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Pediatric Drug Development

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https://www.readbyqxmd.com/read/28938089/drug-discovery-and-chemical-biology-of-cancer-epigenetics
#1
REVIEW
Scott Ribich, Darren Harvey, Robert A Copeland
Comprehensive whole-exome sequencing, DNA copy-number determination, and transcriptomic analyses of diverse cancers have greatly expanded our understanding of the biology of many tumor types. In addition to mutations in the common cell-of-origin specific driver mutations, these studies have also revealed a large number of loss-of-function and gain-of-function mutations in chromatin-modifying proteins (CMPs). This has revealed that epigenetic dysregulation is a common feature of most pediatric and adult cancers...
September 21, 2017: Cell Chemical Biology
https://www.readbyqxmd.com/read/28937537/a-comparison-of-biologic-screening-and-diagnostic-indicators-to-detect-in-utero-opiate-and-cocaine-exposure-among-mother-infant-dyads
#2
Meagan Stabler, Peter Giacobbi, Ilana Chertok, Leann Long, Lesley Cottrell, Panitan Yossuck
BACKGROUND: Opioid and cocaine antenatal substance use can result in significant obstetric and pediatric health implications. Accurate detection of in utero exposed neonates can improve patient care and health outcomes. Therefore, the effectiveness of mother-infant biological and diagnostic indicators collected at labor and delivery to provide accurate detection of in utero opiate and cocaine exposure was assessed. METHODS: A retrospective medical chart review included 335 mother-infant dyads exposed to antenatal substances who were delivered between January 2009 and March 2014...
September 19, 2017: Therapeutic Drug Monitoring
https://www.readbyqxmd.com/read/28933291/pharmacotranscriptomic-biomarkers-in-glucocorticoid-treatment-of-pediatric-inflammatory-bowel-disease
#3
Marianna Lucafò, Biljana Stankovic, Nikola Kotur, Alessia Di Silvestre, Stefano Martelossi, Alessandro Ventura, Branka Zukic, Sonya Pavlovic, Giuliana Decorti
Pharmacotranscriptomics aims to reach more accurate drug dosing based on interindividual transcriptome variations. Here, we provide an overview of RNA biomarkers that could predict the response to glucocorticoids (GCs), considered the standard for treatment of inflammatory bowel diseases (IBD), both in adult and pediatric patients. Although new biological agents are very effective in the IBD treatment, GCs are still widely used for induction of remission in IBD patients with moderate to severe disease. It is important to identify patients that are poor responders to GCs therapy, because suboptimal response is frequent and associated with various side effects...
September 20, 2017: Current Medicinal Chemistry
https://www.readbyqxmd.com/read/28932575/pediatric-lung-transplantation
#4
REVIEW
Christian Benden
Pediatric lung transplantation has been undertaken since the 1980s, and it is today considered an accepted therapy option in carefully selected children with end-stage pulmonary diseases, providing carefully selected children a net survival benefit and improved health-related quality of life. Nowadays, >100 pediatric lung transplants are done worldwide every year. Here, specific pediatric aspects of lung transplantation are reviewed such as the surgical challenge, effects of immunosuppression on the developing pediatric immune system, and typical infections of childhood, as it is vital to comprehend that children undergoing lung transplants present a real challenge as children are not 'just small adults'...
August 2017: Journal of Thoracic Disease
https://www.readbyqxmd.com/read/28925976/shiga-toxin-therapeutics-beyond-neutralization
#5
REVIEW
Gregory Hall, Shinichiro Kurosawa, Deborah J Stearns-Kurosawa
Ribotoxic Shiga toxins are the primary cause of hemolytic uremic syndrome (HUS) in patients infected with Shiga toxin-producing enterohemorrhagic Escherichia coli (STEC), a pathogen class responsible for epidemic outbreaks of gastrointestinal disease around the globe. HUS is a leading cause of pediatric renal failure in otherwise healthy children, resulting in a mortality rate of 10% and a chronic morbidity rate near 25%. There are currently no available therapeutics to prevent or treat HUS in STEC patients despite decades of work elucidating the mechanisms of Shiga toxicity in sensitive cells...
