Introduction
Endoscopic eradication therapy (EET), consisting of mucosal resection and ablation
techniques, has emerged as a viable alternative to surgical esophagectomy for definitive
therapy of Barrett’s esophagus (BE)-associated mucosal neoplasia. In the largest reported
series, 1000 consecutive patients with T1 esophageal adenocarcinoma (EAC) underwent
EET with a complete response rate of 96 %. During a mean follow-up of 56.6 months,
only 12 patients required salvage esophagectomy and only two patients died of EAC
[1]. An increasing number of patients in the United States now undergo endoscopic therapy
rather than surgery, with data from the Surveillance Epidemiology and End Results
(SEER) database reporting that the proportion of patients with high grade dysplasia
(HGD) or stage T1 EAC undergoing endoscopic therapy rose from 3 % in 1998 to 29 %
in 2009 [2].
Medical decision making is a critical component of current healthcare delivery, and
patients themselves have a central role in this process [3]. Current practice guidelines of the American Gastroenterological Association endorse
the concept of shared decision-making between physician and patient when considering
endoscopic intervention for BE [4]. Yet there exists a critical gap in understanding how an individual patient newly
diagnosed with esophageal cancer arrives at a decision to undergo either endoscopic
or surgical therapy.
There are precedent data examining patient decision-making in the selection of cancer
treatment options. For instance, patients with breast cancer may face a choice between
less invasive and more invasive treatment options – ranging from breast conserving
surgery, to mastectomy, to contralateral prophylactic mastectomy. Patient anxiety
over future cancer risk has been identified as a factor influencing patient preference
for breast cancer intervention [5]. Data from breast cancer research also provide insight into patient involvement
in the decision-making process, decision confidence, and decision regret.
The objective of this study was to assess patient involvement in decision making,
decision confidence, and decision regret in patients who have undergone EET or esophagectomy
for treatment of BE-associated HGD or T1 EAC, and to identify factors associated with
patient selection of endoscopic versus surgical therapy. The hypothesis was that high
rates of decision confidence and low rates of decision regret would be present in
both treatment groups.
Patients/Material and methods
Patients/Material and methods
Approval to conduct this study was granted by the Vanderbilt University Institutional
Review Board. Patients who had undergone either surgical esophagectomy or endoscopic
therapy, the latter consisting of endoscopic mucosal resection (EMR) and/or radiofrequency
ablation (RFA), both of which were available and performed at our institution, were
retrospectively identified from surgical and endoscopic databases. Patients with a
pre-intervention diagnosis of BE containing HGD or T1a (intramucosal) EAC were eligible
for inclusion.
Eligible subjects were contacted between January and October 2015 and invited to complete
a two-part survey during a scheduled clinic or endoscopy visit or via scripted telephone
interview. The first component of the survey, derived from published literature in
decision making [6], was designed to assess patient involvement in the treatment decision (Supplementary Appendix 1). The second component of the survey was designed to identify factors which influenced
the subject’s treatment decision, to assess the subject’s digestive functional status
following intervention, and to assess decision confidence and decision regret [7] (Supplementary Appendix 2).
Additional clinical and demographic data were extracted from the electronic medical
record, including the specialty (surgeon or gastroenterologist) of the initial consulting
physician within the institution.
Study data were recorded and managed using the Research Electronic Data Capture (REDCap)
tools hosted at Vanderbilt University. REDCap is a secure, web-based application designed
to support data capture for research studies. Statistical analysis was performed using
JMP software (SAS Institute, Cary, NC, United States). Parametric testing with the
Student’s t test was used for comparison of continuous data. Fisher’s exact test was used for
comparison of categorical data. The threshold for statistical significance for all
analyses was set at a two-sided P value of less than 0.05.
Results
In total, 68 patients who underwent EET for HGD/T1a EAC were eligible for study participation;
among these, 35 agreed to participate and completed the surveys. We were unable to
contact 25 subjects during the study period, one subject was deceased, and seven subjects
were contacted but declined enrollment – for a participation rate of 83 % (35/42)
among contacted subjects and an overall participation rate of 51 % (35/68). A total
of 26 patients who underwent esophagectomy for HGD/T1a EAC were eligible for study
participation; among these, 15 agreed to participate and completed the surveys. We
were unable to contact seven subjects, during the study period, two subjects were
deceased, and two declined enrollment – for a participation rate of 88 % (15/17) among
contacted subjects and an overall participation rate of 58 % (15/26).
