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https://www.readbyqxmd.com/read/29785886/treatment-of-homozygous-familial-hypercholesterolaemia-in-paediatric-patients-a-monocentric-experience
#1
Paola S Buonuomo, Marina Macchiaiolo, Giovanna Leone, Paola Valente, Gerarda Mastrogiorgio, Maria Gnazzo, Ippolita Rana, Michaela V Gonfiantini, Maria G Gagliardi, Francesca Romano, Andrea Bartuli
Background Homozygous familial hypercholesterolaemia is a rare life-threatening disease characterized by markedly elevated low-density lipoprotein cholesterol (LDL-C) concentrations and accelerated atherosclerosis. The presence of double gene defects in the LDL-Receptor, either the same defect (homozygous) or two different LDL-raising mutations (compound heterozygotes) or other variants, identify the homozygous phenotype (HopFH). Apheresis is a procedure in which plasma is separated from red blood cells before the physical removal of LDL-C or the LDL-C is directly removed from whole blood...
January 1, 2018: European Journal of Preventive Cardiology
https://www.readbyqxmd.com/read/29785508/lacosamide-a-review-in-focal-onset-seizures-in-patients-with-epilepsy
#2
Sheridan M Hoy
Lacosamide (Vimpat® ) is a functionalized amino acid (available orally and intravenously) approved in the EU and the USA for use as monotherapy and adjunctive therapy for the treatment of focal-onset seizures in adults, adolescents and children aged ≥ 4 years with epilepsy. In adults and adolescents (aged ≥ 16 years), oral lacosamide as adjunctive therapy to other antiepileptic drugs was generally effective in reducing seizure frequency during short-term (up to 18 weeks) treatment, with efficacy sustained over the longer-term (up to 8 years)...
May 21, 2018: CNS Drugs
https://www.readbyqxmd.com/read/29781554/children-who-face-development-risks-due-to-maternal-addiction-during-pregnancy-require-extra-medical-and-psychosocial-resources
#3
Jenny Rangmar, Maria Lilja, Marie Köhler, Antonia Reuter
AIM: This study examined medical and psychosocial risk factors in children born to women with addiction problems during pregnancy and the children's needs for extra medical and psychosocial resources. METHODS: Swedish midwives routinely screen pregnant women for drugs and alcohol and refer women with addictions to the Maternity and Child Healthcare Resource Team. We investigated the medical records of 127 children (51% girls) whose mothers were referred to the Resource Team from 2009-2015...
May 21, 2018: Acta Paediatrica
https://www.readbyqxmd.com/read/29776346/a-systematic-review-of-the-risk-factors-for-clinical-response-to-opioids-for-all-age-patients-with-cancer-related-pain-and-presentation-of-the-paediatric-stop-pain-study
#4
Ersilia Lucenteforte, Laura Vagnoli, Alessandra Pugi, Giada Crescioli, Niccolò Lombardi, Roberto Bonaiuti, Maurizio Aricò, Sabrina Giglio, Andrea Messeri, Alessandro Mugelli, Alfredo Vannacci, Valentina Maggini
BACKGROUND: Inter-patient variability in response to opioids is well known but a comprehensive definition of its pathophysiological mechanism is still lacking and, more importantly, no studies have focused on children. The STOP Pain project aimed to evaluate the risk factors that contribute to clinical response and adverse drug reactions to opioids by means of a systematic review and a clinical investigation on paediatric oncological patients. METHODS: We conducted a systematic literature search in EMBASE and PubMed up to the 24th of November 2016 following Cochrane Handbook and PRISMA guidelines...
May 18, 2018: BMC Cancer
https://www.readbyqxmd.com/read/29767845/final-results-from-a-defibrotide-treatment-ind-study-for-patients-with-hepatic-veno-occlusive-disease-sinusoidal-obstruction-syndrome
#5
Nancy A Kernan, Stephan Grupp, Angela R Smith, Sally Arai, Brandon Triplett, Joseph H Antin, Leslie Lehmann, Tsiporah Shore, Vincent T Ho, Nancy Bunin, Massimo Iacobelli, Wei Liang, Robin Hume, William Tappe, Robert Soiffer, Paul Richardson
Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic stem cell transplant (HSCT) conditioning and chemotherapy. Defibrotide is approved for treatment of hepatic VOD/SOS with pulmonary or renal dysfunction [i.e., multi-organ dysfunction (MOD)] after HSCT in the United States and severe VOD/SOS after HSCT in patients aged older than 1 month in the European Union. Defibrotide was available as an investigational drug by an expanded-access treatment programme (T-IND; NCT00628498)...
