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Biomedical subjects

Anne M Stiggelbout

Publications and source records attributed to Anne M Stiggelbout.

At least 19 recordsLinked to original sources
PubMed2006

Factors affecting patients' perceptions of choice regarding adjuvant chemotherapy for breast cancer.

INTRODUCTION: Patients' perceptions of having had freedom of choice in treatment decision-making are shown to have implications for their quality of life. It is, therefore, important to determine what factors underlie these perceptions. One factor that has been studied is whether or not patients believed that their doctor had offered choice of treatment. However, even when choice is actually offered, patients may still feel that they have no choice. Little attention has been paid to other factors that may contribute to patients' perceptions of having had no choice. Our purpose was to explore (1) whether early-stage breast cancer patients perceived to have had a choice with regard to adjuvant chemotherapy, and (2) their reasons for having perceived no choice. PATIENTS AND METHODS: The determinants of no choice that we examined were: (1) (preferred) involvement in treatment decision-making, (2) perceived clinical benefits of chemotherapy (e.g., cure), (3) perceived psychological benefits (e.g., less worries), and (4) perception of being able to cope with the side effects. All predictors, as well as patient and clinical characteristics, were entered simultaneously in a multiple logistic regression analysis and non-significant predictors were removed step-by-step, until only significant predictors (p<0.05) remained. Because determinants may differ between patients with experience of chemotherapy and those without, both treatment groups were analyzed separately. RESULTS: Four hundred and forty-six patients filled out the questionnaire (62%). Sixty-six percent of patients who had been treated with chemotherapy and 86% of inexperienced patients had perceived no treatment choice. In both treatment groups, patients who preferred a shared role or a passive role in treatment decision-making were statistically significantly more likely to have perceived no treatment choice than patients who preferred an active role. Surprisingly, beliefs about the clinical and psychological benefits of chemotherapy did not influence patients' perceptions of having had choice of treatment. CONCLUSION: If we adhere to patient autonomy and shared decision making, we may consider the perception of no treatment choice as a negative outcome of treatment decision-making. However, if this is the result of the patients' preference for less active involvement in treatment decision-making, the topic of perception of no treatment choice may be viewed in a different light.

Adult↗
PubMed2006

Reliability and validity of the Beck depression inventory in patients with Parkinson's disease.

We evaluated the validity, reliability, and potential responsiveness of the Beck Depression Inventory (BDI) in patients with Parkinson's disease (PD). In part 1 of the study, 92 patients with PD underwent a structured clinical interview for DSM major depression and based on this patients were considered depressed (PD-D) or nondepressed (PD-ND). Subsequently, patients filled in the BDI. In part 2, a postal survey consisting the BDI was performed in 185 PD patients and 112 controls. Test-retest reliability was assessed in 60 PD patients. The factor analysis revealed a cognitive-affective and a somatic factor. Cronbachs alpha for the BDI was 0.88. Mean BDI indicated significant differences (P<0.001) between the PD and control group, between the PD-ND and PD-D group, and between PD-ND and control group. In part 1, the receiver operating characteristic curves showed that the area under the curve for the total BDI was 0.88. A cutoff was calculated for the BDI (14/15) that had the highest sum of sensitivity (0.71) and specificity (0.90). In part 2, the test-retest reliability for the BDI total score was 0.89 (intraclass correlation coefficient). The smallest real difference was 3.3 for the total BDI. The BDI is a valid, reliable, and potential responsive instrument to assess the severity of depression in PD. However, an adjusted cutoff is recommended.

Aged↗
PubMed2006

Effects of magnetic stimulation in the treatment of pelvic floor dysfunction.

