Article

Variation in dermatologist beliefs about the safety and effectiveness of treatments for moderate to severe psoriasis

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Abstract

Background: Multiple systemic treatments are available for moderate to severe psoriasis, but dermatologists' perceptions of these treatments are unknown. Physician perceptions can influence prescribing patterns and patient outcomes, and may help to explain variations in clinical practice. Objective: We sought to describe the variation in dermatologist's beliefs about the safety and effectiveness of psoriasis treatments and evaluate how these relate to dermatologist characteristics and treatment preferences. Methods: We conducted a cross-sectional mail survey of a random sample of 500 National Psoriasis Foundation (NPF) members and 500 American Academy of Dermatology (AAD) members who treat psoriasis. Results: Of 989 clinicians who could be contacted, 246 NPF members and 141 AAD members returned the survey (39% response rate). Respondents perceived infliximab, ustekinumab, cyclosporine, and adalimumab to have the highest likelihood of skin clearance in 3 months (67%-75%). Etanercept, adalimumab, ultraviolet B, and ustekinumab had the lowest perceived likelihood of side effects requiring treatment discontinuation (9%-11%). Up to 49% of respondents "didn't know" the effectiveness or likelihood of side effects; calculated coefficients of variation were higher for perceived likelihood of side effects than perceived effectiveness. There were few significant associations between safety and effectiveness perceptions and respondent characteristics, and treatment preferences were not consistently predictive of perceptions. Limitations: Only dermatologists with interest in treating psoriasis were surveyed and general perceptions were elicited via survey format. Perceptions may differ between survey respondents and nonrespondents. Conclusions: Psoriasis providers demonstrate wide variation in their perception of the effectiveness and especially safety of systemic treatments.

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... Создание новых лекарственных препаратов с применением генно-инженерных технологий явилось одним из наиболее перспективных современных достижений биотехнологии в области медицины. положительные оценки соотношения пользы и риска продемонстрированы в первоначальных краткосрочных исследованиях этих препаратов [34][35][36][37][38] и были подтверждены необходимыми долгосрочными оценками безопасности относительно нежелательных явлений [39]. ...
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The article describes high incidence and morbidity rate of psoriasis, substantial reduction in the life quality and psychosocial disadaptation of patients, and presents certain particular features of psoriasis pathogenesis taking into consideration the role of immune mechanisms and relation between the disease and other chronic processes in the organism, as a result of which psoriasis is considered to be a multimorbid condition. The multimorbidity of psoriasis is an important factor for selecting a therapy, especially for patients with severe forms of the disease.
... g. delay in receiving systemic treatment.34 Other studies have documented patients' and physicians' concerns about safety as a barrier to prescription of certain systemic therapies.30,35,36 A strength of our study is the large number of included patients; however, patient numbers were low in some of the cohort analyses limiting interpretation of the results. ...
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Background Topical treatments are first‐line therapies, prescribed to most patients with chronic plaque psoriasis. Objective This non‐interventional, longitudinal study examined data regarding the treatment pathways of French patients with psoriasis vulgaris using a pharmacy database. Methods From this database, patients with an initial prescription of a topical treatment of interest (i.e., calcipotriol alone and/or calcipotriol/betamethasone) between March and October 2013 were included in the study. The primary objective was to capture the switch from a topical treatment, from treatment initiation to receipt of a systemic therapy over a period of 3 years. Results A total of 26,605 patients were included in the study. The mean age was 58.5 years. The majority of patients (94.7%) maintained topical treatment during the 3 years, receiving a mean of 1.1 different therapies. Of 1400 patients who switched to a systemic therapy, 93.1% switched to a non‐biological (mean time to switching >400 days), maintaining this for the remainder of the follow‐up period. The most commonly prescribed first non‐biological systemic therapy was methotrexate (37%). Less than 1% of patients switched to a biological therapy during follow up. Cohort analyses suggest that patients progressing to use of a systemic therapy within 12 months were those with more severe disease. Conclusions There was a low rate of transition from topical to systemic therapies in patients with chronic plaque psoriasis during the first 3 years of treatment, suggesting stability of disease severity over time with topical therapy alone, potentially due to good patient adherence. This article is protected by copyright. All rights reserved.
... Возможно развитие так называемого ускользания терапевтического эффекта. Данный феномен представляет собой следствие иммуногенности, которая формируется за счет выработки аутоантител и/или нейтрализующих антител [12,13]. ...
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Objective: We aim to present preliminary on about the efficacy of a new drug for plaque psoriasis and psoriatic arthritis treatment - an oral selective PDE-4 inhibitor (apremilast) in 3 psoriasis patients and evaluate apremilast efficacy and safety. Materials and methods: The study enrolled patients with moderate-to-severe plaque psoriasis and psoriatic arthritis who demonstrated lack of efficacy, negative side effects or intolerance to systemic therapy with methotrexate, acitretin, and phototherapy in anamnesis. Patients were administered with apremilast according to the prescription (start 10 mg daily, stepwise increase to 30 mg taken orally twi ce a day). The severity was estimated by PASI, BSA, sPGA, ptPGA; the patient's quality of life was determined by DLQI. The efficacy was evaluated at week 14. Results: All patients reached significant clinical improvement (two patients reached δPASI50, one patient δPASI75, improvement of the nail plate state). Conclusion: According to our observations, apremilast is safe and effective for the treatment of moderate-to-severe plaque psoriasis, scalp and nail psoriasis, and psoriatic arthritis.
... Nearly 7.5 million Americans suffer from psoriasis, and physicians are frequently opting to treat these patients with biologics as a first line treatment. [3,4] Ustekinumab is a fully human monoclonal antibody targeting IL-12 and IL-23, inhibiting the Th1 and Th17 immune pathways. After receiving FDA approval for moderateto-severe plaque psoriasis in 2009, ustekinumab quickly became the standard against which many promising novel biologics were compared. ...
