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Haploidentical Transplants

Effective donor lymphohematopoietic reconstitution after haploidentical CD34+-selected hematopoietic stem cell transplantation in children with refractory severe aplastic anemia

Bone Marrow Transplantation volume 33, pages 411–418 (2004)Cite this article

Summary:

Peritransplant toxicity and a delay in effective immune reconstitution have limited the utility of alternate donor transplantation for children with refractory severe aplastic anemia. We have assessed the effectiveness of infusing large numbers of highly purified haploidentical CD34+ cells after immunoablative conditioning in three patients who had failed intensive immunosuppression, lacked unrelated donors, and had active or recent serious infections. One patient rejected the first infusion, but engrafted after a second infusion from the same donor. This patient died 4 months after hematopoietic stem cell transplantation with no evidence of lymphoid reconstitution. Two patients experienced mixed chimerism requiring treatment with antibodies and/or donor lymphocyte infusion. Both currently survive more than 1 year after transplantation with normal blood counts, 100% donor engraftment, effective lymphoid reconstitution, and no chronic graft-versus-host disease. We observed functional thymopoiesis as measured by lymphocyte immunophenotyping, T cell receptor excision circles and T cell receptor Vβ spectratyping complexity analysis. Further study is required to validate the initial promise of these preliminary observations.

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Acknowledgements

We would like to express their appreciation to the BMT Clinical Research Office for data management and to the BMT practitioners for patient care. This work was supported in part by NIH Cancer Center Support Grant P 30CA 21765 and the American Lebanese Syrian Associated Charities (ALSAC).

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Authors and Affiliations

  1. Division of Stem Cell Transplantation, Department of Hematology/Oncology, St Jude Children's Research Hospital, Memphis, TN, USA

    P Woodard, J M Cunningham, E Benaim, X Chen, G Hale, E Horwitz, J Houston, K Kasow, W Leung, U Yusuf & R Handgretinger

  2. Divisions of Hematology, Department of Hematology/Oncology, St Jude Children's Research Hospital, Memphis, TN, USA

    W Wang

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  1. P Woodard
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Correspondence to P Woodard.

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Woodard, P., Cunningham, J., Benaim, E. et al. Effective donor lymphohematopoietic reconstitution after haploidentical CD34+-selected hematopoietic stem cell transplantation in children with refractory severe aplastic anemia. Bone Marrow Transplant 33, 411–418 (2004). https://doi.org/10.1038/sj.bmt.1704358

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