Use of Hematopoietic Stem Cells as a Treatment for Sickle Cell Anemia

Systematic Review

Abstract

Sickle cell anemia is a genetic disease caused by a mutation in the beta-globin gene, which interferes with the production of red blood cells. The body produces sickle-shaped red blood cells instead of biconcave ones, leading to clinical complications. Among the therapies, the use of hematopoietic stem cells is the only curative therapy available. Therefore, the aim of the study was to search the literature for relevant information on stem cell transplantation and sickle cell anemia. The study is a systematic review that was guided by the PICO strategy, using the PubMed, MEDLINE, Cochrane and BVS databases. The search was carried out for studies published between 2019 and 2023, in English and Portuguese. The inclusion/exclusion selection of studies was made from data extraction via RAYYAN^®, where unrelated studies, case reports and review articles were excluded. A total of 786 articles were identified and, after screening, 4 articles related to the recovery/cure of patients with sickle cell anemia through THCT were included. The studies demonstrated that transplantation offers high survival rates with significant improvement in hematologic parameters and reduction of inflammatory and hemolytic markers. Although there are many benefits, challenges such as donor availability, graft failure, and late mortality are also encountered. This way, other non-curative alternative therapies such as Hydroxyurea^®, L-glutamine^®, Voxelotor^® should be considered, which help in symptomatic control. Therefore, hematopoietic stem cell transplantation represents a promising alternative for the treatment of sickle cell anemia, with a high potential for cure and significant improvement in the quality of life of patients.