Montse Roset

Clinical patterns over time in irritable bowel syndrome: symptom instability and severity variability.

OBJECTIVES:The clinical course of irritable bowel syndrome (IBS) remains poorly known. In 209 IBS patients meeting Rome II criteria (137 females and 72 males) we evaluated: (1) changes in frequency and intensity of abdominal pain/discomfort, abnormal number of bowel movements, loose or watery stools, defecatory urgency, hard or lumpy stools, straining during bowel movements, and feeling of incomplete evacuation); (2) use of resources, HRQoL, and psychological well being.METHODS:Observational, prospective, multicenter study. Symptoms were registered in a diary over two 28-day periods with an interval of 4 wk; direct resource use and indirect costs were noted weekly. Three HRQoL questionnaires were administered.RESULTS:High-intensity symptoms were present on more than 50% of the days. Sixty-one percent were classified in the same IBS subtype on both occasions (κ= 0.48), while 49% had the same symptom predominance and intensity (κ= 0.40). The greatest instability was observed among diarrhea (D-IBS) and constipation (C-IBS) subtypes: only 46% and 51% remained in the same pattern with a tendency to shift to alternating diarrhea/constipation subtype (A-IBS); however, practically no patient changed from D-IBS to C-IBS, or vice versa. The most reliable symptom characteristic was frequency, followed by intensity and number of episodes. Symptom frequency and intensity were directly related to resource use and HRQoL impairment.CONCLUSIONS:IBS symptoms are instable over time and variables in intensity. Many patients with D-IBS or C-IBS move to A-IBS; however, shift from D-IBS to C-IBS, or vice versa, is very infrequent.

Splitting irritable bowel syndrome: from original Rome to Rome II criteria.

OBJECTIVES:Diagnosis of irritable bowel syndrome (IBS) and other functional bowel disorders (FBD) is based on symptom evaluation. Clinical criteria have changed over time, yielding different proportions of subjects fulfilling diagnostic requirements. According to new diagnostic criteria (Rome II), subjects considered some years ago to have IBS no longer do so. The aim of this article is to evaluate how patients diagnosed as having IBS according to original Rome criteria have been split, and to which clinical diagnosis they belong today.METHODS:Two hundred and eleven subjects meeting original Rome IBS diagnostic criteria were studied: 65 also met Rome II criteria while 146 did not. Subjects were extracted from an epidemiological survey, using home-based personal interviews, on 2,000 subjects randomly selected as representative of the Spanish population. Clinical complaints, personal well-being, resource utilization, and health-related quality of life (HRQOL) were compared.RESULTS:Of the subjects meeting original Rome but not Rome II criteria, the present diagnosis should be: 40%“minor” IBS (IBS symptoms of less than 12 wk duration), 37% functional constipation, 12% alternating bowel habit, 7% functional diarrhea, 3% functional abdominal bloating, and 1% unspecified functional bowel disorder (FBD). Thus, 52 subjects (36%) should not be diagnosed with IBS because they really had other FBD, 59 (40%) because of symptoms consistent with IBD diagnosis but not the required duration or frequency, and 35 (24%) because of symptoms consistent with some other FBD diagnosis but not meeting the required duration. Clinical complaints, personal well-being, resource utilization, and HRQOL were more severely affected in IBS than in other FBD as a group, and in “major” rather than in “minor” forms.CONCLUSIONS:Many subjects meeting original Rome criteria for IBS do not meet Rome II criteria: approximately one quarter of subjects do not have sufficient symptom duration or frequency to be diagnosed with IBS and almost half are now considered as having other (“major” or “minor”) FBD.

