130 research outputs found
Paediatric non-neuronopathic Gaucherdisease: recommendations for treatment and monitoring
No full textIn individuals with non-neuronopathic Gaucher disease, childhood manifestations are usually predictive of a more severe phenotype. Although children with Gaucher disease are at risk of irreversible disease complications, early intervention with an optimal dose of enzyme therapy can prevent the development of complications and ensure adequate, potentially normal, development through childhood and adolescence. Very few, if any, children diagnosed by signs and symptoms should go untreated. Evidence suggests that disease severity, disease progression and treatment response in different organs where glucocerebroside accumulates are often non-uniform in affected individuals. Therefore, serial monitoring of the affected compartments is important. This should include a thorough physical examination at 6- to 12-monthly intervals. Neurological assessment should be performed to rule out neurological involvement and should be undertaken periodically thereafter in children who are considered to have risk factors for developing neuronopathic disease. Haematological and biochemical markers, such as haemoglobin, platelet counts and chitotriosidase levels, should be assessed every 3 months initially, but when clinical goals have been met through treatment with enzyme therapy, the frequency can be reduced to every 12 to 24 months. Careful monitoring of bone disease is vitally important, as the resulting sequelae are associated with the greatest level of morbidity. By combining various imaging modalities, the skeletal complications of non-neuronopathic Gaucher disease can be effectively monitored so that irreversible skeletal pathology is avoided and pain due to bone involvement is diminished or eliminated. Monitoring must include regular psychosocial, functional status and quality-of-life evaluation, as well as consistent assessment of therapeutic goal attainment and necessary dosage adjustments based on the patient's progress. CONCLUSION: Through comprehensive and serial monitoring, ultimately, a therapeutic dose of enzyme therapy that achieves sustained benefits can be found for each child with non-neuronpathic Gaucher disease
Pediatric non-neuronopathic Gaucher disease: presentation, diagnosis and assessment. Consensus statements
No full textIn individuals with non-neuronopathic Gaucher disease, childhood manifestations are usually predictive of a more severe phenotype. Although children with Gaucher disease are at risk of irreversible disease complications, early intervention with an optimal dose of enzyme therapy can prevent the development of complications and ensure adequate, potentially normal, development through childhood and adolescence. Very few, if any, children diagnosed by signs and symptoms should go untreated. Evidence suggests that disease severity, disease progression and treatment response in different organs where glucocerebroside accumulates are often non-uniform in affected individuals. Therefore, serial monitoring of the affected compartments is important. This should include a thorough physical examination at 6- to 12-monthly intervals. Neurological assessment should be performed to rule out neurological involvement and should be undertaken periodically thereafter in children who are considered to have risk factors for developing neuronopathic disease. Haematological and biochemical markers, such as haemoglobin, platelet counts and chitotriosidase levels, should be assessed every 3 months initially, but when clinical goals have been met through treatment with enzyme therapy, the frequency can be reduced to every 12 to 24 months. Careful monitoring of bone disease is vitally important, as the resulting sequelae are associated with the greatest level of morbidity. By combining various imaging modalities, the skeletal complications of non-neuronopathic Gaucher disease can be effectively monitored so that irreversible skeletal pathology is avoided and pain due to bone involvement is diminished or eliminated. Monitoring must include regular psychosocial, functional status and quality-of-life evaluation, as well as consistent assessment of therapeutic goal attainment and necessary dosage adjustments based on the patient's progress. CONCLUSION: Through comprehensive and serial monitoring, ultimately, a therapeutic dose of enzyme therapy that achieves sustained benefits can be found for each child with non-neuronpathic Gaucher disease
Diagnostic evaluation, monitoring, and perioperative management of spinal cord compression in patients with Morquio syndrome.
