Cenário atual do transplante alogênico de medula óssea em doença falciforme: revisão da literatura e aplicabilidade à realidade nacional

Abstract

Sickle cell disease (SCD) comprises a group of hereditary disorders in which there is the presence of hemoglobin S (Hb S). A point mutation occurs in the HBB gene that encodes the beta chains of hemoglobin. This change results in erythrocytes with altered morphology, in the development of the disease, which is characterized by vaso-occlusive phenomena and hemolytic anemia. The social cost of SCD must be considered as it occurs more frequently in lowerincome populations. UNICEF data indicates that 40-60% of adults with SCD are unemployed in the US. The use of hydroxyurea (HU) proved to be effective in increasing fetal hemoglobin, reducing vaso-occlusive crises, the incidence of acute chest syndrome (ACS) and the need for transfusions. However, HU does not have curative potential for the disease. Hematopoietic stem cell transplantation (HSCT) from a related donor, with identical human leukocyte antigen (HLA), is potentially curative. The availability of a compatible donor is restricted. Advances in conditioning regimens, prophylaxis for graft-host disease (GVHD) and better knowledge regarding post-transplant complications have made it possible to expand the spectrum of patients indicated for HSCT. Considering HSCT for patients with more severe disease or those at greater risk of complications may be a viable alternative, as well as a possibility to reduce public health costs.