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Pediatric Drug Development

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https://www.readbyqxmd.com/read/29778713/concluding-commentary-on-current-trends-to-enhance-the-clinical-safety-of-pediatric-transfusion-focusing-on-prevention-of-untoward-complications-of-hsc-transplantation-newer-strategies-for-improving-the-standards-of-safety-quality-of-stem-cells-expansion-for
#1
REVIEW
Irena Sniecinski, Jerard Seghatchian
Clinical practice and related diagnostic, development and research [DDR] strategies in pediatric transfusion and transplantation cover a broad range of multidisciplinary studies, performed by many professionals involved in this most challenging clinical field [1]. This commentary on the current position and future perspectives in pediatric transfusion field is aimed to highlight major unresolved transfusion complications in pediatric patients, namely red blood cell and platelet alloimmunisation, and new ones such as nosocomial infection, thrombosis and multi-organ failure...
May 16, 2018: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29777528/state-of-the-art-review-on-physiologically-based-pharmacokinetic-modeling-in-pediatric-drug-development
#2
REVIEW
Venkata Yellepeddi, Joseph Rower, Xiaoxi Liu, Shaun Kumar, Jahidur Rashid, Catherine M T Sherwin
Physiologically based pharmacokinetic modeling and simulation is an important tool for predicting the pharmacokinetics, pharmacodynamics, and safety of drugs in pediatrics. Physiologically based pharmacokinetic modeling is applied in pediatric drug development for first-time-in-pediatric dose selection, simulation-based trial design, correlation with target organ toxicities, risk assessment by investigating possible drug-drug interactions, real-time assessment of pharmacokinetic-safety relationships, and assessment of non-systemic biodistribution targets...
May 18, 2018: Clinical Pharmacokinetics
https://www.readbyqxmd.com/read/29775757/molecular-characterization-of-physis-tissue-by-rna-sequencing
#3
Christopher R Paradise, Catalina Galeano-Garces, Daniela Galeano-Garces, Amel Dudakovic, Todd A Milbrandt, Daniel B F Saris, Aaron J Krych, Marcel Karperien, Gabriel B Ferguson, Denis Evseenko, Scott M Riester, Andre J van Wijnen, A Noelle Larson
The physis is a well-established and anatomically distinct cartilaginous structure that is crucial for normal long-bone development and growth. Abnormalities in physis function are linked to growth plate disorders and other pediatric musculoskeletal diseases. Understanding the molecular pathways operative in the physis may permit development of regenerative therapies to complement surgically-based procedures that are the current standard of care for growth plate disorders. Here, we performed next generation RNA sequencing on mRNA isolated from human physis and other skeletal tissues (e...
May 15, 2018: Gene
https://www.readbyqxmd.com/read/29774098/establishment-of-primary-cell-culture-and-an-intracranial-xenograft-model-of-pediatric-ependymoma-a-prospect-for-therapy-development-and-understanding-of-tumor-biology
#4
Lorena Favaro Pavon, Tatiana Tais Sibov, Silvia Regina Caminada de Toledo, Daniela Mara de Oliveira, Francisco Romero Cabral, Jean Gabriel de Souza, Pamela Boufleur, Luciana C Marti, Jackeline Moraes Malheiros, Edgar Ferreira da Cruz, Fernando F Paiva, Suzana M F Malheiros, Manoel A de Paiva Neto, Alberto Tannús, Sérgio Mascarenhas de Oliveira, Nasjla Saba Silva, Andrea Maria Cappellano, Antonio Sérgio Petrilli, Ana Marisa Chudzinski-Tavassi, Sérgio Cavalheiro
Background: Ependymoma (EPN), the third most common pediatric brain tumor, is a central nervous system (CNS) malignancy originating from the walls of the ventricular system. Surgical resection followed by radiation therapy has been the primary treatment for most pediatric intracranial EPNs. Despite numerous studies into the prognostic value of histological classification, the extent of surgical resection and adjuvant radiotherapy, there have been relatively few studies into the molecular and cellular biology of EPNs...
April 24, 2018: Oncotarget
https://www.readbyqxmd.com/read/29770297/current-status-and-future-perspectives-of-the-pumpkin-trial
#5
REVIEW
Iki Adachi
Similar to the adult experience, the use of continuous-flow ventricular assist devices (VADs) has been increasing in the pediatric population. According to the PediMACS registry, continuous-flow VAD currently accounts for >60% of the durable device implantations in the U.S. Nonetheless, the continuous-flow VADs currently in use are designed for adults; this inevitably causes the patient-device size mismatch issue, especially when applied for small children. Pulsatile VADs, therefore, represent the only practical option for this group of patients despite the known risk profile of pulsatile VADs...