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Purpose of Review This review will highlight the latest data on ustekinumab, as well as provide anecdotal evidence and insight into unanswered questions regarding its safety and the populations’ best suited for its use. Recent Findings In numerous clinical trials, ustekinumab has been found to be safe and efficacious. Many targeted psoriasis medications affecting the same pathway have since been approved as treatments. Recent data supports the notion that ustekinumab does not increase risk of cardiovascular events, and in fact, may be protective against them. Summary Targeted biologic medications for psoriasis have given insight into the complex interactions of the immune system. With these medications, patients suffering from psoriasis can now achieve up to 100 % skin clearance. Ustekinumab (Stelara®; Janssen Biotech, Inc.), a fully human monoclonal antibody against the p40 subunit of interleukin (IL) 12 and IL 23, was approved in 2009 for the treatment of moderate-to-severe plaque psoriasis and has become a standard against which other biologics are tested. Future studies should be directed toward exploring the long-term safety of ustekinumab, as well as efficacy of ustekinumab beyond 5 years of therapy.
... Comparative effectiveness data are important to both the physician and patient for differentiating between treatment options when selecting a therapy, especially as the options for biologic therapy continue to expand. 1 Consequently, there is increasing demand for comparative effectiveness data in the current health care environment to better inform patients and physicians when choosing appropriate treatments for psoriasis. [2][3][4] Selection of therapy depends on numerous factors, including efficacy, safety, response over time, convenience, and affordability. Furthermore, some studies have demonstrated a correlation between measures of healthrelated quality of life (HRQoL) and effectiveness, indicating the importance of patient-reported outcomes. ...
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... Surveys have shown that dermatologists' preferences of first-line therapy for treating moderate-to-severe psoriasis as well as beliefs about the safety and effectiveness of these therapies exhibit wide variability. 44,45 These existing differences underline the need for measures such as guideline training to strengthen physicians' compliance to guidelines. ...
Article
Despite the availability of effective therapeutics and evidence-based treatment guidelines, a substantial proportion of patients with moderate-to-severe psoriasis does not receive appropriate care. This under-provision of health care may cause further worsening of health, remarkable limitations of the patient's quality of life, and indirect costs for the health care system. In order to provide guideline-compliant care for every psoriasis patient, it is important to identify barriers obstructing optimal care. Studies have identified various barriers on the physician's and on the patient's side; however, respective studies approached only single barriers, and not all of them in the context of psoriasis. Other publications that describe barriers systematically did not focus on psoriasis either. The objective of this literature review was to identify barriers and facilitators, based on studies analysing quality of care and single barriers, resulting in a comprehensive model of causal factors. Our analyses revealed three categories of barriers - patient-related, physician-related and external factors: On the patient side, we found non-adherence to therapies to be an important barrier, often in close association with psychiatric factors. Barriers on the physician's side predominantly are incomplete knowledge of the guidelines as well as the complexity of psoriasis comorbidity. In some countries, payment for patients with complex disease status is poor and inconsistent reimbursement regulations potentially interfere with optimal care. The current analysis indicates that most barriers are interdependent. Thus, measures approaching related barriers simultaneously are required. To improve care for psoriasis patients, further studies systematically addressing all potentially relevant barriers in conjoint are needed.
... 1 Around 10% of the patients diagnosed with psoriasis by health systems receive systemic therapy. 2 Surveys have shown that dermatologists' preferences for first-line therapy of moderateto-severe psoriasis, and beliefs about the safety and effectiveness of these therapies, exhibit wide variability. 3,4 Whether these expressed beliefs are reflected in practice is unknown. ...
Article
BACKGROUND: Several national prospective registries of psoriatic patients treated with systemic therapies are running, with the aim of describing the population treated, safety and effectiveness of these treatments, especially biologics. Psonet is an initiative to pool data from these registries. OBJECTIVES: To describe psoriasis therapy in Psonet countries, using baseline data of patients included in these registries. METHODS: We collected data from Psocare (Italy), Dermbio (Denmark), Biobadaderm (Spain), Clalit Health Services (Israel), Australasian Psoriasis Registry, Psobest (Germany), and AMC Medical Center Registry (Netherlands). We described previous use of drugs at the time that patients started a new classic systemic drug or any biologic drug. RESULTS: Data from 20,232 patients was pooled in our analysis (9,668 treated with biologics, 10,564 with other systemic therapies). At a given time in the life course of psoriasis, we have shown large between country heterogeneity on the previous use of systemic drugs for psoriasis and relevant rates of use of biologic drugs in potentially off-label use (first line use and use in psoriasis forms different from plaque psoriasis). Variability in therapy is larger than variability in available patient characteristics likely to influence therapy. CONCLUSIONS: We have shown the presence of large heterogeneity on the use of systemic drugs for psoriasis in participating countries, including differences in patient access to biologics amongst the participating countries. This might be an indicator of unwarranted clinical variation in some countries: a marker of inefficient or less safe use of systemic drugs for psoriasis, and requires further study.
Chapter
Biologic medicines can be very effective treatments for a variety of illnesses, most notably several cancers and chronic autoimmune diseases such as rheumatoid arthritis, ulcerative colitis, Crohn's disease, and psoriasis. The quality use of medicines approach is helpful as a means of both guiding appropriate therapy and providing the education and supports to see such guidance implemented. Quality use of medicines promotes cost‐effective use of the medicines while building the evidence base with real‐world data to continue to provide patients with the best care. Pharmacy and Therapeutics Committees, insurers, and third‐party payers will find these helpful in their formulary decisions and ongoing reviews of management pathways. Uptake of biologic medicines, including biosimilars, varies across Europe. Adherence to and persistence with medication for chronic conditions is a frequently reported issue for all pharmacological therapies and a number of factors have been found to be important.
