doi:10.1111/codi.12538
Original article
Patient and clinician preferences for surgical and medical treatment options in ulcerative colitis C. M. Byrne*†, K.-K. Tan*†, J. M. Young*, W. Selby‡ and M. J. Solomon*† *Surgical Outcomes Research Centre (SOuRCe), Sydney Local Health District and University of Sydney, Sydney, Australia, †Department of Colorectal Surgery, Royal Prince Alfred Hospital, Sydney, Australia and ‡AW Morrow Department of Gastroenterology and Liver Centre, Royal Prince Alfred Hospital, Sydney, Australia Received 4 September 2013; accepted 6 November 2013; Accepted Article online 21 December 2013
Abstract Aim When treating patients with refractory ulcerative colitis (UC), the choice between escalating medical management or surgery can be difficult. The aim of this study was to quantify the preferences of patients and clinicians for the treatment options in UC.
more prepared to gamble or trade to avoid any surgery than were colorectal surgeons. All groups were aligned in their decision to undergo yearly colonoscopy surveillance rather than to undergo definitive surgery that would result in a stoma.
Method Ulcerative colitis outpatients were interviewed to measure their preferences for five scenarios examining the management of acute and chronic UC, using a prospective measure of preference method that generates two utility scores: willingness and amount of expected life to trade or gamble. A self-administered questionnaire was mailed to Australian and New Zealand colorectal surgeons and gastroenterologists.
Conclusion Patient preferences for the treatment of UC were more aligned to those of gastroenterologists than those of colorectal surgeons. Despite postoperative studies revealing an equal quality of life for pouch and stoma patients, this study confirmed that a pouch is the preferred surgical option.
Results Fifty-five patients (26 medical and 29 surgical), 91 surgeons and 78 gastroenterologists were surveyed. In the acute setting, 89% of patients, 69% of gastroenterologists and 55% of surgeons were willing to trade part of their life expectancy to avoid a permanent stoma, while for chronic disease 71% of patients were prepared to trade to avoid an operation with a permanent stoma compared with 55% for an operation with a pouch (P = 0.01). Both patients and gastroenterologists were
Introduction Two therapeutic pathways exist for the treatment of patients with refractory ulcerative colitis (UC). Medical management using anti-inflammatory and/or immunosuppressant medications is frequently effective for the majority of patients with UC, but in up to 30% of these patients, surgery is still necessary for indications such as failed medical therapy and neoplasia [1,2]. Whilst prolonged use of corticosteroid therapy is associated with Correspondence to: Michael J Solomon, Department of Colorectal Surgery, Surgical Outcomes Research Centre (SOuRCe), Royal Prince Alfred Hospital, Missenden Road, Camperdown, NSW 2050, Australia. E-mail: [email protected]
Keywords Preferences, surgery, ulcerative colitis, decision-making What does this paper add to the literature? Patient preferences for the treatment of UC were more aligned to those of gastroenterologists than those of colorectal surgeons. Despite postoperative studies revealing an equal quality of life for pouch and stoma patients, this study has confirmed that a pouch is the preferred surgical option.
numerous health problems, long-standing colitis is associated with an increased risk of bowel cancer and other associated complications [3–7]. If the aforementioned difficulties are encountered, surgery is then necessary. In an elective setting, two surgical options exist. Total proctocolectomy with end ileostomy removes all colonic mucosa, albeit with a permanent stoma. Although a restorative ileo-anal pouch operation avoids the need for a permanent stoma, it has its own set of short- and long-term difficulties. There is also a higher rate of subsequent surgery for pouch-related complications [8,9]. Patients with UC and their clinicians are thus confronted by numerous potentially conflicting factors when choosing between surgical or medical therapy.
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Before choosing any therapy, the preference of the patient is an important consideration. This is especially pertinent when the intervention has the potential to influence their quality of life (QOL) considerably [10]. Despite the notion that patient preferences should always be taken into account as part of informed decision-making during routine surgical practice involving a choice between therapies, the reality is somewhat different [11]. In a study of 1032 tape-recorded patient consultations by 60 primary care physicians and 60 surgeons, it was found that of the 3552 decisions made less than 20% actually included any assessment of patient preference [12]. It is not surprising that studies have suggested that more patients would prefer an increased role in the decision-making process [13,14]. Understanding patient preferences is important for clinicians when up to 40% of medical decisions are made by doctors for their patients [13,15–18]. There is also mounting evidence that surgeons and physicians have different preferences for treatment options from their patients and from each other [10,11,19–21]. To our knowledge, there have been no studies that have directly examined the decision-making process in UC when choosing between medical and surgical treatment options, and in particular between the choice of surgery resulting in a permanent stoma vs restorative procedures. This study was conducted with the particular aim of determining patient preferences for surgical intervention in UC prospectively rather than the postoperative quality of life measures in the surgical cohort alone. Secondly, we wanted to quantify the worth of living with a permanent stoma as opposed to a pouch, and finally to compare patient preferences with those of surgeons and gastroenterologists.
Method Patient recruitment
A consecutive sample of UC patients was selected retrospectively from clinical databases of their treating specialist. An information sheet, consent form and questionnaire were sent. The ethics review board stipulated that the patients had to ‘opt on’ by returning a signed consent form and no direct approach could be made by the researchers. One reminder letter was sent. Medical patients were defined as those who had never undergone colectomy. Script development and hypothetical scenarios
Standardized verbal scripts were developed to explain three treatment scenarios in acute UC and two in
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chronic UC. A prelude to the first set of scenarios described a patient with a 2-year history of active, corticosteroid-dependent UC affecting over half the colon who was now suffering an acute, severe, exacerbation that had not responded to high-dose corticosteroid therapy. Three treatment choices were presented: 1 Standard escalating medical therapy vs total proctocolectomy with an ileal J pouch; 2 standard escalating medical therapy vs total proctocolectomy with an end ileostomy; 3 total proctocolectomy with an ileal J pouch vs total proctocolectomy with an end ileostomy. A prelude to the remaining scenarios described an asymptomatic patient with a 20-year history of UC whose recent colonoscopy demonstrated ‘burnt-out’ inactive disease affecting three-quarters of the colon with no dysplasia on biopsies. Two treatment choices presented were: 1 Yearly colonoscopic surveillance vs total proctocolectomy with an ileal J pouch; 2 yearly colonoscopic surveillance vs total proctocolectomy with an end ileostomy. Prospective measure of preference
A clinician (C.M.B.) conducted telephone interviews using the standardized scripts explaining each treatment scenario in detail, including the benefits, risks and the likely long-term outcome. The major features of the two treatment options were summarized as bullet points in a visual aid. For each treatment choice, one option was presented as the standard treatment and the other as the alternative treatment. Once the patient had reached a level of understanding sufficient to make an informed treatment choice, patient preferences were elicited [10,11,19,22]. The previously described prospective measure of preference (PMP) method was utilized to elicit preferences [10,11,22]. This is a prospective modification of the time trade-off and standard gamble tools that have been previously described and validated in colorectal settings [10,11,19,22]. The PMP method produces two measures of preference. The first is a dichotomous variable (yes/no) of either willingness to gamble (WTG) or willingness to trade (WTT) life expectancy, and this is summarized as the proportion of study participants who would be willing to either gamble or trade (0–1.00). The second measure, either a prospective measure of preference standard gamble (PMPg) or a prospective measure of preference time trade-off (PMPt), is expressed as the group average utility of life expectancy given away ranging from 0 to 1.
