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pediatric therapeutics

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https://www.readbyqxmd.com/read/28098869/core%C3%A2-shell-lipid-polymer-nanoparticles-for-combined-chemo%C3%A2-and-gene-therapy-of-childhood-head-and-neck-cancers
#1
Tiesong Zhang, Jing Ma, Chao Li, Ken Lin, Fan Lou, Hongchao Jiang, Yingqin Gao, Yanli Yang, Cheng Ming, Biao Ruan
Pediatric head and neck cancers account for overall 12% of all pediatric cancers. Despite recent advances in therapeutic modalities, children with tumor metastasis have poor prognosis. Therefore, there is an unmet need for new and effective treatment modalities for pediatric head and neck cancers. The present study describes a simple and efficient method for fabrication of cationic lipid‑polymer hybrid nanoparticles (CLPNs) for co‑delivery of cisplatin (CDDP) and DNA (CDDP/DNA CLPNs) for the therapy of childhood head and neck cancers...
January 13, 2017: Oncology Reports
https://www.readbyqxmd.com/read/28098235/molecular-characterization-of-pediatric-restrictive-cardiomyopathy-from-integrative-genomics
#2
Tara N Rindler, Robert B Hinton, Nathan Salomonis, Stephanie M Ware
Pediatric restrictive cardiomyopathy (RCM) is a genetically heterogeneous heart disease with limited therapeutic options. RCM cases are largely idiopathic; however, even within families with a known genetic cause for cardiomyopathy, there is striking variability in disease severity. Although accumulating evidence implicates both gene expression and alternative splicing in development of dilated cardiomyopathy (DCM), there have been no detailed molecular characterizations of underlying pathways dysregulated in RCM...
January 18, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28093934/efficacy-and-safety-of-oral-sildenafil-in-treatment-of-pediatric-head-and-neck-lymphatic-malformations
#3
Shengcai Wang, Jie Zhang, Wentong Ge, Yuanhu Liu, Yongli Guo, Yue Liu, Ning Wang, Yuzhu He, Jun Tai, Xin Ni
CONCLUSIONS: Low dose oral sildenafil citrate tablets are safe and effective to decrease the lesions volume in some LMs children, especially with macrocystic lesion or mixed type. OBJECTIVE: To evaluate the efficacy and safety of oral sildenafil in treatment of pediatric head and neck lymphatic malformations (LMs). SUBJECTS AND METHODS: In this open-label study, 21 children diagnosed with LMs were given oral sildenafil and stopped at week 24...
January 17, 2017: Acta Oto-laryngologica
https://www.readbyqxmd.com/read/28093429/paediatric-ed-bipap-continuous-quality-improvement-programme-with-patient-analysis-2005-2013
#4
Thomas Abramo, Abby Williams, Samaiya Mushtaq, Mark Meredith, Rawle Sepaule, Kristen Crossman, Cheryl Burney Jones, Suzanne Godbold, Zhuopei Hu, Todd Nick
OBJECTIVE: In paediatric moderate-to-severe asthmatics, there is significant bronchospasm, airway obstruction, air trapping causing severe hyperinflation with more positive intraplural pressure preventing passive air movement. These effects cause an increased respiratory rate (RR), less airflow and shortened inspiratory breath time. In certain asthmatics, aerosols are ineffective due to their inadequate ventilation. Bilevel positive airway pressure (BiPAP) in acute paediatric asthmatics can be an effective treatment...
January 16, 2017: BMJ Open
https://www.readbyqxmd.com/read/28092280/challenges-and-opportunities-to-enhance-global-drug-development-in-neonates
#5
Alexandra M Smith, Jonathan M Davis
PURPOSE OF REVIEW: This study reviews the history of neonatal drug development, recent legislative efforts designed to facilitate the study of therapeutic agents in neonates, and future steps necessary to advance drug development. RECENT FINDINGS: Although many federal regulations have been introduced over the past 15 years to encourage pediatric and neonatal drug development, the majority of medications that are used in the Neonatal Intensive Care Unit are not approved by the Food and Drug Administration (FDA) for use in neonates...