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Purpose of Review Despite a robust therapeutic landscape, significant gaps exist in the quality of care of psoriatic disease. Thus, an improved understanding of the challenges in providing quality care and the implementation of effective strategies to overcome them is needed. In this review, we summarize the burden of psoriatic disease, discuss the challenges in the care of psoriatic patients, and outline how combined dermatology-rheumatology clinics bridge many of these gaps. Recent Findings Multiple challenges are faced in providing high-quality care to patients with psoriasis and psoriatic arthritis from the pre-diagnosis phase of disease to the follow-up period. Challenges are mainly driven by lack of education of patients and healthcare providers, inefficient communication between specialists, lack of a holistic approach to patients, and limitations of available therapies. The Psoriasis and Psoriatic Arthritis Clinics Multicenter Advancement Network (PPACMAN) is working on demonstrating the effectiveness of combined dermatology-rheumatology clinics in addressing some of these challenges. Recent findings show that combined clinic models may improve quality of care by raising awareness of psoriatic disease, fostering educational activities for both patients and physicians, and allowing for comprehensive evaluation and management of patients through improved communications between disciplines. Summary Psoriasis and psoriatic arthritis are complex diseases that often require an interdisciplinary approach. Thus, combined dermatology-rheumatology clinics and local-regional partnerships are potentially effective in improving quality of care in psoriatic disease.
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The National Psoriasis Foundation (NPF) is developing an agenda for patient-centered research to help patients and their caregivers make more informed health care decisions by engaging psoriasis patients in prioritizing comparative effectiveness research (CER) topics. The NPF has created a novel patient-centered research platform known as Citizen Pscientist (CP), allowing patients with psoriasis and psoriatic arthritis to register and contribute their health data. The CP Governance Council administered an online 23-question CER survey to the CP community and held a structured meeting on December 3, 2016, with patients and researchers to review CER survey results and discuss patient-centered research priorities. Of the 2,945 patients surveyed, 792 patients responded. Three CER topics were deemed to be of high priority for the research agenda: 1) Treat-to-target therapy for psoriasis, 2) Psoriatic arthritis screening questionnaires for early detection and treatment of psoriatic arthritis, and 3) Comparative effectiveness of home-based phototherapy for psoriasis.
Article
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Background: Patients’ perception of disease management can influence compliance to treatment and thus affect outcome. Objective: To survey patients and physicians on their perceptions of biologic therapy for treating psoriasis in an outpatient setting. Methods: The subjective impact of intravenous treatment of severe psoriasis on patients and physicians in the outpatient setting was determined via two surveys. Results: Between September–November 2014, 24 dermatologists and 90 patients were surveyed. Treatment with biologic agents in the outpatient setting was associated with a high level of patient satisfaction: 93.3% of patients considered their psoriasis well controlled and 46.1% reported complete control. Patients highly valued the feeling of greater disease control (72.2%), regular follow-up (66.7%), and rapid improvement of psoriasis (58.9%) when attending an outpatient setting. Other positive aspects of outpatient treatment were control of other health issues and perceived improvements in quality of life. Outpatient attendance was high, with 90% of patients keeping scheduled appointments and 79.2% of physicians acknowledged that they were able to monitor their patients’ condition more closely. Conclusion: Administration of treatment in an outpatient setting may provide a feeling of improved quality of life and disease control Introduction
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Background: The prevalence of childhood psoriasis is estimated at between 0.4% and 0.7%. Clinical aspects of the diseases depend on age. The aim of this study was to investigate the clinical aspects of psoriasis according to age and sex. Patients and methods: A cross-sectional, multicentre study of children with psoriasis was performed by investigators belonging to the Research Group of the French Society of Paediatric Dermatology. The study was conducted from April 2012 to March 2013. Inclusion criteria were age less than 18 years and clinical diagnosis of psoriasis. The children were classified into 3 groups by age: infants: <2 years; children: ≥2 years and <13 years; adolescents≥13 years. The information collected included demographic data, clinical, epidemiological, and therapeutic aspects of the psoriasis, as well as analysis of comorbidities. Results: Three hundred and thirteen children were included: 27 (8.6%) infants, 207 (66.1%) children, and 79 (25.2%) adolescents. Plaque psoriasis was the most frequent clinical type of psoriasis seen in children and adolescents (>41%), but it accounted for only 25.9% of psoriasis of infants (P<0.0001). Napkin psoriasis (37.0%) and inverse psoriasis (22.2%) were the most common forms of psoriasis seen in infants and were described significantly more frequently in this group than in the two other groups (P<0.003). Nail involvement was more common in adolescents (37.2%, P=0.03) and children (32.9%) than in infants (14.8%) and affected boys more than girls (43.6% vs 22.0%, P<0.0001). Girls presented scalp psoriasis more frequently (17.7% vs 8.7%, P=0.02). Local vitamin-D treatment and systemic therapies were used more frequently in children and adolescents than in infants. There was no significant difference for treatment use, including for acitretin, according to gender. Discussion: Plaque psoriasis was the most common clinical type of psoriasis in children but affected less than 50% of the children. Age had a significant impact on extra-cutaneous skin disorders and on treatment used, while sex had little incidence. The frequency of comorbidities was not affected by age. Conclusion: Childhood psoriasis thus presents specific characteristics dependent on the age of the child. The results of studies exclusively dealing with adults cannot be extrapolated to children.
Article
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Over the past decade, biologics have become the gold standard in the treatment of moderate-to-severe psoriasis for patients who have failed or who have contraindications to traditional systemic treatments. However, although practical recommendations on how to treat a suboptimal response to biologics exist in other chronic inflammatory diseases, they are only just beginning to emerge for psoriasis. This article aims to formulate recommendations in the case of a suboptimal response of psoriasis to biologics in the Belgian setting. A Belgian taskforce of psoriasis experts was convened to review the results of a literature search and formulate recommendations based on the available evidence and provide expert opinion to address gaps in the evidence. The taskforce has proposed a treatment algorithm for patients with a primary non-response or a secondary loss of response to help address an unmet need. Expert recommendations have been developed to address treatment strategies in case of a primary or secondary suboptimal response to biologics in the treatment of moderate-to-severe psoriasis in Belgium.
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Explore the feasibility of Treat to Target in the area of psoriasis as seen in other therapeutic areas such as hypertension, hyperlipidemia, diabetes and rheumatoid arthritis. Review validated, measurable targets for psoriasis, including physician global assessment (PGA), psoriasis area and severity index (PASI) and dermatology life quality index (DLQI). Examine principles brought forth in the published European consensus on psoriasis and develop a Canadian consensus on Treat to Target in psoriasis. As PASI and DLQI are not routinely used in the community setting, we are recommending target at a PGA of zero (clear). Recommend that the target is a PGA of zero (clear) as it provides a simple and measurable result that the patient and physician can clearly understand. © 2014 Canadian Dermatology Association.
