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pediatric clinical pharmacology

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https://www.readbyqxmd.com/read/28516235/clinical-imaging-and-genotypical-features-of-three-deceased-and-five-surviving-cases-with-ada2-deficiency
#1
Sezgin Sahin, Amra Adrovic, Kenan Barut, Serdal Ugurlu, Eda Tahir Turanli, Huri Ozdogan, Ozgur Kasapcopur
Deficiency of adenosine deaminase type 2 (DADA2) is a rare form of autoinflammatory disorder with limited reported cases. In this paper, we have presented the clinico-immunological, radiological and genetic characteristics of five surviving and three deceased childhood-onset DADA2 patients. We aimed to compare surviving and deceased patients in terms of clinical features and treatment modalities. Moreover, we have evaluated the causes of death in our DADA2 subjects together with the previously reported cases...
May 17, 2017: Rheumatology International
https://www.readbyqxmd.com/read/28510298/drugs-for-children
#2
S Ito
The "Therapeutic Orphan" status of children is gradually improving, but our efforts to eliminate off-label/off-evidence use must be sustained for safer and more effective pediatric drug therapy. This is even more important in resource-scarce settings, because of the increasing child population in those regions. The juncture of pediatric pharmacology and global child health represents an emerging field of clinical pharmacology illuminating a crucial societal need on a global scale.
June 2017: Clinical Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/28506869/model-based-clinical-dose-optimization-for-phenobarbital-in-neonates-an-illustration-of-the-importance-of-data-sharing-and-external-validation
#3
Swantje Völler, Robert B Flint, Leo M Stolk, Pieter L J Degraeuwe, Sinno H P Simons, Paula Pokorna, David M Burger, Ronald de Groot, Dick Tibboel, Catherijne A J Knibbe
BACKGROUND: Particularly in the pediatric clinical pharmacology field, data-sharing offers the possibility of making the most of all available data. In this study, we utilize previously collected therapeutic drug monitoring (TDM) data of term and preterm newborns to develop a population pharmacokinetic model for phenobarbital. We externally validate the model using prospective phenobarbital data from an ongoing pharmacokinetic study in preterm neonates. METHODS: TDM data from 53 neonates (gestational age (GA): 37 (24-42) weeks, bodyweight: 2...
May 12, 2017: European Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/28490206/pharmacokinetics-pharmacodynamics-and-pharmacogenetics-associated-with-nonsteroidal-anti-inflammatory-drugs-and-opioids-in-pediatric-cancer-patients
#4
Jonathan Constance, Sarah Campbell, Amit A Somani, Venkata Yellepeddi, Katie Owens, Catherine Sherwin
Advancing appropriate and adequate analgesic pharmacotherapy in pediatric patients with cancer is an area of clinical need. Few studies have been performed to evaluate the selection of an analgesic and appropriate dosing corresponding to analgesic effect among pediatric cancer patients. This review describes information related to pharmacokinetic, pharmacodynamic, and pharmacogenomic (when applicable) considerations for analgesics that are commonly used to manage pain experienced by pediatric patients with cancer...
May 11, 2017: Expert Opinion on Drug Metabolism & Toxicology
https://www.readbyqxmd.com/read/28487636/elamipretide-ss-31-ameliorates-isoflurane-induced-long-term-impairments-of-mitochondrial-morphogenesis-and-cognition-in-developing-rats
#5
Jing Wu, Shuangying Hao, Xiao-Ru Sun, Hui Zhang, Huihui Li, Hongting Zhao, Mu-Huo Ji, Jian-Jun Yang, Kuanyu Li
Mitochondria are supposed to be involved in the early pathogenesis of general anesthesia (GA)-induced neurotoxicity and long-term cognitive deficits in developing brains. However, effective pharmacologic agents targeted on mitochondria during GA exposure are lacking. This study explores the protective effects of mitochondrion-targeted antioxidant elamipretide (SS-31) on mitochondrial morphogenesis and cognition in developing rats exposed to isoflurane. Rat pups at postnatal day (PND) 7 were exposed to 1.5% isoflurane for 6 h following intraperitoneal administration of elamipretide or vehicle with 30 min interval...