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To determine WTG and the PMPg patients were asked to quantify the amount of their remaining life expectancy they would gamble (or risk) to avoid an alternative treatment option rather than the current option. To determine WTT and the PMPt patients were asked how much life expectancy they would trade (or give up) to avoid the alternative treatment option. PMPg involves an uncertain outcome (that is, a potential risk of losing life expectancy) whereas PMPt involves a certain, known outcome (that is, a definite amount of life given up). The greater the PMPg or PMPt the more averse patients are to the alternative treatment. PMP measures of more than 0.10 are indicative of strong preference. These measures have demonstrated strong test–retest correlations (R = 0.72–0.96) [19]. Specialist doctor recruitment
The patient questionnaire was converted to a selfadministered format to be mailed to specialist doctors. The questionnaire sent to clinicians elicited identical information to the patient questionnaire. Clinicians were asked to answer as if they were a patient with UC (surrogate patient). All 136 members and training fellows of the Colorectal Surgical Society of Australia and New Zealand (colorectal surgeons) and a random sample of 300 members of the Gastroenterological Society of Australia (gastroenterologists) were invited to participate.
Preferences in ulcerative colitis
Demographic and clinical details
The characteristics of the patient participants are shown in Table 1. Their mean age was 47 years and the mean duration of colitis was over 13 years. Almost 85% had used corticosteroids previously. Two-thirds had taken other immunosuppressant medication, while 40% reported complications of medical therapy. Over half had undergone abdominal surgery for UC and almost all of these had experienced an ileostomy, either temporary or permanent. Just over half of the patients had no current symptoms from their colitis. The mean duration of interview was 37 min. Escalating medical therapy vs total proctocolectomy with a J pouch
The first scenario explored the choice between escalating medical therapy with increasingly potent immunosuppressant therapy vs a restorative proctocolectomy with J pouch surgery. As shown in Table 2, both patients and gastroenterologists were aligned in their opinion and were more willing than colorectal surgeons to gamble or trade their life expectancy for escalating medical therapy rather than an operation. These differences were statistically significant. There was no statistical difference between the responses of UC patients and gastroenterologists.
Table 1 Demographic and past medical details of patients with ulcerative colitis.
Statistical analysis
Statistical analysis was performed using SPSS Version 13, blinded to participants’ group status. For each scenario WTG and WTT were compared between groups using the chi-square test, whereas the PMPg and PMPt were compared using the Wilcoxon rank sum test. Within groups PMPg and PMPt were compared between scenarios using the McNemar test or the Wilcoxon signed rank test as appropriate.
Results Response rate
A total of 55 (34%) out of 162 UC patients responded to a recruitment letter from their specialist (29 of 74 surgical patients and 26 of 88 medical patients). Ninety-one completed questionnaires were received from 127 (72%) eligible surgeons, while 78 (27%) completed questionnaires from 272 gastroenterologists were returned.
Characteristic Number Mean age (years) Gender (M/F) Mean disease duration (years) Previous corticosteroid use Other immunosuppressant use Complications of medications Emergency hospital admission Discussion with surgeon Previous abdominal surgery for ulcerative colitis Previous stoma Complications of surgery Current symptoms None Mild Moderate Marked Severe
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Number of patients (%) 55 47.7 26/29 13.3 47 33 20 32 35 30
(85) (60) (36) (58) (64) (56)
29 (54) 16 (29) 29 12 7 2 4
(54) (22) (13) (4) (7)
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Table 2 Preference measures and comparison between the various groups in scenarios 1 and 2 (preferences in severe acute-onchronic ulcerative colitis (UC) to have escalating medical therapy to avoid (1) J pouch surgery and (2) permanent stoma surgery).
Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(1) J pouch surgery UC patients (55) Colorectal surgeons (91) Gastroenterologists (74)
0.64 0.49 0.80
0.16 (0–0.20) 0.00 (0–0.10) 0.10 (0.05–0.20)
0.54 0.41 0.59
0.05 (0–0.13) 0.00 (0–0.05) 0.05 (0–0.10)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
0.08 0.59 0.011
0.01 0.38 0.002
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.10 0.038 < 0.001
0.004 0.73 < 0.001
Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(2) Permanent stoma surgery UC patients (55) Colorectal surgeons (91) Gastroenterologists (74)
0.75 0.68 0.85
0.15 (0–0.29) 0.10 (0–0.15) 0.13 (0.05–0.25)
0.71 0.51 0.69
0.10 (0–0.20) 0.01 (0–0.10) 0.05 (0–0.15)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.38 0.14 0.011
0.006 0.73 0.019
0.015 0.69 0.027
0.003 0.38 0.028
WTG, proportion willing to gamble; WTT, proportion willing to trade; PMPg, prospective measure of preference gamble; PMPg, prospective measure of preference trade; IQR, interquartile range. Bold values indicate statistically significant results. *P-value for v2 test. **P-value for Wilcoxon rank sum test.
Escalating medical therapy vs total proctocolectomy with a permanent stoma
When the option changed between escalating medical treatment vs a proctocolectomy with a permanent stoma, there was again a higher proportion of patients across all the groups willing to gamble to avoid this operation, including gastroenterologists (85% from 80%), patients (75% from 64%) and surgeons (68% from 49%) (Table 2). But as in the earlier option, both the gastroenterologists and patients were consistently similar and more willing than colorectal surgeons to gamble or trade. Proctocolectomy with a J pouch vs proctocolectomy with a permanent stoma
In the next scenario, large proportions of all groups were willing to gamble to undergo J pouch surgery to avoid a permanent stoma (Table 3). PMPg was 0.15 for both patients and gastroenterologists and 0.10 for sur-
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geons. Eighty-nine per cent of patients, 69% of gastroenterologists and 55% of surgeons were willing to trade to avoid the operation with the permanent stoma. The PMPt was 0.10 for patients, 0.07 for gastroenterologists and 0.05 for surgeons. More patients were willing to gamble or trade than both colorectal surgeons and gastroenterologists to achieve this outcome. This difference was statistically significant. Screening for cancer vs prophylactic total proctocolectomy
The last two scenarios were in a more elective setting whereby a patient with chronic UC has to choose between having cancer screening via yearly colonoscopy or surgery to avoid a 15% current risk of cancer that would increase by 1% per year. Higher numbers of participants in all three groups were willing to gamble or trade more life expectancy to avoid undergoing a restorative proctocolectomy than in previous scenarios
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Table 3 Preference measures and comparison between the various groups in scenario 3 (preferences in severe acute-on-chronic ulcerative colitis (UC) to have J pouch surgery to avoid permanent stoma).
Group (n)
WTG
Median PMPg (IQR)
WTT
UC patients (26) Colorectal surgeons (91) Gastroenterologists (74)
0.93 0.75 0.84
0.15 (0.10–0.26) 0.10 (0.02–0.20) 0.15 (0.05–0.30)
0.89 0.55 0.69
Comparison groups
WTG*
PMPg**
WTT*
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.009 0.14 0.18
0.004 0.61 0.028
< 0.001 0.006 0.08
Median PMPt (IQR) 0.10 (0.05–0.20) 0.05 (0–0.10) 0.07 (0–0.18) PMPt** < 0.001 0.019 0.06
WTG, proportion willing to gamble; WTT, proportion willing to trade; PMPg, prospective measure of preference gamble; PMPg, prospective measure of preference trade; IQR, interquartile range. Bold values indicate statistically significant results. *P-value for v2 test. **P-value for Wilcoxon rank sum test.
