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https://www.readbyqxmd.com/read/29351570/joint-adolescent-adult-early-phase-clinical-trials-to-improve-access-to-new-drugs-for-adolescents-with-cancer-proposals-from-the-multi-stakeholder-platform-accelerate
#1
N Gaspar, L V Marshall, D Binner, R Herold, R Rousseau, P Blanc, R Capdeville, J Carleer, C Copland, Y Kerloeguen, K Norga, L Pacaud, M-A Sevaux, C Spadoni, J Sterba, F Ligas, T Taube, M Uttenreuther-Fischer, S Chioato, M A O'Connell, B Geoerger, J-Y Blay, J C Soria, S Kaye, B Wulff, L Brugières, G Vassal, A D J Pearson
The impressive progress recently observed in adult cancers through the introduction of new drugs has not yet been translated to adolescents between 12 and 17 years of age. Currently adolescents are grouped with children, so their access to new, effective drugs already available for adults is delayed because paediatric drug development starts late relative to adult programmes. Moreover, specific early phase trials designed exclusively for adolescents in rare diseases recruit poorly, even if conducted internationally...
January 16, 2018: Annals of Oncology: Official Journal of the European Society for Medical Oncology
https://www.readbyqxmd.com/read/29350487/pediatric-patients-with-cutaneous-melanoma-a-european-study
#2
Ines B Brecht, Angela De Paoli, Gianni Bisogno, Daniel Orbach, Dominik T Schneider, Ulrike Leiter, Sonja Offenmueller, Giovanni Cecchetto, Jan Godzinski, Ewa Bien, Teresa Stachowicz-Stencel, Tal Ben-Ami, Stefano Chiaravalli, Andrea Maurichi, Gian Luca De Salvo, Silvia Sorbara, Christine Bodemer, Claus Garbe, Yves Reguerre, Andrea Ferrari
INTRODUCTION: Cutaneous melanoma is rare in childhood and published studies have mainly been retrospective single-institution series or small case series. Given the absence of clinical protocols dedicated to pediatric melanoma, the treatment approach is generally extrapolated from the ones applied to adults. METHODS: Coordinated by the European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT), this study collected patients prospectively registered between 2002 and 2012 under national cooperative projects dedicated to rare pediatric tumors in Italy, Poland, Germany, and France...
January 19, 2018: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/29275494/differences-in-pediatric-headache-prescription-patterns-by-diagnosis
#3
Jonathan Rabner, Allison Ludwick, Alyssa LeBel
BACKGROUND: Few studies have reported prescription patterns for headache medication. OBJECTIVE: The aim was to present the rates of specific medication prescribed to pediatric patients diagnosed with migraine, tension-type headache (TTH), and new daily persistent headache (NDPH), as well as differences in those prescription patterns by diagnosis, age, and gender. PATIENTS AND METHODS: A query using the i2b2 platform yielded 14,591 patients [migraine 10,547 (72...
December 23, 2017: Paediatric Drugs
https://www.readbyqxmd.com/read/29236237/outcome-of-children-and-adolescents-with-central-nervous-system-tumors-in-phase-i-trials
#4
Fernando Carceller, Francisco Bautista, Irene Jiménez, Raquel Hladun-Álvaro, Cécile Giraud, Luca Bergamaschi, Madhumita Dandapani, Isabelle Aerts, François Doz, Didier Frappaz, Michela Casanova, Bruce Morland, Darren R Hargrave, Gilles Vassal, Andrew D J Pearson, Birgit Geoerger, Lucas Moreno, Lynley V Marshall
Central nervous system (CNS) tumors are a leading cause of death in pediatric oncology. New drugs are desperately needed to improve survival. We evaluated the outcome of children and adolescents with CNS tumors participating in phase I trials within the Innovative Therapies for Children with Cancer (ITCC) consortium. Patients with solid tumors aged < 18 years at enrollment in their first dose-finding trial between 2000 and 2014 at eight ITCC centers were included retrospectively. Survival was evaluated using univariate/multivariate analyses...
December 13, 2017: Journal of Neuro-oncology
https://www.readbyqxmd.com/read/29213035/-problems-of-juvenile-myoclonic-epilepsy-a-view-through-the-prism-of-time
#5
V A Karlov, V S Zolovkina
The authors present a comparative analysis of the results of the study of 72 adolescent patients with juvenile myoclonic epilepsy (JME), which were published in 2010, and a new sample of patients over the last 5 years with the publications of domestic and foreign authors. Difficult clinical cases are described. These data indicate positive shifts in the diagnosis and treatment of JME. However, the question of diagnosis is still a hard problem for neurologists. The questions to be resolved are: gender aspect of the disease, to what extent levetiracetam could be a drug of first choice and to what extent valproats could be replaced; monitoring of children born to mothers with JME, a role of paternal inheritance...
