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New drugs for children and adolescents

Si Houn Hahn, David Kronn, Nancy D Leslie, Loren D M Pena, Pranoot Tanpaiboon, Michael J Gambello, James B Gibson, Richard Hillman, David W Stockton, John W Day, Raymond Y Wang, Kristina An Haack, Raheel Shafi, Susan Sparks, Yang Zhao, Catherine Wilson, Priya S Kishnani
PurposePompe disease results from lysosomal acid α-glucosidase (GAA) deficiency and its associated glycogen accumulation and muscle damage. Alglucosidase alfa (recombinant human GAA (rhGAA)) received approval in 2006 as a treatment for Pompe disease at the 160 L production scale. In 2010, larger-scale rhGAA was approved for patients up to 8 years old without cardiomyopathy. NCT01526785 evaluated 4,000 L rhGAA efficacy/safety in US infantile- or late-onset Pompe disease (IOPD, LOPD) patients up to 1 year old transitioned from 160 L rhGAA...
March 22, 2018: Genetics in Medicine: Official Journal of the American College of Medical Genetics
Mery Munoz-Persy, Alfredo J Lucendo
Eosinophilic esophagitis (EoE) is a unique form of non-IgE-mediated food allergy characterized by esophageal eosinophilic infiltration that commonly causes dysphagia and food impaction in children and adolescents. Assessing the efficacy of dietary restrictions or drug therapies to achieve clinical and histologic resolution of EoE through randomized controlled trials and meta-analyses has resulted in new evidence-based guidelines. Avoiding food triggers is the only therapy targeting the cause of the disease...
March 17, 2018: European Journal of Pediatrics
Irving Zucker
Breastfeeding women are excluded from clinical trials of psychoactive drugs because of ethical concerns. Animal testing, which often is predictive of adverse effects in humans, represents the only avenue available for assessing drug safety for human offspring exposed to drugs during lactation. I determined whether behavioral outcomes for children exposed during breastfeeding to antidepressants, anxiolytics, antipsychotics, anti-seizure medications, analgesics, sedatives, and marijuana can be predicted by rodent studies of offspring exposed to drugs during lactation...
March 16, 2018: Psychopharmacology
Zhenqin Zhang, Zhenzhu Zhu, Cheng Luo, Chengcheng Zhu, Changli Zhang, Zijian Guo, Xiaoyong Wang
Osteosarcoma (OS) is the most common primary pediatric bone tumor lethal to children and adolescents. Chemotherapeutic agents such as cisplatin are not effective for OS because of their poor accessibility to this cancer and severe systemic toxicity. In this study, a lipophilic platinum(II) complex bearing a bisphosphonate bone-targeting moiety, cis-[PtL(NH3 )2 Cl]NO3 {BPP; L = tetraethyl [2-(pyridin-2-yl)ethane-1,1-diyl]bisphosphonate}, was prepared and characterized by NMR, electrospray ionization mass spectrometry, and single-crystal X-ray crystallography...
March 7, 2018: Inorganic Chemistry
Emanuele Pontali, Dina Visca, Rosella Centis, Lia D'Ambrosio, Antonio Spanevello, Giovanni Battista Migliori
PURPOSE OF REVIEW: Multidrug-resistant (MDR) tuberculosis (TB) and extensively drug-resistant (XDR)-TB epidemics are key obstacles towards TB control and elimination. RECENT FINDINGS: Diagnosis of MDR/XDR-TB is difficult and requires several weeks. New diagnostic tools are being tested and proposed allowing for shorter time to diagnosis and reduced delays in starting an adequate treatment regimen. MDR/XDR-TB treatment strategies are currently on an evolving stage...
February 20, 2018: Current Opinion in Pulmonary Medicine
Roberto Visentin, Enrique Campos-Náñez, Michele Schiavon, Dayu Lv, Martina Vettoretti, Marc Breton, Boris P Kovatchev, Chiara Dalla Man, Claudio Cobelli
BACKGROUND: A new version of the UVA/Padova Type 1 Diabetes (T1D) Simulator is presented which provides a more realistic testing scenario. The upgrades to the previous simulator, which was accepted by the Food and Drug Administration in 2013, are described. METHOD: Intraday variability of insulin sensitivity (S I ) has been modeled, based on clinical T1D data, accounting for both intra- and intersubject variability of daily S I . Thus, time-varying distributions of both subject's basal insulin infusion and insulin-to-carbohydrate ratio were calculated and made available to the user...
February 1, 2018: Journal of Diabetes Science and Technology
Marina Khrizman, Ann Pakalnis
Migraine is one of the most common neurologic conditions in pediatrics. It can be a significant stressor, causing absences from school and interruption of parents' work and family schedules. The mainstay of treatment remains educating patients about healthy lifestyle practices and the influences of sleep, stressors, and hydration on triggering migraine attacks. Psychological therapies such as biofeedback or cognitive-behavioral therapy may be beneficial in some patients, especially those with prominent psychological comorbidities...
February 1, 2018: Pediatric Annals
David M Clive, Vijay K Vanguri
The syndrome of tubulointerstitial nephritis and uveitis (TINU) is a multisystemic autoimmune disorder that may occur in response to various environmental triggers, including drugs and microbial pathogens. Evidence exists of HLA antigen-related genetic predisposition to developing TINU. The resulting inflammation affects chiefly the ocular uvea and renal tubules, although other organs may be involved. TINU is uncommon; only about 200 cases are on record since its original description 40 years ago, although it is possible that new ones are no longer being reported...
February 8, 2018: American Journal of Kidney Diseases: the Official Journal of the National Kidney Foundation
Linnéa Karlsson Lind, Katarina Wide, Björn Wettermark, Mia von Euler
This study aimed to investigate the utilization of antiepileptic drugs (AEDs) in children and adolescents with epilepsy and other diagnoses in a nationwide population between 2007 and 2014. Data on dispensed prescriptions of AEDs were collected from the Swedish Prescribed Drug Register and linked to diagnosis data from the National Patient Register covering all in- and outpatient consultations from Swedish hospitals. Children aged 0-17 years who had received at least one prescription for AEDs were selected...
February 10, 2018: Basic & Clinical Pharmacology & Toxicology
Tatsuhiko Urakami
The principal treatment for children and adolescents with type 2 diabetes is dietary and exercise management. However, the blood glucose levels of some patients receiving this treatment fail to improve; thus, pharmacological treatment is eventually required. The pathophysiology of type 2 diabetes in pediatric patients appears to be similar to that in adults; thus, the range of antidiabetic drugs used in adults is likely to be effective in pediatric patients as well. However, in the majority of countries, including Japan, only metformin, glimepiride, and insulin have been approved for use in pediatric patients...
2018: Clinical Pediatric Endocrinology: Case Reports and Clinical Investigations: Official Journal of the Japanese Society for Pediatric Endocrinology
Ekaterina Stepanova, Susannah Dowling, Molly Phelps, Robert L Findling
Autism spectrum disorder (ASD) is characterized by impairment in social communication and restricted patterns of behavior. Although there is no pharmacological treatment approved by the US Food and Drug Administration (FDA) for the core symptoms of ASD, there is mounting support in the literature for the management of behavioral symptoms associated with this developmental disorder, in particular, irritability and hyperactivity. Aripiprazole and risperidone are currently approved by the FDA for the treatment of irritability in youth with ASD...
December 2017: Dialogues in Clinical Neuroscience
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
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
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
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...
March 2018: Journal of Neuro-oncology
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
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
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
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...
February 2018: Current Opinion in Pediatrics
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...
December 2017: Seizure: the Journal of the British Epilepsy Association
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