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Pediatric Drug Development

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https://www.readbyqxmd.com/read/28419087/novel-targeted-therapy-for-neuroblastoma-silencing-the-mxd3-gene-using-sirna
#1
Connie Duong, Sakiko Yoshida, Cathy Chen, Gustavo Barisone, Elva Diaz, Yueju Li, Laurel Beckett, Jong Chung, Reuben Antony, Jan Nolta, Nitin Nitin, Noriko Satake
BACKGROUND: Neuroblastoma is the second most common extracranial cancer in children. Current therapies for neuroblastoma, which use a combination of chemotherapy drugs, have limitations for high-risk subtypes and can cause significant long-term adverse effects in young patients. Therefore, a new therapy is needed. In this study, we investigated the transcription factor MXD3 as a potential therapeutic target in neuroblastoma. METHODS: MXD3 expression was analyzed in five neuroblastoma cell lines by immunocytochemistry and quantitative real time reverse transcription PCR and in 18 primary patient tumor samples by immunohistochemistry...
April 18, 2017: Pediatric Research
https://www.readbyqxmd.com/read/28405936/population-pharmacokinetics-some-observations-in-pediatric-modeling-for-drug-clearance
#2
Iftekhar Mahmood, Million A Tegenge
The objective of this study is to evaluate the predictive performance of several models to predict drug clearance in preterm and term neonates. Five models using different types of allometric and linear models were developed. Two sets of data were used to develop these models (data from preterm neonates to adults and data from preterm and term neonates). Models were also developed with (normalized to 70 kg) or without body weight normalization (body weight 1 kg). From the literature, clearance values for four drugs from neonates to adults were obtained...
April 12, 2017: Clinical Pharmacokinetics
https://www.readbyqxmd.com/read/28399927/bk-viruria-and-viremia-in-children-with-systemic-lupus-erythematosus
#3
Nirupama Gupta, Cuong Q Nguyen, Renee F Modica, Melissa E Elder, Eduardo H Garin
BACKGROUND: BK virus (BKV) is a ubiquitous polyoma virus that lies dormant in the genitourinary tract once acquired in early childhood. In states of cellular immunodeficiency, the virus can reactivate to cause hemorrhagic cystitis and nephritis. Children with systemic lupus erythematosus (SLE) have an increased risk of developing infectious complications secondary to their immunocompromised state from the administration of several immuno-modulatory drugs. Currently, there are no data regarding the prevalence of BK viruria or viremia in children with SLE...
April 11, 2017: Pediatric Rheumatology Online Journal
https://www.readbyqxmd.com/read/28398693/development-and-qualification-of-physiologically-based-pharmacokinetic-models-for-drugs-with-atypical-distribution-behavior-a-desipramine-case-study
#4
T S Samant, V Lukacova, S Schmidt
Desipramine is a secondary tricyclic amine, which is primarily metabolized by cytochrome 2D6. It shows a high volume of distribution (Vss) (10-50 L/kg) due to its high lipophilicity, unspecific phospholipid binding, and lysosomal trapping. The objective of this study was to develop and qualify a physiologically based pharmacokinetic (PBPK) model for desipramine, which accounts for the high Vss of the drug following intravenous and oral administration of doses up to 100 mg. The model also accounts for the extended time to reach maximum concentration after oral dosing due to enterocyte trapping...
April 11, 2017: CPT: Pharmacometrics & Systems Pharmacology
https://www.readbyqxmd.com/read/28398275/natural-killer-t-cell-lymphomas-in-pediatric-and-adolescent-patients
#5
REVIEW
Amanda M Termuhlen
Natural killer/T-cell (NK/T-cell) lymphomas are rare in children and adolescents and consist predominantly of nasal-type extranodal NK/T-cell lymphomas. More than half of pediatric/adolescent patients with NK/T-cell lymphomas present with localized nasal/sinus involvement, but the disease may involve many organs. NK/T-cell lymphoma cells are cytotoxic and associated with necrosis and angioinvasion; they express CD56, CD2, cytoplasmic CD3 epsilon, and to a variable degree CD30. The cells contain Epstein-Barr virus (EBV)-encoded RNA...
