The management of iron deficiency in inflammatory bowel disease--an online tool developed by the RAND/UCLA appropriateness method.

BACKGROUND: Iron deficiency is a common and undertreated problem in inflammatory bowel disease (IBD). AIM: To develop an online tool to support treatment choice at the patient-specific level. METHODS: Using the RAND/UCLA Appropriateness Method (RUAM), a European expert panel assessed the appropriateness of treatment regimens for a variety of clinical scenarios in patients with non-anaemic iron deficiency (NAID) and iron deficiency anaemia (IDA). Treatment options included adjustment of IBD medication only, oral iron supplementation, high-/low-dose intravenous (IV) regimens, IV iron plus erythropoietin-stimulating agent (ESA), and blood transfusion. The panel process consisted of two individual rating rounds (1148 treatment indications; 9-point scale) and three plenary discussion meetings. RESULTS: The panel reached agreement on 71% of treatment indications. 'No treatment' was never considered appropriate, and repeat treatment after previous failure was generally discouraged. For 98% of scenarios, at least one treatment was appropriate. Adjustment of IBD medication was deemed appropriate in all patients with active disease. Use of oral iron was mainly considered an option in NAID and mildly anaemic patients without disease activity. IV regimens were often judged appropriate, with high-dose IV iron being the preferred option in 77% of IDA scenarios. Blood transfusion and IV+ESA were indicated in exceptional cases only. CONCLUSIONS: The RUAM revealed high agreement amongst experts on the management of iron deficiency in patients with IBD. High-dose IV iron was more often considered appropriate than other options. To facilitate dissemination of the recommendations, panel outcomes were embedded in an online tool, accessible via http://ferroscope.com/.

[The management of prostate cancer: a retrospective chart review in 808 French men undergoing biopsy]. / Prise en charge du cancer de la prostate: analyse rétrospective de 808 hommes biopsiés en France.

OBJECTIVE: We explored the characteristics of a sample of men who had undergone a biopsy in clinical practice in France and evaluated initial treatment choice in men with a positive biopsy. METHODS: This was a multi-centre, retrospective chart review including men who had undergone a biopsy in France. Clinical variables were collected using an electronic data capture system. RESULTS: Eight hundred and eight men were included; 632 men (78%) had an initial biopsy and 176 men (22%) had one or more repeat biopsy. The mean age was 64 years and 9% of men were 75 years or more. The mean (median) PSA was 11.6 (7.0) ng/mL; 25% of men had a PSA greater than 10 ng/mL. Twenty-eight percent of men had a suspicious DRE. A total of 52% of men had a positive initial and 26% a positive repeat biopsy. One hundred and eleven patients (34%) had low-risk PCa (stage T1c-2a, PSA<10 ng/mL, Gleason sum<7) and 195 (59%) were at intermediate/high risk of disease progression. The most common treatment was radical therapy (54% of patients), even in men with low-risk PCa (40% of patients). A total of 38% of low-risk patients chose active surveillance. CONCLUSIONS: The French biopsy sample appeared to be at a relatively high risk of having PCa at initial biopsy. Radical therapy was the most common treatment choice in men with a positive biopsy. In patients with low-risk PCa, radical therapy and active surveillance were used most often and to the same extent.

Integrating risk profiles for disease progression in the treatment choice for patients with lower urinary tract symptoms/benign prostatic hyperplasia: a combined analysis of external evidence and clinical expertise.

The RAND appropriateness method was used to explore the relevance of risk factors for disease progression in the treatment choice for patients with lower urinary tract symptoms/benign prostatic hyperplasia (LUTS/BPH). A total of, 12 international experts assessed the appropriateness of various treatments for 243 risk profiles. Highest appropriateness rates were found for alpha1-adrenoceptor antagonists (68% of profiles) and combination therapy (46%). A large prostate volume was the dominant argument in favour of 5alpha-reductase inhibitors and combination therapy, but was irrelevant for the choice of surgery. Considerable postvoid residual, severe symptoms and poor maximum flow rate were the most important factors in favour of surgery.

Risk factors for disease progression in patients with lower urinary tract symptoms/benign prostatic hyperplasia (LUTS/BPH): a systematic analysis of expert opinion.

Disease progression has become an important issue for the management of lower urinary tract symptoms/benign prostatic hyperplasia (LUTS/BPH). Although several risk factors have been identified, no specific patient risk profiles have been established that can be useful in the day-to-day management of LUTS/BPH. In this study, an international panel of urologists developed a risk classification based on the attribution of a risk score to 243 unique patient profiles. From the perspective of clinical decision making, it was concluded that postvoid residual, symptom severity and maximum flow rate are the most relevant determinants of the risk of disease progression.

