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Early administration of selected CD34 peripheral blood stem cell following bone marrow transplantation to prevent graft rejection: experience in haploidentical transplant for sickle cell disease

Bone Marrow Transplantation (2025)Cite this article

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Sickle cell disease (SCD) is a severe inherited hemoglobinopathy associated with vaso-occlusive crises (VOCs), progressive organ damage, and early death. To date, allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment [1]. Most HSCTs are performed from an HLA-matched sibling donor, but haplo-identical transplants with graft-versus-host disease (GVHD) prophylaxis based on post-transplant high-dose cyclophosphamide (PTCy) may be a suitable alternative for patients lacking matched donor. Initial experience of haplo-identical transplant with a reduced intensity conditioning regimen (RIC) approach was associated with high incidence of graft failure, leading to intensification of the conditioning regimen [2,3,4]. As engraftment is a major issue in transplant for SCD, a high dose of CD34+ cells is required to promote engraftment; however, bone marrow is usually preferred over peripheral blood stem cells (PBSC) to limit GVHD.

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Authors and Affiliations

  1. Cells Therapy Unit, Saint-Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France

    Clara. Barthélemy, Justine Nasone & Lionnel Faivre

  2. Adolescent and Young Adult Hematology Unit, Saint Louis University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France

    Matthieu Jestin, Florian Chevillon, Nicolas Boissel & Nathalie Dhédin

  3. Therapeutic Apheresis Unit, Saint-Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France

    Anne Brignier & Nathalie Parquet

  4. Bone Marrow Transplant Unit, Saint-Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France

    Régis Peffault de Latour

Authors
  1. Clara. Barthélemy
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  2. Matthieu Jestin
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  3. Anne Brignier
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  4. Nathalie Parquet
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  5. Florian Chevillon
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  9. Lionnel Faivre
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  10. Nathalie Dhédin
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Contributions

CB: collected and assembled data, analyzed and interpreted data, wrote the manuscript.—MJ, AB, NP, FC, NB, JN, RPL, and LF: provided study materials or patients, reviewed manuscript and gave final approval to the manuscript.—ND: conceived and designed the study, analyzed and interpreted data, wrote the manuscript.

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Correspondence to Nathalie Dhédin.

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Barthélemy, C., Jestin, M., Brignier, A. et al. Early administration of selected CD34 peripheral blood stem cell following bone marrow transplantation to prevent graft rejection: experience in haploidentical transplant for sickle cell disease. Bone Marrow Transplant (2025). https://doi.org/10.1038/s41409-025-02685-0

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