April 2018: Translational Pediatrics
https://www.readbyqxmd.com/read/29764494/precision-medicine-becomes-reality-tumor-type-agnostic-therapy
#6
REVIEW
Li Yan, Wei Zhang
Precision medicine just witnessed two breakthroughs in oncology in 2017. Pembrolizumab (Keytruda), Merck's anti-programmed cell death-1 (PD-1) monoclonal antibody (mAb), received accelerated approval in May 2017 by the US Food and Drug Administration for the treatment of adult and pediatric patients with unresectable or metastatic solid tumors that have been identified as having microsatellite instability-high (MSI-H) or deficient DNA mismatch repair (dMMR). Shortly after, nivolumab (Opdivo), Bristol-Myers Squibb's anti-PD-1 mAb, gained an accelerated approval in August 2017 for adult and pediatric patients with MSI-H or dMMR metastatic colorectal cancer that has progressed after standard chemotherapy...
March 31, 2018: Cancer communications
https://www.readbyqxmd.com/read/29763644/immunomodulatory-effect-of-celecoxib-on-hmgb1-tlr4-pathway-in-a-recurrent-seizures-model-in-immature-rats
#7
Mariana Morales-Sosa, Sandra Orozco-Suárez, AngélicaVega-García, Sara Caballero-Chacón, Iris A Feria-Romero
Epileptic seizures constitute an important problem in pediatric neurology during the developmental period. The frequency and nosological significance of seizures, as well as their association with epileptogenesis, may be related to underlying mechanisms such as neuroinflammation. Those mechanisms of response activate inflammatory molecules induced in the neurons, activated glial cells and endothelial cells via the key HMGB1/TLR4 pathway. In this study, the drug celecoxib (CCX) was used as a blocker of the cyclooxygenase 2 (COX-2) and HMGB1/TLR-4 pathways...
May 12, 2018: Pharmacology, Biochemistry, and Behavior
https://www.readbyqxmd.com/read/29763632/lack-of-value-of-juvenile-animal-toxicity-studies-for-supporting-the-safety-of-pediatric-oncology-phase-i-trials
#8
REVIEW
Thomas Visalli, Nancy Bower, Tushar Kokate, Paul A Andrews
Toxicity studies in juvenile animals (JAS) are sometimes performed to support clinical trials in pediatric oncology patients, and there are differing conclusions on the value of JAS for pediatric drug development. This manuscript provides a review of the pediatric clinical data for 25 molecularly-targeted and 4 biologic anticancer therapeutics. Other publications that evaluated the value of JAS in pediatric drug development focus on differences in toxicity between juvenile animals and adult animals. The present paper examines pediatric-specific clinical findings to focus on dose setting in pediatric oncology patients and safety monitoring in terms of the potential value of JAS...
May 12, 2018: Regulatory Toxicology and Pharmacology: RTP
https://www.readbyqxmd.com/read/29760294/breath-profiles-of-children-on-ketogenic-therapy
#9
Vera Ruzsanyi, Miklós Péter Kalapos, Christine Schmidl, Daniela Karall, Sabine Scholl-Buergi, Matthias Baumann
Ketogenic diets (KD) were initially introduced to clinical practice as alimentary approaches with the aim to control drug-resistant epilepsies. Therapy control, particularly at initiation, happens through regular blood analysis and control of urine ketone levels. However, there is a lack of fast, reliable and preferably non-invasive methods of analysis. The exhaled breath contains hundreds of volatile organic compounds (VOCs), which can be modified by diet. The instrumental development of VOC detection converges in the direction of low cost sensors, which can facilitate the self-monitoring of the patients in the future if reliable breath markers are available...
May 15, 2018: Journal of Breath Research
https://www.readbyqxmd.com/read/29759018/accelerating-pediatric-cancer-drug-development-challenges-and-opportunities-for-pediatric-master-protocols
#10
Tahira Khan, Mark Stewart, Samuel Blackman, Raphaël Rousseau, Martha Donoghue, Kenneth Cohen, Nita Seibel, Mark Fleury, Bouchra Benettaib, Raleigh Malik, Gilles Vassal, Gregory Reaman
Although outcomes for children with cancer have significantly improved over the past 40 years, there has been little progress in the treatment of some pediatric cancers, particularly when advanced. Additionally, clinical trial options and availability are often insufficient. Improved genomic and immunologic understanding of pediatric cancers, combined with innovative clinical trial designs, may provide an enhanced opportunity to study childhood cancers. Master protocols, which incorporate the use of precision medicine approaches, coupled with the ability to quickly assess the safety and effectiveness of new therapies, have the potential to accelerate early-phase clinical testing of novel therapeutics and which may result in more rapid approval of new drugs for children with cancer...
