Tratamentos atualmente disponíveis para a doença falciforme: uma revisão integrativa

Abstract

Sickle cell disease (SCD) is an autosomal recessive genetic condition resulting from defects in the hemoglobin structure associated or not with defects in its synthesis. Moreover, there are four main pathophysiological processes involved in the clinical manifestations of the disease, which are: polymerization of hemoglobin S, vaso-occlusion, hemolysis mediated by endothelial dysfunction, and inflammation. However, therapeutic options remain limited and the most common approach still includes the management of acute and chronic complications and the guidance of the family regarding the care and recognition of more severe conditions. In this context, the present study consists of an integrative review that aims to obtain a greater understanding of the treatments currently available for sickle cell disease, as well as to verify possible complications related to treatment. Therefore, searches were carried out in the databases: SciELO, MEDLINE, and LILACS. Additionally, the selection involved a preliminary screening in which titles, abstracts, and the entire article were read. Thereafter, 23 articles were selected to compose the integrative review. Furthermore, the vast majority of publications were from the United States, were randomized clinical trials, and had a level of evidence II. Regarding the content of the articles, the following was observed: hydroxyurea in childhood significantly improves the prognosis of the disease, transfusions can cause alloimmunization mainly in those patients with polymorphism, and iron overload can be treated with the drugs deferasirox, deferoxamine, and deferiprone, owing to their safe use. In addition, drugs such as crizanlizumab, voxelotor, and l-glutamine were found to be new drugs in use due to their effectiveness in modifying the course of sickle cell disease. Nevertheless, hematopoietic stem cell transplantation (HSCT) is the only promising treatment for the cure of SCD