Cost of Clinical Management of Acromegaly in Spain

AbstractIntroduction and Background: The cost of the therapeutic management of acromegaly depends on the selection of resources used, surgery and/or pharmacological treatment, by the specialist responsible for treatment, related to the characteristics of the patient and tumour. The objective of this work is to evaluate these costs for an illness that is rare but that is associated with a high morbidity in the context of routine clinical practice. Methods: This was an epidemiological, prospective, naturalistic, multicentre study in Spain, in which 38 endocrinologists participated. Adult patients with acromegaly and a pituitary microadenoma or macroadenoma were included in the study. Patients were assigned, according to first-line treatment, to the following two groups: surgery first-line group (surgery in the 6 months before inclusion or during the follow-up period) and pharmaceutical first-line group (treatment with somatostatin analogues [SAs] for at least 6 months and with or without surgery after starting treatment with SAs). Data were collected during routine visits made during a follow-up period of 2 years. All resources were estimated at 2009 prices (€) and adjusted according to the Spanish consumer price index in 2010. Results: Seventy-four patients were included, the majority of them with macroadenoma (70%). Eighty-eight percent of patients were treated surgically (76% as a first-line treatment), while 12% of patients received only SAs. Treatment with SAs was used at some point in the study by 85% of patients. The mean annual total cost of acromegaly is h9668 per patient (€9223 for the surgery group and €11 054 for the pharmaceutical group). Seventy-one percent of the direct cost of the disease corresponds to treatment with SAs. The cost of a patient treated only with surgery is €2501 on an annual basis, versus €9745 for a patient receiving only pharmacological treatment. In cases where a combination of both types of treatment is required, the annual total cost ranges from €10 866 to €12364. Conclusion: The annual direct cost per patients of acromegaly in Spain is €9668. Even though surgery is the preferred option for treatment for a great number of patients, SAs must be added to the treatment regimen of the majority of such patients. The costs associated with this treatment are greater than the cost of treatment with SAs alone.

Disease-specific quality of life evaluation and its determinants in Cushing’s syndrome: what have we learnt?

Cushing’s syndrome (CS) has a considerable negative impact on patient health-related quality of life (HRQoL). Two disease-specific instruments (the CushingQoL and the Tuebingen CD-25 questionnaire) are now available to assess the impact of the disease and its treatment on HRQoL. The purpose of this review was to summarize the characteristics of the studies which have used these two instruments to date and summarize their findings regarding (a) the determinants of disease-specific HRQoL in patients with CS and (b) the impact of treatment for CS on disease-specific HRQoL. A total of 7 studies were identified, 5 with the CushingQoL and 2 with the Tuebingen CD-25. Most were observational studies, though the CushingQoL had been used in one randomized clinical trial. In terms of clinical factors, there was some evidence for an association between UFC levels and disease-specific HRQoL, though the presence and strength of the association varied between studies. There was also some evidence that a more recent diagnosis of CS could lead to poorer HRQoL, and that length of time with adrenal insufficiency may also affect HRQoL. There was no evidence for an impact on disease-specific HRQoL of etiology or of the clinical signs and symptoms associated with CS, such as bruising, rubor, and fat deposits. One factor which did have a significant negative effect on HRQoL was the presence of depression. No clear picture emerged as to the effect of demographic variables such as age and gender on HRQoL scores, though there was some evidence for poorer HRQoL in female patients. As regards treatment, the two interventions studied to date (transsphenoidal surgery and pasireotide) both showed significant gains in HRQoL, with moderate to large effect sizes. This type of review is useful in summarizing knowledge to date and suggesting future research directions.

Bayesian regression models for cost-effectiveness analysis.

Recent studies have shown how cost-effectiveness analysis can be undertaken in a regression framework. This contribution explores the use of practical regression models for estimating cost-effectiveness from a Bayesian perspective. Two different Bayesian models are described. The first considers the outcome measure to be a quantitative variable. In the second model the individual outcome measure is a binary variable with value 1 if any objective has been achieved. We describe the implementation of the model using data from a trial that compares two highly active antiretroviral therapies in HIV asymptomatic patients. Data on direct cost and data effectiveness (percentage of patients with undetectable viral load and quality of life) were recorded. If we consider the quality of life as an effectiveness measure, the new treatment is preferred for a willingness to pay more than 142.3 € for an increase in the quality of life. For illustrative purposes, if we compare the results with an analogous model that does not include covariates, the critical value becomes 247.4 €. For the binary measure of effectiveness the control treatment dominates the new treatment.