OBJECTIVE: To correlate, in a pilot study, the clinical results of extracorporeal magnetic innervation therapy (ExMI) of the pelvic floor muscles with functional changes in the pelvic floor musculature, urodynamics and quality of life. PATIENTS AND METHODS: In all, 74 patients (65 women and nine men) with urge incontinence, urgency/frequency, stress incontinence, mixed incontinence and defecation problems were included in a prospective study of ExMI using a 'electromagnetic chair'. All patients were treated twice weekly for 8 weeks. Digital palpation and biofeedback with a vaginal or anal probe were used for registration of the pelvic floor musculature. A urodynamic evaluation, a voiding diary, a pad-test, the King's Health Questionnaire (KHQ) and a visual analogue scale (VAS) were completed by the patient at baseline and at the end of the study. RESULTS: In the group as a whole, there were no significant differences in the voiding diary, pad-test, quality of life, VAS score, biofeedback registration and urodynamics before and after treatment. Additional stratification was applied to the total patient group, related to the pretreatment rest tone of the pelvic floor, the basal amplitude registered on electromyography, to age and to previous treatments. However, there were no significant differences in the data before and after treatment within all subgroups (stress incontinence, urge incontinence, urgency/frequency, defecation problems, overactive pelvic floor, age, previous treatments), except for the KHQ domain of 'role limitations', where there was a significant improvement in all groups. CONCLUSION: ExMI did not change pelvic floor function in the present patients. The varying outcomes of several studies on ExMI stress the need for critical studies on the effect and the mode of action of electrostimulation and magnetic stimulation. In our opinion 'the chair' is suitable to train awareness of the location of the pelvic floor. However, active pelvic floor muscle exercises remain essential.

Adult↗
PubMed2005

Impact of short-term preoperative radiotherapy on health-related quality of life and sexual functioning in primary rectal cancer: report of a multicenter randomized trial.

BACKGROUND: Few prospective studies have been performed about the impact of preoperative radiotherapy (PRT) or total mesorectal excision (TME) on health-related quality of life (HRQL) and sexual functioning in patients with resectable rectal cancer. This report describes the HRQL and sexual functioning of 990 patients who underwent TME and were randomly assigned to short-term PRT (5 x 5 Gy). PATIENTS AND METHODS: The Rotterdam Symptom Check List supplemented with additional items was used with questionnaires before treatment and at 3, 6, 12, 18, and 24 months after surgery. Patients without a recurrence the first 2 years were analyzed (n = 990). RESULTS: Few differences were found in HRQL between patients treated with or without PRT. Daily activities were significantly less for PRT patients 3 months postoperatively. Irradiated patients recovered slower from defecation problems than TME-only patients (P = .006). PRT had a negative effect on sexual functioning in males (P = .004) and females (P < .001). Irradiated males had more ejaculation disorders (P = .002), and erectile functioning deteriorated over time (P < .001). PRT had similar effects in patients who underwent a low anterior resection (LAR) versus an abdominoperineal resection (APR). Patients with an APR scored better on the physical (P = .004) and psychologic dimension (P = .007) than LAR patients, but worse on voiding (P = .0007). CONCLUSION: Short-term PRT leads to more sexual dysfunction, slower recovery of bowel function, and impaired daily activity postoperatively. However, this does not seriously affect HRQL. The comparison between LAR and APR patients demonstrates that the existence of a permanent stoma is not the only determinant of HRQL.

Female↗
PubMed2005

The impact of diagnosis and treatment of rectal cancer on paid and unpaid labor.

