Gastrointestinal Unit, Massachusetts General Hospital, Boston, MA, USA
Institute for Technology Assessment, Massachusetts General Hospital, Boston, MA, USA
Department of Health Policy and Management (GSG), Harvard School of Public Health, Boston, MA, USA
Abstract
Background
The management of Barrett's esophagus (BE), particularly high grade dysplasia (HGD), is an area of much debate and controversy. Surgical esophagectomy, intensive endoscopic surveillance and mucosal ablative techniques, especially photodynamic therapy (PDT), have been proposed as possible management strategies. The purpose of this study was to determine the health related quality of life associated with Barrett's esophagus and many of the pivotal health states associated with Barrett's HGD management.
Methods
20 patients with Barrett's esophagus were enrolled in a pilot survey study at a large urban hospital. The utility of Barrett's esophagus without dysplasia (current health state) as well as various health states associated with HGD management (hypothetical states as the subject did not have HGD) were measured using a validated health utility instrument (Paper Standard Gamble). These specific health states were chosen for the study because they are considered pivotal in Barrett's HGD decision making. Information regarding Barrett's HGD was presented to the subject in a standardized format that was designed to be easily comprehendible.
Results
The average utility scores (0–1 with 0 = death and 1 = perfect health) for the various Barrett's esophagus associated states were: BE without dysplasia-0.95; Post-esophagectomy for HGD with dysphagia-0.92; Post-PDT for HGD with recurrence uncertainty-0.93; Post-PDT for HGD with recurrence uncertainty and dysphagia-0.91; Intensive endoscopic surveillance for HGD-0.90.
Conclusion
We present the scores for utilities associated with Barrett's esophagus as well as various states associated with the management of HGD. The results of our study may be useful in advising patients and providers regarding expected outcomes of the various HGD management strategies as well as providing utility scores for future cost-effectiveness analyses.
Background
Barrett's esophagus (BE) is a result of chronic reflux disease and is a risk factor for esophageal adenocarcinoma
Although surgical esophagectomy is considered by many as the standard management for esophageal cancer in those patients who are operative candidates, a consensus regarding the optimal management of HGD does not exist. Publications have reported a wide range 27–73%
Mucosal ablation is an area of much current investigation and provides an intermediate option between surgery and endoscopic surveillance, with the most data available for photodynamic therapy (PDT). PDT is an endoscopic ablative treatment that has successfully treated patients with BE and early esophageal cancer or HGD who have traditionally been poor operative candidates for esophagectomy
Methods
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
Study administration and materials
The standard gamble instrument is considered the gold standard method for utility measurement
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
Table
References for characteristics of health states
Characteristic
Patient Simplified Description
Published Values
References
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]
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]
EGD every 3–6 months for 2 years and then yearly
Hur [15]
Intensive Endoscopy
EGD every 3 months
Sampliner [33]
Abbreviations: EGD-upper endoscopy; Sx-surgery; PDT-photodynamic therapy.
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
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.
Results
Clinical and demographic features
The mean age of the subjects in the study was 64.6 years and 55% (11/20) were male. 20% of the subjects had undergone a Nissen fundoplication surgery, 15% had a history of dysphagia and 10% had a history of Barrett's low grade dysplasia that subsequently regressed on follow-up endoscopic biopsies (Table
Patient Characteristics
Characteristics
Mean
Range
Age
64.6
49–77
Sex
Male
55%
Female
45%
Prior Nissen
Fundoplication
20%
History of dysphagia
15%
History of low grade dysplasia
10%
Questionnaire responses
The paper standard gamble utility scores are presented in Table
Utilities Associated with Various Barrett's Esophagus Health States
Health State
Mean (Median)
Range
SD
Barrett's esophagus (no dysplasia)
0.95 (0.98)
0.775–0.995
0.067
Post-Esophagectomy with dysphagia
0.92 (0.96)
0.725–0.995
0.079
Post-PDT (no dysphagia)
0.93 (0.98)
0.550–0.995
0.107
Post-PDT with dysphagia
0.91 (0.96)
0.550–0.995
0.117
Intensive Endoscopic Surveillance
0.90 (0.95)
0.550–0.995
0.138
Abbreviations: PDT-photodynamic therapy.
The average rating of the interview quality or subject comprehension graded by the interviewer was 2.75 with all interviews rated either 2 or 3 (1–3 scale).
Discussion
We present estimates of utilities for Barrett's esophagus as well as various health states associated with Barrett's HGD management and therapy using the Paper Standard Gamble instrument. Although other studies have analyzed quality of life in patients with gastroesophageal reflux disease (GERD) and BE, our analysis is the first to present utilities associated with many of the pivotal health states associated with Barrett's HGD management.
Gerson et al. recently published the results of an analysis which used a computer program to elicit utilities from patients with GERD as well as a subset of patients who also had BE
A limitation to the study was the relatively small sample size. This is of particular concern as large variations in quality of life were found among those who provided scores for BE. The congruency in our BE without dysplasia utility score and those of Gerson et al.
Except for utilities scores for BE without dysplasia, the other utilities were evaluated for hypothetical states. Community or population utilities approximate societal values, which can be estimated by sampling general society. Especially if a disease or health state is rare, the societal value for a disease health state would be approximated by interviewing individuals who probably will not have the disease but would be asked to imagine a specific health state and then to assign a value to it using an instrument for this purpose
Although our study subjects all had BE and some familiarity with many aspects of the hypothetical health states described, in order to present the information surrounding this clinical issue to participants who were presumed not to have prior medical training, it was necessary to simplify medical complexities to make it comprehendible and also to limit cognitive burden (see Methods section). The process of simplification could have theoretically led to biases, which could have influenced the participants' choices, which is a possible limitation to the study estimates for the hypothetical utility scores. The possibility of biases in these types of studies is, to a large part, unavoidable. However, the best efforts were made by the investigators to construct simplified presentations that were objective and based on published literature.
Conclusion
Our study findings confirm the BE without dysplasia utility score previously reported
Abbreviations
BE Barrett's esophagus;
EGD esophagogastroduodenoscopy;
HGD high grade dysplasia;
PDT photodynamic therapy.
Competing interests
The author(s) declare that they have no competing interests.
Authors' contributions
CH participated in the design, administration, statistical analysis, and manuscript preparation. EW participated in the design, statistical analysis and manuscript preparation. NSN and GSG contributed to study design and manuscript preparation.
Hur additional file. Appendix: 1. Paper Standard Gamble Survey; 2. Imagined Paper Standard Gamble; 3. Post Successful Esophagectomy with Dysphagia State Description; 4. Post Successful PDT (no dysphagia) Description; 5. Post Successful PDT with Dysphagia Description; 6. HGD Management with Intensive Endoscopic Surveillance Description
Click here for file
Acknowledgements
Supported by the American Gastroenterological Association's Research Scholars Award and by the National Institutes of Health (1K07 CA107060).