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biologic therapy in children

Fabienne Hartmann-Fritsch, Daniela Marino, Ernst Reichmann
BACKGROUND: The treatment of severe full-thickness skin defects represents a significant and common clinical problem worldwide. A bio-engineered autologous skin substitute would significantly reduce the problems observed with today's gold standard. METHODS: Within 15 years of research, the Tissue Biology Research Unit of the University Children's Hospital Zurich has developed autologous tissue-engineered skin grafts based on collagen type I hydrogels. Those products are considered as advanced therapy medicinal products (ATMPs) and are routinely produced for clinical trials in a clean room facility following the guidelines for good manufacturing practice (GMP)...
September 2016: Transfusion Medicine and Hemotherapy
L Hepburn, D J Hijnen, B R Sellman, T Mustelin, M A Sleeman, R D May, I Strickland
Atopic dermatitis (AD) is a complex, chronic inflammatory skin disorder affecting more than 10% of UK children and is a major cause of occupation-related disability. A subset of patients, particularly those with severe AD, are persistently colonised with Staphylococcus aureus (S. aureus) and exacerbation of disease is commonly associated with this bacterium by virtue of increased inflammation and allergic sensitisation, aggravated by skin barrier defects. Understanding the complex biology of S. aureus is an important factor when developing new drugs to combat infection...
October 25, 2016: British Journal of Dermatology
Francesca Tocchioni, Chiara Tani, Laura Bartolini, Maria Moriondo, Francesco Nieddu, Patrizia Pecile, Chiara Azzari, Antonio Messineo, Marco Ghionzoli
Bacterial growth of peritoneal fluid specimens obtained during surgical procedures for acute appendicitis may be useful to optimize further antibiotic therapy in complicated cases. DNA amplification represents a fast technique to detect microbial sequences. We aimed to compare the potential of DNA amplification versus traditional bacterial growth culture highlighting advantages and drawbacks in a surgical setting. Peritoneal fluid specimens were collected during surgery from 36 children who underwent appendectomy between May and December 2012...
September 19, 2016: Pediatric Reports
M J Santos, M Conde, A F Mourão, F O Ramos, M Cabral, I Brito, M P Ramos, R C Marques, S M Gomes, M Guedes, M J Gonçalves, P Estanqueiro, C Zilhão, M Rodrigues, C Henriques, M Salgado, H Canhão, J E Fonseca, J M Gomes
OBJECTIVE: To provide evidence-based guidance for the rational and safe prescription of biological therapies in children and adolescents with juvenile idiopathic arthritis (JIAs) considering the latest available evidence and the new licensed biologics. METHODS: Rheumatologists and Pediatricians with expertise in Pediatric Rheumatology updated the recommendations endorsed by the Portuguese Society of Rheumatology and the Portuguese Society of Pediatrics based on published evidence and expert opinion...
July 2016: Acta Reumatológica Portuguesa
Gerd Horneff, Ariane Klein, Prasad T Oommen, Anton Hospach, Ivan Foeldvari, Isa Feddersen, Kirsten Minden
OBJECTIVES: While tumour necrosis factor (TNF)-α-inhibitor treatment improved outcome of juvenile idiopathic arthritis (JIA) management markedly, concerns have been raised about an association of TNF-α-inhibitor treatment and an increased risk for malignancies especially lymphoma. METHODS: Cases of suspected malignancies documented in the German Biker Registry are reviewed in detail. RESULTS: Until Dec 31, 2015, 3695 JIA patients were prospectively followed with a total of more than 13,198 observation years...
September 8, 2016: Clinical and Experimental Rheumatology
Tao Xiong, Hongju Chen, Rong Luo, Dezhi Mu
BACKGROUND: The rising prevalence of autism spectrum disorder (ASD) has increased the need for evidence-based treatments to lessen the impact of symptoms. Presently, no therapies are available to effectively treat individuals with all of the symptoms of this disorder. It has been suggested that hyperbaric oxygen therapy may alleviate the biochemical dysfunction and clinical symptoms of ASD. OBJECTIVES: To determine whether treatment with hyperbaric oxygen:1. improves core symptoms of ASD, including social communication problems and stereotypical and repetitive behaviors;2...
October 13, 2016: Cochrane Database of Systematic Reviews
Bérangère S Joly, Alain Stepanian, Thierry Leblanc, David Hajage, Hervé Chambost, Jérôme Harambat, Fanny Fouyssac, Vincent Guigonis, Guy Leverger, Tim Ulinski, Thérésa Kwon, Chantal Loirat, Paul Coppo, Agnès Veyradier
BACKGROUND: Thrombotic thrombocytopenic purpura is a rare thrombotic microangiopathy, related to a severe ADAMTS13 deficiency (a disintegrin and metalloprotease with thromboSpondin type 1 repeats, member 13; activity <10% of normal). Childhood-onset thrombotic thrombocytopenic purpura is very rare and initially often misdiagnosed, especially when ADAMTS13 deficiency is acquired (ie, not linked to inherited mutations of the ADAMTS13 gene). We aimed to investigate initial presentation, management, and outcome of acquired thrombotic thrombocytopenic purpura in children...
