UvA-DARE (Digital Academic Repository) Therapeutic targets in sickle cell disease Sins, J.W.R. Link to publication
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1 UvA-DARE (Digital Academic Repository) Therapeutic targets in sickle cell disease Sins, J.W.R. Link to publication Citation for published version (APA): Sins, J. W. R. (2017). Therapeutic targets in sickle cell disease General rights It is not permitted to download or to forward/distribute the text or part of it without the consent of the author(s) and/or copyright holder(s), other than for strictly personal, individual use, unless the work is under an open content license (like Creative Commons). Disclaimer/Complaints regulations If you believe that digital publication of certain material infringes any of your rights or (privacy) interests, please let the Library know, stating your reasons. In case of a legitimate complaint, the Library will make the material inaccessible and/or remove it from the website. Please Ask the Library: or a letter to: Library of the University of Amsterdam, Secretariat, Singel 425, 1012 WP Amsterdam, The Netherlands. You will be contacted as soon as possible. UvA-DARE is a service provided by the library of the University of Amsterdam ( Download date: 17 Jan 2019
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3 Dedicated to all patients with sickle cell disease
4 Therapeutic Targets in Sickle Cell Disease Author: Joep Sins ISBN/EAN: Cover design: Enzo Pérès-Labourdette Lay-out: Karen van Luttervelt Printed by: Optima Grafische Communicatie, Rotterdam, The Netherlands The research in this thesis was financially supported by the Netherlands Organisation for Health Research and Development (ZonMw), the National Institutes of Health (NIH, US), the Academic Medical Center, JANIVO Stichting, Egbers Stichting, Fonds NutsOhra, the Belgian Hematology Society, Baxalta US Inc. (now part of Shire), Stichting Maarten Kappelle, Christine Bader Stichting Irene Kinderziekenhuis, Fulbright Center Nederland, KNAW Ter Meulen Fonds en Stichting Atheros. Financial support for printing this thesis was kindly provided by the Academic Medical Center, Zambon Nederland B.V., CSL Behring B.V., Bayer B.V., Nordic Pharma B.V., Baxalta Netherlands B.V., Celgene B.V. and Pfizer B.V. All rights reserved. No part of this thesis may be reproduced or transmitted in any form or by any means without the written permission of the author. Copyright 2017, Joep Sins, Amsterdam, The Netherlands
5 Therapeutic Targets in Sickle Cell Disease ACADEMISCH PROEFSCHRIFT ter verkrijging van de graad van doctor aan de Universiteit van Amsterdam op gezag van de Rector Magnificus prof. dr. ir. K.I.J. Maex ten overstaan van een door het College voor Promoties ingestelde commissie, in het openbaar te verdedigen in de Agnietenkapel op vrijdag 20 oktober 2017, te uur door Joep Willem Rudolph Sins geboren te Castricum
6 PROMOTIE COMMISSIE Promotores Prof. dr. C.J. Fijnvandraat AMC - Universiteit van Amsterdam Prof. dr. M.H.J. van Oers AMC - Universiteit van Amsterdam Copromotores Dr. B.J. Biemond AMC - Universiteit van Amsterdam Prof. dr. T.W. Kuijpers AMC - Universiteit van Amsterdam Overige leden Prof. dr. M.J. Kersten AMC - Universiteit van Amsterdam Prof. dr. J.B. van Goudoever AMC - Universiteit van Amsterdam Prof. dr. R.P.J. Oude-Elferink AMC - Universiteit van Amsterdam Prof. dr. J.J. Zwaginga LUMC - Universiteit Leiden Dr. M. Peters AMC - Universiteit van Amsterdam Dr. M.H. Cnossen EMC - Erasmus Universiteit Rotterdam Faculteit der Geneeskunde
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9 The Creator looked down on Anansi and his family, and said: Anansi, thank you for finding my ball of light. For your services, I will take the ball of light and place it here in the sky for all time. It will rise when the Sun sets and set when the Sun rises. However, it will not always be here. Some nights you will see it and some you will not. On those nights when it is not here, you will know that it will soon come again. Remember, no matter how dark it gets, the light will surely come. And that, my friends, is how the moon came to be put in the sky. Anansi is an African folktale character. He often takes the shape of a spider and is considered to be the spirit of all knowledge of stories. He is also one of the most important characters of West African and Caribbean folklore. Adapted from: Anansi and How the Moon Got Put in the Sky, as told by Kwesi Soti Mtundu (a.k.a. Craig C. Brookins), Am J Community Psychol Dec;32(3-4):283-4.
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11 Contents Chapter 1 General introduction and thesis outline 11 PART I CLINICAL CHARACTERISTICS, PATHOPHYSIOLOGY AND CURRENT TREATMENT OPTIONS Chapter 2 Daily pain in adults with sickle cell disease - a different perspective Chapter 3 Dynamics of von Willebrand factor reactivity in sickle cell disease during vaso-occlusive crisis and steady state Chapter 4 Prevention of acute, vaso-occlusive pain in sickle cell disease: a systematic review PART II N-ACETYLCYSTEINE AND OXIDATIVE STRESS Chapter 5 Effect of N-acetylcysteine on pain in daily life in patients with sickle cell disease: a randomized clinical trial Chapter 6 Effects of oral N-acetylcysteine on oxidative stress in patients with sickle cell disease PART III ALLO-IMMUNIZATION Chapter 7 Early occurrence of red blood cell allo-immunization in patients with sickle cell disease Chapter 8 Non-classical FCGR2C haplotype is associated with protection from red blood cell allo-immunization in sickle cell disease Chapter 9 Summary and general discussion 243 APPENDIX Nederlandse samenvatting Contributing authors List of Publications PhD Portfolio Acknowledgements About the author
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