PURPOSE: This study was designed to describe the consequences of diagnosis and treatment of rectal cancer for paid and unpaid labor over time and to identify sociodemographic-related factors, treatment-related factors, and quality of life-related factors associated with paid and unpaid labor. METHODS: Data were assessed prospectively in two samples of patients with primary rectal cancer, participating in a multicenter clinical trial, who were randomized to receive surgery with or without 5 x 5-Gy preoperative radiotherapy. For paid labor, 292 patients who indicated paid labor before treatment filled out quality of life questionnaires, which included questions on paid labor at 3, 6, 12, 18, and 24 months after surgery. For unpaid labor, another sample of 92 patients also filled out the Health and Labor questionnaire, which included questions on unpaid labor, before treatment, and at 3 and 12 months after treatment. RESULTS: From 3 to 18 months after surgery, paid labor resumption increased from 19 to 63 percent (P < 0.001). At 24 months after surgery, paid labor resumption was 61 percent. In a multivariate analysis, age older than 55 years (P <or= 0.001), lower education level (P <or= 0.003), shorter time since surgery (P < 0.001), preoperative radiotherapy (P = 0.02), lower valuation of overall health (P < 0.01), more physical symptom distress (P < 0.001), and more limitations in daily activities (P < 0.001) were all associated with less or later resumption of paid labor. The average amount of unpaid labor increased from 17.3 hours per week at 3 months to 21 hours per week at 12 months after surgery. In a multivariate analysis, only shorter time since surgery (P = 0.03) and male gender (P < 0.001) were related to less unpaid labor. CONCLUSIONS: Diagnosis and treatment of rectal cancer affect paid and unpaid labor. The impact on paid labor is most pronounced. Multiple other sociodemographic and quality of life-related variables also were associated with paid labor. Patient information and decision making on preoperative radiotherapy should include the effects on paid labor, and interventions focused on promoting paid labor participation in patients with rectal cancer should be tailored to the specific characteristics and needs of those patients.

Aged↗
PubMed2005

Understanding VAS valuations: qualitative data on the cognitive process.

Eliciting people's value is a central pursuit in health economics. We explored approaches to valuing a health state on a visual analog scale (VAS). Additionally, we examined whether dual processing (an interaction between automatic and controlled information processing) occurred during VAS valuation. In the first experiment, respondents were probed for their approach after valuation on a VAS. After inductive generalization, we grouped the approaches: (1) 'Sort-of ' (automatic processing), (2) 'Bisection of line first', (3) 'Numerical expression', and (4) 'Dividing into smaller segments'. In the second experiment, a short questionnaire followed the VAS in which these approaches were systematically assessed, as was awareness of the approach used, intention to re-use the approach the next time (confidence), and basis of the approach. Data showed that the 'Sort-of' approach was used most often, followed by the 'Bisection-first' approach. We argue that dual processing occurs during performance on the VAS. Awareness of the approach used was lower when an intuitive approach was used. A reasoned approach had a higher correlation with confidence. Thus, awareness of approach may improve reliability. Reducing the number of health states to be valued concurrently diminishes the complexity of the task; this may enhance the validity of the VAS.

Adult↗
PubMed2005

Choice-based evaluation for the improvement of upper-extremity function compared with other impairments in tetraplegia.

OBJECTIVES: To assess preference of reconstructive treatment of upper extremities in subjects with tetraplegia compared with preference of treatment of 3 other impairments and to determine the effect of subjects' characteristics on preference of upper-extremity reconstruction. DESIGN: Survey. SETTING: Two specialized spinal cord injury centers in the Netherlands. PARTICIPANTS: A consecutive sample of 47 patients with tetraplegia in stable condition. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURE: The quality weight of 5 tetraplegic health states determined with the time trade-off technique and expressed as a single value (the "utility") on a scale between 0 (worst possible situation) and 1 (best possible situation). RESULTS: The response rate was 92%. The utility of tetraplegia +/- standard deviation was .57+/-.30. The utilities of tetraplegia without impairment in one of the following functions were .69+/-.33 for sexuality, .69+/-.33 for standing/walking, .63+/-.31 for bladder and bowel function, and .65+/-.32 for upper-extremity function. The differences between these utilities and the utility of tetraplegia were significant (P<.05). No significant differences were found between the utilities of the impairments. Improvement of a specific impairment contributed between 14% and 28% to the potential overall gain in the tetraplegic health state utility. CONCLUSIONS: The combination of impairments determines the low utility of the tetraplegic health state. No priority for improvement of any of the investigated impairments was found. This emphasizes the need for the meticulous selection of patients for treatment of specific conditions. Further research should try to determine the crucial factors in the decision-making process of patients for specific interventions.

