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10 new medicines for pediatrics

J Stuart Elborn, Jane Davies, Marcus A Mall, Patrick A Flume, Barry Plant
The content for this activity is based on the satellite symposium, "Current Strategies for the Long-term Assessment, Monitoring, and Management for Cystic Fibrosis Patients Treated with CFTR Modulator Therapy" that was presented at the 39th European Cystic Fibrosis Society Conference on June 10, 2016 (Online access: The emergence of novel targeted agents, that directly correct CFTR loss function alleles, has created new treatment opportunities for patients with cystic fibrosis with advanced disease...
December 13, 2016: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
R Ferri, S Fulda, R P Allen, M Zucconi, O Bruni, S Chokroverty, L Ferini-Strambi, B Frauscher, D Garcia-Borreguero, M Hirshkowitz, B Högl, Y Inoue, A Jahangir, M Manconi, C L Marcus, D L Picchietti, G Plazzi, J W Winkelman, R S Zak
This report presents the results of the work by a joint task force of the International and European Restless Legs Syndrome Study Groups and World Association of Sleep Medicine that revised and updated the current standards for recording and scoring leg movements (LM) in polysomnographic recordings (PSG). First, the background of the decisions made and the explanations of the new rules are reported and then specific standard rules are presented for recording, detecting, scoring and reporting LM activity in PSG...
October 2016: Sleep Medicine
Robert W Grundmeier, Aaron J Masino, T Charles Casper, Jonathan M Dean, Jamie Bell, Rene Enriquez, Sara Deakyne, James M Chamberlain, Elizabeth R Alpern
BACKGROUND: Important information to support healthcare quality improvement is often recorded in free text documents such as radiology reports. Natural language processing (NLP) methods may help extract this information, but these methods have rarely been applied outside the research laboratories where they were developed. OBJECTIVE: To implement and validate NLP tools to identify long bone fractures for pediatric emergency medicine quality improvement. METHODS: Using freely available statistical software packages, we implemented NLP methods to identify long bone fractures from radiology reports...
November 9, 2016: Applied Clinical Informatics
Charles A Mkony, Ephata E Kaaya, Alex J Goodell, Sarah B Macfarlane
BACKGROUND: Faced with one of the lowest physician-to-population ratios in the world, the Government of Tanzania is urging its medical schools to train more physicians. The annual number of medical students admitted across the country rose from 55 in the 1990s to 1,680 approved places for the 2015/16 academic year. These escalating numbers strain existing faculty. OBJECTIVE: To describe the availability of faculty in medical schools in Tanzania. DESIGN: We identified faculty lists published on the Internet by five Tanzanian medical schools for the 2011/12 academic year and analyzed the appointment status, rank, discipline, and qualifications of faculty members...
2016: Global Health Action
William Wei Lim Chin, Angelika Joos
: Over the past two decades, there has been growing concern over the lack of proper medication for children. This review attempts to evaluate the current progress of EU Pediatric Regulation made since 2007. The lack of properly evaluated pediatric medication has for long been a source of concern in the European Union. The drugs that were used in the past were often not properly evaluated, and dosage was arbitrarily calculated. Therefore, it was necessary to establish the Pediatric Regulation (EC no...
December 2016: European Journal of Pediatrics
Fareen Zaver, Michael Hansen, Evan Leibner, Andrew Little, Michelle Lin
INTRODUCTION: By critically appraising open access, educational blogs and podcasts in emergency medicine (EM) using an objective scoring instrument, this installment of the ALiEM (Academic Life in Emergency Medicine) Blog and Podcast Watch series curated and scored relevant posts in the specific areas of pediatric EM. METHODS: The Approved Instructional Resources - Professional (AIR-Pro) series is a continuously building curriculum covering a new subject area every two months...
September 2016: Western Journal of Emergency Medicine
M B Nielsen, K Seitz
The impact factor (IF) for 2015 was recently released and this could be the time to once again reflect on its use as a metric of a journal. Problems and concerns regarding the IF have been addressed extensively elsewhere 1 2. The principle of the IF for a given year is that it represents the average number of citations of articles published in the journal in the two previous years.While authors frequently cite the IF as a determining factor for submission, the IF does not predict how many times individual articles will be cited...
