bluuebunny/crossref_metadata_2025_split huggingface dataset:
Enzyme-Replacement Therapy With Agalsidase Alfa in Children With Fabry Disease
Authors: Ries Markus , Clarke Joe T.R. , Whybra Catharina , Timmons Margaret , Robinson Chevalia , Schlaggar Bradley L. , Pastores Gregory , Lien Y. Howard , Kampmann Christoph , Brady Roscoe O. , Beck Michael , Schiffmann Raphael 
Published: September 2006

CONTEXT. Fabry disease is an X-linked multisystem disorder. Enzyme-replacement therapy in adults has limited efficacy in treating major sequelae of advanced Fabry disease, such as kidney failure or stroke. This prompted a study of the safety and efficacy of enzyme replacement at an earlier stage of Fabry disease.
OBJECTIVES. Our purpose with this work was to evaluate safety and to explore efficacy of enzyme treatment with agalsidase alfa in pediatric patients with Fabry disease.
METHODS. We conducted a 6-month open-label study at 3 tertiary care centers with 24 children (19 boys and 5 girls) with a mean age of 11.8 (range: 6.5–18) years, to examine safety parameters, including infusion reactions and antiagalsidase alfa antibodies.
RESULTS. Agalsidase alfa was well tolerated, and all of the patients completed the study. Six boys and 1 girl had mild-to-moderate infusion reactions. One boy developed transient immunoglobulin G antibodies against agalsidase alfa. The boys showed a significant reduction in plasma globotriaosylceramide on treatment. Mean estimated glomerular filtration rate, cardiac structure, and function were normal and did not change over 26 weeks. Heart rate variability, as determined by 2-hour ambulatory monitoring, was decreased in the boys compared with the girls at baseline. All indices of heart rate variability improved significantly in the boys. Three patients with anhidrosis, as determined by quantitative sudomotor axon reflex testing, developed sweating. Six of 11 patients could reduce or cease their use of antineuropathic analgesics.
CONCLUSIONS. Enzyme replacement with agalsidase alfa was safe in this study. The exploratory efficacy analysis documented increased clearance of globotriaosylceramide and improvement of autonomic function. Prospective long-term studies are needed to assess whether enzyme replacement initiated early in patients with Fabry disease is able to prevent major organ failure in adulthood.
Diagnosis and Management of Food-Induced Anaphylaxis: A National Survey of Pediatricians
Authors: Krugman Scott D. , Chiaramonte Delia R. , Matsui Elizabeth C. 
Published: September 2006

BACKGROUND. Food allergy is a common pediatric problem, affecting as many as 6% of young children, yet it is unclear whether pediatricians are well prepared to manage food-induced anaphylaxis.
OBJECTIVE. The purpose of this work was to assess pediatricians' knowledge of diagnosis and management of children with food-induced anaphylaxis.
METHODS. A survey designed to assess food allergy diagnosis and management was mailed to a US national random sample of 1130 pediatricians. Survey questions were based on a clinical scenario involving a child having an anaphylactic reaction after ingesting peanut. Primary outcome measures included correct responses to the 11 questions about anaphylaxis.
RESULTS. A total of 468 pediatricians (41%) responded to the survey. The majority of the respondents were women (58%), spent &gt;50% of their time in a clinical setting (78%), and reported providing care for food allergy patients (86%). Overall, 70% of the pediatricians agreed that the clinical scenario was consistent with anaphylaxis, and 72% chose to administer epinephrine. However, only 56% of respondents agreed with both the diagnosis of anaphylaxis and treating with epinephrine. Most pediatricians (70%) did not recognize that a 30-minute observation period after anaphylaxis was too short. Pediatricians who reported providing care for food allergy patients were more likely to agree with the diagnosis of anaphylaxis (73% vs 59%), with treating the reaction with epinephrine (73% vs 64%), and with prescribing self-injectable epinephrine (83% vs 66%) than pediatricians who did not care for food allergy patients. The more certain that pediatricians were that the child was having an anaphylactic reaction, the more likely they were to agree with treating the reaction with epinephrine. In general, recent continuing medical education was not predictive of improved knowledge.
CONCLUSION. Although the majority of pediatricians seem to have some knowledge of food-induced anaphylaxis, a substantial proportion has knowledge deficits that may hinder their ability to provide optimal care to children with food-induced anaphylaxis. Pediatricians who provide health care for patients with food allergy may be better equipped to manage food-induced anaphylaxis than those who do not. Because continuing medical education was not a significant predictor of increased knowledge, ensuring that pediatric residents develop experience managing patients with food allergies may be a better strategy to educate primary care pediatricians about food allergy.
Relative Benefits of Stimulant Therapy With OROS Methylphenidate Versus Mixed Amphetamine Salts Extended Release in Improving the Driving Performance of Adolescent Drivers With Attention-Deficit/Hyperactivity Disorder
Authors: Cox Daniel J. , Merkel R. Lawrence , Moore Melissa , Thorndike Frances , Muller Carrie , Kovatchev Boris 
Published: September 2006