September 19, 2017: Toxins
https://www.readbyqxmd.com/read/28925064/improving-realism-in-clinical-trial-simulations-via-real-world-data
#6
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/28923290/use-of-adult-sensory-panel-to-study-individual-differences-in-the-palatability-of-a-pediatric-hiv-treatment-drug
#7
Julie A Mennella, Phoebe S Mathew, Elizabeth D Lowenthal
PURPOSE: The recommended first-line treatment for young children infected with HIV includes the liquid formulation of the co-formulated protease inhibitors lopinavir/ritonavir (Kaletra(®) [Abbott Laboratories, Chicago, Illinois]). Clinical reports indicate that some children readily accept the taste of Kaletra, whereas others strongly reject it, which can deter therapeutic adherence and outcomes. METHODS: As a proof-of-concept approach, a sensory panel of genotyped adults was used to document the range of individual differences in the taste and palatability (hedonics) of the liquid formulation of Kaletra and other taste stimuli, including common excipients...
September 15, 2017: Clinical Therapeutics
https://www.readbyqxmd.com/read/28922349/cytochrome-p450-and-flavin-containing-monooxygenase-families-age-dependent-differences-in-expression-and-functional-activity
#8
Nicole R Zane, Yao Chen, Michael Zhuo Wang, Dhiren R Thakker
BACKGROUND: Age-dependent differences in pharmacokinetics exist for metabolically cleared medications. Differential contributions in the cytochrome P450 3A (CYP3A), CYP2C, and flavin-containing monooxygenases (FMO) families play an important role in the metabolic clearance of a large number of drugs administered to children. METHODS: Unlike previous semi-quantitative characterization of age-dependent changes in the expression of genes and proteins (Western Analysis), this study quantifies both gene and absolute protein expression in the same fetal, pediatric and adult hepatic tissue...
September 18, 2017: Pediatric Research
https://www.readbyqxmd.com/read/28922236/efficacy-outcome-measures-for-pediatric-procedural-sedation-clinical-trials-an-acttion-systematic-review
#9
Mark R Williams, Michael Nayshtut, Amie Hoefnagel, Andrew McKeown, Douglas W Carlson, Joseph Cravero, Jenifer Lightdale, Keira P Mason, Stephen Wilson, Dennis C Turk, Robert H Dworkin, Denham S Ward
Objective evaluations comparing different techniques and approaches to pediatric procedural sedation studies have been limited by a lack of consistency among the outcome measures used in assessment. This study reviewed those existing measures, which have undergone psychometric analysis in a pediatric procedural sedation setting, to determine to what extent and in what circumstances their use is justified across the spectrum of procedures, age groups, and techniques. The results of our study suggest that a wide range of measures has been used to assess the efficacy and effectiveness of pediatric procedural sedation...
September 14, 2017: Anesthesia and Analgesia
https://www.readbyqxmd.com/read/28921643/bridging-adult-experience-to-pediatrics-in-oncology-drug-development
#10
Ruby Leong, Hong Zhao, Gregory Reaman, Qi Liu, Yaning Wang, Clinton F Stewart, Gilbert Burckart
Pediatric drug development in the United States has grown under the current regulations made permanent by the Food and Drug Administration Safety and Innovation Act of 2012. Over 1200 pediatric studies have now been submitted to the US FDA, but there is still a high rate of failure to obtain pediatric labeling for the indication pursued. Pediatric oncology represents special problems in that the disease is most often dissimilar to any cancer found in the adult population. Therefore, the development of drug dosing in pediatric oncology patients represents a special challenge...
October 2017: Journal of Clinical Pharmacology
https://www.readbyqxmd.com/read/28906365/the-antibiotic-susceptibility-patterns-of-uropathogens-among-children-with-urinary-tract-infection-in-shiraz
#11
Gholamreza Pouladfar, Mitra Basiratnia, Mojtaba Anvarinejad, Pejman Abbasi, Fatemeh Amirmoezi, Samaneh Zare
Urinary tract infection (UTI) is one of the most common bacterial infections in pediatrics. Delay in diagnosis and treatment can cause significant morbidity. The physicians knowledge regarding the symptoms, microorganisms that caused UTI, and effective antibiotics in a geographical area can help them to select the appropriate antibiotics. This study was performed to determine the prevalence of bacteria that cause UTI and their susceptibility to common antibiotics as well as the common symptoms and associated factors in children of Shiraz, Southern Iran...
