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Pediatric Drug Development

Constanze Hantel, Igor Shapiro, Giada Poli, Costanza Chiapponi, Martin Bidlingmaier, Martin Reincke, Michaela Luconi, Sara Jung, Felix Beuschlein
In recent years it has been recognized that clinical translation of novel therapeutic strategies for patients with adrenocortical carcinoma (ACC) often fails. These disappointing results indicate that the currently utilized tumor models only poorly reflect relevant pathophysiology and, thereby, do not predict clinical applicability of novel pharmacological approaches. However, also the development of new preclinical ACC models has remained a challenge with only one human cell line (NCI-H295R) and one recently established human pediatric xenograft model (SJ-ACC3) being available for this highly heterogeneous malignancy...
October 15, 2016: Oncotarget
Muhammad Irfan, Sumeira Rabel, Quratulain Bukhtar, Muhammad Imran Qadir, Farhat Jabeen, Ahmed Khan
Over the past few decades, tendency toward innovative drug delivery systems has majorly increased attempts to ensure efficacy, safety and patient acceptability. As discovery and development of new chemical agents is a complex, expensive and time consuming process, so recent trends are shifting toward designing and developing innovative drug delivery systems for existing drugs. Out of those, drug delivery system being very eminent among pediatrics and geriatrics is orally disintegrating films (ODFs). These fast disintegrating films have superiority over fast disintegrating tablets as the latter are associated with the risks of choking and friability...
September 2016: Saudi Pharmaceutical Journal: SPJ: the Official Publication of the Saudi Pharmaceutical Society
Dawood Yusef, Blanca E Gonzalez, Charles B Foster, Johanna Goldfarb, Carla Saracusa, Sarah Worley, Camille Sabella
We report a significantly higher occurrence of adverse events associated with prolonged courses of piperacillin-tazobactam compared with other antibacterial agents used for pediatric outpatient parenteral antimicrobial therapy. These adverse events were characterized by a constellation of clinical findings including fever, hematologic abnormalities, and transaminitis. Adverse events related to piperacillin-tazobactam should be considered in patients who develop a febrile illness associated with a prolonged course of therapy...
October 3, 2016: Pediatric Infectious Disease Journal
Michael T Long, Matthew P Murray
Tongue entrapments within bottles are very rare childhood mishaps. The most immediate hazard in a tongue entrapment is airway obstruction. Tongue entrapment is an airway emergency; contingency planning to maintain airway patency, oxygenation, and ventilation is critical. Here, we report the case of a 5-year-old girl presenting to a pediatric emergency department with an increasingly popular novelty soda bottle, featuring a unique and dangerous design, entrapped on her tongue. Operative removal was anticipated...
September 30, 2016: Pediatric Emergency Care
Luka Verrest, Thomas P C Dorlo
INTRODUCTION: Neglected tropical diseases (NTDs) affect more than one billion people, mainly living in developing countries. For most of these NTDs, treatment is suboptimal. To optimize treatment regimens, clinical pharmacokinetic studies are required where they have not been previously conducted to enable the use of pharmacometric modeling and simulation techniques in their application, which can provide substantial advantages. OBJECTIVES: Our aim was to provide a systematic overview and summary of all clinical pharmacokinetic studies in NTDs and to assess the use of pharmacometrics in these studies, as well as to identify which of the NTDs or which treatments have not been sufficiently studied...
October 15, 2016: Clinical Pharmacokinetics
Tawanda Gumbo, Mamodikoe K Makhene, James A Seddon
There has been a recent expansion of preclinical models to predict the efficacy of regimens to treat adults with tuberculosis. Despite increasing global interest in childhood tuberculosis, these same tools have not been employed to develop pediatric regimens. Children differ from adults in bacillary burden, spectrum of disease, the metabolism and distribution of antituberculosis drugs, and the toxicity experienced. The studies documented in this series describe a proof-of-concept approach to pediatric regimen development...
November 1, 2016: Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America
Devyani Deshpande, Shashikant Srivastava, Eric Nuermberger, Jotam G Pasipanodya, Soumya Swaminathan, Tawanda Gumbo
BACKGROUND:  The regimen of linezolid and moxifloxacin was found to be efficacious in the hollow fiber system model of pediatric intracellular tuberculosis. However, its kill rate was slower than the standard 3-drug regimen of isoniazid, rifampin, and pyrazinamide. We wanted to examine the effect of adding a third oral agent, faropenem, to this dual combination. METHODS:  We performed a series of studies in the hollow fiber system model of intracellular Mycobacterium tuberculosis, by mimicking pediatric pharmacokinetics of each antibiotic...
