Read by QxMD icon Read

New drugs for children and adolescents

Michela Casanova, Eleonora Basso, Chiara Magni, Luca Bergamaschi, Stefano Chiaravalli, Roberto Carta, Elisa Tirtei, Maura Massimino, Franca Fagioli, Andrea Ferrari
Pazopanib is an oral multikinase inhibitor that has proved effective in adults treated for relapsing soft tissue sarcoma and synovial sarcoma in particular. Two cases are reported here of pediatric patients with pretreated relapsing synovial sarcoma whose tumors showed a prolonged response to pazopanib given on compassionate grounds. These results suggest that new agents found effective in adult patients might achieve similar results in adolescents with the same disease. Facilitating the availability of new drugs for children and adolescents is a major challenge for pediatric oncologists...
August 25, 2016: Tumori
Sascha Abbas, Peter Ihle, Jürgen-Bernhard Adler, Susanne Engel, Christian Günster, Martin Holtmann, Axel Kortevoss, Roland Linder, Werner Maier, Gerd Lehmkuhl, Ingrid Schubert
Children and adolescents with mental health problems need effective and safe therapies to support their emotional and social development and to avoid functional impairment and progress of social deficits. Though psychotropic drugs seem to be the preferential treatment, psychotherapy and psychosocial interventions are essential in mental health care. For Germany, current data on the utilization of psychotherapy and psychosocial interventions in children with mental health problems is lacking. To analyse why certain children and adolescents with mental or behavioural disorders do and others do not receive non-drug treatment, we assessed predictors associated with specific non-drug psychiatric/psychotherapeutic treatment including psychosocial interventions, psychotherapy and other non-drug treatments...
September 14, 2016: European Child & Adolescent Psychiatry
Klaus Rose, Thomas Müller
BACKGROUND: Both the United States (US) Food and Drug Administration (FDA) and the European Union (EU) European Medicines Agency (EMA) order pediatric clinical trials as a condition for approval of new compounds. We evaluate clinical value and likelihood of sufficient recruitment for pediatric multiple sclerosis (pMS) studies and discuss US and EU pediatric legislation with pMS as a paradigm. METHODS: We analyzed pMS clinical trials requested by the FDA and the EMA and industry-sponsored pMS studies registered on www...
September 2016: Therapeutic Advances in Neurological Disorders
Athena P Kourtis, Ayesha Mirza
Access to high-quality reproductive health care is important for adolescents and young adults with HIV infection to prevent unintended pregnancies, sexually transmitted infections, and secondary transmission of HIV to partners and children. As perinatally HIV-infected children mature into adolescence and adulthood and new HIV infections among adolescents and young adults continue to occur in the United States, medical providers taking care of such individuals often face issues related to sexual and reproductive health...
September 2016: Pediatrics
Tanuja Chitnis, Angelo Ghezzi, Barbara Bajer-Kornek, Alexey Boyko, Gavin Giovannoni, Daniela Pohl
Over the last 20 years, there have been significant advances in multiple sclerosis (MS) therapeutics, with regulatory approval for 13 therapies in adults by the European Medicines Agency (EMA) and Food and Drug Administration. However, there is only limited approval for interferon-β and glatiramer acetate use in children 12 years and older by the EMA. Availability of disease-modifying therapies to children and adolescents with MS is variable by region, and is extremely limited in some regions of the world...
August 30, 2016: Neurology
Lauri J Virta, Maiju M Saarinen, Kaija-Leena Kolho
BACKGROUND AND AIMS: The burden of inflammatory bowel disease (IBD) in health care is high. We conducted research on the temporal changes in the incidence of pediatric IBD (PIBD) using nationwide registry-based data in Finland. METHODS: All PIBD cases diagnosed at less than 20 years of age during 1987-2014 (in total, 5,415 patients) were retrieved from a database documenting reimbursements for drug costs. Incidence rates were calculated by dividing the number of annual new PIBD cases by the size of the pediatric population at risk during each calendar year...
August 23, 2016: Journal of Crohn's & Colitis
Douglas S Ross, Henry B Burch, David S Cooper, M Carol Greenlee, Peter Laurberg, Ana Luiza Maia, Scott A Rivkees, Mary Samuels, Julie Ann Sosa, Marius N Stan, Martin A Walter
BACKGROUND: Thyrotoxicosis has multiple etiologies, manifestations, and potential therapies. Appropriate treatment requires an accurate diagnosis and is influenced by coexisting medical conditions and patient preference. This document describes evidence-based clinical guidelines for the management of thyrotoxicosis that would be useful to generalist and subspecialty physicians and others providing care for patients with this condition. METHODS: The American Thyroid Association (ATA) previously cosponsored guidelines for the management of thyrotoxicosis that were published in 2011...