March 2017: Clinical Advances in Hematology & Oncology: H&O
https://www.readbyqxmd.com/read/28398096/advances-in-therapy-for-the-prevention-of-hiv-transmission-from-mother-to-child
#6
Marcela A Moretton, Facundo Bertera, Eduardo Lagomarsino, Jennifer Riedel, Diego A Chiappetta, Christian Höcht
Actually, ~17.8 million women and 1.8 million children (<15 years) are currently infected with the Human Immunodeficiency Virus (HIV)/Acquired Immunodeficiency Syndrome (AIDS). Particularly, the majority of pediatric infections (>90%) resulted from "HIV mother-to-child transmission" (MTCT), both in pregnancy, labour, delivery and later by breastfeeding. Due to its high pediatric incidence, MTCT represents a public health concern. Areas covered: In this review, we focus on available treatments and antiretroviral drugs recommended by the World Health Organization, and the main clinical investigations in antiretroviral pharmacotherapy to prevent the MTCT...
April 11, 2017: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/28396659/disease-phenotype-and-outcome-depending-on-the-age-at-disease-onset-in-patients-carrying-the-r92q-low-penetrance-variant-in-tnfrsf1a-gene
#7
Estíbaliz Ruiz-Ortiz, Estíbaliz Iglesias, Alessandra Soriano, Segundo Buján-Rivas, Marta Español-Rego, Raul Castellanos-Moreira, Adrià Tomé, Jordi Yagüe, Jordi Antón, José Hernández-Rodríguez
BACKGROUND: Tumor necrosis factor receptor-associated periodic syndrome (TRAPS) is an autosomal-dominant autoinflammatory disease caused by mutations in the TNFRSF1A gene. R92Q, a low-penetrance variant, is usually associated with a milder TRAPS phenotype than structural or pathogenic mutations. No studies differentiating R92Q-related disease in patients with pediatric and adult onset have been performed to date. OBJECTIVE: To analyze clinical features and disease outcomes in patients diagnosed with TRAPS associated with R92Q variant and to investigate differences between patients with pediatric and adult disease onset...
2017: Frontiers in Immunology
https://www.readbyqxmd.com/read/28393062/the-likelihood-of-preventing-respiratory-exacerbations-in-children-and-adolescents-with-either-chronic-suppurative-lung-disease-or-bronchiectasis
#8
REVIEW
Kerry-Ann F O'Grady, Keith Grimwood
Chronic suppurative lung disease (CSLD) and bronchiectasis in children and adolescents are important causes of respiratory morbidity and reduced quality of life (QoL), also leading to subsequent premature death during adulthood. Acute respiratory exacerbations in pediatric CSLD and bronchiectasis are important markers of disease control clinically, given that they impact upon QoL and increase health-care-associated costs and can adversely affect future lung functioning. Preventing exacerbations in this population is, therefore, likely to have significant individual, familial, societal, and health-sector benefits...
2017: Frontiers in Pediatrics
https://www.readbyqxmd.com/read/28387160/long-term-treatment-in-pediatric-asthma-an-update-on-chemical-pharmacotherapy
#9
Carolina Vitale, Angelantonio Maglio, Corrado Pelaia, Alessandro Vatrella
Asthma is the most common chronic disease in childhood, affecting approximately 10% of all children, and is the leading cause of hospitalization in developed countries. In this paper we aimed to review the evidence on chemical pharmacotherapy for long-term treatment of pediatric asthma, according to the latest updates. Area covered: Long-term treatment, essential for controlling symptoms and reducing future risks including exacerbations and decline in lung function, includes control agents such as inhaled corticosteroids, long-acting beta2-adrenergic agonists, and leukotriene modifiers...
April 20, 2017: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/28386633/rlip76-inhibition-a-promising-developmental-therapy-for-neuroblastoma
#10
REVIEW
Sharad S Singhal, Lokesh Nagaprashantha, Preeti Singhal, Sulabh Singhal, Jyotsana Singhal, Sanjay Awasthi, David Horne
Refractory and relapsed neuroblastoma (NB) present with significant challenges in clinical management. Though primary NBs largely with wild-type p53 respond well to interventions, dysfunctional signaling in the p53 pathways in a MYCN oncogene driven background is found in a number of children with NB. The p53-mutant NB is largely unresponsive to available therapies and p53-independent targeted therapeutics represents a vital need in pediatric oncology. We analyzed the findings on mercapturic acid pathway (MAP) transporter RLIP76, which has broad and critical effects on multiple pathways as essential for carcinogenesis, oxidative stress and drug-resistance, is over-expressed in NB...