May 16, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29754420/the-predictive-value-of-the-heart-rate-variability-derived-analgesia-nociception-index-in-children-anaesthetised-with-sevoflurane-an-observational-pilot-study
#6
Frank Weber, Noortje J E Geerts, Hilde G Roeleveld, Annejet T Warmenhoven, Chantal A Liebrand
BACKGROUND: The heart rate variability (HRV) derived Analgesia Nociception Index (ANI™ ) is a continuous non-invasive tool to assess the nociception/anti-nociception balance in unconscious patients. It has been shown to be superior to hemodynamic variables in detecting insufficient anti-nociception in children, while little is known about its predictive value. METHODS: The primary objective of this prospective observational pilot study in paediatric surgical patients under sevoflurane anaesthesia, was to compare the predictive value of the ANI and heart rate to help decide to give additional opioids...
May 13, 2018: European Journal of Pain: EJP
https://www.readbyqxmd.com/read/29753906/continuous-inkjet-printing-of-enalapril-maleate-onto-orodispersible-film-formulations
#7
Yasmin Thabet, Dominique Lunter, Joerg Breitkreutz
Piezoelectric inkjet printing onto orodispersible films (ODFs) was proven to be a successful technique applying flexible doses of active pharmaceutical ingredients (APIs) onto edible substrates. The reported API printing and ODF production was conducted in a non-continuous production approach. Within this study, drug-free and hydrochlorothiazide (HCT) containing ODFs should be imprinted in-line with enalapril maleate (EM) ink during continuous ODF production. Macrogol inks based on various solvents and solvent-water mixtures were developed providing dynamic viscosities from 7 to 17 mPa∗s...
May 10, 2018: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/29752817/national-treatment-guidelines-decreased-the-use-of-racemic-adrenaline-for-bronchiolitis-in-four-finnish-university-hospitals
#8
Sauli Palmu, Minna Mecklin, Paula Heikkilä, Katri Backman, Ville Peltola, Marjo Renko, Matti Korppi
AIM: Inhaled racemic adrenaline was used for bronchiolitis in many hospitals in Finland prior to new national current care guidelines for bronchiolitis in 2014, which limited its recommendations to on-demand rescue therapy. We studied the drug's use before and after the new guidelines to gauge changes in prescribing habits. METHODS: This 2012-2016 study analysed how many 0.5 ml doses of racemic adrenaline were used for children by emergency rooms, paediatric wards and paediatric intensive care units at four university hospitals and estimated drug and staff costs...
May 12, 2018: Acta Paediatrica
https://www.readbyqxmd.com/read/29751121/evaluation-of-dried-blood-spot-sampling-for-pharmacokinetic-research-and-therapeutic-drug-monitoring-of-anti-tuberculosis-drugs-in-children
#9
Lisa C Martial, Jordy Kerkhoff, Nilza Martinez, Mabel Rodríguez, Rosarito Coronel, Gladys Molinas, Myriam Roman, Roscio Gomez, Sarita Aguirre, Erwin Jongedijk, Justine Huisman, Daan J Touw, Domingo Pérez, Gilberto Chaparro, Felipe Gonzalez, Rob E Aarnoutse, Jan-Willem Alffenaar, Cecile Magis-Escurra
BACKGROUND: Dried blood spot sampling (DBS) for pharmacokinetic (PK) studies and Therapeutic Drug Monitoring has unique advantages over venous sampling. We aimed to evaluate a DBS method for first-line anti-TB drugs in children, implemented DBS to assess PK parameters. METHODS: Paraguayan children were treated according to the revised paediatric WHO dosing scheme. A PK curve was performed both with DBS and conventional venous sampling for rifampicin, pyrazinamide and ethambutol...
May 8, 2018: International Journal of Antimicrobial Agents
https://www.readbyqxmd.com/read/29750764/linezolid-containing-treatment-regimens-for-tuberculosis-in-children
#10
Luis M Prieto, Begoña Santiago, Teresa Del Rosal, Begoña Carazo, Ana B Jiménez, Beatriz Pérez-Gorricho, Felipe Rubio, Alfredo Tagarro, Daniel Blázquez, David Moreno-Pérez, María J Mellado, Fernando Baquero-Artigao
BACKGROUND: In recent years there is an increasing interest in the use of linezolid for the treatment of tuberculosis (TB). METHODS: Patients under 18 years of age who received linezolid within the Spanish Pediatric TB Network (pTBred) from 2001 to 2016 were retrospectively included. Treatment characteristics, adverse events (AEs) and outcomes were analyzed. RESULTS: Fifteen children were included (53% male) with a median age of 3.6 years (IQR: 1...