(Table 4). Because of the similar high scores between all three groups there were no significant differences observed. A similar response was observed when the choice was changed to involve an operation resulting in a permanent stoma. This scenario had the highest PMPg of all scenarios studied (between 0.18 and 0.20). Comparison between a permanent stoma and pouch surgery
The proportion of participants who were willing to gamble or trade in scenarios 1 and 2 was compared by the McNemar test for paired proportions (Table 5). Seventy-one per cent of patients were prepared to trade to avoid the operation with the permanent stoma compared with 55% for the operation with the pouch (v2 = 6.31, 1 df, P = 0.01). A significantly greater number of surgeons traded to avoid a permanent stoma (52%) than to avoid pouch surgery (39%; v2 = 4.89, 1 df, P = 0.03). Ten per cent of gastroenterologists changed their preference, which was not significantly different and reflected the high proportion of gastroenterologists who preferred to avoid either surgical option.
Discussion The comparisons between the three groups revealed interesting findings. The preferences of the patients and gastroenterologists were not too dissimilar and they would rather choose to have escalating medical therapy than any operation in an acute setting. If surgery was
unavoidable they were more willing to choose J-pouch surgery to avoid a permanent stoma. Their preferences were consistently different from those of colorectal surgeons in all the acute scenarios and the differences were statistically significant. Our findings surprisingly contradicted an earlier study that evaluated patients and clinicians managing Crohn’s disease [10]. In that study, more patients with Crohn’s disease had similar preferences to colorectal surgeons and were more receptive to an operation than escalating medical therapy. For those scenarios, the differences between the patients and gastroenterologists were statistically different. The differences we see in the results for patients with Crohn’s disease and those with UC are very enlightening. We believe that in the setting of Crohn’s disease, a limited resection compared with an extensive resection is often performed and a stoma is often not necessary – hence the similarities between the surgeons and the patients. In UC, however, any operation requires the resection of the entire colon and rectum. It is therefore not unexpected that patients with Crohn’s disease also choose to avoid extensive surgery when possible, especially if it entails a stoma [10]. The fear of having a stoma is genuine, as the complications faced by patients with a stoma are not negligible [23]. The role of stoma therapists in educating and supporting patients through the initial phase of adapting the stoma is critical to ensure that the QOL of these patients is not compromised [23]. Our finding also reflects that the gastroenterologists were aware that a majority of patients with Crohn’s disease and a minority of patients with UC will need resectional surgery in their lifetime [24,25].
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Table 4 Preference measures and comparison between the various groups in scenarios 4 & 5 (Preferences in long-standing ulcerative colitis (UC) to have yearly colonoscopy surveillance to avoid (4) J pouch surgery or (5) permanent stoma surgery). Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(4) J pouch surgery UC medical patients (26) Colorectal surgeons (91) Gastroenterologists (74)
0.84 0.84 0.87
0.15 (0.09–0.25) 0.10 (0.05–0.25) 0.15 (0.10–0.30)
0.77 0.66 0.72
0.10 (0.02–0.17) 0.05 (0–0.13) 0.08 (0–0.15)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.97 0.63 0.61
0.82 0.37 0.24
0.17 0.58 0.37
0.10 0.34 0.49
Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(5) Permanent stoma surgery UC medical patients (55) Colorectal surgeons (91) Gastroenterologists (74)
0.87 0.89 0.87
0.18 (0.10–0.30) 0.20 (0.10–0.40) 0.20 (0.10–0.43)
0.80 0.73 0.77
0.10 (0.04–0.20) 0.05 (0–0.20) 0.10 (0.03–0.20)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
Surgical UC patients vs colorectal surgeons Surgical UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.98 0.90 0.91
0.73 0.42 0.64
0.22 0.69 0.38
0.30 0.90 0.36
WTG, proportion willing to gamble; WTT, proportion willing to trade; PMPg, prospective measure of preference gamble; PMPg, prospective measure of preference trade; IQR, interquartile range. *P-value for v2 test. **P-value for Wilcoxon rank sum test.
This study highlights the deficiency of comparing outcomes of surgery using postoperative QOL measures alone truly to understand patient preferences in decisional conflicts [22]. Several studies have argued for the excellent and comparable QOL for permanent stomas compared with pelvic pouches in UC or colo-anal anastomoses in low rectal cancers [8,19,24]. Postoperative QOL does not seem to relate strongly to prospective patient choice, and probably reflects expectations of informed consent rather than true choice in decisional conflicts [22]. This paper has suggested that this is the case in decision-making in patients with UC. In the setting of a prophylactic proctocolectomy vs screening in long-standing UC, most patients and doctors were willing to gamble or trade life expectancy to avoid prophylactic colectomy. These scenarios had the highest concordance of preferences of all scenarios, reflecting that patients and doctors did not accept that a 40% lifetime risk of colorectal cancer was worth prophylactic surgery, either with or without permanent stoma. Our findings have significant implications regarding the impact that clinicians’ own preferences have on actual treatment pathways and outcomes. The actual treatment that is received by the patient may be almost
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always dependent on the type of specialist and also on the individual doctor who reviews the patient. We hope that our study raises awareness among all practising gastroenterologists and colorectal surgeons on the potentially significant disparity between the preferences of patients and the treating clinician. It may be prudent to involve other specialists early in the treatment of these patients to ensure that certain details are not overlooked and a balanced multidisciplinary decision is made. Patient consultation with stoma therapists as well as other postoperative UC patients is practised in our unit to allow patients to obtain a broader understanding of the surgical considerations and decision-making process. One of the limitations of this study is the lower patient accrual which was due to the ethical constraints and a new Privacy Act. Patients had to ‘opt on’ to the study, which resulted in a lower response rate than anticipated and little protection against selection bias. This study was conducted at a tertiary referral centre from patients treated by one gastroenterologist (W.S.) and one surgeon (M.J.S.). Approximately half of the patients had undergone definitive surgery and half were being treated with med-
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Table 5 Comparison of willingness to trade to avoid surgery involving pouch (no stoma) or permanent stoma (scenarios 1 and 2).