April 6, 2017: Pharmaceutical Research
https://www.readbyqxmd.com/read/28384261/cost-effectiveness-of-meglumine-antimoniate-versus-miltefosine-caregiver-dot-for-the-treatment-of-pediatric-cutaneous-leishmaniasis
#11
Brandon A Berger, Alexandra Cossio, Nancy Gore Saravia, Maria Del Mar Castro, Sergio Prada, Allison H Bartlett, Mai T Pho
BACKGROUND: Oral miltefosine has been shown to be non-inferior to first-line, injectable meglumine antimoniate (MA) for the treatment of cutaneous leishmaniasis (CL) in children. Miltefosine may be administered via in-home caregiver Directly Observed Therapy (cDOT), while patients must travel to clinics to receive MA. We performed a cost-effectiveness analysis comparing miltefosine by cDOT versus MA for pediatric CL in southwest Colombia. METHODOLOGY/PRINCIPLE FINDINGS: We developed a Monte Carlo model comparing the cost-per-cure of miltefosine by cDOT compared to MA from patient, government payer, and societal perspectives (societal = sum of patient and government payer perspective costs)...
April 6, 2017: PLoS Neglected Tropical Diseases
https://www.readbyqxmd.com/read/28377092/interdisciplinary-medication-decision-making-by-pharmacists-in-pediatric-hospital-settings-an-ethnographic-study
#12
Ellie Rosenfeld, Sharon Kinney, Carlye Weiner, Fiona Newall, Allison Williams, Noel Cranswick, Ian Wong, Narelle Borrott, Elizabeth Manias
OBJECTIVE: Children are particularly vulnerable to experiencing medication incidents in hospitals. Making sound medication decisions is therefore of paramount importance. Prior research has principally described pharmacists' role in reducing medication errors. There is a dearth of information about pharmacists' interactions with pediatric hospital staff across disciplines in resolving medication issues. The aim of this study was to examine interdisciplinary medication decision making by pharmacists in pediatric hospital settings...
March 22, 2017: Research in Social & Administrative Pharmacy: RSAP
https://www.readbyqxmd.com/read/28376825/antiretroviral-therapy-supply-chain-quality-control-and-assurance-in-improving-people-living-with-hiv-therapeutic-outcomes-in-cameroon
#13
M P Ngogang Djobet, David Singhe, Julienne Lohoue, Christopher Kuaban, Jeanne Ngogang, Ernest Tambo
BACKGROUND: Evaluation of medication efficacy and safety is an essential guarantee to successful therapeutic outcome in public health practices. However, larger distribution chain supply in developing countries such as Cameroon is often challenged by counterfeit drugs, poor manufacturing, storage and degradation leading to health and patient adverse consequences. Yet, access to supply chain management in strengthening ARVs quality assurance and outcomes remains poorly documented. More than 53,000 patients have been enrolled on free ARVs medications, but little is documented on quality assurance and validity of safety for affected populations along the supply chain management since 2008...
April 4, 2017: AIDS Research and Therapy
https://www.readbyqxmd.com/read/28376230/target-and-agent-prioritization-for-the-children-s-oncology-group-national-cancer-institute-pediatric-match-trial
#14
Carl E Allen, Theodore W Laetsch, Rajen Mody, Meredith S Irwin, Megan S Lim, Peter C Adamson, Nita L Seibel, D Williams Parsons, Y Jae Cho, Katherine Janeway
Over the past decades, outcomes for children with cancer have improved dramatically through serial clinical trials based in large measure on dose intensification of cytotoxic chemotherapy for children with high-risk malignancies. Progress made through such dose intensification, in general, is no longer yielding further improvements in outcome. With the revolution in sequencing technologies and rapid development of drugs that block specific proteins and pathways, there is now an opportunity to improve outcomes for pediatric cancer patients through mutation-based targeted therapeutic strategies...
May 1, 2017: Journal of the National Cancer Institute
https://www.readbyqxmd.com/read/28369788/model-based-approach-for-optimizing-study-design-and-clinical-drug-performances-of-extended-release-formulations-of-methylphenidate-for-the-treatment-of-adhd
#15
R Gomeni, Fmm Bressolle-Gomeni, T J Spencer, S V Faraone, L Fang, A Babiskin
Methylphenidate (MPH) is currently used to treat children with attention deficit hyperactivity disorder (ADHD). Several extended-release (ER) formulations characterized by a dual release process were developed to improve efficacy over an extended duration. In this study, a model-based approach using literature data was developed to: a) evaluate the most efficient pharmacokinetic (PK) model to characterize the complex PK profile of MPH ER formulations; b) provide PK endpoint metrics for comparing ER formulations; c) define criteria for optimizing development of ER formulations using a convolution-based model linking in-vitro release, in-vivo release and hour-by-hour behavioral ratings of ADHD symptoms; and d) define an optimized trial design for assessing the activity of MPH in pediatric populations...
