Advanced Therapies in Pediatric Endocrinology and Diabetology

Description

Dealing with current hot topics, this book aims to provide an update on advances in the treatment of endocrine disorders. The chapters cover innovative therapeutic approaches to type 1 diabetes, such as artificial pancreas and islet transplantation. Additionally, novel pharmacological interventions for patients with obesity and the metabolic syndrome, as well as with adrenal and gonadal disorders are provided. Furthermore, recent developments in growth-promoting therapies, such as the C-type natriuretic peptide analog to treat achondroplasia, as well as antioxidant and gene replacement therapies in patients with adrenoleukodystrophy, are described. Worldwide renowned experts share their experience and cutting edge research in Advanced Therapies in Pediatric Endocrinology and Diabetology, which makes it a useful and interesting resource for every pediatric endocrinologist, not only those active in clinical research, but also those involved in the management of children with endocrine diseases in daily practice.

Informations

• Publié par: S. Karger AG
• Date de parution: 10 décembre 2015
• Nombre de lectures: 0
• EAN13: 9783318056372
• Langue: English
• Poids de l'ouvrage: 1 Mo

Extrait

Advanced Therapies in Pediatric Endocrinology and Diabetology
With an unrestricted educational grant from
Endocrine Development
Vol. 30
Series Editor
P.-E. Mullis Bern
Workshop, Rome, October 27-28, 2014
Advanced Therapies in Pediatric Endocrinology and Diabetology
Volume Editors
Marco Cappa Rome
Stefano Cianfarani Rome
Lucia Ghizzoni Turin
Sandro Loche Cagliari
Mohamad Maghnie Genoa
26 figures, 6 in color and 10 tables, 2016
Endocrine Development Founded 1999 by Martin O. Savage, London
_______________________ Marco Cappa Endocrinology and Diabetology in Diabetes Units D.P.U.O. Bambino Gesù Children's Hospital IRCCS Rome, Italy
_______________________ Stefano Cianfarani Molecular Endocrinology Unit D.P.U.O. Bambino Gesù Children's Hospital IRCCS ‘Tor Vergata’ University Rome, Italy
_______________________ Lucia Ghizzoni Division of Endocrinology, Diabetology and Metabolism Department of Internal Medicine University of Turin Turin, Italy
_______________________ Sandro Loche Regional Hospital for Microcytaemia Cagliari, Italy