To avoid stoma operation
v2 and P-value P-value of McNemar test
To avoid pouch operation
Number willing to trade
Number not trading
Total
Ulcerative colitis patients
Trade Not trade Total
29 (53) 10 (18) 39 (71)
1 (2) 15 (27) 16 (29)
30 (55) 25 (45) 55 (100)
6.31 (0.012)
Surgeons
Trade Not trade Total
31 (34) 16 (18) 47 (52)
5 (5) 39 (43) 44 (48)
36 (39) 55 (61) 91 (100)
4.89 (0.027)
Gastroenterologists
Trade Not trade Total
41 (57) 9 (13) 50 (70)
2 (3) 21 (27) 23 (30)
30 (60) 43 (40) 73 (100)
3.41 (0.065)
ical therapy. The heterogeneous nature of the patient group is a significant drawback because the medical patients were being asked to deliberate about future treatment choices while the surgical patients may have already made or considered such options during treatment of their conditions. The current clinical status of surgical patients (pouch with or without stoma vs end ileostomy vs no stoma; single vs repeated admissions) could also potentially affect their response to the questionnaire. A subgroup analysis was actually performed by the authors comparing the responses of the medical and surgical patient groups, which interestingly found no statistical difference in all the scenarios. Moreover, as the number of patients in each group was very small, the authors decided to group the two sets of patients together as one group to facilitate comparison with the gastroenterologists and colorectal surgeons. There is also debate over which preference tool is most appropriate, as all measures of preference are cognitively complex; some respondents are unwilling to trade or gamble life expectancy; some make illogical or contradictory decisions and the gamble and trade tools can generate different utilities for the same health state [26–28]. The authors also acknowledge that the various scenarios for the purpose of this project oversimplified the different treatment options that a patient with steroid-refractory acute UC would face. This has been done to facilitate the conduct of the questionnaire and the subsequent data analysis. This study has demonstrated a wide range of preferences for treatment scenarios in UC, particularly in the acute setting. Here patients had markedly different preferences from surgeons but not from gastroenterologists. The preferences of colorectal surgeons were frequently different from those of gastroenterologists. Prospective preferences favour a pelvic pouch much more than a per-
manent stoma in all groups. Clinicians treating UC should be aware of the large variation in preferences for treatment and should seek to understand patient preferences when at critical junctures in determining therapy in UC and ensure that there has been adequate multidisciplinary consultation and informed choice.
Acknowledgements We would like to thank all patients and clinicians who took part in this study.
Author contributions Christopher M. Byrne, Ker-Kan Tan: contributed to the data analysis, interpretation, drafting and revision of manuscript, and final approval of the manuscript. Jane M Young, Warwick Selby, Michael J. Solomon: contributed to the study design, data collection and analysis, revision and final approval of the manuscript.
Financial disclosure Dr Byrne was supported by the Notaras Fellowship from the University of Sydney, the Section of Colon and Rectal Surgery of the Royal Australasian College of Surgeons and the Training Board of the Colorectal Surgical Society of Australasia for the conduct of this study.
References 1 Friedman S, Blumberg RS. Inflammatory bowel disease. In: Harrison’s Principles of Internal Medicine, 15th edition (eds Braunwald E, Fauci AS, Kasper DL, Hauser SL, Longo DL, Jameson JL). New York: McGraw Hill, 2001. pp. 1679–92.
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291
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2 Nicholls RJ, Tekkis PP. Ulcerative Colitis. In: Colorectal Surgery: Companion to Specialist Colorectal Surgical Practice. 3rd edition (ed. Phillips RK). London: W.B. Saunders Company, 2005. pp. 129–62. 3 Blomqvist P, Feltelius N, Lofberg R, Ekbom A. A 10-year survey of inflammatory bowel diseases-drug therapy, costs and adverse reactions. Aliment Pharmacol Ther 2001; 15: 475–81. 4 Stuck A, Minder C, Frey F. Risk of infectious complications in patients taking glucocorticosteroids. Rev Infect Dis 1989; 11: 954–63. 5 Warman J, Korelitz B, Fleisher M, Janardhanam R. Cumulative experience with short- and long-term toxicity to 6-mercaptopurine in the treatment of Crohn’s disease and ulcerative colitis. J Clin Gastroenterol 2003; 37: 220–5. 6 Yang Y, Lichtenstein G. Corticosteroids in Crohn’s disease. Am J Gastroenterol 2002; 97: 803–23. 7 Solomon MJ, Schnitzler M. Cancer and inflammatory bowel disease: bias, epidemiology, surveillance, and treatment. World J Surg 1998; 22: 352–8. 8 Camilleri-Brennan J, Munro A, Steele RJ. Does an ileoanal pouch offer a better quality of life than a permanent ileostomy for patients with ulcerative colitis? J Gastrointest Surg 2003; 7: 814–9. 9 Kohler L, Troidl H. The ileoanal pouch: a risk-benefit analysis. Br J Surg 1995; 82: 443–7. 10 Byrne CM, Solomon MJ, Young JM, Selby W, Harrison JD. Patient preferences between surgical and medical treatment in Crohn’s disease. Dis Colon Rectum 2007; 50: 586–97. 11 Solomon MJ, Pager CK, Keshava A et al. What do patients want? Patient preferences and surrogate decision making in the treatment of colorectal cancer. Dis Colon Rectum 2003; 46: 1351–7. 12 Braddock CH, Edwards KA, Hasenberg NM, Laidley TL, Levinson W. Informed decision making in outpatient practice: time to get back to basics. JAMA 1999; 282: 2313–20. 13 Protheroe J, Fahey T, Montgomery AA, Peters TJ. The impact of patients’ preferences on the treatment of atrial fibrillation: observational study of patient based decision analysis. BMJ 2000; 320: 1380–4. 14 Bowling A, Ebrahim S. Measuring patients’ preferences for treatment and perceptions of risk. Qual Health Care 2001; 10(Suppl): i2–8. 15 Butow PN, Maclean M, Dunn SM. The dynamics of change: cancer patients’ preferences for information, involvement and support. Ann Oncol 1997; 8: 857–63.
292
16 Stiggelbout AM, Kiebert GM. A role for the sick role. Patient preferences regarding information and participation in clinical decision-making. CMAJ 1997; 157: 383–9. 17 Ramfelt E, Langius A, Bjorvell H, Nordstrom G. Treatment decision-making and its relation to the sense of coherence and the meaning of the disease in a group of patients with colorectal cancer. Eur J Cancer Care (Engl) 2000; 9: 158–66. 18 Henman MJ, Butow PN, Brown RF. Lay contructions of decision-making in cancer. Psychooncology 2002; 11: 295– 306. 19 Harrison JD, Solomon MJ, Young JM et al. Patient and physician preferences for surgical and adjuvant treatment options for rectal cancer. Arch Surg 2008; 143: 389–94. 20 Suarez-Almazor ME, Conner-Spady B, Kendall CJ, Russell AS, Skeith K. Lack of congruence in the ratings of patients’ health status by patients and their physicians. Med Decis Making 2001; 21: 113–21. 21 Bruera E, Willey JS, Palmer JL, Rosales M. Treatment decisions for breast carcinoma – patient preferences and physician perceptions. Cancer 2002; 94: 2076–80. 22 Young JM, Solomon MJ, Harrison JD, Salkeld G, Butow P. Measuring patient preference and surgeon choice. Surgery 2008; 143: 582–90. 23 McLeod RS, Baxter NN. Quality of life of patients with inflammatory bowel disease after surgery. World J Surg 1998; 22: 375–81. 24 Targownik LE, Singh H, Nugent Z, Bernstein CN. The epidemiology of colectomy in ulcerative colitis: results from a population-based cohort. Am J Gastroenterol 2012; 107: 1228–35. 25 Peyrin-Biroulet L, Loftus EV Jr, Colombel JF, Sandborn WJ. The natural history of adult Crohn’s disease in population-based cohorts. Am J Gastroenterol 2010; 105: 289– 9726. 26 Stiggelbout AM, de Haes JC. Patient preference for cancer therapy: an overview of measurement approaches. J Clin Oncol 2001; 19: 220–30. 27 Llewellyn-Thomas HA. Investigating patients’ preferences for different treatment options. Can J Nurs Res 1997; 29: 45–64. 28 King JT, Tsevat J, Roberts MS. Positive association between current health and health values for hypothetical disease states. Med Decis Making 2004; 24: 367–78.