January 13, 2017: Current Opinion in Pediatrics
https://www.readbyqxmd.com/read/28091756/-recommendation-on-temperature-management-after-cardiopulmonary-arrest-and-severe-traumatic-brain-injury-in-childhood-beyond-the-neonatal-period-statement-of-the-german-society-for-neonatology-and-pediatric-intensive-care-medicine%C3%A2-gnpi-and-the-scientific-working
#6
S Brenner, C Eich, G Rellensmann, M U Schuhmann, T Nicolai, F Hoffmann
The available data on the effectiveness of therapeutic hypothermia in different patient groups are heterogeneous. Although the benefits have been proven for some collectives, recommendations for the use of hypothermia treatment in other groups are based on less robust data and conclusions by analogy. This article gives a review of the current evidence of temperature management in all age groups and based on this state of knowledge, recommends active temperature management with the primary aim of strict normothermia (36-36...
January 13, 2017: Der Anaesthesist
https://www.readbyqxmd.com/read/28091414/acute-myeloid-leukemia-advancements-in-diagnosis-and-treatment
#7
REVIEW
Meng-Ge Yu, Hu-Yong Zheng
OBJECTIVE: Leukemia is the most common pediatric malignancy and a major cause of morbidity and mortality in children. Among all subtypes, a lack of consensus exists regarding the diagnosis and treatment of acute myeloid leukemia (AML). Patient survival rates have remained modest for the past three decades in AML. Recently, targeted therapy has emerged as a promising treatment. DATA SOURCES: We searched the PubMed database for recently published research papers on diagnostic development, target therapy, and other novel therapies of AML...
2017: Chinese Medical Journal
https://www.readbyqxmd.com/read/28090779/preparation-and-in-vitro-in-vivo-evaluation-of-metformin-hydrochloride-rectal-dosage-forms-for-treatment-of-patients-with-type-ii-diabetes
#8
Abdel-Azim Zaghloul, Ahmad Lila, Fathy Abd-Allah, Aly Nada
BACKGROUND: Metformin hydrochloride (MtHCL) is an oral antidiabetic drug and has many other therapeutic benefits. It has poor bioavailability, narrow absorption window and extensive liver metabolism. Moreover, children and elders face difficulty to swallow the commercial oral tablets. OBJECTIVES: Preparation, in vitro/in vivo evaluation of MtHCL suppositories for rectal administration to solve some of these problems. METHODS: Suppository fatty bases (Witepsol®, Suppocire® and Massa®; different grades) and PEG bases 1000, 4000 and 6000 (different ratios), were used to prepare rectal suppository formulations each containing 500 mg drug...
January 16, 2017: Journal of Drug Targeting
https://www.readbyqxmd.com/read/28090473/three-year-old-male-with-multiple-dieulafoy-lesions-treated-with-epinephrine-injections-via-therapeutic-endoscopy
#9
Christina L Baldwin, Michael Wilsey
Dieulafoy lesions, vascular anomalies typically found along the gastrointestinal tract, have been viewed as rare and obscure causes of sudden intestinal bleeding, especially in pediatric patients. Since their discovery in the late 19th century, the reported incidence has increased. This is due to an increased awareness of, and knowledge about, their presentation and to advanced endoscopic diagnosis and therapy. Our patient was a three-year-old male, without a complex medical history. He presented to the emergency department with acute hematemesis with blood clots and acute anemia requiring blood transfusion...
December 2016: Pediatric Gastroenterology, Hepatology & Nutrition
https://www.readbyqxmd.com/read/28088906/epratuzumab-and-blinatumomab-as-therapeutic-antibodies-for-treatment-of-pediatric-acute-lymphoblastic-leukemia-current-status-and-future-perspectives
#10
Raffaella Franca, Diego Favretto, Marilena Granzotto, Giuliana Decorti, Marco Rabusin, Gabriele Stocco
BACKGROUND: More than 85% of children affected by acute lymphoblastic leukemia (ALL) are successfully treated; however relapse remains a remarkable clinical concern, with 50-60% of relapsing patients facing a fatal outcome. Management of relapsed patients includes standardized intensive risk-adapted regimens based on conventional drugs, and hematopoietic stem cells transplantation for patients with unfavourable features. Biological drugs, in particular the monoclonal antibody epratuzumab and the bi-functional recombinant single chain peptide blinatumomab, have been recently recognized as novel potential agents to be integrated in salvage ALL therapy to further improve rescue outcome...
