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biologic therapy in children

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https://www.readbyqxmd.com/read/28725977/the-effect-of-vitamin-d-and-calcium-supplementation-in-pediatric-steroid-sensitive-nephrotic-syndrome
#1
Sushmita Banerjee, Surupa Basu, Ananda Sen, Jayati Sengupta
BACKGROUND: Low serum levels of total 25-hydroxycholecalciferol (25(OH)D) occur in nephrotic syndrome (NS). We aimed to assess the effects of vitamin D3 and calcium supplementation on 25(OH)D levels, bone mineralization, and NS relapse rate in children with steroid-sensitive NS. METHODS: A randomized controlled trial (RCT) was performed in children with steroid-sensitive NS. The treatment group received vitamin D3 (60,000 IU orally, weekly for 4 weeks) and calcium supplements (500 to 1,000 mg/day for 3 months) after achieving NS remission...
July 19, 2017: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
https://www.readbyqxmd.com/read/28725955/biologics-in-juvenile-idiopathic-arthritis-a-narrative-review
#2
REVIEW
Federica Vanoni, Francesca Minoia, Clara Malattia
In the past years, pediatric rheumatology has seen a revolution in the treatments for rheumatic diseases, particularly juvenile idiopathic arthritis. Even if nonsteroidal anti-inflammatory drugs (NSAID), intra-articular corticosteroids (IAC) injections, and methotrexate remain the mainstay of the treatment for JIA patients, in aggressive disease, these treatments may be not sufficient to reach disease remission and to prevent long-term disability. Comprehension of immunological mechanisms involved in the pathogenesis of the diseases allowed to conceive new drugs targeting specific steps of the immune response...
July 20, 2017: European Journal of Pediatrics
https://www.readbyqxmd.com/read/28713235/connecting-the-brain-to-itself-through-an-emulation
#3
Mijail D Serruya
Pilot clinical trials of human patients implanted with devices that can chronically record and stimulate ensembles of hundreds to thousands of individual neurons offer the possibility of expanding the substrate of cognition. Parallel trains of firing rate activity can be delivered in real-time to an array of intermediate external modules that in turn can trigger parallel trains of stimulation back into the brain. These modules may be built in software, VLSI firmware, or biological tissue as in vitro culture preparations or in vivo ectopic construct organoids...
2017: Frontiers in Neuroscience
https://www.readbyqxmd.com/read/28700361/persistent-pain-in-cancer-survivors
#4
Elaine G Boland, Sam H Ahmedzai
PURPOSE OF REVIEW: As people are living longer after a diagnosis and primary treatment for cancer, or indeed living with cancer as a chronic disease, new problems are emerging in this growing population of so-called 'survivors'. Persistent or chronic pain is one of the commonest complaints, arising from the tissue damage caused by the original neoplasm, consequences of surgery and other therapies, and - especially in older people - multimorbidity. This review explores some of the principle causes and mechanisms of this phenomenon and reviews the evidence for their management...
July 11, 2017: Current Opinion in Supportive and Palliative Care
https://www.readbyqxmd.com/read/28698772/examining-the-causes-of-autism
#5
David G Amaral
Autism is a broad, complex, and increasingly important brain disorder. New data from the Center for Disease Control and Prevention indicate that one in sixty-eight children is born with some degree of autism. Autism is also more common in males by a four to one ratio. Making it especially difficult to discuss in finite, conclusive terms is the fact that there is no biological test for autism; diagnosis is based on behavior, and the only verified treatment is intensive behavior therapy. Our author, one of the nation's foremost researchers on autism, examines the prenatal factors that contribute to the disorder...