January 1, 2018: Therapeutic Innovation & Regulatory Science
https://www.readbyqxmd.com/read/29756718/-drug-reaction-with-eosinophilia-and-systemic-symptoms-due-to-carbamazepine-pediatric-case
#11
Rocío Del P Pereira-Ospina, Ana M Bejarano-Quintero, José M Suescún-Vargas, Javier Y Pinzón-Salamanca
Severe skin reactions include Stevens-Johnson Syndrome, toxic epidermal necrolysis and Drug reaction with Eosinophilia and Systemic Symptoms (DRESS) syndrome, which are uncommon in the pediatric population (incidence 1/1000- 10 000 children), but they have bad prognosis. Drug-sensitive Syndrome with eosinophilia and systemic symptoms consists in rash, hematological abnormalities, lymphadenopathy and organ involvement. We report the case of a 12-year-old male patient who developed this pathology after initiating anticonvulsant therapy with carbamazepine...
June 1, 2018: Archivos Argentinos de Pediatría
https://www.readbyqxmd.com/read/29753346/clinical-outcome-assessments-use-of-normative-data-in-a-pediatric-rare-disease
#12
Dawn Phillips, Beth Leiro
Pediatric rare diseases present unique challenges in clinical trial design and in selection of clinical outcome assessments (COAs) used to support claims in medical product labeling. COAs that discriminate level of function relative to a normative sample are particularly important in the pediatric rare disease setting because the literature is often void of natural history data. Pediatric rare disease clinical trials will often include a wide age distribution. Gross and fine motor skills, communication, cognition, and independence in activities of daily living vary by age, and it may be difficult to distinguish between treatment effect and change due to developmental maturation...
May 2018: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/29751747/a-fatal-case-associated-with-respiratory-syncytial-virus-infection-in-a-young-child
#13
Lili Xu, Hengmiao Gao, Jiansheng Zeng, Jun Liu, Cong Lu, Xiaolei Guan, Suyun Qian, Zhengde Xie
BACKGROUND: Respiratory syncytial virus (RSV) is the most common viral cause of pediatric bronchiolitis and pneumonia worldwide. Risk factors for high mortality and prolonged morbidity after RSV infection include premature birth, bronchopulmonary dysplasia, congenital heart disease, and Down syndrome. However, some previously healthy, full-term children who are infected with RSV also require hospitalization and even experience severe sequelae or death. CASE PRESENTATION: In this report, we present the case of an RSV-associated death of a child who was born at full-term and developed normally up to the age of 2 years old...
May 11, 2018: BMC Infectious Diseases
https://www.readbyqxmd.com/read/29739184/moving-anesthesiology-educational-resources-to-the-point-of-care-experience-with-a-pediatric-anesthesia-mobile-app
#14
Katherine S Monroe, Michael A Evans, Shivani G Mukkamala, Julie L Williamson, Craig S Jabaley, Edward R Mariano, Vikas N O'Reilly-Shah
Background: Educators in all disciplines recognize the need to update tools for the modern learner. Mobile applications (apps) may be useful, but real-time data is needed to demonstrate the patterns of utilization and engagement amongst learners. Methods: We examined the use of an anesthesia app by two groups of learners (residents and anesthesiologist assistant students [AAs]) during a pediatric anesthesiology rotation. The app calculates age and weight-based information for clinical decision support and contains didactic materials for self-directed learning...
May 9, 2018: Korean Journal of Anesthesiology
https://www.readbyqxmd.com/read/29737457/a-physiologically-based-pharmacokinetic-model-to-describe-ciprofloxacin-pharmacokinetics-over-the-entire-span-of-life
#15
Jan-Frederik Schlender, Donato Teutonico, Katrin Coboeken, Katrin Schnizler, Thomas Eissing, Stefan Willmann, Ulrich Jaehde, Heino Stass
BACKGROUND: Physiologically-based pharmacokinetic (PBPK) modeling has received growing interest as a useful tool for the assessment of drug pharmacokinetics by continuous knowledge integration. OBJECTIVE: The objective of this study was to build a ciprofloxacin PBPK model for intravenous and oral dosing based on a comprehensive literature review, and evaluate the predictive performance towards pediatric and geriatric patients. METHODS: The aim of this report was to establish confidence in simulations of the ciprofloxacin PBPK model along the development process to facilitate reliable predictions outside of the tested adult age range towards the extremes of ages...