January 12, 2017: Current Medicinal Chemistry
https://www.readbyqxmd.com/read/28087825/incidence-and-predictors-of-epilepsy-after-pediatric-arterial-ischemic-stroke
#11
Lori L Billinghurst, Lauren A Beslow, Nicholas S Abend, Michael Uohara, Laura Jastrzab, Daniel J Licht, Rebecca N Ichord
OBJECTIVE: To determine the cumulative incidence and clinical predictors of remote symptomatic seizures and epilepsy after pediatric arterial ischemic stroke (AIS). METHODS: We performed a retrospective analysis of 218 participants with neonatal AIS (NAIS), presumed perinatal AIS (PPAIS), and childhood AIS (CAIS) from a single-center prospective consecutive cohort enrolled from 2006 to 2014. Medical records were reviewed for timing, semiology, and treatment of acute symptomatic seizures, remote symptomatic seizures (RSS), and epilepsy...
January 13, 2017: Neurology
https://www.readbyqxmd.com/read/28081040/azathioprine-therapy-in-a-pediatric-tpmt-deficient-patient-still-an-option
#12
S A W van Moorsel, N Bevers, M Meurs, L K van Rossum, P M Hooymans, D R Wong
We describe the case of a pediatric patient on azathioprine therapy with previously undiagnosed homozygote thiopurine S-methyltransferase (TPMT) deficiency, resulting in myelotoxic thiopurine metabolite levels. The patient was successfully treated with a very low azathioprine dose of 50 mg once a week (4% of standard dose), guided by frequent thiopurine metabolite measurement and a close clinical surveillance. We demonstrate that azathioprine therapy still might be an effective and safe therapeutic option in pediatric thiopurine S-methyltransferase-deficient IBD patients...
February 2017: Therapeutic Drug Monitoring
https://www.readbyqxmd.com/read/28079914/intravenous-immunoglobulin-in-pediatric-rheumatology-when-to-use-it-and-what-is-the-evidence
#13
Martha M Rodriguez, Linda Wagner-Weiner
Intravenous immunoglobulin (IVIG) is given to children with a variety of rheumatologic illnesses. The mechanism of action by which it exerts therapeutic effects is not well understood and likely differs in the medical conditions for which it is given. IVIG is approved by the US Food and Drug Administration and is the standard of care for Kawasaki disease, but most IVIG use in pediatric rheumatology is "off-label. " The literature supports the use of IVIG for juvenile dermatomyositis, although it is unclear whether its use should be limited to those children with more severe or refractory disease...
January 1, 2017: Pediatric Annals
https://www.readbyqxmd.com/read/28079913/intravenous-immunoglobulin-in-the-treatment-of-hematologic-disorders-in-pediatrics
#14
Gabriela Villanueva, Jill L O de Jong, Jennifer L McNeer
Intravenous immunoglobulin (IVIG) is pooled immunoglobulin G derived from human blood donors. It was introduced in the early 1980s to treat immunodeficiency disorders. Since then, its use has expanded to other fields such as neurology, rheumatology, and hematology. IVIG has been used to provide passive immunity in qualitative and quantitative immunoglobulin disorders, to neutralize antibodies in immune-mediated diseases, and as an immune modulatory agent. The difficulty of producing IVIG in high quantities, in addition to a growing list of "off-label" indications, has resulted in a worldwide shortage and increase in cost...
January 1, 2017: Pediatric Annals
https://www.readbyqxmd.com/read/28079912/intravenous-immunoglobulin-in-the-treatment-of-primary-immunodeficiency-diseases
#15
Deirdre De Ranieri, Nana Sarkoah Fenny
Intravenous immunoglobulin (IVIG) has been used as antibody replacement therapy in primary immunodeficiency diseases (PIDDs) for more than 50 years. Its role as a therapeutic agent has expanded over the past couple of decades as its anti-inflammatory and immune-modulatory mechanisms of action have been elucidated. It is now used "off-label" to treat other autoimmune diseases. This article focuses on the role of IVIG in the treatment of PIDDs characterized by absent or deficient antibody production. Replacement doses are given on a monthly basis in these conditions as a prophylactic measure to prevent acute and serious bacterial infections...