2017: Zhurnal Nevrologii i Psikhiatrii Imeni S.S. Korsakova
https://www.readbyqxmd.com/read/29201174/effects-of-activated-carbon-n-acetylcysteine-sustained-release-microcapsule-on-dipeptidyl-peptidase-iv-expression-in-young-rats-with-non-alcoholic-fatty-liver-disease
#6
Hongping Zhou, Tingting Shi, Jun Yan, Xiaojin Chen, Li Liao, Shiyong Zhao, Hongying Fang, Rangxiao Zhuang
Non-alcoholic fatty liver disease (NAFLD) in children has become the most common liver disease influencing adolescent health and one of the most influencing chronic liver diseases among children in Chinese wealthy families, particularly in coastal regions. However, the medicine available for the treatment of NAFLD is deficient. In order to solve this problem, our team studied the activated carbon N-acetylcysteine (NAC) sustained-release microcapsule, which improves the oxidation resistance, bioavailability and drug stability of acetylcysteine and reduces toxic and side effects...
November 2017: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/29182223/tuberculosis-in-poland-in-2015
#7
Maria Korzeniewska-Koseła
AIM OF THE STUDY: To evaluate the main features of TB epidemiology in 2015 in Poland and to compare with the data on the same phenomena in the EU/EEA countries. METHODS: Analysis of case – based data on TB patients from National TB Register, data on anti-TB drug susceptibility testing results in cases notified in 2015, data from National Institute of Public Health- National Institute of Hygiene on cases of tuberculosis as AIDS-defining disease, data from Central Statistical Office on deaths from tuberculosis based on death certificates, data from ECDC report „ European Centre for Disease Prevention and Control/WHO Regional Office for Europe...
2017: Przegla̧d Epidemiologiczny
https://www.readbyqxmd.com/read/29176356/evolving-paradigms-for-new-agent-development-in-pediatric-oncology
#8
Andrea Flynn, Elizabeth Fox
PURPOSE OF REVIEW: To discuss considerations of new paradigms for clinical drug development in pediatric oncology that incorporate our expanding knowledge and complexity of molecular alterations associated with cancer; advances in cancer immunology and cellular therapy; the increasing number of new anticancer drugs, therapeutic approaches, and potential combinations; and recent initiatives by regulatory agencies to improve access to safe and effective therapies. RECENT FINDINGS: Cancer in children and adolescents is a rare event with significant long-term impact on individuals and society...
November 14, 2017: Current Opinion in Pediatrics
https://www.readbyqxmd.com/read/29172138/non-epileptic-seizures-in-children-views-and-approaches-at-a-uk-child-and-adolescent-psychiatry-conference
#9
Andrew McWilliams, Colin Reilly, Isobel Heyman
PURPOSE: To describe clinical experiences of paediatric non-epileptic seizures (NES) among doctors attending a UK child and adolescent psychiatry conference. METHODS: Sixty-six participants completed a semi-structured bespoke questionnaire. Results are reported using descriptive statistics. RESULTS: Two-thirds of respondents regarded the best name for the disorder to be "non-epileptic seizures". Although most doctors saw new cases each year, two-thirds described their own service as poorly equipped to manage NES...
October 31, 2017: Seizure: the Journal of the British Epilepsy Association
https://www.readbyqxmd.com/read/29154107/aripiprazole-in-treatment-of-gilles-de-la-tourette-syndrome-new-therapeutic-option
#10
REVIEW
Piotr Janik, Natalia Szejko
Aripiprazole is a dopamine D2- and serotonin 5-hydroxytryptamine (5-HT)1A receptor partial agonist and 5-HT2A receptor antagonist primarily used for the treatment of schizophrenia, bipolar disorder or depression with psychotic ideation. However, recently a number of new possible indications have been suggested, among them Gilles de la Tourette syndrome (GTS). In two randomized, double-blind, placebo-controlled studies in children and adolescents with GTS has been confirmed the efficacy of aripiprazole in tic reduction...
