Efficacy of hematopoietic stem cell transplantation in patients with sickle cell anemia
DOI:
https://doi.org/10.33448/rsd-v14i10.49840Keywords:
Sickle cell anemia, Progenitor cell, Hematopoietic stem cell transplantation, Clinical efficacy, Quality of life.Abstract
Objective: To conduct an integrative literature review on the efficacy of HSCT in the treatment of sickle cell anemia. Methodology: This is a descriptive study consisting of an analysis of theoretical reviews, gathering, evaluating, and synthesizing results within the variables expressed in the study, using PubMed/MEDLINE and SciElo databases to develop a search strategy. Results: After careful analysis, six articles were selected. These articles addressed variables such as age group, disease stage, presence of comorbidities, among others. These articles demonstrated high efficacy, especially in children under 12 years of age with an HLA-compatible donor, in addition to evaluating the good prognosis regarding successful engraftment after transplantation. Conclusion: HSCT currently represents the only curative alternative for sickle cell anemia. However, the lack of compatible donors, the high cost, and several variables hinder access to this method. Therefore, this work proves relevant not only from an academic and scientific perspective, but also offers critical and current analysis, contributing to clinical decisions and strategies that can expand access to this treatment, transforming the lives of patients.
References
Aljaafari, B. A., et al. (2023). Quality of life after hematopoietic stem cell transplantation in adolescents and adults with sickle cell disease. Pediatric Blood & Cancer, 70(10). https://pubmed.ncbi.nlm.nih.gov/37581462/
Ascef, B., Marques, M. O., Kashiura, D., Bertinato, J., & De Oliveira Jr., H. A. (2025). PD54 Impact of experiencing vaso-occlusive crisis in patients with sickle cell disease: Systematic review and meta-analysis of prognostic studies. International Journal of Technology Assessment in Health Care, 40(Suppl. 1), S117–S118. https://pubmed.ncbi.nlm.nih.gov/37092659/
Azevedo, J. T. C., Costa, T. C. de M., Lima, K. C., et al. (2021). Long-term effects of allogeneic hematopoietic stem cell transplantation on systemic inflammation in sickle cell disease patients. Frontiers in Immunology, 12, 774442. https://doi.org/10.3389/fimmu.2021.774442
Beer, S. A., Blättel, J., Reuß, K., et al. (2025). Long-term patient-reported outcomes following allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation, 60(5), 617–624. https://pmc.ncbi.nlm.nih.gov/articles/PMC12061752/
Brasil. Ministério da Saúde. (2024). Protocolo Clínico e Diretrizes Terapêuticas da Doença Falciforme. Portaria Conjunta nº 16, de 1º de novembro de 2024. Recuperado de https://www.gov.br/conitec/pt-br/midias/protocolos/pcdt-da-doenca-falciforme
Brazauskas, R. (2020). Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood, 136(18), 2043–2054. https://pubmed.ncbi.nlm.nih.gov/32518950/
Dovern, E., Aydin, M., & DeBaun, M. R. (2024). Effect of allogeneic hematopoietic stem cell transplantation on sickle cell disease-related organ complications: A systematic review and meta-analysis. American Journal of Hematology, 99(6), 1129–1141. https://doi.org/10.1002/ajh.27297
Gluckman, E., et al. (2020). Alternative donor hematopoietic stem cell transplantation for sickle cell disease. Hematology/Oncology and Stem Cell Therapy, 13(3), 133–142. https://journals.lww.com/hosct/fulltext/2020/13040/alternative_donor_hematopoietic_stem_cell.1.aspx