September 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28903160/safety-of-systemic-agents-for-the-treatment-of-pediatric-psoriasis
#12
Inge M G J Bronckers, Marieke M B Seyger, Dennis P West, Irene Lara-Corrales, Megha Tollefson, Wynnis L Tom, Marcia Hogeling, Leah Belazarian, Claus Zachariae, Emmanuel Mahé, Elaine Siegfried, Sandra Philipp, Zsuzsanna Szalai, Ruth Ann Vleugels, Kristen Holland, Ruth Murphy, Eulalia Baselga, Kelly Cordoro, Jo Lambert, Alex Alexopoulos, Ulrich Mrowietz, Wietske Kievit, Amy S Paller
Importance: Use of systemic therapies for moderate to severe psoriasis in children is increasing, but comparative data on their use and toxicities are limited. Objective: To assess patterns of use and relative risks of systemic agents for moderate to severe psoriasis in children. Design, Setting, and Participants: A retrospective review was conducted at 20 centers in North America and Europe, and included all consecutive children with moderate to severe psoriasis who used systemic medications or phototherapy for at least 3 months from December 1, 1990, to September 16, 2014...
September 13, 2017: JAMA Dermatology
https://www.readbyqxmd.com/read/28900869/enabling-development-of-paediatric-medicines-in-europe-10-years-of-the-eu-paediatric-regulation
#13
Paolo A Tomasi, Gunter F Egger, Chrissi Pallidis, Agnes Saint-Raymond
The year 2017 marks the tenth anniversary of entry into force of the Paediatric Regulation in the European Union (EU). This law aimed to stimulate the development of paediatric medicines and provide more information on their use, as a response to the lack of evidence and approval of medicines for children. The European Medicines Agency (EMA) has had a central role in the implementation of the Regulation. Pharmaceutical companies need to submit a paediatric investigation plan (PIP) to the EMA's Paediatric Committee (PDCO) for every new medicine, unless an exemption (waiver) is granted...
September 12, 2017: Paediatric Drugs
https://www.readbyqxmd.com/read/28890858/extrapolation-of-efficacy-in-pediatric-drug-development-and-evidence-based-medicine-progress-and-lessons-learned
#14
Haihao Sun, Jean W Temeck, Wiley Chambers, Ginger Perkins, Renan Bonnel, Dianne Murphy
BACKGROUND: "Complete Extrapolation" of efficacy from adult or other pediatric data, to the pediatric population, is an important scientific tool that reduces the need for pediatric efficacy trials. Dose finding and safety studies in pediatrics are still needed. "No Extrapolation" requires 2 pediatric efficacy trials. "Partial Extrapolation" eliminates the need to conduct 2 pediatric efficacy trials; 1 efficacy or exposure/response study may be sufficient. We examined pediatric extrapolation from 2009 to 2014 evaluating any changes in extrapolation assumptions and the causes for these changes since a prior analysis published in 2011...
2017: Therapeutic Innovation & Regulatory Science
https://www.readbyqxmd.com/read/28880998/impaired-bone-and-muscle-development-in-young-people-treated-with-antiepileptic-drugs
#15
Peter J Simm, Sebastian Seah, Alex Gorelik, Lauren Gilbert, Jenning Nuguid, George A Werther, Mark T Mackay, Jeremy L Freeman, Sandra J Petty, John D Wark
OBJECTIVE: Antiepileptic drugs (AEDs) are associated with reduced bone density, balance impairment, and increased fracture risk in adults. However, pediatric data are limited. Therefore, we aimed to examine bone, muscle, and balance outcomes in young patients taking AEDs. METHODS: We undertook a case-control study utilizing an AED exposure-discordant matched-pair approach. Subjects were aged 5-18 years with at least 12 months of AED exposure. Pairs were twins, nontwin siblings and first cousins, sex- and age-matched (to within 2 years), allowing for greater power than with unrelated control subjects...