January 2017: Cerebrum: the Dana Forum on Brain Science
https://www.readbyqxmd.com/read/28691434/universal-antiretroviral-therapy-for-hiv-infected-children-a-review-of-the-benefits-and-risks-to-consider-during-implementation
#6
Linda Barlow-Mosha, Victor Musiime, Mary-Ann Davies, Andrew J Prendergast, Philippa Musoke, George Siberry, Martina Penazzato
BACKGROUND: The 2016 World Health Organization (WHO) consolidated guidelines on the use of antiretroviral drugs for treating and preventing HIV infection, recommended to start all HIV-infected children on antiretroviral therapy (ART). Here, we explore the possible benefits and risks of implementing universal ART for all HIV-infected children and adolescents and outline some of the key considerations that led to the 2016 revision of WHO guidelines. METHODS: We conducted a review of the published data from 2000 to 2016, to ascertain the clinical and programmatic benefits, as well as the risks of implementing universal ART for all children...
June 27, 2017: Journal of the International AIDS Society
https://www.readbyqxmd.com/read/28691235/outcomes-of-the-conventional-and-biological-treatment-approaches-for-the-management-of-caries-in-the-primary-dentition
#7
Alaa BaniHani, Monty Duggal, Jack Toumba, Chris Deery
BACKGROUND: In the last few years, conventional restorations including complete removal of carious tissue (CT) with or without pulp therapy for the treatment of carious lesions (CL) in primary teeth have been challenged and a more biological approach has been suggested. This approach involves the use of less invasive techniques which alter the environment of the CL isolating it from the cariogenic biofilm and substrate. Two of these treatment approaches that are becoming increasingly widely accepted and used in paediatric dentistry are the Hall Technique and indirect pulp capping (IPC)...
July 9, 2017: International Journal of Paediatric Dentistry
https://www.readbyqxmd.com/read/28677575/from-class-waivers-to-precision-medicine-in-paediatric-oncology
#8
REVIEW
Andrew D J Pearson, Stefan M Pfister, Andre Baruchel, Jean-Pierre Bourquin, Michela Casanova, Louis Chesler, François Doz, Angelika Eggert, Birgit Geoerger, David T W Jones, Pamela R Kearns, Jan J Molenaar, Bruce Morland, Gudrun Schleiermacher, Johannes H Schulte, Josef Vormoor, Lynley V Marshall, C Michel Zwaan, Gilles Vassal
New drugs are crucially needed for children with cancer. The European Paediatric Regulation facilitates paediatric class waivers for drugs developed for diseases only occurring in adults. In this Review, we retrospectively searched oncology drugs that were class waivered between June, 2012, and June, 2015. 147 oncology class waivers were confirmed for 89 drugs. Mechanisms of action were then assessed as potential paediatric therapeutic targets by both a literature search and an expert review. 48 (54%) of the 89 class-waivered drugs had a mechanisms of action warranting paediatric development...
July 2017: Lancet Oncology
https://www.readbyqxmd.com/read/28662353/neuroblastoma-in-children-update-on-clinicopathologic-and-genetic-prognostic-factors
#9
Atif A Ahmed, Lei Zhang, Naresh Reddivalla, Maxine Hetherington
Neuroblastoma is the most common extracranial solid tumor in childhood accounting for 8-10% of all childhood malignancies. The tumor is characterized by a spectrum of histopathologic features and a heterogeneous clinical phenotype. Modern multimodality therapy results in variable clinical response ranging from cure in localized tumors to limited response in aggressive metastatic disease. Accurate clinical staging and risk assessment based on clinical, surgical, biologic and pathologic criteria are of pivotal importance in assigning prognosis and planning effective treatment approaches...
June 29, 2017: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/28660181/psychosocial-challenges-associated-with-caregiving-in-the-context-of-pediatric-hiv-in-rural-eastern-cape
#10
Antonio G Lentoor
BACKGROUND: While survival among human immunodeficiency virus (HIV)-infected children has increased due to combination antiretroviral therapy, many children remain vulnerable to the adverse effects of poverty and family disruptions as a result of the loss of one or both biological parents to acquired immunodeficiency syndrome. The aim of this qualitative study was to develop an understanding of the psychosocial challenges experienced by caregivers caring for a child with perinatally acquired HIV...
