Hematopoietic stem cell transplantation for hemoglobinopathies in adults
DOI:
https://doi.org/10.46765/2675-374X.2025v7n1e355Keywords:
Hemoglobinopathies, Hematopoietic Stem Cell Transplantation, Sickle Cell Disease. ThalassemiaAbstract
Background: Hemoglobinopathies are the most prevalent monogenic disorders worldwide, and allogeneic hematopoietic stem cell transplantation (HSCT) remains the only established curative treatment. Objective: To provide an updated version of the Brazilian consensus published by the Brazilian Society of Cellular Therapy and Bone Marrow Transplantation, incorporating recent advances in allogeneic HSCT for adults with hemoglobinopathies. Methods: A comprehensive literature review was conducted using the PubMed database through January 2026, followed by a structured consensus building process conducted by an expert panel. Results: Current evidence on indications for HSCT in adults with hemoglobinopathies was summarized, with emphasis on patient selection, conditioning regimens, graft-versus-host disease prophylaxis and management, and recommendations for long term follow-up. Conclusion: Allogeneic HSCT from an HLA-identical sibling remains the standard of care for eligible adults with sickle cell disease. In addition, HSCT from matched unrelated and haploidentical donors has emerged as a promising therapeutic option, supported by encouraging outcomes in selected patients. In contrast, evidence regarding HSCT in adults with thalassemia remains limited, precluding definitive recommendations.
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Copyright (c) 2026 Thalita Cristina de Mello Costa, Adriana Seber, George Maurício Navarro Barros, João Victor Piccolo Feliciano, Roseane Vasconcelos Gouveia, Cíntia Delbem Albino, Ricardo Helman, Gil Cunha De Santis, Vanderson Rocha, Belinda Pinto Simões
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