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pediatric clinical pharmacology

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https://www.readbyqxmd.com/read/28925064/improving-realism-in-clinical-trial-simulations-via-real-world-data
#1
EDITORIAL
Holly Kimko, Kwan Lee
Simulation validity depends on how well sampling distributions used reflect real-patient characteristics, such as drug adherence, disease progression, and pharmacologic handling in the body. We challenge the current use of growth charts from nondisease-specific pediatrics in simulations for drug development. Complementary use of data from clinical trials and the real-world is expected to achieve a more realistic representation of clinical outcomes for decisions in drug development, regulatory approval, and health technology assessment...
September 19, 2017: CPT: Pharmacometrics & Systems Pharmacology
https://www.readbyqxmd.com/read/28883017/nf%C3%AE%C2%BAb-signaling-in-alveolar-rhabdomyosarcoma
#2
Megan M Cleary, Atiya Mansoor, Teagan Settelmeyer, Yuichi Ijiri, Katherine J Ladner, Matthew N Svalina, Brian P Rubin, Denis C Guttridge, Charles Keller
Alveolar rhabdomyosarcoma (aRMS) is a pediatric soft tissue cancer commonly associated with a chromosomal translocation that leads to the expression of a Pax3:Foxo1 or Pax7:Foxo1 fusion protein, the developmental underpinnings of which may give clues to its therapeutic approaches. In aRMS, the NFκB-YY1-miR-29 regulatory circuit is dysregulated, resulting in repression of miR-29 and loss of the associated tumor suppressor activity. To further elucidate the role of NFκB in aRMS, we first tested 55 unique sarcoma cell lines and primary cell cultures in a large-scale chemical screen targeting diverse molecular pathways...
September 1, 2017: Disease Models & Mechanisms
https://www.readbyqxmd.com/read/28874164/radiation-free-cmr-diagnostic-heart-catheterization-in-children
#3
Kanishka Ratnayaka, Joshua P Kanter, Anthony Z Faranesh, Elena K Grant, Laura J Olivieri, Russell R Cross, Ileen F Cronin, Karin S Hamann, Adrienne E Campbell-Washburn, Kendall J O'Brien, Toby Rogers, Michael S Hansen, Robert J Lederman
BACKGROUND: Children with heart disease may require repeated X-Ray cardiac catheterization procedures, are more radiosensitive, and more likely to survive to experience oncologic risks of medical radiation. Cardiovascular magnetic resonance (CMR) is radiation-free and offers information about structure, function, and perfusion but not hemodynamics. We intend to perform complete radiation-free diagnostic right heart catheterization entirely using CMR fluoroscopy guidance in an unselected cohort of pediatric patients; we report the feasibility and safety...
September 6, 2017: Journal of Cardiovascular Magnetic Resonance
https://www.readbyqxmd.com/read/28866891/integration-of-hypnosis-into-pediatric-palliative-care
#4
Stefan J Friedrichsdorf, Daniel P Kohen
At least 8 million children would need specialized pediatric palliative care (PPC) services annually worldwide, and of the more than 42,000 children and teenagers dying annually in the United States, at least 15,000 children would require PPC. Unfortunately, even in resource-rich countries the majority of children dying from serious advanced illnesses are suffering from unrelieved, distressing symptoms such as pain, dyspnea, nausea, vomiting, and anxiety. State of the art treatment and prevention of those symptoms requires employing multi-modal therapies, commonly including pharmacology, rehabilitation, procedural intervention, psychology, and integrative modalities...
June 27, 2017: Annals of Palliative Medicine
https://www.readbyqxmd.com/read/28851284/anesthetic-management-of-a-pediatric-patient-with-electron-transfer-flavoprotein-dehydrogenase-deficiency-etfdh-and-acute-appendicitis-case-report-and-review-of-the-literature
#5
Emmanuel Lilitsis, Elisavet Astyrakaki, Evaggelos Blevrakis, Sofia Xenaki, George Chalkiadakis, Emmanuel Chrysos
BACKGROUND: Mitochondria are the energy producing organelles practically in every human cell except erythrocytes. Indeed mitochondria are widespread in high energy requiring organs like brain, heart and muscles. Currently there are no clinical trials supporting with clear evidence which is the most suitable surgical or anesthetic management of a patient with known mitochondrial disease presenting with surgical disorders. This condition poses possible hazardous problems to the medical attention of those patients...