OBJECTIVE. Automobile accidents are the leading cause of death among adolescents, and collisions are 2 to 4 times more likely to occur among adolescents with attention-deficit/hyperactivity disorder. Studies have demonstrated that stimulants improve driving performance. This study compared 2 long-acting stimulant medications during daytime and evening driving evaluations.METHODS. Adolescent drivers with attention-deficit/hyperactivity disorder were compared on a driving simulator after taking 72 mg of OROS methylphenidate, 30 mg of mixed amphetamine salts extended release, or placebo in a randomized, double-blind, placebo-controlled, crossover study design. During laboratory testing, adolescents drove a driving simulator at 5:00 pm, 8:00 pm, and 11:00 pm. Driving performance was rated by adolescents and investigators.RESULTS. The study included 35 adolescent drivers with attention-deficit/hyperactivity disorder (19 boys/16 girls). The mean age was 17.8 years. The overall Impaired Driving Score demonstrated that OROS methylphenidate led to better driving performance compared with placebo and mixed amphetamine salts extended release, whereas mixed amphetamine salts extended release demonstrated no statistical improvement over placebo. Specifically, relative to placebo, OROS methylphenidate resulted in less time driving off the road, fewer instances of speeding, less erratic speed control, more time executing left turns, and less inappropriate use of brakes. OROS methylphenidate and mixed amphetamine salts extended release worked equally well for male and female adolescents and equally as well with teenagers who have combined and inattentive subtypes of attention-deficit/hyperactivity disorder.CONCLUSIONS. This study validates the use of stimulants to improve driving performance in adolescents with attention-deficit/hyperactivity disorder. In the study, OROS methylphenidate promoted significantly improved driving performance compared with placebo and mixed amphetamine salts extended release.
Electrocardiography in the Diagnosis of Right Ventricular Hypertrophy in Children
Authors: Puchalski Michael D. , Lozier John S. , Bradley David J. , Minich L. LuAnn , Tani Lloyd Y. 
Published: September 2006

OBJECTIVE. Although the electrocardiogram is commonly obtained in the evaluation of patients with pulmonary hypertension, its value as a screening test for right ventricular hypertrophy or pulmonary hypertension is unclear. Therefore, we sought to determine the value of an electrocardiogram in the diagnosis of right ventricular hypertrophy using echocardiography as the gold standard.
METHODS. We identified children without congenital heart disease who underwent evaluation for suspected pulmonary hypertension that included both an electrocardiogram and echocardiography within a specified time frame.
RESULTS. A total of 76 echocardiography-electrocardiogram pairs for pulmonary hypertension were identified. Although there was a significant relationship between electrocardiogram and echocardiography evidence of right ventricular hypertrophy, the sensitivity of an electrocardiogram in diagnosing echocardiography-documented right ventricular hypertrophy was only 69%, and the positive predictive value was 67%. There was no relationship between electrocardiogram changes and Doppler tricuspid regurgitation gradient.
CONCLUSION. Despite a statistically significant relationship between an electrocardiogram and echocardiography in the diagnosis of right ventricular hypertrophy, an electrocardiogram has limited value as a screening tool for right ventricular hypertrophy because of its relatively low sensitivity and positive predictive value.
Chronic Snoring and Sleep in Children: A Demonstration of Sleep Disruption
Authors: Lopes M. Cecilia , Guilleminault Christian 
Published: September 2006