No full textThe Comprehensibility of Plain Language and Non Plain Language Minnesota Civil Jury Instructions
No full textA substantial volume of research (e.g., Charrow & Charrow, 1979; Elwork, Sales & Alfini, 1977, 1982) suggests that jurors do not understand the often-convoluted language of standard jury instructions. Some states have recently simplified their instructions, but others continue to debate whether change is beneficial. This study was designed to investigate whether plain language jury instructions lead to improved comprehension. College students listened to either new, plain language Minnesota jury instructions or older Minnesota jury instructions addressing the same topics. Participants then took a written comprehension test covering legal rules contained in the instructions. Participants also completed a Nelson-Denny vocabulary test (Brown, Bennet, & Hanna, 1981) and provided demographic information. No significant treatment group differences were found for overall comprehension scores. Vocabulary scores were significantly correlated with comprehension scores for both groups of subjects. The results suggest that a juror\u27s verbal proficiency is more important in predicting comprehension of jury instructions than the language style of the instructions. Also, response patterns for some items suggest that people often maintain preconceived notions of legal rules despite clear instruction to the contrary
Organic indoor location : infrastructure and applications
No full textThesis (M. Eng.)--Massachusetts Institute of Technology, Dept. of Electrical Engineering and Computer Science, 2010.This electronic version was submitted by the student author. The certified thesis is available in the Institute Archives and Special Collections.Cataloged from student-submitted PDF version of thesis.Includes bibliographical references (p. 55-57).We describe OIL, a system that uses the existing wireless infrastructure of a building to enable a mobile device to discover its indoor location. One of the main goals behind OIL is to enable non-expert users to contribute the data that is required for localization. Toward this we have developed (1) a server-client architecture for aggregating and distributing data; (2) a caching scheme that enables client devices to estimate indoor location; (3) a simple user interface for contributing data; and (4) a way to indicate how uncertain localization estimates are. We evaluate our system with a nine-day, nineteen-person user study that took place on campus as well as a deployment of the system at an off-campus long-term specialized care facility. We also describe how to use a person's indoor location trace (i.e. the rooms they had visited and the times of each visit) to build a content-based recommendation system for academic seminars. Such a system would learn about a user's preferences implicitly, placing no burden on the user. We evaluate a prototype recommendation system based on data gathered from a user study in which participants ranked seminars.by Benjamin W. Charrow.M.Eng
Documented Transient Third-Degree Atrioventricular Block and Asystole in a Child with Familial Dysautonomia
No full text
How many deaths are attributable to smoking in the United States? Comparison of methods for estimating smoking-attributable mortality when smoking prevalence changes
No full textBackground: The number of smoking-attributable deaths is commonly estimated using current and former smoking prevalences or lung cancer mortality as an indirect metric of cumulative population smoking. Neither method accounts for differences in the timing with which relative risks (RRs) for different diseases change following smoking initiation and cessation. We aimed to develop a method to account for time-dependent RRs. Methods: We used birth cohort lung cancer mortality and its change over time to characterize time-varying cumulative smoking exposure. We analyzed data from the American Cancer Society Cancer Prevention Study II to estimate RRs for disease-specific mortality associated with current and former smoking, and change in RRs over time after cessation. Results: When lung cancer was used to measure cumulative smoking exposure, 254,700 male and 227,000 female deaths were attributed to smoking in the US in 2005. A modified method in which RRs for different diseases decreased at different rates after cessation yielded similar but slightly lower estimates [251,900 (male) and 221,100 (female)]. The lowest estimates resulted from the method based on smoking prevalence [225,800 (male) and 163,700 (female)]. Conclusions: Although all methods estimated a large number of smoking attributable deaths, future efforts should account for temporal changes in smoking prevalence and in accumulation/reversibility of disease-specific risks
Correlates of Complete Childhood Vaccination in East African Countries.
No full textDespite the benefits of childhood vaccinations, vaccination rates in low-income countries (LICs) vary widely. Increasing coverage of vaccines to 90% in the poorest countries over the next 10 years has been estimated to prevent 426 million cases of illness and avert nearly 6.4 million childhood deaths worldwide. Consequently, we sought to provide a comprehensive examination of contemporary vaccination patterns in East Africa and to identify common and country-specific barriers to complete childhood vaccination. Using data from the Demographic and Health Surveys (DHS) for Burundi, Ethiopia, Kenya, Rwanda, Tanzania, and Uganda, we looked at the prevalence of complete vaccination for polio, measles, Bacillus Calmette-Guérin (BCG) and DTwPHibHep (DTP) as recommended by the WHO among children ages 12 to 23 months. We conducted multivariable logistic regression within each country to estimate associations between complete vaccination status and health care access and sociodemographic variables using backwards stepwise regression. Vaccination varied significantly by country. In all countries, the majority of children received at least one dose of a WHO recommended vaccine; however, in Ethiopia, Tanzania, and Uganda less than 50% of children received a complete schedule of recommended vaccines. Being delivered in a public or private institution compared with being delivered at home was associated with increased odds of complete vaccination status. Sociodemographic covariates were not consistently associated with complete vaccination status across countries. Although no consistent set of predictors accounted for complete vaccination status, we observed differences based on region and the location of delivery. These differences point to the need to examine the historical, political, and economic context of each country in order to maximize vaccination coverage. Vaccination against these childhood diseases is a critical step towards reaching the Millennium Development Goal of reducing under-five mortality by two-thirds by 2015 and thus should be a global priority
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