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Original article
Patient and clinician preferences for surgical and medical treatment options in ulcerative colitis C. M. Byrne*†, K.-K. Tan*†, J. M. Young*, W. Selby‡ and M. J. Solomon*† *Surgical Outcomes Research Centre (SOuRCe), Sydney Local Health District and University of Sydney, Sydney, Australia, †Department of Colorectal Surgery, Royal Prince Alfred Hospital, Sydney, Australia and ‡AW Morrow Department of Gastroenterology and Liver Centre, Royal Prince Alfred Hospital, Sydney, Australia Received 4 September 2013; accepted 6 November 2013; Accepted Article online 21 December 2013
Abstract Aim When treating patients with refractory ulcerative colitis (UC), the choice between escalating medical management or surgery can be difficult. The aim of this study was to quantify the preferences of patients and clinicians for the treatment options in UC.
more prepared to gamble or trade to avoid any surgery than were colorectal surgeons. All groups were aligned in their decision to undergo yearly colonoscopy surveillance rather than to undergo definitive surgery that would result in a stoma.
Method Ulcerative colitis outpatients were interviewed to measure their preferences for five scenarios examining the management of acute and chronic UC, using a prospective measure of preference method that generates two utility scores: willingness and amount of expected life to trade or gamble. A self-administered questionnaire was mailed to Australian and New Zealand colorectal surgeons and gastroenterologists.
Conclusion Patient preferences for the treatment of UC were more aligned to those of gastroenterologists than those of colorectal surgeons. Despite postoperative studies revealing an equal quality of life for pouch and stoma patients, this study confirmed that a pouch is the preferred surgical option.
Results Fifty-five patients (26 medical and 29 surgical), 91 surgeons and 78 gastroenterologists were surveyed. In the acute setting, 89% of patients, 69% of gastroenterologists and 55% of surgeons were willing to trade part of their life expectancy to avoid a permanent stoma, while for chronic disease 71% of patients were prepared to trade to avoid an operation with a permanent stoma compared with 55% for an operation with a pouch (P = 0.01). Both patients and gastroenterologists were
Introduction Two therapeutic pathways exist for the treatment of patients with refractory ulcerative colitis (UC). Medical management using anti-inflammatory and/or immunosuppressant medications is frequently effective for the majority of patients with UC, but in up to 30% of these patients, surgery is still necessary for indications such as failed medical therapy and neoplasia [1,2]. Whilst prolonged use of corticosteroid therapy is associated with Correspondence to: Michael J Solomon, Department of Colorectal Surgery, Surgical Outcomes Research Centre (SOuRCe), Royal Prince Alfred Hospital, Missenden Road, Camperdown, NSW 2050, Australia. E-mail: [email protected]
Keywords Preferences, surgery, ulcerative colitis, decision-making What does this paper add to the literature? Patient preferences for the treatment of UC were more aligned to those of gastroenterologists than those of colorectal surgeons. Despite postoperative studies revealing an equal quality of life for pouch and stoma patients, this study has confirmed that a pouch is the preferred surgical option.
numerous health problems, long-standing colitis is associated with an increased risk of bowel cancer and other associated complications [3–7]. If the aforementioned difficulties are encountered, surgery is then necessary. In an elective setting, two surgical options exist. Total proctocolectomy with end ileostomy removes all colonic mucosa, albeit with a permanent stoma. Although a restorative ileo-anal pouch operation avoids the need for a permanent stoma, it has its own set of short- and long-term difficulties. There is also a higher rate of subsequent surgery for pouch-related complications [8,9]. Patients with UC and their clinicians are thus confronted by numerous potentially conflicting factors when choosing between surgical or medical therapy.
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Before choosing any therapy, the preference of the patient is an important consideration. This is especially pertinent when the intervention has the potential to influence their quality of life (QOL) considerably [10]. Despite the notion that patient preferences should always be taken into account as part of informed decision-making during routine surgical practice involving a choice between therapies, the reality is somewhat different [11]. In a study of 1032 tape-recorded patient consultations by 60 primary care physicians and 60 surgeons, it was found that of the 3552 decisions made less than 20% actually included any assessment of patient preference [12]. It is not surprising that studies have suggested that more patients would prefer an increased role in the decision-making process [13,14]. Understanding patient preferences is important for clinicians when up to 40% of medical decisions are made by doctors for their patients [13,15–18]. There is also mounting evidence that surgeons and physicians have different preferences for treatment options from their patients and from each other [10,11,19–21]. To our knowledge, there have been no studies that have directly examined the decision-making process in UC when choosing between medical and surgical treatment options, and in particular between the choice of surgery resulting in a permanent stoma vs restorative procedures. This study was conducted with the particular aim of determining patient preferences for surgical intervention in UC prospectively rather than the postoperative quality of life measures in the surgical cohort alone. Secondly, we wanted to quantify the worth of living with a permanent stoma as opposed to a pouch, and finally to compare patient preferences with those of surgeons and gastroenterologists.
Method Patient recruitment
A consecutive sample of UC patients was selected retrospectively from clinical databases of their treating specialist. An information sheet, consent form and questionnaire were sent. The ethics review board stipulated that the patients had to ‘opt on’ by returning a signed consent form and no direct approach could be made by the researchers. One reminder letter was sent. Medical patients were defined as those who had never undergone colectomy. Script development and hypothetical scenarios
Standardized verbal scripts were developed to explain three treatment scenarios in acute UC and two in
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chronic UC. A prelude to the first set of scenarios described a patient with a 2-year history of active, corticosteroid-dependent UC affecting over half the colon who was now suffering an acute, severe, exacerbation that had not responded to high-dose corticosteroid therapy. Three treatment choices were presented: 1 Standard escalating medical therapy vs total proctocolectomy with an ileal J pouch; 2 standard escalating medical therapy vs total proctocolectomy with an end ileostomy; 3 total proctocolectomy with an ileal J pouch vs total proctocolectomy with an end ileostomy. A prelude to the remaining scenarios described an asymptomatic patient with a 20-year history of UC whose recent colonoscopy demonstrated ‘burnt-out’ inactive disease affecting three-quarters of the colon with no dysplasia on biopsies. Two treatment choices presented were: 1 Yearly colonoscopic surveillance vs total proctocolectomy with an ileal J pouch; 2 yearly colonoscopic surveillance vs total proctocolectomy with an end ileostomy. Prospective measure of preference
A clinician (C.M.B.) conducted telephone interviews using the standardized scripts explaining each treatment scenario in detail, including the benefits, risks and the likely long-term outcome. The major features of the two treatment options were summarized as bullet points in a visual aid. For each treatment choice, one option was presented as the standard treatment and the other as the alternative treatment. Once the patient had reached a level of understanding sufficient to make an informed treatment choice, patient preferences were elicited [10,11,19,22]. The previously described prospective measure of preference (PMP) method was utilized to elicit preferences [10,11,22]. This is a prospective modification of the time trade-off and standard gamble tools that have been previously described and validated in colorectal settings [10,11,19,22]. The PMP method produces two measures of preference. The first is a dichotomous variable (yes/no) of either willingness to gamble (WTG) or willingness to trade (WTT) life expectancy, and this is summarized as the proportion of study participants who would be willing to either gamble or trade (0–1.00). The second measure, either a prospective measure of preference standard gamble (PMPg) or a prospective measure of preference time trade-off (PMPt), is expressed as the group average utility of life expectancy given away ranging from 0 to 1.
