expectancy, compared with observation, for patients with acute DVT, and an 18% gain for patients having survived a PE. Anticoagulant therapy yielded gains of 9% and 16%, respectively. Compared with anticoagulant therapy, filter was less costly due to the avoidance of additional expenses incurred by bleeding events. Prophylactic therapy was the least effective of the three strategies examined. Vena caval filter placement and long-term anticoagulation therapy yield similar outcomes in the setting of cancer-related hypercoagulable states. However, filter insertion is less expensive than anticoagulation. Given the short life expectancy and morbidity of patients with end-stage malignancy, patient preferences for health states must be considered in the decision-making process. If active treatment is pursued, vena caval filter should be used as a primary therapy. Prophylactic therapy is not warranted in any circumstance.