October 2016: Thyroid: Official Journal of the American Thyroid Association
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
Sascha Abbas, Peter Ihle, Jürgen-Bernhard Adler, Susanne Engel, Christian Günster, Roland Linder, Gerd Lehmkuhl, Ingrid Schübert
BACKGROUND: In view of the well-known increase in prescriptions of stimulants for children and adolescents over the last 20 years, it is important to study trends in the prevalence and incidence of the use of other psychotropic drugs by this age group as well, to enable an early response to potential problems in the current care situation. METHODS: We used nationwide data from German statutory health insurance funds (Allgemeine Ortskrankenkasse [AOK], all insurees; Techniker Kranken - kasse [TK], a 50% randomized sample) concerning all insurees aged 0-17 years (5...
June 6, 2016: Deutsches Ärzteblatt International
N Matin, O Tabatabaie, R Falsaperla, P Pavone, A Serra, S Cocuzza, P Di Mauro, L Licciardello, R Lubrano, G Vitaliti
Immunoglobulin E (IgE) was discovered in 1966 and was found responsible for immune defense against helminths, type I hypersensitivity and allergic diseases. IgE mediates allergic responses by binding to Fc receptors (the high affinity Fc-epsilon receptor I and the low affinity Fc-epsilon receptor II or CD23) expressed on tissue mast cells and blood basophils. This binding leads to degranulation and release of pro-inflammatory mediators. Considering the pivotal role of IgE in allergic diseases, antibodies against IgE potentiate an array of new therapeutic strategies and in this regard omalizumab (rhuMAb-E25, Xolair) has been developed as a monoclonal biologic drug to block serum IgEs...
April 2016: Journal of Biological Regulators and Homeostatic Agents
Lauren E Hirsch, Tamara Pringsheim
BACKGROUND: Autism spectrum disorders (ASD) include autistic disorder, Asperger's disorder and pervasive developmental disorder - not otherwise specified (PDD-NOS). Antipsychotics have been used as a medication intervention for irritability related to ASD. Aripiprazole, a third-generation, atypical antipsychotic, is a relatively new drug that has a unique mechanism of action different from that of other antipsychotics. This review updates a previous Cochrane review on the safety and efficacy of aripiprazole for individuals with ASD, published in 2011 (Ching 2011)...
2016: Cochrane Database of Systematic Reviews
J Pietsch, K Scheffel, D Acquarone, I Koch, M Deters, M Hermanns-Clausen
Aim of the study: Based on an abundance of information on the internet, psychotropic plants and mushrooms are of increasing relevance as legal and cheap alternatives to other illicit drugs. Due to the lack of a German database, the abuse of selected biogenic drugs using reported exposures to 3 German Poison Information Centres (PICs) was examined. Methods: Information on abuse of biogenic drugs was collected from the German PICs in Berlin, Erfurt and Fribourg (2007-2013). Results: 602 drug abuses were reported to the selected PICs...
June 14, 2016: Das Gesundheitswesen
Eric J Dziuban, Emilia D Rivadeneira
Antiretroviral treatment coverage for children living with HIV is low, and new efforts are underway to expand eligibility so that all children and adolescents qualify for treatment regardless of immune suppression or clinical stage. While recent trials provide direct evidence of the benefit of this approach in adults, no such studies have been performed in children. This paper examines the available body of evidence regarding universal HIV treatment for children and adolescents. The benefits and challenges for individual patient health, as well as programmatic-level outcomes, are assessed...
June 10, 2016: Pediatric Infectious Disease Journal
Andrew D J Pearson, Ralf Herold, Raphaël Rousseau, Chris Copland, Brigid Bradley-Garelik, Debbie Binner, Renaud Capdeville, Hubert Caron, Jacqueline Carleer, Louis Chesler, Birgit Geoerger, Pamela Kearns, Lynley V Marshall, Stefan M Pfister, Gudrun Schleiermacher, Jeffrey Skolnik, Cesare Spadoni, Jaroslav Sterba, Hendrick van den Berg, Martina Uttenreuther-Fischer, Olaf Witt, Koen Norga, Gilles Vassal
An urgent need remains for new paediatric oncology drugs to cure children who die from cancer and to reduce drug-related sequelae in survivors. In 2007, the European Paediatric Regulation came into law requiring industry to create paediatric drug (all types of medicinal products) development programmes alongside those for adults. Unfortunately, paediatric drug development is still largely centred on adult conditions and not a mechanism of action (MoA)-based model, even though this would be more logical for childhood tumours as these have much fewer non-synonymous coding mutations than adult malignancies...