Gastro-protective treatment in patients using NSAIDs. Development of appropriateness criteria by a multidisciplinary expert panel.

OBJECTIVE: To refine the appropriate indications for gastro-protective treatment in patients using non-steroidal anti-inflammatory drugs (NSAIDs). METHODS: Using the RAND Appropriateness Method, a multidisciplinary expert panel was asked to rate the appropriateness of gastro-protection for 4608 different cases. Logistic regression was used to construct a decision framework to identify patients for whom gastro-protective medication should be considered. RESULTS: Complete consensus existed on the appropriate use of gastro-protection in patients with a history of (un)complicated peptic ulcer. For other patients. agreement was found for 39% over 1536 cases. Logistic regression demonstrated strong consistency of the panel ratings (Hosmer Lemeshow coefficient 0.92), allowing the development of a comprehensive decision support model. CONCLUSIONS: Using the RAND Appropriateness Method, we were able to develop a clear and internally consistent decision framework for the appropriate use of gastro-protection in patients taking NSAIDs. The validity of this model should be tested in further studies and practice.

The appropriate management of symptomatic gastro-oesophageal reflux disease (GORD) in primary care: a systematic analysis of expert opinion.

OBJECTIVE: To explore the appropriate indications for endoscopy and short-term anti-secretory treatment in patients with symptoms of gastro-oesophageal reflux disease (GORD). METHODS: The RAND Appropriateness Method (RAM) was used to systematically investigate the opinions of an expert panel (6 gastroenterologists and 6 general practitioners) on the appropriateness of either endoscopy or short-term medication for 768 different patient scenarios (cases). Each case was defined by the unique combination of diagnostic characteristics considered to be relevant in treatment choice. Panel members firstly individually rated the appropriateness of all indications using a 9-point scale (9 = extremely appropriate, 1 = extremely inappropriate). Subsequently, the panel discussed the results and re-rated some of the indications. Based on the median score and agreement figures, the individual ratings were converted to panel statements (appropriate, inappropriate, and uncertain) for each of the indications. Logistic regression was used to study the relationship between diagnostic characteristics and panel outcomes. RESULTS: Disagreement was seen in only 18% of cases. Statistical analysis revealed consistent patterns that determined the panel judgements. The most pronounced patterns and regression results were used to indicate situations in which either medication or referral was considered appropriate by the panel. CONCLUSION: The RAND panel method proved to be useful in the systematic analysis of expert opinion on the appropriate management of symptomatic GORD. Nevertheless, as the recommendations still reflect the subjective opinion of the panel members, their validity and usefulness for daily practice should be the subject of further investigations.

Sexual function before and after various treatments for symptomatic benign prostatic hyperplasia.

OBJECTIVE: To determine the effect of various treatments for benign prostatic hyperplasia (BPH) on sexual function. PATIENTS AND METHODS: In a longitudinal multicentre study carried out in the Netherlands, 670 consecutive patients with BPH (aged > or = 50 years) were examined at baseline and 9 months after inclusion. All patients completed a questionnaire about symptomatology, bothersomeness and sexual function before and after treatment. Other diagnostic information was retrieved from the medical records. RESULTS: At baseline, 49-59% of the population (mean age 66 years) reported normal potency and 69% a normal libido. Outcomes at 9 months were stratified by type of treatment, i.e. surgery in 207, alpha-blockers in 43, finasteride in 47 and watchful waiting in 234 men. Patients with multiple or other treatments (131) or recent surgery (eight) were excluded from the analyses. For the four sexual items included, 84% of the patients reported no changes. All treatments showed both improvement and deterioration in 3-14% and 0-16% of the patients, respectively. CONCLUSION: For sexual function, most patients remained stable after treatment for BPH, while positive and negative changes occurred in equal proportions for all treatments. These results question the previously reported high incidence of sexual adverse events, particularly after surgical intervention.

The appropriateness of treatment of benign prostatic hyperplasia: a comparison of Dutch and multinational criteria.