May 8, 2018: Clinical Pharmacokinetics
https://www.readbyqxmd.com/read/29735109/pediatric-iatrogenic-movement-disorders
#16
Deepti Nagesh, Marcie Goeden, Keith A Coffman
The acute development of a movement disorder is often a dramatic and frightening experience for patients and families, often requiring urgent or emergent evaluation by a neurologist. In the assessment of these patients, one relies on the history, physical and neurologic examination to determine the etiology of the condition. We aim to demonstrate that a thorough medication history is an incredibly critical part of this evaluation as iatrogenic movement disorders can arise from exposure not only to psychoactive medications, but from drugs prescribed for a variety of nonneurologic disorders...
April 2018: Seminars in Pediatric Neurology
https://www.readbyqxmd.com/read/29733088/pediatric-abdominal-migraine-current-perspectives-on-a-lesser-known-entity
#17
REVIEW
Jyoti Mani, Shailender Madani
Background: Abdominal migraine (AM) is a common cause of chronic and recurrent abdominal pain in children. It is characterized by paroxysms of moderate to severe abdominal pain that is midline, periumbilical, or diffuse in location and accompanied by other symptoms including headache, anorexia, nausea, vomiting, or pallor. Despite the presence of comprehensive diagnostic criteria under Rome IV classification of functional gastrointestinal disorders (FGIDs) and International Classification of Headache Disorders, it continues to be an underdiagnosed entity...
2018: Pediatric Health, Medicine and Therapeutics
https://www.readbyqxmd.com/read/29729830/integrating-staff-nurses-in-antibiotic-stewardship-opportunities-and-barriers
#18
Elizabeth Monsees, Lori Popejoy, Mary Anne Jackson, Brian Lee, Jennifer Goldman
BACKGROUND: Nursing has been called for greater participation in antibiotic stewardship. Although many of the functions that are integral to successful stewardship are within the scope of bedside nurses, data evaluating nursing engagement in stewardship are limited. The objective of this study was to identify nurses' roles and confidence in engaging in stewardship practices by conducting a survey of pediatric staff nurses employed at a 354-bed freestanding children's hospital with a well-established prospective audit and feedback stewardship program...
May 2, 2018: American Journal of Infection Control
https://www.readbyqxmd.com/read/29726024/impact-of-nursing-interventions-on-adherence-to-treatment-with-antituberculosis-drugs-in-children-and-young-people-a-non-randomized-controlled-trial
#19
Eva Maria Guix-Comellas, Librada Rozas-Quesada, Eneritz Velasco-Arnaiz, Ariadna Ferres-Canals, Joan Maria Estrada-Masllorens, Enriqueta Force-Sanmartín, Antoni Noguera-Julian
AIM: To evaluate the association of a new nursing intervention on the adherence to antituberculosis treatment in a pediatric cohort (<18 years). BACKGROUND: Tuberculosis remains a public health problem worldwide. The risk of developing tuberculosis after primary infection and its severity are higher in children. Proper adherence to antituberculosis treatment is critical for disease control. DESIGN: Non-randomized controlled trial; Phase 1, retrospective (2011-2013), compared with Phase 2, prospective with intervention (2015-2016), in a referral center for pediatric tuberculosis in Spain (NCT03230409)...
May 3, 2018: Journal of Advanced Nursing
https://www.readbyqxmd.com/read/29720452/arginine-vasopressin-in-cerebrospinal-fluid-is-a-marker-of-sociality-in-nonhuman-primates
#20
Karen J Parker, Joseph P Garner, Ozge Oztan, Erna R Tarara, Jiang Li, Valentina Sclafani, Laura A Del Rosso, Katie Chun, Sean W Berquist, Michael G Chez, Sonia Partap, Antonio Y Hardan, Elliott H Sherr, John P Capitanio
Autism spectrum disorder (ASD) is a neurodevelopmental condition characterized by core social impairments. ASD remains poorly understood because of the difficulty in studying disease biology directly in patients and the reliance on mouse models that lack clinically relevant, complex social cognition abilities. We use ethological observations in rhesus macaques to identify male monkeys with naturally occurring low sociality. These monkeys showed differences in specific neuropeptide and kinase signaling pathways compared to socially competent male monkeys...
May 2, 2018: Science Translational Medicine
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