August 29, 2017: BMC Anesthesiology
https://www.readbyqxmd.com/read/28840947/the-conceptualization-of-childhood-in-north-american-pediatric-dentistry-texts-a-discursive-case-study-analysis
#6
Nora Makansi, Franco A Carnevale, Mary Ellen Macdonald
BACKGROUND: In recent year, conceptions of childhood have been evolving towards an increased recognition of children as active agents, capable of participating in the determination of their wellbeing. In pediatric dentistry, the extent to which these conceptions are being discursively endorsed is not well known. AIM: The aim of this investigation was to examine the discursive construction of childhood in seminal North American pedagogical dentistry materials. DESIGN: We conducted a qualitative discourse analysis of a sample of prominent texts using a sociological discourse analysis approach...
August 25, 2017: International Journal of Paediatric Dentistry
https://www.readbyqxmd.com/read/28837801/pharmacologic-and-nonpharmacologic-treatment-for-acute-cough-associated-with-the-common-cold-chest-expert-panel-report
#7
Mark A Malesker, Priscilla Callahan-Lyon, Belinda Ireland, Richard S Irwin
BACKGROUND: Acute cough associated with the common cold (CACC) causes significant impairment in quality of life. Effective treatment approaches are needed for CACC. We conducted a systematic review on the management of CACC to update the recommendations and suggestions of the CHEST 2006 guideline on this topic. METHODS: This systematic review of randomized controlled trials (RCTs) asked the question: Is there evidence of clinically relevant treatment effects for pharmacologic or nonpharmacologic therapies in reducing the duration/severity of acute CACC? Studies of adults and pediatric patients with CACC were included and assessed for relevance and quality...
August 22, 2017: Chest
https://www.readbyqxmd.com/read/28830790/increased-delivery-of-chemotherapy-to-the-vitreous-by-inhibition-of-the-blood-retinal-barrier
#8
Guillem Pascual-Pasto, Nagore G Olaciregui, Javier A W Opezzo, Helena Castillo-Ecija, Maria Cuadrado-Vilanova, Sonia Paco, Ezequiel M Rivero, Monica Vila-Ubach, Camilo A Restrepo-Perdomo, Montserrat Torrebadell, Mariona Suñol, Paula Schaiquevich, Jaume Mora, Guillermo F Bramuglia, Guillermo L Chantada, Angel M Carcaboso
Treatment of retinoblastoma -a pediatric cancer of the developing retina- might benefit from strategies to inhibit the blood-retinal barrier (BRB). The potent anticancer agent topotecan is a substrate of efflux transporters BCRP and P-gp, which are expressed at the BRB to restrict vitreous and retinal distribution of xenobiotics. In this work we have studied vitreous and retinal distribution, tumor accumulation and antitumor activity of topotecan, using pantoprazole as inhibitor of BCRP and P-gp. We used rabbit and mouse eyes as BRB models and patient-derived xenografts as retinoblastoma models...
August 19, 2017: Journal of Controlled Release: Official Journal of the Controlled Release Society
https://www.readbyqxmd.com/read/28817401/challenges-in-the-anesthetic-management-of-ambulatory-patients-in-the-mri-suites
#9
Jasper Deen, Yzabel Vandevivere, Peter Van de Putte
PURPOSE OF REVIEW: MRI is becoming an indispensable diagnostic tool. The need for prolonged motion-free periods has substantially increased the need for deep sedation or anesthesia in a challenging environment. This review summarises recent literature with respect to pharmacological sedative strategies, nonpharmacological alternative approaches, airway management and safety issues in the ambulatory setting. RECENT FINDINGS: Most literature researches the pediatric patient population...
August 16, 2017: Current Opinion in Anaesthesiology
https://www.readbyqxmd.com/read/28777308/key-challenges-in-the-search-for-innovative-drug-treatments-for-special-populations-converging-needs-in-neonatology-pediatrics-and-medical-genetics
#10
Stuart MacLeod
The explosion of knowledge concerning the interplay of genetic and environmental factors determining pathophysiology and guiding therapeutic choice has altered the landscape in pediatric clinical pharmacology and pharmacy. The need for innovative research methods and design expertise for small clinical trials to be undertaken in sparse populations has been accentuated. At the same time, shortfalls in critical human resources represent a key challenge, especially in low- and middle-income countries where the need for new research and education directions is greatest...