Adult↗
PubMed2005

Self-reports of health-care utilization: diary or questionnaire?

OBJECTIVES: The feasibility and convergent validity of a cost diary and a cost questionnaire was investigated. METHODS: Data were obtained as part of a cost-utility analysis alongside a multicenter clinical trial in patients with resectable rectal cancer. A sample of 107 patients from 30 hospitals was asked to keep a weekly diary during the first 3 months after surgery, and a monthly diary from 3 to 12 months after surgery. A second sample of seventy-two patients from twenty-eight hospitals in the trial received a questionnaire at 3, 6, and 12 months after surgery, referring to the previous 3 or 6 months. Format and items of the questions were similar and included a wide range of medical and nonmedical items and costs after hospitalization for surgery. RESULTS: Small differences were found with respect to nonresponse (range, 79 to 86 percent) and missing questions (range, 1 to 6 percent between the diary and questionnaire). For most estimates of volumes of care and of costs, the diary and questionnaire did not differ significantly. Total 3-month nonhospital costs were 1,860 Euros, 1,280 Euros, and 1,050 Euros in the diary sample and 1,860 Euros, 1,090 Euros, and 840 Euros in the questionnaire sample at 3, 6, and 12 months after surgery, respectively (p = .50). However, with respect to open questions, the diary sample tended to report significantly more care. CONCLUSIONS: For the assessment of health-care utilization in economic evaluations alongside clinical trials, a cost questionnaire with structured closed questions may replace a cost diary for recall periods up to 6 months.

Colectomy↗
PubMed2004

Clinical nature and prognosis of locally recurrent rectal cancer after total mesorectal excision with or without preoperative radiotherapy.

PURPOSE: To document the clinical nature and prognosis of locally recurrent rectal cancer after total mesorectal excision (TME) with or without 5 x 5 Gy preoperative radiotherapy (PRT) and to identify patient-, disease-, and treatment-related factors associated with differences in prognosis after local recurrence. PATIENTS AND METHODS: For 96 Dutch patients with a local recurrence who participated in a multicenter randomized clinical trial, data on treatments and follow-up were gathered from surgeons and radiation and medical oncologists. Twenty-three patients (24%) had previously been treated with PRT plus TME, and 73 patients (76%) had been treated with TME alone. Eighty-one patients (84%) were followed until death; median follow-up time of the alive patients after local recurrence was 21 months (range, 5 to 48 months). RESULTS: Survival after local recurrence in the PRT + TME group was significantly shorter than in the TME group (median survival, 6.1 v 15.9 months; hazard ratio for death, 2.1; P =.008). Patients with a local recurrence in the PRT + TME group had distant metastases more often (74% v 40%; P =.004), underwent surgical resection of local recurrence less often (17% v 35%; P =.11), and received radiotherapy for local recurrence at a total dose >/= 45 Gy less often (4% v 42%; P =.001) than patients without PRT. In a multivariate analysis, the difference in survival after local recurrence between randomization groups was no longer statistically significant (hazard ratio for death of PRT, 1.53; P =.16). CONCLUSION: The clinical nature and prognosis of patients with locally recurrent rectal cancer has changed since the introduction of PRT. The majority of patients who present with a local recurrence after previous PRT have simultaneous distant metastases, and median survival has decreased to 6 months.

Colectomy↗
PubMed2004

Review of determinants of patients' preferences for adjuvant therapy in cancer.