Over the last decade, a number of organisations have developed clinical guidelines, typically at a national level, in order to increase appropriate health care. This raises the question as to whether it is possible to develop guidelines, applicable on the national level, at an international level. In order to examine this, we compared the appropriateness criteria for the treatment of benign prostatic hyperplasia ratings developed by two panels, one a single-nationality (Dutch) panel, the other a multinational (European) panel. The panels, both consisting of experienced urologists, used a modified Delphi process to rate 1152 indications for the most common treatments (surgery, alpha-blocker, finasteride and watchful waiting) on a nine-point scale. This article describes the similarities and differences between the ratings produced by the panels. The appropriateness ratings were identical for 84% of the indications (kappa=0.76). The difference in the scores for individual indications was zero in 41% of indications and less than or equal to two in 99% of indications. This study provides strong evidence that a multinational panel can deliver essentially the same appropriateness ratings for BPH as a national panel. Developing appropriateness criteria on an international level may result in significant savings and may help contribute to the reduction of undesirable practice variation.

Changing therapeutic regimens in benign prostatic hyperplasia. Clinical and economic considerations.

About one-quarter of men aged 50 years and older experience voiding problems due to benign prostatic hyperplasia (BPH). Until about 10 years ago, surgery (particularly transurethral resection of the prostate) was the only effective treatment for symptomatic BPH. Over the last decade, several new treatments have been introduced. These include different types of medication (alpha-blockers and finasteride), thermotherapy, laser prostatectomy, needle ablation and vaporisation methods. The diffusion of these less invasive treatment modalities has resulted not only in a decrease in the age-adjusted surgery rates, but also in an increase of the total number of men treated for BPH. A large number of studies on clinical benefits and risks reveal that the conventional types of surgery remain the most effective treatments, whereas new interventional therapies require a shorter hospital stay and result in fewer short term complications. The efficacy of medication is lower than that of interventional treatments. Adverse effects include dizziness and orthostatic hypotension (alpha-blockers) and decreased sexual function (finasteride), but are generally mild. There is some evidence that medication and minimally invasive treatments may preclude eventual surgical treatment, but the precise effect is difficult to estimate because of differences in the study populations and the relatively short study periods. As a result of the dynamic nature of BPH treatment and the lack of long term data, the cost effects of the introduction of the various new treatments are also difficult to assess. Given the aging of the population and the growing percentage of patients with BPH for whom any type of treatment can be considered, a considerable increase of total costs can be expected. Long term prospective studies are necessary to gain insight into the most cost-effective treatment for different patient groups.

Lower urinary tract symptoms suggestive of benign prostatic obstruction: how can clinical expertise contribute to rational management?

OBJECTIVE: To perform a systematic analysis of clinical expertise on treatment for benign prostatic hyperplasia (lower urinary tract symptoms (LUTS) suggestive of benign prostatic obstruction (BPO)) and to investigate the usefulness of these data in further guideline development. METHODS: A modified Delphi method was used to analyse the opinions of a panel of 15 European urologists on the appropriateness of 4 common treatments for 1,152 "indications" (hypothetical cases) for LUTS suggestive of BPO. Each indication consisted of a unique combination of 9 diagnostic variables, found to be relevant in treatment choice in previous research. The study population was restricted to patients for whom current guidelines do not provide clear indications on the most appropriate treatment. The panellists individually rated the appropriateness of three active treatments (surgery, alpha(1)-adrenoceptor antagonists, finasteride) using a 9-point scale, all in comparison with "watchful waiting". Aggregate panel judgements were calculated from individual ratings for each indication (appropriate, inappropriate, and uncertain). The relationship between diagnostic characteristics and panel opinions was analysed using logistic regression methods. The results were compared to those of an identical panel study including 12 Dutch urologists. RESULTS: Strong agreement existed for 42.5% of the indications, while strong disagreement was found in only 0.1%. For patients who had not previously been treated for LUTS, surgery was considered appropriate in 44% of the indications. For alpha(1)-adrenoceptor antagonists and finasteride these percentages were 56 and 6 respectively. Strong contra-indications were found only for finasteride (34%). Logistic regression analysis demonstrated consistent panel opinions, indicating a strong cumulative impact of almost all diagnostic variables on the panel judgement "appropriate". The figures on appropriateness were highly comparable to the results of the Dutch study (overall agreement 84%, kappa 0,76). A computer program was constructed to facilitate the implementation and evaluation of the panel recommendations in daily clinical practice. CONCLUSIONS: Given the consistency of the panel opinions, the results may be useful in complementing evidence-based guidelines for LUTS suggestive of BPO in the grey area of treatment choice.

[Appropriate treatment of benign prostatic hyperplasia; refining the indications for treatment by systematic analysis of expert opinion]. / Passende behandeling bij benigne prostaathyperplasie; verfijning van de indicatiestelling door systematische analyse van klinische expertise.

