Sickle Cell Disease: Review of Clinical Complications and Therapeutic Advances with Emphasis on Hematopoietic Stem Cell Transplantation
DOI:
https://doi.org/10.36557/2674-8169.2025v7n7p373-396Keywords:
Sickle Cell Disease;, Hematopoietic Stem Cell Transplantation;, Clinical Complications;Abstract
Introduction: Sickle cell anemia (SCA) is a hereditary hemoglobinopathy caused by a mutation in the beta-globin chain of hemoglobin, leading to the production of hemoglobin S (HbS). This alteration compromises the structure of erythrocytes, promoting their sickling, increased blood viscosity, tissue ischemia, and severe clinical manifestations such as vaso-occlusive crises (VOCs). In recent years, in addition to traditional approaches, innovative therapies such as crizanlizumab, voxelotor, and hematopoietic stem cell transplantation (HSCT) have stood out for their efficacy in disease control and potential for cure. Objective: To analyze, through a literature review, the therapeutic benefits of bone marrow transplantation and emerging pharmacological treatments in the management of sickle cell anemia, considering aspects such as clinical efficacy, safety, and applicability. Methodology: A narrative literature review was conducted using recognized scientific databases, selecting articles in Portuguese and English that addressed sickle cell anemia, bone marrow transplantation, and recent drug therapies, prioritizing studies with consistent clinical data. Final Considerations: Hematopoietic stem cell transplantation, particularly the allogeneic modality with haploidentical donors and the use of adapted immunosuppressive protocols, currently represents the main curative alternative for patients with sickle cell anemia. Additionally, pharmacological therapies such as crizanlizumab and voxelotor have shown promising results in reducing vaso-occlusive crises and improving hematological parameters. These advances represent a milestone in the treatment of the disease, although greater accessibility and the implementation of long-term multicenter studies are still needed. The combination of early diagnosis, multidisciplinary follow-up, and innovative therapies is essential for improving the quality of life and prognosis of individuals affected by this hematological condition.
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Copyright (c) 2025 Thais Vitoria Barbosa, Evelyn Cristina Schaurich, Anderson Felipe Ferreira, Barbara Sackser Horvath, Everton Padilha, Grazielle Mecabô
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