2017: Frontiers in Cellular Neuroscience
https://www.readbyqxmd.com/read/28481700/pediatric-status-epilepticus-improved-management-with-new-drug-therapies
#6
Alberto Verrotti, Michela Ambrosi, Piero Pavone, Pasquale Striano
Status Epilepticus (SE) is the most common neurological emergency of childhood. It requires prompt administration of appropriately selected anti-seizure medications. Areas covered: Following a distinction between estabilished and emergent drugs, we present pharmacological treatment options and their clinical utility in children, with a short mention on alternatives to drug treatment. We also propose an algorithm for the management of pediatric SE. For this review a Pubmed, Medline and Embase search was performed...
May 19, 2017: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/28461862/effectiveness-of-coenzyme-q10-on-echocardiographic-parameters-of-patients-with-duchenne-muscular-dystrophy
#7
Forod Salehi, Aliakbar Zeinaloo, Hamid Reza Riasi, Alireza Sepehri Shamloo
BACKGROUND: Myocardial damage is a common complication in patients with Duchenne muscular dystrophy (DMD) that occurs due to myocardial replacement by fat and fibrosis. In recent years, efforts have been made toward finding new pharmacological agents with fewer complications which can be used as prophylactic before the symptoms. Coenzyme Q10 plays a central role in production of bioenergy in heart muscle and antioxidant in reperfusion condition of myocardial damaged muscle and leads to membrane stability and prevents cell death...
March 2017: Electronic Physician
https://www.readbyqxmd.com/read/28457820/differential-roles-for-akt-and-mtorc1-in-the-hypertrophy-of-pten-mutant-neurons-a-cellular-model-of-brain-overgrowth-disorders
#8
Ina Nikolaeva, Tatiana M Kazdoba, Beth Crowell, Gabriella D'Arcangelo
Mutations in the PI3K/Akt/mTOR signaling pathway or in the upstream negative regulator Pten cause human brain overgrowth disorders, such as focal cortical dysplasia and megalencephaly, and are characterized by the presence of hypertrophic neurons. These disorders often have a pediatric onset and a high comorbidity with drug-resistant epilepsy; however, effective pharmacological treatments are lacking. We established forebrain excitatory neuron-specific Pten-deficient cultures as an in vitro model of brain overgrowth disorders, and investigated the effects of this Pten mutation on PI3K/Akt/mTOR signaling and neuronal growth...
April 27, 2017: Neuroscience
https://www.readbyqxmd.com/read/28445187/the-effects-of-inherited-nudt15-polymorphisms-on-thiopurine-active-metabolites-in-japanese-children-with-acute-lymphoblastic-leukemia
#9
Takaya Moriyama, Rina Nishii, Ting-Nien Lin, Kentaro Kihira, Hidemi Toyoda, Nersting Jacob, Motohiro Kato, Katsuyoshi Koh, Hiroto Inaba, Atsushi Manabe, Kjeld Schmiegelow, Jun J Yang, Hiroki Hori
Thiopurines [e.g. mercaptopurine (MP)] are widely used as chemotherapeutic agents in the treatment of pediatric acute lymphoblastic leukemia with dose-limiting hematopoietic toxicity. Recently, germline variants in NUDT15 have been identified as a major genetic cause for MP-related bone marrow suppression, and there is increasing interest in the clinical implementation of NUDT15 genotype-guided MP dose individualization. Therefore, we sought to evaluate the effects of NUDT15 on thiopurine metabolism and identify pharmacologic markers to inform NUDT15 genotype-guided MP dosing...
June 2017: Pharmacogenetics and Genomics
https://www.readbyqxmd.com/read/28430759/evaluation-of-guidelines-for-injured-children-at-high-risk-for-venous-thromboembolism-a-prospective-observational-study
#10
Rachel M Landisch, Sheila J Hanson, Laura D Cassidy, Kristin Braun, Rowena C Punzalan, David M Gourlay
BACKGROUND: Pharmacologic prophylaxis for venous thromboembolism (VTE) is a widely accepted practice in adult trauma patients to prevent associated morbidity and mortality. However, VTE prophylaxis has not been standardized in injured pediatric patients. Our institution identified factors potentially associated with a high risk of VTE in critically injured children that led to prospective implementation of VTE prophylaxis guidelines. We hypothesize that the guidelines are accurate in predicting children at risk for VTE...