October 3, 2016: Lancet Haematology
Didier Frappaz, Alexandre Vasiljevic, Pierre-Aurelien Beuriat, Claire Alapetite, Jacques Grill, Alexandru Szathmari, Cécile Faure-Conter
Ependymomas represent 10% of pediatric brain tumors. In the recent WHO 2016 classification, pathology is enriched by localization and molecular biology. Whatever the age, total removal by one or several looks when required remains a major prognostic factor. In children, focal radiation remains a standard, while the role of chemotherapy is matter of randomized studies. In infants, front line chemotherapy is the standard. Inclusion in the SIOP ependymoma II protocol is encouraged. In case of relapse, further surgery and radiation are advised, while inclusion in innovative trials including re-irradiation, and phase I-II should be encouraged...
October 2016: Bulletin du Cancer
Edyta Szymanska, Maciej Dadalski, Sylwia Szymanska, Wieslawa Grajkowska, Maciej Pronicki, Jaroslaw Kierkus
INTRODUCTION: The clinical efficacy of infliximab (IFX) for induction of remission in both adults and children with active Crohn's disease (CD) has been well documented. Recently, so-called "deep remission" defined as mucosal healing has become the ultimate endpoint of the most recent therapeutic advances for CD. However, endoscopic evidence of mucosal healing is not necessarily associated with histological evidence of suppression of inflammation. AIM: Since data on that issue are limited, especially in the paediatric population, the aim of this study was to assess the impact of induction therapy with IFX on deep microscopic remission in paediatric patients with CD...
2016: Przegla̜d Gastroenterologiczny
Raymond M Johnson, Kelly R Bergmann, John J Manaloor, Xiaoqing Yu, James E Slaven, Anupam B Kharbanda
Background.  Pediatric Kawasaki disease (KD) and human immunodeficiency virus (HIV)(+) adult Kawasaki-like syndrome (KLS) are dramatic vasculitides with similar physical findings. Both syndromes include unusual arterial histopathology with immunoglobulin (Ig)A(+) plasma cells, and both impressively respond to pooled Ig therapy. Their distinctive presentations, histopathology, and therapeutic response suggest a common etiology. Because blood is in immediate contact with inflamed arteries, we investigated whether KD and KLS share an inflammatory signature in serum...
September 2016: Open Forum Infectious Diseases
Nicole Coles, Catherine Birken, Jill Hamilton
Severe obesity in childhood is increasing in prevalence and is associated with considerable morbidity. Studies into pediatric obesity have focused largely on interventions that do not necessarily target the unique biologic or psychological underpinnings for the weight gain in the individual child or adolescent. Outcomes show modest improvement and are of questionable benefit for patients with severe obesity. Although weight is a commonly used outcome, other psychological and metabolic parameters including normalization of physical activity and eating behaviors should be primary outcome goals...
September 29, 2016: BMJ: British Medical Journal
Branko M van Hulst, Patrick de Zeeuw, Dienke J Bos, Yvonne Rijks, Sebastiaan F W Neggers, Sarah Durston
BACKGROUND: Changes in reward processing are thought to be involved in the etiology of attention-deficit/hyperactivity disorder (ADHD), as well as other developmental disorders. In addition, different forms of therapy for ADHD rely on reinforcement principles. As such, improved understanding of reward processing in ADHD could eventually lead to more effective treatment options. However, differences in reward processing may not be specific to ADHD, but may be a trans-diagnostic feature of disorders that involve ADHD-like symptoms...
September 28, 2016: Journal of Child Psychology and Psychiatry, and Allied Disciplines
Stéphanie Fontaine Carbonnel, Pascal Rippert, Isabelle Poirot, Dominique Gachet, Capucine de Lattre, Carole Vuillerot
OBJECTIVE: Since 2005, in France, corticosteroid therapy is now widely used in Duchenne muscular dystrophy (DMD). This treatment has changed our practice of pediatric rehabilitation teams. We describe here our 10-year clinical experience in treating DMD patients by CS according to international guidelines i.e. prednisone 0.75mg/kg/day started from the plateau of motor function. MATERIALS/PATIENTS AND METHODS: We conducted a prospective study. Information was given on the expected effects and side effects...