September 7, 2017: Epilepsia
https://www.readbyqxmd.com/read/28879787/moving-forward-with-treatment-options-for-hiv-infected-children
#16
Jean-Christophe Beghin, Jean Cyr Yombi, Jean Ruelle, Dimitri Van der Linden
Current international guidelines recommend to treat all HIV-1 infected patients regardless of CD4 cell count. Despite the remarkable worldwide progress for universal access to antiretroviral during the last decade, the pediatric population remains fragile due to lack of randomized studies, inappropriate antiretroviral formulations, adherence difficulties, drug toxicity and development of resistance. Areas covered: This review summarizes the latest recommendations and advances for the treatment of HIV-infected children and highlights the potential complications of a lifelong antiretroviral treatment initiated early in life...
September 7, 2017: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/28877075/microsatellite-instability-a-predictive-biomarker-for-cancer-immunotherapy
#17
Liisa Chang, Minna Chang, Hanna M Chang, Fuju Chang
Immunotherapy has shown promising results in various types of cancers. Checkpoint inhibitor drugs developed for cancer immunotherapy have been approved by the US Food and Drug Administration (FDA) for patients with advanced melanoma, non-small cell lung cancer, renal cell carcinoma, bladder cancers, and refractory Hodgkin lymphoma. In the latest announcement, the FDA has granted accelerated approval to pembrolizumab for pediatric and adult patients with microsatellite instability-high (MSI-H) or mismatch repair-deficient solid tumors...
September 4, 2017: Applied Immunohistochemistry & Molecular Morphology: AIMM
https://www.readbyqxmd.com/read/28860717/development-of-idursulfase-therapy-for-mucopolysaccharidosis-type-ii-hunter-syndrome-the-past-the-present-and-the-future
#18
REVIEW
David Ah Whiteman, Alan Kimura
Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare, multisystemic, progressive lysosomal storage disease caused by deficient activity of the iduronate-2-sulfatase (I2S) enzyme. Accumulation of the glycosaminoglycans dermatan sulfate and heparan sulfate results in a broad range of disease manifestations that are highly variable in presentation and severity; notably, approximately two-thirds of individuals are affected by progressive central nervous system involvement. Historically, management of this disease was palliative; however, during the 1990s, I2S was purified to homogeneity for the first time, leading to cloning of the corresponding gene and offering a means of addressing the underlying cause of MPS II using enzyme replacement therapy (ERT)...
2017: Drug Design, Development and Therapy
https://www.readbyqxmd.com/read/28860113/physiologically-based-pharmacokinetic-pbpk-model-of-the-cyp2d6-probe-atomoxetine-extrapolation-to-special-populations-and-drug-drug-interactions
#19
Weize Huang, Mariko Nakano, Jennifer E Sager, Isabelle Ragueneau-Majlessi, Nina Isoherranen
Physiologically based pharmacokinetic (PBPK) modeling of drug disposition and drug-drug interactions has become a key component of drug development. PBPK modeling has also been considered as an approach to predict drug disposition in special populations. However, whether models developed and validated in healthy populations can be extrapolated to special populations is not well established. The goal of this study was to determine whether a drug specific PBPK model validated using healthy populations could be used to predict drug disposition in specific populations and in organ impairment...
August 31, 2017: Drug Metabolism and Disposition: the Biological Fate of Chemicals
https://www.readbyqxmd.com/read/28856931/acceptability-of-orodispersible-films-for-delivery-of-medicines-to-infants-and-preschool-children
#20
Mine Orlu, Sejal R Ranmal, Yucheng Sheng, Catherine Tuleu, Paul Seddon
Orodispersible films (ODFs) possess potential to facilitate oral drug delivery to children; however, documentation of their acceptability in this age group is lacking. This study is the first to explore the initial perceptions, acceptability and ease of use of ODFs for infants and preschool children, and their caregivers through observed administration of the type of dosage form. Placebo ODFs were administered to children stratified into aged 6 to 12 months, 1 year, 2 years, 3 years, 4 years and 5 years old and into those with an acute illness or long-term stable condition in hospital setting...
November 2017: Drug Delivery
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