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Pediatric Transplants

Salvage allogeneic hematopoietic SCT for primary graft failure in children

Abstract

Primary graft failure (pGF) is associated with considerable morbidity and mortality. Salvage hematopoietic SCT (HSCT) can rescue pGF patients; however, the optimal preconditioning regimen and stem cell source are yet to be determined, particularly in children. In this study, we retrospectively analyzed 102 pediatric patients who received salvage allogeneic HSCT for pGF. Salvage HSCT from matched or one-Ag-mismatched related donors (rMM01) provided superior OS compared with that from two- or three-Ags-mismatched related donors (rMM23) or cord blood transplantation (CBT). CBT showed a trend toward a slightly lower engraftment rate and late engraftment achievement compared with rMM23; however, the OS rate was similar between the two groups (47.6±7.7% for rMM23 and 45.7±8.6% for CBT, at 1 year after salvage HSCT). Multivariate analysis showed that preconditioning regimens with fludarabine or irradiation were associated with a higher engraftment rate and those with alkylating agents were associated with better OS. In conclusion, our results showed that rMM01 was the most suitable donor for salvage HSCT for pediatric pGF, and that CBT was an equally important option compared with rMM23 for patients without rMM01.

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Acknowledgements

We thank all the clinicians and leaders of hospitals and centers who provided precise data via a registry of the Japan Society for Hematopoietic Cell Transplantation (JSHCT). Script kindly given by Dr Yoshinobu Kanda was used for data manipulation.

Author contributions

MK, KM, RS and TF designed the research. HY, MI, HK, JI, K Koh, YH, HT, AS, AK, HS, K Kawa and K Kato collected the data. MK analyzed the data, and MK and TF wrote the manuscript. All the authors discussed the results and commented on the manuscript.

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Correspondence to M Kato.

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Kato, M., Matsumoto, K., Suzuki, R. et al. Salvage allogeneic hematopoietic SCT for primary graft failure in children. Bone Marrow Transplant 48, 1173–1178 (2013). https://doi.org/10.1038/bmt.2013.36

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