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Engraftment

Engraftment and survival following hematopoietic stem cell transplantation for osteopetrosis using a reduced intensity conditioning regimen

Bone Marrow Transplantation volume 38, pages 783–787 (2006)Cite this article

Abstract

Autosomal recessive osteopetrosis (OP) is a disease characterized by osteoclast dysfunction, leading to multisystem morbidity and death of most affected children. Hematopoietic stem cell transplantation (HSCT) is the treatment of choice for OP, but this patient population is particularly prone to post-transplant complications and death after myeloablative conditioning. To determine the potential of achieving improved overall outcomes in these patients by decreasing pre-transplant mortality, we investigated engraftment and survival following a reduced intensity regimen including busulfan, fludarabine and total lymphoid irradiation. We report outcomes in 11 patients. All six patients who received a bone marrow or peripheral stem cell graft engrafted with >75% donor chimerism. In contrast, all five recipients of unrelated cord blood as a stem cell source for a first graft failed to demonstrate donor hematopoietic chimerism. The day 100 and 6-month mortality was low at 9%. One year after HSCT, six of 11 patients (55%) were surviving. Our data suggest that this regimen results in low peri-transplant mortality without compromising engraftment when a marrow or peripheral stem cell graft is used. An umbilical cord blood graft, however, should be used with caution for patients with OP when this or a similar reduced intensity regimen is used.

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Acknowledgements

This work was supported by Children's Cancer Research Fund.

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Authors and Affiliations

  1. Division of Hematology, Department of Pediatrics, Oncology, Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA

    J Tolar, S Grewal & P Orchard

  2. Bone Marrow Transplantation Division, Internal Medicine Department, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, PR, Brazil

    C Bonfim

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  1. J Tolar
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  2. C Bonfim
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  4. P Orchard
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Correspondence to J Tolar.

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Tolar, J., Bonfim, C., Grewal, S. et al. Engraftment and survival following hematopoietic stem cell transplantation for osteopetrosis using a reduced intensity conditioning regimen. Bone Marrow Transplant 38, 783–787 (2006). https://doi.org/10.1038/sj.bmt.1705533

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