PURPOSE: Many studies have determined cancer patients' preferences for adjuvant therapy, for example, by asking patients the extent of benefit they would need in order to accept the therapy. However, little is known about the determinants that influence these preferences. Our research goal was to explore which determinants underlie patients' preferences by means of a literature review. METHODS: PubMed searches were conducted to identify studies in which cancer patients' preferences for adjuvant therapy had been elicited by means of a treatment preference instrument. Twenty-three papers were evaluated with regard to reported relationships between preferences and potential determinants. A total of 40 determinants were recorded and classified into one of seven categories: (1) treatment-related determinants, (2) sociodemographic characteristics and current quality of life, (3) clinical characteristics, (4) measurement instrument-related determinants, (5) time-related determinants, (6) cognitive/affective determinants, and (7) specialist-related determinants. RESULTS: The benefit and toxicity of treatment, experience of the treatment, and having dependents (eg, children) living at home were important determinants of patients' preferences. Furthermore, qualitative data suggested that cognitive/affective and specialist-related determinants might have a large impact on patients' treatment preferences. CONCLUSION: Our results show that patients' preferences cannot fully be explained on the basis of treatment-related determinants and patient and clinical characteristics. More research is needed in the area of cognitive/affective and specialist-related determinants because of the lack of quantitative results. Furthermore, we recommend carrying out larger studies in which the (internal) relationships between determinants and preferences are assessed in the context of a cognitive cost-benefit model.

Chemotherapy, Adjuvant↗
PubMed2004

Assessing comorbidity in patients with Parkinson's disease.

The aim of this study was to assess the accuracy of an interview-based assessment of comorbidity, in patients with Parkinson's disease (PD). The Cumulative Illness Rating Scale-Geriatric (CIRS-G) was completed (1) in an interview with 31 PD patients and their caregivers, and (2) by reviewing the patient's medical charts from their general practitioners. Based on the interview, all patients had some comorbidity, 84% had one or more moderate comorbid diseases. The most frequently affected organ systems were "lower gastrointestinal" and "genitourinary". The mean +/- SD total score of the interview-based (chart-based) CIRS-G was 6.9 +/- 3.8 (7.6 +/- 3.5) with a mean of 4.3 +/- 1.9 (5.0 +/- 1.9) affected organ systems and a mean of 2.1 +/- 1.7 (2.3 +/- 1.6) organ systems with at least moderate comorbidity per patient. The agreement (intraclass correlation coefficients) between the interview-based and chart-based assessments for the six summary scores ranged from 0.69 to 0.81. The agreement for the 14 organ systems ranged from 0.13 to 1.00 (weighted kappa); 12 had a K(w) above 0.40 (moderate agreement). The comorbidity summary scores had a moderate correlation with age and disability. The interview-based assessment of the CIRS-G is easy to apply and is an accurate method to assess comorbidity in patients with PD.

Activities of Daily Living↗
PubMed2004

Assessment of autonomic dysfunction in Parkinson's disease: the SCOPA-AUT.

We developed a questionnaire to assess autonomic symptoms in patients with Parkinson's disease (PD) and evaluated its reliability and validity. Based on the results of a postal survey in 46 PD patients, 21 multiple system atrophy patients, and 8 movement disorders specialists, items were included according to their frequency, burden, and clinical relevance. The questionnaire was evaluated in 140 PD patients and 100 controls, and test-retest reliability was established in a sample of 55 PD patients. The SCOPA-AUT consists of 25 items assessing the following regions: gastrointestinal (7), urinary (6), cardiovascular (3), thermoregulatory (4), pupillomotor (1), and sexual (2 items for men and 2 items for women) dysfunction. Test-retest reliability was good. Autonomic problems increased significantly with increasing disease severity for all autonomic regions, except sexual dysfunction. We conclude that SCOPA-AUT is a reliable and valid questionnaire that evaluates autonomic dysfunction in PD.

Aged↗
PubMed2004

Empirical data and moral theory. A plea for integrated empirical ethics.