March 29, 2017: Clinical Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/28368673/development-and-evaluation-of-orally-disintegrating-tablets-comprising-taste-masked-mirtazapine-granules
#16
Simay Yıldız, Eren Aytekin, Burçin Yavuz, Sibel Bozdağ Pehlivan, İmran Vural, Nurşen Ünlü
INTRODUCTION: Orally disintegrating tablets (ODTs) provide an important treatment option for pediatric, geriatric, and psychiatric patients. In our previous study, we have performed the initial studies for the formulation development and characterization of new ODT formulations containing a bitter taste drug, mirtazapine, coated with 6% (w/w) Eudragit® E-100 (first group of formulations, FGF) without taste evaluation. In present study, coating ratio of the drug was increased to 8% (w/w) (second group of formulations, SGF) to examine the effect of increased coating ratio of drug on in vitro characterization of the formulations including in vitro taste masking study...
April 3, 2017: Pharmaceutical Development and Technology
https://www.readbyqxmd.com/read/28366443/modeling-the-mutational-and-phenotypic-landscapes-of-pelizaeus-merzbacher-disease-with-human-ipsc-derived-oligodendrocytes
#17
Zachary S Nevin, Daniel C Factor, Robert T Karl, Panagiotis Douvaras, Jeremy Laukka, Martha S Windrem, Steven A Goldman, Valentina Fossati, Grace M Hobson, Paul J Tesar
Pelizaeus-Merzbacher disease (PMD) is a pediatric disease of myelin in the central nervous system and manifests with a wide spectrum of clinical severities. Although PMD is a rare monogenic disease, hundreds of mutations in the X-linked myelin gene proteolipid protein 1 (PLP1) have been identified in humans. Attempts to identify a common pathogenic process underlying PMD have been complicated by an incomplete understanding of PLP1 dysfunction and limited access to primary human oligodendrocytes. To address this, we generated panels of human induced pluripotent stem cells (hiPSCs) and hiPSC-derived oligodendrocytes from 12 individuals with mutations spanning the genetic and clinical diversity of PMD-including point mutations and duplication, triplication, and deletion of PLP1-and developed an in vitro platform for molecular and cellular characterization of all 12 mutations simultaneously...
April 6, 2017: American Journal of Human Genetics
https://www.readbyqxmd.com/read/28363856/bitterness-prediction-in-silico-a-step-towards-better-drugs
#18
Malkeet Singh Bahia, Ido Nissim, Masha Niv
Bitter taste is innately aversive and thought to protect against consuming poisons. Bitter taste receptors (Tas2Rs) are G-protein coupled receptors, expressed both orally and extra-orally and proposed as novel targets for several indications, including asthma. Many clinical drugs elicit bitter taste, suggesting the possibility of drugs re-purposing. On the other hand, the bitter taste of medicine presents a major compliance problem for pediatric drugs. Thus, efficient tools for predicting, measuring and masking bitterness of active pharmaceutical ingredients (APIs) are required by the pharmaceutical industry...
March 28, 2017: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/28362694/the-challenge-of-treating-children-with-hepatitis-c-virus-infection
#19
Giuseppe Indolfi, Claire Thorne, Manal H El-Sayed, Carlo Giaquinto, Regino P Gonzalez-Peralta
The development of oral hepatitis C virus direct acting antivirals has revolutionized the therapeutic field. Nowadays, multiple safe and highly effective antiviral regimens are commercially available to treat adults with hepatitis C infection. These new regimens for the first time, genuinely raise the prospects of eradicating hepatitis C virus. However, many challenges remain: from identifying infected individuals to optimizing treatment and ensuring global access to antiviral therapy to all population groups, including children...
March 30, 2017: Journal of Pediatric Gastroenterology and Nutrition
https://www.readbyqxmd.com/read/28362356/severe-pneumonia-caused-by-influenza-a-h1n1-virus-successfully-managed-with-extracorporeal-life-support-in-a-comorbid-former-preterm-infant
#20
Genny Raffaeli, Giacomo Cavallaro, Lorenza Pugni, Ernesto Leva, Andrea Artoni, Simona Neri, Chiara Baracetti, Mauro Cotza, Valerio Gentilino, Leonardo Terranova, Susanna Esposito, Fabio Mosca
Influenza A (H1N1) virus infection is a global health burden, leading to significant pediatric morbidity and mortality. Prematurity, young age and comorbidities are important risk factors for unfavorable outcomes. Preventive strategies, such as healthcare workers and household contacts vaccination as well as the implementation of infection control practices during the epidemic season, are crucial to protect the most vulnerable populations. Early diagnosis, timely administration of antiviral drugs and supportive therapy are crucial to lead to a complete recovery...
March 31, 2017: International Journal of Environmental Research and Public Health
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