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Pediatric Drug Development

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https://www.readbyqxmd.com/read/29325220/profile-risk-factors-and-outcome-of-acute-kidney-injury-in-pediatric-acute-on-chronic-liver-failure
#1
Bikrant Bihari Lal, Seema Alam, Vikrant Sood, Dinesh Rawat, Rajeev Khanna
There are no studies on acute kidney injury (AKI) in pediatric acute-on-chronic liver failure (ACLF). This study was planned with aim to describe the clinical presentation and outcome of AKI among pediatric ACLF patients METHODS: Data of all children 1-18 years of age presenting with ACLF (APASL definition) was reviewed. AKI was defined as per KDIGO guidelines. Poor outcome was defined as death or need for liver transplant within 3 months of development of AKI RESULTS: A total of 84 children with ACLF presented to us in the study period...
January 11, 2018: Liver International: Official Journal of the International Association for the Study of the Liver
https://www.readbyqxmd.com/read/29324237/dna-induction-of-mdm2-promotes-proliferation-of-human-renal-mesangial-cells-and-alters-peripheral-b-cells-subsets-in-pediatric-systemic-lupus-erythematosus
#2
Chen-Xing Zhang, Ji Chen, Li Cai, Jing Wu, Jia-Yuan Wang, Lan-Fang Cao, Wei Zhou, Tong-Xin Chen
The study is aimed to investigate the role of MDM2 in the pathogenesis of lupus nephritis (LN) in pediatric SLE (pSLE). We confirmed that MDM2 expression was increased in peripheral blood mononuclear cells (PBMCs) as well as renal specimen of SLE compared with that of controls by western blot and immunofluorescence staining. Percentage of apoptotic and necrotic CD4+T, CD8+T and B cells were detected by flow cytometry respectively and levels of plasma cell free DNA (cfDNA) were quantified in SLE and healthy controls (HC)...
January 8, 2018: Molecular Immunology
https://www.readbyqxmd.com/read/29322843/immunotherapy-for-brain-tumors-understanding-early-successes-and-limitations
#3
Nicole A P Lieberman, Nicholas A Vitanza, Courtney A Crane
Adverse effects and toxicities related to standard treatments for brain tumors significantly reduce patients' quality of life. Although most immunotherapy approaches for solid tumors have not been successful, several early-phase clinical trials are beginning to reveal a potential role for immunotherapy in the treatment of brain tumors. In particular, methods that activate the innate immune system and induce a polyclonal anti-cancer response have demonstrated that brain tumors are susceptible to immune-mediated tumor destruction...
January 11, 2018: Expert Review of Neurotherapeutics
https://www.readbyqxmd.com/read/29319897/application-of-a-physiologically-based-pharmacokinetic-model-for-the-prediction-of-bumetanide-plasma-and-brain-concentrations-in-the-neonate
#4
Maria D Donovan, Khaled Abduljalil, John F Cryan, Geraldine B Boylan, Brendan T Griffin
Bumetanide is a loop diuretic that is proposed to possess a beneficial effect on disorders of the central nervous system, including neonatal seizures. Therefore, prediction of unbound bumetanide concentrations in brain is relevant from a pharmacological prospective. A physiologically-based pharmacokinetic (PBPK) model was developed for the prediction of bumetanide disposition in plasma and brain in adult and pediatric populations. A compound file was built for bumetanide integrating physicochemical data and in vitro data...
January 10, 2018: Biopharmaceutics & Drug Disposition
https://www.readbyqxmd.com/read/29319159/leveraging-big-data-in-pediatric-development-programs-proceedings-from-the-2016-american-college-of-clinical-pharmacology-annual-meeting-symposium
#5
REVIEW
Lily Yeruk Mulugeta, Lynne Yao, Diane Mould, Brian Jacobs, Jeffrey Florian, Brian Smith, Vikram Sinha, Jeffrey S Barrett
This article discusses the use of big data in pediatric drug development. The article covers key topics discussed at the ACCP annual meeting symposium in 2016 including the extent to which big data or real-world data can inform clinical trial design and substitute for efficacy and safety data typically obtained in clinical trials. The current states of use, opportunities, and challenges with the use of big data in future pediatric drug development are discussed.