May 2017: Journal of Trauma and Acute Care Surgery
https://www.readbyqxmd.com/read/28428678/productive-cough-in-children-and-adolescents-view-from-primary-health-care-system
#11
REVIEW
Edin Begic, Zijo Begic, Amra Dobraca, Edo Hasanbegovic
Cough is one of the most common symptoms that doctor faces in working with pediatric population, and according to some characteristics of cough, doctors can often conclude localization, and sometimes even the nature of the disease that causes it. Cough is not only the physiological defense reaction, but a symptom of a disease. According to duration it can be acute, chronic and recidivist, recurrent and persistent, strong or discreet, caused by changes in body position and changes in outside temperature. Pathoanatomically it is divided into lobar, lobular, alveolar and interstitial, pathogenetically to bronchogenic and hematogenous, as well as in immuno competent and immunocompromised, and clinically on the local and inpatient (72 hours after hospital admission)...
February 2017: Medical Archives
https://www.readbyqxmd.com/read/28402139/long-term-open-label-safety-study-of-edivoxetine-monotherapy-in-children-and-adolescents-with-attention-deficit-hyperactivity-disorder
#12
Emel Serap Monkul Nery, Mark Bangs, Peng Liu, Jonna Ahl, David Perahia
OBJECTIVE: The purpose of this study was to assess the long-term safety and tolerability of edivoxetine, a selective norepinephrine reuptake inhibitor, which was being developed as monotherapy in pediatric attention-deficit/hyperactivity disorder (ADHD). METHODS: This was an open-label study of edivoxetine once daily dosing (0.1-0.3 mg/kg) as treatment for ADHD in children (6-11 years) and adolescents (12-17 years) to assess safety for up to 5 years. The safety assessments included the incidence of adverse events, vital signs, electrocardiograms, laboratory tests, percentile changes in weight, height, and body mass index, and Tanner staging...
April 12, 2017: Journal of Child and Adolescent Psychopharmacology
https://www.readbyqxmd.com/read/28401674/placebo-like-response-in-absence-of-treatment-in-children-with-autism
#13
Rebecca M Jones, Caroline Carberry, Amarelle Hamo, Catherine Lord
Caregiver report is the most common measure of change in pediatric psychiatry. Yet, placebo response rates pose significant challenges to reliably detect a treatment response. The present study simulated an eight-week clinical trial protocol for Autism Spectrum Disorder (ASD) for the purpose of testing the feasibility and validity of several outcome measures. Twenty caregivers answered questions about their child's behavior on their smartphone each week and completed a battery of paper questionnaires during weeks one and eight...
April 12, 2017: Autism Research: Official Journal of the International Society for Autism Research
https://www.readbyqxmd.com/read/28401060/therapeutic-targeting-of-histone-modifications-in-adult-and-pediatric-high-grade-glioma
#14
REVIEW
Maria J Williams, Will G B Singleton, Stephen P Lowis, Karim Malik, Kathreena M Kurian
Recent exciting work partly through The Cancer Genome Atlas has implicated epigenetic mechanisms including histone modifications in the development of both pediatric and adult high-grade glioma (HGG). Histone lysine methylation has emerged as an important player in regulating gene expression and chromatin function. Lysine (K) 27 (K27) is a critical residue in all seven histone 3 variants and the subject of posttranslational histone modifications, as it can be both methylated and acetylated. In pediatric HGG, two critical single-point mutations occur in the H3F3A gene encoding the regulatory histone variant H3...
2017: Frontiers in Oncology
https://www.readbyqxmd.com/read/28396105/eosinophilic-esophagitis-a-case-study-of-a-12-year-old-boy
#15
Katherine Kiely
Eosinophilic esophagitis is an underrecognized chronic condition in which symptoms of gastroesophageal reflux are accompanied by eosinophilia in the esophagus. This disorder affects both children and adults and is associated with a history of other atopic disorders, particularly food allergies. This case study discusses the epidemiology of eosinophilic esophagitis and the proper procedures for diagnosis, referral, and management using the dietary restrictions and pharmacologic treatment included in the clinical recommendations issued by the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition; the American Academy of Allergy, Asthma, and Immunology; and the American College of Gastroenterologists...