November 1, 2016: Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America
Shashikant Srivastava, Devyani Deshpande, Jotam G Pasipanodya, Tania Thomas, Soumya Swaminathan, Eric Nuermberger, Tawanda Gumbo
Children with tuberculosis are treated with drug regimens copied from adults despite significant differences in antibiotic pharmacokinetics, pathology, and the microbial burden between childhood and adult tuberculosis. We sought to develop a new and effective oral treatment regimen specific to children of different ages. We investigated and validated the concept that target drug concentrations associated with therapy failure and death in children are different from those of adults. On that basis, we proposed a 4-step program to rapidly develop treatment regimens for children...
November 1, 2016: Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America
Thomas Naert, Robin Colpaert, Tom Van Nieuwenhuysen, Dionysia Dimitrakopoulou, Jannick Leoen, Jurgen Haustraete, Annekatrien Boel, Wouter Steyaert, Trees Lepez, Dieter Deforce, Andy Willaert, David Creytens, Kris Vleminckx
Retinoblastoma is a pediatric eye tumor in which bi-allelic inactivation of the Retinoblastoma 1 (RB1) gene is the initiating genetic lesion. Although recently curative rates of retinoblastoma have increased, there are at this time no molecular targeted therapies available. This is, in part, due to the lack of highly penetrant and rapid retinoblastoma animal models that facilitate rapid identification of targets that allow therapeutic intervention. Different mouse models are available, all based on genetic deactivation of both Rb1 and Retinoblastoma-like 1 (Rbl1), and each showing different kinetics of retinoblastoma development...
October 14, 2016: Scientific Reports
Henrika Wickström, Johan O Nyman, Mathias Indola, Heidi Sundelin, Leif Kronberg, Maren Preis, Jukka Rantanen, Niklas Sandler
Printing technologies were recently introduced to the pharmaceutical field for manufacturing of drug delivery systems. Printing allows on demand manufacturing of flexible pharmaceutical doses in a personalized manner, which is critical for a successful and safe treatment of patient populations with specific needs, such as children and the elderly, and patients facing multimorbidity. Printing of pharmaceuticals as technique generates new demands on the quality control procedures. For example, rapid quality control is needed as the printing can be done on demand and at the point of care...
October 13, 2016: AAPS PharmSciTech
Diana Karpman, Peter Höglund
Orphan drugs designed to treat rare diseases are often overpriced per patient. Novel treatments are sometimes even more expensive for patients with ultra-rare diseases, in part due to the limited number of patients. Pharmaceutical companies that develop a patented life-saving drug are in a position to charge a very high price, which, at best, may enable these companies to further develop drugs for use in rare disease. However, is there a limit to how much a life-saving drug should cost annually per patient? Government interventions and regulations may opt to withhold a life-saving drug solely due to its high price and cost-effectiveness...
October 13, 2016: Pediatric Nephrology: Journal of the International Pediatric Nephrology Association
Şükrü Çekiç, Yakup Canıtez, Nihat Sapan
AIM: Stevens Johnson syndrome and toxic epidermal necrolysis are severe acute mucocutaneous diseases. In this study, we evaluated the clinical aspects of Steven Johnson syndrome, toxic epidermal necrolysis and Stevens-Johnson syndrome/toxic epidermal necrolysis overlap patients who admitted to our clinics in the last five years. MATERIAL AND METHODS: Eleven patients diagnosed as Stevens-Johnson syndrome, toxic epidermal necrolysis and Stevens-Johnson syndrome/toxic epidermal necrolysis overlap in Department of Pediatric Allergy in Uludağ University School of Medicine were included in this study...
September 2016: Türk Pediatri Arşivi
Başak Akyıldız, Serkan Özsoylu, Mehmet Adnan Öztürk, Abdullah İnci, Önder Düzlü, Alparslan Yıldırım
Bee venom is a complex substance which acts in different ways; local or systemic anaphylaxis associated with IgE and direct toxic effects of the large volume of injected venom. We report a 10- year-old boy who was the vulnerable of 5989 honey bee attacks. To the best of our knowledge, this case had the ultimate number of honey bee stings in the literature, until now. He was admitted to pediatric intensive care unit after 3 hours of incident. Plasmapheresis was started to remove circulating venom that can cause many systemic side effects...
