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Haploidentical SCT in children: an update and future perspectives

Abstract

Transplantation of haploidentical stem cells has become a well-established approach, which makes a potential donor available for almost all patients. This review focuses on current results and new strategies, especially in pediatric patients with malignant diseases. CD34+ positive selection was the most common procedure for graft manipulation in the past years, whereas T and B cell depletion is a promising new method. GVHD could herewith be effectively reduced and primary engraftment was reported in 83–100% of patients after transplantation of high stem cell doses. For patients with ALL in remission, disease-free survival at 3 years ranged between 22 and 48%. TRM, mainly because of viral infections, was improved by the use of reduced-intensity conditioning (which helped to speed up T cell recovery) and by close monitoring of viral loads and prophylactic/preemptive therapy. The role of donor-derived Ag-specific T cells against viral and fungal antigens is currently under investigation. Patients with active disease at the time of transplantation had a poor outcome and several attempts to improve these results are currently evaluated, such as co-infusion of natural killer cells, co-transplantation of MSC, use of new antileukemic drugs and post-transplant immunotherapy.

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Acknowledgements

We thank Barbara Lang, MD, for helping prepare the manuscript and David Martin, MD, for critically reviewing it. This work was supported by grants from the Deutsche Forschungsgemeinschaft (SFB 685), the AKF program of the University of Tuebingen, the German José Carreras Leukemia Foundation and the Reinhold Beitlich Stiftung, Tuebingen, Germany.

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Correspondence to P Lang.

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Lang, P., Handgretinger, R. Haploidentical SCT in children: an update and future perspectives. Bone Marrow Transplant 42 (Suppl 2), S54–S59 (2008). https://doi.org/10.1038/bmt.2008.285

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