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To compare the effectiveness of biologic systemic therapy, nonbiologic systemic therapy, and phototherapy for treatment of psoriasis. A cross-sectional design was used. Ten outpatient dermatology sites across the United States participating in the Dermatology Clinical Effectiveness Research Network contributed to the study. A total of 713 patients with plaque psoriasis receiving systemic monotherapy (ie, methotrexate sodium, adalimumab, etanercept, or ustekinumab) or narrowband UV-B phototherapy. The primary outcome of the study was clear or almost clear skin on the Physician Global Assessment scale. Secondary outcomes were score on the Psoriasis Area and Severity Index, affected body surface area, and score on the Dermatology Life Quality Index. The proportion of patients with clear or almost clear ratings on the Physician Global Assessment scale differed among treatments: methotrexate (23.8%), adalimumab (47.7%), etanercept (34.2%), ustekinumab (36.1%), and narrowband UV-B (27.6%) (P < .001). In adjusted analyses, patients receiving adalimumab (relative response rate, 2.15; 95% CI, 1.60-2.90), etanercept (1.45; 1.06-1.97), and ustekinumab (1.57; 1.06-2.32) were more likely to have clear or almost clear skin vs patients receiving methotrexate. Patients receiving phototherapy showed no significant difference (1.35; 95% CI, 0.93-1.96) compared with those receiving methotrexate. No response difference was observed with respect to quality of life. Treatment doses were double the recommended doses in 36.1% of patients taking etanercept and 11.8% of those taking adalimumab;10.6% of patients undergoing phototherapy received the recommended treatment frequency. The effectiveness of psoriasis therapies in clinical practice may be lower than that reported in previous trials. Although relative differences in objective response rates among therapies may exist, absolute differences are small and may not be clinically significant. Dosing of common therapies varied from trial recommendations. These results provide novel benchmarks emphasizing the critical importance of studying effectiveness in real-world practice.
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Psoriasis is a common chronic inflammatory T-helper cell-1/17 mediated skin disease. Recent studies suggest that psoriasis, particularly if severe, may be an independent risk factor for atherosclerosis, myocardial infarction (MI), and stroke. We conducted a cohort study using the General Practice Research Database to determine if severe psoriasis patients have an increased risk of cardiovascular (CV) mortality. Severe psoriasis was defined as patients who received a psoriasis diagnosis and systemic therapy consistent with severe psoriasis (n = 3603). Up to four unexposed patients without psoriasis were selected from the same practices and start dates for each psoriasis patient (n = 14 330). For every death, the cause was determined by review of the electronic medical record. Severe psoriasis was an independent risk factor for CV mortality (HR 1.57; 95% CI 1.26, 1.96) when adjusting for age, sex, smoking, diabetes, hypertension, and hyperlipidaemia. Overall, severe psoriasis patients experienced one extra CV death per 283 patients per year, even when adjusting for major CV risk factors. The relative risk of CV mortality was modified by age. For example, the RR of CV death for a 40-year-old and 60-year-old with severe psoriasis was 2.69 (1.45, 4.99) and 1.92 (1.41, 2.62), respectively. The findings were robust to multiple sensitivity analyses. Patients with severe psoriasis have an increased risk of CV mortality that is independent of traditional CV risk factors. Additional studies are needed to determine the mechanism of this association and the impact that control of psoriasis has on CV risk.
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The Skillings-Mack statistic (Skillings and Mack, 1981, Technometrics 23: 171–177) is a general Friedman-type statistic that can be used in almost any block design with an arbitrary missing-data structure. The missing data can be either missing by design, for example, an incomplete block design, or missing completely at random. The Skillings–Mack test is equivalent to the Friedman test when there are no missing data in a balanced complete block design, and the Skillings–Mack test is equivalent to the test suggested in Durbin (1951, British Journal of Psychology, Statistical Section 4: 85–90) for a balanced incomplete block design. The Friedman test was implemented in Stata by Goldstein (1991, Stata Technical Bulletin 3: 26–27) and further developed in Goldstein (2005, Stata Journal 5: 285). This article introduces the skilmack command, which performs the Skillings–Mack test. The skilmack command is also useful when there are many ties or equal ranks (N.B. the Friedman statistic compared with the x ² distribution will give a conservative result), as well as for small samples; appropriate results can be obtained by simulating the distribution of the test statistic under the null hypothesis.
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Psoriasis is a chronic Th-1 and Th-17 inflammatory disease. Chronic inflammation has also been associated with atherosclerosis and thrombosis. The purpose of this study was to determine the risk of stroke in patients with psoriasis. We conducted a population-based cohort study of patients seen by general practitioners participating in the General Practice Research Database in the United Kingdom, 1987–2002. Mild psoriasis was defined as any patient with a diagnostic code of psoriasis, but no history of systemic therapy. Severe psoriasis was defined as any patient with a diagnostic code of psoriasis and a history of systemic therapy consistent with severe psoriasis. The unexposed (control) population was composed of patients with no history of a psoriasis diagnostic code. When adjusting for major risk factors for stroke, both mild (hazard ratio (HR) 1.06, 95% confidence interval (CI) 1.0–1.1) and severe (1.43, 95% CI 1.1–1.9) psoriasis were independent risk factors for stroke. The excess risk of stroke attributable to psoriasis in patients with mild and severe disease was 1 in 4,115 per year and 1 in 530 per year, respectively. Patients with psoriasis, particularly if severe, have an increased risk of stroke that is not explained by major stroke risk factors identified in routine medical care. JID JOURNAL CLUB ARTICLE: For questions, answers, and open discussion about this article, please go to http://network.nature.com/group/jidclub
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Traditional conceptualizations of evidence-based medicine rely heavily on randomized controlled trials. Although initiatives to broaden definitions of evidence have been advanced, they generally have not tied evidentiary criteria formally and quantitatively to the benefits and costs involved in a decision to adopt or reject an intervention. Decision analysis provides a framework for combining information to inform the adoption decision in this manner. Value-of-information analysis, a related methodology, helps to determine whether it is worthwhile to collect additional information as well as the type of research that would be most helpful.