Overall, 50 subjects were enrolled and completed the survey, 70 % (35/50) of whom
had undergone endoscopic therapy and 30 % (15/50) of whom had undergone esophagectomy.
Mean age at time of survey completion was 66 years in the endoscopic treatment group
compared to 62 years in the esophagectomy group (P = 0.17). All patients in the esophagectomy group were at least 1 year post-esophagectomy
at the time of survey administration. The majority of patients were male (80 % of
endoscopic group vs 93 % of esophagectomy group, P = 0.41).
Among the 50 patients surveyed, all lesions were discovered by a gastroenterologist.
Eighty percent of the lesions were discovered outside of our health system, and all
lesions were confirmed by expert pathology review before treatment. Of the total cohort,
48 % (24/50) saw both a surgeon and a gastroenterologist in consultation. Of the 12
patients who had seen a surgeon in initial consultation, eight were treated with EET
and four were treated with esophagectomy. In contrast, of the 12 patients who had
seen a gastroenterologist in initial consultation, four were treated with EET and
eight were treated with esophagectomy. Fifty-two percent (26/50) of patients did not
visit with both a surgeon and a gastroenterologist. Of these 26, three met with a
surgeon and were treated with esophagectomy, while 23 met with a gastroenterologist
and were treated with EET.
Patient involvement in decision-making
Seventy-one percent (25/35) of patients who underwent EET reported that esophagectomy
was discussed as a treatment option and 80 % (12/15) of subjects who underwent esophagectomy
reported that EET was discussed as a treatment option.
Sixty-six percent (23/35) of EET patients and 93 % (14/15) of esophagectomy patients
recall being asked whether they preferred EET or surgery (P = 0.04).
Factors involved in patient decision
Among subjects who underwent EET and identified factors influencing treatment choice,
risk of surgery was reported by 55 % (16/29) as the most important reason for selecting
EET rather than surgery ([
Fig. 1a
]). Among subjects who underwent esophagectomy, fear of cancer spread was reported
by 67 % (10/15) and desire to avoid multiple treatment sessions was reported by 33 %
(5/15) of subjects as the most important reasons for selecting esophagectomy rather
than EET ([
Fig. 1b
]).
Fig. 1 a Most important factor influencing choice for endoscopic eradication therapy over
esophagectomy. b Most important factor influencing choice for esophagectomy over endoscopic eradication
therapy.
Eight percent (4/50) of subjects reported that they had a family member or friend
who had undergone endoscopic treatment for BE or EAC, while 12 % (6/50) reported they
had a family member or friend who had undergone surgery for BE or EAC. No patients
in the EET group and 20 % (3/15) of patients in the esophagectomy group reported having
had the opportunity to speak before treatment with another patient who had undergone
similar treatment for this condition. No patients in the EET group and 73 % (11/15)
of patients in the esophagectomy group responded that they would have liked the opportunity
to speak before treatment with another patient who had undergone similar treatment.
Decision confidence and decision regret
Subjects who underwent esophagectomy were more likely to report post-treatment dysphagia
compared to subjects who underwent EET (47 % vs 14 %, P = 0.03), and were more likely to report that their ability to eat a normal diet had
changed (73 % vs 6 %, P < 0.0001) ([
Fig. 2
]).
Fig. 2 Percentage of patients reporting post-treatment dietary change or dysphagia after
undergoing endoscopic eradication therapy or esophagectomy.
On a five-point ordinal scale (“Compared to how I felt before undergoing treatment/surgery,
my current overall health is: much better, somewhat better, the same, somewhat worse,
or much worse), subjects who underwent esophagectomy were less likely than those who
underwent EET to view their overall post-treatment health favorably compared to pretreatment
health (P = 0.047).
Yet when asked whether they had in hindsight selected the right treatment approach,
there was no difference in decision confidence (mean 9.8 for EET vs 9.3 for esophagectomy
on a 0 – 10 scale, P = 0.12) ([
Fig. 3a
]). There was also no difference in decision regret: 97 % (34/35) of EET patients
and 80 % (12/15) of esophagectomy patients indicated they would select the same treatment
option (definitely yes or probably yes) if faced again with the same treatment decision
(P = 0.08) ([
Fig. 3b
]).
Fig. 3 a Decision confidence for treatment choice of EET vs esophagectomy. b Decision regret for treatment choice of EET vs esophagectomy.