OBJECTIVE. Chronic snoring that does not adhere to the criteria for a diagnosis of obstructive sleep apnea syndrome may be associated with learning and behavioral problems. We investigated the sleep structure of chronic snorers who had an apnea-hypopnea index of &lt;1 event per hour and analyzed the cyclic alternating pattern.
METHODS. Fifteen successively seen chronic snorers (9.8 ± 4 years) with an apnea-hypopnea index of &lt;1 and 15 aged-matched control subjects (10.3 ± 5 years) underwent an investigation of their sleep with the determination of non–apneic-hypopneic breathing abnormalities polysomnographic scoring using current criteria and analysis of the cyclic alternating pattern.
RESULTS. Chronic snorers have evidence of flow limitations and tachypnea during sleep even if they do not present with apneas, hypopneas, and decrease in oxygen saturations. They also present with abnormal cyclic alternating pattern rates and changes in phase A of cyclic alternating pattern compared with control subjects.
CONCLUSIONS. An apnea-hypopnea index value cannot be the sole determinant in evaluating sleep-disordered breathing in children. Children who have chronic snoring and do not respond to the criteria for obstructive sleep apnea syndrome can present with an abnormal sleep electroencephalogram as evidenced by a significant increase in cyclic alternating pattern rates, with a predominance of abnormalities in slow wave sleep.
Trends in Sunburns, Sun Protection Practices, and Attitudes Toward Sun Exposure Protection and Tanning Among US Adolescents, 1998–2004
Authors: Cokkinides Vilma , Weinstock Martin , Glanz Karen , Albano Jessica , Ward Elizabeth , Thun Michael 
Published: September 2006

BACKGROUND. Sun exposure in childhood is an important risk factor for developing skin cancer as an adult. Despite extensive efforts to reduce sun exposure among the young, there are no population-based data on trends in sunburns and sun protection practices in the young. The aim of this study was to describe nationally representative trend data on sunburns, sun protection, and attitudes related to sun exposure among US youth.METHODS. Cross-sectional telephone surveys of youth aged 11 to 18 years in 1998 (N = 1196) and in 2004 (N = 1613) were conducted using a 2-stage sampling process to draw population-based samples. The surveys asked identical questions about sun protection, number of sunburns experienced, and attitudes toward sun exposure. Time trends were evaluated using pooled logistic regression analysis.RESULTS. In 2004, 69% of subjects reported having been sunburned during the summer, not significantly less than in 1998 (72%). There was a significant decrease in the percentage of those aged 11 to 15 years who reported sunburns and a nonsignificant increase among the 16- to 18-year-olds. The proportion of youth who reported regular sunscreen use increased significantly from 31% to 39%. Little change occurred in other recommended sun protection practices.CONCLUSIONS. A small reduction in sunburn frequency and modest increases in sun protection practices were observed among youth between 1998 and 2004, despite widespread sun protection campaigns. Nevertheless, the decrease in sunburns among younger teens may be cause for optimism regarding future trends. Overall, there was rather limited progress in improving sun protection practices and reducing sunburns among US youth between 1998 and 2004.
Associations Between Sleep Problems, Anxiety, and Depression in Twins at 8 Years of Age
Authors: Gregory Alice M. , Rijsdijk Frühling V. , Dahl Ronald E. , McGuffin Peter , Eley Thalia C. 
Published: September 2006