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To determine WTG and the PMPg patients were asked to quantify the amount of their remaining life expectancy they would gamble (or risk) to avoid an alternative treatment option rather than the current option. To determine WTT and the PMPt patients were asked how much life expectancy they would trade (or give up) to avoid the alternative treatment option. PMPg involves an uncertain outcome (that is, a potential risk of losing life expectancy) whereas PMPt involves a certain, known outcome (that is, a definite amount of life given up). The greater the PMPg or PMPt the more averse patients are to the alternative treatment. PMP measures of more than 0.10 are indicative of strong preference. These measures have demonstrated strong test–retest correlations (R = 0.72–0.96) [19]. Specialist doctor recruitment
The patient questionnaire was converted to a selfadministered format to be mailed to specialist doctors. The questionnaire sent to clinicians elicited identical information to the patient questionnaire. Clinicians were asked to answer as if they were a patient with UC (surrogate patient). All 136 members and training fellows of the Colorectal Surgical Society of Australia and New Zealand (colorectal surgeons) and a random sample of 300 members of the Gastroenterological Society of Australia (gastroenterologists) were invited to participate.
Preferences in ulcerative colitis
Demographic and clinical details
The characteristics of the patient participants are shown in Table 1. Their mean age was 47 years and the mean duration of colitis was over 13 years. Almost 85% had used corticosteroids previously. Two-thirds had taken other immunosuppressant medication, while 40% reported complications of medical therapy. Over half had undergone abdominal surgery for UC and almost all of these had experienced an ileostomy, either temporary or permanent. Just over half of the patients had no current symptoms from their colitis. The mean duration of interview was 37 min. Escalating medical therapy vs total proctocolectomy with a J pouch
The first scenario explored the choice between escalating medical therapy with increasingly potent immunosuppressant therapy vs a restorative proctocolectomy with J pouch surgery. As shown in Table 2, both patients and gastroenterologists were aligned in their opinion and were more willing than colorectal surgeons to gamble or trade their life expectancy for escalating medical therapy rather than an operation. These differences were statistically significant. There was no statistical difference between the responses of UC patients and gastroenterologists.
Table 1 Demographic and past medical details of patients with ulcerative colitis.
Statistical analysis
Statistical analysis was performed using SPSS Version 13, blinded to participants’ group status. For each scenario WTG and WTT were compared between groups using the chi-square test, whereas the PMPg and PMPt were compared using the Wilcoxon rank sum test. Within groups PMPg and PMPt were compared between scenarios using the McNemar test or the Wilcoxon signed rank test as appropriate.
Results Response rate
A total of 55 (34%) out of 162 UC patients responded to a recruitment letter from their specialist (29 of 74 surgical patients and 26 of 88 medical patients). Ninety-one completed questionnaires were received from 127 (72%) eligible surgeons, while 78 (27%) completed questionnaires from 272 gastroenterologists were returned.
Characteristic Number Mean age (years) Gender (M/F) Mean disease duration (years) Previous corticosteroid use Other immunosuppressant use Complications of medications Emergency hospital admission Discussion with surgeon Previous abdominal surgery for ulcerative colitis Previous stoma Complications of surgery Current symptoms None Mild Moderate Marked Severe
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Number of patients (%) 55 47.7 26/29 13.3 47 33 20 32 35 30
(85) (60) (36) (58) (64) (56)
29 (54) 16 (29) 29 12 7 2 4
(54) (22) (13) (4) (7)
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Table 2 Preference measures and comparison between the various groups in scenarios 1 and 2 (preferences in severe acute-onchronic ulcerative colitis (UC) to have escalating medical therapy to avoid (1) J pouch surgery and (2) permanent stoma surgery).
Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(1) J pouch surgery UC patients (55) Colorectal surgeons (91) Gastroenterologists (74)
0.64 0.49 0.80
0.16 (0–0.20) 0.00 (0–0.10) 0.10 (0.05–0.20)
0.54 0.41 0.59
0.05 (0–0.13) 0.00 (0–0.05) 0.05 (0–0.10)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
0.08 0.59 0.011
0.01 0.38 0.002
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.10 0.038 < 0.001
0.004 0.73 < 0.001
Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(2) Permanent stoma surgery UC patients (55) Colorectal surgeons (91) Gastroenterologists (74)
0.75 0.68 0.85
0.15 (0–0.29) 0.10 (0–0.15) 0.13 (0.05–0.25)
0.71 0.51 0.69
0.10 (0–0.20) 0.01 (0–0.10) 0.05 (0–0.15)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.38 0.14 0.011
0.006 0.73 0.019
0.015 0.69 0.027
0.003 0.38 0.028
WTG, proportion willing to gamble; WTT, proportion willing to trade; PMPg, prospective measure of preference gamble; PMPg, prospective measure of preference trade; IQR, interquartile range. Bold values indicate statistically significant results. *P-value for v2 test. **P-value for Wilcoxon rank sum test.
Escalating medical therapy vs total proctocolectomy with a permanent stoma
When the option changed between escalating medical treatment vs a proctocolectomy with a permanent stoma, there was again a higher proportion of patients across all the groups willing to gamble to avoid this operation, including gastroenterologists (85% from 80%), patients (75% from 64%) and surgeons (68% from 49%) (Table 2). But as in the earlier option, both the gastroenterologists and patients were consistently similar and more willing than colorectal surgeons to gamble or trade. Proctocolectomy with a J pouch vs proctocolectomy with a permanent stoma
In the next scenario, large proportions of all groups were willing to gamble to undergo J pouch surgery to avoid a permanent stoma (Table 3). PMPg was 0.15 for both patients and gastroenterologists and 0.10 for sur-
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geons. Eighty-nine per cent of patients, 69% of gastroenterologists and 55% of surgeons were willing to trade to avoid the operation with the permanent stoma. The PMPt was 0.10 for patients, 0.07 for gastroenterologists and 0.05 for surgeons. More patients were willing to gamble or trade than both colorectal surgeons and gastroenterologists to achieve this outcome. This difference was statistically significant. Screening for cancer vs prophylactic total proctocolectomy
The last two scenarios were in a more elective setting whereby a patient with chronic UC has to choose between having cancer screening via yearly colonoscopy or surgery to avoid a 15% current risk of cancer that would increase by 1% per year. Higher numbers of participants in all three groups were willing to gamble or trade more life expectancy to avoid undergoing a restorative proctocolectomy than in previous scenarios
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Table 3 Preference measures and comparison between the various groups in scenario 3 (preferences in severe acute-on-chronic ulcerative colitis (UC) to have J pouch surgery to avoid permanent stoma).
Group (n)
WTG
Median PMPg (IQR)
WTT
UC patients (26) Colorectal surgeons (91) Gastroenterologists (74)
0.93 0.75 0.84
0.15 (0.10–0.26) 0.10 (0.02–0.20) 0.15 (0.05–0.30)
0.89 0.55 0.69
Comparison groups
WTG*
PMPg**
WTT*
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.009 0.14 0.18
0.004 0.61 0.028
< 0.001 0.006 0.08
Median PMPt (IQR) 0.10 (0.05–0.20) 0.05 (0–0.10) 0.07 (0–0.18) PMPt** < 0.001 0.019 0.06
WTG, proportion willing to gamble; WTT, proportion willing to trade; PMPg, prospective measure of preference gamble; PMPg, prospective measure of preference trade; IQR, interquartile range. Bold values indicate statistically significant results. *P-value for v2 test. **P-value for Wilcoxon rank sum test.
