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Pediatric Drug Development

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https://www.readbyqxmd.com/read/28735109/design-and-rationale-for-a-real-world-observational-cohort-of-patients-with-nonalcoholic-fatty-liver-disease-the-target-nash-study
#1
A Sidney Barritt, Norman Gitlin, Samuel Klein, Anna S Lok, Rohit Loomba, Laura Malahias, Margaret Powell, Miriam B Vos, L Michael Weiss, Kenneth Cusi, Brent A Neuschwander-Tetri, Arun Sanyal
Nonalcoholic fatty liver disease (NAFLD) is highly prevalent and can lead to cirrhosis, hepatocellular carcinoma, and end-stage liver disease. NAFLD comprises the spectrum from simple steatosis (nonalcoholic fatty liver, NAFL), to steatosis with inflammation (nonalcoholic steatohepatitis, NASH). Current primary therapy recommended for NAFLD is weight loss induced by lifestyle modification. The difficulty in achieving this has led to robust pharmacological therapy development. While new drugs may show efficacy in selected phase II/III clinical trial populations, their real-world effectiveness is unknown...
July 19, 2017: Contemporary Clinical Trials
https://www.readbyqxmd.com/read/28734784/drug-delivery-innovations-to-address-global-health-challenges-for-pediatric-and-geriatric-populations-through-improvements-in-patient-compliance
#2
Joshua Boateng
Despite significant advances in pharmaceutical and biotechnological drug discovery, the global population is plagued with many challenging diseases. These are further compounded by anticipated explosion in an ageing population, which presents several problems such as polypharmacy, dysphagia and neurological conditions, resulting in non-compliance and disease complications. For antibiotics, poor compliance, can result in development of drug resistant infections which can be fatal. Further, children, especially, in developing countries die unnecessarily from easily treatable diseases (e...
July 19, 2017: Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/28730261/post-medication-stevens-johnson-syndrome-in-a-girl-hospitalized-for-a-norovirus-and-rotavirus-infection
#3
Simona Dumitra, LuminiŢa Pilat, Alina Iftode, Ozana Nicoleta Bălan, Carmen Ramona StănculeŢ, Elena Claudia Covaci, Alexandru Fica Mircea Onel, Carmen Nicoleta Crişan
Stevens-Johnson syndrome (SJS) is a cutaneous mucosal disorder characterized by extended necrosis and detachment of the epidermis affecting <10% of the body surface, caused by drugs or infections. The authors report a case of a girl with Depakine resistant epilepsy, who develops a SJS in the third week of introducing lamotrigine. The girl also presents an acute diarrheal disease with double viral etiology - rotavirus and norovirus. The clinical image comprises polymorphic erythematous maculopapular exanthema with vesicular and bullous elements, with ulcerations and desquamations at the level of the eyelids, mouth, anogenital area and tegument denuding at the level of the abdomen and limbs...
2017: Romanian Journal of Morphology and Embryology, Revue Roumaine de Morphologie et Embryologie
https://www.readbyqxmd.com/read/28729186/-tuberculosis-treatment-for-children-an-update
#4
María José Mellado Peña, Begoña Santiago García, Fernando Baquero-Artigao, David Moreno Pérez, Roi Piñeiro Pérez, Ana Méndez Echevarría, José Tomás Ramos Amador, David Gómez-Pastrana Durán, Antoni Noguera Julian
Tuberculosis (TB) is the most important infectious disease all over the world, with a high morbidity and mortality. Pediatric tuberculosis has been a neglected epidemic, due to the difficulties in assessing its global impact, reduced incidence and lower infectivity compared to adults. In 2015, the WHO reported 1 million cases of paediatric TB and 169,000 deaths. In Europe, the emergence of MDR TB is a major concern, representing 16% of the new diagnosis in Eastern Europe. In 2014, it was estimated that about 219,000 children were infected by MDR-TB-strains in Europe, and 2,120 developed the disease...
July 17, 2017: Anales de Pediatría: Publicación Oficial de la Asociación Española de Pediatría (A.E.P.)
https://www.readbyqxmd.com/read/28729174/challenges-and-strategies-to-facilitate-formulation-development-of-pediatric-drug-products-safety-qualification-of-excipients
#5
Lorrene A Buckley, Smita Salunke, Karen Thompson, Gerri Baer, Darren Fegley, Mark A Turner
A public workshop entitled "Challenges and strategies to facilitate formulation development of pediatric drug products" focused on current status and gaps as well as recommendations for risk-based strategies to support the development of pediatric age-appropriate drug products. Representatives from industry, academia, and regulatory agencies discussed the issues within plenary, panel, and case-study breakout sessions. By enabling practical and meaningful discussion between scientists representing the diversity of involved disciplines (formulators, nonclinical scientists, clinicians, and regulators) and geographies (eg, US, EU), the Excipients Safety workshop session was successful in providing specific and key recommendations for defining paths forward...
