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- Title
New Therapeutic Options for the Treatment of Sickle Cell Disease.
- Authors
Matte, Alessandro; Zorzi, Francesco; Mazzi, Filippo; Federti, Enrica; Olivieri, Oliviero; De Franceschi, Lucia
- Abstract
Sickle cell disease (SCD; ORPHA232; OMIM # 603903) is a chronic and invalidating disorder distributed worldwide, with high morbidity and mortality. Given the disease complexity and the multiplicity of pathophysiological targets, development of new therapeutic options is critical, despite the positive effects of hydroxyurea (HU), for many years the only approved drug for SCD. New therapeutic strategies might be divided into (1) pathophysiology-related novel therapies and (2) innovations in curative therapeutic options such as hematopoietic stem cell transplantation and gene therapy. The pathophysiology related novel therapies are: a) Agents which reduce sickling or prevent sickle red cell dehydration; b) Agents targeting SCD vasculopathy and sickle cellendothelial adhesive events; c) Anti-oxidant agents. This review highlights new therapeutic strategies in SCD and discusses future developments, research implications, and possible innovative clinical trials.
- Subjects
SICKLE cell anemia; HEMATOPOIETIC stem cell transplantation; SICKLE cell anemia treatment; DRUG therapy; GENE therapy
- Publication
Mediterranean Journal of Hematology & Infectious Diseases, 2019, Vol 11, Issue 1, p1
- ISSN
2035-3006
- Publication type
Article
- DOI
10.4084/MJHID.2019.002