Ethicists differ considerably in their reasons for using empirical data. This paper presents a brief overview of four traditional approaches to the use of empirical data: "the prescriptive applied ethicists," "the theorists," "the critical applied ethicists," and "the particularists." The main aim of this paper is to introduce a fifth approach of more recent date (i.e. "integrated empirical ethics") and to offer some methodological directives for research in integrated empirical ethics. All five approaches are presented in a table for heuristic purposes. The table consists of eight columns: "view on distinction descriptive-prescriptive sciences," "location of moral authority," "central goal(s)," "types of normativity," "use of empirical data," "method," "interaction empirical data and moral theory," and "cooperation with descriptive sciences." Ethicists can use the table in order to identify their own approach. Reflection on these issues prior to starting research in empirical ethics should lead to harmonization of the different scientific disciplines and effective planning of the final research design. Integrated empirical ethics (IEE) refers to studies in which ethicists and descriptive scientists cooperate together continuously and intensively. Both disciplines try to integrate moral theory and empirical data in order to reach a normative conclusion with respect to a specific social practice. IEE is not wholly prescriptive or wholly descriptive since IEE assumes an interdepence between facts and values and between the empirical and the normative. The paper ends with three suggestions for consideration on some of the future challenges of integrated empirical ethics.

Empirical Research↗
PubMed2004

Cost measurement in economic evaluations of health care: whom to ask?

PURPOSES: The purposes of this study were 1) to investigate the feasibility of using providers' administrative systems for the assessment of healthcare utilization in economic evaluations performed alongside multicenter studies, 2) to assess the convergent validity of patients' and providers' reports of care, and 3) to investigate whether differences between providers' and patients' reports are related to age, gender, health, recall period, and volumes of care. METHODS: Data were obtained as part of a cost-utility analysis alongside a multicenter clinical trial in patients with rectal cancer. For a sample of 179 patients from 49 hospitals, data on hospitalizations, outpatient visits, medications, and care products during the first year after treatment were obtained from the patients by questionnaire or diary. For all patients, hospitals were contacted for information on hospitalizations and outpatient visits. For a subsample of 94 patients, 86 pharmacists and 10 suppliers of stoma care products were contacted for information on medications and care products. RESULTS: Response by providers of care was high, ranging from 84% to 100%. With respect to hospital days and outpatient visits, we found no significant differences between patients' and providers' reports. For medications and care products, agreement was lower, with providers reporting up to 2 times more product types and costs than patients. Providers failed to report 20% to 25% of all products, whereas patients failed to report 50% to 60% of all products. CONCLUSIONS: Patients' reports seem as valid as providers' reports for hospital days and outpatient visits. For medications and care products, we recommend the use of reports from providers of care, whenever feasible, because they much less underestimate volumes and costs than patients.

Aged↗
PubMed2003

Cost-utility analysis of preoperative radiotherapy in patients with rectal cancer undergoing total mesorectal excision: a study of the Dutch Colorectal Cancer Group.

PURPOSE: To compare the societal costs and the (quality-adjusted) life expectancy of patients with rectal cancer undergoing total mesorectal excision (TME) with or without short-term preoperative radiotherapy (5 x 5 Gy). PATIENTS AND METHODS: We used a Markov model to project the clinical and economic outcomes of preoperative radiotherapy. Data on local recurrence rates, quality of life, and costs were obtained from the patients of a multicenter randomized clinical trial. In this trial, 1,861 patients with resectable rectal cancer from 108 hospitals were randomly assigned for TME surgery with or without preoperative radiotherapy. Outcome measures of the model were life expectancy, quality-adjusted life expectancy, lifetime costs per patient, and the incremental cost-effectiveness ratio. RESULTS: The base case model estimates that the loss of quality of life due to preoperative radiotherapy is outweighed by the gain in life expectancy. Life expectancy increases by 0.67 years; quality-adjusted life expectancy, by 0.39 years; and costs, by $9,800 per patient. The corresponding cost-effectiveness ratio is $25,100 per quality-adjusted life year. Sensitivity analyses indicate that the cost-effectiveness ratio remains acceptable under a wide range of assumptions. CONCLUSION: Assuming that the reduced local recurrence rate does lead to a survival advantage, the cost-utility analysis estimates that the improved survival outweighs the impaired quality of life and the increased costs. We conclude that short-term preoperative radiotherapy in patients with rectal cancer undergoing TME is both effective and cost-effective.