January 10, 2018: Clinical Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/29306991/asthma-and-medicines-long-term-side-effects-monitoring-and-dose-titration
#6
REVIEW
Satnam Kaur, Varinder Singh
Asthma is a major pediatric respiratory morbidity requiring long-term management. A thorough knowledge of long-term medication side-effects in children is, thus, essential for every physician dealing with childhood asthma. Establishing diagnosis and initiating treatment is just a beginning of the journey. Ongoing monitoring is an essential component of comprehensive asthma management programme. Monitoring includes not only assessment of asthma control but also checking for adherence to treatment, technique of inhaler device use, associated co-morbities, if any, and potential environmental exposure...
January 6, 2018: Indian Journal of Pediatrics
https://www.readbyqxmd.com/read/29305553/preclinical-efficacy-of-daratumumab-in-t-cell-acute-lymphoblastic-leukemia-t-all
#7
Karen L Bride, Tiffaney L Vincent, Soo-Yeon Im, Richard Aplenc, David M Barrett, William L Carroll, Robin Carson, Yunfeng Dai, Meenakshi Devidas, Kimberly P Dunsmore, Tori Fuller, Tina Glisovic-Aplenc, Terzah M Horton, Stephen P Hunger, Mignon L Loh, Shannon L Maude, Elizabeth A Raetz, Stuart S Winter, Stephan A Grupp, Michelle L Hermiston, Brent L Wood, David T Teachey
As a consequence of acquired or intrinsic disease resistance, the prognosis for patients with relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) is dismal. Novel, less toxic drugs are clearly needed. One of the most promising emerging therapeutic strategies for cancer treatment is targeted immunotherapy. Immune therapies have improved outcomes for patients with other hematologic malignancies including B-ALL, however no immune therapy has been successfully developed for T-ALL. We hypothesize targeting CD38 will be effective against T-ALL...
January 5, 2018: Blood
https://www.readbyqxmd.com/read/29289454/audiometric-assessment-of-pediatric-patients-with-cystic-fibrosis
#8
Kathryn L Kreicher, Michael J Bauschard, Clarice S Clemmens, Concetta Maria Riva, Ted A Meyer
BACKGROUND: The purpose of this study was to evaluate hearing impairment in pediatric patients with cystic fibrosis (CF). METHODS: This is a retrospective analysis of the AudGen database generated by Children's Hospital of Philadelphia. Audiograms were analyzed for type of hearing loss (HL), pure-tone-average (PTA), laterality, and change in hearing over time. Medical charts were reviewed to identify factors that influence development and progression of hearing loss...
December 27, 2017: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29287014/treatment-of-chronic-hepatitis-c-virus-infection-in-children-a-position-paper-by-the-hepatology-committee-of-european-society-of-paediatric-gastroenterology-hepatology-and-nutrition
#9
Giuseppe Indolfi, Loreto Hierro, Antal Dezsofi, Jörg Jahnel, Dominique Debray, Nedim Hadzic, Piotr Czubowski, Girish Gupte, Yael Mozer-Glassberg, Wendy van der Woerd, Françoise Smets, Henkjan J Verkade, Björn Fischler
OBJECTIVES: In 2017, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) approved the use of the fixed-dose combination of ledipasvir/sofosbuvir and of the combination of sofosbuvir and ribavirin for treatment of adolescents (12-17 years, weighing more than 35 kg) with chronic hepatitis C virus (HCV) genotype 1, 4, 5 and 6 and genotype 2 and 3 infections, respectively. Although trials with direct acting antivirals (DAAs) are ongoing for younger children, the only available treatment in US and Europe for those < 12 years is still the dual therapy of pegylated interferon (PEG IFN) and ribavirin...
December 28, 2017: Journal of Pediatric Gastroenterology and Nutrition
https://www.readbyqxmd.com/read/29284286/10-years-eu-regulation-of-pediatric-medicines-impact-on-cardiovascular-drug-formulations
#10
Yasmin Thabet, Marta Slavkova, Jörg Breitkreutz
Child-appropriate drug formulations are mandatory for an efficient and safe drug therapy in children. Since the implementation of supportive legislations development of novel drug formulations has significantly been enforced despite the fact that children are a heterogeneous group of patients with varying needs according to age, maturation and disease. Areas covered: In this review, recent advances and current strategies are evaluated how to overcome the specific hurdles in pediatric drug development. For cardiovascular diseases as an example, EMA's decisions on pediatric investigation plans (PIPs) have been evaluated...