August 4, 2017: Children
https://www.readbyqxmd.com/read/28737198/-tetany-secondary-to-phosphate-enema-toxicity-case-report
#11
María José Núñez Sánchez, Sofía Leighton Swaneck, Franco Díaz
Phosphate enemas are frequently used in the treatment of constipation. Errors in dosage and administration can lead to severe complications. OBJECTIVE: To report a case of severe toxicity of phosphate enemas in a child with no risk factors. CASE: 2 years old female, with functional constipation, was brought to emergency department because abdominal pain. She was diagnosed with fecal impaction and received half a bottle of Fleet Adult® (Laboratorio Synthon, Chile) two times, with no clinical resolution, deciding to start proctoclisis in pediatric ward...
June 2017: Revista Chilena de Pediatría
https://www.readbyqxmd.com/read/28735109/design-and-rationale-for-a-real-world-observational-cohort-of-patients-with-nonalcoholic-fatty-liver-disease-the-target-nash-study
#12
A S Barritt, Norman Gitlin, Samuel Klein, Anna S Lok, Rohit Loomba, Laura Malahias, Margaret Powell, Miriam B Vos, L Michael Weiss, Kenneth Cusi, Brent A Neuschwander-Tetri, Arun Sanyal
Nonalcoholic fatty liver disease (NAFLD) is highly prevalent and can lead to cirrhosis, hepatocellular carcinoma, and end-stage liver disease. NAFLD comprises the spectrum from simple steatosis (nonalcoholic fatty liver, NAFL), to steatosis with inflammation (nonalcoholic steatohepatitis, NASH). Current primary therapy recommended for NAFLD is weight loss induced by lifestyle modification. The difficulty in achieving this has led to robust pharmacological therapy development. While new drugs may show efficacy in selected phase II/III clinical trial populations, their real-world effectiveness is unknown...
October 2017: Contemporary Clinical Trials
https://www.readbyqxmd.com/read/28696783/course-of-neuropsychiatric-symptoms-after-introduction-and-removal-of-nonsteroidal-anti-inflammatory-drugs-a-pediatric-observational-study
#13
Ellen J Spartz, G Mark Freeman, Kayla Brown, Bahare Farhadian, Margo Thienemann, Jennifer Frankovich
OBJECTIVE: Accumulating evidence suggests that anti-inflammatory interventions can modulate neuropsychiatric symptoms. Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) is characterized by an abrupt and dramatic onset of obsessive-compulsive (OC) symptoms and/or severely restrictive food intake and at least two coinciding, equally debilitating neuropsychiatric symptoms. When associated with group A Streptococcus, the syndrome is labeled Pediatric Autoimmune Neuropsychiatric Disorder Associated with Streptococcal infections (PANDAS)...
September 2017: Journal of Child and Adolescent Psychopharmacology
https://www.readbyqxmd.com/read/28678061/pain-in-children-with-cancer-prevalence-characteristics-and-parent-management
#14
Perri R Tutelman, Christine T Chambers, Jennifer N Stinson, Jennifer A Parker, Conrad V Fernandez, Holly O Witteman, Paul C Nathan, Melanie Barwick, Fiona Campbell, Lindsay A Jibb, Karen Irwin
OBJECTIVES: Pain is a common and distressing symptom of pediatric cancer, as reported by both children and their parents. Increasingly, children with cancer are cared for as outpatients, yet little is known about how parents manage their cancer-related pain. The aim of the current study was to examine pain prevalence and characteristics, and the pharmacological, physical and psychological pain management strategies used by parents to manage their child's cancer pain. METHODS: In total, 230 parents and caregivers (89% mothers) of children (mean age=8...
July 3, 2017: Clinical Journal of Pain
https://www.readbyqxmd.com/read/28677996/headache-in-pediatric-and-adolescent-patients-with-chronic-kidney-disease-with-and-without-hemodialysis-a-comparative-cohort-study
#15
Miriam Davidovits, Tal Eidlitz Markus
Background Several studies have reported dialysis-related headache in adults. We investigated headache and its characteristics in pediatric and adolescent patients with chronic kidney disease and patients treated with dialysis, and compared characteristics of patients with and without headache in the entire cohort and separately among dialysis and among chronic kidney disease patients. Methods Patients and their parents who attended a nephrology clinic and hemodialysis unit were interviewed regarding the existence of headache and its characteristics...
