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Copyright ©The Author(s) 2016. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Clin Pediatr. Feb 8, 2016; 5(1): 25-34
Published online Feb 8, 2016. doi: 10.5409/wjcp.v5.i1.25
Novel insights in the management of sickle cell disease in childhood
Lorenzo Iughetti, Elena Bigi, Donatella Venturelli
Lorenzo Iughetti, Elena Bigi, Pediatric Unit, Department of Medical and Surgical Sciences for Mothers, Children and Adults, University of Modena and Reggio Emilia, 41124 Modena, Italy
Donatella Venturelli, Transfusion Medicine Department, University Hospital of Modena, 41124 Modena, Italy
Author contributions: Iughetti L and Bigi E wrote the paper; Iughetti L and Venturelli D supervised the manuscript drafting.
Conflict-of-interest statement: The authors declare no conflicts of interest regarding this manuscript.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Lorenzo Iughetti, Associate Professor of Pediatrics, Pediatric Unit, Department of Medical and Surgical Sciences for Mothers, Children and Adults, University of Modena and Reggio Emilia, via del Pozzo 71, 41124 Modena, Italy. iughetti.lorenzo@unimore.it
Telephone: +39-059-4225382
Received: July 31, 2015
Peer-review started: July 31, 2015
First decision: September 29, 2015
Revised: October 13, 2015
Accepted: December 18, 2015
Article in press: December 21, 2015
Published online: February 8, 2016
Processing time: 181 Days and 19.2 Hours
Abstract

Sickle cell disease (SCD) is a life-threatening genetic disorder characterized by chronic hemolytic anemia, vascular injury and multiorgan dysfunctions. Over the last few decades, there have been significant improvements in SCD management in Western countries, especially in pediatric population. An early onset of prophylaxis with Penicillin and a proper treatment of the infections have increased the overall survival in childhood. Nevertheless, management of painful episodes and prevention of organ damage are still challenging and more efforts are needed to better understand the mechanisms behind the development of chronic organ damages. Hydroxyurea (Hydroxycarbamide, HU), the only medication approved as a disease-modifying agent by the United States Food and Drug Administration and the European Medicines Agency, is usually under-used, especially in developing countries. Currently, hematopoietic stem-cell transplantation is considered the only curative option, although its use is limited by lack of donors and transplant-related toxicity. SCD symptoms are similar in children and adults, but complications and systemic organ damages increase with age, leading to early mortality worldwide. Experts in comprehensive care of young patients with SCD, especially those approaching the transition age to adulthood, are missing, leading people to rely on urgent care, increasing health care utilization costs and inappropriate treatments. It would be important to establish programs of comprehensive healthcare for patients with SCD from birth to adulthood, to improve their quality and expectancy of life.

Keywords: Clinical management; Hydroxyurea; Sickle cell disease; Children; Hematopoietic stem-cell transplantation

Core tip: The correct management of sickle cell disease (SCD) requires a comprehensive medical care. Both a wider use of hydroxyurea and an early treatment of pain in children are needed to improve long-term outcomes. Moreover, we report in details the possibility offered by hematopoietic stem cell transplantation as a future curative option for SCD patients.