August 2016: Ultraschall in der Medizin
M Saps, M A L van Tilburg, J V Lavigne, A Miranda, M A Benninga, J A Taminiau, C Di Lorenzo
BACKGROUND: There is little published evidence of efficacy for the most commonly used treatments. Thus, there is an urgent need to conduct clinical trials on existing and novel therapies. PURPOSE: In order to address these issues the Rome Foundation and members of the Pediatric Committee of the European Medicines Agency formed a subcommittee on clinical trials to develop guidelines for the design of clinical trials in children with irritable bowel syndrome (IBS)...
July 31, 2016: Neurogastroenterology and Motility: the Official Journal of the European Gastrointestinal Motility Society
H Eugene Hoyme, Wendy O Kalberg, Amy J Elliott, Jason Blankenship, David Buckley, Anna-Susan Marais, Melanie A Manning, Luther K Robinson, Margaret P Adam, Omar Abdul-Rahman, Tamison Jewett, Claire D Coles, Christina Chambers, Kenneth L Jones, Colleen M Adnams, Prachi E Shah, Edward P Riley, Michael E Charness, Kenneth R Warren, Philip A May
The adverse effects of prenatal alcohol exposure constitute a continuum of disabilities (fetal alcohol spectrum disorders [FASD]). In 1996, the Institute of Medicine established diagnostic categories delineating the spectrum but not specifying clinical criteria by which diagnoses could be assigned. In 2005, the authors published practical guidelines operationalizing the Institute of Medicine categories, allowing for standardization of FASD diagnoses in clinical settings. The purpose of the current report is to present updated diagnostic guidelines based on a thorough review of the literature and the authors' combined expertise based on the evaluation of >10 000 children for potential FASD in clinical settings and in epidemiologic studies in conjunction with National Institute on Alcohol Abuse and Alcoholism-funded studies, the Collaborative Initiative on Fetal Alcohol Spectrum Disorders, and the Collaboration on FASD Prevalence...
August 2016: Pediatrics
Alejandro J Lopez, Justin K Scheer, Nader S Dahdaleh, Alpesh A Patel, Zachary A Smith
STUDY DESIGN: A systematic review. OBJECTIVE: The available literature on interspinous rigid fixation/fusion devices (IFD) was systematically reviewed to explore the devices' efficacy and complication profile. SUMMARY OF BACKGROUND DATA: The clinical application of new spinal technologies may proceed without well-established evidence, as is the case with IFDs. IFDs are plate-like devices that are attached to the lateral aspects of 2 adjacent spinous processes to promote rigidity at that segment...
July 19, 2016: Clinical Spine Surgery
Chiara Bernagie, Marina Danckaerts, Martien Wampers, Marc De Hert
BACKGROUND: Both the US FDA and the European Medicines Agency (EMA) have approved aripiprazole for use in adolescents for specific indications. Given the assumed favorable side-effect profile of aripiprazole, its use in children and adolescents has increased for both official and off-label indications (anxiety disorders, eating disorders, personality disorders). However, several cases of children and adolescents with new-onset extrapyramidal symptoms (EPS) after commencing treatment with aripiprazole have been reported, and a more systematic appraisal of this possible risk is lacking...
September 2016: CNS Drugs
Renato Tambucci, Claudia Basti, Maria Maresca, Giangennaro Coppola, Alberto Verrotti
Eslicarbazepine acetate (ESL) is a once daily new third generation antiepileptic drug that shares the basic chemical structure of carbamazepine and oxcarbazepine - a dibenzazepine nucleus with the 5-carboxamide substituent, but is structurally different at the 10,11-position. ESL is a pro-drug metabolized to its major active metabolite eslicarbazepine. Despite the fact that the exact mechanism of action has not been fully elucidated, it is thought to involve inhibition of voltage-gated sodium channels (VGSC)...
2016: Neuropsychiatric Disease and Treatment
Andrea Stracciolini, Yi-Meng Yen, Pierre A d'Hemecourt, Cara L Lewis, Dai Sugimoto
UNLABELLED: To compare sports-related hip injuries on the basis of sex and age in a cohort of young athletes. A 5% random probability sample of all new patients' charts over a 10-year time period was selected for investigation. The most common hip injury diagnoses, sport at time of injury, mechanism (acute/traumatic vs. overuse), and types (bony vs. soft tissue) were compared by sex and age (preadolescent vs. adolescent). Descriptive and χ-analyses were carried out. The interaction of sex and age with respect to hip injury over time was examined by two-way (sex, age) analysis of variance...
