Introduction: Sickle cell disease is the most prevalent inherited hemoglobinopathy worldwide, characterized by chronic
hemolysis, systemic inflammation, and recurrent episodes of vaso-occlusion, which are responsible for high morbidity
and mortality. In recent decades, therapeutic advances have expanded treatment options, including disease-modifying
therapies, hematopoietic stem cell transplantation, and gene therapy.
Objective: To analyze the scientific evidence regarding therapeutic advances, disease-modifying therapies, and future
prospects for the treatment of sickle cell anemia.
Methods: A systematic review conducted in accordance with PRISMA guidelines, using the databases PubMed, Scopus,
Web of Science, Embase, the Cochrane Library, and ScienceDirect. Studies published between 2016 and 2026 were
included, covering pharmacological therapies, hematopoietic stem cell transplantation, gene therapy, and gene editing.
After the selection process, 124 studies comprised the final sample.
Results: Hydroxyurea remained the primary disease-modifying drug, while crizanlizumab, voxelotor, and L-glutamine
demonstrated additional benefits in reducing vaso-occlusive crises, hemolysis, and clinical complications. Hematopoietic
stem cell transplantation showed the greatest curative potential, and gene therapies based on lentiviral vectors and
CRISPR-Cas9 revealed promising results in terms of reducing clinical events and achieving transfusion independence.
However, limitations related to cost, specialized infrastructure, access, and the lack of long-term follow-up still restrict
their widespread implementation.
Conclusion: The evidence demonstrates that advances in disease-modifying therapies and potentially curative strategies
are transforming the management of sickle cell disease, reinforcing the need for long-term studies and public policies
that expand access to innovative technologies.
Rosane Rezende de Souza Giuliani, Felipe Santos Teixeira Martiniano, Nicole Mioto Medeiros et al.· Advances in Hematology and O...· 0 citations
Treatment-resistant depression (TRD) represents one of the most severe forms of major depressive disorder and is associated with high morbidity and mortality, functional impairment, increased suicidal ideation, and significant socioeconomic impacts. In this context, ketamine and esketamine have emerged as innovative therapeutic alternatives due to their rapid antidepressant effect and their potential to reduce suicidal ideation within a few hours of administration. The aim of this study was to critically analyze the available scientific evidence regarding the therapeutic use of ketamine and esketamine in treatment-resistant depression, emphasizing their clinical efficacy, mechanisms of action, safety profile, and future prospects. A systematic literature review was conducted in accordance with the PRISMA 2020 guidelines. Searches were conducted in the PubMed/MEDLINE, Embase, Scopus, Web of Science, Cochrane Library, and PsycINFO databases, using search terms related to treatment-resistant depression, ketamine, esketamine, NMDA receptors, neuroplasticity, and suicidal ideation. After applying the eligibility criteria, 92 studies were included for qualitative analysis. The evidence demonstrated that both intravenous ketamine and intranasal esketamine promote a rapid and significant reduction in depressive symptoms and suicidal ideation, especially in patients with multiple treatment failures. The mechanisms of action involve modulation of glutamatergic neurotransmission, activation of AMPA receptors, increased expression of brain-derived neurotrophic factor (BDNF), and restoration of brain neuroplasticity. The main adverse events were transient dissociative symptoms, temporary elevation of blood pressure, nausea, and dizziness, presenting a favorable safety profile when used under specialized monitoring. It is concluded that ketamine and esketamine represent important therapeutic advances in the treatment of treatment-resistant depression, particularly due to the rapid clinical response and the potential to reduce the risk of suicide. However, further studies are needed to define optimal maintenance protocols, assess long-term safety, and expand the use of these therapies in evidence-based clinical practice.
Marcos A. Couto, Flávia Eduarda Pereira Januário, Gabriela de Luna Costa Pinheiro et al.· International Journal of Pha...· 0 citations
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