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Pediatric Drug Development

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https://www.readbyqxmd.com/read/28640707/response-assessment-in-neuro-oncology-clinical-trials
#1
Patrick Y Wen, Susan M Chang, Martin J Van den Bent, Michael A Vogelbaum, David R Macdonald, Eudocia Q Lee
Development of novel therapies for CNS tumors requires reliable assessment of response and progression. This requirement has been particularly challenging in neuro-oncology for which contrast enhancement serves as an imperfect surrogate for tumor volume and is influenced by agents that affect vascular permeability, such as antiangiogenic therapies. In addition, most tumors have a nonenhancing component that can be difficult to accurately quantify. To improve the response assessment in neuro-oncology and to standardize the criteria that are used for different CNS tumors, the Response Assessment in Neuro-Oncology (RANO) working group was established...
June 22, 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
#2
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/28627392/the-great-barrier-belief-the-blood-brain-barrier-and-considerations-for-juvenile-toxicity-studies
#3
REVIEW
Georg Schmitt, Neil Parrott, Eric Prinssen, Paul Barrow
Juvenile animal studies can be warranted to support the development of pediatric medicines. Drugs acting on the CNS or those which penetrate into the brain merit particular attention. The blood-brain barrier is functionally mature at birth, but undergoes functional postnatal modulation to provide a suitable microenvironment for the developing brain. In the past, dosing in rat juvenile studies has often commenced at 4 or 7days of age. However, rodents are very neurologically immature at birth compared with humans...
June 13, 2017: Reproductive Toxicology
https://www.readbyqxmd.com/read/28612637/treatment-options-for-children-and-adolescents-with-inflammatory-bowel-disease-is-granulomonocytapheresis-an-effective-alternative-to-drug-therapy
#4
Tomotaka Tanaka, Takayuki Yamamoto, Koji Sawada, Rodolfo Sacco
Patients with inflammatory bowel diseases (IBD) require life-long medications, which even if effective have the potential to cause adverse effects as additional morbidity factors. In pediatric patients, drug therapy has more serious limitations, including impaired physical and mental development. A non-drug therapeutic option is believed to be depletion of elevated and activated granulocytes and monocytes known to release inflammatory cytokines, like the CD14+CD16+ monocyte phenotype known to release tumor necrosis factor-α...
June 14, 2017: Expert Review of Gastroenterology & Hepatology
https://www.readbyqxmd.com/read/28612538/-risk-factors-and-prognosis-of-secondary-epilepsy-in-children-with-viral-encephalitis
#5
Liang Huang, Dan Yu, Rong Luo, Min Li, Hui Zhou, Xiao-Tang Cai, Zhi-Ling Wang
OBJECTIVES: To investigate the clinical features of viral encephalitis in children at acute stage, and the relationship of these clinical features with post viral encephalitic epilepsy. METHODS: The children with viral encephalitis treated in West China Second University Hospital between January 2010 and January 2014 were enrolled. The clinical features included general conditions, clinical manifestations, video electroencephalogram (VEEG), neuroimaging, virology, prognosis and antiepileptic drugs...
March 2017: Sichuan da Xue Xue Bao. Yi Xue Ban, Journal of Sichuan University. Medical Science Edition
https://www.readbyqxmd.com/read/28612093/clinical-trial-design-in-juvenile-idiopathic-arthritis
#6
Stephen J Balevic, Mara L Becker, Michael Cohen-Wolkowiez, Laura E Schanberg
Randomized clinical trials provide the gold standard evidence base to guide clinical practice. Despite major advances in trial design, pediatric clinical trials are still difficult to perform and pose unique challenges, including the need to consider the impact of developmental changes in trial design. Advances within pediatric rheumatology combined with the need to comply with legislative requirements have driven new approaches to performing pediatric clinical trials such as utilization of large research networks, incorporation of patient and family stakeholders in the planning and implementation of clinical trials, and the development of novel trial designs...
