Patients
Patients with documented (by histology) Barrett's esophagus over the age of 18 who were either having an endoscopy or a clinic visit within the Massachusetts General Hospital (large, urban hospital) GI Associates' practice were identified by one of the investigators using the practice's patient scheduling system. Subjects gave informed consent prior to participation and received no remuneration.
After permission was obtained from the patient's physician, the investigator invited the potential subject to participate. A total of 26 patients were asked to participate in this study and 20 completed the study. The institutional review board overseeing human research at the Massachusetts General Hospital approved the study.
Patients recruited in the endoscopy unit (18/20) were approached prior to their endoscopy, and if willing, a future telephone appointment was made to administer the questionnaire. The subject was also given written copies of the questionnaires (described in next section) in a packet to take home for review prior to the telephone call. Alternatively, if the subject was recruited in the outpatient clinic (2/20), the questionnaire was administered in person after the scheduled physician visit.
Regardless of the method used to administer the survey, the investigator attempted to standardize the interview as much as possible.
Patients with Barrett's esophagus were chosen for the study because they would be familiar with endoscopic surveillance and may have considered many of the issues regarding HGD management, thereby making them an informed and realistic patient population facing these decisions. Although patients with HGD or prior HGD were excluded, patients with prior LGD were included.
The description of the patient recruitment and separate data acquired from these recruited subjects have been previously published [
17]. However, the data presented in this manuscript are the results of a new analysis using distinct data that have not yet been published (except in abstract form) [
18].
Study administration and materials
The standard gamble instrument is considered the gold standard method for utility measurement [
19]. To assess utilities in our subjects with BE, we used a previously validated paper version of the standard gamble [
16,
20] (Appendix [see Additional file
1]).
After measuring the subjects' utility for their current health state (Barrett's esophagus without dysplasia), each enrolled patient was then asked to assess four hypothetical health states that might result from management strategies for Barrett's HGD. These states included: 1) Post-surgical esophagectomy for HGD without concern regarding BE or dysplasia recurrence but with dysphagia; 2) Post-successful PDT for HGD with concern about an unknown chance of recurrence but no dysphagia; 3) Post-successful PDT for HGD with concern about an unknown chance of recurrence and with dysphagia; 4) Intensive Endoscopic Surveillance (Appendix for complete descriptions of all the health states [see Additional file
1]). Each of these hypothetical health states was presented in a standardized format including risks of BE and HGD recurrence, future endoscopic surveillance regimens and possible morbidity or side effects.
Table
1 presents the estimates for various aspects of the strategies portrayed in the health state descriptions with references to the published literature upon which they are based. In constructing the descriptions of the various health states, a careful balance was sought between accurately portraying the medical complexities involved in each state and minimizing "cognitive burden" (i.e., effort required to perceive, think and remember) as described in Furlong et al.'s guide to health state questionnaires [
21]. A summary in bullet format was provided for each health state to help the subject keep the important factors in mind while undergoing utility assessment.
Table 1
References for characteristics of health states
Esophagectomy
| | | |
Sx Success Rate | "cured" | Recurrence 0–2%/year* | Rice [7], Ferguson [27] |
Dysphagia Treatment | "3 endoscopies" | | Headrick [28] |
Endoscopic Dilation Perforation | "1 in 200" | >0.25%/dilation | Bueno [29] |
Follow-up Surveillance
| | | |
EGD every year | | | Hur [30] |
Photodynamic Therapy
| | | |
Recurrence Risk | "chance of recurrence" | | Barham [31], Bonavina [32] |
Dysphagia Treatment | "3 endoscopies" | | Headrick [28] |
Endoscopic Dilation Perforation | "1 in 200" | >0.25%/dilation | Bueno [29] |
Follow-up Surveillance
| | | |
EGD every 3–6 months for 2 years and then yearly | | | Hur [15] |
Intensive Endoscopy
| | | |
EGD every 3 months | | | Sampliner [33] |
In the standard gamble (SG) utility assessment method, patients are offered an option such as an imaginary pill that will result in either perfect health or death. The maximum amount of risk of death that a patient is willing to assume for a chance at perfect health is determined and used to derive the utility of the health state in question [
22]. The SG instrument was originally administered face-to-face with trained interviewers, but the more recently developed Paper Standard Gamble was developed and validated so that instrument could be self-administered [
16]. In our study, although the Paper Standard Gamble (Appendix [see Additional file
1]) was used, a study investigator provided each subject with directions regarding the instrument and allowed the patient to ask questions, either in person (2/20), or by telephone (18/20) during utility measurements for each health state presented. All surveys were administered by a single investigator (C.H.) who tried, if it all possible, to limit the number of questions asked by subjects during the interview, in an attempt to maintain study standardization.
On average, this portion of the questionnaire took approximately 15 minutes to complete. At the end of the interview, the interviewer qualitatively assessed the perceived quality of the subject's comprehension on a scale of 1–3 (1-poor, 2-fair, 3-excellent).
The subject's demographic and clinical data were retrieved from the patient's electronic medical record after the interview was completed. The study instruments and algorithm were tested and refined on four (non-patient) subjects for feasibility and comprehension prior to use with actual study subjects. The primary refinements that resulted from this 'pre-testing' were further simplifications of many of the medical terms used to describe the various health states.
Data analysis
This was a descriptive, cross-sectional study where the results are presented as average (mean and median) scores with ranges and standard deviations. No statistical analyses or power calculations were performed for this pilot study.