Summary:
For many children with life-threatening hematological diseases, hematopoietic stem cell transplantation (HSCT) is the only curative option. In children lacking a matched related or unrelated donor and with the certainty that, left untreated, death will ensue alternative donors must be sought. Haplo-identical peripheral blood stem cell transplantation (PBSCT) from a healthy parent is a feasible alternative. To reduce the risk of fatal graft-versus-host disease (GvHD) as a complication of transplant across major histocompatibility antigens, intense T-cell depletion is required. Large numbers of purified, cytokine mobilized peripheral stem cells (the so-called mega-dose concept) are required to compensate for the significantly increased risk of either graft failure or early rejection. In our unit, despite this approach, graft dysfunction has, in a significant group of children, proved problematic and, despite salvage attempts at re-transplantation, usually fatal. In children with hematological malignant disease, our overall relapse-free survival is 41%. However, successful transplant outcome has been associated with considerable delays in immune reconstitution that can be implicated in subsequent viral reactivation. We are investigating new strategies to improve the outcome of haplo-identical PBSCT, which may allow us to offer this form of treatment to more children requiring urgent HSCT.
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Acknowledgements
The medical, nursing and ancillary staff of the IHOBA, Willem-Alexander Kinder en Jeugd Centrum, Leiden University Medical Center, Leiden, the Netherlands. The staff of the Europdonor Foundation, Leiden, the Netherlands. JDJ Bakker-Steeneveld and CM Jol-van der Zijde, Data Managers, IHOBA, Willem-Alexander Kinder en Jeugd Centrum, Leiden University Medical Center, Leiden, the Netherlands. Professor Dr Dietrich Niethammer, Klinik für Kinderheilkunde und Jugendmedizin, Abteilung der Universität Tubingen, Tubingen, Germany. Professor Dr Thomas Klingebiel, Klinik für Kinderheilkunde III, Zentrum für Kinderheilkunde und Jugendmedizin der Universität Frankfurt, Frankfurt, Germany.
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Ball, L., Lankester, A., Bredius, R. et al. Graft dysfunction and delayed immune reconstitution following haploidentical peripheral blood hematopoietic stem cell transplantation. Bone Marrow Transplant 35 (Suppl 1), S35–S38 (2005). https://doi.org/10.1038/sj.bmt.1704842
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DOI: https://doi.org/10.1038/sj.bmt.1704842
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