June 13, 2017: Paediatric Drugs
https://www.readbyqxmd.com/read/28610634/medication-related-visits-in-a-pediatric-emergency-department-an-8-years-retrospective-analysis
#7
Cristiano Rosafio, Serena Paioli, Cinzia Del Giovane, Valentina Cenciarelli, Nilla Viani, Paolo Bertolani, Lorenzo Iughetti
BACKGROUND: There are limited data on the characterization of medication-related visits (MRVs) to the emergency department (ED) in pediatric patients in Italy. We have estimated the frequency, severity, and classification of MRVs to the ED in pediatric patients. METHODS: We retrospectively analyzed data for children seeking medical evaluation for a MRV over an 8 years period. A medication-related ED visit was identified by using a random pharmacist assessment, emergency physician assessment, and in case of conflicting events, by a third investigators random assessment...
June 13, 2017: Italian Journal of Pediatrics
https://www.readbyqxmd.com/read/28610606/biologic-therapies-for-refractory-juvenile-dermatomyositis-five-years-of-experience-of-the-childhood-arthritis-and-rheumatology-research-alliance-in-north-america
#8
C H Spencer, K Rouster-Stevens, H Gewanter, G Syverson, R Modica, K Schmidt, H Emery, C Wallace, S Grevich, K Nanda, Y D Zhao, S Shenoi, S Tarvin, S Hong, C Lindsley, J E Weiss, M Passo, K Ede, A Brown, K Ardalan, W Bernal, M L Stoll, B Lang, R Carrasco, C Agaiar, L Feller, H Bukulmez, R Vehe, H Kim, H Schmeling, D Gerstbacher, M Hoeltzel, B Eberhard, R Sundel, S Kim, A M Huber, A Patwardhan
BACKGROUND: The prognosis of children with juvenile dermatomyositis (JDM) has improved remarkably since the 1960's with the use of corticosteroid and immunosuppressive therapy. Yet there remain a minority of children who have refractory disease. Since 2003 the sporadic use of biologics (genetically-engineered proteins that usually are derived from human genes) for inflammatory myositis has been reported. In 2011-2016 we investigated our collective experience of biologics in JDM through the Childhood Arthritis and Rheumatology Research Alliance (CARRA)...
June 13, 2017: Pediatric Rheumatology Online Journal
https://www.readbyqxmd.com/read/28602129/from-the-children-s-oncology-group-evidence-based-recommendations-for-peg-asparaginase-nurse-monitoring-hypersensitivity-reaction-management-and-patient-family-education
#9
Deborah Woods, Kari Winchester, Alison Towerman, Katie Gettinger, Christina Carey, Karen Timmermann, Rachel Langley, Emily Browne
PEG-aspariginase is a backbone chemotherapy agent in pediatric acute lymphoblastic leukemia and in some non-Hodgkin lymphoma therapies. Nurses lack standardized guidelines for monitoring patients receiving PEG-asparaginase and for educating patients/families about hypersensitivity reaction risks. An electronic search of 6 databases using publication years 2000-2015 and multiple professional organizations and clinical resources was conducted. Evidence sources were reviewed for topic applicability. Each of the final 23 sources was appraised by 2 team members...
June 1, 2017: Journal of Pediatric Oncology Nursing: Official Journal of the Association of Pediatric Oncology Nurses
https://www.readbyqxmd.com/read/28597827/inflammatory-bowel-disease-in-childhood-and-adolescence
#10
Jan Däbritz, Patrick Gerner, Axel Enninger, Martin Claßen, Michael Radke
BACKGROUND: The incidence of inflammatory bowel disease (IBD) in childhood and adolescence is 5-11 cases per 100 000 persons per year, corresponding to a new diagnosis of IBD in 800-1470 patients in Germany each year. METHODS: This review is based on pertinent publications retrieved by a selective search in PubMed, including guidelines from Germany and abroad. RESULTS: Children and adolescents with IBD often have extensive involvement and an aggressive course of disease...
May 12, 2017: Deutsches Ärzteblatt International
https://www.readbyqxmd.com/read/28597754/characteristics-of-pediatric-pulmonary-hypertension-trials-registered-on-clinicaltrials-gov
#11
Jordan D Awerbach, Richard A Krasuski, Kevin D Hill
The investigation of pediatric pulmonary hypertension (PH) drugs has been identified as a high priority by the United States National Institutes of Health (NIH). Studying pediatric PH is challenging due to the rare and heterogeneous nature of the disease. We sought to define the pediatric PH clinical trials landscape, to evaluate areas of trial success or failure, and to identify potential obstacles to the study of pediatric PH drugs. Interventional pediatric (ages 0-17 years) PH trials registered on ClinicalTrials...