July 17, 2017: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/28725955/biologics-in-juvenile-idiopathic-arthritis-a-narrative-review
#6
REVIEW
Federica Vanoni, Francesca Minoia, Clara Malattia
In the past years, pediatric rheumatology has seen a revolution in the treatments for rheumatic diseases, particularly juvenile idiopathic arthritis. Even if nonsteroidal anti-inflammatory drugs (NSAID), intra-articular corticosteroids (IAC) injections, and methotrexate remain the mainstay of the treatment for JIA patients, in aggressive disease, these treatments may be not sufficient to reach disease remission and to prevent long-term disability. Comprehension of immunological mechanisms involved in the pathogenesis of the diseases allowed to conceive new drugs targeting specific steps of the immune response...
July 20, 2017: European Journal of Pediatrics
https://www.readbyqxmd.com/read/28724202/population-pharmacokinetics-of-lenalidomide-in-healthy-volunteers-and-patients-with-hematologic-malignancies
#7
Jamie N Connarn, Renfang Hwang, Yue Gao, Maria Palmisano, Nianhang Chen
A population pharmacokinetic (PopPK) model of lenalidomide was developed using data pooled from 13 clinical studies (dose range, 5-400 mg) in participants who were considered to have adequate capability for renal excretion of lenalidomide (creatinine clearance [CrCl] > 50 mL/min). The analysis population included 305 healthy volunteers and 83 patients with multiple myeloma or myelodysplastic syndromes. A 1-compartment model with linear absorption and elimination described well the observed data for both healthy volunteers and patients...
July 19, 2017: Clinical Pharmacology in Drug Development
https://www.readbyqxmd.com/read/28719598/the-predictive-value-of-abcb1-abcg2-cyp3a4-5-and-cyp2d6-polymorphisms-for-risperidone-and-aripiprazole-plasma-concentrations-and-the-occurrence-of-adverse-drug-reactions
#8
C Rafaniello, M Sessa, F F Bernardi, M Pozzi, S Cheli, D Cattaneo, S Baldelli, M Molteni, R Bernardini, F Rossi, E Clementi, C Bravaccio, S Radice, A Capuano
We investigated in ninety Caucasian pediatric patients the impact of the main polymorphisms occurring in CYP3A, CYP2D6, ABCB1 and ABCG2 genes on second-generation antipsychotics plasma concentrations, and their association with the occurrence of adverse drug reactions. Patients with the CA/AA ABCG2 genotype had a statistically significant lower risperidone plasma concentration/dose ratio (Ct/ds) (P-value: 0.007) and an higher estimated marginal probability of developing metabolism and nutrition disorders as compared to the ABCG2 c...
July 18, 2017: Pharmacogenomics Journal
https://www.readbyqxmd.com/read/28719387/pediatric-mitochondrial-diseases-and-the-heart
#9
Gregory M Enns
PURPOSE OF REVIEW: Mitochondrial disorders are an increasingly recognized cause of heart dysfunction, with the primary manifestations being cardiomyopathy and conduction defects. This review focuses on the complex genetics of mitochondrial disease and recently discovered conditions that affect mitochondrial function. RECENT FINDINGS: Next-generation sequencing techniques, especially whole-exome sequencing, have led to the discovery of a number of conditions that cause mitochondrial dysfunction and subsequent cardiac abnormalities...
July 17, 2017: Current Opinion in Pediatrics
https://www.readbyqxmd.com/read/28716726/early-life-risperidone-enhances-locomotor-responses-to-amphetamine-during-adulthood
#10
Bobbie Lee Stubbeman, Clifford J Brown, Justin R Yates, Mark E Bardgett
Antipsychotic drug prescriptions for pediatric populations have increased over the past 20 years, particularly the use of atypical antipsychotic drugs such as risperidone. Most antipsychotic drugs target forebrain dopamine systems, and early-life antipsychotic drug exposure could conceivably reset forebrain neurotransmitter function in a permanent manner that persists into adulthood. This study determined whether chronic risperidone administration during development modified locomotor responses to the dopamine/norepinephrine agonist, D-amphetamine, in adult rats...