April 7, 2017: Journal of Pediatric Health Care
https://www.readbyqxmd.com/read/28389455/dinutuximab-for-maintenance-therapy-in-pediatric-neuroblastoma
#16
REVIEW
Lauren McGinty, Jill Kolesar
PURPOSE: The pharmacology, clinical efficacy, safety, dosage and administration, and role in therapy of dinutuximab for the treatment of high-risk pediatric neuroblastoma are reviewed. SUMMARY: Dinutuximab (Unituxin, United Therapeutics) is a novel monoclonal antibody recently approved for use in combination with granulocyte- macrophage colony-stimulating factor, interleukin-2, and isotretinoin for the treatment of pediatric patients with high-risk neuroblastoma...
April 15, 2017: American Journal of Health-system Pharmacy: AJHP
https://www.readbyqxmd.com/read/28373409/activation-of-tgf-%C3%AE-activated-kinase-1-promotes-colon-mucosal-pathogenesis-in-inflammatory-bowel-disease
#17
Zhiwei Liu, Fansheng Kong, Jefferson E Vallance, Eleana Harmel-Laws, Surya Amarachintha, Kris A Steinbrecher, Michael J Rosen, Sandip Bhattacharyya
The etiology and mechanisms for inflammatory bowel disease (IBD) are incompletely known. Determination of new, clinically important mechanisms for intestinal inflammation is imperative for developing effective therapies to treat IBD We sought to define a widespread mechanism for colon mucosal inflammation via the activation of TGF-β activated Kinase 1 (TAK1), a central regulator of cellular inflammatory actions. Activation of TAK1 and the downstream inflammatory signaling mediators was determined in pediatric patients with ulcerative colitis (UC) or Crohn's disease (CD) as well as in DSS-induced and spontaneous IBD in mice...
April 2017: Physiological Reports
https://www.readbyqxmd.com/read/28353057/levetiracetam-clinical-pharmacokinetic-monitoring-in-pediatric-patients-with-epilepsy
#18
REVIEW
Jason Tan, Vanessa Paquette, Marc Levine, Mary H H Ensom
Levetiracetam is a broad-spectrum antiepileptic drug (AED) with a unique mechanism of action. Older AEDs can cause serious short- and long-term adverse drug reactions and complications, rendering them undesirable to use in pediatric patients. Characteristics that make levetiracetam a near-ideal AED include its broad spectrum of activity, good tolerability profile, and minimal drug-drug interactions. Clinical pharmacokinetic monitoring (CPM) is often recommended in pediatric patients for certain AEDs due to large interindividual pharmacokinetic differences and unpredictable drug disposition...
March 28, 2017: Clinical Pharmacokinetics
https://www.readbyqxmd.com/read/28349046/venous-thromboembolism-in-critical-illness-and-trauma-pediatric-perspectives
#19
REVIEW
Ranjit S Chima, Sheila J Hanson
Critically ill children and those sustaining severe traumatic injuries are at higher risk for developing venous thromboembolism (VTE) than other hospitalized children. Multiple factors including the need for central venous catheters, immobility, surgical procedures, malignancy, and dysregulated inflammatory state confer this increased risk. As well as being at higher risk of VTE, this population is frequently at an increased risk of bleeding, making the decision of prophylactic anticoagulation even more nuanced...
2017: Frontiers in Pediatrics
https://www.readbyqxmd.com/read/28336302/-vasopressors-and-inotropes-use-in-pediatrics
#20
Adrián García-Canales, Rocío Alejandra Peña-Juárez, Luz de María Sandoval-Franco
The cardiovascular system is a dynamic system whose function is to ensure adequate delivery of oxygen, nutrients and hormones to the tissues, necessary for cell metabolism, also synthesizes and modifies vasoactive components to regulate vascular tone and myocardial function. These components have become critical in the management of pediatric patients in critical condition with heart failure and shock which has proven its beneficial effects, however its use and abuse brings harmful effects, increase mortality, associated with arrhythmias and increase myocardial oxygen consumption favoring the presence of ischemia, therefore is necessary to know the mechanism of action and indications of these drugs to minimize the harmful effects...
March 20, 2017: Archivos de Cardiología de México
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