2017: Frontiers in Public Health
https://www.readbyqxmd.com/read/28649891/new-targeted-therapies-for-relapsed-pediatric-acute-lymphoblastic-leukemia
#11
Joanna Pierro, Laura E Hogan, Teena Bhatla, William L Carroll
The improvement in outcomes for children with acute lymphoblastic leukemia (ALL) is one of the greatest success stories of modern oncology however the prognosis for patients who relapse remains dismal. Recent discoveries by high resolution genomic technologies have characterized the biology of relapsed leukemia, most notably pathways leading to the drug resistant phenotype. These observations open the possibility of targeting such pathways to prevent and/or treat relapse. Likewise, early experiences with new immunotherapeutic approaches have shown great promise...
July 5, 2017: Expert Review of Anticancer Therapy
https://www.readbyqxmd.com/read/28647791/premonitory-symptoms-of-migraine-in-childhood-and-adolescence
#12
REVIEW
N Karsan, P Prabhakar, P J Goadsby
PURPOSE OF REVIEW: Premonitory symptoms in migraine; symptoms occurring before the onset of migraine pain or aura, are an increasingly recognised area of interest within headache research. It has been recently documented in the literature that these symptoms also occur in children and adolescents, with a comparable phenotype to adults. This review discusses the wide presentation of premonitory symptoms in migraine in children and adolescents, and the importance of understanding how these early symptoms are mediated in order to ensure that targeted abortive therapies are developed in the future...
July 2017: Current Pain and Headache Reports
https://www.readbyqxmd.com/read/28644430/the-clinical-landscape-for-sma-in-a-new-therapeutic-era
#13
REVIEW
K Talbot, E F Tizzano
Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for translational medicine. The childhood onset motor neuron disorder spinal muscular atrophy (SMA) has been viewed as one of the more tractable targets for molecular therapy, due to a detailed understanding of the molecular genetic basis of the disease. In SMA, inactivating mutations in the SMN1 gene can be partially compensated for by limited expression of SMN protein from a variable number of copies of the SMN2 gene, which provides both a molecular explanation for phenotypic severity and a target for therapy...
June 23, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28643357/nut-carcinoma-in-children-and-adults-a-multicenter-retrospective-study
#14
Lauriane Lemelle, Gaëlle Pierron, Paul Fréneaux, Sophie Huybrechts, Alexandra Spiegel, Dominique Plantaz, Morbize Julieron, Sophie Dumoucel, Antoine Italiano, Fréderic Millot, Christophe Le Tourneau, Guy Leverger, Pascal Chastagner, Matthieu Carton, Daniel Orbach
BACKGROUND: Nuclear protein of the testis (NUT) carcinoma (formerly NUT midline carcinoma) is an aggressive tumor defined by the presence of NUT rearrangement with a poor prognosis. This rare cancer is underdiagnosed and poorly treated. OBJECTIVE: The primary objective of this study was to describe the clinical, radiologic, and biological features of NUT carcinoma. The secondary objective was to describe the various treatments and assess their efficacy. METHODS: This retrospective multicenter study was based on review of the medical records of children and adults with NUT carcinoma with specific rearrangement or positive anti-NUT nuclear staining (>50%)...
June 23, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28640705/genomic-analysis-of-childhood-brain-tumors-methods-for-genome-wide-discovery-and-precision-medicine-become-mainstream
#15
Stephen C Mack, Paul A Northcott
Recent breakthroughs in next-generation sequencing technology and complementary genomic platforms have transformed our capacity to interrogate the molecular landscapes of human cancers, including childhood brain tumors. Numerous high-throughput genomic studies have been reported for the major histologic brain tumor entities diagnosed in children, including interrogations at the level of the genome, epigenome, and transcriptome, many of which have yielded essential new insights into disease biology. The nature of these discoveries has been largely platform dependent, exemplifying the usefulness of applying different genomic and computational strategies, or integrative approaches, to address specific biologic and/or clinical questions...