OBJECTIVE: To assess systematically the opinion of urology experts regarding the appropriateness of indications for treatment of benign prostatic hyperplasia (BPH) and to evaluate the potential use of these expert opinions for the refinement of treatment guidelines. DESIGN: Modified Delphi procedure. METHODS: A panel of 12 Dutch urologists judged the appropriateness of three common treatments (surgery, alpha-adrenergic antagonists, finasteride) for 1152 hypothetical cases of BPH. These cases consisted of all combinations of 9 diagnostic characteristics considered relevant to treatment choice. The study population was restricted to patients for whom current (evidence-based) guidelines do not provide clear indications on the most appropriate treatment. The panel members individually rated the appropriateness of the three active treatments using a 1 to 9 scale, each in comparison with 'watchful waiting'. By combining the results on agreement and appropriateness, aggregate panel judgements were calculated for each indication (appropriate, inappropriate, uncertain). The relationship between diagnostic characteristics and panel opinions was studied using logistic regression methods. RESULTS: For patients without previous treatment for BPH, surgery was considered appropriate in 44% of cases. For alpha-blocking drugs and finasteride, these values were 70% and 3% respectively. Logistic regression analysis revealed a strong and consistent relationship between the several diagnostic characteristics and the panel judgement 'appropriate indication'. CONCLUSION: Systematic analysis of clinical expertise can offer a meaningful contribution to the refinement of indications for BPH treatments.

Treatment choice for benign prostatic hyperplasia: a matter of urologist preference?

PURPOSE: New treatment modalities for benign prostatic hyperplasia (BPH) have considerably altered the decision making process in daily clinical practice. Guidelines provide a framework for treatment choice but leave much room for physician personal opinions. We identified and quantified determinants of treatment choice for BPH among urologists focusing on urologist treatment preferences. MATERIALS AND METHODS: The study population consisted of 670 consecutive patients with BPH 50 years old or older newly referred to 1 of 39 urologists in a stratified sample of 13 hospitals throughout The Netherlands. Data on patient characteristics were retrieved from patient questionnaires (symptomatology, bothersomeness, sexual function), medical records (diagnostic outcomes, co-morbidity) and urologist questionnaire (initial treatment choice and main considerations for this decision). Urologist treatment preferences were inventoried using a mailed questionnaire. Polychotomous logistic regression analysis was used to study the impact of patient characteristics and urologist preferences on treatment choice. RESULTS: Among the patient characteristics maximum flow rate, residual urine and prostate volume were strongly associated with the probability of surgery and watchful waiting. However, the influence of urologist preferences on actual decisions was also significant. Adjusted for case mix the differences in low and high preferences revealed a 2.2 times greater probability of surgery. For alpha-blockers and finasteride these ratios were 1.8 and 9.4, respectively. An additional independent effect was seen for urologist extent of experience. CONCLUSIONS: The influence of urologist personal preferences on treatment choice in BPH is considerable. Given the different efficacy and side effects of the various treatments, further consensus development is needed to enhance appropriate treatment decisions and eliminate undue costs.

The economic impact of introducing transurethral microwave thermotherapy in the treatment of benign prostatic hyperplasia: a scenario analysis.

The goal of this study was to assess the economic impact of introducing transurethral microwave thermotherapy (TUMT) in the treatment of benign prostatic hyperplasia (BPH). Different scenarios were constructed using both randomized clinical trial data and observational data on resource use related to BPH treatments. These include a baseline scenario, demand scenarios reflecting the number of men who will be treated by TUMT when it is introduced, and supply scenarios reflecting the number of hospitals that will provide TUMT. In the baseline scenario, costs of BPH treatment equal Netherlands guilders (NLG) 203 million. If the demand for BPH treatment does not increase following the adoption of TUMT, costs may vary between NLG 187 and 189 million, depending on how TUMT is provided. If the demand increases up to 25% following the introduction of TUMT, costs may vary between NLG 457 and 466 million, depending on how TUMT is provided. The introduction of TUMT seems to be cost-saving, but savings depend on the number of men who seek treatment for BPH. There is no indication for a controlled provision.

Interpretation of uroflowmetry curves by urologists.