December 28, 2017: Expert Opinion on Drug Delivery
https://www.readbyqxmd.com/read/29280786/nephrotoxicity-with-vancomycin-in-the-pediatric-population-a-systematic-review-and-meta-analysis
#11
Theresa M Fiorito, Megan K Luther, Penelope H Dennehy, Kerry L LaPlante, Kelly L Matson
BACKGROUND: Vancomycin is frequently used to treat methicillin-resistant Staphylococcus aureus (MRSA) infections in pediatric patients. Vancomycin exposure may lead to an increase in frequency of nephrotoxicity. Our aim was to conduct a systematic review to describe predictors of nephrotoxicity associated with vancomycin, including documented trough concentrations ≥15mg/L. We also aimed to use a meta-analysis to assess the impact of a vancomycin trough ≥15 mg/L on nephrotoxicity...
December 26, 2017: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/29278643/response-biomarkers-in-neonatal-intervention-studies
#12
Lauren E Kelly, Kimberly Caswell, Mary Short, Prabhu S Parimi, Martin Offringa, Thomas Diacovo
BACKGROUND: Up to 90% of all drugs used in neonatal intensive care units (NICUs) have not been clinically tested for safety and efficacy. To further drug development for neonates the pharmaceutical industry is moving towards rigorous testing, necessitating the need to development and validate biomarkers in neonates to predict response. The objective of this review is to evaluate the quality of response biomarker reporting in neonatal clinical trials. METHODS: A validated literature search strategy was applied...
August 21, 2017: Pediatric Research
https://www.readbyqxmd.com/read/29276869/incidence-risk-factors-and-treatment-outcomes-of-drug-extravasation-in-pediatric-patients-in-china
#13
Ya-Min Yan, Mei Gong, Jia-Ling Chen, Dan Li, Ting-Ting Xu, Huan Zou, Ai-Qiu Li, Qiao-Ling Fan, Qun-Feng Lu
Yan YM, Gong M, Chen JL, Li D, Xu TT, Zou H, Li AQ, Fan QL, Lu QF. Incidence, risk factors and treatment outcomes of drug extravasation in pediatric patients in China. Turk J Pediatr 2017; 59: 162-168. Extravasation injury is a common phenomenon in hospitals. Failure to detect and treat extravasation injury can lead to irreversible local injuries, tissue necrosis and malfunction of the affected tissue. Until now, it is largely unknown about incidence, risk factors and treatment outcomes of extravasation in Chinese pediatric patients...
2017: Turkish Journal of Pediatrics
https://www.readbyqxmd.com/read/29274889/new-onset-autoimmune-hepatitis-during-anti-tumor-necrosis-factor-alpha-treatment-in-children
#14
Amanda Ricciuto, Binita M Kamath, Thomas D Walters, Karen Frost, Nicholas Carman, Peter C Church, Simon C Ling, Anne M Griffiths
OBJECTIVES: To evaluate a large anti-tumor necrosis factor (TNF)-treated pediatric inflammatory bowel disease cohort for drug-induced liver injury (DILI) following presentation of an index case with suspected DILI with autoimmune features after infliximab exposure. To characterize the incidence, natural history, and risk factors for liver enzyme elevation with anti-TNF use. STUDY DESIGN: We reviewed the index case and performed a retrospective cohort study of 659 children receiving anti-TNF therapy between 2000 and 2015 at a tertiary pediatric inflammatory bowel disease center...
December 21, 2017: Journal of Pediatrics
https://www.readbyqxmd.com/read/29274818/population-pharmacokinetics-and-dosing-simulations-of-ceftazidime-in-chinese-neonates
#15
Honghong Wang, Xingang Li, Shusen Sun, Guifu Mao, Ping Xiao, Chan Fu, Zhuoxin Liang, Min Zheng, Yuling Huang, Haihong Tang, Renhao Ou, Ni Yang, Xi Ling, Zhigang Zhao
An accurate dosage determination is required in neonates when antibiotics are used. The adult data cannot be simply extrapolated to the pediatric population due to significant individual differences. We aimed to identify factors impacting ceftazidime exposure in neonates and to provide drug dosing guidance to clinicians. 43 neonates aged less than 60 days with proven or suspected infections were enrolled in this study. After intravenous administration, blood samples were collected, and plasma ceftazidime concentration was determined using a high-performance liquid chromatography method...
December 21, 2017: Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/29260752/-safety-of-the-russian-antiviral-drug-kagocel
#16
T G Borovskaya
The review gives summarized information on the preclinical data and clinical trials evaluating the safety of the antiviral drug Kagocel. It notes that the manufacturer of the drug pay special attention to the control of its impurity content. There is information on the development and validation of highly sensitive and specific high-performance liquid chromatography procedures, the application of which can guarantee that free gossypol impurities are absent in the drug. The results of preclinical toxicity study of Kagocel in experiments on laboratory animals are briefly reviewed; particular attention is paid to the investigation of the drug's safety for the reproductive system of immature animals...