OBJECTIVES. Associations between sleep and internalizing problems are complex and poorly understood. To better understand these covarying difficulties, genetic and environmental influences were estimated by using a twin design.
METHODS. Three hundred 8-year-old twin pairs reported on their anxiety and depression by completing the Screen for Childhood Anxiety Related Emotional Disorders and the Children's Depression Inventory. Parents reported on their children's sleep problems by completing the Child Sleep Habits Questionnaire.
RESULTS. Children reported by their parents to have different types of sleep problems self-reported more depression symptoms than those without. The correlation between total sleep-problem score and depression was moderate. That between sleep problems and anxiety was smaller and was not examined further. The association between sleep problems and depression was mainly explained by genes, and there was substantial overlap between the genes influencing sleep problems and those influencing depression. There was smaller influence from environmental factors making family members alike, and environmental factors making family members different decreased the association between sleep problems and depression.
CONCLUSIONS. A range of sleep difficulties are associated with depression in school-aged children, and the overall association between the 2 difficulties may be largely influenced by genes.
Comparing Perceptions of Training for Medicine-Pediatrics and Categorically Trained Physicians
Authors: Freed Gary L. ,   and the Research Advisory Committee of the American Board of Pediatrics
Published: September 2006

OBJECTIVE. The objective of this study was to compare the perceptions of clinical preparedness among graduates of categorical pediatric and internal medicine residency programs versus medicine-pediatric training programs and whether these varied for recent or older graduates.
METHODS. A mail survey study was conducted in the fall of 2004 of a random sample of 245 and 246 internal medicine physicians who applied for or took a general certifying examination between 1980 and 1997 (older graduates) and between 1998 and 2003 (recent graduates), respectively, and a random sample of 248 and 247 pediatricians who applied for or took a general certifying examination between 1980 and 1997 (older graduates) and between 1998 and 2003 (recent graduates). Analyses compared results from a recent, similar survey of medicine-pediatric physicians.
RESULTS. Response rate for pediatricians was 78% and for internal medicine physicians was 64%. Categorically trained pediatricians were more likely than medicine-pediatrics–trained physicians to report that they were very well prepared to care for infants (65% vs 50%) but less likely well trained to care for adolescents (17% vs 45%). Categorically trained internal medicine residents were less likely to feel very well prepared to care for both adults and elders than their medicine-pediatrics–trained counterparts.
CONCLUSIONS. Categorically trained pediatricians and internal medicine physicians believed that they were no better prepared to care for their patients than medicine-pediatrics–trained physicians. Regardless of their beliefs of their preparedness, medicine-pediatrics physicians in practice spend a significant majority of their time in the care of adults rather than children. Our findings indicate that this is not because of their perception of a lack of training or preparation in the care of children relative to categorically trained pediatricians. Other factors, including but not limited to patient demographic changes, reimbursement for care, and the job market, potentially play a significant role.
Detection of Acetaminophen Protein Adducts in Children With Acute Liver Failure of Indeterminate Cause
Authors: James Laura P. , Alonso Estella M. , Hynan Linda S. , Hinson Jack A. , Davern Timothy J. , Lee William M. , Squires Robert H. ,   the Pediatric Acute Liver Failure Study Group
Published: September 2006