September 2016: Annals of Physical and Rehabilitation Medicine
Francisco Bautista, Jasper Van der Lugt, Pamela R Kearns, Francis J Mussai, C Michel Zwaan, Lucas Moreno
Survival rates in pediatric leukemia have greatly improved in the last decades but still a substantial number of patients will relapse and die. New agents are necessary to overcome the limitations of conventional chemotherapy and hematopoietic stem cell transplantation and to reduce their undesirable long-term toxicities. The identification of driving molecular alterations of leukemogenesis in subsets of patients will allow the incorporation of new-targeted therapies. Areas covered: In this article the authors present a detailed review of the most recent advances in targeted therapies for pediatric leukemias...
November 2016: Expert Opinion on Drug Discovery
María Baro Fernández, Vanesa Pérez Alonso
INTRODUCTION: Pediatric low-grade gliomas (PLGG) are the most common primary central nervous system tumor in children. Patients in whom gross total resection can be achieved have an excellent overall (OS) and event-free survival (EFS) and do not require adjuvant therapy. However, children with unresectable tumors often experience multiple progressions and require additional treatment. CONVENTIONAL CHEMOTHERAPY: Radiotherapy results in long-term tumor control, but it is associated with significant toxicity, making chemotherapy the preferred therapeutic option...
October 2016: Child's Nervous System: ChNS: Official Journal of the International Society for Pediatric Neurosurgery
Alvaro Lassaletta, Michal Zapotocky, Eric Bouffet, Cynthia Hawkins, Uri Tabori
Hemispheric low-grade gliomas account for the second most common location in pediatric low-grade gliomas (PLGGs) after the cerebellum. The pathological spectrum includes gangliogliomas, dysembryoplastic neuroepithelial tumors (DNETs), diffuse astrocytomas, pilocytic astrocytomas, and pleomorphic xanthoastrocytomas (PXAs), among others. Clinically, hemispheric PLGGs represent a well-recognized cause of intractable epilepsy in children and adolescents. With an excellent long-term outcome, surgery remains the cornerstone and patients with gross total resection typically do not need any further therapies...
October 2016: Child's Nervous System: ChNS: Official Journal of the International Society for Pediatric Neurosurgery
Mario Abinun, Jonathan P Lane, Mark Wood, Mark Friswell, Terence J Flood, Helen E Foster
Severe infections are emerging as major risk factors for death among children with juvenile idiopathic arthritis (JIA). In particular, children with refractory JIA treated with long-term, multiple, and often combined immunosuppressive and antiinflammatory agents, including the new biological disease-modifying antirheumatic drugs (DMARDs), are at increased risk for severe infections and death. We investigated 4 persons with JIA who died during 1994-2013, three of overwhelming central venous catheter-related bacterial sepsis caused by coagulase-negative Staphylococus or α-hemolytic Streptococcus infection and 1 of disseminated adenovirus and Epstein-Barr virus infection)...
October 2016: Emerging Infectious Diseases
Jeremy C Kane, Laura K Murray, Judith Cohen, Shannon Dorsey, Stephanie Skavenski van Wyk, Jennica Galloway Henderson, Mwiya Imasiku, John Mayeya, Paul Bolton
BACKGROUND: The effectiveness of mental health interventions such as trauma-focused cognitive behavioral therapy (TF-CBT) may vary by client, caregiver, and intervention-level variables, but few randomized trials in low- and middle-income countries (LMIC) have conducted moderation analyses to investigate these characteristics. This study explores moderating factors to TF-CBT treatment response among a sample of orphans and vulnerable children (OVC) in Zambia. METHODS: Data were obtained from a completed randomized trial of TF-CBT among 257 OVC in Zambia...
October 2016: Journal of Child Psychology and Psychiatry, and Allied Disciplines
Benjamin A Farber, Neerav Shukla, Irene Isabel P Lim, Jennifer M Murphy, Michael P La Quaglia
INTRODUCTION: Non-central nervous system (non-CNS) rhabdoid tumors tend to present at a young age and have an extremely aggressive course, with dismal overall survival rates. Inactivation of the tumor suppressor gene SMARCB1 has been shown in rhabdoid tumors regardless of anatomic location, suggesting a common genetic basis. We retrospectively analyzed our institutional experience with non-CNS rhabdoid tumors to determine overall survival and prognostic variables. METHODS: We reviewed records of pediatric patients (age<22y) with non-CNS rhabdoid tumor at our institution between 1980 and 2014...
August 31, 2016: Journal of Pediatric Surgery
Domenico Corica, Claudio Romano
The incidence of inflammatory bowel disease (IBD) has increased steadily worldwide, both in adult and in children; approximately 25% of IBD patients are diagnosed before the age of 18. The natural history of IBD is usually more severe in children than in adults, and can be associated with linear growth impairment, delayed puberty onset, reduced bone mass index, malnutrition, and the need for surgery. Biological therapies, especially blocking tumor necrosis factor-α (TNFα), have radically modified the treatment strategies and disease course of IBD in children...
September 15, 2016: Journal of Clinical Gastroenterology
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