July 14, 2017: European Journal of Pharmacology
https://www.readbyqxmd.com/read/28716517/lethal-suicide-attempt-with-a-mixed-drug-intoxication-of-metoprolol-and-propafenone-a-first-pediatric-case-report
#11
Ivana Kacirova, Milan Grundmann, Martin Kolek, Erika Vyskocilova-Hrudikova, Romana Urinovska, Petr Handlos
INTRODUCTION: The β1 adrenergic receptor blocker metoprolol is often prescribed together with the antiarrhythmic drug propafenone. Both are metabolized by cytochrome P450 2D6 and propafenone is also an inhibitor of this enzyme. We present a pediatric case showing metoprolol and propafenone intoxication in combination. CASE: A 14-year-old girl was admitted to a local emergency department after ingestion of metoprolol (probably 1g) and propafenone (probably 1.5-3g) in a suicide attempt...
June 29, 2017: Forensic Science International
https://www.readbyqxmd.com/read/28715810/population-pharmacokinetics-of-diazoxide-in-children-with-hyperinsulinemic-hypoglycemia
#12
Rika Kizu, Kazuko Nishimura, Reiko Sato, Kenjiro Kosaki, Toshiaki Tanaka, Yusuke Tanigawara, Tomonobu Hasegawa
BACKGROUND: Diazoxide is the first-line treatment for pediatric hyperinsulinemic hypoglycemia (HI). This study aimed to elucidate the pharmacokinetics of diazoxide in children with HI. METHODS: We obtained 81 blood samples from 22 children with HI. Measured serum diazoxide concentrations were used for population pharmacokinetic analysis. Patient factors influencing pharmacokinetics were estimated using nonlinear mixed-effects model analysis. Relationships between drug exposure and adverse drug reactions were also investigated...
July 14, 2017: Hormone Research in Pædiatrics
https://www.readbyqxmd.com/read/28715290/trainees-knowledge-attitudes-and-practices-towards-caring-for-the-substance-exposed-mother-infant-dyad
#13
Davida M Schiff, Barry Zuckerman, Elisha M Wachman, Megan Bair-Merritt
BACKGROUND: As rates of substance use disorder during pregnancy rise, pediatric trainees are increasingly caring for infants with neonatal abstinence syndrome (NAS). This study evaluated the knowledge, attitudes, and practices of trainees caring for substance-exposed newborns and their families, comparing differences by level and type of training, and personal experience with addiction or trauma. METHODS: A cross-sectional survey of medical students, pediatric, medicine/pediatrics and family medicine residents in 2015-2106...
July 17, 2017: Substance Abuse
https://www.readbyqxmd.com/read/28709570/systemic-therapies-in-atopic-dermatitis-the-pipeline
#14
Yael Renert-Yuval, Emma Guttman-Yassky
Atopic dermatitis (AD), the most prevalent inflammatory skin disease, is characterized by robust T-cell activation. The disease has several subtypes, all having a common T helper type 2 (Th2)/Th22 polarization, but it also displays differential immune skewing, such as increased Th17/interleukin 23 skewing in the skin of intrinsic, Asian, and early pediatric AD patients. Current systemic treatments for moderate to severe AD are largely unsatisfactory, associated with significant adverse effects and low compliance...
July 2017: Clinics in Dermatology
https://www.readbyqxmd.com/read/28708753/effectiveness-of-preoperative-intranasal-dexmedetomidine-compared-with-oral-midazolam-for-the-prevention-of-emergence-delirium-in-the-pediatric-patient-undergoing-general-anesthesia-a-systematic-review
#15
James FitzSimons, Laura S Bonanno, Stephanie Pierce, Jennifer Badeaux
BACKGROUND: Emergence delirium is defined as a cognitive disturbance during emergence from general anesthesia resulting in hallucinations, delusions and confusion manifested by agitation, restlessness, involuntary physical movement and extreme flailing in bed. Postoperative emergence delirium develops in 12% to 18% of all children undergoing general anesthesia for surgery. This post-anesthetic phenomenon changes cognitive and psychomotor behavior, and puts pediatric patients and health care personnel at risk of injury...
