Adolescents and MP3 Players: Too Many Risks, Too Few Precautions

OBJECTIVE. The goal was to assess risky and protective listening behaviors of adolescent users of MP3 players and the association of these behaviors with demographic characteristics and frequency of use. METHODS. In 2007, 1687 adolescents (12–19 years of age) in 68 classes in 15 Dutch secondary schools were invited to complete questionnaires about their music-listening behaviors. RESULTS. Ninety percent of participants reported listening to music through earphones on MP3 players; 32.8% were frequent users, 48.0% used high volume settings, and only 6.8% always or nearly always used a noise-limiter. Frequent users were _4 times more likely to listen to high-volume music than were infrequent users, and adolescents in practical prevocational schools were more than twice as likely to listen to high-volume music as were those attending preuniversity education. CONCLUSIONS. When using MP3 players, adolescents are very likely to engage in risky listening behaviors and are unlikely to seek protection. Frequent MP3 player use is an indicator of other risky listening behaviors, such as listening at high volumes and failing to use noise-limiter

Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study

Background: Though enzyme-replacement therapy (ERT) with alglucosidase alfa has significantly improved the prospects for patients with classic infantile Pompe disease, some 50 % of treated infants do not survive ventilator-free beyond the age of 3 years. We investigated whether higher and more frequent dosing of alglucosidase alfa improves outcome. Methods: Eight cross-reactive immun

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

Background: Infantile Pompe disease is a rare metabolic disease. Patients generally do not survive the first year of life. Enzyme replacement therapy (ERT) has proven to have substantial effects on survival in infantile Pompe disease. However, the costs of therapy are very high. In this paper, we assess the cost-effectiveness of enzyme replacement therapy in infantile Pompe disease. Methods. A patient simulation model was used to compare costs and effects of ERT with costs of effects of supportive therapy (ST). The model was filled with data on survival, quality of life and costs. For both arms of the model, data on survival w

Viscosity and fission time scale of^{156}Dy

In the fusion-fission reaction Ar-40+Cd-116-->Dy-156-->fission, performed at beam energies E(b) = 216 MeV and 238 MeV, gamma rays were measured in coincidence with fission fragments. The gamma-ray spectra are interpreted using a modified version of the statistical-model code CASCADE. From a comparison of the experimental and calculated spectra it is deduced that the nuclear viscosity is in the range 0.01 <gamma <4. The extracted fission time scale is of the order of 10(-19) s

Classic infantile Pompe patients approaching adulthood: A cohort study on consequences for the brain

Aim: To examine the long-term consequences of glycogen storage in the central nervous system (CNS) for classic infantile Pompe disease using enzyme replacement therapy. Method: Using neuropsychological tests and brain magnetic resonance imaging (MRI), we prospectively assessed a cohort of 11 classic infantile Pompe patients aged up to 17 years. Results: From approximately age 2 years onwards, brain MRI showed involvement of the periventricular white matter and centrum semiovale. After 8 years of age, additional white-matter abnormalities occurred in the corpus callosum, internal and external capsule, and subcortical areas. From 11 years of age, white-matter abnormalities were also found in the brainstem. Although there seemed to be a characteristic pattern of involvement over time, there were considerable variations between patients, reflected by variations in neuropsychological development. Cognitive development ranged from stable and normal to declines that lead to intellectual disabilities. Interpretation: As treatment enables patients with classic infantile Pompe disease to reach adulthood, white-matter abnormalities are becoming increasingly evident, affecting the neuropsychological development. Therefore, we advise follow-up programs are expanded to capture CNS involvement in larger, international patient cohorts, to incorporate our findings in the counselling of parents before the start of treatment, and to include the brain as an additional target in the development of next-generation therapeutic strategies for classic infantile Pompe disease. What this paper adds: In our long-term survivors treated intravenously with enzyme replacement therapy, we found slowly progressive symmetric white-matter abnormalities. Cognitive development varied from stable and normal to declines towards intellectual disabilities

Impact of enzyme replacement therapy on survival in adults with Pompe disease: Results from a prospective international observational study

Background: Pompe disease is a rare metabolic myopathy for which disease-specific enzyme replacement therapy (ERT) has been available since 2006. ERT has shown efficacy concerning muscle strength and pulmonary function in adult patients. However, no data on the effect of ERT on the survival of adult patients are currently available. The aim of this study was to assess the effect of ERT on survival in adult patients with Pompe disease. Methods. Data were collected as part of an international observational study conducted between 2002 and 2011, in which patients were followed on an annual basis. Time-dependent Cox's proportional hazards models were used for univariable and multivariable analyses. Results: Overall, 283 adult patients with a median age of 48 years (range, 19 to 81 years) were included in the study. Seventy-two percent of patients started ERT at some time during follow-up, and 28% never received ERT. During follow-up (median, 6 years; range, 0.04 to 9 years), 46 patients died, 28 (61%) of whom had never received ERT. After adjustment for age, sex, country of residence, and disease severity (based on wheelchair and ventilator use), ERT was positively associated with survival (hazard ratio, 0.41; 95% CI, 0.19 to 0.87). Conclusion: This prospective study was the first to demonstrate the positive effect of ERT on survival in adults with Pompe disease. Given the relatively recent registration of ERT for Pompe disease, these findings further support its beneficial impact in adult patients

Marital resemblance for obsessive–compulsive, anxious and depressive symptoms in a population-based sample.