January 1, 2017: Cephalalgia: An International Journal of Headache
https://www.readbyqxmd.com/read/28673313/mdm2-x-inhibitors-under-clinical-evaluation-perspectives-for-the-management-of-hematological-malignancies-and-pediatric-cancer
#16
REVIEW
Veronica Tisato, Rebecca Voltan, Arianna Gonelli, Paola Secchiero, Giorgio Zauli
The two murine double minute (MDM) family members MDM2 and MDMX are at the center of an intense clinical assessment as molecular target for the management of cancer. Indeed, the two proteins act as regulators of P53, a well-known key controller of the cell cycle regulation and cell proliferation that, when altered, plays a direct role on cancer development and progression. Several evidence demonstrated that functional aberrations of P53 in tumors are in most cases the consequence of alterations on the MDM2 and MDMX regulatory proteins, in particular in patients with hematological malignancies where TP53 shows a relatively low frequency of mutation while MDM2 and MDMX are frequently found amplified/overexpressed...
July 3, 2017: Journal of Hematology & Oncology
https://www.readbyqxmd.com/read/28669553/expert-consensus-guidelines-for-stocking-of-antidotes-in-hospitals-that-provide-emergency-care
#17
Richard C Dart, Lewis R Goldfrank, Brian L Erstad, David T Huang, Knox H Todd, Jeffrey Weitz, Vikhyat S Bebarta, E Martin Caravati, Fred M Henretig, Theodore R Delbridge, William Banner, Sandra M Schneider, Victoria E Anderson
We provide recommendations for stocking of antidotes used in emergency departments (EDs). An expert panel representing diverse perspectives (clinical pharmacology, medical toxicology, critical care medicine, hematology/oncology, hospital pharmacy, emergency medicine, emergency medical services, pediatric emergency medicine, pediatric critical care medicine, poison centers, hospital administration, and public health) was formed to create recommendations for antidote stocking. Using a standardized summary of the medical literature, the primary reviewer for each antidote proposed guidelines for antidote stocking to the full panel...
June 29, 2017: Annals of Emergency Medicine
https://www.readbyqxmd.com/read/28660776/gender-affirming-pharmacological-interventions-for-youth-with-gender-dysphoria-when-treatment-guidelines-are-not-enough
#18
Leena Nahata, Gayathri Chelvakumar, Scott Leibowitz
Youth with gender dysphoria, also known as transgender youth, are increasingly presenting to multidisciplinary clinics within academic pediatric centers across the United States. Gender-affirming pharmacological interventions for adolescents with gender dysphoria may be used to promote positive psychological well-being and mental health outcomes. Interventions range from completely reversible to partially irreversible, based on the age and sexual maturity of the adolescent. For each intervention, dilemmas and controversies exist regarding age at treatment initiation, treatment duration, safety, and cost...
June 1, 2017: Annals of Pharmacotherapy
https://www.readbyqxmd.com/read/28660740/prolonged-first-line-peg-asparaginase-treatment-in-pediatric-acute-lymphoblastic-leukemia-in-the-nopho-all2008-protocol-pharmacokinetics-and-antibody-formation
#19
Louise Tram Henriksen, Sofie Gottschalk Højfeldt, Kjeld Schmiegelow, Thomas Leth Frandsen, Peder Skov Wehner, Henrik Schrøder, Birgitte Klug Albertsen
BACKGROUND: As pegylated asparaginase is becoming the preferred first-line asparaginase preparation in the chemotherapy regimens of childhood acute lymphoblastic leukemia (ALL), there is a need to evaluate this treatment. METHODS: The aim of this study was to evaluate the pharmacokinetics of prolonged upfront biweekly PEG-asparaginase (where PEG is polyethylene glycol) treatment by measuring serum l-asparaginase activity and formation of anti-PEG-asparaginase antibodies...
June 29, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28650778/success-of-dental-treatments-under-behavior-management-sedation-and-general-anesthesia
#20
COMPARATIVE STUDY
Sigalit Blumer, Liora Costa, Benjamin Peretz
OBJECTIVE: To present comparative study aims to assist the practitioner to choose between behavior modification (BM) techniques, pharmacologic sedation (N2O-O2 alone or combined with midazolam 0.5 mg/ kg) or routine general anesthesia (GA) for the most successful approach in enabling pediatric dental care. STUDY DESIGN: Dental records of 56 children treated in a university dental clinic between 2006-2016 were reviewed, and data on age, gender, required treatment (amalgam restorations, composite restorations, pulpotomy, and stainless steel crowns [SSC]), treatment approaches and therapeutic success at final follow-up were retrieved...
2017: Journal of Clinical Pediatric Dentistry
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