November 2015: Turkish Journal of Pediatrics
Preeti Sule, Ronak Tilvawala, Parnia Behinaein, Grant K Walkup, Jeffrey D Cirillo
Although tuberculosis (TB) is one of the most common causes of morbidity and mortality in humans worldwide and diagnostic methods have been in place for more than 100 years, diagnosis remains a challenge. The main problems with diagnosis relate to the time needed to obtain a definitive result, difficulty in obtaining sputum, the primary clinical material used, and the ability of the causative agent, Mycobacterium tuberculosis, to cause disease in nearly any tissue within the body. In order to decrease incidence of TB, discovery of a novel interventions will be required, since current technologies have only been able to control numbers of infections, not reduce them...
September 28, 2016: Tuberculosis
P Zarzosa, N Navarro, I Giralt, C Molist, A Almazán-Moga, I Vidal, A Soriano, M F Segura, R Hladun, A Villanueva, S Gallego, J Roma
The use of preclinical models is essential in translational cancer research and especially important in pediatric cancer given the low incidence of each particular type of cancer. Cell line cultures have led to significant advances in cancer biology. However, cell lines have adapted to growth in artificial culture conditions, thereby undergoing genetic and phenotypic changes which may hinder the translational application. Tumor grafts developed in mice from patient tumor tissues, generally known as patient-derived xenografts (PDXs), are interesting alternative approaches to reproducing the biology of the original tumor...
October 7, 2016: Clinical & Translational Oncology
Marc S Schwartz, Eric P Wilkinson
OBJECTIVE: Auditory brainstem implants (ABIs), which have previously been used to restore auditory perception to deaf patients with neurofibromatosis type 2 (NF2), are now being utilized in other situations, including treatment of congenitally deaf children with cochlear malformations or cochlear nerve deficiencies. Concurrent with this expansion of indications, the number of centers placing and expressing interest in placing ABIs has proliferated. Because ABI placement involves posterior fossa craniotomy in order to access the site of implantation on the cochlear nucleus complex of the brainstem and is not without significant risk, we aim to highlight issues important in developing and maintaining successful ABI programs that would be in the best interests of patients...
September 26, 2016: Laryngoscope
Alexis José Ormeño Julca
Exposure to potentially toxic substances represents 0.3% of annual visits to a pediatric emergency department, being a situation likely to be prevented and that generates a non-negligible morbidity and mortality. The most common route of exposure is the oral and drugs are the products most frequently involved. Esophagitis caustic ingestion of corrosive substances in childhood is now a public health problem in developing countries. Clinical manifestations vary from no injuries serious commitment to high risk of death observed more frequently in patients between one and three years old...
July 2016: Revista de Gastroenterología del Perú: órgano Oficial de la Sociedad de Gastroenterología del Perú
Shanghao Li, Daniel Amat, Zhili Peng, Steven Vanni, Scott Raskin, Guillermo De Angulo, Abdelhameed M Othman, Regina M Graham, Roger M Leblanc
Among various cancers, pediatric brain tumors represent the most common cancer type in children and the second most common cause of cancer related deaths. Anticancer drugs and therapies, such as doxorubicin (Dox), have severe side effects on patients during chemotherapy, especially for children as their bodies are still under development. These side effects are believed to be due to the lack of a delivery system with high efficacy and targeting selectivity, resulting in serious damages of normal cells. To improve the efficacy and selectivity, the transferrin (Trans) receptor mediated endocytosis can be utilized for drug delivery system design, as transferrin receptors are expressed on the blood brain barrier (BBB) and often over expressed in brain tumor cells...
September 22, 2016: Nanoscale
Katrina Ducis, Jian Guan, Michael Karsy, Robert J Bollo
Epilepsy is a common disease in the pediatric population, and the majority of cases are controlled with medications and lifestyle modification. For the children whose seizures are pharmacoresistant, continued epileptic activity can have a severely detrimental impact on cognitive development. Early referral of children with drug-resistant seizures to a pediatric epilepsy surgery center for evaluation is critical to achieving optimal patient outcomes. There are several components to a thorough presurgical evaluation, including a detailed medical history and physical examination, noninvasive testing including electroencephalogram, magnetic resonance imaging (MRI) of the brain, and often metabolic imaging...
July 2016: Translational pediatrics
J Alex Strahan, William H Walker, Taylor R Montgomery, Nancy G Forger
Minocycline, an antibiotic of the tetracycline family, inhibits microglia in many paradigms and is among the most commonly used tools for examining the role of microglia in physiological processes. Microglia may play an active role in triggering developmental neuronal cell death, although findings have been contradictory. To determine whether microglia influence developmental cell death, we treated perinatal mice with minocycline (45 mg/kg) and quantified effects on dying cells and microglial labeling using immunohistochemistry for activated caspase-3 (AC3) and ionized calcium-binding adapter molecule 1 (Iba1), respectively...
October 5, 2016: Developmental Neurobiology
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