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Turchetti G.,SantAnna School of Advanced Studies | Scalone L.,Fondazione Charta | Scalone L.,University of Milan Bicocca | Della Casa Alberighi O.,IRCCS Giannina Gaslini Institute | And 5 more authors.
Clinical and Experimental Rheumatology | Year: 2012

Pharmacoeconomic analysis is aimed at supporting choices between alternatives available for the efficient management of specific conditions. Aim of the paper is to provide an overview of the main features of pharmacoeconomic evaluations, with the objective of providing the reader with the basic tools necessary to read and interpret or to design and conduct a pharmacoeconomic analysis in RA and in other rheumatic diseases. The paragraphs will cover in detail the definition of health economic evaluation and pharmacoeconomics, the alternatives to be compared, the perspective of the analysis, costs and effects (presenting in detail direct costs and effects, indirect costs and effects, intangible costs and effects and source of data), and pharmacoeconomic techniques. Pharmacoeconomic analyses have to be conducted accurately to provide valuable information to guide the choice of options representing the best value for money without compromising the quality of care delivered. For this reason, as these analyses generally present some limitations, a very close and strong relationship between pharmacoeconomists and clinicians is crucial both in the design of pharmacoeconomic studies and in the interpretation of their results, and also in the development of more satisfactory methods and indicators. © Copyright Clinical and Experimental Rheumatology 2012.

Barrett J.S.,Childrens Hospital of Philadelphia | Della Casa Alberighi O.,IRCCS Giannina Gaslini Institute | Laer S.,Heinrich Heine University Dusseldorf | Meibohm B.,University of Tennessee Health Science Center
Clinical Pharmacology and Therapeutics | Year: 2012

This review summarizes the present status of physiologically based pharmacokinetic (PBPK) modeling and simulation (M&S) and its application in support of pediatric drug research. We address the reasons that PBPK is suited to the current needs of pediatric drug development and pharmacotherapy in light of the evolution in pediatric PBPK methodologies and approaches, which were originally developed for the purpose of toxicologic evaluation. Also discussed is the current degree of confidence in using PBPK to support pediatric drug development and registration and the key factors essential for robust results and broader adoption of pediatric PBPK M&S.

Paglialonga F.,Pediatric Nephrology and Dialysis Unit | Consolo S.,Pediatric Nephrology and Dialysis Unit | Pecoraro C.,Nephrology and Dialysis Unit | Vidal E.,University of Padua | And 6 more authors.
Pediatric Nephrology | Year: 2016

Background: Chronic haemodialysis (HD) in small children has not been adequately investigated. Methods: This was a retrospective investigation of the use of chronic HD in 21 children aged <2 years (n = 12 aged <1 year) who were registered in the Italian Pediatric Dialysis Registry. Data collected over a period of >10 years were analysed. Results: The median age of the 21 children at start of HD was 11.4 [interquartile range (IQR) 6.2–14.6] months, and HD consisted mainly of haemodiafiltration for 3–4 h in ≥4 sessions/week. A total of 51 central venous catheters were placed, and the median survival of tunnelled and temporary lines was 349 and 31 days, respectively (p < 0.001). Eight children (38 %) showed evidence of central vein thrombosis. Although 19 % of patients received growth hormone and 63.6 % received enteral feeding, the weight and height of these patients remained suboptimal. During the HD period the haemoglobin level increased in all patients, but not to normal levels (from 8.5 to 9.6 g/dl) despite erythropoietin administration (503–600 U/kg/week). The hospitalisation rate was 1.94/patient-year. Seventeen patients underwent renal transplantation at a median age of 3.0 years. Four patients, all affected by severe comorbidities, died during follow-up (in 2 cases due to absence of a vascular access). The 5- and 10-year cumulative survival was 82.4 and 68.7 %, respectively. Conclusions: Extracorporeal dialysis is feasible in children aged <2 years, but comorbidities, vascular access, growth and anaemia remain major concerns. © 2015, IPNA.

Montaldo E.,IRCCS Giannina Gaslini Institute | Vacca P.,University of Genoa | Vitale C.,University of Genoa | Moretta F.,University of Verona | And 4 more authors.
Immunology Letters | Year: 2016

The interest in innate lymphoid cells (ILC) has rapidly grown during the last decade. ILC include distinct cell types that are collectively involved in host protection against pathogens and tumor cells and in the regulation of tissue homeostasis. Studies in mice enabled a broad characterization of ILC function and of their developmental requirements. In humans all mature ILC subsets have been characterized and their role in the pathogenesis of certain disease is emerging. Nonetheless, still limited information is available on human ILC development. Indeed, only the cell precursors committed toward NK cells or ILC3 have been described. Here, we review the most recent finding on human mature ILC, discussing their tissue localization and function. Moreover, we summarize the available data regarding human ILC development. © 2016 European Federation of Immunological Societies.

Srinivasaraghavan R.,Jawaharlal Institute of Postgraduate Medical Education & Research | Krishnamurthy S.,Jawaharlal Institute of Postgraduate Medical Education & Research | Chandar R.,Jawaharlal Institute of Postgraduate Medical Education & Research | Cassandrini D.,IRCCS Fondazione Stella Maris | And 3 more authors.
Pediatric Dermatology | Year: 2014

Chanarin-Dorfman syndrome (CDS) is a rare nonlysosomal neutral lipid storage disorder characterized by congenital ichthyosis, lipid vacuoles in leukocytes (Jordan's anomaly), and hepatomegaly. The authors herein report an 18-month-old boy with ichthyosis and hepatomegaly diagnosed with CDS and confirmed to have a novel c.506-3C>G mutation in the ABHD5/CGI-58 gene. Our case also illustrates that retinoids such as acitretin could be useful in the treatment of skin manifestations in CDS even in the presence of liver derangement. © 2013 Wiley Periodicals, Inc.

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