Results of a 2-year retrospective cohort study of newly prescribed triptan users in European nationwide practice databases

Objective: This study was conducted to characterize prescription refill patterns for triptans among European patients with new prescriptions of triptans. Background: Persistency with prescriptions of triptan monotherapy for migraine headache among newly prescribed users in European primary-care practices has not been well described. Methods: Using electronic medical databases in the UK (N = 3618), France (N = 2051) and Germany (N = 954), we conducted a retrospective cohort analysis to identify refill patterns over 2 years among migraineurs receiving new prescriptions of triptan monotherapy in 2006. Results: Of all patients, >33% of migraineurs with new triptan prescriptions received ≥1 refill of their index triptan prescriptions (UK, 44.3%; France, 34.2%; Germany, 37.7%). More than 50% never received index-triptan refill prescriptions (UK, 55.7%; France, 65.8%; Germany, 63.3%). Small proportions of patients (<7.0%) switched to alternative triptans, and even fewer switched to different prescription-medication classes (UK and Germany, 2.3%; France, 4.0%). More than 48% of patients received no further prescriptions for migraine after index prescriptions (UK, 48.5%; France, 54.9%; Germany, 54.7%). After the second year, >83.0% of patients in each country had no further prescriptions for migraine medications, <14.0% remained persistent with index prescriptions, <4.0% switched to other triptans, and <3.0% switched to alternative medication classes. Conclusions: In migraine patients who received new prescriptions of triptan monotherapy from their primary-care physicians, poor triptan prescription refill frequency was observed in Europe. Although consistent with potential clinical challenges in migraine management, our findings should be interpreted with caution given certain inherent limitations associated with the database study design. Further research is warranted to confirm our findings and to identify reasons for, or predictors of, triptan discontinuation.

Conjoint analysis to understand preferences of patients with multiple sclerosis for disease-modifying therapy attributes in Spain: a cross-sectional observational study

Objective To assess patients’ preferences for a range of disease-modifying therapy (DMT) attributes in multiple sclerosis (MS). Design A cross-sectional observational study. Setting The data reported were from 17 MS units throughout Spain. Participants Adult patients with relapsing-remitting MS. Main outcome A conjoint analysis was applied to assess preferences. A total of 221 patients completed a survey with 10 hypothetical DMT profiles developed using an orthogonal design and rating preferences from 1 (most acceptable) to 10 (least acceptable). Medication attributes included preventing relapse, preventing disease progression, side effect risk, route and frequency of administration. Results Patients placed the greatest relative importance on the side effect risk domain (32.9%), followed by route of administration (26.1%), frequency of administration (22.7%), prevention of disease progression (10.0%) and prevention of relapse (8.3%). These results were independent of the Expanded Disability Status Scale score. The importance assigned to side effect risk was highest for patients with a recent diagnosis. Patients who had previously received more than one DMT gave a higher importance to relapse rate reduction than patients receiving their first DMT. Conclusions Patient DMT preferences were mainly driven by risk minimisation, route of administration and treatment schedule. The risk–benefit spectrum of available DMT for MS is becoming increasingly complicated. Understanding which treatment characteristics are meaningful to patients may help to tailor information for them and facilitate shared decision-making in clinical practice.

Healthcare resource utilization and cost among males with lower urinary tract symptoms with a predominant storage component in Spain: The epidemiological, cross-sectional MERCURY study

To assess the relationship between storage‐predominant LUTS and healthcare resource consumption and cost among males in Spain.

