Summary consensus statement of the PEK review board regarding the PEK process and the PEK products.
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Neurofibromatosis 1 (NF1) is an autosomal dominant tumor predisposition syndrome in which affected individuals have a greatly increased risk of developing malignant peripheral nerve sheath tumors (MPNSTs). These cancers are difficult to detect and have a poor prognosis. Because patients may present to specialists from widely differing disciplines, the association with NF1 is often not appreciated, and there is no cohesive pattern of care. A multidisciplinary group of 33 clinicians and scientists with specialist knowledge in MPNST and NF1 reviewed the current published and unpublished data in this field, and distilled their collective experience to produce a consensus summary on MPNST in NF1. The known clinical, pathological, and genetic information on MPNST in NF1was collated, and a database was established to record information in a uniform manner. Subgroups with a higher risk of developing MPNSTwere identified within the NF1 population. The consortium formulated proposals and guidelines for clinical and pathological diagnosis, surgical management, and medical treatment of MPNST in individuals with NF1.A multidisciplinary team approach to the management of this complex disorder is advocated. Progress can be made by adopting the guidelines proposed by this consortium and by widespread dissemination of standardized information. Collaborative research should be promoted with the aim of harnessing advances in molecular genetics to develop targeted therapies for MPNST in people with NF1.
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We developed a short-length document that clearly delineates a prudent approach to and criteria for reimbursement of positive airway pressure (PAP) costs for the treatment of obstructive sleep apnea (OSA). Treatment modalities for OSA with PAP include continuous positive airway pressure, bilevel or variable PAP, and autotitrating PAP. This guidance on the appropriate criteria for PAP use in OSA is based on widely acknowledged peer-reviewed studies and widely accepted clinical practice. These criteria reflect current opinion on the appropriate clinical management of OSA in lieu of data pending from the Sleep Heart Health Study and upcoming outcome studies. This document is not intended to provide a complete review and analysis of the OSA clinical literature. The key to the success of this document is to foster consensus within and outside the clinical sleep community by providing a common sense and easily understood approach to the treatment of OSA with PAP.
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OBJECTIVE: To estimate the risk and prevalence of Mycobacterium tuberculosis (MTB) infection and tuberculosis (TB) incidence, prevalence, and mortality, including disease attributable to human immunodeficiency virus (HIV), for 212 countries in 1997. PARTICIPANTS: A panel of 86 TB experts and epidemiologists from more than 40 countries was chosen by the World Health Organization (WHO), with final agreement being reached between country experts and WHO staff. EVIDENCE: Incidence of TB and mortality in each country was determined by (1) case notification to the WHO, (2) annual risk of infection data from tuberculin surveys, and (3) data on prevalence of smear-positive pulmonary disease from prevalence surveys. Estimates derived from relatively poor data were strongly influenced by panel member opinion. Objective estimates were derived from high-quality data collected recently by approved procedures. CONSENSUS PROCESS: Agreement was reached by (1) participants reviewing methods and data and making provisional estimates in closed workshops held at WHO's 6 regional offices, (2) principal authors refining estimates using standard methods and all available data, and (3) country experts reviewing and adjusting these estimates and reaching final agreement with WHO staff. CONCLUSIONS: In 1997, new cases of TB totaled an estimated 7.96 million (range, 6.3 million-11.1 million), including 3.52 million (2.8 million-4.9 million) cases (44%) of infectious pulmonary disease (smear-positive), and there were 16.2 million (12.1 million-22.5 million) existing cases of disease. An estimated 1.87 million (1.4 million-2.8 million) people died of TB and the global case fatality rate was 23% but exceeded 50% in some African countries with high HIV rates. Global prevalence of MTB infection was 32% (1.86 billion people). Eighty percent of all incident TB cases were found in 22 countries, with more than half the cases occurring in 5 Southeast Asian countries. Nine of 10 countries with the highest incidence rates per capita were in Africa. Prevalence of MTB/HIV coinfection worldwide was 0.18% and 640000 incident TB cases (8%) had HIV infection. The global burden of tuberculosis remains enormous, mainly because of poor control in Southeast Asia, sub-Saharan Africa, and eastern Europe, and because of high rates of M tuberculosis and HIV coinfection in some African countries.
