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Successful haploidentical BMT with post-transplant cyclophosphamide for refractory autoimmune pancytopenia after cord blood transplant in pediatric myelodysplastic syndrome

Bone Marrow Transplantation volume 52, pages 653–655 (2017)Cite this article

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A recent approach to HLA-haploidentical donor transplantation with post-transplant cyclophosphamide (PTCy) has extended the availability of hematopoietic stem cell transplantation (HSCT) for patients who lack an HLA-matched donor. By selectively depleting the proliferating alloreactive T cells that are responsible for GvHD and graft rejection while preserving the resting memory T cells that are essential for post-transplant immunologic recovery, PTCy contributes to a low rate of both post-transplant infections and treatment-related toxicities.1 In adults, some physicians have adopted the HLA-haploidentical BMT with PTCy strategy for non-malignant diseases such as severe aplastic anemia.2 However, only limited evidence for this strategy is available in children. In this report, we present the case of a girl with myelodysplastic syndrome, who developed autoimmune pancytopenia after a cord blood transplantation (CBT) from her brother. Because conventional therapies, including steroids, intravenous immunoglobulin, rituximab and even bortezomib were not fully effective, we successfully retransplanted her from her HLA-haploidentical father with reduced intensity conditioning and PTCy. We also discuss the feasibility of HLA-haploidentical HSCT in children.

Due to the allowance of more HLA disparities compared with that of BM or PBSC, CB has become an important alternative source for patients lacking a suitable donor, particularly in children. Furthermore, its prompt availability establishes CBT as a therapeutic rescue option for graft failure after a previous HSCT. When there is an immediate requirement, HSCT from an HLA-haploidentical relative is another option. However, a T-cell-depleted HLA-haploidentical HSCT has a high incidence of engraftment failure mainly because the remaining host alloreactive T cells may escape the conditioning procedure, whereas a T-cell-repleted HLA-haploidentical HSCT has great concerns for severe GvHD.7

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Authors and Affiliations

  1. Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan

    H Shima, K Isshiki, Y Yamada, F Yamazaki, T Takahashi & H Shimada

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  1. H Shima
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Correspondence to H Shima.

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Shima, H., Isshiki, K., Yamada, Y. et al. Successful haploidentical BMT with post-transplant cyclophosphamide for refractory autoimmune pancytopenia after cord blood transplant in pediatric myelodysplastic syndrome. Bone Marrow Transplant 52, 653–655 (2017). https://doi.org/10.1038/bmt.2016.341

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