_______________________ Mohamad Maghnie Department of Pediatrics IRCCS G. Gaslini University of Genoa Genoa, Italy
Library of Congress Cataloging-in-Publication Data
Advanced therapies in pediatric endocrinology and diabetology: workshop, Rome, October 27-28, 2014 / volume editors Marco Cappa, Stefano Cianfarani, Lucia Ghizzoni, Sandro Loche, Mohamad Maghnie.
p. ; cm. -- (Endocrine development ; vol. 30)
Includes bibliographical references and index.
ISBN 978-3-318-05636-5 (hard cover: alk. paper) -- ISBN 978-3-318-05637-2 (electronic version)
I. Cappa, Marco, editor. II. Cianfarani, Stefano, editor. III. Ghizzoni, Lucia, editor. IV. Loche, Sandro, editor. V. Maghnie, Mohamad, editor. VI. Series: Endocrine development ; v. 30.
[DNLM: 1. Endocrine System Diseases--therapy--Congresses. 2. Child--Congresses. W1 EN3635 v.30 2016 / WS 330]
RJ418
618.92'4--dc23
2015035218
Bibliographic Indices. This publication is listed in bibliographic services, including Current Contents ® and PubMed/MEDLINE.
Disclaimer. The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publisher and the editor(s). The appearance of advertisements in the book is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements.
Drug Dosage. The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any change in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug.
All rights reserved. No part of this publication may be translated into other languages, reproduced or utilized in any form or by any means electronic or mechanical, including photocopying, recording, microcopying, or by any information storage and retrieval system, without permission in writing from the publisher.
© Copyright 2016 by S. Karger AG, P.O. Box, CH-4009 Basel (Switzerland)
www.karger.com
Printed in Germany on acid-free and non-aging paper (ISO 9706) by Kraft Druck, Ettlingen
ISSN 1421-7082
e-ISSN 1662-2979
ISBN 978-3-318-05636-5
e-ISBN 978-3-318-05637-2
Contents
Preface
Cappa, M.; Cianfarani, S. (Rome); Ghizzoni, L. (Turin); Loche, S. (Cagliari); Maghnie, M. (Genoa)
Toward Automation of Insulin Delivery - Management Solutions for Type 1 Diabetes
Nimri, R. (Petah Tikva); Phillip, M. (Petah Tikva/Tel Aviv)
Islet Transplantation in Pediatric Patients: Current Indications and Future Perspectives
Bertuzzi, F.; Antonioli, B.; Tosca, M.C.; Galuzzi, M.; Bonomo, M.; Marazzi, M.; Colussi, G. (Milan)
Glucagon-Like Peptide-1 Receptor Agonist Treatment for Pediatric Obesity
Kelly, A.S. (Minneapolis, Minn.)
Docosahexaenoic Acid and Its Role in G-Protein-Coupled Receptor 120 Activation in Children Affected by Nonalcoholic Fatty Liver Disease
Della Corte, C.; Mosca, A. (Rome); Ionata, A. (Isernia); Nobili, V. (Rome)
Noninvasive Prenatal Diagnosis of Congenital Adrenal Hyperplasia
Khattab, A.; Yuen, T.; Sun, L.; Yau, M.; Barhan, A.; Zaidi, M. (New York, N.Y.); Lo, Y.M.D. (Hong Kong, SAR); New, M.I. (New York, N.Y.)
Recent Advances in Hydrocortisone Replacement Treatment
Mallappa, A. (Bethesda, Md.); Debono, M. (Sheffield)
Experience with the Histrelin Implant in Pediatric Patients
Eugster, E.A. (Indianapolis, Ind.)
Different Medications for Hypogonadotropic Hypogonadism
Rastrelli, G.; Vignozzi, L.; Maggi, M. (Florence)
Long-Acting Growth Hormone: An Update
Saenger, P.H.; Mejia-Corletto, J. (New York, N.Y.)
C-Type Natriuretic Peptide Analog as Therapy for Achondroplasia
Legeai-Mallet, L. (Paris)
Therapeutic Neuroendocrine Agonist and Antagonist Analogs of Hypothalamic Neuropeptides as Modulators of the Hypothalamic-Pituitary-Gonadal Axis
Newton, C.L.; Anderson, R.C.; Millar, R.P. (Pretoria/Cape Town)
Aromatase Inhibitors in the Treatment of Short Stature
Hero, M. (Helsinki)
Gene Therapy for Rare Central Nervous System Diseases Comes to Age
Aubourg, P. (Le Kremlin-Bicêtre)
Novel Therapeutic Targets and Drug Candidates for Modifying Disease Progression in Adrenoleukodystrophy
Pujol, A. (Barcelona)
Author Index
Subject Index
Preface
This volume of Endocrine Development is the 6th volume of a series started in 2007 with the proceedings of an outstanding symposium held in Genoa on congenital endocrinopathies, which was followed by documentations of workshops every other year in different Italian cities (Rome, Cagliari and Turin). The aim of the workshops is to provide an update on advanced therapies of endocrine disorders.
The program of this workshop covered several topics organized in thematic sessions and plenary lectures. Outstanding experts discussed a number of issues, including new modalities for treating type 1 diabetes like the artificial pancreas and islet transplantation in pediatric patients, novel therapeutic approaches for obesity and the metabolic syndrome, as well as adrenal and gonadal disorders.
Recent advances in growth-promoting therapies, such as the C-type natriuretic peptide analogue to treat achondroplasia as well as oxidative stress blockers and gene therapy for patients with adrenoleukodystrophy, have been discussed. The chapters presented are the results of fruitful exchange of scientific information and discussions of experts in the field and we are really grateful to all the participating speakers and authors for sharing their experience and knowledge with us.
We are sure that the quality of this book will be as excellent as the workshop.
Marco Cappa, Rome Stefano Cianfarani, Rome Lucia Ghizzoni, Turin Sandro Loche, Cagliari Mohamad Maghnie, Genoa
Cappa M, Cianfarani S, Ghizzoni L, Loche S, Maghnie M (eds): Advanced Therapies in Pediatric Endocrinology and Diabetology. Endocr Dev. Basel, Karger, 2016, vol 30, pp 1-13 (DOI: 10.1159/000439321)
______________________
Toward Automation of Insulin Delivery - Management Solutions for Type 1 Diabetes
Revital Nimri a Moshe Phillip a , b
a The Jesse Z and Sara Lea Shafer Institute for Endocrinology and Diabetes, National Center for Childhood Diabetes, Schneider Children's Medical Center of Israel, Petah Tikva, and b Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
______________________
Abstract
In the past decade, the field of type 1 diabetes was characterized by the efforts to integrate technology into the daily management of diabetes. Automated insulin delivery systems have emerged followed by the improvements in technology of pumps and sensors and automated close-loop systems that were developed around the world for overnight as well as for day and night use. Initially, these closed-loop systems were tested clinically in research centers, then at diabetes camps or hotels, and recently at patients’ homes. The systems were tested in a wide range of populations of patients with type 1 diabetes: children, adolescents, adults, newly diagnosed, well and suboptimally controlled patients, the critically ill and pregnant women. The extensive clinical evaluation found these close-loop systems to be safe and efficient in controlling blood glucose levels. Now is the time to take these systems from research to industry and to get a regulatory approval of convenient devices for the use at home. Automated insulin delivery systems have the potential to change the way diabetes is treated and managed for the benefit of patients. This chapter summarizes the recent advances in this field.
© 2016 S. Karger AG, Basel
A century passed since the discovery of insulin in Toronto and two decades since the results of the Diabetes Control and Complications Trial were published and the world acknowledgment that good glycemic control is mandatory to reduce or postpone diabetes-rel