47 research outputs found
Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?
Higher Efficiency of Percutaneous Microwave (MWA) Than Radiofrequency Ablation (RFA) in Achieving Complete Response in Cirrhotic Patients with Early Hepatocellular Carcinoma
Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test
The aim of this study was to establish the possible effect of glucocorticoid treatment on upper limb function in a cohort of 91 non-ambulant DMD boys and adults of age between 11 and 26 years.
All 91 were assessed using the Performance of Upper Limb test. Forty-eight were still on glucocorticoid after loss of ambulation, 25 stopped steroids at the time they lost ambulation and 18 were GC naive or had steroids while ambulant for less than a year.
At baseline the total scores ranged between 0 and 74 (mean 41.20). The mean total scores were 47.92 in the glucocorticoid group, 36 in those who stopped at loss of ambulation and 30.5 in the naive group (p <0.001).
The 12-month changes ranged between -20 and 4 (mean -4.4). The mean changes were -3.79 in the glucocorticoid group, -5.52 in those who stopped at loss of ambulation and -4.44 in the naive group. This was more obvious in the patients between 12 and 18 years and at shoulder and elbow levels.
Our findings suggest that continuing glucocorticoids throughout teenage years and adulthood after loss of ambulation appears to have a beneficial effect on upper limb function. (C) 2015 The Authors. Published by Elsevier B.V
Timed rise from floor as a predictor of disease progression in Duchenne muscular dystrophy: An observational study
The role of timed items, and more specifically, of the time to rise from the floor, has been reported as an early prognostic factor for disease progression and loss of ambulation. The aim of our study was to investigate the possible effect of the time to rise from the floor test on the changes observed on the 6MWT over 12 months in a cohort of ambulant Duchenne boys.A total of 487 12-month data points were collected from 215 ambulant Duchenne boys. The age ranged between 5.0 and 20.0 years (mean 8.48 ±2.48 DS).The results of the time to rise from the floor at baseline ranged from 1.2 to 29.4 seconds in the boys who could perform the test. 49 patients were unable to perform the test at baseline and 87 at 12 month The 6MWT values ranged from 82 to 567 meters at baseline. 3 patients lost the ability to perform the 6mwt at 12 months. The correlation between time to rise from the floor and 6MWT at baseline was high (r = 0.6, p<0.01).Both time to rise from the floor and baseline 6MWT were relevant for predicting 6MWT changes in the group above the age of 7 years, with no interaction between the two measures, as the impact of time to rise from the floor on 6MWT change was similar in the patients below and above 350 m. Our results suggest that, time to rise from the floor can be considered an additional important prognostic factor of 12 month changes on the 6MWT and, more generally, of disease progression
Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data
The aim of the study was to establish 24 month changes in upper limb function using a revised version of the performance of upper limb test (PUL 2.0) in a large cohort of ambulant and non-ambulant boys with Duchenne muscular dystrophy and to identify possible trajectories of progression. Of the 187 patients studied, 87 were ambulant (age range: 7\u201315.8 years), and 90 non-ambulant (age range: 9.08\u201324.78). The total scores changed significantly over time (p<0.001). Non-ambulant patients had lower total scores at baseline (mean 19.7) when compared to the ambulant ones (mean 38.4). They also had also a bigger decrease in total scores over 24 months compared to the ambulant boys (4.36 vs 2.07 points). Multivariate model analysis showed that the Performance of Upper Limb changes reflected the entry level and ambulation status, that were independently associated to the slope of Performance of Upper Limb changes. This information will be of help both in clinical practice and at the time of designing clinical trials
Reliability of the Performance of Upper Limb assessment in Duchenne muscular dystrophy
Abstract The Performance of Upper Limb was specifically designed to assess upper limb function in Duchenne muscular dystrophy. The aim of this study was to assess (1) a cohort of typically developing children from the age of 3 years onwards in order to identify the age when the activities assessed in the individual items are consistently achieved, and (2) a cohort of 322 Duchenne children and young adults to establish the range of findings at different ages. We collected normative data for the scale validation on 277 typically developing subjects from 3 to 25 years old. A full score was consistently achieved by the age of 5 years. In the Duchenne cohort there was early involvement of the proximal muscles and a proximal to distal progressive involvement. The scale was capable of measuring small distal movements, related to activities of daily living, even in the oldest and weakest patients. Our data suggest that the assessment can be reliably used in both ambulant and non ambulant Duchenne patients in a multicentric setting and could therefore be considered as an outcome measure for future trials
Metrological traceability for the analysis of environmental pollutants in the atmosphere
The importance of carrying out accurate and reliable measurements is a fundamental topic in many different fields, in particular for
the safeguard of the environment and the climatic conditions of the planet. This is the basis for the planning of correct actions to
prevent environmental damages and potential harmful effects for the health of the human beings. The application of metrology to
chemical monitoring can assure the reliability of measurement results.
At the Istituto Nazionale di Ricerca Metrologica (INRiM), the Italian Metrology Institute, different activities are carried out for the
analysis of gaseous and organic pollutants in the atmosphere. This paper deals with some examples of such activities.
For gaseous pollutants primary gravimetric mixtures are produced for the calibration of the instrumentation devoted to the analysis of
carbon dioxide (CO2) and nitrogen oxides (NOx), with the aim of assuring traceability to the measurements of CO2 at ambient level and
at the vehicles emission level, and of NOx at ambient level. On the other hand, research is carried out in the field of organic
micropollutants, in particular regarding the establishment of metrological traceability for the atmospheric concentrations of Polycyclic
Aromatic Hydrocarbons (PAHs) adsorbed on particulate matter (PM)
Nitrogen oxides analysis: INRiM activity
The monitoring of nitrogen oxides (NOX) is
prescribed by European directives by means of
chemiluminescence. This paper presents the activity carried out
at INRiM for the preparation and analysis of NOX mixtures,
with the aim of establishing metrological traceability. Together
with the use of chemiluminescence, the application of Fourier
Transform Infrared (FTIR) spectroscopy is also under
investigation as it can allow the identification of some
impurities occurring in the gas mixtures which cannot be
discriminated by chemiluminescence
Production and validation of primary carbon dioxide reference standards at INRIM
The accurate and reliable determination of carbon dioxide (CO2) levels in atmosphere is
important for the role of this gas in the greenhouse effect. In addition, the rising level of CO2 absorbed by
the oceans from the atmosphere causes an acidification of the marine waters, with fallouts on the global
ecosystem. In this framework, the application of the metrological concepts is necessary to assure the
reliability of the measurement results. At INRIM, activities for the preparation and verification of CO2
primary reference mixtures are carried out. A facility for the production of primary CO2 standards by
gravimetry has been used since 2004. Due to the huge amount of gas mixtures that are needed to cover
different ranges of molar fractions during the calibration of instrumentation, a new system for dynamic
dilution was recently set up. An overview of the above-mentioned activities, with a focus on some crucial
issues related to mixture verification and stability studies, is presented in this work