130 research outputs found
Toward an international consensus-Integrating lipoprotein apheresis and new lipid-lowering drugs
Background: Despite advances in pharmacotherapy of lipid disorders, many dyslipidemic patients do not attain sufficient lipid lowering to mitigate risk of atherosclerotic cardiovascular disease. Several classes of novel lipid-lowering agents are being evaluated to reduce atherosclerotic cardiovascular disease risk. Lipoprotein apheresis (LA) is effective in acutely lowering the plasma concentrations of atherogenic lipoproteins including low-density lipoprotein cholesterol and lipoprotein(a), and novel lipid-lowering drugs may dampen the lipid rebound effect of LA, with the possibility that LA frequency may be decreased, in some cases even be discontinued. Sources of material: This document builds on current American Society for Apheresis guidelines and, for the first time, makes recommendations from summarized data of the emerging lipid-lowering drug classes (inhibitors of proprotein convertase subtilisin/kexin type 9 or microsomal triglyceride transfer protein, high-density lipoprotein mimetic), including the available evidence on combination therapy with LA with respect to the management of patients with dyslipidemia. Abstract of findings: Recommendations for different indications are given based on the latest evidence. However, except for lomitapide in homozygous familial hypercholesterolemia and alirocumab/evolocumab in heterozygous familial hypercholesterolemia subjects, limited data are available on the effectiveness and safety of combination therapy. More studies on combining LA with novel lipid-lowering drugs are needed. Conclusion: Novel lipid-lowering agents have potential to improve the performance of LA, but more evidence is needed. The Multidisciplinary International Group for Hemapheresis TherapY and Metabolic DIsturbances Contrast scientific society aims to establish an international registry of clinical experience on LA combination therapy to expand the evidence on this treatment in individuals at high cardiovascular disease risk
Can we improve outcome of congenital diaphragmatic hernia?
This review gives an overview of the disease spectrum of congenital diaphragmatic hernia (CDH). Etiological factors, prenatal predictors of survival, new treatment strategies and long-term morbidity are described. Early recognition of problems and improvement of treatment strategies in CDH patients may increase survival and prevent secondary morbidity. Multidisciplinary healthcare is necessary to improve healthcare for CDH patients. Absence of international therapy guidelines, lack of evidence of many therapeutic modalities and the relative low number of CDH patients calls for cooperation between centers with an expertise in the treatment of CDH patients. The international CDH Euro-Consortium is an example of such a collaborative network, which enhances exchange of knowledge, future research and development of treatment protocols
Reducing the Clinical and Public Health Burden of Familial Hypercholesterolemia A Global Call to Action
Q1Q1Artículo completoE1-E13IMPORTANCE Familial hypercholesterolemia (FH) is an underdiagnosed and undertreated
genetic disorder that leads to premature morbidity and mortality due to atherosclerotic
cardiovascular disease. Familial hypercholesterolemia affects 1 in 200 to 250 people around
the world of every race and ethnicity. The lack of general awareness of FH among the public
and medical community has resulted in only 10% of the FH population being diagnosed and
adequately treated. The World Health Organization recognized FH as a public health priority
in 1998 during a consultation meeting in Geneva, Switzerland. The World Health Organization
report highlighted 11 recommendations to address FH worldwide, from diagnosis and
treatment to family screening and education. Research since the 1998 report has increased
understanding and awareness of FH, particularly in specialty areas, such as cardiology and
lipidology. However, in the past 20 years, there has been little progress in implementing the
11 recommendations to prevent premature atherosclerotic cardiovascular disease in an entire
generation of families with FH.
OBSERVATIONS In 2018, the Familial Hypercholesterolemia Foundation and the World Heart
Federation convened the international FH community to update the 11 recommendations.
Two meetings were held: one at the 2018 FH Foundation Global Summit and the other during
the 2018 World Congress of Cardiology and Cardiovascular Health. Each meeting served as
a platform for the FH community to examine the original recommendations, assess the gaps,
and provide commentary on the revised recommendations. The Global Call to Action on
Familial Hypercholesterolemia thus represents individuals with FH, advocacy leaders,
scientific experts, policy makers, and the original authors of the 1998 World Health
Organization report. Attendees from 40 countries brought perspectives on FH from low-,
middle-, and high-income regions. Tables listing country-specific government support for
FH care, existing country-specific and international FH scientific statements and guidelines,
country-specific and international FH registries, and known FH advocacy organizations
around the world were created.
CONCLUSIONS AND RELEVANCE By adopting the 9 updated public policy recommendations
created for this document, covering awareness; advocacy; screening, testing, and diagnosis;
treatment; family-based care; registries; research; and cost and value, individual countries
have the opportunity to prevent atherosclerotic heart disease in their citizens carrying a gene
associated with FH and, likely, all those with severe hypercholesterolemia as well
Recovering a probabilistic knowledge structure by constraining its parameter space
In the Basic Local Independence Model (BLIM) of Doignon and Falmagne (Knowledge Spaces, Springer, Berlin, 1999), the probabilistic relationship between the latent knowledge states and the observ- able response patterns is established by the introduction of a pair of parameters for each of the problems: a lucky guess probability and a careless error probability. In estimating the parameters of the BLIM with an empirical data set, it is desirable that such probabilities remain reasonably small. A special case of the BLIM is proposed where the parameter space of such probabilities is constrained. A simulation study shows that the constrained BLIM is more effective than the unconstrained one, in recovering a probabilistic knowledge structure
A logistic approach to knowledge structures
Models for quantitative (or numerical) testing like e.g. educational testing have a relatively long tradition in psychology, while the qualitative (or nonnumerical) approach to psychometrics is more recent. The approach presented in this paper can be regarded as an attempt to integrate, to some extent, the numerical and nonnumerical fields. In numerical testing a subject is characterized by some real-valued parameter representing her level or ability. In the nonnumerical approach the knowledge state of an individual is represented by the subset of problems that the individual is capable of solving. We propose a model in which the relationship between the ability levels and the knowledge states is worked out on a probabilistic basis. The central idea is that the ability parameters and the knowledge states are not independent. A logistic model is derived which specifies the probabilities of the knowledge states conditional on the ability levels. We show that the Rasch model arises as a special case of the proposed model
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