(Table 4). Because of the similar high scores between all three groups there were no significant differences observed. A similar response was observed when the choice was changed to involve an operation resulting in a permanent stoma. This scenario had the highest PMPg of all scenarios studied (between 0.18 and 0.20). Comparison between a permanent stoma and pouch surgery
The proportion of participants who were willing to gamble or trade in scenarios 1 and 2 was compared by the McNemar test for paired proportions (Table 5). Seventy-one per cent of patients were prepared to trade to avoid the operation with the permanent stoma compared with 55% for the operation with the pouch (v2 = 6.31, 1 df, P = 0.01). A significantly greater number of surgeons traded to avoid a permanent stoma (52%) than to avoid pouch surgery (39%; v2 = 4.89, 1 df, P = 0.03). Ten per cent of gastroenterologists changed their preference, which was not significantly different and reflected the high proportion of gastroenterologists who preferred to avoid either surgical option.
Discussion The comparisons between the three groups revealed interesting findings. The preferences of the patients and gastroenterologists were not too dissimilar and they would rather choose to have escalating medical therapy than any operation in an acute setting. If surgery was
unavoidable they were more willing to choose J-pouch surgery to avoid a permanent stoma. Their preferences were consistently different from those of colorectal surgeons in all the acute scenarios and the differences were statistically significant. Our findings surprisingly contradicted an earlier study that evaluated patients and clinicians managing Crohn’s disease [10]. In that study, more patients with Crohn’s disease had similar preferences to colorectal surgeons and were more receptive to an operation than escalating medical therapy. For those scenarios, the differences between the patients and gastroenterologists were statistically different. The differences we see in the results for patients with Crohn’s disease and those with UC are very enlightening. We believe that in the setting of Crohn’s disease, a limited resection compared with an extensive resection is often performed and a stoma is often not necessary – hence the similarities between the surgeons and the patients. In UC, however, any operation requires the resection of the entire colon and rectum. It is therefore not unexpected that patients with Crohn’s disease also choose to avoid extensive surgery when possible, especially if it entails a stoma [10]. The fear of having a stoma is genuine, as the complications faced by patients with a stoma are not negligible [23]. The role of stoma therapists in educating and supporting patients through the initial phase of adapting the stoma is critical to ensure that the QOL of these patients is not compromised [23]. Our finding also reflects that the gastroenterologists were aware that a majority of patients with Crohn’s disease and a minority of patients with UC will need resectional surgery in their lifetime [24,25].
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Table 4 Preference measures and comparison between the various groups in scenarios 4 & 5 (Preferences in long-standing ulcerative colitis (UC) to have yearly colonoscopy surveillance to avoid (4) J pouch surgery or (5) permanent stoma surgery). Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(4) J pouch surgery UC medical patients (26) Colorectal surgeons (91) Gastroenterologists (74)
0.84 0.84 0.87
0.15 (0.09–0.25) 0.10 (0.05–0.25) 0.15 (0.10–0.30)
0.77 0.66 0.72
0.10 (0.02–0.17) 0.05 (0–0.13) 0.08 (0–0.15)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
UC patients vs colorectal surgeons UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.97 0.63 0.61
0.82 0.37 0.24
0.17 0.58 0.37
0.10 0.34 0.49
Group (n)
WTG
Median PMPg (IQR)
WTT
Median PMPt (IQR)
(5) Permanent stoma surgery UC medical patients (55) Colorectal surgeons (91) Gastroenterologists (74)
0.87 0.89 0.87
0.18 (0.10–0.30) 0.20 (0.10–0.40) 0.20 (0.10–0.43)
0.80 0.73 0.77
0.10 (0.04–0.20) 0.05 (0–0.20) 0.10 (0.03–0.20)
Comparison groups
WTG*
PMPg**
WTT*
PMPt**
Surgical UC patients vs colorectal surgeons Surgical UC patients vs gastroenterologists Colorectal surgeons vs gastroenterologists
0.98 0.90 0.91
0.73 0.42 0.64
0.22 0.69 0.38
0.30 0.90 0.36
WTG, proportion willing to gamble; WTT, proportion willing to trade; PMPg, prospective measure of preference gamble; PMPg, prospective measure of preference trade; IQR, interquartile range. *P-value for v2 test. **P-value for Wilcoxon rank sum test.
This study highlights the deficiency of comparing outcomes of surgery using postoperative QOL measures alone truly to understand patient preferences in decisional conflicts [22]. Several studies have argued for the excellent and comparable QOL for permanent stomas compared with pelvic pouches in UC or colo-anal anastomoses in low rectal cancers [8,19,24]. Postoperative QOL does not seem to relate strongly to prospective patient choice, and probably reflects expectations of informed consent rather than true choice in decisional conflicts [22]. This paper has suggested that this is the case in decision-making in patients with UC. In the setting of a prophylactic proctocolectomy vs screening in long-standing UC, most patients and doctors were willing to gamble or trade life expectancy to avoid prophylactic colectomy. These scenarios had the highest concordance of preferences of all scenarios, reflecting that patients and doctors did not accept that a 40% lifetime risk of colorectal cancer was worth prophylactic surgery, either with or without permanent stoma. Our findings have significant implications regarding the impact that clinicians’ own preferences have on actual treatment pathways and outcomes. The actual treatment that is received by the patient may be almost
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always dependent on the type of specialist and also on the individual doctor who reviews the patient. We hope that our study raises awareness among all practising gastroenterologists and colorectal surgeons on the potentially significant disparity between the preferences of patients and the treating clinician. It may be prudent to involve other specialists early in the treatment of these patients to ensure that certain details are not overlooked and a balanced multidisciplinary decision is made. Patient consultation with stoma therapists as well as other postoperative UC patients is practised in our unit to allow patients to obtain a broader understanding of the surgical considerations and decision-making process. One of the limitations of this study is the lower patient accrual which was due to the ethical constraints and a new Privacy Act. Patients had to ‘opt on’ to the study, which resulted in a lower response rate than anticipated and little protection against selection bias. This study was conducted at a tertiary referral centre from patients treated by one gastroenterologist (W.S.) and one surgeon (M.J.S.). Approximately half of the patients had undergone definitive surgery and half were being treated with med-
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Table 5 Comparison of willingness to trade to avoid surgery involving pouch (no stoma) or permanent stoma (scenarios 1 and 2).