July 20, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28640697/current-clinical-challenges-in-childhood-ependymoma-a-focused-review
#16
Thomas E Merchant
Ependymoma is a locally aggressive tumor with metastatic potential that arises in diverse locations throughout the brain and spine in children. Tumor and treatment may result in significant morbidity. Cure remains elusive for many patients owing to diverse biology and resistance to conventional therapy. The implementation of systematic postoperative irradiation in clinical trials during the past 20 years has increased the proportion of patients achieving durable disease control with excellent results, as measured by objective functional outcome measures...
July 20, 2017: Journal of Clinical Oncology: Official Journal of the American Society of Clinical Oncology
https://www.readbyqxmd.com/read/28632924/metabolomics-as-a-driver-in-advancing-precision-medicine-in-sepsis
#17
Michelle Eckerle, Lilliam Ambroggio, Michael Puskarich, Brent Winston, Alan E Jones, Theodore J Standiford, Kathleen A Stringer
OBJECTIVE: The objective of this review is to explain the science of metabolomics-a science of systems biology that measures and studies endogenous small molecules (metabolites) that are present in a single biological sample-and its application to the diagnosis and treatment of sepsis. In addition, we discuss how discovery through metabolomics can contribute to the development of precision medicine targets for this complex disease state and the potential avenues for those new discoveries to be applied in the clinical environment...
June 20, 2017: Pharmacotherapy
https://www.readbyqxmd.com/read/28628351/sleep-in-adolescents-and-young-adults-in-the-year-after-cancer-treatment
#18
Lauren C Daniel, Richa Aggarwal, Lisa A Schwartz
PURPOSE: Adolescents and young adults (AYA) with cancer are a vulnerable group facing more intense treatments, higher symptom burden, and poorer treatment outcomes relative to younger children. Sleep disruption is common during cancer treatment and sleep problems persist into adulthood for some survivors of AYA cancer. The developmental period of adolescence/emerging adulthood confers greater biological and behavioral risk for insufficient sleep relative to older or younger ages. Thus, understanding AYA sleep disturbances shortly after completing treatment can inform interventions to manage cancer-related symptoms and improve quality of life...
June 19, 2017: Journal of Adolescent and Young Adult Oncology
https://www.readbyqxmd.com/read/28627000/pencil-beam-scanned-protons-for-the-treatment-of-patients-with-ewing-sarcoma
#19
Damien C Weber, Fritz R Murray, Dora Correia, Alessandra Bolsi, Martina Frei-Welte, Alessia Pica, Antony J Lomax, Ralf Schneider, Barbara Bachtiary
BACKGROUND: Few data exist regarding the clinical outcome of patients with Ewing sarcoma (EWS) treated with pencil beam scanning proton therapy (PT). We report the outcome of children, adolescents and young adults (AYA) treated with PT at the Paul Scherrer Institute. MATERIALS: Thirty-eight patients (median age, 9.9 years) received a median dose of 54.9 Gy(RBE) (where RBE is relative biologic effectiveness). Size of the tumor ranged from 1.7 to 24 cm. Most common primary site was axial/pelvic (n = 27; 71%)...
June 19, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28620836/sox2-immunity-and-tissue-resident-memory-in-children-and-young-adults-with-glioma
#20
Juan C Vasquez, Anita Huttner, Lin Zhang, Asher Marks, Amy Chan, Joachim M Baehring, Kristopher T Kahle, Kavita M Dhodapkar
Therapies targeting immune checkpoints are effective in tumors with a high mutation burden that express multiple neo-antigens. However, glial tumors including those seen in children carry fewer mutations and there is an unmet need to identify new antigenic targets of anti-tumor immunity. SOX2 is an embryonal stem cell antigen implicated in the biology of glioma initiating cells. Expression of SOX2 by pediatric glial tumors and the capacity of the immune system in these patients to recognize SOX2 has not been previously studied...
June 15, 2017: Journal of Neuro-oncology
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