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The coefficient of variation (V) is a statistical measure commonly used for comparing diversity in work groups. It has been employed by organizational researchers to index the relative internal variability of top-management teams, task groups, boards of directors, departments, and other social aggregates on numerous dimensions. Commenting on its widespread application, this article reviews cautions and pitfalls associated with its use for this purpose. Research implications associated with using V are also discussed.
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The Patient Protection and Affordable Care Act of 2010 created the Patient-Centered Outcomes Research Institute (PCORI) to fund and promote comparative clinical effectiveness research (CER) that will “assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis.”1 CER is not a new concept,2,3 but appreciation of its potential for providing patients and their clinicians with uniquely valuable information on what works, tailored to the clinical situation and to patient priorities, has increased rapidly in recent years.
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Reimbursement decisions are typically based on cost-effectiveness analyses. While a cost-effectiveness analysis can identify the optimum strategy, there is usually some degree of uncertainty around this decision. Sources of uncertainty include statistical sampling error in treatment efficacy measures, underlying baseline risk, utility measures and costs, as well as uncertainty in the structure of the model. The optimal strategy is therefore only optimal on average, and a decision to adopt this strategy might still be the wrong decision if all uncertainty could be eliminated. This means that there is a quantifiable expected (average) loss attaching to decisions made under uncertainty, and hence a value in collecting information to reduce that uncertainty. Value of information (VOI) analyses can be used to provide guidance on whether more research would be cost-effective, which particular model inputs (parameters) have the most bearing on decision uncertainty, and can also help with the design and sample size of further research. Here, we introduce the key concepts in VOI analyses, and highlight the inputs required to calculate it. The adoption of the new biologic treatments for RA and PsA tends to be based on placebo-controlled trials. We discuss the possible role of VOI analyses in deciding whether head-to-head comparisons of the biologic therapies should be carried out, illustrating with examples from other fields. We emphasize the need for a model of the natural history of RA and PsA, which reflects a consensus view.
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Despite increasing therapies for moderate to severe psoriasis, dermatologists' treatment preferences are unknown. We sought to assess dermatologists' preferences for first-line treatments and their selection determinants. We surveyed 1000 US dermatologists (500 National Psoriasis Foundation and 500 American Academy of Dermatology members who treat psoriasis) about their preferences for first-line treatment of moderate to severe psoriasis in healthy adults of childbearing age using standardized patient vignettes. The response rate was 39% (N = 387). Preferred therapies for male and female patients were: ultraviolet (UV) B (40% and 56%, respectively), etanercept (15% and 19%), methotrexate (16% and 4%), and adalimumab (12% and 10%). Of respondents, 66% administered phototherapy in their practice. After adjusting for all physician characteristics, those preferring first-line UVB for male or female patients were significantly more likely to have phototherapy in their practice (odds ratio [OR] 3.4, 95% confidence interval [CI] 1.8-6.6 and OR 2.8, 95% CI 1.5-5.3, respectively) and to have used UVB in more than 10 patients in the last 3 months (OR 8.0, 95% CI 3.9-16.4; OR 9.6, 95% CI 4.3-21.6). Dermatologists in the Midwest were more likely than those in the Northeast to prefer adalimumab first line for male and female patients. We surveyed only dermatologists with interest in treating psoriasis and elicited their treatment preferences for a single base case scenario. Treatment preferences may differ between survey respondents and nonrespondents. UVB is most commonly preferred as a first-line treatment for moderate to severe psoriasis in healthy adults, and preferences vary based on region, phototherapy availability, and prior treatment use.
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Value of information (VOI) techniques can provide estimates of the expected benefits from clinical research studies that can inform decisions about the design and priority of those studies. Most VOI studies use decision-analytic models to characterize the uncertainty of the effects of interventions on health outcomes, but the complexity of constructing such models can pose barriers to some practical applications of VOI. However, because some clinical studies can directly characterize uncertainty in health outcomes, it may sometimes be possible to perform VOI analysis with only minimal modeling. This article 1) develops a framework to define and classify minimal modeling approaches to VOI, 2) reviews existing VOI studies that apply minimal modeling approaches, and 3) illustrates and discusses the application of the minimal modeling to 2 new clinical applications to which the approach appears well suited because clinical trials with comprehensive outcomes provide preliminary estimates of the uncertainty in outcomes. The authors conclude that minimal modeling approaches to VOI can be readily applied in some instances to estimate the expected benefits of clinical research.
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The new national emphasis on comparative effectiveness research is likely to generate an unprecedented volume of new findings. It is essential to anticipate the obstacles that front-line health care professionals will face in translating these results into better clinical decision making. We review the current barriers to the dissemination of evidence-based clinical recommendations, including problems with continuing medical education, provider incentives, and quality assurance. We then propose solutions, including more effective educational outreach programs, requirements for practitioners to master important findings, and alignment of incentives to encourage evidence-based practice. Such strategies can lead to policies that could encourage the uptake of new comparative effectiveness data and encourage their translation into better clinical practice.