OBJECTIVE. Acetaminophen cysteine protein adducts are a widely recognized correlate of acetaminophen-mediated hepatic injury in laboratory animals. The objective of this study was to use a new assay for the detection of acetaminophen cysteine protein adducts in children with acute liver failure to determine the role of acetaminophen toxicity in acute liver failure of unknown cause.
METHODS. Serum samples from children with acute liver failure were measured for acetaminophen cysteine protein adducts using high-performance liquid chromatography with electrochemical detection. For comparison, samples from children with well-characterized acetaminophen toxicity and children with known other causes of acute liver failure also were measured for acetaminophen cysteine protein adducts. The analytical laboratory was blinded to patient diagnoses.
RESULTS. Acetaminophen cysteine protein adduct was detected in 90% of samples from children with acute liver failure that was attributed to acetaminophen toxicity, 12.5% of samples from children with acute liver failure of indeterminate cause, and 9.6% of samples from children with acute liver failure that was attributed to other causes. Adduct-positive patients from the indeterminate cause subgroup had higher levels of serum aspartate aminotransferase and alanine aminotransferase and lower levels of bilirubin. Adduct-positive patients also had lower rates of transplantation and higher rates of spontaneous remission.
CONCLUSIONS. A small but significant percentage of children with acute liver failure of indeterminate cause tested positive for acetaminophen cysteine protein adducts, strongly suggesting acetaminophen toxicity as the cause of acute liver failure. An assay for the detection of acetaminophen cysteine protein adducts can aid the diagnosis of acetaminophen-related liver injury in children.
Self-Perceived Health-Related Quality of Life of Former Extremely Low Birth Weight Infants at Young Adulthood
Authors: Saigal Saroj , Stoskopf Barbara , Pinelli Janet , Streiner David , Hoult Lorraine , Paneth Nigel , Goddeeris John 
Published: September 2006

OBJECTIVES. The goals were to compare the self-reported, health-related quality of life of former extremely low birth weight and normal birth weight infants at young adulthood and to determine whether there were any changes over time.METHODS. A prospective, longitudinal, population-based study with concurrent control subjects was performed. We interviewed 143 of 166 extremely low birth weight survivors (birth weight: 501–1000 g; 1977–1982 births) and 130 of 145 sociodemographically comparable, normal birth weight, reference subjects. Neurosensory impairments were present for 27% extremely low birth weight and 2% normal birth weight young adults. Health Utilities Index 2 was used to assess health status, and standard gamble technique was used to measure directly the self-reported, health-related, quality of life and 4 hypothetical health states.RESULTS. Extremely low birth weight young adults reported more functional limitations in cognition, sensation, mobility, and self-care, compared with control subjects. There were no differences between groups in the mean self-reported, health-related, quality of life or between impaired (n = 38) and nonimpaired (n = 105) extremely low birth weight subjects. However, with a conservative approach of assigning a score of 0 for 10 severely disabled, extremely low birth weight subjects, the mean health-related quality of life was significantly lower than control values. Repeated-measures analysis of variance to compare health-related quality-of-life measurements obtained for young adults and teens showed the same decline in scores over time for both groups. There were no differences between groups in the ratings provided for the hypothetical health states.CONCLUSIONS. At young adulthood, health-related quality of life was not related to size at birth or to the presence of disability. There was a small decrease in health-related quality-of-life scores over time for both groups.
Improving Pediatric Prevention via the Internet: A Randomized, Controlled Trial
Authors: Christakis Dimitri A. , Zimmerman Frederick J. , Rivara Frederick P. , Ebel Beth 
Published: September 2006

BACKGROUND. Innovations to improve the delivery of pediatric preventive care are needed.METHODS. We enrolled children, 0 to 11 years of age, into a factorial, randomized, controlled trial of a tailored, evidence-based, Web site (MyHealthyChild) that provided information on prevention topics before a scheduled well-child visit. There were 2 components of the intervention, namely, parental Web content and provider notification. Parental Web content provided information to parents about prevention topics; provider notification communicated to physicians topics that were of interest to parents. We assigned 887 children randomly to 4 groups (usual care, content only, content and notification, or notification only). Outcomes were determined with telephone follow-up surveys conducted 2 to 4 weeks after the visit. Poisson regression analysis was used to determine the independent effects of each intervention on the number of topics discussed and the number of preventive practices implemented.RESULTS. Parents in the notification/content group and in the notification-only group reported discussing more MyHealthyChild topics with their provider. Parents in the notification/content group and in the content-only group reported implementing more MyHealthyChild topic suggestions (such as use of a safety device).CONCLUSIONS. A Web-based intervention can activate parents to discuss prevention topics with their child's provider. Delivery of tailored content can promote preventive practices.