May 10, 2018: Pediatric Infectious Disease Journal
https://www.readbyqxmd.com/read/29749488/c%C3%A2-myc-inhibitor-10058%C3%A2-f4-increases-the-efficacy-of-dexamethasone-on-acute-lymphoblastic-leukaemia-cells
#11
Mei Lv, Yi Wang, Wenmiao Wu, Shujun Yang, Huiling Zhu, Bei Hu, Ying Chen, Cong Shi, Yi Zhang, Qitian Mu, Guifang Ouyang
The long‑term survival rate in paediatric acute lymphoblastic leukaemia (ALL) exceeds 80%; however, the outcome of adult ALL remains to be poor. Glucocorticoids (GCs) are the preferred drugs in the traditional treatment of ALL patients. In the anti‑leukaemia molecular mechanisms of GCs, c‑Myc inhibition serves a critical role. In the present study, a c‑Myc inhibitor that increased the sensitivity to GCs in NALM6 cells of the B‑cell‑ALL cell line and CEM cells of the T‑cell‑ALL cell line was investigated...
April 27, 2018: Molecular Medicine Reports
https://www.readbyqxmd.com/read/29743330/protocol-for-a-randomised-trial-of-higher-versus-lower-intensity-patient-provider-communication-interventions-to-reduce-antibiotic-misuse-in-two-paediatric-ambulatory-clinics-in-the-usa
#12
Kathy Goggin, Andrea Bradley-Ewing, Angela L Myers, Brian R Lee, Emily A Hurley, Kirsten B Delay, Sarah Schlachter, Areli Ramphal, Kimberly Pina, David Yu, Kirsten Weltmer, Sebastian Linnemayr, Christopher C Butler, Jason G Newland
INTRODUCTION: Children with acute respiratory tract infections (ARTIs) are prescribed up to 11.4 million unnecessary antibiotic prescriptions annually. Inadequate parent-provider communication is a chief contributor, yet efforts to reduce overprescribing have only indirectly targeted communication or been impractical. This paper describes our multisite, parallel group, cluster randomised trial comparing two feasible interventions for enhancing parent-provider communication on the rate of inappropriate antibiotic prescribing (primary outcome) and revisits, adverse drug reactions and parent-rated quality of shared decision-making, parent-provider communication and visit satisfaction (secondary outcomes)...
May 9, 2018: BMJ Open
https://www.readbyqxmd.com/read/29742880/diffuse-intrinsic-pontine-glioma-clinical-features-molecular-genetics-and-novel-targeted-therapeutics
#13
REVIEW
Ryan K Mathew, James T Rutka
Diffuse intrinsic pontine glioma (DIPG) is a deadly paediatric brain cancer. Transient response to radiation, ineffective chemotherapeutic agents and aggressive biology result in rapid progression of symptoms and a dismal prognosis. Increased availability of tumour tissue has enabled the identification of histone gene aberrations, genetic driver mutations and methylation changes, which have resulted in molecular and phenotypic subgrouping. However, many of the underlying mechanisms of DIPG oncogenesis remain unexplained...
May 2018: Journal of Korean Neurosurgical Society
https://www.readbyqxmd.com/read/29739700/catalysing-the-development-and-introduction-of-paediatric-drug-formulations-for-children-living-with-hiv-a-new-global-collaborative-framework-for-action
#14
Martina Penazzato, Melynda Watkins, Sébastien Morin, Linda Lewis, Fernando Pascual, Marissa Vicari, Janice Lee, Sally Hargreaves, Meg Doherty, George K Siberry
Progress in the development and introduction of paediatric formulations for key infectious diseases is poor in low-income and middle-income countries (LMICs). Although major steps have been made in the scale-up of antiretroviral medicines in LMICs, the development and deployment of formulations for infants and children is suboptimal. Of the children living with HIV globally (most in Africa), only 43% are receiving antiretroviral therapy (ART), many with suboptimal formulations. These shortfalls pose a series of challenges to meeting global treatment targets of 1·6 million children (aged 0-14 years) on ART by the end of 2018 (95% coverage) and to ensuring that 95% of those on ART are virologically suppressed...