Discussion
To our knowledge, this is the first study to assess decision confidence, decision
regret, and patient involvement in decision making among patients choosing between
endoscopic or surgical therapy for BE associated neoplasia. The demographic characteristics
of the cohort mirror those of the general population with BE, therefore the study
results are likely to be representative of the decision process experienced by patients
with BE associated neoplasia evaluated at a tertiary referral center and selecting
between endoscopic and surgical therapy.
Although patients with BE neoplasia are evaluated in multidisciplinary fashion at
our institution, each consultant discussed treatment options, both EET and esophagectomy,
with the patient typically before formal multidisciplinary review. Prior data reported
by the study authors in a similar patient population showed a strong correlation between
provider specialty and treatment choice [8]. We did not observe a similar degree of specialty bias in our study, though this
potential bias may have been attenuated by the size of the cohort.
There are potential advantages and disadvantages to either EET or esophagectomy for
treatment of BE neoplasia. EET is less invasive and carries low rates of procedural
morbidity; however, it does require a longitudinal commitment to multiple treatment
sessions followed by long-term (perhaps indefinite) post-treatment surveillance given
recurrence risk. Surgical therapy, on the other hand, is more invasive and has higher
up-front morbidity, yet offers the allure of a “one-and-done” cure in a single intervention.
In this context, risk of surgery was reported by the majority of patients in the EET
cohort as the most important reason for selecting EET rather than esophagectomy. Fear
of cancer spread was reported by the majority of patients in the esophagectomy cohort
as the most important reason for selecting esophagectomy rather than EET. These findings
are consistent with data reported by Rosmolen et al. from a European cohort demonstrating
that patients who undergo EET for BE neoplasia are more likely to experience fear
of cancer recurrence post-treatment than patients who undergo esophagectomy [9].
Curiously, fear of cancer spread was reported by 41 % of subjects in the EET arm as
a reason for selecting EET rather than esophagectomy. This was not an expected finding,
and the reason(s) for this finding may include poor representation of the posed question,
misunderstanding of the relative ability of EET or esophagectomy to prevent cancer
spread, or a perception that esophagectomy might contribute to cancer spread. Rephrasing
this question to reflect the ability of EET to stop progression to cancer, rather
than prevent spread of cancer may alter these results. Regardless, the fear of cancer,
both its progression and potential spread, looks to play an important role in selection
of therapy.
Cost influences medical decision making and may make the one time monetary outlay
of esophagectomy more appealing. Depending on insurance type and plan, the upfront
cost for esophagectomy is likely higher than for EET, but may prove to be more cost
efficient in the long term by eliminating the downstream cost of multiple treatment
sessions and indefinite endoscopic surveillance that is inherent with EET. In our
cohort, 13.8 % of patients choosing EET indicated that medical insurance played a
role in their decision making compared to 6.7 % of patients choosing esophagectomy.
No patient indicated that medical insurance status was the most important factor in
the treatment decision. As is often the case in a privately insured medical system,
it is unlikely that patients were aware of the overall individual cost at the time
of treatment decision. We did not collect specific insurance type as a part of our
survey, and did not calculate each patient’s out of pocket cost. If individual cost
to the patient was more transparent and readily available, it may impact treatment
decision.
Subjects who had undergone esophagectomy were more likely to report post-treatment
dysphagia, were more likely to report that their ability to tolerate a normal oral
diet had changed, and were less likely to view their post-treatment health favorably
compared to subjects who had undergone EET. However, high rates of decision confidence
were reported with no meaningful difference in comparing subjects who had undergone
EET vs esophagectomy (9.8 vs 9.3 on a 10-point scale, P = 0.12). Rates of decision regret were also similar: 80 % of subjects who underwent
esophagectomy and 97 % of subjects who underwent EET reported they “definitely” or
“probably” would select the same treatment choice again (P = 0.08). While a difference in this measure might be discernible in a study with
larger sample size, on a base level, these findings indicate that the majority of
patients are satisfied with their treatment choice, irrespective of modality, and
despite apparent differences in symptom-specific outcomes. This may serve as a striking
reminder that, despite the emergence of EET in an era in which endoscopists may be
conditioned to view the need for surgical therapy as a failure of endoscopic management,
the option of surgical intervention may remain a desirable option for some patients.
A retrospective study of this nature may be subject to recall bias. For instance,
93 % of esophagectomy patients recall being asked which treatment they preferred;
however, a percentage which exceeds the 80 % of patients who report that discussion
of the alternative treatment option in fact occurred.