Adult↗
PubMed2003

A short psychosocial questionnaire for patients with Parkinson's disease: the SCOPA-PS.

The purpose of this study was to develop a short questionnaire for psychosocial functioning in patients with Parkinson's Disease (PD). The SCales for Outcomes in Parkinson's disease-PsychoSocial questionnaire (SCOPA-PS) was tested in a survey and compared with other instruments and with medical information. This survey was sent to 205 patents with idiopathic PD. Eighty-six percent of the questionnaires were returned. Cronbach's alpha was 0.83. Two-week test-retest reliability was 0.85 (intraclass correlation coefficient). Construct validity with other scales (Spearman's rho) was 0.82 for the Parkinson's Disease Questionnaire-39-item version (PDQ-39), 0.76 for the PDQ-8, 0.69 for the Hospital Anxiety and Depression Scale, -0.61 for the Euroqol, and -0.60 for a visual analogue scale evaluating Quality-of-Life. The summary index revealed a significant increase with increasing disease severity. The SCOPA-PS is a new, short psychosocial questionnaire for patients with PD with good clinimetric properties.

Activities of Daily Living↗
PubMed2002

High dose chemotherapy and hematopoietic stem cell transplantation: a study of treatment preference in patients with rheumatoid arthritis and rheumatologists.

OBJECTIVE: Patients with intractable rheumatoid arthritis (RA) may benefit from treatment with high dose chemotherapy followed by rescue with autologous hematopoietic peripheral blood stem cell transplant (HSCT). We investigated whether the risks of this approach are acceptable to patients with RA and rheumatologists and whether risk taking by patients was associated with disease characteristics, socioeconomic variables, and/or personality traits. METHODS: A survey in the outpatient clinic was conducted among 2 cohorts of 45 (cohort A) and 51 (cohort B) RA patients with active disease. Patients received information about the potential benefit of HSCT (2/3 chance of a good clinical response, 1/3 no response) and treatment related morbidity and mortality. Cure was assumed not to be a realistic perspective. Cohort A was asked to choose between their own disease state for an indefinite time or HSCT. Nonparametric tests were performed to evaluate putative predictive factors that led patients to accept transplant related mortality (TRM): swollen joint count, tender joint count, visual analog scale (VAS) measures of disease activity and pain, erythrocyte sedimentation rate, Health Assessment Questionnaire (HAQ), socioeconomic variables, RA Quality of Life Questionnaire (RAQoL), and the Life Orientation Test. Cohort B was asked to consider a worst case scenario with respect to their disease activity. The minimal duration of benefit was assessed, given a TRM of 0.01% and 2%. To evaluate treatment preference of physicians, 96 Dutch rheumatologists responded to a hypothetical clinical case analogous to the interviews with RA patients. The minimum duration of benefit was assessed, given a TRM of 2% and the maximal TRM acceptable to rheumatologists if duration of benefit was 2 years in 2/3 patients. RESULTS: In cohort A, 5 of 45 patients were willing to accept risk of death. VAS disease activity (p = 0.006), VAS pain (p = 0.021), and HAQ (p = 0.05) were significantly higher in patients willing to accept risk of death. Religiosity (p = 0.093), a higher Ritchie Articular Index (p = 0.096), and low quality of life (by RAQoL) (p = 0.133) showed trends toward risk taking. In cohort B, 22 of 50 patients (44%) were willing to accept a risk of TRM related to HSCT. For the 22 patients the median required duration of benefit given a TRM of 2% was 5 years (range 1-15). Physicians also required a median duration of benefit of 5 years. CONCLUSION: We evaluated risk taking in patients with RA and physicians based on a realistic perspective in which the tradeoff between short term risks and possible longterm benefit of HSCT was investigated. Based on current efficacy data for HSCT (2 years improvement in 2/3 patients), half the patients would accept the current TRM of 2%, based on registry results. Patients willing to accept TRM had higher VAS disease activity, VAS pain, and HAQ. Doctors were more willing to accept mortality in the treatment of RA.

Adult↗