July 2017: JBI Database of Systematic Reviews and Implementation Reports
https://www.readbyqxmd.com/read/28708590/whole-exome-analysis-in-osteosarcoma-to-identify-a-personalized-therapy
#16
Caterina Chiappetta, Massimiliano Mancini, Francesca Lessi, Paolo Aretini, Veronica De Gregorio, Chiara Puggioni, Raffaella Carletti, Vincenzo Petrozza, Prospero Civita, Sara Franceschi, Antonio G Naccarato, Carlo Della Rocca, Chiara M Mazzanti, Claudio Di Cristofano
Osteosarcoma is the most common pediatric primary non-hematopoietic bone tumor. Survival of these young patients is related to the response to chemotherapy and development of metastases. Despite many advances in cancer research, chemotherapy regimens for osteosarcoma are still based on non-selective cytotoxic drugs. It is essential to investigate new specific molecular therapies for osteosarcoma to increase the survival rate of these patients. We performed exomic sequence analyses of 8 diagnostic biopsies of patients with conventional high grade osteosarcoma to advance our understanding of their genetic underpinnings and to correlate the genetic alteration with the clinical and pathological features of each patient to identify a personalized therapy...
July 5, 2017: Oncotarget
https://www.readbyqxmd.com/read/28706488/postnatal-maturation-of-the-glomerular-filtration-rate-in-conventional-growing-piglets-as-potential-juvenile-animal-model-for-preclinical-pharmaceutical-research
#17
Elke Gasthuys, Mathias Devreese, Joske Millecam, Stanislas Sys, Katrien Vanderperren, Joris Delanghe, Johan Vande Walle, Marjolein Heyndrickx, Siska Croubels
Adequate animal models are required to study the preclinical pharmacokinetics (PK), pharmacodynamics (PD) and safety of drugs in the pediatric subpopulation. Over the years, pigs were presented as a potential animal model, since they display a high degree of anatomical and physiological similarities with humans. To assess the suitability of piglets as a preclinical animal model for children, the ontogeny and maturation processes of several organ systems have to be unraveled and compared between both species...
2017: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/28700523/dried-blood-spots-combined-with-ultra-high-performance-liquid-chromatography-mass-spectrometry-for-the-quantification-of-the-antipsychotics-risperidone-aripiprazole-pipamperone-and-their-major-metabolites
#18
Camille Tron, Sanne M Kloosterboer, Bart C H van der Nagel, Rixt A Wijma, Bram Dierckx, Gwen C Dieleman, Teun van Gelder, Birgit C P Koch
BACKGROUND: Risperidone, aripiprazole, and pipamperone are antipsychotic drugs frequently prescribed for the treatment of comorbid behavioral problems in children with autism spectrum disorders. Therapeutic drug monitoring (TDM) could be useful to decrease side effects and to improve patient outcome. Dried blood spot (DBS) sample collection seems to be an attractive technique to develop TDM of these drugs in a pediatric population. The aim of this work was to develop and validate a DBS assay suitable for TDM and home sampling...
August 2017: Therapeutic Drug Monitoring
https://www.readbyqxmd.com/read/28700522/dried-blood-spot-sampling-for-tacrolimus-and-mycophenolic-acid-in-children-analytical-and-clinical-validation
#19
Lisa C Martial, Karin E J Hoogtanders, Michiel F Schreuder, Elisabeth A Cornelissen, Jac van der Heijden, Manuela A Joore, Erik M Van Maarseveen, David M Burger, Sander Croes, Roger J M Brüggemann, Rob E Aarnoutse
BACKGROUND: Tacrolimus and mycophenolic acid (MPA) are the backbone of immunosuppressive therapy after pediatric kidney transplantation. Dosing of these drugs is individualized by therapeutic drug monitoring. Dried blood spot (DBS) sampling may prove beneficial over conventional venous sampling. We aimed to develop and clinically validate a DBS method for tacrolimus and MPA in children. METHODS: A joint DBS liquid chromatography-mass spectrometry assay for tacrolimus and MPA was developed...
August 2017: Therapeutic Drug Monitoring
https://www.readbyqxmd.com/read/28697215/update-on-the-systemic-risks-of-superpotent-topical-steroids
#20
Mio Nakamura, Michael Abrouk, Henry Zhu, Benjamin Farahnik, John Koo, Tina Bhutani
<p>INTRODUCTION: The potential for systemic effects due to percutaneous absorption of superpotent topical steroids has been a longstanding concern. The Food and Drug Administration currently recommends limiting the use of superpotent topical steroids to 50g per week for 2 or 4 consecutive weeks depending on the formulation, which is mostly based on the exact duration with which phase 3 clinical trials were allowed to be conducted per the FDA. This article reviews all published clinical incidence of adrenal adverse effects in the medical literature, specifically Cushing's syndrome (CS) and pathologic adrenal suppression (PAAS), to try to ascertain a more realistic limit for the safe use of superpotent topical steroids as it pertains to its potential systemic effects...
July 1, 2017: Journal of Drugs in Dermatology: JDD
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