November 9, 2017: Neurologia i Neurochirurgia Polska
https://www.readbyqxmd.com/read/29140932/characteristics-of-treatment-experienced-hiv-infected-african-children-and-adolescents-initiating-darunavir-and-or-etravirine-based-antiretroviral-treatment
#11
Bethany Corrigan, Irene Mukui, Lloyd Mulenga, Nobuhle Mthethwa, Mosilinyane Letsie, Stephanie Bruno, Natella Rakhmanina
BACKGROUND: Data are limited on the selection and sequencing of second and third-line pediatric antiretroviral treatment (ART) in resource-limited settings. This study aimed to evaluate characteristics of African pediatric patients initiated on darunavir (DRV) and/or etravirine (ETR) through a specific drug donation program. METHODS: This was a cross-sectional study of baseline immunologic, virologic and demographic characteristics of children and adolescents initiating DRV- and/or ETR-based ART...
November 14, 2017: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/29114859/antiepileptic-drug-behavioral-side-effects-and-baseline-hyperactivity-in-children-and-adolescents-with-new-onset-epilepsy
#12
Shanna M Guilfoyle, Katherine Follansbee-Junger, Aimee W Smith, Angela Combs, Shannon Ollier, Brooke Hater, Avani C Modi
OBJECTIVE: To examine baseline psychological functioning and antiepileptic drug (AED) behavioral side effects in new onset epilepsy and determine, by age, whether baseline psychological functioning predicts AED behavioral side effects 1 month following AED initiation. METHODS: A retrospective chart review was conducted between July 2011 and December 2014 that included youths with new onset epilepsy. As part of routine interdisciplinary care, caregivers completed the Behavior Assessment System for Children, 2nd Edition: Parent Rating Scale to report on baseline psychological functioning at the diagnostic visit and the Pediatric Epilepsy Side Effects Questionnaire to identify AED behavioral side effects at the 1-month follow-up clinic visit following AED initiation...
January 2018: Epilepsia
https://www.readbyqxmd.com/read/29098969/-upward-trend-in-the-use-of-psychostimulants-by-adults
#13
L van der Koog, J van der Schans, M A Tjioe, J H J Bos, B J Bijker, E Hak, C C M Schuiling-Veninga
OBJECTIVE: To gain insight into the trends in the use of psychostimulants among adults. DESIGN: Retrospective database study. METHODS: We selected data on adults (≥ 18 years) who had had a minimum of 2 prescriptions for a psychostimulant drug within 1 year from IADB.nl, a Netherlands database of filled prescriptions (59 public pharmacies, approximately 600,000 patients). We calculated both the number of new users and the total number of users of psychostimulants per year in the period 2004-2014...
2017: Nederlands Tijdschrift Voor Geneeskunde
https://www.readbyqxmd.com/read/29091743/movement-disorders-in-children-and-adolescents-receiving-antipsychotic-pharmacotherapy-a-population-based-study
#14
Robert G Biscontri, Sarita Jha, David M Collins, Shawn Bugden, Laurence Y Katz, Silvia Alessi-Severini
OBJECTIVES: To describe a cohort of young users of risperidone and quetiapine in the province of Manitoba (Canada) and assess the risk for movement disorders in the two treatments. METHODS: This was a population-based study conducted on all residents of the province of 19 years of age and younger who received prescriptions for risperidone or quetiapine between April 1, 1996, and March 31, 2011. Incident rates of antipsychotic use were reported. The risk for movement disorders in patients treated with quetiapine compared with those treated with risperidone was assessed by time-to-event analysis using Cox proportional hazards models...
November 1, 2017: Journal of Child and Adolescent Psychopharmacology
https://www.readbyqxmd.com/read/28991007/the-optimal-approach-to-nutrition-and-cystic-fibrosis-latest-evidence-and-recommendations
#15
Francis M Hollander, Nicole M de Roos, Harry G M Heijerman
PURPOSE OF REVIEW: Cystic fibrosis (CF) is a progressive genetic disease that affects multiple organ systems. Therapy is directed to maintain and optimize nutritional status and pulmonary function, as these are key factors in survival. In this review, the most recent findings regarding nutritional management associated with pulmonary function and outcome will be explored. RECENT FINDINGS: Evidence-based and expert-based guidelines emphasize the need for adequate nutritional intake to improve nutritional status...