PURPOSE: Uroflowmetry has become a routine investigation in patients with symptoms of the lower urinary tract. Little is known about the variation in the use of uroflowmetry and in the interpretation of its outcomes. We investigated the diagnostic value of uroflowmetry as a freestanding test, and examined the interobserver and intra-observer variation in the interpretation of uroflowmetry curves. MATERIALS AND METHODS: A representative panel of 58 urologists was questioned about the relevance of visual inspection and flow parameters for interpretation. In addition, they individually assessed 25 randomly selected uroflowmetry curves (from patients with no abnormalities and those with various lower urinary tract symptoms) regarding normal findings and the most likely diagnosis. To investigate intra-observer agreement 4 of these curves were studied twice. RESULTS: Voided volume (81%), visual inspection (77%) and maximum flow rate (77%) were most frequently mentioned as relevant for interpretation. Large differences existed between panel opinions and actual case information. For 43% of the normal cases the panel members considered the curves as abnormal. Of the abnormal cases 6% of the curves were regarded as normal. The urologists predicted correctly the actual diagnosis in 36% of all cases. Interobserver agreement was moderate for normalcy (kappa 0.46, standard error 0.087) and poor for the most likely diagnosis (kappa 0.30, standard error 0.043). Intra-observer agreement was also not satisfactory. On average, for the 4 cases studied twice 29% of the panel members chose another option for normalcy, while 41% mentioned another diagnosis the second time. CONCLUSIONS: These results necessitate reconsideration of the diagnostic use of uroflowmetry in daily urological practice.

[Variation in the diagnosis and treatment of benign prostatic hyperplasia in urological practice]. / Variatie in diagnostiek en behandeling van benigne prostaathyperplasie in de urologische praktijk.

OBJECTIVE: To analyse practice variation among urologists and its determinants with respect to diagnostics and therapy choice in benign prostatic hyperplasia (BPH). DESIGN: Cross-sectional. SETTING: Stratified sample of 12 urological practices throughout the Netherlands. METHODS: On 670 consecutive, newly referred BPH patients > or = 50 years, data were collected about symptomatology, discomfort and sexual functioning (patient questionnaire), diagnostic procedures and outcomes (medical record), and (considerations for) therapy choice (urologist questionnaire). Characteristics of patients as well as of urologists were integrally studied to explain the variation. RESULTS: With respect to diagnostics, highest variation (interquartile ranges) was found for ultrasonography of the prostate (19-86%), kidneys (19-68%), and bladder (42-91%), and lowest variation for digital rectal examination (97-100%) and blood tests and urinalysis (88-100% and 86-99% respectively). For therapy choice, interquartile ranges were 24-42% (surgery), 32-49% (watchful waiting), 5-29% (alpha-blockers), and 0-17% (5-alpha-reductase-inhibitor). Variation in diagnostics was associated with characteristics of urologists and work setting as well as of patients. For differences in therapy choice, symptomatology, discomfort, sexual activity, peak flow, volume of residual urine, prostate volume, comorbidity, experience of the urologist, and the type of hospital were the most important explanatory variables. CONCLUSION: Variation in both diagnostics and therapy choice is considerable. This variation is associated with characteristics of patients as well as of urologists and work setting.

The symptom questionnaire for benign prostatic hyperplasia: an ambiguous indicator for an ambiguous disease.

OBJECTIVE: To assess the criterion validity of the American Urological Association (AUA) symptom index for benign prostatic hyperplasia (BPH) in urological practice. PATIENTS AND METHODS: The study comprised 1414 consecutive men > or = 50 years of age, newly referred to a urologist in a stratified sample of 12 hospitals throughout the Netherlands, who completed the AUA symptom questionnaire at their first visit. The 39 urologists completed a questionnaire on the diagnosis. The discriminative power of the index for BPH versus other urological diagnoses was calculated by receiver operating characteristic (ROC) analysis. Subsequently, criterion sensibility was studied by assessing the effect of particular variables on the probability of the diagnosis of BPH. RESULTS: ROC areas were low for BPH versus prostate cancer (0.57, SE 0.04), chronic prostatitis/prostatodynia (0.65, SE 0.03), and other diseases of the lower urinary tract (0.57, SE 0.04). Satisfactory to good values were found for BPH versus diseases of the upper urinary tract (0.79, SE 0.03), impotence/infertility (0.79, SE 0.04), penile-scrotal diseases (0.85, SE 0.02), and no abnormalities (0.84, SE 0.03). The diagnosis of BPH, as made by urologists in daily practice, appeared to be a weak criterion. Irrespective of other characteristics, the chance of being diagnosed with BPH was affected by differences in definition and the type of hospital. CONCLUSION: Although the AUA index discriminated fairly well between BPH and diseases of the upper urinary tract, as well as genital diseases, it has no potential for distinguishing BPH from other diseases of the lower urinary tract. In addition, the lack of consensus in defining clinical BPH underscores the weakness of the index as a tool in the diagnostic process of this disease.