BACKGROUND AND PURPOSE: To develop recommendations for the establishment of comprehensive stroke centers capable of delivering the full spectrum of care to seriously ill patients with stroke and cerebrovascular disease. Recommendations were developed by members of the Brain Attack Coalition (BAC), which is a multidisciplinary group of members from major professional organizations involved with the care of patients with stroke and cerebrovascular disease. SUMMARY OF REVIEW: A comprehensive literature search was conducted from 1966 through December 2004 using Medline and Pub Med. Articles with information about clinical trials, meta-analyses, care guidelines, scientific guidelines, and other relevant clinical and research reports were examined and graded using established evidence-based medicine approaches for therapeutic and diagnostic modalities. Evidence was also obtained from a questionnaire survey sent to leaders in cerebrovascular disease. Members of BAC reviewed literature related to their field and graded the scientific evidence on the various diagnostic and treatment modalities for stroke. Input was obtained from the organizations represented by BAC. BAC met on several occasions to review each specific recommendation and reach a consensus about its importance in light of other medical, logistical, and financial factors. CONCLUSIONS: There are a number of key areas supported by evidence-based medicine that are important for a comprehensive stroke center and its ability to deliver the wide variety of specialized care needed by patients with serious cerebrovascular disease. These areas include: (1) health care personnel with specific expertise in a number of disciplines, including neurosurgery and vascular neurology; (2) advanced neuroimaging capabilities such as MRI and various types of cerebral angiography; (3) surgical and endovascular techniques, including clipping and coiling of intracranial aneurysms, carotid endarterectomy, and intra-arterial thrombolytic therapy; and (4) other specific infrastructure and programmatic elements such as an intensive care unit and a stroke registry. Integration of these elements into a coordinated hospital-based program or system is likely to improve outcomes of patients with strokes and complex cerebrovascular disease who require the services of a comprehensive stroke center.
International declarations that articulate core values, goals and standards have played an important role in enhancing the quality of care in a number of areas of medicine. This document attempts this task for early intervention in psychotic disorders. It was originally inspired by the St Vincents declaration on the care of diabetes and carefully developed by David Shiers and Jo Smith with support from the Initiative to Reduce the Impact of Schizophrenia, National Institute for Mental Health in England and Rethink, resulting in the UK-focused Newcastle Declaration. The World Health Organization and the International Early Psychosis Association then collaborated to produce an international version of the declaration, which articulates the universal principles of early intervention and tries to blend these with local capacities and cultural diversity.
During a special session, the faculty identified several specific areas related to the role of dopamine and norepinephrine in depression and antidepressant treatment that either warrant the clinician's attention or are in need of more research. Areas of interest include fatigue and lethargy in depression, treatment strategies for treatment-resistant depression, the somatic presentation of depression, neurobiology of fatigue and its role in determining treatment, symptom rating scales, and sexual side effects. In addition, the faculty discussed the importance of patient psychoeducation and self-management as well as the ways in which disease models of depression affect treatment.
The main recommendations for the use of ciclosporin in the management of psoriasis are: (i) intermittent short courses (average of 12 weeks duration) of ciclosporin are preferable; (ii) ciclosporin should be given in the dose range 2.5-5.0 mg kg(-1) day(-1) (doses greater than 5.0 mg kg(-1) day(-1) should only be given in exceptional circumstances); (iii) treatment regimens should be tailored to the needs of each patient; (iv) selection of patients should take into account psychosocial disability, as well as clinical extent of disease and failure of previous treatment; (v) each patient's renal function (as measured by serum creatinine) should be thoroughly assessed before and during treatment; (vi) each patient's blood pressure should be carefully monitored before and during treatment; (vii) adherence to treatment guidelines substantially reduces the risk of adverse events; (viii) long-term continuous ciclosporin therapy may be appropriate in a subgroup of patients; however, duration of treatment should be kept below 2 years whenever possible; and (ix) when long-term continuous ciclosporin therapy is necessary, annual evaluation of glomerular filtration rate may be useful to accurately monitor renal function.