2017: Terapevticheskiĭ Arkhiv
https://www.readbyqxmd.com/read/29258474/full-title-peripheral-venous-catheter-complications-in-children-predisposing-factors-in-a-multicenter-prospective-cohort-study
#17
Rim Ben Abdelaziz, Habiba Hafsi, Hela Hajji, Hela Boudabous, Amel Ben Chehida, Ali Mrabet, Khadija Boussetta, Sihem Barsaoui, Azza Sammoud, Mourad Hamzaoui, Hatem Azzouz, Néji Tebib
BACKGROUND: Peripheral venous catheterization (PVC) is frequently used in children. This procedure is not free from potential complications. Our purpose was to identify the types and incidences of PVC complications in children and their predisposing factors in a developing country. METHODS: We conducted a prospective observational multicenter study in five pediatric and pediatric surgery departments over a period of 2 months. Two hundred fifteen PVC procedures were conducted in 98 children...
December 19, 2017: BMC Pediatrics
https://www.readbyqxmd.com/read/29247506/suboptimal-anti-tuberculosis-drug-concentrations-and-outcomes-in-small-and-hiv-coinfected-children-in-india-recommendations-for-dose-modifications
#18
Benjamin Guiastrennec, Geetha Ramachandran, Mats O Karlsson, A K Hemanth Kumar, Perumal Kannabiran Bhavani, N Poorana Gangadevi, Soumya Swaminathan, Amita Gupta, Kelly E Dooley, Radojka M Savic
This work aimed to evaluate the once-daily anti-tuberculosis treatment as recommended by the new Indian pediatric guidelines. Isoniazid, rifampin and pyrazinamide concentration-time profiles and treatment outcome were obtained from 161 Indian children with drug-sensitive tuberculosis undergoing thrice-weekly dosing as per previous Indian pediatric guidelines. The exposure-response relationships were established using a population pharmacokinetic-pharmacodynamic approach. Rifampin exposure was identified as the unique predictor of treatment outcome...
December 16, 2017: Clinical Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/29247424/method-development-for-clinical-comprehensive-evaluation-of-pediatric-drugs-based-on-multi-criteria-decision-analysis-application-to-inhaled-corticosteroids-for-children-with-asthma
#19
Yuncui Yu, Lulu Jia, Yao Meng, Lihua Hu, Yiwei Liu, Xiaolu Nie, Meng Zhang, Xuan Zhang, Sheng Han, Xiaoxia Peng, Xiaoling Wang
BACKGROUND: Establishing a comprehensive clinical evaluation system is critical in enacting national drug policy and promoting rational drug use. In China, the 'Clinical Comprehensive Evaluation System for Pediatric Drugs' (CCES-P) project, which aims to compare drugs based on clinical efficacy and cost effectiveness to help decision makers, was recently proposed; therefore, a systematic and objective method is required to guide the process. METHODS: An evidence-based multi-criteria decision analysis model that involved an analytic hierarchy process (AHP) was developed, consisting of nine steps: (1) select the drugs to be reviewed; (2) establish the evaluation criterion system; (3) determine the criterion weight based on the AHP; (4) construct the evidence body for each drug under evaluation; (5) select comparative measures and calculate the original utility score; (6) place a common utility scale and calculate the standardized utility score; (7) calculate the comprehensive utility score; (8) rank the drugs; and (9) perform a sensitivity analysis...
December 15, 2017: Paediatric Drugs
https://www.readbyqxmd.com/read/29246341/comorbidity-of-type-1-diabetes-mellitus-in-patients-with-juvenile-idiopathic-arthritis
#20
Sandra Schenck, Joachim Rosenbauer, Martina Niewerth, Jens Klotsche, Kirsten Minden, Tobias Schwarz, Ivan Foeldvari, Gerd Horneff, Frank Weller-Heinemann, Reinhard W Holl, Angelika Thon
OBJECTIVES: To determine the prevalence of type 1 diabetes mellitus (T1D) in patients with juvenile idiopathic arthritis (JIA) and to characterize patients having both. STUDY DESIGN: Diabetes comorbidity was recorded in the National Pediatric Rheumatologic Database since 2012. Data from the North Rhine-Westphalian diabetes registry served as the reference population for the prevalence of diabetes in the general population. The National Pediatric Rheumatologic Database data were indirectly standardized for age and sex for comparison with the general population...
January 2018: Journal of Pediatrics
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