Ilonze, C., et al. (2025). Perceived barriers to hematopoietic cell transplantation among adolescents with sickle cell disease and their caregivers. Journal of Pediatric Hematology/Oncology. https://pubmed.ncbi.nlm.nih.gov/40229993/
Iqbal, M., Reljic, T., Corbacioglu, S., et al. (2021). Systematic review/meta-analysis on efficacy of allogeneic hematopoietic cell transplantation in sickle cell disease. Transplant Cell Therapy, 27(2), 167.e1–167.e12. https://pubmed.ncbi.nlm.nih.gov/33830027/
Kassim, A. A., et al. (2024). Haploidentical transplantation with posttransplant cyclophosphamide for sickle cell disease. Blood, 144(1), 50–62. https://pubmed.ncbi.nlm.nih.gov/38493482/
Larue, M., Labopin, M., Schroeder, T., et al. (2024). Long‐term outcome of 2‐year survivors after allogeneic hematopoietic cell transplantation for acute leukemia. Hemasphere, 8(10), e70026. https://pmc.ncbi.nlm.nih.gov/articles/PMC11494155/
Leonard, A. K., Furstenau, D., Inam, Z., et al. (2024). In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation. Blood Advances, 8(7), 1806–1816. https://pmc.ncbi.nlm.nih.gov/articles/PMC11006808/#bib19
Lucena, M. M. de, Gutierres, G. P., Rezende, M. J. de A., Nunes, T. A., & Nonino, A. (2021). Desafios do transplante de células-tronco hematopoieticas no tratamento da anemia falciforme. In XV Congresso Médico Universidade Católica de Brasília. Brasília–DF. Disponível em: https://doity.com.br/anais/xvcmucb/trabalho/165929
Magedanz, L., et al. (2022). Transplante de células-tronco hematopoieticas: Iniquidades na distribuição em território brasileiro, 2001 a 2020. Ciência & Saúde Coletiva, 27(8), 3239–3247. https://doi.org/10.1590/1413-81232022278.03142022
Moraes, A. S., & Rodrigues, C. R. (2022). Células-tronco em terapias hematológicas: Uma revisão de literatura. ResearchGate. https://www.researchgate.net/publication/360924676_Celulas_tronco_em_terapias_hematologicas_uma_revisao_de_literatura
Oliveira, L. S., Silva, E. C., & Sousa, F. M. O. (2022). Quadro clínico de crianças com anemia falciforme que necessitam de transplante de células-tronco hematopoieticas: Uma abordagem sobre a qualidade de vida. Diálogos & Ciência, 2(1), 153–172. https://doi.org/10.7447/1678-0493.2022v2n1p153-172
Pereira, A. S., Shitsuka, D. M., Parreira, F. J., & Shitsuka, R. (2018). Metodologia da pesquisa científica [eBook]. Editora da UFSM. https://repositorio.ufsm.br/handle/1/15824
Phelan, R., Rotz, S., Dandoy, C. E., et al. (2025). Multicenter study on caregiver experiences in pediatric hematopoietic stem cell transplantation: Part II. Transplant Cell Therapy, 31(8), 590.e1–590.e16.
Snyder, H. (2019). Literature review as a research methodology: An overview and guidelines. Journal of Business Research, 104, 333‑339. https://doi.org/10.1016/j.jbusres.2019.07.039
St Martin, A., Hebert, K. M., Serret-Larmande, A., et al. (2022). Long-term survival after hematopoietic cell transplant for sickle cell disease compared to the United States population. Transplant Cell Therapy, 28(6), 325.e1–325.e7. https://doi.org/10.1016/j.jtct.2022.03.014
Xiao, H., Huang, Q., Lai, Y., & Liu, R. (2025). Haploidentical hematopoietic stem cell transplantation in pediatric transfusion-dependent thalassemia: A systematic review and meta-analysis. Transplant Cell Therapy, 31(2), 101.e1–101.e12. https://pubmed.ncbi.nlm.nih.gov/39647520/
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Copyright (c) 2025 Maria Emanuelly Pereira Lima, Alcides Victor Oliveira Sá, Caio Vinícius Silva Oliveira, Denilson de Araújo e Silva, Marcia Gardênia Melo Santana de Lima, Tacyana Pires de Carvalho Costa

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