Mapping AcroQoL scores to EQ-5D to obtain utility values for patients with acromegaly

Abstract Aims: To estimate a preference-based single index for the disease-specific instrument (AcroQoL) by mapping it onto the EQ-5D to assist in future economic evaluations. Materials and methods: A sample of 245 acromegaly patients with AcroQoL and EQ-5D scores was obtained from three previously published European studies. The sample was split into two: one sub-sample to construct the model (algorithm construction sample, n = 184), and the other one to confirm it (validation sample, n = 61). Various multiple regression models including two-part model, tobit model, and generalized additive models were tested and/or evaluated for predictive ability, consistency of estimated coefficients, normality of prediction errors, and simplicity. Results: Across these studies, mean age was 50–60 years and the proportion of males was 36–59%. At overall level the percentage of patients with controlled disease was 37.4%. Mean (SD) scores for AcroQoL Global Score and EQ-5D utility were 62.3 (18.5) and 0.71 (0.28), respectively. The best model for predicting EQ-5D was a generalized regression model that included the Physical Dimension summary score and categories from questions 9 and 14 as independent variables (Adj. R2 = 0.56, with mean absolute error of 0.0128 in the confirmatory sample). Observed and predicted utilities were strongly correlated (Spearman r = 0.73, p < .001) and paired t-Student test revealed non-significant differences between means (p > .05). Estimated utility scores showed a minimum error of ≤10% in 45% of patients; however, error increased in patients with an observed utility score under 0.2. The model’s predictive ability was confirmed in the validation cohort. Limitations and conclusions: A mapping algorithm was developed for mapping of AcroQoL to EQ-5D, using patient level data from three previously published studies, and including validation in the confirmatory sub-sample. Mean (SD) utilities index in this study population was estimated as 0.71 (0.28). Additional research may be needed to test this mapping algorithm in other acromegaly populations.

PDB84 MANAGEMENT OF ACROMEGALY IN CLINICAL PRACTICE CONDITIONS IN SPAIN

PDB80 A CONTROLLED, PROSPECTIVE LONGITUDINAL, INTERVENTIONAL, NATURALISTIC STUDY TO EVALUATE THE UNIQUE HEALTH CARE PROGRAM “CONVERSATION MAP©” FOR TURKISH PATIENTS WITH TYPE-2 DIABETES IN ONE GERMAN MAJOR CITY Korolewa V, Kretschmer B, Clouth J Lilly Deutschland GmbH, Bad Homburg, Germany OBJECTIVES: Considering a cultural and epidemiological disparity between Turkish and German type-2 diabetes patients in Germany, resulting in a health care gap, a customised diabetes program for Turkish migrants is required. The objective was to evaluate “Conversation Map©”, a unique health care education program for Turkish migrants with type-2 diabetes in Germany. METHODS: Ninety-two Turkish patients with poor German language skills were divided into interventional (IV, n = 44) and control (C, n = 48) groups, 42 German (G) patients served as additional control group. The IV group attended a structured once weekly, 2 hr “Conversation Map©” training over 4 weeks in Turkish language. The two control groups had no intervention. Sociodemographic data were recorded at baseline, and standardized questionnaires assessing treatment, diabetes-knowledge (0–6; 6 = everything right), and therapy adherence (“Essential Training Content (ETC)” 0–10; 10 = best) were applied at baseline and after 4 weeks after training in the IV group. Baseline differences between the groups and changes of data after 4 weeks compared to baseline were tested (paired t-test, α = 0.05, two-sided). RESULTS: Ethnic groups were comparable on demographic data, whereas Turkish patients had lower education level (P < 0.001). 55% of all Turkish patients (IV and C) were insulin-dependent and 63% were previously not attending any diabetes-training. Baseline diabetes knowledge of Turkish patients was signifi cantly lower than in the German control group (IV 2.38, T 2.77, G: 4.10, P < 0.001), as well as the ETC score (IV 6.37, C 5.92, G 7.98, P < 0.001). After “Conversation Map©” training the Turkish group achieved signifi cantly higher mean knowledgeand ETC-score values compared to baseline (difference +70%, ETC: +25%, P < 0.001) and were comparable to the German group. CONCLUSIONS: The study demonstrated that the cultural sensitive training “Conversation Map©”, led to a signifi cant increase of disease-knowledge and therapy adherence, helping to improve the health care situation of Turkish patients, with cost savings potential in the long term.