Studies of decision regret in patients undergoing therapy for prostate cancer have
suggested that physiologic dysfunction plays a significant role in decision regret,
such that patients with more physiologic dysfunction are more likely to regret their
treatment decision [10]. We suspect that the lack of a significant difference in decision regret among patients
undergoing esophagectomy despite worse physiologic outcomes is influenced by the concept
of “cognitive dissonance reduction”. In accordance with this theory, individuals tend
to reduce or minimize inconsistencies between previous decisions and current beliefs
or treatment preferences [11]. As a result, subjects would be expected to adjust current beliefs in favor of the
treatment which they had received, and thus report a higher degree of decision confidence
and a lower degree of decision regret than is commensurate with post-treatment symptoms
and health. We expect the effect of cognitive dissonance reduction to be strongest
in patients who experienced a relatively worse functional outcome, yet still maintained
a high degree of decision confidence and a low degree of decision regret; however,
there is likely considerable interpatient variability, and the magnitude of this effect
is hard to measure absolutely. A study with responses measured before a treatment
decision and monitored prospectively might in theory minimize this source of potential
bias.
Participation in our survey was voluntary and our study population included all patients
who underwent therapy during the study period. We did not identify any specific predictive
factors for survey participation, but we did note that patients who declined to participate
in the survey of the EET arm were slightly older than patients who chose to participate
(77 years vs 66 years) and were all male. As with any survey, there is the possibility
of self-selection bias where more respondents with either strongly positive or strongly
negative feelings would be more likely to participate. The lack of variability in
decision confidence and decision regret within groups suggests that this effect is
small.
Our survey was either administered during a scheduled gastroenterology clinic or endoscopy
visit at our institution or by an interviewer identifying himself as affiliated with
Vanderbilt University. This distinction opens the possibility that survey responses
could be influenced by auspices bias, by which responses are influenced by the organization
conducting the survey. It is possible that responses may have differed if the surveyor
identified himself as a surgeon, or did not reveal any institutional affiliation.
This was a single-center retrospective experience, in which one physician served as
both the treating endoscopist and the clinical consultant for all EET cases. This
factor may decrease practice variability in the study, but may lead to a lack of generalizability
and external validity. The size of our cohort, particularly the esophagectomy cohort,
limits the power of statistical analysis, and it is possible that a larger sample
size could influence study findings. A multicenter prospective study would be ideal
to further investigate these findings, but this may be a moving target in the midst
of a paradigm shift: as rates of esophagectomy for BE HGD/T1a EAC decline, future
enrollment of a sizable surgical cohort may prove difficult.
The study instruments were adapted from studies involving breast cancer treatment,
but have not yet been validated in the setting of patient decisions for treatment
of BE associated neoplasia. Also, health literacy was not formally assessed as a part
of our study.
In summary, this retrospective survey of patients who underwent endoscopic eradication
therapy or esophagectomy for BE with HGD or T1a EAC at a tertiary referral center
identified a high degree of patient involvement in the decision-making process. Despite
differences in specific symptom outcomes, measures of decision confidence and decision
regret do not differ statistically between the two treatment groups. Continued attention
to patient preferences, dedication to informed, shared decision making, and further
prospective study of factors influencing treatment selection should result in desirable
treatment outcomes for patients with BE-associated neoplasia irrespective of treatment
modality.
Appendix 1
Involvement in decision making assessment – Retrospective survey #1
Appendix 1
Involvement in decision making assessment – Retrospective survey #1
Endoscopic treatment cohort
Instructions: please choose the one best response for each question
-
Did any of your doctors discuss surgery as an option for you?
Yes No I’m not sure
-
How much did your doctors discuss reasons to have endoscopic therapy with you?
A lot Some A little Not at all I’m not sure
-
How much did your doctors discuss reasons not to have endoscopic therapy with you?
A lot Some A little Not at all I’m not sure
-
How much did your doctors discuss reasons to have surgery with you?
A lot Some A little Not at all I’m not sure
-
How much did your doctors discuss reasons not to have surgery with you?
A lot Some A little Not at all I’m not sure
-
Did any of your doctors ask you whether you preferred endoscopic therapy or surgery?
Yes No I’m not sure
Esophagectomy cohort
Instructions: please choose the one best response for each question
-
Did any of your doctors discuss endoscopic therapy as an option for you?