November 2017: Current Opinion in Pulmonary Medicine
https://www.readbyqxmd.com/read/28980418/medulloblastoma-in-children-and-adolescents-a-systematic-review-of-contemporary-phase-i-and-ii-clinical-trials-and-biology-update
#16
REVIEW
Francisco Bautista, Victoria Fioravantti, Teresa de Rojas, Fernando Carceller, Luis Madero, Alvaro Lassaletta, Lucas Moreno
Survival rates for patients with medulloblastoma have improved in the last decades but for those who relapse outcome is dismal and new approaches are needed. Emerging drugs have been tested in the last two decades within the context of phase I/II trials. In parallel, advances in genetic profiling have permitted to identify key molecular alterations for which new strategies are being developed. We performed a systematic review focused on the design and outcome of early-phase trials evaluating new agents in patients with relapsed medulloblastoma...
November 2017: Cancer Medicine
https://www.readbyqxmd.com/read/28968169/modernizing-clinical-trial-eligibility-recommendations-of-the-american-society-of-clinical-oncology-friends-of-cancer-research-minimum-age-working-group
#17
Lia Gore, S Percy Ivy, Frank M Balis, Eric Rubin, Katherine Thornton, Martha Donoghue, Samantha Roberts, Suanna Bruinooge, Jennifer Ersek, Nancy Goodman, Caroline Schenkel, Gregory Reaman
Purpose Children have historically been excluded from first-in-human studies of promising new cancer drugs and later phase adult clinical trials. Delays in evaluation may result in off-label use without dosing information as the only access to new drugs. A multistakeholder workshop was convened in May 2016 by ASCO and Friends of Cancer Research to identify opportunities for when it would be scientifically appropriate to expand trial eligibility to include children younger than age 18 years in first-in-human and other adult cancer clinical trials...
November 20, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28945987/sedation-and-analgesia-for-procedures-in-the-pediatric-emergency-room
#18
REVIEW
Carlos Eduardo Ramalho, Pedro Messeder Caldeira Bretas, Claudio Schvartsman, Amélia Gorete Reis
OBJECTIVE: Children and adolescents often require sedation and analgesia in emergency situations. With the emergence of new therapeutic options, the obsolescence of others, and recent discoveries regarding already known drugs, it became necessary to review the literature in this area. DATA SOURCES: Non-systematic review in the PubMed database of studies published up to December 2016, including original articles, review articles, systematic reviews, and meta-analyses...
November 2017: Jornal de Pediatria
https://www.readbyqxmd.com/read/28915908/advances-in-paediatrics-in-2016-current-practices-and-challenges-in-allergy-autoimmune-diseases-cardiology-endocrinology-gastroenterology-infectious-diseases-neonatology-nephrology-neurology-nutrition-pulmonology
#19
REVIEW
Carlo Caffarelli, Francesca Santamaria, Dora Di Mauro, Carla Mastrorilli, Silvia Montella, Sergio Bernasconi
This review reports main progresses in various pediatric issues published in Italian Journal of Pediatrics and in international journals in 2016. New insights in clinical features or complications of several disorders may be useful for our better understanding. They comprise severe asthma, changing features of lupus erythematosus from birth to adolescence, celiac disease, functional gastrointestinal disorders, Moebius syndrome, recurrent pneumonia. Risk factors for congenital heart defects, Kawasaki disease have been widely investigated...
September 16, 2017: Italian Journal of Pediatrics
https://www.readbyqxmd.com/read/28910830/consensus-guidelines-for-therapeutic-drug-monitoring-in-neuropsychopharmacology-update-2017
#20
C Hiemke, N Bergemann, H W Clement, A Conca, J Deckert, K Domschke, G Eckermann, K Egberts, M Gerlach, C Greiner, G Gründer, E Haen, U Havemann-Reinecke, G Hefner, R Helmer, G Janssen, E Jaquenoud, G Laux, T Messer, R Mössner, M J Müller, M Paulzen, B Pfuhlmann, P Riederer, A Saria, B Schoppek, G Schoretsanitis, M Schwarz, M Silva Gracia, B Stegmann, W Steimer, J C Stingl, M Uhr, S Ulrich, S Unterecker, R Waschgler, G Zernig, G Zurek, P Baumann
Therapeutic drug monitoring (TDM) is the quantification and interpretation of drug concentrations in blood to optimize pharmacotherapy. It considers the interindividual variability of pharmacokinetics and thus enables personalized pharmacotherapy. In psychiatry and neurology, patient populations that may particularly benefit from TDM are children and adolescents, pregnant women, elderly patients, individuals with intellectual disabilities, patients with substance abuse disorders, forensic psychiatric patients or patients with known or suspected pharmacokinetic abnormalities...
September 14, 2017: Pharmacopsychiatry
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