January 1, 2017: Pediatric Annals
https://www.readbyqxmd.com/read/28079601/infliximab-optimization-based-on-therapeutic-drug-monitoring-in-pediatric-ibd
#16
Tatyana Hofmekler, Madeline Bertha, Courtney McCracken, Benadette Martineau, Elizabeth McKinnon, Bess T Schoen, Barbara O McElhanon, Gayathri Tenjarla, Subra Kugathasan, Cary Sauer
BACKGROUND: Infliximab (IFX) is an effective treatment for the management of moderate to severe inflammatory bowel disease (IBD). Low serum IFX levels are associated with the development of antibodies to IFX (ATI) which subsequently associated with clinical relapse and increased morbidity. The purpose of this study is to examine the relationship between dose and interval to IFX level. Secondary goal is to evaluate the relationship between IFX level and ATI in a pediatric IBD population...
June 21, 2016: Journal of Pediatric Gastroenterology and Nutrition
https://www.readbyqxmd.com/read/28078488/hydroxyurea-treatment-effect-on-children-with-sickle-cell-disease-and-obstructive-sleep-apnea
#17
Anthony J Grady, Jane S Hankins, Brent Haberman, Robert Schoumacher, Rose Mary Stocks
BACKGROUND: While hydroxyurea is the mainstay of treatment for many of the comorbidities associated with sickle cell disease, its effect on obstructive sleep apnea has not been fully investigated. The purpose of this project is to help characterize the effects of hydroxyurea on obstructive sleep apnea in children with sickle cell disease and determine its therapeutic role in the condition. METHODS: Chart review was conducted on two pediatric patients with sickle cell disease who experienced resolution of obstructive sleep apnea following hydroxyurea administration...
January 11, 2017: Sleep & Breathing, Schlaf & Atmung
https://www.readbyqxmd.com/read/28074348/challenges-and-opportunities-in-the-development-of-hiv-medications-in-pediatric-patients
#18
Gueorgui Dubrocq, Natella Rakhmanina, B Ryan Phelps
Successful management of pediatric HIV disease requires high therapeutic efficacy and adherence, which can be achieved by providing affordable, easy to store, and palatable antiretrovirals. Current challenges in pediatric antiretroviral drug development include poor palatability, large pill size, limited oral liquid formulations, and few incentives for development by drug manufacturers as the number of children with HIV continues to decline due to successful worldwide preventive interventions and improved access to antiretrovirals...
January 10, 2017: Paediatric Drugs
https://www.readbyqxmd.com/read/28074288/clinical-research-participation-among-adolescent-and-young-adults-at-an-nci-designated-comprehensive-cancer-center-and-affiliated-pediatric-hospital
#19
Stacy D Sanford, Jennifer L Beaumont, Mallory A Snyder, Jennifer Reichek, John M Salsman
PURPOSE: Minimal clinical trial participation among adolescents and young adults (AYAs) with cancer limits scientific progress and ultimately their clinical care and outcomes. These analyses examine the current state of AYA clinical research participation at a Midwestern comprehensive cancer center and affiliated pediatric hospital to advise program development and increase availability of trials and AYA participation. Enrollment is examined across all diagnoses, the entire AYA age spectrum (15-39), and both cancer therapeutic and supportive care protocols...
January 10, 2017: Supportive Care in Cancer: Official Journal of the Multinational Association of Supportive Care in Cancer
https://www.readbyqxmd.com/read/28074279/identification-and-clinical-course-of-166-pediatric-cardiac-tumors
#20
Lin Shi, Lanping Wu, Huijuan Fang, Bo Han, Jialun Yang, Xiaojin Ma, Fang Liu, Yongwei Zhang, Tingting Xiao, Min Huang, Meirong Huang
: The aim of this study was to investigate the pathological classifications, clinical features, and natural history of pediatric cardiac tumors to provide a basis for the selection of an appropriate therapeutic method. The medical records of in- or outpatients with cardiac tumors at four hospitals were classified to analyze various types of tumor growth locations, clinical manifestations, surgical indications, and long-term follow-up results. There were 166 patients, including 158 with primary cardiac tumors, six with metastatic cardiac tumors, and two with unclassified cardiac tumors...
January 10, 2017: European Journal of Pediatrics
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