July 2016: European Journal of Cancer
Stéphane Auvin
INTRODUCTION: The pharmacological management of epilepsy is continually modified by the increase in our knowledge about the efficacy and the safety on antiepileptic drugs. AREAS COVERED: This review covers the published data (2010-2015) on the pharmacological management of epilepsy in children and adolescent. We review the data from the most recent randomized controlled and open-label trials. EXPERT OPINION: Even if there is an increasing number of antiepileptic drugs approved for focal seizure in children and adolescent with epilepsy, each new approval would be considered as a significant addition to the current therapeutic options...
August 2016: Expert Opinion on Pharmacotherapy
Philip Asherson, Jan Buitelaar, Stephen V Faraone, Luis A Rohde
For many years, attention-deficit hyperactivity disorder (ADHD) was thought to be a childhood-onset disorder that has a limited effect on adult psychopathology. However, the symptoms and impairments that define ADHD often affect the adult population, with similar responses to drugs such as methylphenidate, dexamphetamine, and atomoxetine, and psychosocial interventions, to those seen in children and adolescents. As a result, awareness of ADHD in adults has rapidly increased and new clinical practice has emerged across the world...
June 2016: Lancet Psychiatry
Fernando Carceller, Francisco J Bautista, Lucy A Fowkes, Lynley V Marshall, Sara I Sirvent, Julia C Chisholm, Andrew D J Pearson, Dow-Mu Koh, Lucas Moreno
INTRODUCTION: RECIST guidelines constitute the reference for radiological response assessment in most paediatric trials of anticancer agents. However, these criteria have not been validated in children. We evaluated the outcomes and patterns of progression of children/adolescents enrolled in phase I trials in two paediatric drug development units. METHODS: Patients aged ≤21 assessed with RECIST (v1.0 or v1.1) were eligible. Clinico-radiological data were analysed using Mann-Whitney U and log-rank tests to correlate response categories and sum of longest diameters (SLD) with time-to-event variables and overall survival (OS)...
August 2016: Pediatric Blood & Cancer
Jerzy R Kowalczyk, Marzena Samardakiewicz, Kathy Pritchard-Jones, Ruth Ladenstein, Samira Essiaf, Edel Fitzgerald, Giulia Petrarulo, Gilles Vassal
BACKGROUND: In recent years, the European Commission has supported an increased focus on rare cancers in order to improve quality of care, disseminate best practice and set up networks to improve access that is essential to continued progress. At European Union (EU) conference in 2009, an agreement was reached to create a 'European Standard of Care for Children with Cancer'. In 2013, the European Paediatric Oncology Society launched a Europe-wide survey in order to assess the implementation of the Standards...
July 2016: European Journal of Cancer
Yi-Qing Gao, Min Gao, Ying Xue
Type 1 diabetes mellitus (T1D) and type 2 diabetes (T2D) mellitus are on the increase in children and adolescents. An increase in T2D is linked to the increasing rates of obesity in children. Usually, in both children and adults, T1D is treated with insulin while T2D is treated with metformin. There are other classes of drugs that are under assessment for their safety and efficacy in relation to pediatric patients. Most of these new drugs, however, have not been studied in children. Thus, the repertoire of drugs that are available to treat diabetes in children is limited...
April 2016: Experimental and Therapeutic Medicine
O V Bykova, I A Nankina, I M Drozdova, O V Kvasova, T T Batysheva, A N Boiko
The vast majority of therapies are being evaluated and introduced for the treatment of adult multiple sclerosis (MS). A role of these drugs in the management of pediatric MS has yet to be defined both in Russia and in the whole world. Despite the fact that today the study of new drugs in the pediatric population have included in routine practices of the big pharmaceutical agencies, such as FDA and EMA, recommendations for the treatment of pediatric patients with MS are based not so much on a long period of systematic clinical research, but on professional consensus of international expert associations, in particular, the International pediatric multiple sclerosis study group (IPMSSG)...
2016: Zhurnal Nevrologii i Psikhiatrii Imeni S.S. Korsakova
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"