To avoid stoma operation
v2 and P-value P-value of McNemar test
To avoid pouch operation
Number willing to trade
Number not trading
Total
Ulcerative colitis patients
Trade Not trade Total
29 (53) 10 (18) 39 (71)
1 (2) 15 (27) 16 (29)
30 (55) 25 (45) 55 (100)
6.31 (0.012)
Surgeons
Trade Not trade Total
31 (34) 16 (18) 47 (52)
5 (5) 39 (43) 44 (48)
36 (39) 55 (61) 91 (100)
4.89 (0.027)
Gastroenterologists
Trade Not trade Total
41 (57) 9 (13) 50 (70)
2 (3) 21 (27) 23 (30)
30 (60) 43 (40) 73 (100)
3.41 (0.065)
ical therapy. The heterogeneous nature of the patient group is a significant drawback because the medical patients were being asked to deliberate about future treatment choices while the surgical patients may have already made or considered such options during treatment of their conditions. The current clinical status of surgical patients (pouch with or without stoma vs end ileostomy vs no stoma; single vs repeated admissions) could also potentially affect their response to the questionnaire. A subgroup analysis was actually performed by the authors comparing the responses of the medical and surgical patient groups, which interestingly found no statistical difference in all the scenarios. Moreover, as the number of patients in each group was very small, the authors decided to group the two sets of patients together as one group to facilitate comparison with the gastroenterologists and colorectal surgeons. There is also debate over which preference tool is most appropriate, as all measures of preference are cognitively complex; some respondents are unwilling to trade or gamble life expectancy; some make illogical or contradictory decisions and the gamble and trade tools can generate different utilities for the same health state [26–28]. The authors also acknowledge that the various scenarios for the purpose of this project oversimplified the different treatment options that a patient with steroid-refractory acute UC would face. This has been done to facilitate the conduct of the questionnaire and the subsequent data analysis. This study has demonstrated a wide range of preferences for treatment scenarios in UC, particularly in the acute setting. Here patients had markedly different preferences from surgeons but not from gastroenterologists. The preferences of colorectal surgeons were frequently different from those of gastroenterologists. Prospective preferences favour a pelvic pouch much more than a per-
manent stoma in all groups. Clinicians treating UC should be aware of the large variation in preferences for treatment and should seek to understand patient preferences when at critical junctures in determining therapy in UC and ensure that there has been adequate multidisciplinary consultation and informed choice.
Acknowledgements We would like to thank all patients and clinicians who took part in this study.
Author contributions Christopher M. Byrne, Ker-Kan Tan: contributed to the data analysis, interpretation, drafting and revision of manuscript, and final approval of the manuscript. Jane M Young, Warwick Selby, Michael J. Solomon: contributed to the study design, data collection and analysis, revision and final approval of the manuscript.
Financial disclosure Dr Byrne was supported by the Notaras Fellowship from the University of Sydney, the Section of Colon and Rectal Surgery of the Royal Australasian College of Surgeons and the Training Board of the Colorectal Surgical Society of Australasia for the conduct of this study.
References 1 Friedman S, Blumberg RS. Inflammatory bowel disease. In: Harrison’s Principles of Internal Medicine, 15th edition (eds Braunwald E, Fauci AS, Kasper DL, Hauser SL, Longo DL, Jameson JL). New York: McGraw Hill, 2001. pp. 1679–92.
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2 Nicholls RJ, Tekkis PP. Ulcerative Colitis. In: Colorectal Surgery: Companion to Specialist Colorectal Surgical Practice. 3rd edition (ed. Phillips RK). London: W.B. Saunders Company, 2005. pp. 129–62. 3 Blomqvist P, Feltelius N, Lofberg R, Ekbom A. A 10-year survey of inflammatory bowel diseases-drug therapy, costs and adverse reactions. Aliment Pharmacol Ther 2001; 15: 475–81. 4 Stuck A, Minder C, Frey F. Risk of infectious complications in patients taking glucocorticosteroids. Rev Infect Dis 1989; 11: 954–63. 5 Warman J, Korelitz B, Fleisher M, Janardhanam R. Cumulative experience with short- and long-term toxicity to 6-mercaptopurine in the treatment of Crohn’s disease and ulcerative colitis. J Clin Gastroenterol 2003; 37: 220–5. 6 Yang Y, Lichtenstein G. Corticosteroids in Crohn’s disease. Am J Gastroenterol 2002; 97: 803–23. 7 Solomon MJ, Schnitzler M. Cancer and inflammatory bowel disease: bias, epidemiology, surveillance, and treatment. World J Surg 1998; 22: 352–8. 8 Camilleri-Brennan J, Munro A, Steele RJ. Does an ileoanal pouch offer a better quality of life than a permanent ileostomy for patients with ulcerative colitis? J Gastrointest Surg 2003; 7: 814–9. 9 Kohler L, Troidl H. The ileoanal pouch: a risk-benefit analysis. Br J Surg 1995; 82: 443–7. 10 Byrne CM, Solomon MJ, Young JM, Selby W, Harrison JD. Patient preferences between surgical and medical treatment in Crohn’s disease. Dis Colon Rectum 2007; 50: 586–97. 11 Solomon MJ, Pager CK, Keshava A et al. What do patients want? Patient preferences and surrogate decision making in the treatment of colorectal cancer. Dis Colon Rectum 2003; 46: 1351–7. 12 Braddock CH, Edwards KA, Hasenberg NM, Laidley TL, Levinson W. Informed decision making in outpatient practice: time to get back to basics. JAMA 1999; 282: 2313–20. 13 Protheroe J, Fahey T, Montgomery AA, Peters TJ. The impact of patients’ preferences on the treatment of atrial fibrillation: observational study of patient based decision analysis. BMJ 2000; 320: 1380–4. 14 Bowling A, Ebrahim S. Measuring patients’ preferences for treatment and perceptions of risk. Qual Health Care 2001; 10(Suppl): i2–8. 15 Butow PN, Maclean M, Dunn SM. The dynamics of change: cancer patients’ preferences for information, involvement and support. Ann Oncol 1997; 8: 857–63.
292
16 Stiggelbout AM, Kiebert GM. A role for the sick role. Patient preferences regarding information and participation in clinical decision-making. CMAJ 1997; 157: 383–9. 17 Ramfelt E, Langius A, Bjorvell H, Nordstrom G. Treatment decision-making and its relation to the sense of coherence and the meaning of the disease in a group of patients with colorectal cancer. Eur J Cancer Care (Engl) 2000; 9: 158–66. 18 Henman MJ, Butow PN, Brown RF. Lay contructions of decision-making in cancer. Psychooncology 2002; 11: 295– 306. 19 Harrison JD, Solomon MJ, Young JM et al. Patient and physician preferences for surgical and adjuvant treatment options for rectal cancer. Arch Surg 2008; 143: 389–94. 20 Suarez-Almazor ME, Conner-Spady B, Kendall CJ, Russell AS, Skeith K. Lack of congruence in the ratings of patients’ health status by patients and their physicians. Med Decis Making 2001; 21: 113–21. 21 Bruera E, Willey JS, Palmer JL, Rosales M. Treatment decisions for breast carcinoma – patient preferences and physician perceptions. Cancer 2002; 94: 2076–80. 22 Young JM, Solomon MJ, Harrison JD, Salkeld G, Butow P. Measuring patient preference and surgeon choice. Surgery 2008; 143: 582–90. 23 McLeod RS, Baxter NN. Quality of life of patients with inflammatory bowel disease after surgery. World J Surg 1998; 22: 375–81. 24 Targownik LE, Singh H, Nugent Z, Bernstein CN. The epidemiology of colectomy in ulcerative colitis: results from a population-based cohort. Am J Gastroenterol 2012; 107: 1228–35. 25 Peyrin-Biroulet L, Loftus EV Jr, Colombel JF, Sandborn WJ. The natural history of adult Crohn’s disease in population-based cohorts. Am J Gastroenterol 2010; 105: 289– 9726. 26 Stiggelbout AM, de Haes JC. Patient preference for cancer therapy: an overview of measurement approaches. J Clin Oncol 2001; 19: 220–30. 27 Llewellyn-Thomas HA. Investigating patients’ preferences for different treatment options. Can J Nurs Res 1997; 29: 45–64. 28 King JT, Tsevat J, Roberts MS. Positive association between current health and health values for hypothetical disease states. Med Decis Making 2004; 24: 367–78.
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