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The 2006 Canadian Dermatology Association (CDA) member survey tracked the Canadian dermatology workforce. Information on use of nondermatologist extenders, impact of financial burden on practice style, and wait times was collected in the survey. To survey Canadian dermatologists for specialty-specific physician resource information including demographics, workload, and future career plans and compare it to results from the 2001 survey. In addition, to explore three other areas not covered in the previous survey: patient access to dermatologic care through wait times, the use of nondermatologist extenders, and potential impact of educational financial debt on practice styles. CDA members in 2006 were surveyed by mail. Follow-up mailings were done for nonresponders. Survey results were compared to those of the 2001 survey. Thirty-six percent (216 of 602) of Canadian dermatologists responded (70% in 2001). The national distribution was identical between surveys. The median age increased to 55 years; two-thirds of dermatologists are male. The median retirement age remained at 65 years. There was a shift from rural to urban practice locations; 78% practice in private offices. Three-fifths of dermatologists do mainly medical dermatology, a decrease between surveys. Pediatric dermatology decreased 10%, whereas surgical dermatology increased 52% between surveys. Fewer practitioners perform noninsured services, and half as many perform research or hospital consultations or teach medical students. Financial debt burden had no impact on selection of practice style. Median wait times for nonurgent consultations doubled from 5 to 10 weeks; follow-up visits increased from 4 to 5 weeks; noninsured consultations increased from 4 to 5 weeks. The national median wait time for a third available consultation appointment was 42 days (range 7-161 days). Seventeen percent of dermatologists reported using nondermatologist extenders. Training programs produce only 60% of new practitioners needed to replace retirees over the next 5 years. Existing training programs are at full capacity, and only the creation of new programs can expand training capacity. Although the face of Canadian dermatology shows a productive specialty committed to patient care, teaching, and research, the demographics of the Canadian baby boom generation will have a major negative impact on the effectiveness of Canadian dermatology in the service of the Canadian population. The attrition rate predicted in the 2001 survey and validated by the 2006 survey spotlights the critical imperative for the specialty to adapt to the future of a shrinking workforce in the face of expanding demand for its services.
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Psoriasis is a predictor of morbidity. It is important to determine the extent to which psoriasis remains undiagnosed. To determine the prevalence of psoriasis. We conducted a cross-sectional study using the National Health and Nutrition Examination Survey 2003-2004. The prevalence of diagnosed psoriasis was 3.15% (95% confidence interval [CI], 2.18-4.53), corresponding to 5 million adults. Approximately 17% of these patients have moderate to severe psoriasis based on body surface area report and 25% rate psoriasis a large problem in everyday life. The prevalence of undiagnosed active psoriasis by conservative estimate was 0.4% (95% CI, 0.19-0.82), corresponding to approximately 600,000 US adults, and 2.28% (95% CI, 1.47-3.50) by a broader definition, corresponding to 3.6 million US adults. Undiagnosed patients had a trend toward being more likely to be male, nonwhite, less educated, and unmarried compared with patients who had received a diagnosis. The method for determining the presence of psoriasis had limited ability to detect mild disease and only fair interrater agreement. More than 5 million adults have been diagnosed with psoriasis. A large number have undiagnosed psoriasis and there are important disparities which may be associated with not receiving medical attention.
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Chronic bullous disease of childhood is the commonest acquired blistering disorder of children. Erythromycin has been reported to be beneficial for this condition. A three question survey was e-mailed to all members of the British Society for Paediatric Dermatology to assess the incidence, preferred treatments and experience of oral erythromycin in treating chronic bullous disease of childhood. A second, more detailed questionnaire was sent to members who had used erythromycin. Forty patients were reported to have been treated over the previous 2 years. The preferred treatment was dapsone. Erythromycin alone had been used in five children as first-line oral treatment. In three of these patients the initial improvement was graded as either "good" or "complete resolution." This benefit was only sustained in one child, with the other two relapsing between 4 and 12 weeks. In a further eight children, erythromycin had been used with other oral agents. In five of these children, erythromycin was associated with long-term benefit. These results suggest that erythromycin is unlikely to produce sustained improvement in chronic bullous disease of childhood when used as a sole first-line agent. However, erythromycin can cause an initial improvement, which may be useful whilst awaiting results of diagnostic tests and may confer benefit when used with other systemic treatments.
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Adjustments for making multiple comparisons in large bodies of data are recommended to avoid rejecting the null hypothesis too readily. Unfortunately, reducing the type I error for null associations increases the type II error for those associations that are not null. The theoretical basis for advocating a routine adjustment for multiple comparisons is the "universal null hypothesis" that "chance" serves as the first-order explanation for observed phenomena. This hypothesis undermines the basic premises of empirical research, which holds that nature follows regular laws that may be studied through observations. A policy of not making adjustments for multiple comparisons is preferable because it will lead to fewer errors of interpretation when the data under evaluation are not random numbers but actual observations on nature. Furthermore, scientists should not be so reluctant to explore leads that may turn out to be wrong that they penalize themselves by missing possibly important findings.
Article
To survey a random sample of primary care physicians across six European countries regarding their perceptions of diagnostic and prescribing issues in heart failure, and to consider factors that might be associated with physician under-performance. Qualitative, postal questionnaire-based, validated survey in the native tongue of a random sample of 200 primary care physicians in each of five European countries (France, Germany, Italy, The Netherlands and Spain) and of 250 U.K. primary care physicians. Respondents provided: details of practice characteristics; the usual way a diagnosis of heart failure was established; access to investigations; names of drugs prescribed in heart failure, with estimates of the proportion of patients supplied with particular classes; and physician attitudes regarding the evidence base (in terms of benefits and risks) for treatments used. Outcomes were physician perceptions and attitudes about heart failure diagnosis and treatment. Adjusted response rates varied from 17% (France) to 56% (Britain). Primary care physicians underestimate the prevalence of heart failure. Most patients are diagnosed on symptoms and signs alone, with only 32% having further investigations or referral. Although most primary care physicians stated they prescribe ACE inhibitors in heart failure, this was for only 47-62% of patients, and at doses below those identified as effective in trials. Most prescribing doctors (91%) believe there is strong evidence of reduced mortality in heart failure patients using ACE inhibitors, but 51% also consider ACE inhibitors have substantial risks with their use. Limitations of the data include the general problem of questionnaires, whether responses accord with actual clinical practice, and, specific to these data, the low response rate in some countries (although the study does provide information from nearly 300 randomly selected primary care physicians across Europe). New preliminary insights include exposition of the 'low tech' approach to heart failure diagnosis across Europe: doctors report the use of symptoms and signs alone; the lack of direct (open) access to objective investigations, such as echocardiography, which almost guarantees that misdiagnoses will occur; and the under-utilization and under-dosing with ACE inhibitors. The main factor influencing under-use would appear to be the exaggerated perceptions of treatment risk amongst primary care physicians that dominate the widespread and accurate knowledge of treatment benefits.