April 2017: Pulmonary Circulation
https://www.readbyqxmd.com/read/28597539/developmental-effects-of-antipsychotic-drugs-on-serotonin-receptor-subtypes
#12
Yong Kee Choi, Matthew P Gardner, Frank I Tarazi
Antipsychotic medications are increasingly prescribed to pediatric and adolescent patients with psychotic diseases in spite of limited knowledge on the long-term effects of dissimilar antipsychotic drugs on developing brain. In this study, we quantified the levels of two major serotonin 5-HT1A , and 5-HT2A receptors in brain regions of developing rats after 3 weeks of treatment with typical (fluphenazine) and atypical (clozapine and olanzapine) antipsychotics, and compared to similarly treated adult rats treated with olanzapine, risperidone and quetiapine examined in previous studies...
June 8, 2017: Synapse
https://www.readbyqxmd.com/read/28580210/immunosuppressive-medication-is-not-associated-with-surgical-site-infection-after-surgery-for-intractable-ulcerative-colitis-in-children
#13
Keiichi Uchida, Yoshikazu Ohtsuka, Atsushi Yoden, Hitoshi Tajiri, Hideaki Kimura, Takashi Isihige, Hiroyuki Yamada, Katsuhiro Arai, Takeshi Tomomasa, Kosuke Ushijima, Tomoki Aomatsu, Satoru Nagata, Kohei Otake, Kohei Matsushita, Mikihiro Inoue, Takahiro Kudo, Kenji Hosoi, Kazuo Takeuchi, Toshiaki Shimizu
Pediatric ulcerative colitis (UC) sometimes progresses to an intractable condition for medical therapy. The surgical management of UC is challenging because of difficult procedures and frequent infectious complications. The aim of this study was to survey surgical procedures and infectious complications in pediatric patients with UC in Japan and to assess the relationship between preoperatively administered immunosuppressive drugs and postoperative surgical site infection (SSI). A survey of pediatric patients treated from 2000 to 2012 was sent to 683 facilities nationwide...
May 2017: Intractable & Rare Diseases Research
https://www.readbyqxmd.com/read/28577701/challenges-of-developing-an-observable-parent-reported-measure-a-qualitative-study-of-functional-impact-of-adhd-in-children
#14
Louis S Matza, Mary Kay Margolis, Linda S Deal, Kimberly F Farrand, M Haim Erder
BACKGROUND: Informant-reported outcome measures, usually completed by parents, are often administered in pediatric clinical trials with the intention of collecting data to support claims in a medical product label. Recently, there has been an emphasis on limiting these measures to observable content, as recommended in the US Food and Drug Administration guidance on patient-reported outcomes. This qualitative study explores the concept of observability using the example of childhood attention deficit/hyperactivity disorder (ADHD)...
June 2017: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/28571505/buprenorphine-implants-in-medical-treatment-of-opioid-addiction
#15
Steven Chavoustie, Michael Frost, Ole Snyder, Joel Owen, Mona Darwish, Ryan Dammerman, Victoria Sanjurjo
Opioid use disorder is a chronic, relapsing disease that encompasses use of both prescription opioids and heroin and is associated with a high annual rate of overdose deaths. Medical treatment has proven more successful than placebo treatment or psychosocial intervention, and the partial µ-opioid receptor agonist and κ-opioid receptor antagonist buprenorphine is similar in efficacy to methadone while offering lower risk of respiratory depression. However, frequent dosing requirements and potential for misuse and drug diversion contribute to significant complications with treatment adherence for available formulations...