STUDY DESIGN: Systematic review. OBJECTIVES: To review the various radiographic parameters currently used to assess traumatic thoracolumbar injuries, emphasizing the validity and technique behind each one, to formulate evidence-based guidelines for a standardized radiographic method of assessment of these fractures. SUMMARY OF BACKGROUND DATA: The treatment of thoracolumbar fractures is guided by various radiographic measurement parameters. Unfortunately, for each group of parameters, there has usually been more than 1 proposed measurement technique, thus creating confusion when gathering data and reporting outcomes. Ultimately, this effect results in clinical decisions being based on nonstandardized, nonvalidated outcome measures. METHODS: Computerized bibliographic databases were searched up to January 2004 using key words and Medical Subject Headings on thoracolumbar spine trauma, radiographic parameters, and methodologic terms. Using strict inclusion criteria, 2 independent reviewers conducted study selection, data abstraction, and methodologic quality assessment. RESULTS: There were 18 original articles that ultimately constituted the basis for the review. Of radiographic measurement parameters, 3 major groups were identified, depicting the properties of the injured spinal column: sagittal alignment, vertebral body compression, and spinal canal dimensions, with 14 radiographic parameters reported to assess these properties. CONCLUSIONS: Based on a systematic review of the literature and expert opinion from an experienced group of spine trauma surgeons, it is recommended that the following radiographic parameters should be used routinely to assess thoracolumbar fractures: the Cobb angle, to assess sagittal alignment; vertebral body translation percentage, to express traumatic anterolisthesis; anterior vertebral body compression percentage, to assess vertebral body compression, the sagittal-to-transverse canal diameter ratio, and canal total cross-sectional area (measured or calculated); and the percent canal occlusion, to assess canal dimensions.
STUDY DESIGN: Literature review. OBJECTIVES: Members of the Spine Trauma Study Group evaluated various imaging methods and compiled standardized approaches to making radiographic measurements for the assessment of sacral fractures. SUMMARY OF BACKGROUND DATA: The diagnosis of sacral fractures is frequently missed or delayed, and the treatment is controversial, with significant variations in recommendations regarding nonoperative treatment, neural decompression, and internal fixation. A paucity of specific radiographic measurements and criteria exist to aid the clinician in making sound treatment decisions. This may obligate surgeons to base clinical decisions on nonstandardized, nonvalidated recommendations and measures and, in some cases, anecdotal evidence. We think that a critical first step toward valid and optimal clinical decision-making requires an accurate and clear definition of imaging measurement parameters used to evaluate sacral injuries and gather data for future studies. METHODS: A review of the English-speaking literature was performed to assess cited radiographic measurement techniques of sacral fractures currently in use. This allowed the formulation of detailed radiographic assessments designed to more reliably describe sacral fracture morphometry. RESULTS: These measurements include: anterior-posterior sacral fracture displacement (axial computed tomography [CT] of the pelvis), vertical sacral fracture displacement (coronal CT reconstruction), anterior-posterior translation and kyphotic angulation (sagittal CT reconstructions), and degree of central canal involvement and foraminal encroachment (axial CT with fine cuts and coronal and sagittal reconstructions). Other radiographic factors that should be considered that may impact treatment outcomes include the level and type of sacral fracture, lumbosacral junction and sacroiliac joint involvement, and associated pelvic ring injury. CONCLUSIONS: With adoption of these radiographic guidelines, future studies will have a uniform method in which to describe sacral injuries and therefore allow study of the efficacy of various recommended treatment protocols.
UNLABELLED: Gaucher disease is caused by defective activity of glucocerebrosidase. The resulting accumulation of glucocerebroside in the lysosomes of visceral macrophages in various tissue and organ compartments leads to multiple manifestations, including hepatosplenomegaly, anemia, thrombocytopenia, growth retardation and skeletal disease. The most prevalent form of Gaucher disease is the non-neuronopathic (type 1) variant, which lacks primary involvement of the central nervous system. Traditionally, this has been referred to as the 'adult type'; however, 66% of individuals with symptomatic non-neuronopathic Gaucher disease manifest in childhood. Onset in childhood is usually predictive of a severe, rapidly progressive phenotype and children with non-neuronopathic Gaucher disease are at high risk for morbid complications. Enzyme therapy with recombinant human glucocerebrosidase in childhood can restore health in reversible manifestations and prevent the development of irreversible symptoms. A heightened focus on pediatric Gaucher disease is therefore needed. Although some correlation has been found between genotype and phenotype, mutation analysis is of limited value in disease prognosis. Management of pediatric Gaucher disease should be underpinned by a thorough assessment of the phenotype at baseline with regular monitoring thereafter. Excluding neuronopathic disease is recommended as the first step. Subsequently, baseline evaluation should focus on staging of different storage tissues, particularly the bone the involvement of which results in the greatest long-term morbidity. These organ assessments are recommended because bone disease severity may not correlate with disease severity in other organs and vice versa. In addition, different organs may respond differently to therapy. Initial assessment of each organ system can enable setting of realistic and individualized goals. CONCLUSION: A thorough approach to baseline assessment will improve the understanding of childhood Gaucher disease, optimizing management to minimize impairment of growth and development and prevent irreversible symptoms.
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