Article
Opinions concerning the significance of dysplastic nevi and their management vary among dermatologists. The purpose of this study was to assess how fellows of the American Academy of Dermatology (AAD) perceive and manage dysplastic nevi. Questionnaires were sent to 1216 fellows of the AAD; 456 questionnaires were returned. Almost all respondents (98%) accept the dysplastic nevus, or atypical mole, as an entity. Seventy-five percent of respondents perform follow-up total cutaneous examinations on all their patients with dysplastic nevi, and another 22% on some of them; 86% usually intend to do total removals when they perform biopsies of dysplastic nevi; 75% use margins of 2 mm or less when removing dysplastic nevi; 49% order baseline total-cutaneous photographs of some or all of their patients with multiple dysplastic nevi, although only 12% do so routinely; 67% prefer to re-excise dysplastic nevi when margins are positive, some using histologic atypia as a criterion; 60% recommend an ophthalmologic examination for at least some of their patients with many dysplastic nevi, although only 3% do so routinely; 12% always recommend cutaneous examinations of blood relatives of their patients with dysplastic nevi and another 81% recommend such examinations for at least some of their patients with dysplastic nevi; 23% use dermoscopy; 99% recommend self-examination; almost 100% recommend sunscreen use and 93%, sun avoidance. Most respondents, in agreement with the literature, accept the concept that patients with dysplastic nevi are at increased risk for melanoma and that methods for prevention and early detection of melanomas are appropriate for these patients.
Article
There is no consensus concerning management of Spitz nevi. This study was carried out to ascertain how dermatologists manage Spitz nevi. A questionnaire was sent to 997 fellows of the American Academy of Dermatology, 284 pediatric dermatologists, and 27 directors of pigmented-lesion clinics. The results are based on the 381 questionnaires returned. The vast majority of responding dermatologists (93%) recommend biopsies of suspected Spitz nevi. Of this group, 43% recommend total biopsies and 55% recommend partial biopsies; 2% would recommend either total or partial biopsies, depending on the clinical situation. Sixty-nine percent of physicians would completely excise a lesion that was histologically diagnosed as an incompletely removed Spitz nevus. Seventy percent of general dermatologists and 80% of pediatric dermatologists would recommend excision with a 1- to 2-mm margin of normal-appearing skin around a Spitz nevus. Nine percent of general dermatologists would recommend margins of 4 mm or more; however, all pediatric dermatologists surveyed would recommend margins less than 4 mm. Physicians were less likely to monitor patients whose Spitz nevi were completely removed. Three fourths (74%) of respondents believe Spitz nevi are entirely benign, 4% believe they are precursors to melanoma, and 22% are not sure. Seven percent of general dermatologists and 4% of pediatric dermatologists have seen metastatic melanomas arise at sites of lesions initially diagnosed histologically as Spitz nevi; 40% of pigmented-lesion clinic directors have seen such lesions. We believe that the lack of consensus, both in our survey and in the medical literature, reflects to some extent the lack of certainty in the histologic differentiation of Spitz nevi from melanomas and that concern about melanoma influences management. At the pigmented-lesion clinic of the New York University Skin and Cancer Unit, because of this concern about melanoma, it is usually recommended that Spitz nevi be completely excised.
Article
Based on a series of clinical trials showing no difference in the effectiveness or tolerability of most major classes of antihypertensive medications, the Joint National Commission on High Blood Pressure Treatment recommends that physicians prescribe beta-blockers or diuretics as initial hypertensive therapy unless there are compelling indications for another type of medication. Nevertheless, many physicians continue to favor more expensive medications like angiotensin-converting enzyme (ACE) inhibitors and calcium channel blockers as first line agents. The persistent use of these agents raises questions as to whether physicians perceive ACE inhibitors and calcium channel blockers to be better than beta-blockers and diuretics. We surveyed 1,200 primary care physicians in 1997, and another 500 primary care physicians in 2000, and asked them to estimate the relative effectiveness and side effects of 4 classes of medication in treating a hypothetical patient with uncomplicated hypertension: ACE inhibitors, beta-blockers, calcium channel blockers, and diuretics. In addition, we asked them to indicate whether they ever provided free samples of hypertension medications to their patients. Perceptions of the relative effectiveness and side effects of the 4 classes of hypertension medications did not significantly change over the 3 years, nor did prescription recommendations. Physicians perceive that diuretics are less effective at lowering blood pressure than the other 3 classes (P <.001). They also perceive that beta-blockers are less tolerated than the other 3 classes (P <.001). In a multivariate model, perceptions of effectiveness and tolerability displayed significant associations with prescription preference independent of background variables. The only other variable to contribute significantly to the model was provision of free medication samples to patients. Despite numerous clinical trials showing no difference in the effectiveness or side-effect profiles of these 4 classes of drugs, most physicians believed that diuretics were less effective and beta-blockers were less tolerated than other medications. Moreover, their prescription practices were associated with their provision of free samples provided by pharmaceutical representatives, even after adjusting for other demographic characteristics. Efforts to increase physicians' prescribing of beta-blockers and diuretics may need to be directed at overcoming misunderstandings about the effectiveness and tolerability of these medicines.
Article
The impact of psoriasis on quality of life has been studied in select patient populations. Population-based data detailing the distribution of extent of disease, associated problems in everyday life, and treatment satisfaction for the US population have been lacking. Our population-based survey indicates that approximately 4.5 million adults have been diagnosed as having psoriasis. Most (59%) have little or no involvement, but 650,000 adults have at least three palms of body surface involved and more than 1,000,000 indicate substantial dissatisfaction with their treatment. Only 5% of patients (56,000) who report severe dissatisfaction with current therapy have extensive disease (10 palms). Many individuals with little psoriasis at the time of interview considered the disease to be a large problem in everyday life.
Article
Psoriasis is a common chronic, recurrent, immune mediated disease of the skin and joints. It can have a significant negative impact on the physical, emotional, and, psychosocial wellbeing of affected patients. Psoriasis is found worldwide but the prevalence varies among different ethnic groups. It has a strong genetic component but environmental factors such as infections can play an important role in the presentation of disease. There are several clinical cutaneous manifestations of psoriasis but most commonly the disease presents as chronic, symmetrical, erythematous, scaling papules and plaques. The epidemiology, clinical features, and impact on quality of life of psoriasis are reviewed.