Yes No I’m not sure
-
How much did your doctors discuss reasons to have surgery with you?
A lot Some A little Not at all I’m not sure
-
How much did your doctors discuss reasons not to have surgery with you?
A lot Some A little Not at all I’m not sure
-
How much did your doctors discuss reasons to have endoscopic therapy with you?
A lot Some A little Not at all I’m not sure
-
How much did your doctors discuss reasons not to have endoscopic therapy with you?
A lot Some A little Not at all I’m not sure
-
Did any of your doctors ask you whether you preferred surgery or endoscopic therapy?
Yes No I’m not sure
Appendix 2
Retrospective survey #2
Appendix 2
Retrospective survey #2
Endoscopic treatment cohort
-
Current age years
-
Gender (circle one) Male Female
-
Surgery was offered to me as an alternative to endoscopic treatment:
Yes No
-
I have a family member or friend who underwent endoscopic treatment for Barrett’s
esophagus or esophageal cancer:
Yes No
-
I have a family member or friend who underwent surgery for Barrett’s esophagus or
esophageal cancer:
Yes No
-
Reasons I chose endoscopic treatment rather than surgery include (circle all which
apply)
a) My age
b) My other medical condition(s)/illness(es)
c) Risks of surgery
d) Fear of cancer spread
e) My health insurance
-
The most important reason I chose endoscopic treatment rather than surgery was (circle
one):
a) My age
b) My other medical condition(s)/illness(es)
c) Risks of surgery
d) Fear of cancer spread
e) My health insurance
-
Before undergoing treatment, I had the opportunity to speak with another patient who
had undergone endoscopic treatment for this condition:
Yes No
-
I would have liked the opportunity to speak with another patient who had undergone
endoscopic treatment for this condition:
Yes No
-
Since undergoing treatment, I have difficulty swallowing:
Yes No
-
Since undergoing treatment, my ability to eat a normal diet has changed:
Yes No
-
Compared to how I felt prior to undergoing treatment, my current overall health is
(circle one):
a) Much better
b) Somewhat better
c) The same
d) Somewhat worse
e) Much worse
-
On a scale of 0 to 10 where 0 is not at all confident and 10 is extremely confident,
how confident are you that the decision about treatment was the right one for you”
(circle one response)
0 1 2 3 4 5 6 7 8 9 10
-
If you had the chance to make the decision again, would you have the same type of
treatment (circle one response):
a) Definitely yes
b) Probably yes
c) Not sure
d) Probably no
e) Definitely no
Esophagectomy cohort
-
Current age years
-
Gender (circle one) Male Female
-
I have a family member or friend who underwent endoscopic treatment for Barrett’s
esophagus or esophageal cancer:
Yes No
-
I have a family member or friend who underwent surgery for Barrett’s esophagus or
esophageal cancer:
Yes No
-
Reasons I chose surgery rather than endoscopic treatment include (circle all which
apply)
a) My age
b) My other medical condition(s)/illness(es)
c) Fear of cancer spread
d) My health insurance
e) Desire to avoid multiple treatment sessions
-
The most important reason I chose surgery rather than endoscopic treatment was (circle
one):
a) My age
b) My other medical condition(s)/illness(es)
c) Fear of cancer spread
d) My health insurance
e) Desire to avoid multiple treatment sessions
-
Before undergoing treatment, I had the opportunity to speak with another patient who
had undergone surgery for this condition:
Yes No
-
I would have liked the opportunity to speak with another patient who had undergone
surgery for this condition:
Yes No
-
Since surgery, I have difficulty swallowing:
Yes No
-
Since surgery, my ability to eat a normal diet has changed:
Yes No
-
Compared to how I felt prior to undergoing surgery, my current overall health is (circle
one):
a) Much better
b) Somewhat better
c) The same
d) Somewhat worse
e) Much worse
-
On a scale of 0 to 10 where 0 is not at all confident and 10 is extremely confident,
how confident are you that the decision about treatment was the right one for you”
(circle one response)
0 1 2 3 4 5 6 7 8 9 10
-
If you had the chance to make the decision again, would you have the same type of
treatment (circle one response):
a) Definitely yes
b) Probably yes
c) Not sure
d) Probably no
e) Definitely no
Acknowledgments
Grants and financial support: NIH/NCATS grant UL1 TR000445 for Vanderbilt Institute
for Clinical and Translational Research.