June 21, 2017: Expert Review of Clinical Pharmacology
https://www.readbyqxmd.com/read/28570545/transplantation-of-zebrafish-pediatric-brain-tumors-into-immune-competent-hosts-for-long-term-study-of-tumor-cell-behavior-and-drug-response
#16
Mattie J Casey, Katarzyna Modzelewska, Daniela Anderson, James Goodman, Elena F Boer, Laura Jimenez, Douglas Grossman, Rodney A Stewart
Tumor cell transplantation is an important technique to define the mechanisms controlling cancer cell growth, migration, and host response, as well as to assess potential patient response to therapy. Current methods largely depend on using syngeneic or immune-compromised animals to avoid rejection of the tumor graft. Such methods require the use of specific genetic strains that often prevent the analysis of immune-tumor cell interactions and/or are limited to specific genetic backgrounds. An alternative method in zebrafish takes advantage of an incompletely developed immune system in the embryonic brain before 3 days, where tumor cells are transplanted for use in short-term assays (i...
May 17, 2017: Journal of Visualized Experiments: JoVE
https://www.readbyqxmd.com/read/28561211/-increasing-consumption-of-crystal-meth-in-saxony-and-its-risks-for-mother-and-child-experiences-at-a-level-i-perinatal-center-from-a-pediatric-viewpoint
#17
Jürgen Dinger, Nicole Näther, Pauline Wimberger, Ulrich S Zimmermann, Jochen Schmitt, Jörg Reichert, Mario Rüdiger
Over the past several years, the number of first-time users of methamphetamine considerably increased within the Free State of Saxony. This number also, and especially, includes women of child-bearing age and young mothers. A supportive intervention aimed at reducing the effects of drug abuse should begin in a timely way - during pregnancy at latest, but better before it. To develop an optimal care protocol for both the mother and her child, more knowledge regarding the personal conditions of the women as well as the specific features of later child development after prenatal methamphetamine exposure (PME) is vital...
April 2017: Zeitschrift Für Geburtshilfe und Neonatologie
https://www.readbyqxmd.com/read/28560211/advanced-therapy-medicinal-products-for-rare-diseases-state-of-play-of-incentives-supporting-development-in-europe
#18
Andreas M Farkas, Segundo Mariz, Violeta Stoyanova-Beninska, Patrick Celis, Spiros Vamvakas, Kristina Larsson, Bruno Sepodes
In 2008, the European Union introduced the Advanced Medicines Regulation aiming to improve regulation of advanced therapy medicinal products (ATMPs). We applied the ATMPs classification definitions in this Regulation to understand the link of this emerging group of medicinal products and the use of the Orphan Regulation. A total of 185 products that can be classified as ATMPs based on this Regulation have been submitted for orphan designation. Prior to its introduction in 2008, 4.5% of the products submitted for orphan designation met these criteria...
2017: Frontiers in Medicine
https://www.readbyqxmd.com/read/28559702/tubercular-abdominal-cocoon-in-children-a-single-centre-study-in-remote-area-of-northern-india
#19
Rikki Singal, B Satyashree, Amit Mittal, Bhanu Pratap Sharma, Samita Singal, Muzzafarr Zaman, Parteek Shardha
BACKGROUND: Amongst the numerous causes of intestinal obstruction listed in the literature, sclerosing encapsulating peritonitis also called Abdominal Cocoon (AC) is one of the rarest entities. Its characteristic feature is a thick fibrotic membrane encasing varying lengths of the small and large gut in a cocoon. In India, there is an increasing incidence of tuberculosis, especially in the rural areas. AIMS AND OBJECTIVES: The aim of this study was to investigate the clinical presentation and evaluate the operative findings of tuberculous AC...
2017: Clujul Medical (1957)
https://www.readbyqxmd.com/read/28559222/mobile-app-design-development-and-publication-for-adverse-drug-reaction-assessments-of-causality-severity-and-preventability
#20
Muslimah Ithnin, Mohd Dzulkhairi Mohd Rani, Zuraidah Abd Latif, Paveethra Kani, Asmalita Syaiful, Khairun Nain Nor Aripin, Tengku Amatullah Madeehah Tengku Mohd
BACKGROUND: Adverse drug reactions (ADRs) cause significant morbidity and mortality. Improved assessment of ADRs to identify the causal relationship, the severity, and the preventability will aid ADRs prevention or reduce patient burden. OBJECTIVE: The aim of this study was to develop mobile apps in assisting clinical decision in ADR assessments of causality, severity, and preventability using validated tools. The usability of the apps was assessed. METHODS: We designed mobile apps using validated assessment tools for ADRs...
May 30, 2017: JMIR MHealth and UHealth
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