July 2016: Journal of Pediatric Orthopedics. Part B
A K Parikh, C C Shah
BACKGROUND AND PURPOSE: The American College of Radiology-American Society of Neuroradiology-Society for Pediatric Radiology Practice Parameter for a neck CT suggests that coverage should be from the sella to the aortic arch. It also recommends using CT scans judiciously to achieve the clinical objective. Our purpose was to analyze the potential dose reduction by decreasing the scan length of a neck CT and to assess for any clinically relevant information that might be missed from this modified approach...
June 2016: AJNR. American Journal of Neuroradiology
Robert Flood, Paula Szwargulski, Nadeem Qureshi, Mary Bixby, Steven Laffey, Ryan Pratt, James Gerard
BACKGROUND: Immediate bedding has been shown to increase efficiency in general emergency departments (EDs), but little has been published regarding its use in pediatric emergency medicine. OBJECTIVE: Our aims were to improve door-to-provider (DTP) times and patient satisfaction and to better define the relationships between throughput times and patient satisfaction in a pediatric ED. METHODS: On November 1, 2011, we changed to a new immediate bedding triage process in our academic, urban pediatric Level I trauma center...
May 2016: Journal of Emergency Medicine
Laura Jacobson, Samantha Garbers, Hannah Helmy, Hope Roobol, Julia E Kohn, Megan L Kavanaugh
OBJECTIVE: Intrauterine devices (IUDs) are one of the most effective forms of reversible contraception and can reduce unintended pregnancy rates. We explored practice characteristics associated with IUD services across a network of primary care practices in New York City during 2010-2013. STUDY DESIGN: Data were extracted from electronic health records (EHRs) for 253 primary care practices participating in an EHR quality improvement program in New York City. We used diagnostic and procedure codes to count IUD insertions and removals among females aged 10-49 years during 2010-2013...
March 2016: Contraception
Simon Lygo-Baker, Patricia K Kokotailo, Karen M Young
The important role of medical trainees (interns and residents) as teachers is increasingly recognized in veterinary and human medicine, but often is not supported through adult learning programs or other preparatory training methods. To develop appropriate teaching programs focused on effective clinical teaching, more understanding is needed about the support required for the trainee's teaching role. Following discussion among faculty members from education and veterinary and pediatric medicine, an experienced external observer and expert in higher education observed 28 incoming and outgoing veterinary and pediatric trainees in multiple clinical teaching settings over 10 weeks...
2015: Journal of Veterinary Medical Education
Rivka Sukenik-Halevy, Mark David Ludman, Shay Ben-Shachar, Annick Raas-Rothschild
PURPOSE: Clinical genetics services are time- and labor-intensive. With increasing pressure for cost-effective medical care, the means of providing medical genetics services need to be evaluated in the current era of new genomic technologies. METHODS: An anonymous online survey regarding activities linked to medical genetics practice was administered to an international cohort of professionals. RESULTS: Among 151 responses, the reported average time required for pediatric, oncogenetic, pregnancy with a malformed fetus, and preamniocentesis counseling sessions was 48, 37, 40, and 18 min, respectively...
April 2016: Genetics in Medicine: Official Journal of the American College of Medical Genetics
Paula Aristizabal, Spencer Fuller, Rebeca Rivera, David Beyda, Raul C Ribeiro, William Roberts
In 2007, the 5-year survival rate for children with acute leukemia in Baja California, Mexico was estimated at 10% (vs. 88% in the United States). In response, stakeholders at St. Jude Children's Research Hospital, Rady Children's Hospital San Diego, and the Hospital General de Tijuana (HGT) implemented a transcultural partnership to establish a pediatric oncology program. The aim was to improve clinical outcomes and overall survival for children in Baja California. An initial needs assessment evaluation was performed and a culturally sensitive, comprehensive, 5-year plan was designed and implemented...
2015: Frontiers in Public Health
Tianwu Xie, Choonsik Lee, Wesley E Bolch, Habib Zaidi
PURPOSE: Nuclear cardiology plays an important role in clinical assessment and has enormous impact on the management of a variety of cardiovascular diseases. Pediatric patients at different age groups are exposed to a spectrum of radiation dose levels and associated cancer risks different from those of adults in diagnostic nuclear medicine procedures. Therefore, comprehensive radiation dosimetry evaluations for commonly used myocardial perfusion imaging (MPI) and viability radiotracers in target population (children and adults) at different age groups are highly desired...
June 2015: Medical Physics
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