May 2018: Lancet HIV
https://www.readbyqxmd.com/read/29738796/how-do-hypothalamic-nonapeptides-shape-youth-s-sociality-a-systematic-review-on-oxytocin-vasopressin-and-human-socio-emotional-development
#15
REVIEW
Nuno Torres, Daniel Martins, António J Santos, Diana Prata, Maria Veríssimo
The hypothalamic nonapeptides oxytocin and vasopressin are important modulators of socio-affective behaviours in a wide variety of animal species, including humans. Nevertheless, there is little research addressing their possible roles on socio-affective dimensions of human behaviour across development, during which considerable behavioural and physiological change occurs. Questions still remain about the extent to which findings from adults may directly apply to earlier phases of human development. In this article, we systematically summarize and discuss all existing studies investigating the developmental association of endogenous levels of hypothalamic neuropeptides oxytocin and vasopressin with human social behaviour or on its disruption in paediatric populations...
May 5, 2018: Neuroscience and Biobehavioral Reviews
https://www.readbyqxmd.com/read/29728806/-new-antirheumatics-advances-for-patients-and-society
#16
REVIEW
S Throm, R Hömke
The current repertoire of conventional, biological and targeted synthetic disease-modifying antirheumatic drugs (DMARDs) presents wide-ranging options to help patients with inflammatory rheumatic diseases. These therapeutic possibilities have improved quality of life for many patients and also had a positive impact at the societal level. Among these positive effects are a reduction in short-term inability to work and early retirement; for paediatric patients they represent an opportunity for an almost normal school life including physical education...
May 2018: Zeitschrift Für Rheumatologie
https://www.readbyqxmd.com/read/29727612/electrocorticographic-telemetric-recording-in-unrestrained-mouse-pups
#17
Nicole Chemaly, Astrid Nehlig, Catherine Chiron, Rima Nabbout
BACKGROUND: Early onset epileptic encephalopathies are rare paediatric diseases, with seizures resistant to drugs and impacting development of cognitive and motor functions. Many of them show monogenic aetiology and engineered animal models are crucial to understand the underlying mechanisms and propose treatment trials. These models have mostly been explored in vitro or in vivo under anaesthesia. This may affect the occurrence of epileptic activities and their clinical expression. These study conditions perturb social skills and are limited in time...
May 1, 2018: Journal of Neuroscience Methods
https://www.readbyqxmd.com/read/29723913/observational-pre-post-study-showed-that-a-quality-improvement-project-reduced-paediatric-antibiotic-prescribing-rates-in-primary-care
#18
Simona Di Mario, Carlo Gagliotti, Rossella Buttazzi, Luca Cisbani, Chiara Di Girolamo, Antonio Brambilla, Maria Luisa Moro
AIM: This study assessed the effectiveness of a quality improvement project that aimed to promote more considered antibiotic prescribing in paediatric primary care. Method This was an observational pre-post study that used patient-level prescribing data from the Emilia-Romagna region of Italy to monitor indicators from 2005 to 2016. Multilevel interventions and activities started in 2007 and these included developing guidelines and updates, disseminating evidence, audits and feedback, public information campaigns, engaging health managers and performance incentives...
May 3, 2018: Acta Paediatrica
https://www.readbyqxmd.com/read/29718748/an-update-on-the-toxicological-considerations-for-protease-inhibitors-used-for-hepatitis-c-infection
#19
Lung-Yi Mak, Wai-Kay Seto, Ching-Lung Lai, Man-Fung Yuen
Hepatitis C virus protease inhibitors (PIs) are important components of many direct acting antiviral regimens. Many clinical trials and real-world studies have described the safety data for individual PIs. We aimed to review the safety of both the first and second generation PIs in patients with chronic hepatitis C (CHC). Areas covered: The unique pharmacokinetic properties of PIs partly explain their toxicities. Second generation PIs, when used without interferon and ribavirin, are well-tolerated. Use of PIs in renal impaired patients or those on dialysis appears to be safe...
May 2, 2018: Expert Opinion on Drug Metabolism & Toxicology
https://www.readbyqxmd.com/read/29718336/a-clinical-decision-support-system-algorithm-for-intravenous-to-oral-antibiotic-switch-therapy-validity-clinical-relevance-and-usefulness-in-a-three-step-evaluation-study
#20
H Akhloufi, M Hulscher, C P van der Hoeven, J M Prins, H van der Sijs, D C Melles, A Verbon
Objectives: To evaluate a clinical decision support system (CDSS) based on consensus-based intravenous to oral switch criteria, which identifies intravenous to oral switch candidates. Methods: A three-step evaluation study of a stand-alone CDSS with electronic health record interoperability was performed at the Erasmus University Medical Centre in the Netherlands. During the first step, we performed a technical validation. During the second step, we determined the sensitivity, specificity, negative predictive value and positive predictive value in a retrospective cohort of all hospitalized adult patients starting at least one therapeutic antibacterial drug between 1 and 16 May 2013...
April 26, 2018: Journal of Antimicrobial Chemotherapy
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