Background. Resemblance between spouses can be due to phenotypic assortment, social homogamy and/or marital interaction. A significant degree of assortment can have consequences for the genetic architecture of a population. We examined the existence and cause(s) of assortment for obsessive-compulsive (OC), anxious and depressive symptoms in a population-based twin-family sample. Method. OC, anxious and depressive symptoms were measured in around 1400 twin-spouse pairs and >850 parent pairs. Correlations of twins and their spouse, twin and co-twin's spouse, spouses of both twins and parents of twins were obtained to consider phenotypic assortment versus social homogamy as possible causes of marital resemblance. The association of length of relationship with marital resemblance was also investigated. Finally, we examined whether within-trait or cross-trait processes play a primarily role in marital resemblance. Results. Small but significant within-trait correlations of between 0.1 and 0.2 were seen for spouse similarity in OC, anxious and depressive symptoms. Cross-correlations were significant but lower. There was no correlation between length of relationship and marital resemblance. From the pattern of correlations for twin-spouse, co-twin-spouse and spouses of both twins, phenotypic assortment could not be distinguished from social homogamy. Both within- and cross-assortment processes play a role in marital resemblance. Conclusions. Small within- and across-trait correlations exist for OC, anxious and depressive symptoms. No evidence for marital interaction was found. Spouse correlations are small, which makes it difficult to distinguish between social homogamy and phenotypic assortment. It is unlikely that correlations of this size will have a large impact on genetic studies. © 2008 Cambridge University Press

Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up

Background: Pompe disease is an inheritable metabolic disorder for which enzyme replacement therapy (ERT) has been available since 2006. Effects of ERT have been shown on distance walked, pulmonary function and survival. We investigated whether it also improves quality of life and participation in daily life in adult patients with the disease. Methods: In an international survey, we assessed quality of life (Short Form 36, SF-36) and participation (Rotterdam Handicap Scale, RHS) annually between 2002 and 2012. Repeated measurements mixed effects models were used to describe the data over time. Results: Responses were available for 174 adult patients. In the periods before and after start of ERT, the median follow-up times were 4 years each (range 0.5-8). The SF-36 Physical Component Summary measure (PCS) deteriorated before ERT (-0.73 score points per year (sp/y); CI 95 % -1.07 to -0.39), while it improved in the first 2 years of ERT (1.49 sp/y; CI 0.76 to 2.21), and remained stable thereafter. The Mental Component Summary measure (MCS) remained stable before and during ERT. After declining beforehand (-0.49 sp/year; CI -0.64 to-0.34), the RHS stabilized under ERT. Conclusion: In adult patients with Pompe disease, ERT positively affects quality of life and participation in daily life. Our results reinforce previous findings regarding the effect of ERT on muscle strength, pulmonary function and survival

Costs and effects of conventional vision screening and photoscreening in the Dutch preventive child health care system

Background: Little is known about costs and effects of vision screening strategies to detect amblyopia. Aim of this study was to compare costs and effects of conventional (optotype) vision screening, photoscreening or a combination in children aged 3-6 years. Methods: Population-based, cross-sectional study in preventive child health care in The Hague. Children aged 3 years (3y), 3 years and 9 months (3y9m) or 5-6 years (5/6y) received the conventional chart vision screening and a test with a photoscreener (Plusoptix 512C). Costs were based on test duration and additional costs for devices and diagnostic work-up. Results: Two thousand, one hundred and forty-four children were included. The estimated costs per child screened were (sic)17.44, (sic)20.37 and (sic)6.90 for conventional vision screening at 3y, 3y9m and 5/6y, respectively. For photoscreening, these estimates were (sic)6.61, (sic)7.52 and (sic)9.40 and for photoscreening followed by vision screening if the result was unclear (combination) (sic)9.32 (3y) and (sic)9.33 (3y9m). The number of children detected with amblyopia by age were 9, 14 and 5 (conventional screening), 6, 13 and 3 (photoscreening) and 10 (3y) and 15 (3y9m) (combination), respectively. The estimated costs per child diagnosed with amblyopia were (sic)1500, (sic)1050 and (sic)860 for conventional vision screening, (sic)860, (sic)420 and (sic)1940 for photoscreensic)ing and (sic)730 (3y) and (sic)450 (3y9m) for the combination. Conclusions: Combining photoscreening with vision screening seems promising to detect amblyopia in children aged 3y/3y9m, whereas conventional screening seems preferable at 5/6y. As the number of study children with amblyopia is small, further research on the effects of these screening alternatives in detecting children with amblyopia is recommended.Research into fetal development and medicin