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Conditioning

Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: a prospective study and review of the literature

Bone Marrow Transplantation volume 52, pages 726–732 (2017)Cite this article

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Abstract

Allogeneic hematopoietic cell transplantation (HCT) effectively treats several non-malignant disorders such as selected lysosomal disorders, cerebral adrenoleukodystrophy and hemoglobinopathies. However, rates of graft failure (GF) in non-malignant populations exceed those of patients with malignant indications for HCT. Salvage conditioning regimens and outcomes for second HCT for GF vary immensely in the literature. We report 17 consecutive pediatric patients with non-malignant disorders who underwent a second allogenic HCT for GF using a non-myeloablative, low-dose busulfan-based regimen. Graft sources for the second transplant included umbilical cord blood, unrelated bone marrow and unrelated PBSCs. Median age at time of second HCT was 6.6 years (1.1–14.6 years). Fourteen of seventeen patients (82%) achieved engraftment, with a 3-year overall survival of 82% (95% CI, 54–94%). Day 100 transplant-related mortality was 12% (95% CI, 0–27%). CMV and adenovirus reactivation occurred in 30% and fungal infections in 18%. The incidence of grade II–IV acute GvHD disease was 35% (95% CI, 13–58%) with only 6% grade III–IV (95% CI, 0–17%). In summary, we illustrate excellent overall survival and acceptable toxicity using a non-myeloablative conditioning regimen for second HCT as salvage therapy for first GF in patients with non-malignant conditions.

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Acknowledgements

This work was supported in part by grants from the Children’s Cancer Research Fund and National Cancer Institute P01 CA65493.

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Authors and Affiliations

  1. Division of Pediatric Blood and Marrow Transplant, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA,

    K Mallhi, P J Orchard, W P Miller, J Tolar & T C Lund

  2. Biostatistics Core, Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA

    Q Cao

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Correspondence to T C Lund.

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Mallhi, K., Orchard, P., Miller, W. et al. Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: a prospective study and review of the literature. Bone Marrow Transplant 52, 726–732 (2017). https://doi.org/10.1038/bmt.2016.356

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