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December 8, 2025BloodOpen Access

Haploidentical hematopoietic stem cell transplantation in adults with severe sickle cell disease: Long-term outcomes using a modified conditioning regimen with peripheral blood stem cells.

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Authors

EZElisa ZucchettiMSMatias SanchezMKMatthew Koshy

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Overview

Observational analysis reports 95% survival in adults with severe sickle cell disease after haplo-HSCT, indicating effective outcomes with modified total body irradiation.

Key Points

  • Twenty-two adults with severe sickle cell disease underwent haploidentical hematopoietic stem cell transplantation with a modified conditioning regimen.
  • Long-term survival rate was 95%, with 90% remaining free of vaso-occlusive crisis after median follow-up of 4.8 years.
  • Conditioning included total body irradiation, antithymocyte globulin, and peripheral blood stem cells instead of bone marrow.
  • Findings highlight the potential of haplo-HSCT as a safe and effective curative approach for severe sickle cell disease.

Cite This Study

Zucchetti et al. (2025) studied this question.

synapsesocial.com/papers/69362f7f4fa91c937236e51chttps://doi.org/10.1182/blood-2025-2487
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Encouraging Outcomes of Hematopoeitic Stem Cell Transplantation in Pediatric Sickle Cell Disease- A Decade-Long Experience from the Developing World2025
  2. 2Hematopoietic stem cell transplantation with reduced toxicity myeloablative conditioning regimen for children and adults with sickle cell disease: A brazilian single center experience.2025
  3. 3Reduced intensity haploidentical bone marrow transplantation in children with severe sickle cell disease (SCD): BMT CTN 15072025 · 4 citations
  4. 4Longterm survival and late effects following hematopoietic cell transplantation for sickle cell disease into a modern era: A center for international blood and marrow transplant research (CIBMTR) analysis2025 · 2 citations
  5. 5Expanding access in pediatrics: Outcomes of haploidentical transplantation with reduced intensity and peripheral blood stem cells2025