Article
There is a perception among many academic dermatologists that departments of dermatology face severe challenges with recruitment and retention of faculty. In an era when evidence points to a shortage of dermatologists and residency graduates have plentiful private practice offers in almost every geographic area, some fear that academic programs will face even steeper challenges attracting and keeping enough dermatologists on staff. To compare the practice patterns of academic dermatologists with those of the dermatology workforce in other settings, data from the American Academy of Dermatology 2002 Practice Profile Survey were analyzed (1425 respondents, 35% response rate). The mean age of academic dermatologists (45.6 years) was younger than that of those in other practice settings (51.9 years solo practice, 49.0 years multispecialty group), and older age cohorts were significantly less likely to be working in academics (P < .001). Academic physicians were much more likely than those in solo practice or dermatology-only groups (62.2% vs 18.3%-39.4%) to report that their institution or practice was seeking new dermatologists. The average waiting time for new patient appointments varied from a low of 31.1 days in solo practices to a high of 55.9 days in academic practices. Academic dermatologists saw 32% to 41% fewer patients per week, but spent much more time (24.1 vs 5.5-8.6 h/wk) participating in research, hospital consults, medical writing, administrative activities, and teaching than dermatologists in any other setting. Academic dermatologists reflected a relatively small proportion of survey respondents, and may not be representative of the nation's dermatology faculty (although the percentage of academics in the survey was similar to that in the overall workforce). Possible response biases could also have affected the survey results. The survey results identify a number of differences between the practice patterns of academic dermatologists and their colleagues in other settings, and suggest that academic departments of dermatology may be facing unique workforce challenges.
Article
To provide guidelines for identifying composite hypotheses and addressing the probability of false rejection for multiple hypotheses. Examples from the literature in health services research are used to motivate the discussion of composite hypothesis tests and multiple hypotheses. This article is a didactic presentation. It is not rare to find mistaken inferences in health services research because of inattention to appropriate hypothesis generation and multiple hypotheses testing. Guidelines are presented to help researchers identify composite hypotheses and set significance levels to account for multiple tests. It is important for the quality of scholarship that inferences are valid: properly identifying composite hypotheses and accounting for multiple tests provides some assurance in this regard.
Article
Psoriasis is the most common T-helper cell type 1 (T(H)1) immunological disease. Evidence has linked T(H)1 diseases to myocardial infarction (MI). Psoriasis has been associated with cardiovascular diseases, but has only been investigated in hospital-based studies that did not control for major cardiovascular risk factors. To determine if within a population-based cohort psoriasis is an independent risk factor for MI when controlling for major cardiovascular risk factors. A prospective, population-based cohort study in the United Kingdom of patients with psoriasis aged 20 to 90 years, comparing outcomes among patients with and without a diagnosis of psoriasis. Data were collected by general practitioners as part of the patient's medical record and stored in the General Practice Research Database between 1987 and 2002, with a mean follow-up of 5.4 years. Adjustments were made for hypertension, diabetes, history of myocardial infarction, hyperlipidemia, age, sex, smoking, and body mass index. Patients with psoriasis were classified as severe if they ever received a systemic therapy. Up to 5 controls without psoriasis were randomly selected from the same practices and start dates as the patients with psoriasis. A total of 556,995 control patients and patients with mild (n = 127,139) and severe psoriasis (n = 3837) were identified. Incident MI. There were 11,194 MIs (2.0%) within the control population and 2319 (1.8%) and 112 (2.9%) MIs within the mild and severe psoriasis groups, respectively. The incidences per 1000 person-years for control patients and patients with mild and severe psoriasis were 3.58 (95% confidence interval [CI], 3.52-3.65), 4.04 (95% CI, 3.88-4.21), and 5.13 (95% CI, 4.22-6.17), respectively. Patients with psoriasis had an increased adjusted relative risk (RR) for MI that varied by age. For example, for a 30-year-old patient with mild or severe psoriasis, the adjusted RR of having an MI is 1.29 (95% CI, 1.14-1.46) and 3.10 (95% CI, 1.98-4.86), respectively. For a 60-year-old patient with mild or severe psoriasis, the adjusted RR of having an MI is 1.08 (95% CI, 1.03-1.13) and 1.36 (95% CI, 1.13-1.64), respectively. Psoriasis may confer an independent risk of MI. The RR was greatest in young patients with severe psoriasis.
The psoriasis and psoriatic arthritis pocket guide: treatment algorithms and management options. Portland (OR): National Psoriasis Foundation
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Van Voorhees AS, Feldman SR, Koo JY, Lebwohl MG, Menter A. The psoriasis and psoriatic arthritis pocket guide: treatment algorithms and management options. Portland (OR): National Psoriasis Foundation; 2009.
The psoriasis and psoriatic arthritis pocket guide: treatment algorithms and management options
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Van Voorhees, AS.; Feldman, SR.; Koo, JY.; Lebwohl, MG.; Menter, A. The psoriasis and psoriatic arthritis pocket guide: treatment algorithms and management options. Portland, OR: National Psoriasis Foundation; 2009.
Medical decision making : an international journal of the Society for Medical Decision Making
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Meltzer DO, Hoomans T, Chung JW, Basu A. Minimal modeling approaches to value of information analysis for health research. Medical decision making : an international journal of the Society for Medical Decision Making. 2011; 31:E1-E22. [PubMed: 21712493]
Dermatologist preferences for first-line therapy of moderate to severe psoriasis in healthy adult patients
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Wan J, Abuabara K, Troxel AB, Shin DB, Van Voorhees AS, Bebo BF Jr, et al. Dermatologist preferences for first-line therapy of moderate to severe psoriasis in healthy adult patients. Journal of the American Academy of Dermatology. 2012; 66:376-86. [PubMed: 21856040]
The amazing vanishing Canadian dermatologist: results from the
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test for comparisons between 2 groups, Kruskal-Wallis test for comparisons among 3 or more groups 2 Spearman correlation coefficient and
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Mann-Whitney test for comparisons between 2 groups, Kruskal-Wallis test for comparisons among 3 or more groups 2 Spearman correlation coefficient and p-value 3